Gnanasakthy A, Mordin M, Evans E, Doward L, DeMuro C. A review of patient-reported outcome labeling in the United States (2011-2015). Value Health. 2017;20(3):420-9. doi: http://dx.doi.org/10.1016/j.jval.2016.10.006.


OBJECTIVE: A review of new drug approvals (NDAs) by the Food and Drug Administration (FDA) for 2006-2010 showed that 24.1% of new drugs had patient-reported outcome (PRO)-related labeling. The purpose of this study is to review PRO-related labeling for NDAs for 2011-2015 and to compare key findings reported previously.

METHODS: A review of the FDA drug approval reports for NDAs was conducted using the webpage of the FDA Drug Approval reports to determine the number of NDAs for 2011-2015. For all identified NDAs, drug approval package and product labels were reviewed to identify PRO endpoint status and PRO labeling. NDAs for 2006-2015 were grouped by disease category as per ICD-10 classification. Data were summarized for all NDAs and for approvals in diseases that traditionally rely on PROs for evaluating treatment benefit (PRO-dependent). Results were compared to NDAs for 2006-2010.

RESULTS: In 2011-2015, 16.5% of the 182 NDAs had PRO labeling. For PRO-dependent NDAs, this figure was 46.9% and 46.0% for 2006-1010 and 2011-2015, respectively. The majority of PRO labeling for 2011-2015 was based on primary endpoints (76.7%)s. Almost all PRO labeling was for concepts proximal to the disease.

CONCLUSION: There is potential for increased PRO labeling, especially for drug approvals in diseases that traditionally rely on PROs for evaluating treatment benefit to satisfy regulatory needs. Less PRO labeling based on secondary endpoints may be indicative of drug manufacturers’ reluctance to aid and enhance the value propositions of their products to all stakeholders, including patients.

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