OBJECTIVES: Previous reviews of patient-reported outcome (PRO) label claims did not distinguish products with orphan designation. Prior analyses suggest less evidence is required for orphan drug approvals compared to non-orphan drugs. This study aimed to identify orphan drug products approved by the US Food and Drug Administration (FDA) between 2006 and 2013, prevalence of PRO label claims for these products, and evidence supporting PRO claim approval.
METHODS: Using the Drugs@FDA database, new molecular entities and biologic licensed agents approved between January 2006 and September 2013 with orphan review classification were identified, with subsequent label review. For products with PRO label claims, medical and summary review sections from FDA drug approval packages were reviewed to identify reviewing division, indication, review designation (priority, standard), PRO endpoint status, PRO measure type, number of phase 3 trials supporting claims, and PRO-related SEALD comments. Descriptive data were recorded in Microsoft Excel; frequency of measured characteristics was analyzed.
RESULTS: Of 43 orphan products identified, 5 (12%) included PRO label claims. Five orphan products achieved 7 PRO claims. Priority and standard review prevalence were similar (44% vs. 40%, respectively) for orphan products; the majority (4 of 5) of products with PRO claims were priority review. A slight majority (60%; 3 of 5 orphan products) included PRO claims supported by = 1 phase 3 trial. Signs/symptoms measures and secondary PRO endpoints were most common (57% each). FDA reviewing division varied. One product received PRO-related SEALD comments.
CONCLUSIONS: For the time period evaluated, orphan products rarely included a PRO label claim. These claims may not be needed for orphan product differentiation to the degree that they are for non-orphan products. PRO claims achieved for orphan products appear to require less supporting evidence for approval than non-orphan products, consistent with orphan drug approval expectations but in contrast to FDA PRO guidance criteria.