National Academies Press: OpenBook
« Previous: 8 Pediatric Studies of Biologics
Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×

References

AAP (American Academy of Pediatrics). 1974. General guidelines for the evaluation of drugs to be approved for use during pregnancy and for treatment of infants and children. Evanston, IL: American Academy of Pediatrics.

AAP. 2008. Pediatric drug testing legislative and regulatory history. Evanston, IL: American Academy of Pediatrics. http://www.aap.org/en-us/advocacy-and-policy/federal-advocacy/Documents/bpcapreahistory.pdf (accessed March 23, 2012).

AAP. 2012. Pediatric drug and device laws: Reauthorization text with summary. Evanston, IL: American Academy of Pediatrics. http://www.aap.org/en-us/advocacy-and-policy/federal-advocacy/Documents/PediatricDrugDeviceLawsReauthorizationText_Summary.pdf (accessed August 15, 2012).

AAP Committee on Drugs. 1997. “Inactive” ingredients in pharmaceutical products: Udpate. Pediatrics 99(2):268-278. http://pediatrics.aappublications.org/content/99/2/268.full.pdf (accessed March 23, 2012).

AAP Committee on the Fetus and Newborn. 2004. Age terminology during the perinatal period. Pediatrics 114(5):1362-1364. http://pediatrics.aappublications.org/content/114/5/1362.full.pdf (accessed March 23, 2012).

Abrahamyan, L., C. S. Li, J. Beyene, A. R. Willan, and B. M. Feldman. 2011. Survival distributions impact the power of randomized placebo-phase design and parallel groups randomized clinical trials. Journal of Clinical Epidemiology 64(3):286-292.

Adebowale, A. 1999. Clinical pharmacology and biopharmaceutics review(s) for children’s Motrin cold suspension (ibuprofen and pseudoephedrine). NDA 21128. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/nda/2000/21-128_CHILDREN’S%20MOTRIN%20COLD%20SUSPENSION_BIOPHARMR.PDF (accessed March 23, 2012).

Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×

Adebowale, A. 2002. Clinical pharmacology and biopharmaceutics review(s) for children’s Advil cold suspension (ibuprofen and pseudoephedrine). NDA 21373. April 5. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/nda/2002/21-373_Ibuprofen%20Pseudoephedrine_biopharmr.pdf (accessed March 23, 2012).

Akinbami, L. 2006. Health E-Stat: Asthma prevalence, health care use and mortality: United States, 2003-2005. Atlanta, GA: Centers for Disease Control and Prevention Publications and Information Products. http://www.cdc.gov/nchs/data/hestat/asthma03-05/asthma03-05.htm (accessed March 23, 2012).

Alivisatos, R. 2008. Medical officer review of supplemental labeling revisions (prior approval) for Retrovir (zidovudine, ZDV). NDA 19910/19655/20518. September 18. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/ucm072896.pdf (accessed March 23, 2012).

Anand, K. J. S., J. V. Aranda, C. B. Berde, S. Buckman, E. V. Capparelli, W. A. Carlo, P. Hummel, J. Lantos, C. C. Johnston, V. T. Lehr, A. M. Lynn, L. G. Maxwell, T. F. Oberlander, T. N. K. Raju, S. G. Soriano, A. Taddio, and G. A. Walco. 2005. Analgesia and anesthesia for neonates: Study design and ethical issues. Clinical Therapeutics 27(6):814-843.

Arnon, S. S. 2007. Creation and development of the public service orphan drug human botulism immune globulin. Pediatrics 119(4):785-789. www.cdph.ca.gov/programs/ibtpp/Documents/Peds-Creatn-Devlpmt-BIG-IV-2007.pdf (accessed March 23, 2012).

Ault, A. 2006. Liver failure warning upgraded for telithromycin. Internal Medicine News. http://www.internalmedicinenews.com/index.php?id=2049&type=98&tx_ttnews%5Btt_news%5D=11666&cHash=da03e20e36 (accessed March 23, 2012).

Ayache, S. 2009. Clinical review for Angiomax (bivalirudin). NDA 20873. September 8. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/UCM195876.pdf (accessed March 23, 2012).

Ayalew, K. 2002. Clinical review: Use of Videx and Zerit to treat HIV infected infants and young children. NDA 20154/20155/20156/20412/20413. March 26. Silver Spring, MD: Food and Drug Administration.

Baer, G. R. 2009. Ethical issues in neonatal drug development. In Pediatric drug development: Concepts and applications, edited by A. E. Mulberg, S. A. Silber, and J. N. van den Anker. Hoboken, NJ: John Wiley & Sons, Inc. Pp. 103-114.

Baker-Smith, C. M., J. Daniel K. Benjamin, H. G. Grabowski, E. D. Reid, B. Mangum, J. V. Goldsmith, M. D. Murphy, R. Edwards, E. L. Eisenstein, J. Sun, R. M. Califf, and J. S. Li. 2008. The economic returns of pediatric clinical trials of antihypertensive drugs. American Heart Journal 156(4):682-688.

Ball, L. K. 2007. Warning letter to Gregory Irace, CEO of Sanofi-Aventis US LLC, regarding Ketek, October 23. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/ICECI/EnforcementActions/WarningLetters/2007/ucm076552.htm (accessed March 23, 2012).

Ballentine, C. 1981. Taste of raspberries, taste of death: The 1937 elixir sulfanilamide incident. FDA Consumer, June. http://www.fda.gov/AboutFDA/WhatWeDo/History/ProductRegulation/SulfanilamideDisaster/default.htm (accessed March 23, 2012).

BARDA (Biomedical Advanced Research and Development Authority). 2011. Strategic plan 2011-2016. Washington, DC: Biomedical Advanced Research and Development Authority, U.S. Department of Health and Human Services. http://www.phe.gov/about/barda/Documents/barda-strategic-plan.pdf (accessed March 23, 2012).

Barr, J., G. Brenner-Zada, E. Heiman, G. Pareth, M. Bulkowstein, R. Greenberg, and M. Berkovitch. 2002. Unlicensed and off-label medication use in a neonatal intensive care unit: A prospective study. American Journal of Perinatology 19(2):67-72.

Barron, J. J., H. Tan, J. Spalding, A. W. Bakst, and J. Singer. 2007. Proton pump inhibitor utilization patterns in infants. Journal of Pediatric Gastroenterology and Nutrition 45(4):421-427. http://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/PediatricAdvisoryCommittee/UCM214708.pdf (accessed March 23, 2012).

Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×

Bastings, E. 2002. Clinical review of Relpax (eletriptan hydrobromide). NDA 21016. Parts 1, 2, 3, and 4. November 27. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/nda/2002/21016_Relpax.cfm (accessed March 23, 2012).

Bayer Healthcare. 2010. Prescribing information for Betapace AF (sotalol HCl). Wayne, NJ: Bayer Healthcare. http://berlex.bayerhealthcare.com/html/products/pi/BetapaceAF_PI.pdf (accessed March 23, 2012).

Baylor, N. W. 2006. Approval letter for Zostavax (zoster vaccine, live). BLA 125123. May 25. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/Biologics BloodVaccines/Vaccines/ApprovedProducts/ucm132873.htm (accessed March 23, 2012).

Behrman, R. 2001a. Written request for Axert (almotriptan). NDA 21001. November 5. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/ucm074348.pdf (accessed March 23, 2012).

Behrman, R. 2001b. Written request for Plavix (clopidogrel bisulfate). NDA 20839. October 15. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/UCM241806.pdf (accessed March 23, 2012).

Behrman, R. 2003. Written request for Abilify (aripiprazole). NDA 21436. February 11. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/ucm073289.pdf (accessed March 23, 2012).

Beitz, J. 2005. Data Monitoring Committee report on griseofulvin. Washington, DC: U.S. Department of Health and Human Services. http://bpca.nichd.nih.gov/clinical/requests/upload/griseofulvin.pdf (accessed March 23, 2012).

Beitz, J. 2006a. Approval letter for Myozyme (alglucosidase alfa). BL 125141/0. April 28. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/appletter/2006/125141s0_LTR.pdf (accessed March 23, 2012).

Beitz, J. 2006b. Written request for Lamisil (terbinafine hydrochloride), Amendment 5. NDA 20192/20539/20749/20846/20980/21124. May 15. Silver Spring, MD: Food and Drug Administration.

Beitz, J. 2009a. Approval letter for Creon (pancrelipase). NDA 207725. April 30. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/appletter/2009/020725s000ltr.pdf (accessed March 23, 2012).

Beitz, J. 2009b. Approval letter for Ulesfia (benzyl alcohol). NDA 022129. April 9. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/appletter/2009/022129s000ltr.pdf (accessed March 23, 2012).

Beitz, J. G. 2010. Approval letter for Beyaz tablets (drospirenone; ethinyl estradiol; levomefolate calcium). NDA 022532. September 24. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/nda/2010/022532Orig1s000Approv.pdf (accessed March 23, 2012).

Belew, Y. 2008. Clinical review for Viramune (nevirapine). NDA 20636/20933. June 21. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/ucm072777.pdf (accessed March 23, 2012).

Benevento, J. D., R. D. Jager, A. G. Noble, P. Latkany, W. F. Mieler, M. Sautter, S. Meyers, M. Mets, M. A. Grassi, P. Rabiah, K. Boyer, C. Swisher, R. McLeod, and other members of the Toxoplasmosis Study Group. 2008. Toxoplasmosis associated neovascular lesions treated successfully with ranibizumab and anti-parasitic therapy. Archives of Ophthalmology 126(8):1152-1156.

Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×

Benjamin, D. K., J. S. Hirschfeld, C. K. Cunningham, and R. E. J. McKinney. 2004. Growth as a part of the composite endpoint in paediatric antiretroviral clinical trials. Journal of Antimicrobial Chemotherapy 54(4):701-703.

Benjamin, D. K., Jr., P. B. Smith, D. Murphy, R. Roberts, L. Mathis, D. Avant, R. M. Califf, and J. S. Li. 2006. Peer-reviewed publication of clinical trials completed for pediatric exclusivity. Journal of the American Medical Association 296(10):1266-1273. http://jama.ama-assn.org/content/296/10/1266.full.pdf (accessed March 23, 2012).

Benjamin, D. K., Jr., P. B. Smith, P. Jadhav, J. V. Gobburu, D. Murphy, V. Hasselblad, C. Baker-Smith, R. M. Califf, and J. S. Li. 2008. Pediatric Antihypertensive Trial failures: Analysis of end points and dose range. Hypertension 51(4):834-840. http://hyper.ahajournals.org/content/51/4/834.full.pdf+html (accessed March 23, 2012).

Benjamin, D. K., Jr., P. B. Smith, J. M. Sun, D. Murphy, D. Avant, L. Mathis, W. Rodriguez, R. M. Califf, and J. S. Li. 2009. Pediatric drug trials: Safety and transparency. Archives of Pediatrics & Adolescent Medicine 163(12):1080-1086. http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2792563/pdf/nihms129534.pdf (accessed March 23, 2012).

Berde, C. B., G. A. Walco, E. J. Krane, K. J. S. Anand, J. V. Aranda, K. D. Craig, C. D. Dampier, J. C. Finkel, M. Grabois, C. C. Johnston, J. Lantos, A. Lebel, L. G. Maxwell, P. McGrath, T. F. Oberlander, L. E. Schanberg, B. Stevens, A. Taddio, C. L. von Baeyer, M. Yaster, and W. T. Zempsky. 2012. Pediatric Analgesic Clinical Trial Designs, Measures, and Extrapolation: Report of an FDA scientific workshop. Pediatrics 129(2):354-364. http://pediatrics.aappublications.org/content/129/2/354.long (accessed March 23, 2012).

Berezny, K., P. B. Smith, and D. K. Benjamin, Jr. 2011. Letter to the editor: Future of the NICHD Pediatric Trial Network. e-Journal of Neonatology Research 1(2):109-111. http://www.neonatologyresearch.com/wp-content/uploads/2011/06/LTTR-Ped-Trials-Network1.pdf (accessed March 23, 2012).

BIO (Biotechnology Industry Organization). 2011. Statement for the Institute of Medicine Committee on Pediatric Studies Conducted Under BPCA and PREA, April 28. Washington, DC: Biotechnology Industry Organization. http://www.bio.org/node/178 (accessed March 23, 2012).

Biomarkers Definitions Working Group. 2001. Biomarkers and surrogate endpoints: Preferred definitions and conceptual framework. Clinical Pharmacology and Therapeutics 69(3):89-95.

Birnkrant, D. 2008. Approval letter for PegIntron (peginterferon alfa-2b). BL 103949/5171/5174. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/appletter/2008/103949s5171,%20s5174ltr.pdf (accessed March 23, 2012).

Birnkrant, D. 2010. Approval letter for Viread (tenofovir disoproxil fumarate). NDA 21356. October 1, 2010. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/appletter/2010/021356s034ltr.pdf (accessed March 23, 2012).

Birnkrant, D. 2011a. Approval letter for Viramune XR (nevirapine). NDA 201152. March 25. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/appletter/2011/201152s000ltr.pdf (accessed March 23, 2012).

Birnkrant, D. B. 2011b. Approval letter for Copegus (ribavirin) and Pegasys (peginterferon alfa-2a) supplemental. NDA 21511/BLA 103964. August 22. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/appletter/2011/021511s023,103964s5213ltr.pdf (accessed March 23, 2012).

