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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 dis-
tributions 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_BIO
PHARMR.PDF (accessed March 23, 2012).
Adebowale, A. 2002. Clinical pharmacology and biopharmaceutics review(s) for children’s Ad-
vil 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).
231
OCR for page 232
232 SAFE AND EFFECTIVE MEDICINES FOR CHILDREN
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 Publi-
cations 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/Development
ApprovalProcess/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. Analge-
sia 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 botu-
lism 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 inci-
dent. FDA Consumer, June. http://www.fda.gov/AboutFDA/WhatWeDo/History/Product
Regulation/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 Author-
ity, 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/CommitteesMeeting
Materials/PediatricAdvisoryCommittee/UCM214708.pdf (accessed March 23, 2012).
OCR for page 233
233
REFERENCES
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 Ophthal-
mology 126(8):1152-1156.
OCR for page 234
234 SAFE AND EFFECTIVE MEDICINES FOR CHILDREN
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 ex-
clusivity. 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. Washing-
ton, DC: Biotechnology Industry Organization. http://www.bio.org/node/178 (accessed
March 23, 2012).
Biomarkers Definitions Working Group. 2001. Biomarkers and surrogate endpoints: Pre-
ferred 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.
OCR for page 235
235
REFERENCES
BMS (Bristol-Myers Squibb). 2010. Prescribing information for BuSpar (buspirone HCl).
Princeton, NJ: Bristol-Myers Squibb. http://packageinserts.bms.com/pi/pi_buspar.pdf (ac-
cessed 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 in-
formation for Plavix (clopidogrel bisulfate). Bridgewater, NJ: Bristol-Myers Squibb/
Sanofi Pharmaceuticals Partnership. http://products.sanofi.us/PLAVIX/plavix.pdf (ac -
cessed 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. D eliverable 6: Final report on the PMR/PMC backlog
review. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/
downloads/Drugs/GuidanceComplianceRegulatoryInformation/Post-marketingPhaseIV
Commitments/UCM232880.pdf (accessed March 23, 2012).
Boxwell, D. 2011. Pediatric Advisory Committee memo regarding Kaletra (lopinavir/ritona-
vir). NDA 21251. January 24. Silver Spring, MD: Food and Drug Administration. http://
www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/Pediatric
AdvisoryCommittee/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/LicensedProducts
BLAs/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/Cardiovascular
andRenalDrugsAdvisoryCommittee/UCM221824.pdf (accessed March 23, 2012).
Brion, L. P., E. F. Bell, and T. S. Raghuveer. 2003. Vitamin E supplementation for preven-
tion 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 pre-
mature 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).
OCR for page 236
236 SAFE AND EFFECTIVE MEDICINES FOR CHILDREN
Cadence Pharmaceuticals. 2010. Prescribing information for Ofirmev (acetaminophen). San
Diego, CA: Cadence Pharmaceuticals. http://www.ofirmev.com/pdf/OFIRMEVPrescribing
Information.pdf (accessed March 23, 2012).
Caldwell, P. H. Y., P. N. Butow, and J. C. Craig. 2003. Parents’ attitudes to children’s participa-
tion 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%20
U nofficial%20Legislative%20History%20of%20the%20Biologics%20Price%20
Competition%20and%20Innovation%20Act%20of%202.pdf (accessed March 23,
2012).
Castile, R. 2004. Novel techniques for assessing infant and pediatric lung function and struc-
ture. 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/BloodBlood
P roducts/ApprovedProducts/LicensedProductsBLAs/FractionatedPlasmaProducts/
UCM119473.pdf (accessed March 23, 2012).
CDER (Center for Drug Evaluation and Research). 2010. Manual of policies and proce-
dures (MAPP): Annotated clinical review template. MAPP 6010.3 Rev. 1. Silver Spring,
MD: Food and Drug Administration. http://www.fda.gov/downloads/AboutFDA/Centers
Offices/CDER/ManualofPoliciesProcedures/UCM236903.pdf (accessed March 23, 2012).
CDER. 2011a. Desk reference guide: New drug application and biologic license application re-
views. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads
/AboutFDA/CentersOffices/CDER/ManualofPoliciesProcedures/UCM218757.pdf (ac-
cessed 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 Administra-
tion. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/Development
Resources/UCM207644.pdf (accessed March 23, 2012).
