The increasing use of clinical outcome assessment in FDA labels and G-BA, NICE and HAS assessments from 2012-2022.

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 essential to assess primary endpoints or determine cost-effectiveness.

Methods: The inclusion of "non-mandatory COAs" was analysed in FDA labels and HTAs of German, French and English bodies, from 2012-2022. Changes in COA use are reported overall, by COA type, concept, and therapeutic area (TA).

Results: In FDA labels the inclusion of non-mandatory 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. Non-mandatory 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 (PROs) were the most reported COAs in FDA labels and HTAs in both time periods. In FDA labels and HTAs, most TAs showed increases in non-mandatory COA inclusion. In FDA labels, COA instruments mostly measured signs and symptoms. In HTAs, they mostly measured health status and quality of life.

Conclusion: The findings show increasing trends in the use of non-mandatory COA in HTAs, and to a lesser extent FDA labels, suggesting a greater awareness of COA benefits and a changing practice of inclusion towards more patient-centricity.