Biss, T. T., P. J. Avery, L. R. Brandão, E. A. Chalmers, M. D. Williams, J. D. Grainger, J. B. S. Leathart, J. P. Hanley, A. K. Daly, and F. Kamali. 2012. VKORC1 and CYP2C9 genotype and patient characteristics explain a large proportion of the variability in warfarin dose requirement among children. Blood 119(3):868-873.

Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×

BMS (Bristol-Myers Squibb). 2010. Prescribing information for BuSpar (buspirone HCl). Princeton, NJ: Bristol-Myers Squibb. http://packageinserts.bms.com/pi/pi_buspar.pdf (accessed March 23, 2012).

BMS. 2011. Prescribing information for Abilify (aripiprazole). Princeton, NJ: Bristol-Myers Squibb. http://www.abilify.com/pdf/pi.aspx (accessed February 2, 2012).

BMS/SPP (Bristol-Myers Squibb/Sanofi Pharmaceuticals Partnership). 2011. Prescribing information for Plavix (clopidogrel bisulfate). Bridgewater, NJ: Bristol-Myers Squibb/Sanofi Pharmaceuticals Partnership. http://products.sanofi.us/PLAVIX/plavix.pdf (accessed March 23, 2012).

Booz Allen Hamilton. 2008. Postmarketing commitments study final report. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/AboutFDA/Reports ManualsForms/Reports/UserFeeReports/Studies/UCM109785.pdf (accessed March 23, 2012).

Booz Allen Hamilton. 2010. Deliverable 6: Final report on the PMR/PMC backlog review. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Post-marketingPhaseIVCommitments/UCM232880.pdf (accessed March 23, 2012).

Boxwell, D. 2011. Pediatric Advisory Committee memo regarding Kaletra (lopinavir/ritonavir). NDA 21251. January 24. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/PediatricAdvisoryCommittee/UCM272409.pdf (accessed March 23, 2012).

BPL (Bio Products Laboratory). 2009. Pediatric plan—July 30, 2009 for Gammaplex (immune globulin). BLA 125329. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/BiologicsBloodVaccines/BloodBloodProducts/ApprovedProducts/LicensedProductsBLAs/FractionatedPlasmaProducts/ucm186133.htm (accessed March 23, 2012).

Brar, S. 2010. Powerpoint presentation: Introduction to the sildenafil written request [third of three presentations]. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/CardiovascularandRenalDrugsAdvisoryCommittee/UCM221824.pdf (accessed March 23, 2012).

Brion, L. P., E. F. Bell, and T. S. Raghuveer. 2003. Vitamin E supplementation for prevention of morbidity and mortality in preterm infants. Cochrane Database of Systematic Reviews (4):CD003665. http://apps.who.int/rhl/reviews/CD003665.pdf (accessed March 23, 2012).

Bromberg, D. 2010. Presentation on behalf of the American Academy of Pediatrics at the FDA Public Hearing on Approval Pathway for Biosimilar and Interchangeable Biological Products. Meeting transcript for November 2. http://www.regulations.gov/#!documentDetail;D=FDA-2010-N-0477-0011;oldLink=false (accessed March 23, 2012).

Burns, L. E., J. E. Hodgeman, and A. B. Cass. 1959. Fatal and circulatory collapse in premature infants receiving chloramphenicol. New England Journal of Medicine 261(26):1318-1321.

Bussel, J. B., G. R. Buchanan, D. J. Nugent, D. J. Gnarra, L. Bomgaars, V. S. Blanchette, Y. M. Wang, K. Nie, and S. Jun. 2011. A randomized, double-blind study of romiplostim to determine its safety and efficacy in children with immune thrombocytopenia. Blood 118(1):28-36. http://bloodjournal.hematologylibrary.org/content/early/2011/04/15/blood-2010-10-313908.full.pdf (accessed March 23, 2012).

BVGH (BIO Ventures for Global Health). 2011. Priority review vouchers: Companies can benefit from global health investment. Washington, DC: BIO Ventures for Global Health. http://www.bvgh.org/What-We-Do/Incentives/Priority-Review-Vouchers.aspx (accessed March 23, 2012).

Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×

Cadence Pharmaceuticals. 2010. Prescribing information for Ofirmev (acetaminophen). San Diego, CA: Cadence Pharmaceuticals. http://www.ofirmev.com/pdf/OFIRMEVPrescribingInformation.pdf (accessed March 23, 2012).

Caldwell, P. H. Y., P. N. Butow, and J. C. Craig. 2003. Parents’ attitudes to children’s participation in randomized controlled trials. Journal of Pediatrics 142(5):554-559.

Cangene. 2010. Prescribing information for WinRho (Rho (D) immune globulin intravenous (human)). http://www.winrho.com/pi.pdf (accessed March 23, 2012).

Carr, R. R., and M. H. H. Ensom. 2003. Drug disposition and therapy in adolescence: The effects of puberty. Journal of Pediatric Pharmacology and Therapeutics 8(2):86-96.

Carver, K. H., J. Elikan, and E. Lietzan. 2010. An unofficial legislative history of the Biologics Price Competition and Innovation Act of 2009. Food and Drug Law Journal 65(4):671-818. http://www.cov.com/files/Publication/a2ef648b-5bc9-47c8-94ed-25c8e5f2367e/Presentation/PublicationAttachment/d4eaab3d-e65a-4fff-b5a2-33417bb65152/An%20Unofficial%20Legislative%20History%20of%20the%20Biologics%20Price%20Competition%20and%20Innovation%20Act%20of%202.pdf (accessed March 23, 2012).

Castile, R. 2004. Novel techniques for assessing infant and pediatric lung function and structure. Pediatric Infectious Disease Journal 23(11 Suppl.):S246-S253.

CBER (Center for Biologics Evaluation and Research). 2007. Package insert for Rhophylac (Rho (D) immune globulin intravenous (human)). Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/BiologicsBloodVaccines/BloodBloodProducts/ApprovedProducts/LicensedProductsBLAs/FractionatedPlasmaProducts/UCM119473.pdf (accessed March 23, 2012).

CDER (Center for Drug Evaluation and Research). 2010. Manual of policies and procedures (MAPP): Annotated clinical review template. MAPP 6010.3 Rev. 1. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/AboutFDA/CentersOffices/CDER/ManualofPoliciesProcedures/UCM236903.pdf (accessed March 23, 2012).

CDER. 2011a. Desk reference guide: New drug application and biologic license application reviews. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/AboutFDA/CentersOffices/CDER/ManualofPoliciesProcedures/UCM218757.pdf (accessed March 23, 2012).

CDER. 2011b. Guidance for industry: Dosage delivery devices for orally ingested OTC liquid drug products. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM188992.pdf (accessed March 23, 2012).

CDER. 2011c. Written request template. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/UCM207644.pdf (accessed March 23, 2012).

CDER/CBER. 1998a. Guidance for industry: General considerations for pediatric pharmacokinetic studies for drugs and biological products. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm072114.pdf (accessed March 23, 2012).

CDER/CBER. 1998b. Guidance for industry: Providing clinical evidence of effectiveness for human drug and biological products. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm078749.pdf (accessed March 23, 2012).

CDER/CBER. 1999a. Guidance for industry: Population pharmacokinetics. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm072137.pdf (accessed March 23, 2012).

Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×

CDER/CBER. 1999b. Guidance for industry: Qualifying for pediatric exclusivity under Section 505A of the federal Food, Drug, and Cosmetic Act. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/UCM049924.pdf (accessed March 23, 2012).

CDER/CBER. 2000. Guidance for industry: Pediatric oncology studies in response to a written request. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm071976.pdf (accessed March 23, 2012).

CDER/CBER. 2005. Draft guidance for industry: How to comply with the Pediatric Research Equity Act. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/UCM077855.pdf (accessed March 23, 2012).

CDER/CBER. 2006. Guidance for industry: Clinical studies section of labeling for human prescription drug and biological products—Content and format. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM075059.pdf (accessed March 23, 2012).

CDER/CBER. 2008. Guidance for industry: Tropical disease priority review vouchers. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM080599.pdf (accessed March 23, 2012).

CDER/CBER. 2009. Manual of policies and procedures: Postmarket policies. MAPP 6010.9. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/AboutFDA/CentersOffices/CDER/ManualofPoliciesProcedures/UCM120877.pdf (accessed March 23, 2012).

CDER/CBER. 2010a. Guidance for industry: Adaptive design clinical trials for drugs and biologics. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm201790.pdf (accessed March 23, 2012).

CDER/CBER. 2010b. Guidance for industry and investigators: Safety reporting requirements for INDs and BA/BE studies. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM227351.pdf (accessed March 23, 2012).

CDER/CBER. 2011. Guidance for industry: Postmarketing studies and clinical trials—Implementation of Section 505(o)(3) of the federal Food, Drug, and Cosmetic Act. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM172001.pdf (accessed March 23, 2012).

CDER/CBER/CDRH (Center for Devices and Radiological Health). 2006. Clinical review for Protonix (pantoprazole sodium) for delayed-release oral suspension. NDA 22020. August 10. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/UCM195520.pdf (accessed March 23, 2012).

CDRH (Center for Devices and Radiological Health). 2004. Guidance for industry and FDA staff: Premarket assessment of pediatric medical devices. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/MedicalDevices/DeviceRegulationandGuidance/GuidanceDocuments/ucm089742.pdf (accessed March 23, 2012).

CFF (Cystic Fibrosis Foundation). Undated. Research overview: Therapeutics development network. Bethesda, MD: Cystic Fibrosis Foundation. http://www.cff.org/research/TDN/ (accessed March 23, 2012).

Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×

Chambers, W. A. 2010. Approval letter for Moxeza (moxifloxacin hydrochloride ophthalmic solution). NDA 22428. November 19. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/appletter/2010/022428s000ltr.pdf (accessed March 23, 2012).

Chen, I. L. 2009. Clinical review for Protonix (pantoprazole sodium) for delayed-release oral suspension. NDA 22020. August 10. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/Development Resources/UCM195520.pdf (accessed March 23, 2012).

Chen, Y. F. 2010. Statistical review for Plavix (clopidogrel bisulfate). NDA 20839. November 23. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/UCM242306.pdf (accessed March 23, 2012).

Children’s Hospital of Wisconsin. 2010. Infantile hemangiomas. Milwaukee, WI: Children’s Hospital of Wisconsin. http://www.chw.org/display/PPF/DocID/28482/router.asp (accessed March 23, 2012).

Chowdhury, B. A. 2011. Approval letter for Rezira (hydrocodone and pseudoephedrine). NDA 22442. June 8. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/appletter/2011/022442s000ltr.pdf (accessed March 23, 2012).

Clark, R. H., B. T. Bloom, A. R. Spitzer, and D. R. Gerstmann. 2006. Reported medication use in the neonatal intensive care unit: Data from a large national data set. Pediatrics 117(6):1979-1987. http://pediatrics.aappublications.org/content/117/6/1979.full.pdf (accessed March 23, 2012).

Coffey, M. J., B. Wilfond, and L. Friedman Ross. 2004. Ethical assessment of clinical asthma trials including children subjects. Pediatrics 113(1):87-94. http://pediatrics.aappublications.org/content/113/1/87.full.pdf (accessed March 23, 2012).

Cohen, B. A. 2007. Ulcerated hemagiomas of infancy: Risk factors and management strategies. eMedical Dermatology Review 1(4). http://www.hopkinscme.edu/ofp/eMedDermReview/newsletters/emedderm_1007.html (accessed March 23, 2012).

Cohen, N., and S. Ness. 2009. Applications of pharmacogenomics to study design in pediatrics. In Pediatric drug development: Concepts and applications, edited by A. E. Mulberg, S. A. Silber, and J. N. van den. Anker. Hoboken, NJ: John Wiley & Sons, Inc. Pp. 259-275.

Conroy, S., and J. McIntyre. 2005. The use of unlicensed and off-label medicines in the neonate. Seminars in Fetal and Neonatal Medicine 10(2):115-122.

Cooper, W. O., L. A. Habel, C. M. Sox, K. A. Chan, P. G. Arbogast, T. C. Cheetham, K. T. Murray, V. P. Quinn, C. M. Stein, S. T. Callahan, B. H. Fireman, F. A. Fish, H. S. Kirshner, A. O’Duffy, F. A. Connell, and W. A. Ray. 2011. ADHD drugs and serious cardiovascular events in children and young adults. New England Journal of Medicine 365(20):1896-1904.

Coté, T. 2009. The state of the Orphan Drug Act. Presentation to Institute of Medicine Committee on Accelerating Rare Diseases Research and Orphan Product Development, Washington, DC, August 12.

Cox, E. 2009. Approval letter for Besivance (besifloxacin ophthalmic suspension). NDA 22308. May 28. Silver Spring, MD: Food and Drug Administration. http://www.access data.fda.gov/drugsatfda_docs/appletter/2009/022308s000ltr.pdf (accessed March 23, 2012).

Cox, E. M. 2010. Approval letter for Teflaro (ceftaroline fosamil). NDA 200327. October 29. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/appletter/2010/200327s000ltr.pdf (accessed March 23, 2012).

Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×

Cox, E. 2011. Approval letter for Edurant (rilpivirine). NDA 202022. May 20. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/appletter/2011/202022s000ltr.pdf (accessed March 23, 2012).

CRDAC (Cardiovascular and Renal Drugs Advisory Committee). 2010. Summary minutes of Cardiovascular and Renal Drugs Advisory Committee Meeting, July 29. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/CardiovascularandRenalDrugsAdvisoryCommittee/UCM225329.pdf (accessed March 23, 2012).

Crewalk, J.-A. 2008. Clinical review for PegIntron (peginterferon alfa-2b). BLA 103949. December 8. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/UCM171341.pdf (accessed March 23, 2012).

CSL Behring. 2010. Prescribing information for Rhophylac (Rho (D) immune globulin intravenous (human)). http://labeling.cslbehring.com/PI/US/Rhophylac/EN/Rhophylac-Prescribing-Information.pdf (accessed March 23, 2012).

CTSA CCHOC (Clinical and Translational Science Awards, Consortium Child Health Oversight Committee). 2011. Schedule of Clinical and Translational Science Awards and Pediatric Academic Societies Meeting, April 30-May 2, Denver, CO. http://www.pas-meeting.org/2011denver/Schedules/CTSA.pdf (accessed March 23, 2012).

Cuzzolin, L., A. Atzei, and V. Fanos. 2006. Off-label and unlicensed prescribing for newborns and children in different settings: A review of the literature and a consideration about drug safety. Expert Opinion on Drug Safety 5(5):703-718. http://informahealthcare.com/doi/abs/10.1517/14740338.5.5.703 (accessed March 23, 2012).

Day, R. L., and W. A. Silverman. 1957. Premature and newborn infants: Report of a seminar. Pediatrics 20(1):143-154.

de Castro, R. A. C., J. A. A. DeCastro, M. Y. C. Barez, M. V. Frias, J. Dixit, and M. Genereux. 2007. Thrombocytopenia associated with dengue hemorrhagic fever responds to intravenous administration of anti-D immune globulin. American Journal of Tropical Medicine and Hygiene 76(4):737-742. http://www.ajtmh.org/content/76/4/737.full.pdf (accessed March 23, 2012).

de Graaf, J., R. A. van Lingen, S. H. Simons, K. J. Anand, H. J. Duivenvoorden, N. Weisglas-Kuperus, D. W. Roofhooft, L. J. G. Jebbink, R. R. Veenstra, D. Tibboel, and M. van Dijk. 2011. Long-term effects of routine morphine infusion in mechanically ventilated neonates on children’s functioning: Five-year follow-up of a randomized controlled trial. Pain 152(6):1391-1397.

Di Fiore, J., M. Arko, B. Herynk, R. Martin, and A. M. Hibbs. 2010. Characterization of cardiorespiratory events following gastroesophageal reflux in preterm infants. Journal of Perinatology 30(10):683-687.

Dor, A., T. Burke, R. Whittington, T. Clark, R. Lopert, and S. Rosenbaum. 2007. Assessing the effects of federal pediatric drug safety policies. Washington, DC: The George Washington University Medical Center. http://www.gwumc.edu/sphhs/departments/healthpolicy/dhp_publications/pub_uploads/dhpPublication_1A4EE090-5056-9D20-3DB4FCF9FA438C01.pdf (accessed March 23, 2012).

Doyle, L. W., R. A. Ehrenkranz, and H. L. Halliday. 2010. Dexamethasone treatment after the first week of life for bronchopulmonary dysplasia in preterm infants: A systematic review. Neonatology 98(4):289-296. http://content.karger.com/produktedb/produkte.asp?DOI=000286212&typ=pdf (accessed March 23, 2012).

Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×

Dunne, J. 2010. Extrapolation (IOM Task 3). Presentation to Institute of Medicine Committee on Pediatric Studies Conducted Under BPCA and PREA, Meeting 1, Washington, DC, December 17. http://www.iom.edu/~/media/Files/Activity%20Files/Children/PediatricBPCAPREA/FDA%20Revised%20IOM%20presentation%2001072011final.pdf (accessed March 23, 2012).

Dunne, J., and D. Murphy. 2011. European Union legislation. Presentation to Institute of Medicine Committee on Pediatric Studies Conducted Under BPCA and PREA, Meeting 2, Washington, DC, February 2.

Dunne, J., D. Murphy, and G. Wharton. 2011a. The globalization of pediatric trials: Should we be worried? Pediatrics 127(1):e252-e253. http://pediatrics.aappublications.org/content/127/1/e252.full.pdf+html (accessed March 23, 2012).

Dunne, J., W. Rodriguez, D. Murphy, N. Beasley, G. Burckart, J. Fille, L. Lewis, H. Sachs, P. Sheridan, P. Starke, and L. Yao. 2011b. Extrapolating efficacy maximizing the use of adult & other data in pediatric drug development. Poster presented at Pediatric Academic Societies Annual Meeting, Denver, CO, April 30-May 3.

EMA (European Medicines Agency). 2009a. Guideline on reporting the results of population pharmacokinetic analyses. London, United Kingdom: European Medicines Agency. http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003067.pdf (accessed March 23, 2012).

EMA. 2009b. Principles of interactions: Between EMEA and FDA pediatric therapeutics. London, United Kingdom: European Medicines Agency. http://www.ema.europa.eu/docs/en_GB/document_library/Other/2009/12/WC500017969.pdf (accessed March 23, 2012).

EMA. 2011. Questions and answers on the cessation of supply of Regranex (becaplermin). London, United Kingdom: European Medicines Agency. http://www.ema.europa.eu/docs/en_GB/document_library/Medicine_QA/2011/05/WC500106713.pdf (accessed March 23, 2012).

Epstein, J. S. 2008. Approval letter for ARTISS (fibrin sealant (human)). BLA 125266. March 21. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/Biologics BloodVaccines/BloodBloodProducts/ApprovedProducts/LicensedProductsBLAs/FractionatedPlasmaProducts/ucm073064.htm (accessed March 23, 2012).

Fang, C. 2009. Clinical review for Ofirmev (acetaminophen). NDA 22450. October 17. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/UCM215816.pdf (accessed March 23, 2012).

FDA (Food and Drug Administration). 1977. Guidance for industry: General considerations for the clinical evaluation of drugs in infants and children. HEW (FDA) 77-3041. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm071687.pdf (accessed March 23, 2012).

FDA. 1996. Guidance for industry: The content and format for pediatric use supplements. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm071957.pdf (accessed March 23, 2012).

FDA. 1998. Regulations requiring manufacturers to assess the safety and effectiveness of new drugs and biological products in pediatric patients. Federal Register 63(231, December 2):66631-66672. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/ohrms/dockets/98fr/120298c.txt (accessed March 23, 2012).

FDA. 2000. Guidance for industry: Recommendations for complying with the Pediatric Rule (21 CFR 314.55(a) and 601.27(a)). Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/ohrms/dockets/ac/03/briefing/3927B1_06_GFI-Complying%20with%20the%20Pediatric%20Rule.pdf (accessed March 23, 2012).

FDA. 2001a. Prescribing information for Betapace (sotalol hydrochloride). NDA 19865. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/nda/2001/19-865S010_Betapace_prntlbl.pdf (accessed March 23, 2012).

Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×

TABLE B-3 Recent Pediatric Labeling Changes Identified by FDA and Comparison to Commonly Used Resources

      Information from:
Drug Labeling Change Date of Labeling Change The Harriet Lane Handbook Neofax
Topiramate Lack of efficacy for treatment of seizures for ages 1–24 months
Growth retardation lab abnormalities for ages 1–24 months
12/22/2009 No dosing information for ages <2 years36
New safety findings not mentioned36
No information provided35
Esomeprazole Lack of efficacy for GERDa for ages <1 year 6/18/2009 No dosing information for ages <1 year36
No reference to lack of efficacy for ages <1 year
No information provided35
Lamotrigine Lack of efficacy for ages 1–24 months, seizures
Associated with increased risk of infectious adverse reactions
5/8/2009 No dosing information for ages <2 years36
New safety findings not mentioned36
No information provided35
Azithromycin Efficacy for community-acquired pneumonia not established for ages <6 months
Efficacy for sinusitis not established for pediatric population
10/8/2008 Dosing for otitis media and community-acquired pneumonia provided for ages ≥6 months36
Dosing for acute sinusitis provided for ages6 months36
Dosing provided for infants35
Caspofungin Safety and efficacy not studied for ages <3 months 7/29/2008 Dosing provided for ages <3 months36 Dosing provided for infants35

a GERD = gastroesophageal reflux disease.

negative findings about safety or efficacy or both. Neither The Harriet Lane Handbook nor Neofax routinely notes when dosing is recommended for off-label indications or age groups.35,36,48 Neofax does, however, provide references for its dosing recommendations.35

Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×

FDA. 2010c. PDUFA reauthorization performance goals and procedures fiscal years 2013 through 2017. Silver Spring, MD: Food and Drug Administration. (accessed March 23, 2012).

FDA. 2010d. Press release: FDA approves pancreatic enzyme product, Pancreaze. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm208135.htm (accessed March 23, 2012).

FDA. 2011a. Advancing regulatory science at FDA: A strategic plan. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/ScienceResearch/SpecialTopics/RegulatoryScience/UCM268225.pdf (accessed March 23, 2012).

FDA. 2011b. Approved drug products with therapeutic equivalence evaluations. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/UCM071436.pdf (accessed March 23, 2012).

FDA. 2011c. Table of pharmacogenomic biomarkers in drug labels. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/Drugs/ScienceResearch/ResearchAreas/Pharmacogenetics/ucm083378.htm (accessed March 23, 2012).

FDA. 2012. Guidance agenda: New & revised draft guidances CDER is planning to publish during calendar year 2012. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM079647.pdf (accessed March 23, 2012).

FDA Science Board. 2007. FDA science and mission at risk: Report of the Subcommittee on Science and Technology. Silver Spring, MD: Science Board, Food and Drug Administration. http://www.fda.gov/ohrms/dockets/ac/07/briefing/2007-4329b_02_01_FDA%20Report%20on%20Science%20and%20Technology.pdf (accessed March 23, 2012).

Feeney, J. 2000. Medical review for Neurontin (gabapentin). NDA 21216. October 6. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/nda/2000/21-216.pdf_Neurontin_Medr_P1.pdf (accessed March 23, 2012).

Feldman, B., E. Wang, A. Willan, and J. P. Szalai. 2001. The randomized placebo-phase design for clinical trials. Journal of Clinical Epidemiology 54(6):550-557.

Feudtner, C., D. Dai, K. R. Hexem, X. Luan, and T. A. Metijian. 2012. Prevalence of polypharmacy exposure among hospitalized children in the United States. Archives of Pediatrics & Adolescent Medicine 166(1):9-16.

FIFCFS (Federal Interagency Forum on Child and Family Statistics). 2009. Table Pop2: Children as a percentage of the population: Persons in selected age groups as a percentage of the total US population, and children ages 0-17 as a percentage of the dependent population, 1950-2009 and projected 2010-2050. Childstats.gov. http://www.childstats.gov/americaschildren/tables/pop2.asp (accessed March 23, 2012).

Findling, R. L., N. K. McNamara, R. J. Stansbrey, N. C. Feeny, C. M. Young, F. V. Peric, and E. A. Youngstrom. 2006. The relevance of pharmacokinetic studies in designing efficacy trials in juvenile major depression. Journal of Child and Adolescent Psychopharmacology 16(1-2):131-145. http://www.liebertonline.com/doi/abs/10.1089/cap.2006.16.131 (accessed March 23, 2012).

FNIH (Foundation for the National Institutes of Health). 2011. Past programs: Best Pharmaceuticals for Children Act. Bethesda, MD: Foundation for the National Institutes of Health. http://www.fnih.org/work/past-programs/best-pharmaceuticals-children (accessed March 23, 2012).

Frattarelli, D. 2011. Statement on behalf of the American Academy of Pediatrics to the Joint Meeting of the FDA Nonprescription Drugs Advisory Committee and the Pediatric Advisory Committee, Silver Spring, MD, May 17. http://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/NonprescriptionDrugsAdvisoryCommittee/UCM264148.pdf (accessed March 23, 2012).

Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×

Freund, C. L., and E. W. Clayton. 2003. Pharmacogenomics and children: Meeting the ethical challenges. American Journal of Pharmacogenomics 3(6):399-404.

Frieden, I. J. 2008. Addendum: Commentary on becaplermin gel (Regranex) for hemangiomas. Pediatric Dermatology 25(6):590.

Gabry, K. E., and H. V. Joffe. 2008. Clinical review of Apidra (insulin glulisine). NDA 21629. October 2. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/ucm072460.pdf (accessed March 23, 2012).

Gallo-Torres, H. E. 2002. Justification for studies in pediatric patients. May 15. Briefing document for the Pediatric Advisory Subcommittee Members from the medical team leader of the Gastrointestinal and Coagulation Drug Products Division regarding June 11, 2002, Advisory Committee Meeting on the proton-pump inhibitor (PPI) template. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/ohrms/dockets/ac/02/briefing/3870B1_02_Briefing%20document.pdf (accessed March 23, 2012).

GAO (Government Accountability Office). 2007. Pediatric drug research: Studies conducted under Best Pharmaceuticals for Children Act. GAO-07-557. Washington, DC: Government Accountability Office. http://www.gao.gov/new.items/d07557.pdf (accessed March 23, 2012).

