This review was conducted of older data - FDA has seemingly become more flexible in approving orphan drugs in the last several years. It is possible that the data we have after the extension trial will be sufficient for concurrent approval for AD and HD as Jaylucks from YMB suggests.
An analysis of medical officer reviews for drugs approved from 2007 to 2009 was conducted. (Before 2007, these reviews were not consistently public.) For the 44 drugs approved during this period, the author located full medical officer reviews for 30. (The remaining 14 drugs included 9 clotting factors or immune globulins, 4 previously approved drugs, and 1 other product.) For the 30 drugs collectively, the medical reviews reported a total of 71 trials evaluating efficacy. The trials enrolled a median of 179 participants, and treatment lasted a median of 8.5 weeks. Of the 71 trials, 55 were considered pivotal trials that provided key evidence of efficacy. They included 30 phase III studies, 17 phase II studies, 1 phase I study, and 4 phase IV studies (which were conducted following FDA approval of the drug for a different indication).
In this sample, 13 of the 30 orphan drugs were approved based on a single efficacy trial, including 8 based on a single phase III trial; 4 based on a single phase II trial; and 1 based on a single phase I trial. Among the 55 pivotal trials, 27 had a double-blind design, 5 were single-blind, and the remaining 23 did not have blinding. Twenty-six trials used placebo controls, and 11 used active comparators. Thirty-eight studies were randomized. Thirteen were single-arm studies.
Some data point to differences in the evidence supporting approvals of orphan compared to nonorphan drugs. An analysis of accelerated approvals for NMEs in oncology found that 73 percent of those approved from 1995 through 2008 for nonorphan indications were supported by phase III studies compared to 45 percent of Regulatory Framework for Drugs for Rare Diseases - Rare Diseases and NMEs approved for orphan indications (Richey et al., 2009). The authors also found that the orphan NMEs were more likely than the regular NMEs to have difficulty completing the follow-up confirmatory studies. Another study by Mitsumoto and colleagues (2009) compared approvals for neurological drugs and found that of 20 recently approved nonorphan drugs, all had at least two randomized, placebo-controlled, double-blind clinical studies compared to 32 percent of the 19 approved orphan drugs. The mean number of trial participants in the former was 506 compared to 164 for the latter.
http://www.ncbi.nlm.nih.gov/books/NBK56185/
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Orphan Drug Approval Analysis 2007-2009
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