Online Program

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Thursday, September 13
Thu, Sep 13, 4:15 PM - 5:30 PM
Thurgood Marshall South
Developing a PRO for Use as a Phase III Endpoint for Regulatory Approval and Marketing: Implementation of the 2009 FDA Final Guidance for Industry

A Review of Patient-Reported Outcome Labeling in the United States (2011--2015) (300729)

*Joseph C Cappelleri, Pfizer 

Keywords: drug labeling, FDA, patient-reported outcome, PRO labeling

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

A review of FDA drug approval reports for NDAs was conducted using the FDA website to determine the number of NDAs for the period 2011-2015. For all identified NDAs, drug approval package and product labeling were reviewed to identify PRO endpoint status and PRO labeling. Data were summarized for all NDAs and for approvals in diseases that traditionally rely on PROs for evaluating treatment benefit (PRO-dependent).

In the period 2011-2015, 16.5% of the 182 NDAs had PRO labeling. For PRO-dependent NDAs, this figure was 46.9% and 46.0% for the periods 2006-2010 and 2011-2015, respectively. Most of the PRO labeling for the period 2011-2015 was based on primary endpoints (76.7%). Almost all PRO labeling was for concepts proximal to the disease.

There is potential for increased PRO labeling, especially for drug approvals that traditionally rely on PRO measures for evaluating treatment benefit. Less PRO labeling based on secondary endpoints may be indicative of drug manufacturers’ reluctance to invest in and expand upon secondary measures that may enhance the value proposition for a regulatory claim. Source: Gnanasakthy et al. Value in Health 2017; 20:420-429.