GAO. 2011. Products Studied under Two Related Laws, but Improved Tracking Needed by FDA. GAO-11-457. Washington, DC: Government Accountability Office. http://www.gao.gov/new.items/d11457.pdf (accessed June 29, 2012).

Genentech. 2010a. Prescribing information for Rocephin (ceftriaxone sodium). South San Francisco, CA: Genentech. http://www.gene.com/gene/products/information/rocephin/pdf/pi.pdf (accessed March 23, 2012).

Genentech. 2010b. Prescribing information for Xolair (omalizumab). South San Francisco, CA: Genentech. http://www.gene.com/gene/products/information/pdf/xolair-prescribing.pdf (accessed March 23, 2012).

Genzyme. 2008. Prescribing information for thymoglobulin (anti-thymocyte globulin (rabbit)). Cambridge, MA: Genzyme. http://www.thymoglobulin.com/home/thymo_pdf_pi.pdf (accessed March 23, 2012).

Gerhard, T. 2011. Second generation antipsychotics and risk of type 2 diabetes in publicly insured children and adolescents. Presentation to Institute for Health, Health Care Policy, and Aging Research, Ernest Mario School of Pharmacy, Rutgers University, New Brunswick, NJ. http://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/PediatricAdvisoryCommittee/UCM272847.pdf (accessed March 23, 2012).

Gershanik, J., B. Boecler, H. Ensley, S. McCloskey, and W. George. 1982. The gasping syndrome and benzyl alcohol poisoning. New England Journal of Medicine 307(22):1384-1388.

Giacoia, G. P., and D. R. Mattison. 2005. Newborns and drug studies: The NICHD/FDA newborn drug development initiative. Clinical Therapeutics 27(6):796-813.

GIDAC (Gastrointestinal Drugs Advisory Committee, CDER, FDA). 2010. Summary minutes of the GIDAC meeting, November 5. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/GastrointestinalDrugsAdvisoryCommittee/UCM235453.pdf (accessed March 23, 2012).

Gilbert-McClain, L. 2010. Approval letter for Xolair (omalizumab). BL 103976/5166. January 4. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/appletter/2010/103976s5166ltr.pdf (accessed March 23, 2012).

Gilead Sciences. 2008. Prescribing information for Emtriva (emtricitabine). Foster City, CA: Gilead Sciences. http://www.gilead.com/pdf/emtriva_pi.pdf (accessed March 23, 2012).

Giuliano, C. A., M. L. Dehoorne-Smith, and P. B. Kale-Pradhan. 2011. Pancreatic enzyme products: Digesting the changes. Annals of Pharmacotherapy 45(5):658-666.

Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×

Glickman, S. W., J. G. McHutchison, E. D. Peterson, C. B. Cairns, R. A. Harrington, R. M. Califf, and K. A. Schulman. 2009. Ethical and scientific implications of the globalization of clinical research. New England Journal of Medicine 360(8):816-823. http://www.nejm.org/doi/pdf/10.1056/NEJMsb0803929 (accessed March 23, 2012).

Gobburu, J., and J. Canal. 2000. Clinical pharmacology and biopharmaceutics review for Betapace (sotalol). NDA 19865. August 17. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/nda/2001/19-865S010_Betapace_biopharmr.pdf (accessed March 23, 2012).

Goldberger, M. 2004. Approval letter for Ketek (telithromycin). NDA 21144. April 1. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/appletter/2004/21144ltr.pdf (accessed March 23, 2012).

Golding, B. 2004. Approval letter for Rhophylac (Rho (D) immune globulin intravenous (human)), February 12. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/BiologicsBloodVaccines/BloodBloodProducts/ApprovedProducts/LicensedProductsBLAs/FractionatedPlasmaProducts/ucm119474.htm (accessed March 23, 2012).

Golding, B. 2008a. Approval letter for Gamunex-C (immune globulin intravenous (human)). BLA 125046. September 12. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/BiologicsBloodVaccines/BloodBloodProducts/ApprovedProducts/LicensedProductsBLAs/FractionatedPlasmaProducts/ucm064976.htm (accessed March 23, 2012).

Golding, B. 2008b. Approval letter for Kogenate FS (antihemophilic factor). BLA 103332/5794. October 10. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/BiologicsBloodVaccines/BloodBloodProducts/ApprovedProducts/LicensedProductsBLAs/FractionatedPlasmaProducts/ucm059035.htm (accessed March 23, 2012).

Goodman, C. 2010a. Information and process to support priority setting from The Lewin Group. Presentation to Institute of Medicine Committee on Pediatric Studies Conducted Under BPCA and PREA, Meeting 1, Washington, DC, December 17.

Goodman, J. L. 2010b. Treating rare and neglected pediatric diseases: Promoting the development of new treatments and cures. Statement before the Committee on Health, Education, Labor, and Pensions of the United States Senate. http://www.fda.gov/NewsEvents/Testimony/ucm219765.htm (accessed March 23, 2012).

Goodwin, P., C. L. Shields, A. Ramasubramanian, G. C. Brown, and J. A. Shields. 2009. Ranibizumab for coloboma-related choroidal neovascular membrane in a child. Journal of AAPOS 13(6):616-617.

Grant, S. M. 2011. Summary review for Plavix (clopidogrel bisulfate). NDA 20839. May 5. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/nda/2011/020839Orig1s051SumR.pdf (accessed March 23, 2012).

Green, P., and D. Moeny. 2010. Proton pump inhibitors BPCA drug use review and duration of use analysis. Memorandum to Dianne Murphy. April 30. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/PediatricAdvisoryCommittee/UCM214657.pdf (accessed June 29, 2012).

Gregory-Evans, K., P. Rai, and J. Patterson. 2009. Successful treatment of subretinal neovascularization with intravitreal ranibizumab in a child with optic nerve head drusen. Journal of Pediatric Ophthalmology & Strabismus 46(4):e1-e4.

Griebel, D. 2009. Division director review for Protonix (pantoprazole sodium). NDA 022020/020987. November 12. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/UCM195516.pdf (accessed March 23, 2012).

Griebel, D. 2010. Approval action for Carbaglu (carglumic acid). Memorandum to Julie Beitz, Director, Office of Drug Evaluation III, Center for Drug Evaluation and Research, FDA. NDA 22562. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/nda/2010/022562s000sumr.pdf (accessed March 23, 2012).

Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×

Gruber, R., R. Joober, N. Grizenko, B. L. Leventhal, E. H. Cook, Jr., and M. A. Stein. 2009. Dopamine transporter genotype and stimulant side effect factors in youth diagnosed with attention-deficit/hyperactivity disorder. Journal of Child and Adolescent Psychopharmacology 19(3):233-239.

GSK (GlaxoSmithKline). 2010. Prescribing information for Ziagen (abacavir sulfate). Research Triangle Park, NC: GlaxoSmithKline. http://us.gsk.com/products/assets/us_ziagen.pdf (accessed March 23, 2012).

Harris, G. 2006. Halt is urged for trials of antibiotic in children. New York Times, June 8.

Harris, G., and E. Koli. 2005. Lucrative drug, danger signals and the F.D.A. New York Times, June 10.

Harris, R. 2009. Clinical review of Axert (almotriptan). NDA 21001. March 12. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/UCM170825.pdf (accessed March 23, 2012).

Hartwell, P. 2000. Clinical review of Diprivan (propofol 1% solution). NDA 19627. May 5. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/nda/2001/019627_S035_DIPRIVAN_MEDR.pdf (accessed March 23, 2012).

Hecht, A. 1984. A long reach back to assure drug quality. FDA Consumer 8(15). http://findarticles.com/p/articles/mi_m1370/is_v18/ai_3541522/ (accessed March 23, 2012).

Hertz, S. H. 2010a. Approval letter for Ofirmev (acetaminophen), NDA 022450. November 2. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/appletter/2010/022450s000ltr.pdf (accessed March 23, 2012).

Hertz, S. H. 2010b. Summary review for oxycodone hydrochloride capsule and oral solution. NDA 200534/200535. October 20. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/nda/2010/200534Orig1s000SumR.pdf (accessed March 23, 2012).

Hibbs, A. M. 2011. Pharmacology review: Pharmacology for gastroesophageal reflux disease. NeoReviews 12(3):e159-e166.

Hirschfeld, S. 2003. Clinical review of Hycamtin (topotecan). NDA 20671. February 21. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/UCM163149.pdf (accessed March 23, 2012).

Honig, S. 2002. Medical review for Navelbine (vinorelbine). NDA 20388. July 16. (pp. 34-61 of approval package). Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/nda/2002/020388_S014_NAVELBINE_INJECTION_AP.pdf (accessed April 3, 2012).

Hospira. 2006. Prescribing information for Corlopam (fenoldopam mesylate injection). Lake Forest, IL: Hospira. http://products.hospira.com/assets/pdfs/EN-1271.pdf (accessed April 3, 2012).

Hospira. 2007. Prescribing information for Voluven (hydroxyethyl starch in sodium chloride injection). Lake Forest, IL: Hospira. http://products.hospira.com/assets/pdfs/EN-2475.pdf (accessed April 3, 2012).

Hotez, P. J., D. H. Molyneux, A. Fenwick, J. Kumaresan, S. E. Sachs, J. D. Sachs, and L. Savioli. 2007. Control of neglected tropical diseases. New England Journal of Medicine 357(10):1018-1027.

Howie, S. R. C. 2010. Blood sample volumes in child health research: Review of safe limits. Geneva, Switzerland: World Health Organization. http://www.who.int/bulletin/volumes/89/1/10-080010/en/index.html (accessed April 3, 2012).

Hubbard, L., and W. Chambers. 2003. Clinical review of Vigamox (moxifloxacin hydrochloride ophthalmic solution, 0.5%). NDA 21598. April 15. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/nda/2003/21-598_Vigamox_medr.PDF (accessed April 3, 2012).

Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×

Hudson, K. L. 2011. Genomics, health care, and society. New England Journal of Medicine 365(11):1033-1041.

Hughes, B. 2011. Priority voucher flops. Nature Biotechnology 29(958). http://www.nature.com/nbt/journal/v29/n11/full/nbt1111-958a.html (accessed April 3, 2012).

Ibrahim, A. 2003. Clinical review for Camptosar (irinotecan). NDA 20571. December 22. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsat fda_docs/nda/2004/20-571s021_Camptosar_medr.pdf (accessed April 3, 2012).

ICH (International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use). 1996. Guidance for industry: E6 good clinical practice consolidated guidance. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM073122.pdf (accessed April 3, 2012).

IOM (Institute of Medicine). 1992. Food and Drug Administration advisory committees. Washington, DC: National Academy Press.

IOM. 2000. Rational therapeutics for infants and children. Workshop summary. Washington, DC: National Academy Press.

IOM. 2004. Ethical conduct of clinical research involving children. Washington, DC: The National Academies Press.

IOM. 2007. Challenges for the FDA: The future of drug safety. Workshop summary. Washington, DC: The National Academies Press.

IOM. 2008. Addressing the barriers to pediatric drug development. Workshop summary. Washington, DC: The National Academies Press.

IOM. 2010. Rare diseases and orphan products. Washington, DC: The National Academies Press.

Issa, A. M. 2002. Ethical perspectives on pharmacogenomic profiling in the drug development process. Nature 1(4):300-308. http://www.nature.com/nrd/journal/v1/n4/pdf/nrd771.pdf (accessed April 3, 2012).

Jain, N. 2008. Final review memo for Kogenate FS (antihemophilic factor). BLA 103332/5794. September 10. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/BiologicsBloodVaccines/BloodBloodProducts/ApprovedProducts/LicensedProductsBLAs/FractionatedPlasmaProducts/UCM209295.pdf (accessed April 3, 2012).

Jenkins, J. 2001. Written request for Suprane (desflurane). NDA 20118. December 31. Silver Spring, MD: Food and Drug Administration.

Johnson, S. 2000. Clinical review for Advair Diskus (salmeterol/fluticasone propionate). NDA 21077. Parts 1, 2, and 3. August 23. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/nda/2000/21077_AdvairDiskus.cfm (accessed April 3, 2012).

Junod, S. W. 2002. Biologics centennial: 100 years of biologics regulation. In Update from the Food and Drug Law Institute. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/aboutfda/whatwedo/history/productregulation/selectionsfromfdliupdateseriesonfdahistory/ucm091754.htm (accessed April 3, 2012).

Kaiser, J. 2003. Medical officer’s efficacy review of Omalizumab (recombinant anti-human IgE). BLA 103976/0. June 20. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApproved/ApprovalApplications/TherapeuticBiologicApplications/ucm113459.pdf (accessed April 3, 2012).

Karimi-Shah, B. 2007. Clinical review for Asmanex (mometasone furoate). NDA 21067. December 3. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/ucm072501.pdf (accessed April 3, 2012).

Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×

Karkowsky, A. M. 2000. Clinical review for Betapace (sotalol). NDA 19865. September 18. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/nda/2001/19-865S010_Betapace_medr.pdf (accessed April 3, 2012).

Katz, K. A. 2007. Clinical review for Locoid (hydrocortisone butyrate). NDA 22076. May 11. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/nda/2007/022076s000_MedR.pdf (accessed April 3, 2012).