CDER/CBER. 1998a. Guidance for industry: General considerations for pediatric pharma-
cokinetic studies for drugs and biological products. Silver Spring, MD: Food and Drug
Administration. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatory
Information/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 Administra-
tion. 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/Guidance
ComplianceRegulatoryInformation/Guidances/ucm072137.pdf (accessed March 23,
2012).
OCR for page 237
237
REFERENCES
CDER/CBER. 1999b. Guidance for industry: Qualifying for pediatric exclusivity under Sec-
tion 505A of the federal Food, Drug, and Cosmetic Act. Silver Spring, MD: Food
and Drug Administration. http://www.fda.gov/downloads/Drugs/DevelopmentApproval
Process/DevelopmentResources/UCM049924.pdf (accessed March 23, 2012).
CDER/CBER. 2000. Guidance for industry: Pediatric oncology studies in response to a writ-
ten 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 hu-
man prescription drug and biological products—Content and format. Silver Spring,
MD: Food and Drug Administration. http://www.fda.gov/downloads/Drugs/Guidance
ComplianceRegulatoryInformation/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 (ac-
cessed March 23, 2012).
CDER/CBER. 2010a. Guidance for industry: Adaptive design clinical trials for drugs and bio-
logics. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/downloads
/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm201790.pdf (ac-
cessed 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/DeviceRegulation
andGuidance/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).
OCR for page 238
238 SAFE AND EFFECTIVE MEDICINES FOR CHILDREN
Chambers, W. A. 2010. Approval letter for Moxeza (moxifloxacin hydrochloride ophthalmic
solution). NDA 22428. November 19. Silver Spring, MD: Food and Drug Administra-
tion. 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 Administra-
tion. 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 (ac-
cessed 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 (ac-
cessed March 23, 2012).
Coffey, M. J., B. Wilfond, and L. Friedman Ross. 2004. Ethical assessment of clinical asthma tri-
als 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 pe-
diatrics. 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 neo-
nate. 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).
OCR for page 239
239
REFERENCES
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 Over-
sight Committee). 2011. Schedule of Clinical and Translational Science Awards and Pedi-
atric 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 intrave-
nous 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. Assess-
ing 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).
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/Pediatric
BPCAPREA/FDA%20Revised%20IOM%20presentation%2001072011final.pdf (ac-
cessed March 23, 2012).
OCR for page 240
240 SAFE AND EFFECTIVE MEDICINES FOR CHILDREN
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 popula-
tion 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/Fraction-
atedPlasmaProducts/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.
h ttp://www.fda.gov/ohrms/dockets/ac/03/briefing/3927B1_06_GFI-Complying%20
with%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/drugsat
fda_docs/nda/2001/19-865S010_Betapace_prntlbl.pdf (accessed March 23, 2012).
OCR for page 241
241
REFERENCES
FDA. 2001b. The pediatric exclusivity provision: Status report to Congress. Silver Spring,
MD: Food and Drug Administration. http://www.fda.gov/downloads/drugs/development
approvalprocess/developmentresources/ucm049915.pdf (accessed March 23, 2012).
FDA. 2002. Science and the regulation of biological products: From a rich history to a chal-
lenging future. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/
downloads/aboutfda/whatwedo/history/productregulation/100yearsofbiologicsregulation
/ucm070313.pdf (accessed March 23, 2012).
FDA. 2006a. Guidance for FDA staff and industry: Marketed unapproved drugs—Compliance
policy guide. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/
downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM070290.
pdf (accessed March 23, 2012).
FDA. 2006b. Guidance for industry: Labeling for human prescription drug and biological
products—Implementing the new content and format requirements. Silver Spring, MD: Food
and Drug Administration. http://www.fda.gov/downloads/Drugs/GuidanceCompliance
RegulatoryInformation/Guidances/ucm075082.pdf (accessed March 23, 2012).
FDA. 2006c. Process for handling referrals to FDA under 21 CFR 50.54—Additional
safeguards for children in clinical investigations. Silver Spring, MD: Food and Drug
Administration. http://www.fda.gov/RegulatoryInformation/Guidances/ucm127541.htm
(accessed March 23, 2012).
