Gnanasakthy A, Mordin M, Haydysch E, DeMuro C. FDA patient-reported outcome labeling of novel therapies (2011-2015). Poster presented at the 2016 ISPOR 21st Annual International Meeting; May 25, 2016. Washington, DC. [abstract] Value Health. 2016 May; 19(3):A285-6.

OBJECTIVES: For the period covering 2006 and 2010, 24.1% of newly approved drugs by the FDA had at least one PRO related labeling (Gnanasakthy et. al. (2012)). This study aimed to assess PRO related labeling of new products covering the period between 2011 and 2015 to examine differences compared to the previous assessment.

METHODS: Using the Drugs@FDA database, new drugs approved by FDA between January 2011 and December 2015 were identified. For all identified drug products, medical review sections from publicly available drug approval packages (DAPs) were reviewed. Product labeling (indication, clinical trials sections) were reviewed to determine PRO related labeling and the corresponding endpoint status.

RESULTS: Of the 182 new drugs identified, 30 (16.5%) were granted PRO claims. The vast majority of labeling related to primary endpoints (76.7%), and were based on simple event diaries (e.g. seizures) or measures developed prior to the release of the FDA PRO guidance. Importantly, 27.5% of all approved drugs were for an oncology-related condition (versus 15.5% between 2006 and 2010). The proportion of claims granted is lower than what was reported between 2006 and 2010 (24.1%), and prior to the release of the PRO guidance (30%, 1997-2002).

CONCLUSIONS: PRO claims continue to be approved by FDA, although at a lower rate than previously reported. This may be partly explained by the increase in the number of oncology products with study designs that may not support PRO labeling as per regulatory guidance. The lack of PRO labeling based on secondary endpoints only may be a reflection of sponsors’ reluctance to capture PRO related value messages to regulatory standards when PROs are not required to support the indication. The acceptance of PRO measures developed prior to the release of the PRO guidance for the purpose of labeling reflects 'regulatory flexibility' adopted by the FDA reviewers.

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