Katz, R. G. 2000. Approval letter for Neurontin (gabapentin). NDA 21216/20235/20882/21129. October 12. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/nda/2000/21-216.pdf_Neurontin_Approv.pdf (accessed April 3, 2012).

Katz, R. 2009a. Action memo for NDA 22-115 for the use of Lamictal (lamotrigine) XR tablets. NDA 22115, May 6. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/UCM187171 (accessed April 3, 2012).

Katz, R. 2009b. Approval letter for Axert (almotriptan). NDA 21001. April 30. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/appletter/2009/021001s010,s011ltr.pdf (accessed April 3, 2012).

Katz, R. G. 2010. Approval letter for Nuedexta (dextromethorphan hydrobromide and quinidine sulfate). NDA 021879. October 29. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/appletter/2010/021879s000ltr.pdf (accessed April 3, 2012).

Kearns, G. L., S. M. Abdel-Rahman, S. W. Alander, D. L. Blowey, J. S. Leeder, and R. E. Kauffman. 2003. Developmental pharmacology—Drug disposition, action, and therapy in infants and children. New England Journal of Medicine 349(12):1157-1167.

Kelly, R. S., A. D. Hunt, Jr., and S. G. Tashman. 1951. Studies on the absorption and distribution of chloramphenicol. Pediatrics 8(3):362-367.

Kempe, C. H. 1955. The use of antibacterial agents: Summary of round table discussion. Pediatrics 15(2):221-230.

Kesselheim, A. S., J. A. Myers, and J. Avorn. 2011. Characteristics of clinical trials to support approval of orphan vs nonorphan drugs for cancer. Journal of the American Medical Association 305(22):2320-2326.

Kjaersgaard, M., E. Wendtland, and H. Hasle. 2009. Subcutaneous anti-D treatment of idiopathic thrombocytopenic purpura in children. Pediatric Blood and Cancer 53(7):1315-1317.

Klein, R., and K. Struble. 2011. Kaletra (lopinavir/ritonavir) oral solution label changes related to toxicity in preterm neonates. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/ForConsumers/ByAudience/ForPatientAdvocates/HIVandAIDSActivities/ucm244639.htm (accessed April 3, 2012).

Kohly, R. P., R. H. Muni, P. J. Kertes, and W. C. Lam. 2011. Management of pediatric choroidal neovascular membranes with intravitreal anti-VEGF agents: A retrospective consecutive case series. Canadian Journal of Ophthalmology 46(1):46-50.

Korvick, J. A. 2008. Approval memo for Prilosec (omeprazole magnesium). NDA 22056. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/ucm072796.pdf (accessed April 3, 2012).

Kozlowski, S., J. Woodcock, K. Midthun, and R. Behrman Sherman. 2011. Developing the nation’s biosimilars program. New England Journal of Medicine 365(5):385-388. http://www.nejm.org/doi/pdf/10.1056/NEJMp1107285 (accessed April 3, 2012).

Krudys, K., and V. Arya. 2008. Clinical pharmacology for Valcyte (valganciclovir hydrochloride). NDA 22257/000. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/UCM222178.pdf (accessed April 3, 2012).

Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×

Krug, S. E., V. F. Tait, and L. Aird. 2011. Helping the helpers to help children: Advances by the American Academy of Pediatrics. Pediatrics 128(Suppl. 1):537-539. http://pediatrics.aappublications.org/content/128/Supplement_1/S37.full (accessed April 3, 2012).

Ku, J., and E. Hausman. 2009. Clinical review for Creon (pancrelipase). NDA 20725. April 30. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/UCM261988.pdf (accessed April 3, 2012).

Kuehn, B. M. 2010. Frances Kelsy honored for FDA legacy. Journal of the American Medical Association 304(19):2109-2112. http://jama.ama-assn.org/content/304/19/2109.full.pdf (accessed April 3, 2012).

Kukich, S., and S. Walker. 2007. Approval letter for Locoid (hydrocortisone butyrate). NDA 22076. May 18. Silver Spring, MD: Food and Drug Administration. http://www.access data.fda.gov/drugsatfda_docs/appletter/2007/022076s000ltr.pdf (accessed April 3, 2012).

Kweder, S. L. 1999. Written request for Zerit (stavudine). NDA 20412, 20413. August 20. Silver Spring, MD: Food and Drug Administration.

Laine, C., R. Horton, C. D. DeAngelis, J. M. Drazen, F. A. Frizelle, F. Godlee, C. Haug, P. C. Hébert, S. Kotzin, A. Marusic, P. Sahni, T. V. Schroeder, H. C. Sox, M. B. van der Weyden, and F. W. A. Verheugt. 2007. Clinical trial registration—Looking back and moving ahead. New England Journal of Medicine 356(26):2734-2736.

Laughren, T. P. 2000. Recommendation for approvable action for pediatric supplement for Buspar (buspirone): Negative results for Buspar in the treatment of generalized anxiety disorder in pediatric patients (to be filed with the 3.20.2000 submission of the supplement). NDA 18731/043. Memorandum from Team Leader, Psychiatric Drug Products, December 29 (pp. 33-37 of approval package). Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/nda/2001/18-731S043_review.pdf (accessed April 3, 2012).

Laughren, T. P. 2006. Approval letter for Risperdal (risperidone). NDA 20272/20588/21444. October 6. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/appletter/2006/020272s36,s41,20588s24,s28,s29,21444s8,s15LTR.pdf (accessed April 3, 2012).

Laughren, T. P. 2009a. Approval letter for Abilify (aripiprazole). NDA 021436. November 19. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/appletter/2009/021436s027ltr.pdf (accessed April 3, 2012).

Laughren, T. P. 2009b. Approval letter for Intuniv (guanfacine). NDA 22037. September 2. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/appletter/2009/022037s000ltr.pdf (accessed April 3, 2012).

Laughren, T. P. 2009c. Recommendation for complete response action for SPD503 (guanfacine) extended release tablets for the treatment of attention deficit hyperactivity disorder (ADHD). NDA 22037. July 27. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/UCM226659.pdf (accessed April 3, 2012).

Lee, B. H., B. J. Stoll, S. A. McDonald, and R. D. Higgins. 2008. Neurodevelopmental outcomes of extremely low birth weight infants exposed prenatally to dexamethasone versus betamethasone. Pediatrics 121(2):289-296.

Levin, R. L. 2007. Clinical review for Intuniv (guanfacine hydrochloride). NDA 22037. June 12. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/UCM226549.pdf (accessed April 3, 2012).

Levorson, R. 2010. Clinical review for Viread (tenofovir DF). NDA 021356. March 23. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/UCM209151.pdf (accessed April 3, 2012).

Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×

Li, J. S., E. L. Eisenstein, H. G. Grabowski, E. D. Reid, B. Mangum, K. A. Schulman, J. V. Goldsmith, M. D. Murphy, R. M. Califf, and D. K. Benjamin. 2007. Economic return of clinical trials performed under the pediatric exclusivity program. Journal of the American Medical Association 297(5):480-488.

Lima, J. J., K. V. Blake, K. G. Tantisira, and S. T. Weiss. 2009. Pharmacogenetics of asthma. Current Opinion in Pulmonary Medicine 15(1):57-62.

Lovell, D. J., E. H. Giannini, A. Reiff, G. D. Cawkwell, E. D. Silverman, J. J. Nocton, L. D. Stein, A. Gedalia, N. T. Ilowite, C. A. Wallace, J. Whitmore, and B. K. Finck. 2000. Etanercept in children with polyarticular juvenile rheumatoid arthritis. New England Journal of Medicine 342(11):763-769.

Lovell, D. J., N. Ruperto, S. Goodman, A. Reiff, L. Jung, K. Jarosova, D. Nemcova, R. Mouy, C. Sandborg, J. Bohnsack, D. Elewaut, I. Foeldvari, V. Gerloni, J. Rovensky, K. Minden, R. K. Vehe, L. W. Weiner, G. Horneff, H. I. Huppertz, N. Y. Olson, J. R. Medich, R. Carcereri-De-Prati, M. J. McIlraith, E. H. Giannini, and A. Martini. 2008. Adalimumab with or without methotrexate in juvenile rheumatoid arthritis. New England Journal of Medicine 359(8):810-820.

Malarkey, M. A., and N. W. Baylor. 2011. Approval letter for adenovirus type 4 and type 7 vaccine, live, oral. BLA 125296. March 16. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/BiologicsBloodVaccines/Vaccines/ApprovedProducts/ucm247511.htm (accessed April 3, 2012).

Malarkey, M. A., and J. S. Epstein. 2009. Approval letter for ATryn (antithrombin (recombinant)). BL 125284. February 6. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/BiologicsBloodVaccines/BloodBloodProducts/ApprovedProducts/LicensedProductsBLAs/FractionatedPlasmaProducts/ucm134046.htm (accessed April 3, 2012).

Malarkey, M. A., and J. S. Epstein. 2010. Approval letter for Glassia (alpha-1 proteinase inhibitor (human)). BLA 125325. July 1. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/BiologicsBloodVaccines/BloodBloodProducts/ApprovedProducts/LicensedProductsBLAs/FractionatedPlasmaProducts/ucm217888.htm (accessed April 3, 2012).

Malarkey, M. A., and J. S. Epstein. 2011. Approval letter for Kedbumin (albumin (human)). BLA 125384/0. June 3. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/BiologicsBloodVaccines/BloodBloodProducts/ApprovedProducts/LicensedProductsBLAs/FractionatedPlasmaProducts/ucm257838.htm (accessed April 3, 2012).

Maldonado, S., J. Berlin, J. Siegel, and J. Waldstreicher. 2011. Globalized pediatric research. Pediatrics 127(1):e251-e252.

Marshall, J. D., S. M. Abdel-Rahman, K. Johnson, R. E. Kauffman, and G. L. Kearns. 1999. Rifapentine pharmacokinetics in adolescents. The Pediatric Infectious Disease Journal 18(10):882-888.

Mathis, L., and N. Jain. 2011. Pediatric labeling. Presentation to Institute of Medicine Committee on Pediatric Studies Conducted Under BPCA and PREA, Meeting 2, Washington, DC, February 2.

MCC (Masonic Cancer Center). 2010. Information for HOPE study participants: For mothers of children who have been diagnosed with hepatoblastoma. Minneapolis, MN: Masonic Cancer Center, University of Minnesota. http://www.cancer.umn.edu/research/programs/pehopestudyinfo1.html (accessed April 3, 2012).

McCracken, G. H., Jr., C. M. Ginsburg, T. C. Zweighaft, and J. Clahsen. 1980. Pharmacokinetics of rifampin in infants and children: Relevance to prophylaxis against Haemophilus influenza type B disease. Pediatrics 66(1):17-21.

Meissner, H. C., and A. L. Smith. 1979. The current status of chloramphenicol. Pediatrics 64(3):348-356.

Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×

Metz, B. J., M. C. Rubenstein, M. L. Levy, and D. W. Metry. 2004. Response of ulcerated perineal hemangiomas of infancy to becaplermin gel, a recombinant human platelet-derived growth factor. Archives of Dermatology 140(7):867-870.

Meyer, R. 2000. Approval letter for Advair Diskus (fluticasone propionate and salmeterol xinafoate). NDA 21077. August 24. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/nda/2000/21077_Advair%20Diskus_Approv.pdf (accessed April 3, 2012).

Meyer, R. 2004. Approval letter for Apidra (insulin glulisine (rDNA origin)). NDA 21629. April 16. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/appletter/2004/21629ltr.pdf (accessed April 3, 2012).

Meyer, R. J. 2006. Written request amendment for Suprane (desflurane). NDA 20118. March 6. Silver Spring, MD: Food and Drug Administration.

Miller, F. G., and A. F. Shorr. 2002. Unnecessary use of placebo controls: The case of asthma clinical trials. Archives of Internal Medicine 162(15):1673-1677.

Milne, C. 2009. Pharmaceutical economics and applications to pediatrics: Business case development. In Pediatric drug development: Concepts and applications, edited by A. E. Mulberg, S. A. Silber, and J. N. van den Anker. Hoboken, NJ: John Wiley & Sons, Inc. Pp. 39-57.

Mishina, E. V. 2007. Clinical pharmacology review for guanfacine PK. NDA 22037. June 4. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/UCM226551.pdf (accessed April 3, 2012).

Molloy, E. J., J. M. DiFore, and R. J. Martin. 2005. Does gastroesophageal reflux cause apnea in preterm infants? Biology of the Neonate 87(4):254-261.

Moran, C., C. D. Thornburg, and R. C. Barfield. 2011. Ethical considerations for pharmacogenomic testing in pediatric clinical care and research. Pharmacogenomics 12(6):889-895.

Mosholder, A. 2000. Clinical review for Concerta (methylphenidate HCl). NDA 20121. March 23. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/nda/2000/21-121_Concerta_medr.pdf (accessed April 3, 2012).

Mousa, H., F. W. Woodley, M. Metheney, and J. Hayes. 2005. Testing the association between gastroesophageal reflux and apnea in infants. Journal of Pediatric Gastroenterology and Nutrition 41(2):169-177.