FDA. 2008. Update of safety review: Follow-up to the March 27, 2008 communication about
the ongoing safety review of Regranex (becaplermin). June 6. Silver Spring, MD: Food
and Drug Administration. http://www.fda.gov/Drugs/DrugSafety/PostmarketDrugSafety
InformationforPatientsandProviders/DrugSafetyInformationforHeathcareProfessionals/
ucm072148.htm (accessed March 23, 2012).
FDA. 2009a. FDA’s HSP/BIMO initiative accomplishments—Update. Silver Spring, MD: Food
and Drug Administration. http://www.fda.gov/ScienceResearch/SpecialTopics/Running
ClinicalTrials/ucm134452.htm (accessed March 23, 2012).
FDA. 2009b. Follow-up to the June 4, 2008 early communication about the ongoing safety re-
view of tumor necrosis factor blockers (marketed as Remicade, Enbrel, Humira, Cimzia,
and Simponi). August 4. Silver Spring, MD: Food and Drug Administration. http://www.
fda.gov/Drugs/DrugSafety/PostmarketDrugSafetyInformationforPatientsandProviders
/DrugSafetyInformationforHeathcareProfessionals/ucm174449.htm (accessed March 23,
2012).
FDA. 2009c. IOM recommendations: FDA actions update. Silver Spring, MD: Food and
Drug Administration. http://www.fda.gov/Safety/SafetyofSpecificProducts/ucm184598.
htm (accessed March 23, 2012).
FDA. 2009d. Press announcement: FDA gives update on botulinum toxin safety warnings;
established names of drugs changed. Silver Spring, MD: Food and Drug Administration.
http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/2009/ucm175013.htm
(accessed March 23, 2012).
FDA. 2010a. Drug development and review definitions. Silver Spring, MD: Food and Drug
Administration. http://www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsare
DevelopedandApproved/ApprovalApplications/InvestigationalNewDrugINDApplication/
ucm176522.htm (accessed March 23, 2012).
FDA. 2010b. Information sheet guidance for sponsors, clinical investigators, and IRBs:
Frequently asked questions—Statement of investigator. Silver Spring, MD: Food and
Drug Administration. http://www.fda.gov/downloads/RegulatoryInformation/Guidances/
UCM214282.pdf (accessed March 23, 2012).
OCR for page 250
250 SAFE AND EFFECTIVE MEDICINES FOR CHILDREN
Metz, B. J., M. C. Rubenstein, M. L. Levy, and D. W. Metry. 2004. Response of ulcerated peri-
neal 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 de-
velopment. 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 pharmacoge-
nomic 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 Commit-
tee April 27. Memorandum from Dianne Murphy to members of the Pediatric Advisory
Committee and invited expert consultants. Silver Spring, MD: Food and Drug Administra-
tion. 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).
OCR for page 251
251
REFERENCES
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 Thera-
peutics 30(11):2112-2119.
NAS (National Academy of Sciences). Undated. The drug efficacy study of the National Re-
search Council’s Division of Medical Sciences, 1966-1969. Washington, DC: National
Academy of Sciences. http://www7.nationalacademies.org/archives/drugefficacy.html (ac-
cessed April 3, 2012).
National Commission (National Commission for the Protection of Human Subjects of Bio-
medical and Behavioral Research). 1977. Report and recommendations: Research involv-
ing children. Washington, DC: U.S. Government Printing Office.
NBAC (National Bioethics Advisory Commission). 2001. Ethical and policy issues in interna-
tional 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/Advisory
Committees/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%20
02022011Final.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 pharma-
cogenomics. 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).
OCR for page 252
252 SAFE AND EFFECTIVE MEDICINES FOR CHILDREN
NHLBI (National Heart, Lung, and Blood Institute). 1991. Guidelines for the diagnosis and
management of asthma. Expert panel report. Bethesda, MD: National Asthma Educa-
tion 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 (ac-
cessed 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 Formu-
lation 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 prioriti-
zation, 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 work-
ing 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 pe-
diatric 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 initia-
tive. Workshop summary. Bethesda, MD: National Institute of Child Health and Hu-
man 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 (ac-
cessed April 3, 2012).
