Introduction to a Supplement on Innovative Approaches to Studying Health Outcomes in Rare Diseases

Author:

Kesselheim Aaron S.,Gagne Joshua J.

Publisher

Springer Science and Business Media LLC

Subject

Internal Medicine

Reference8 articles.

1. Coté T, Kelkar A, Xu K, Braun MM, Phillips MI. Orphan products: an emerging trend in drug approvals. Nat Rev Drug Discov. 2010;9(1):84.

2. NIDDK. National diabetes statistics. 2011. Available at: http://diabetes.niddk.nih.gov/dm/pubs/statistics/ .

3. Kesselheim AS. Innovation and the Orphan Drug Act, 1983–2009: the regulatory and clinical characteristics of approved orphan drugs. In: Field MJ, Boat TF, eds. Accelerating Rare Diseases Research and Orphan Product Development. Washington, DC: National Academy Press; 2010.

4. Joppi R, Bertele’ V, Garattini S. Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU. Eur J Clin Pharmacol. 2013;69(4):1009–24.

5. Kesselheim AS. An empirical review of major legislation affecting drug development: past experiences, effects, and unintended consequences. Milbank Q. 2011;89(3):450–502.

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