Murphy, D. 2000. Presentation on placebo-controlled pediatric trials for the FDA Pediatric Subcommittee of the Anti-Infective Drugs Advisory Committee, September 11, Bethesda, MD. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/ohrms/dockets/ac/00/transcripts/3641t1.rtf (accessed April 3, 2012).

Murphy, D. 2011. Overview of the May 16, 2011 meeting of the Pediatric Advisory Committee April 27. Memorandum from Dianne Murphy to members of the Pediatric Advisory Committee and invited expert consultants. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/PediatricAdvisoryCommittee/UCM255156.pdf (accessed April 3, 2012).

Murray, J. 2006. Written request for Prezista (darunavir). NDA 21976. November 17. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/UCM258689.pdf (accessed April 3, 2012).

Murray, J. 2007. Written request amendment for Prezista (darunavir). NDA 21976. August 16. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/UCM258694.pdf (accessed April 3, 2012).

Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×

Murray, J. 2008. Approval letter for Prezista (darunavir). NDA 21976. December 18. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/appletter/2008/021976s009ltr.pdf (accessed April 3, 2012).

Nahata, M. C. 2009. Safety of “inert” additives or excipients in paediatric medicines. Archives of Disease in Childhood, Fetal Neonatal Edition 94(6):F392-F393.

Nahata, M. C., and L. V. Allen, Jr. 2008. Extemporaneous drug formulations. Clinical Therapeutics 30(11):2112-2119.

NAS (National Academy of Sciences). Undated. The drug efficacy study of the National Research Council’s Division of Medical Sciences, 1966-1969. Washington, DC: National Academy of Sciences. http://www7.nationalacademies.org/archives/drugefficacy.html (accessed April 3, 2012).

National Commission (National Commission for the Protection of Human Subjects of Biomedical and Behavioral Research). 1977. Report and recommendations: Research involving children. Washington, DC: U.S. Government Printing Office.

NBAC (National Bioethics Advisory Commission). 2001. Ethical and policy issues in international research: Clinical trials in developing countries, vol. 1. Bethesda, MD: National Bioethics Advisory Commission. http://bioethics.georgetown.edu/nbac/clinical/Vol1.pdf (accessed April 3, 2012).

NCI (National Cancer Institute). 2011. Late effects of treatment for childhood cancer, fertility section. Bethesda, MD: National Cancer Institute. http://www.cancer.gov/cancertopics/pdq/treatment/lateeffects/HealthProfessional/AllPages#Section_395 (accessed April 3, 2012).

Neergaard, L. 2000. A warning for tiny hearts: Safety of heartburn drug Propulsid in question. Pittsburgh Post-Gazette, February 15.

Nelson, R. 2010. The scientific and ethical path forward in pediatric product development. Presentation at the NIH Human Subjects Research Course. Silver Spring, MD: Food and Drug Administration. http://www.bioethics.nih.gov/hsrc/slides/Nelson%20-%20NIH%20HSP%20Course%2010-20-2010.pdf (accessed April 3, 2012).

Nelson, R. 2011a. Evaluating exploratory IND studies according to the ethical framework of 21 CFR 50 Subpart D. Presentation to FDA Pediatric Advisory Committee, May 11. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/PediatricAdvisoryCommittee/UCM254788.pdf (accessed April 3, 2012).

Nelson, R. 2011b. FDA presentation to Committee on Pediatric Studies Conducted Under BPCA and PREA, Meeting 2, Washington, DC, February 2. http://www.iom.edu/~/media/Files/Activity%20Files/Children/PediatricBPCAPREA/FDA%20Presentation%2002022011Final.pdf (accessed April 3, 2012).

Neubert, A., K. Lukas, T. Leis, H. Dormann, K. Brune, and W. Rascher. 2010. Drug utilisation on a preterm and neonatal intensive care unit in Germany: A prospective, cohort-based analysis. European Journal of Clinical Pharmacology 66(1):87-95.

Neville, K. A., M. L. Becker, J. L. Goldman, and G. L. Kearns. 2011. Developmental pharmacogenomics. Pediatric Anesthesia 21(3):255-265.

Nevitt, M. P. 2009. Clinical review of Zymar (gatifloxacin ophthalmic solution, 0.3%). NDA 21493. August 3. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/UCM187155.pdf (accessed April 3, 2012).

Newacheck, P. W., and A. E. Benjamin. 2004. Intergenerational equity and public spending: The United States should embrace a new doctrine of fairness to ensure that vulnerable populations are not forced to compete for resources. Health Affairs 23(5):142-146. http://content.healthaffairs.org/content/23/5/142.full.html (accessed April 3, 2012).

Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×

NHLBI (National Heart, Lung, and Blood Institute). 1991. Guidelines for the diagnosis and management of asthma. Expert panel report. Bethesda, MD: National Asthma Education Program, National Heart, Lung, and Blood Institute. http://www.nhlbi.nih.gov/guidelines/asthma/asthgdln.htm (accessed April 3, 2012).

NHLBI. 2007. Guidelines for the diagnosis and management of asthma. Expert panel report 3. Bethesda, MD: National Asthma Education and Prevention Program, National Heart, Lung, and Blood Institute. http://www.nhlbi.nih.gov/guidelines/asthma/asthgdln.pdf (accessed April 3, 2012).

NICHD (National Institute of Child Health and Human Development). 1998. Letter of intent: Network of Pediatric Pharmacology Research Units (PPRU). Bethesda, MD: National Institute of Child Health and Human Development. http://grants.nih.gov/grants/guide/rfa-files/RFA-HD-98-002.html (accessed April 3, 2012).

NICHD. 2003. Minutes for Best Pharmaceuticals for Children Act prioritization meeting, December 12, 2003. Bethesda, MD: National Institute of Child Health and Human Development. http://bpca.nichd.nih.gov/about/process/upload/list_meeting_12122003.pdf (accessed April 3, 2012).

NICHD. 2006. Minutes from Best Pharmaceuticals for Children Act (BPCA) Pediatric Formulation Initiative (PFI) working meeting, December 6-7. Bethesda, MD: National Institute of Child Health and Human Development. http://bpca.nichd.nih.gov/collaborativeefforts/upload/pfi_meeting_12062005.pdf (accessed April 3, 2012).

NICHD. 2008. Meeting minutes for Best Pharmaceuticals for Children Act scientific prioritization, June 30 and July 1, 2008. Bethesda, MD: National Institute of Child Health and Human Development. http://bpca.nichd.nih.gov/collaborativeefforts/upload/2008_bpca_scientific_prioritization_meeting_minutes.pdf (accessed April 3, 2012).

NICHD. 2010. Best Pharmaceuticals for Children Act (BPCA) adolescent therapeutics working group conference call and webcast, April 16. Bethesda, MD: National Institute of Child Health and Human Development. http://bpca.nichd.nih.gov/about/process/upload/Adolescent_Therapeutics_WG_final_04_23_10_T.pdf (accessed April 3, 2012).

NICHD. 2011. Best Pharmaceuticals for Children Act (BPCA) priority list of needs in pediatric therapeutics. Bethesda, MD: National Institute of Child Health and Human Development. http://bpca.nichd.nih.gov/about/process/upload/2011_Priority_List_and_Summary_for_web_posting_04_07_11.pdf (accessed April 3, 2012).

NICHD/FDA (Food and Drug Administration). 2004. Newborn drug development initiative. Workshop summary. Bethesda, MD: National Institute of Child Health and Human Development. http://bpca.nichd.nih.gov/collaborativeefforts/upload/nddi_meeting_03292004.pdf (accessed April 3, 2012).

Nicklin, S., and S. A. Spencer. 2004. Recruitment failure in early neonatal research (letter). Archives of Disease in Childhood, Fetal Neonatal Edition 89(3):F281. http://www.ncbi.nlm.nih.gov/pmc/articles/PMC1721676/pdf/v089p0F281.pdf (accessed April 3, 2012).

NIH (National Institutes of Health). 1998. NIH policy and guidelines on the inclusion of children as participants in research involving human subjects, March 6. Online edition. Bethesda, MD: National Institutes of Health.

NIH. 2008. Pediatric Pharmacology and Therapeutics Research Consortium (PPTRC). RFA-HD-08-021. Bethesda, MD: National Institutes of Health. http://grants.nih.gov/grants/guide/rfa-files/rfa-hd-08-021.html (accessed April 3, 2012).

NIH. 2011a. Grant overview information. Bethesda, MD: National Institutes of Health. http://grants.nih.gov/grants/guide/pa-files/PAR-11-302.html (accessed April 3, 2012).

NIH. 2011b. The NIH almanac: Chronology of events. Bethesda, MD: National Institutes of Health. http://www.nih.gov/about/almanac/historical/chronology_of_events.htm (accessed April 3, 2012).

Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×

Nishi, Y. 2010. Hepatoblastoma concerns and growth hormone therapy in small for gestational age children. Growth, Genetics and Hormones 26(1). http://www.gghjournal.com/volume26/1/ab01.cfm (accessed April 3, 2012).

NOVA (National Organization of Vascular Anomalies). 2010. Regranex and topicals. Greensboro, NC: National Organization of Vascular Anomalies. http://www.novanews.org/information/hemangioma/hemangioma-treatment/regranex-and-topicals (accessed April 3, 2012).

NPC (Novartis Pharmaceuticals Corporation). 2012. Prescribing information for Zometa (zoledronic acid) injection. East Hanover, NJ: Novartis Pharmaceuticals Corporation. http://www.pharma.us.novartis.com/product/pi/pdf/Zometa.pdf (accessed January 13, 2012).

NRC (National Research Council). 1969. Drug efficacy study: A report to the Commissioner of Food and Drugs. Washington, DC: National Technical Information Service.

O’Connor, A. B. 2009. The need for improved access to FDA reviews. Journal of the American Medical Association 302(2):191-193.

O’Donnell, C. P., R. J. Stone, and C. J. Morley. 2002. Unlicensed and off-label drug use in an Australian neonatal intensive care unit. Pediatrics 110(5):e52. http://pediatrics.aappublications.org/content/110/5/e52.full (accessed April 3, 2012).

OIG/HHS (Office of the Inspector General, U.S. Department of Health and Human Services). 2001. The globalization of clinical trials: A growing challenge in protecting human subjects. Washington, DC: U.S. Department of Health and Human Services. http://oig.hhs.gov/oei/reports/oei-01-00-00190.pdf (accessed April 3, 2012).

OIG/HHS. 2006. FDA’s monitoring of postmarketing study commitments. Washington, DC: U.S. Department of Health and Human Services. http://www.oig.hhs.gov/oei/reports/oei-01-04-00390.pdf (accessed April 3, 2012).

OIG/HHS. 2011. Review of Medicare Part B Avastin and Lucentis Treatments for Age-Related Macular Degeneration. A-01-10-00514. http://oig.hhs.gov/oas/reports/region10/11000514.pdf (accessed April 3, 2012).

O’Leary, M., M. Krailo, J. R. Anderson, and G. H. Reaman. 2008. Progress in childhood cancer: 50 years of research collaboration, a report from the Children’s Oncology Group. Seminars in Oncology 35(5):484-493.

Olechnowicz, J. Q., M. Eder, C. Simon, S. Zyzanski, and E. Kodish. 2002. Assent observed: Children’s involvement in leukemia treatment and research discussions. Pediatrics 109(5):806-814.

Osterman, M. J. K., J. A. Martin, and F. Menacker. 2009. Expanded health data from the new birth certificate, 2006. National Vital Statistics Reports 58(5). Hyattsville, MD: National Center for Health Statistics. http://www.cdc.gov/nchs/data/nvsr/nvsr58/nvsr58_05.pdf (accessed April 3, 2012).

PAC (Pediatric Advisory Committee). 2009. Transcript of Pediatric Advisory Committee meeting, December 8. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/PediatricAdvisoryCommittee/UCM198447.pdf (accessed April 3, 2012).

Panel on Antiretroviral Therapy and Medical Management of HIV-Infected Children. 2011. Guidelines for the use of antiretroviral agents in pediatric HIV infection. August 11. http://aidsinfo.nih.gov/contentfiles/PediatricGuidelines.pdf (accessed April 3, 2012).

Parent, A.-S., G. Teilmann, A. Juul, N. E. Skakkebaek, J. Toppari, and J.-P. Bourguignon. 2003. The timing of normal puberty and the age limits of sexual precocity: Variations around the world, secular trends, and changes after migration. Endocrine Reviews 24(5):668-693.

Pariser, A. 2010. FDA regulation and review of small clinical trials. Presentation to Institute of Medicine Committee on Accelerating Rare Diseases Research and Orphan Product Development, Washington, DC. February 4.

Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×

PAS/AIDAC (Pediatric Advisory Subcommittee of the Anti-Infective Drugs Advisory Committee). 2002. Meeting transcript for subcommittee meeting, July 11, 2002. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/ohrms/dockets/ac/02/transcripts/3870T1.htm (accessed April 3, 2012).

PAS/AIDAC. 2003. Summary minutes of the Pediatrics Subcommittee of the Anti-Infective Drugs Advisory Committee meeting, March 3, 2003. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/ohrms/dockets/ac/03/minutes/3932M1.doc (accessed April 3, 2012).

Pasquali, S. K., D. S. Burstein, D. K. Benjamin, Jr., P. B. Smith, and J. S. Li. 2010. Globalization of pediatric research: Analysis of clinical trials completed for pediatric exclusivity. Pediatrics 126(3):e687-e692. http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2932835/pdf/nihms218026.pdf (accessed April 3, 2012).

