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- A S Kesselheim.
- Division of Pharmacoepidemiology and Pharmacoeconomics, Department of Medicine, Brigham and Women's Hospital and Harvard Medical School, Boston, Massachusetts, USA. akesselheim@partners.org
- Clin. Pharmacol. Ther. 2012 Aug 1; 92 (2): 153-5.
AbstractThe Orphan Drug Act seeks to meet a utilitarian goal of advancing therapeutic options for patients with rare diseases. However, data show that orphan drugs are often approved with more limited premarket testing than that carried out for nonorphan drugs and consequently expose patients to more risk and less certain efficacy. Therefore, the ethical principles of justice and beneficence may require attention to informed consent among patients receiving the drugs and greater investment in postmarket surveillance and confirmational testing.
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