The Increasing Use of Clinical Outcome Assessments in US Food and Drug Administration Labels and the German Gemeinsamer Bundesausschuss, National Institute for Health and Care Excellence, and the French Haute Autorité de Santé Assessments From 2012 to 2022

Abstract

Objectives

Clinical outcome assessments (COAs) are increasingly used in clinical trials. However, knowledge gaps remain around their inclusion in market access decision making for pharmaceuticals and the perceived added value in capturing the patient experience. This study examined trends in the inclusion of COAs in US Food and Drug Administration (FDA) labels and European health technology assessments (HTAs) beyond those that are essential for assessing primary endpoints or determining cost-effectiveness.

Methods

The inclusion of nonmandatory COAs was analyzed in FDA labels and HTAs of German, French, and English bodies, from 2012 to 2022. Changes in COA use are reported overall, by COA type, concept, and therapeutic area.

Results

In FDA labels, the inclusion of nonmandatory COA increased from 18.6% to 28.4% from 2012-2016 to 2017-2022. The average number of unique COAs in FDA labels approximately doubled. Nonmandatory COA inclusions in HTAs increased from 45.2% to 64.9%. The average number of unique COAs per HTA also approximately doubled. Patient-reported outcomes were the most reported COAs in FDA labels and HTAs in both time periods. In FDA labels and HTAs, most therapeutic areas showed increases in nonmandatory COA inclusion. In FDA labels, COA instruments mostly measured signs and symptoms. In HTAs, they mostly measured health status and quality of life.

Conclusions

The findings show increasing trends in the use of nonmandatory COA in HTAs and, to a lesser extent, FDA labels, suggesting a greater awareness of COA benefits and a changing practice of inclusion toward more patient centricity.