Pazdur, R. 2004. Approval letter for Camptosar (irinotecan hydrochloride). NDA 20571. June 24. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/appletter/2004/20571se8-021ltr.pdf (accessed April 3, 2012).

Pazdur, R. 2008. Approval letter for Nplate (romiplostim). BL 125268/0. August 22. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/appletter/2008/125268s000ltr.pdf (accessed April 3, 2012).

PeRC (Pediatric Review Committee). 2010. Retrospective review of information submitted and actions taken in response to PREA 2003. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/UCM197636.pdf (accessed April 3, 2012).

Peter, C. S., N. Sprodowski, B. Bohnhorst, J. Silny, and C. F. Poets. 2002. Gastroesophageal reflux and apnea of prematurity: No temporal relationship. Pediatrics 109(1):8-11.

PhRMA (Pharmaceutical Research and Manufacturers of America). 2011a. Report for the Institute of Medicine: Challenges and successes in neonatal drug development. October 3. Washington, DC: Pharmaceutical Research and Manufacturers of America. http://www.phrma.org/sites/default/files/406/phrmareporttoiomneonataldrugdev10-3-2011final.pdf (accessed April 3, 2012).

PhRMA. 2011b. Statement before the Institute of Medicine Committee on Pediatric Studies Conducted Under BPCA and PREA, April 28. http://www.phrma.org/phrma-statement-institute-medicine-committee-pediatric-studies-conducted-under-bpca-prea (accessed April 3, 2012).

Pikis, A. 2009. Clinical review for Valcyte (valganciclovir). NDA 22257. August 20. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/UCM222175.pdf (accessed April 3, 2012).

Protherics, Inc. 2010. Prescribing information for CroFab (crotalidae polyvalent immune fab (ovine)). Brentwood, TN: Protherics, Inc. http://www.crofab.com/viewpdf.do?documentId=1 (accessed April 3, 2012).

Proytcheva, M. A. 2009. Issues in neonatal cellular analysis. American Journal of Clinical Pathology 131(4):560-573. http://ajcp.ascpjournals.org/content/131/4/560.full.pdf (accessed April 3, 2012).

Raczkowski, V. 1999. Written request for Diprivan (propofol). NDA 19627. April 22. Silver Spring, MD: Food and Drug Administration.

Raczkowski, V. 2001. Written request for Protonix (pantoprazole sodium). NDA 20987/20988. December 31. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/ucm121765.pdf (accessed April 3, 2012).

Rakhmanina, N. Y., and J. N. van den Anker. 2009. Developmental pharmacology issues: Neonates, infants, and children. In Pediatric drug development: Concepts and applications, edited by A. E. Mulberg, S. A. Silber, and J. N. van den Anker. Hoboken, NJ: John Wiley & Sons, Inc. Pp. 231-242.

Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×

Rappaport, B. A. 2008. Approval letter for Orencia (abatacept). BLA 125118/045. April 7. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/appletter/2008/125118s045ltr.pdf (accessed April 3, 2012).

Rappaport, B. A. 2011a. Approval letter for Abstral (fentanyl). NDA 022510. January 7. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/appletter/2011/022510s000ltr.pdf (accessed April 3, 2012).

Rappaport, B. A. 2011b. Approval letter for Gralise (gabapentin). NDA 22544. January 28. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/appletter/2011/022544s000ltr.pdf (accessed April 3, 2012).

Rappaport, B. A. 2011c. Overview of the March 10, 2011, ALSDAC meeting to discuss the neurotoxicity of anesthetic and sedative drugs in juvenile animals memo. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/AnestheticAndLifeSupportDrugsAdvisoryCommittee/UCM245769.pdf (accessed April 3, 2012).

Rappaport, B., R. D. Mellon, A. Simone, and J. Woodcock. 2011. Defining safe use of anesthesia in children. New England Journal of Medicine 364(15):1387-1390. http://www.nejm.org/doi/pdf/10.1056/NEJMp1102155 (accessed April 3, 2012).

Reed, M., and P. Gal. 2004. Principles of drug therapy. In Nelson textbook of pediatrics, 17th ed, edited by R. Berhman, R. Kliegman, and H. Jenson. Philadelphia, PA: WB Saunders. Pp. 2427-2431.

Richman, P. G. 2007. Approval letter for Twinrix (hepatitis A inactivated and hepatitis B vaccine). BLA 103850. March 28. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/BiologicsBloodVaccines/Vaccines/ApprovedProducts/ucm110089.htm (accessed April 3, 2012).

Risso, S. T. 2003. Approval letter for Xolair (omalizumab). BLA 103976/0. June 20. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/appletter/2003/omalgen062003L.htm (accessed April 3, 2012).

Roberts, R., W. Rodriguez, D. Murphy, and T. Crescenzi. 2003. Pediatric drug labeling: Improving the safety and efficacy of pediatric therapies. Journal of the American Medical Association 290(7):905-911. http://jama.ama-assn.org/content/290/7/905.full.pdf (accessed April 3, 2012).

Robertson, A. F. 2003a. Reflections on errors in neonatology. II. The “heroic” years, 1950 to 1970. Journal of Perinatology 23(2):154-161. http://www.nature.com/jp/journal/v23/n2/pdf/7210843a.pdf (accessed April 3, 2012).

Robertson, A. F. 2003b. Reflections on errors in neonatology. III. The “experienced” years, 1970 to 2000. Journal of Perinatology 23(3):240-249. http://www.nature.com/jp/journal/v23/n3/pdf/7210873a.pdf (accessed April 3, 2012).

Robertson, R. 2011. Healthpoint biotherapeutics acquires Regranex gel. PR Newswire, June 2. http://www.prnewswire.com/news-releases/healthpoint-biotherapeutics-acquires-regranex-gel-123016133.html (accessed April 3, 2012).

Roca, R. 2008. Approval letter for Humira (adalimumab). BLA 125057/114. February 21. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/appletter/2008/125057s114ltr.pdf (accessed April 3, 2012).

Rodriguez, W., A. Selen, D. Avant, C. Chaurasia, T. Crescenzi, G. Gieser, J. Di Giacinto, S. Huang, P. Lee, L. Mathis, D. Murphy, S. Murphy, R. Roberts, H. C. Sachs, S. Suarez, V. Tandon, and R. S. Uppoor. 2008. Improving pediatric dosing through pediatric initiatives: What we have learned. Pediatrics 121(3):530-539. http://pediatrics.aappublications.org/content/121/3/530.full.html (accessed April 3, 2012).

Rogawski, M. A., and H. J. Federoff. 2011. Disclosure of clinical trial results when product development is abandoned. Science Translational Medicine 3(102):102cm29.

Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×

Roman, D. 2007. Medical review for Arimidex (anastrozole). NDA 22214/000. February 19. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/ucm129540.pdf (accessed April 3, 2012).

Rose, M. 2010. Clinical review for Plavix (clopidogrel bisulfate). NDA 20839. January 10. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/nda/2011/020839Orig1s051MedR.pdf (accessed April 3, 2012).

Rosebraugh, C. J. 2010. Letter from Curtis J. Rosebraugh, M.D., M.P.H., Director, Office of Drug Evaluation II, FDA, to Mary Ellen Anderson, Senior Director, Regulatory Affairs, INO Therapeutics, April 30. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/UCM232507.pdf (accessed April 3, 2012).

Ross, D. B. 2007. The FDA and the case of Ketek. New England Journal of Medicine 356(16):1601-1604. http://www.nejm.org/doi/full/10.1056/NEJMp078032 (accessed April 3, 2012).

Ross, J. 2010. Elements of FDAAA 2007 and their implementation within CBER; comments on IOM Tasks 5, 6, and 7. Presentation to Institute of Medicine Committee on Pediatric Studies Conducted Under BPCA and PREA, Meeting 1, Washington, DC, December 17.

Roth-Cline, M., J. Gerson, P. Bright, C. S. Lee, and R. M. Nelson. 2011. Ethical considerations in conducting pediatric research. Handbook of Experimental Pharmacology 205:219-244.

Ruperto, N., D. J. Lovell, P. Quartier, N. Rubio-Pérez, C. A. Silva, C. Abud-Mendoza, R. Burgos-Vargas, V. Gerloni, J. A. Melo-Gomes, C. Saad-Magalhães, F. Sztajnbok, C. Goldenstein-Schainberg, M. Scheinberg, I. C. Penades, M. Fischbach, J. Orozco, P. J. Hashkes, C. Hom, L. Jung, L. Lepore, S. Oliveira, C. A. Wallace, L. H. Sigal, A. J. Block, A. Covucci, A. Martini, and E. H. Giannini. 2008. Abatacept in children with juvenile idiopathic arthritis: A randomised, double-blind, placebo-controlled withdrawal trial. The Lancet 372(9636):383-391.

SACGHS (Secretary’s Advisory Committee on Genetics, Health, and Society). 2008. Realizing the potential of pharmacogenomics: Opportunities and challenges. http://oba.od.nih.gov/oba/SACGHS/reports/SACGHS_PGx_report.pdf (accessed April 3, 2012).

SACHRP (Secretary’s Advisory Committee on Human Research Protections). 2005. Meeting minutes, November 2. Washington, DC: U.S. Department of Health and Human Services. http://www.hhs.gov/ohrp/archive/sachrp/mtgings/mtg11-05/1102DHHS.TXT (accessed April 3, 2012).

Sanofi-Aventis. 2009. Prescribing information for Priftin (rifapentine). Bridgewater, NJ: Sanofi-Aventis. http://products.sanofi.us/priftin/Priftin.pdf (accessed April 3, 2012).

Sanofi-Aventis. 2011. Prescribing information for Plavix (clopidogrel bisulfate). Bridgewater, NJ: Sanofi-Aventis. http://products.sanofi.us/plavix/plavix.pdf (accessed April 3, 2012).

Schmidt, B., P. J. Anderson, L. W. Doyle, D. Dewey, R. E. Grunau, E. V. Asztalos, P. G. Davis, W. Tin, D. Moddemann, A. Solimano, A. Ohlsson, K. J. Barrington, and R. S. Roberts. 2012. Survival without disability to age 5 after neonatal caffeine therapy for apnea of prematurity. Journal of the American Medical Association 307(3):275-282.

Schneider, B. S. 2003. Clinical review for Fosamax (alendronate sodium). NDA 20560. June 7. Silver Spring, MD: Food and Drug Administration.

Schrager, L. K. 2011. Clinical review for adenovirus type 4 and type 7 vaccine, live, oral enteric coated tablets biologics license application. BLA 125296. March 15. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/BiologicsBloodVaccines/Vaccines/ApprovedProducts/UCM250619.pdf (accessed April 3, 2012).

Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×

Schultheis, L., and R. Roca. 2008. Clinical review for Zemuron (rocuronium bromide). NDA 20214. June 11. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/ucm072814.pdf (accessed April 3, 2012).

Sherman, R. B., J. Woodcock, J. Norden, C. Grandinetti, and R. J. Temple. 2011. New FDA regulation to improve safety reporting in clinical trials. New England Journal of Medicine 365(1):3-5. http://www.nejm.org/doi/pdf/10.1056/NEJMp1103464 (accessed April 3, 2012).

Shibuya, R. B. 2006. Clinical review for Suprane (desflurane). NDA 20118. October 30. Silver Spring, MD: Food and Drug Administration.

Shirkey, H. 1968. Therapeutic orphans. Journal of Pediatrics 72(1):119-120.

Siegel, J. 2008a. Adalimumab (Humira). BLA supplement 125057/114. January 7. Cross Discipline Team Leader memorandum. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/UCM071555.pdf (accessed April 3, 2012).

Soreth, J., E. Cox, S. Kweder, J. Jenkins, and S. Galson. 2007. Correspondence: Ketek—The FDA perspective. New England Journal of Medicine 356(16):1675-1676. http://www.nejm.org/doi/pdf/10.1056/NEJMc076135 (accessed April 3, 2012).

Spaulding, J. A. 2009. Clinical review for Ofirmev (acetaminophen). NDA 022450. October 6. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/UCM215814.pdf (accessed April 3, 2012).

Starke, P. 2003. Clinical summary of Flovent (fluticase propionate). NDA 20548. June 4. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/ohrms/dockets/ac/04/briefing/2004-4067b1_12_flovent%20medical.pdf (accessed April 3, 2012).

Starke, P. 2009. Clinical review for Xolair (omalizumab). BLA 103976/5149. December 4. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/UCM202179.pdf (accessed April 3, 2012).

Stein, E. A. 2007. Statins and children: Whom do we treat and when? Circulation 116(6):594-595.

Summers, J. 2008. Clinical review for Neulasta (pegfilgrastim). STN 125031/105. November 2. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/UCM182634.pdf (accessed April 3, 2012).

Sun, W. 2008. Approval letter for BioThrax (anthrax vaccine adsorbed). BLA 103821. December 11. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/BiologicsBloodVaccines/Vaccines/ApprovedProducts/ucm124462.htm (accessed April 3, 2012).

Sun, W. 2011. Approval letter for Fluzone Intradermal (influenza virus vaccine). BL 103914. May 9. http://www.fda.gov/BiologicsBloodVaccines/Vaccines/ApprovedProducts/ucm255160.htm (accessed April 3, 2012).

