OBJECTIVES: Patient reported outcome (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 (HRQoL). This study reviewed PRO information reported in labels for novel drug approvals (NDAs) approved from 2016 to 2021 and compared findings to previously published results from 2006 to 2015.

METHODS: 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, 2021. Two investigators independently reviewed the NDAs. For all NDAs, the prescribing information labels were reviewed to identify the usage of PRO measures in clinical studies and approval by the FDA. Drugs were classified as PRO dependent or non-PRO dependent using the ICD-10 code. The PRO outcomes assessed were characterized as HRQoL, functional status, symptoms, health behaviors, and patient experience with care. Findings were compared to those of NDAs approved in 2006 to 2015.

RESULTS: From 2016 to 2021, 19.30% of 202 total NDAs approved had PRO measures included in the labels, compared with 19.46% of 298 total NDAs approved between 2006-2015. 38% of 80 approved NDAs in PRO-dependent disease categories from 2016-to-2021 received PRO labeling approval compared to 46.46% from 2006 to 2015. Also, 7.37% of 122 NDAs approved in non-PRO-dependent disease categories from 2016-to-2021 received PRO labeling approval compared to 6.03% from 2006-to-2015. Amongst the ICD-10 disease categories, the diseases of the nervous system had the most PRO claims (11 out of 39).

CONCLUSIONS: Further comparative analyses are being made to evaluate the trends on PRO information in NDAs from 2006-2021. However, PROs are becoming part of mainstream drug development and aid in the understanding of ‘clinical significance’.