OBJECTIVES: Wilke and colleagues (2004) previously conducted a review of effectiveness endpoints reported in the labels of new drug products approved in the United States (US) between 1997-2002 to determine the extent and type of PRO endpoints utilized. They reported that 30% of product labels reviewed included PROs. Our study aimed to build upon this work by describing the current state of PRO label claims granted for new molecular entities (NMEs) and biologic license applications (BLAs) following release of the draft and final FDA PRO Guidance documents (i.e., since February 2006).
METHODS: Using the FDA Drug Approval Reports webpage, all FDA approved NMEs and BLAs between February 2006 and December 2010 were identified. Generic products with tentative approvals granted in this period were excluded. For all identified drug products, medical review sections from publicly available summary basis of approvals (SBAs) were reviewed to identify PRO endpoint status. Product labels (indication, clinical trials sections) were reviewed to determine the number and type of PRO claims.
RESULTS: Of the 116 NMEs/BLAs identified, 28 (24.1%) were granted PRO claims. The majority (n24) were for signs and symptoms. Nine of the signs and symptom claims were painrelated. Of the 28 products with PRO claims, a PRO was a primary endpoint for 20 (71%). All 20 of these primary endpoints were symptom-related and the majority (12 of 20) were collected via diary.
CONCLUSIONS: PRO claims continue to be approved by FDA, with 24% of NMEs and BLAs granted PRO claims. Successful PRO label claims over the past five years have been largely in support of treatment benefit for symptoms specified as primary endpoints. The proportion of NMEs with PRO label claims during the post-guidance period (24.1%) was lower than that of the pre-guidance period (30%).