OCR for page 253
253
REFERENCES
Nishi, Y. 2010. Hepatoblastoma concerns and growth hormone therapy in small for gesta-
tional 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.aap
publications.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 sub-
jects. 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/region
10/11000514.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 ob-
served: 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 Commit-
tee meeting, December 8. Silver Spring, MD: Food and Drug Administration. http://
www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/Pediatric
AdvisoryCommittee/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.
OCR for page 254
254 SAFE AND EFFECTIVE MEDICINES FOR CHILDREN
PAS/AIDAC (Pediatric Advisory Subcommittee of the Anti-Infective Drugs Advisory Com-
mittee). 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. Globaliza-
tion 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 Administra-
tion. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/Development
Resources/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/Development
ApprovalProcess/DevelopmentResources/UCM222175.pdf (accessed April 3, 2012).
Protherics, Inc. 2010. Prescribing information for CroFab (crotalidae polyvalent im-
mune 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 (ac-
cessed 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 applica-
tions, edited by A. E. Mulberg, S. A. Silber, and J. N. van den Anker. Hoboken, NJ: John
Wiley & Sons, Inc. Pp. 231-242.
OCR for page 255
255
REFERENCES
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/drugs
atfda_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/drugs
atfda_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/drugs
atfda_docs/appletter/2011/022544s000ltr.pdf (accessed April 3, 2012).
Rappaport, B. A. 2011c. Overview of the March 10, 2011, ALSDAC meeting to discuss the neu-
rotoxicity of anesthetic and sedative drugs in juvenile animals memo. Silver Spring, MD:
Food and Drug Administration. http://www.fda.gov/downloads/AdvisoryCommittees/
C ommitteesMeetingMaterials/Drugs/AnestheticAndLifeSupportDrugsAdvisory
Committee/UCM245769.pdf (accessed April 3, 2012).
Rappaport, B., R. D. Mellon, A. Simone, and J. Woodcock. 2011. Defining safe use of anes-
thesia 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: Im-
proving 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 (ac-
cessed 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/drugs
atfda_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.
OCR for page 256
256 SAFE AND EFFECTIVE MEDICINES FOR CHILDREN
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/drugs
atfda_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 consid-
erations 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/Biologics
BloodVaccines/Vaccines/ApprovedProducts/UCM250619.pdf (accessed April 3, 2012).
OCR for page 257
257
REFERENCES
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 Medi-
cine 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 Administra-
tion. http://www.fda.gov/downloads/Drugs/DevelopmentApprovalProcess/Development
Resources/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. Decem-
ber 11. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/Biologics
BloodVaccines/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).
OCR for page 258
258 SAFE AND EFFECTIVE MEDICINES FOR CHILDREN
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. Ra-
mey, 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).
OCR for page 259
259
REFERENCES
Wang, D. 2000. Clinical pharmacology and biopharmaceutics reviews for Lodine XL (etodolac
extended release). NDA 20584. August 8. Silver Spring, MD: Food and Drug Adminis-
tration. 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 gastroesopha-
geal 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/Biologics
BloodVaccines/CellularGeneTherapyProducts/ApprovedProducts/ucm260486.htm (ac-
cessed 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.access
data.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. No-
vember 19. Silver Spring, MD: Food and Drug Administration. http://www.fda.gov/
downloads/Drugs/DevelopmentApprovalProcess/DevelopmentResources/UCM245688.
pdf (accessed April 3, 2012).
OCR for page 260
260 SAFE AND EFFECTIVE MEDICINES FOR CHILDREN
WMA (World Medical Association). 2008. Declaration of Helsinki: Ethical principles for medi-
cal 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 (ac-
cessed 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 con-
tinuing 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 pre-
maturity. New England Journal of Medicine 350(13):1304-1313.
Zhang, J. 2008. Clinical review for Abilify (aripiprazole). NDA 21436-017, 21713-012,
2 1729-004, 21866-004. August 14, 2008. Silver Spring, MD: Food and Drug Ad-
ministration. 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 pharma-
cokinetics of pentobarbital in neonates, infants, and children after open heart surgery.
Journal of Pediatrics 159(3):414-419.
