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Rare Diseases and Orphan Products: Accelerating Research and Development (2011)
Board on Health Sciences Policy (HSP)

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Rare Diseases and Orphan Products: Accelerating Research and Development

AdvaMed. 2009. Statement Presented by Susan Alpert to IOM Committee on Accelerating Rare Diseases Research and Orphan Product Development, Washington, DC. November 23.

Aetna. 2010. Clinical Policy Bulletin: Heart Transplantation. http://www.aetna.com/cpb/medical/data/500_599/0586.html (accessed August 10, 2010).

AIMBE (American Institute for Medical and Biological Engineering). 2005. AIMBE Hall of Fame salutes PET as one of 24 achievements in medical, biological engineering. Molecular Imaging News, March 10.

Aiuti, A., I. Brigida, F. Ferrua, B. Cappelli, R. Chiesa, S. Marktel, and M. Roncarolo. 2009. Hematopoietic stem cell gene therapy for adenosine deaminase deficient-SCID. Immunologic Research 44(1-3):150-159.

Alliance for a Stronger FDA. 2009. Written Statement of the Alliance for a Stronger FDA, U.S. Food and Drug Administration Regarding FY2011 Appropriations for the U.S. Food and Drug Administration Submitted to the Appropriations Subcommittee on Agriculture, Rural Development, Food and Drug Administration, and Related Agencies, U.S. Senate, March 26, 2010. http://fdaalliance.files.wordpress.com/2009/11/alliance-statement-senate-appropriations-committee-fy-11.doc (accessed August 10, 2010).

Anand, G. 2005. How drugs for rare diseases became lifeline for companies. Wall Street Journal, November 15. http://online.wsj.com/article/SB113202332063297223.html (accessed August 10, 2010).

Anderson, T. 2009. Novartis under fire for accepting new reward for old drug. Lancet 373(9673):1414. http://www.thelancet.com/journals/lancet/article/PIIS0140-6736(09)60804-7/fulltext (accessed August 24, 2010).

Anglim, P. P., T. A. Alonzo, and I. A. Laird-Offringa. 2008. DNA methylation-based biomarkers for early detection of non-small cell lung cancer: an update. Molecular Cancer 7(81).

Ariyanchira, S. 2008. BioMarket trends: orphan drug arena driven by biologics. Genetic Engineering and Biotechnology News 21(1). http://www.genengnews.com/gen-articles/biomarket-trends-orphan-drug-arena-driven-by-biologics/2318/ (accessed August 10, 2010).

Arnon, S. S. 2007. Creation and development of the public service orphan drug human botulism immune globulin. Pediatrics 119(4):785-789. http://www.cdph.ca.gov/programs/ibtpp/Documents/Peds-Creatn-Devlpmt-BIG-IV-2007.pdf (accessed August 10, 2010).

Asamoah, A. K., and J. M. Sharfstein. 2010. Transparency at the Food and Drug Administration. New England Journal of Medicine 362:25. http://www.nejm.org/doi/pdf/10.1056/NEJMp1005202 (accessed August 20, 2010).

Asbury, C. H. 1985. Orphan Drugs, Medical Versus Market Value. Lexington, MA: Lexington Books.

Asbury, C. H. 1991. The Orphan Drug Act: the first 7 years. Journal of the American Medical Association 265(7):893-897.

ASH (American Society of Hematology). 2007. Toward a New Research Paradigm: Building a New Sickle Cell Disease Research Agenda. http://www.hematology.org/Advocacy/Policy-Statements/2680.aspx (accessed August 10, 2010).

Ashlock, M. 2010. Increasing the therapeutic options for individuals with rare diseases. Presentation to IOM Committee on Accelerating Rare Diseases Research and Orphan Product Development, Washington, DC. February 23.

ASHP (American Society of Health-System Pharmacists). 2010. Manufacturer PAPs. http://www.ashp.org/Import/PRACTICEANDPOLICY/PracticeResourceCenters/PatientAssistancePrograms/ManufacturerPAPs.aspx (accessed August 10, 2010).

Atkins, D., J. Siegel, and J. Slutsky. 2005. Making policy when the evidence is in dispute. Health Affairs 24(1):102-113.

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