Abstract
In the US marketplace, regulations have been introduced that incentivize manufacturers to invest in drugs for orphan conditions. An orphan designation can lead to manufacturer benefits such as extended exclusivity as well as faster market approval. The objective of this research was to compare clinical trial attributes used in pivotal studies for both orphan and non-orphan oncology drugs. We identified all new oncology approvals between August 2011 and September 2015. We excluded indication expansions. We then collected detailed data on pivotal clinical trials used to gain approval such as study design, number of trials at approval for each indication, comparator type, sample size, trial phase, and major efficacy endpoints. We stratified data based on orphan designation and compared the aforementioned variables. 26 trials for orphan drugs and 12 trials for non-orphan drugs were approved between August 2011 and September 2015. Pivotal trials of orphan drugs had smaller participant numbers (median, 250 [interquartile range {IQR}, 185-392] vs. 288 [IQR, 157-802]) and orphan trials were less likely to be randomized (50% vs. 67%). Orphan and non-orphan pivotal trials varied in their blinding with orphan having a smaller proportion of blinded trials (17% vs. 33%). Primary study outcomes varied between the two orphan designations, with overall survival only occurring as a primary endpoint in non-orphan drug trials, while orphan trials were more likely to assess disease response (63% vs. 43%). There was variation in quality between pivotal trials for orphan and non-orphan oncology drugs. While there were limited differences in clinical trial attributes such as trial size and trial design aspects (e.g. randomization and blinding) noticeable differences were observed in clinical trial phases and primary endpoints between orphan and non-orphan oncology products.
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