Sutherland, J. M. 1959. Fatal cardiovascular collapse of infants receiving large amounts of chloramphenicol. A. M. A. Journal of Diseases of Children 97(6):761-767.

Taddio, A., L. O’Brien, M. Ipp, D. Stephens, M. Goldbach, and G. Koren. 2009. Reliability and validity of observer ratings of pain using the visual analog scale (VAS) in infants undergoing immunization injections. Pain 147(1-3):141-146.

Temple, R. 1999. Written request amended for Buspar (buspirone HCI). NDA 18731. March 15. Silver Spring, MD: Food and Drug Administration.

Temple, R. 2003. Written request for Abilify (aripiprazole). NDA 21436. February 11. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/ucm073289.pdf (accessed April 3, 2012).

Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×

Temple, R. 2010. Draft questions for Sildenafil written request. July 29. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/CardiovascularandRenalDrugsAdvisoryCommittee/UCM220247.pdf (accessed April 3, 2012).

Temple, R., and S. S. Ellenberg. 2000. Placebo-controlled trials and active-control trials in the evaluation of new treatments. Annals of Internal Medicine 133(6):455-463.

Temple, R., and R. Meyer. 2003. Continued need for placebo in many cases, even when there is effective therapy. Archives of Internal Medicine 163(3):371-373.

Teva Pharmaceuticals. 2008. Prescribing information for rocuronium bromide. Petach Tikva, Israel: Teva Pharmaceuticals. http://www.tevausa.com/assets/base/products/pi/Rocuronium_PI_11-2008.pdf (accessed April 3, 2012).

Throckmorton, D. C. 2004. Approval letter for Norvasc (amlodipine besylate). NDA 19787. January 8. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/appletter/2004/19787se5-030ltr.pdf (accessed April 3, 2012).

’t Jong, G., A. G. Vulto, M. de Hoog, K. J. M. Schimmel, D. Tibboel, and J. N. van den Anker. 2001. A survey of the use of off-label and unlicensed drugs in a Dutch children’s hospital. Pediatrics 108(5):1089-1093. http://pediatrics.aappublications.org/content/108/5/1089.abstract (accessed April 3, 2012).

Tomlinson, D., C. L. von Baeyer, J. N. Stinson, and L. Sung. 2010. A systematic review of faces scales for the self-report of pain intensity in children. Pediatrics 126(5):e1168-e1198.

Turner, M. A., S. Lewis, D. B. Hawcutt, and D. Field. 2009. Prioritising neonatal medicines research: UK Medicines for Children Research Network scoping survey. BMC Pediatrics 9:50. http://www.biomedcentral.com/content/pdf/1471-2431-9-50.pdf (accessed April 3, 2012).

Ungar, D., S. Joffe, and E. Kodish. 2006. Children are not small adults: Documentation of assent for research involving children. Journal of Pediatrics 149(1 Suppl.):S31-S33.

USAID (U.S. Agency for International Development). 2009. About the neglected tropical disease program. Washington, DC: U.S. Agency for International Development. http://www.neglecteddiseases.gov/about/index.html (accessed April 3, 2012).

Vaidyanathan, J. B. 2008. Clinical review for Zometa (zoledronic acid). NDA 21223. March 5. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/ucm072899.pdf (accessed April 3, 2012).

Vanco, D. J. 2011. Clinical review for Corifact (Factor XIII concentrate (human)). BLA 125385. February 11. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/BiologicsBloodVaccines/BloodBloodProducts/ApprovedProducts/LicensedProductsBLAs/FractionatedPlasmaProducts/UCM247120.pdf (accessed April 3, 2012).

Wade, K. C., D. Wu, D. A. Kaufman, R. M. Ward, D. K. Benjamin, Jr., J. E. Sullivan, N. Ramey, B. Jayaraman, K. Hoppu, P. C. Adamson, M. R. Gastonguay, and J. S. Barret. 2008. Population pharmacokinetics of fluconazole in young infants. Antimicrobial Agents and Chemotherapy 52(11):4043-4049.

Walker, E. P. 2010a. FDA panel grapples with changing study endpoint. Medpage Today. http://www.medpagetoday.com/PublicHealthPolicy/ClinicalTrials/21465 (accessed April 3, 2012).

Walker, S. J. 2010b. Approval letter for Sorilux (calcipotriene). NDA 022563. October 6. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugs atfda_docs/appletter/2010/022563s000ltr.pdf (accessed April 3, 2012).

Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×

Wang, D. 2000. Clinical pharmacology and biopharmaceutics reviews for Lodine XL (etodolac extended release). NDA 20584. August 8. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/nda/2000/20-584S005_Lodine_biopharmr.pdf (accessed April 3, 2012).

Wang, X. 2008. Clinical review for Ventolin HFA (albuterol sulfate HFA inhalation aerosol). NDA 20983. February 22. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/ucm071709.pdf (accessed April 3, 2012).

Ward, R. M., and R. A. Lugo. 2005. Drug therapy in the newborn. In Avery’s neonatology: Pathophysiology and management of the newborn, 6th ed. edited by M. G. MacDonald, M. M. K. Seshia, and M. D. Mullet. Philadelphia, PA: Lippincott Williams & Wilkins. Pp. 1507-1556.

Ward, R. M., W. E. Benitz, J. D. K. Benjamin, L. Blackmon, G. P. Giacoia, M. Hudak, T. Lasky, W. Rodriguez, and A. Selen. 2006. Criteria supporting the study of drugs in the newborn. Clinical Therapeutics 28(9):1385-1398.

Ward, R. M., B. Tammara, S. E. Sullivan, D. L. Stewart, N. Rath, X. Meng, M. K. Maguire, and G. M. Comer. 2010. Single-dose, multiple-dose, and population pharmacokinetics of pantoprazole in neonates and preterm infants with a clinical diagnosis of gastroesophageal reflux disease (GERD). European Journal of Clinical Pharmacology 66(6):555-561.

Wax, P. M. 1995. Elixirs, diluents, and the passage of the 1938 Federal Food, Drug and Cosmetic Act. Annals of Internal Medicine 122(6):456-461. http://www.annals.org/cgi/content/full/122/6/456 (accessed April 3, 2012).

Weiss, C. F., A. J. Glazko, and J. K. Weston. 1960. Chloramphenicol in newborn infant—A physiologic explanation of its toxicity when given in excessive doses. New England Journal of Medicine 262(16):787-794.

Wendler, D., L. Belsky, K. M. Thompson, and E. J. Emanuel. 2005. Quantifying the federal minimal risk standard: Implications for pediatric research without a prospect of direct benefit. Journal of the American Medical Association 294(7):826-832.

WHO (World Health Organization). 2011a. Essential medicines for children. Geneva, Switzerland: World Health Organization. http://www.who.int/childmedicines/getinvolved/en/index.html (accessed April 3, 2012).

WHO. 2011b. WHO model lists of essential medicines. Geneva, Switzerland: World Health Organization. http://www.who.int/medicines/publications/essentialmedicines/en/index.html (accessed April 3, 2012).

Willman, D. 2000. Propulsid: A heartburn drug, now linked to children’s deaths. Los Angeles Times, December 20.

Wilson, J. T. 1999. An update on the therapeutic orphan. Pediatrics 104(3):585-590.

Witten, C. M., and M. A. Malarkey. 2011. Approval letter for Laviv (azficel-T). BL 125348/0. June 21. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/BiologicsBloodVaccines/CellularGeneTherapyProducts/ApprovedProducts/ucm260486.htm (accessed April 3, 2012).

Witter, J. 1999. Clinical review for Lodine XL (etodolac extended released). NDA 20584. December 12. Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/nda/2000/20-584S005_Lodine_medr.pdf (accessed April 3, 2012).

Witzmann, K. 2010. Medical officer review for INOmax (nitric oxide). NDA 20845. November 19. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/UCM245688.pdf (accessed April 3, 2012).

Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×

WMA (World Medical Association). 2008. Declaration of Helsinki: Ethical principles for medical research involving human subjects. http://www.wma.net/en/30publications/10policies/b3/17c.pdf (accessed April 3, 2012).

Working Group (Working Group on Antiretroviral Drug Development in HIV Infected and Exposed Neonates). 2003. Memorandum to the Pediatric Advisory Subcommittee of the Anti-Infective Drugs Advisory Committee. In Background package for March 3, 2003, advisory committee meeting. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/ohrms/dockets/ac/03/briefing/3932B1_01_FDA%20Backgrounder.pdf (accessed April 3, 2012).

Xiao, S. 2009. Clinical review for Benicar (olmesartan medoxomil). NDA 21286. December 4. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/UCM201695.pdf (accessed April 3, 2012).

Yan, S. 2008. Statistical review for Zomig spray (zolmitriptan). NDA 21450. October 14. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/UCM249246.pdf (accessed April 3, 2012).

Yancey, C. L. 2004. Clinical review for Arava (leflunomide). NDA 20905. March 5 (pp. 36-133 of approval package). Silver Spring, MD: Food and Drug Administration. http://www.accessdata.fda.gov/drugsatfda_docs/nda/2004/020905_S012_ARAVA.pdf (accessed April 3, 2012).

Yang, C. P., M. A. Veltri, B. Anton, M. Yaster, and I. D. Berkowitz. 2011. Food and Drug Administration approval for medications used in the pediatric intensive care unit: A continuing conundrum. Pediatric Critical Care Medicine 12(5):e195-e199. http://journals.lww.com/pccmjournal/Abstract/publishahead/Food_and_Drug_Administration_approval_for.99476.aspx (accessed April 3, 2012).

Yeh, T. F., Y. J. Lin, H. C. Lin, C. C. Huang, W. S. Hsieh, C. H. Lin, and C. H. Tsai. 2004. Outcomes at school age after postnatal dexamethasone therapy for lung disease of prematurity. New England Journal of Medicine 350(13):1304-1313.

Zhang, J. 2008. Clinical review for Abilify (aripiprazole). NDA 21436-017, 21713-012, 21729-004, 21866-004. August 14, 2008. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/-DevelopmentResources/ucm071737.pdf (accessed April 3, 2012).

Zuppa, A. F., S. C. Nicolson, J. S. Barrett, and M. R. Gastonguay. 2011. Population pharmacokinetics of pentobarbital in neonates, infants, and children after open heart surgery. Journal of Pediatrics 159(3):414-419.

Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×
Page 231
Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×
Page 232
Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×
Page 233
Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×
Page 234
Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×
Page 235
Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×
Page 236
Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×
Page 237
Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×
Page 238
Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×
Page 239
Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×
Page 240
Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×
Page 241
Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×
Page 242
Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×
Page 243
Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×
Page 244
Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×
Page 245
Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×
Page 246
Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×
Page 247
Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×
Page 248
Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×
Page 249
Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×
Page 250
Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×
Page 251
Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×
Page 252
Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×
Page 253
Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×
Page 254
Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×
Page 255
Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×
Page 256
Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×
Page 257
Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×
Page 258
Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×
Page 259
Suggested Citation:"References." Institute of Medicine. 2012. Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act. Washington, DC: The National Academies Press. doi: 10.17226/13311.
×
Page 260
Next: Appendix A: Study Activities, Methods, and Public Meetings »
Safe and Effective Medicines for Children: Pediatric Studies Conducted Under the Best Pharmaceuticals for Children Act and the Pediatric Research Equity Act Get This Book
×
Buy Paperback | $65.00 Buy Ebook | $54.99
MyNAP members save 10% online.
Login or Register to save!
Download Free PDF

The Best Pharmaceuticals for Children Act (BPCA) and the Pediatric Research Equity Act (PREA) were designed to encourage more pediatric studies of drugs used for children. The FDA asked the IOM to review aspects of pediatric studies and changes in product labeling that resulted from BPCA and PREA and their predecessor policies, as well as assess the incentives for pediatric studies of biologics and the extent to which biologics have been studied in children. The IOM committee concludes that these policies have helped provide clinicians who care for children with better information about the efficacy, safety, and appropriate prescribing of drugs. The IOM suggests that more can be done to increase knowledge about drugs used by children and thereby improve the clinical care, health, and well-being of the nation's children.

  1. ×

    Welcome to OpenBook!

    You're looking at OpenBook, NAP.edu's online reading room since 1999. Based on feedback from you, our users, we've made some improvements that make it easier than ever to read thousands of publications on our website.

    Do you want to take a quick tour of the OpenBook's features?

    No Thanks Take a Tour »
  2. ×

    Show this book's table of contents, where you can jump to any chapter by name.

    « Back Next »
  3. ×

    ...or use these buttons to go back to the previous chapter or skip to the next one.

    « Back Next »
  4. ×

    Jump up to the previous page or down to the next one. Also, you can type in a page number and press Enter to go directly to that page in the book.

    « Back Next »
  5. ×

    Switch between the Original Pages, where you can read the report as it appeared in print, and Text Pages for the web version, where you can highlight and search the text.

    « Back Next »
  6. ×

    To search the entire text of this book, type in your search term here and press Enter.

    « Back Next »
  7. ×

    Share a link to this book page on your preferred social network or via email.

    « Back Next »
  8. ×

    View our suggested citation for this chapter.

    « Back Next »
  9. ×

    Ready to take your reading offline? Click here to buy this book in print or download it as a free PDF, if available.

    « Back Next »
Stay Connected!