Clinical pharmacology and therapeutics
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The 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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Clin. Pharmacol. Ther. · Aug 2012
Raising orphans: how clinical development programs of drugs for rare and common diseases are different.
We compared clinical trials described in package inserts from noncancer orphan and nonorphan drugs from 1 January 2001 to 31 December 2011. Among the 37 orphan and 58 nonorphan drugs approved by the US Food and Drug Administration (US FDA) during this period, orphans had fewer clinical trials (2.8 vs. 3.5, P < 0.05) and fewer total participants (390 vs. 2,566, P < 0.001), but proportions with randomization, blinding, and placebo-controlled clinical end points were similar, as were development times. We conclude that small studies of appropriate design can support US FDA approval of new medicines for rare diseases.