PNS243 REVIEW OF PATIENT REPORTED OUTCOME (PRO) MEASURES FOR NOVEL DRUG APPROVALS (NDA) IN THE UNITED STATES: 2016-2018

PRO data is being used to support labeling claims of products’ effects on patients’ functional status, experience of care, symptoms and symptom burden, and health related quality of life. This study reviewed PRO information reported in labels for NDAs approved from 2016 to 2018 and compared findings to previously published results from 2006 to 2015. A review of FDA NDAs labeling was conducted using the FDA website to determine the number of NDAs between January 1, 2016 to December 31, 2018. Three investigators independently reviewed the NDAs and collectively analyzed results. For all NDAs, the prescribing information labels were reviewed to identify the usage of PRO measures in clinical studies and approval by the FDA. The NDAs were grouped according to ICD-10 disease categories and PRO measure types. The ICD-10 categories were further divided into PRO-dependent categories that rely on PROs as primary or secondary endpoints for evaluation of treatment benefit, and non-PRO dependent categories that rely on survival, biomarkers, or clinical outcome assessments other than PROs. Findings were compared to those of NDAs approved in 2006 to 2015. From 2016 to 2018, 25.20% of 127 total NDAs approved had PRO measures included in the labels, compared with 19.46% of 298 total NDAs approved between 2006 and 2015. 46.46% of 99 NDAs from 2006 to 2015 and 47.46% of 49 approved NDAs in PRO-dependent disease categories from 2016 to 2018 received PRO labeling approval. 6.03% of 199 NDAs from 2006 to 2015 compared to 5.88% of 56 NDAs approved in non-PRO-dependent disease categories from 2016 to 2018 received PRO labeling approval. This study showed an increasing trend in overall PRO measure usage in FDA NDA labels between 2006 to 2015 and 2016 to 2018, especially within PRO-dependent disease categories.