The importance of patient-centered outcome (PCO) evidence is increasingly recognized, but its inclusion in Health Technology Assessment (HTA) submissions remains inconsistent. We explored the impact of PCO evidence on HTA decision-making.

A framework was developed to assess the impact of PCO evidence (excluding EQ-5D) on HTA appraisals. An impact rating was determined by reviewing company, committee and Evidence Review Group (ERG) opinion. This was applied to publicly available appraisal documents (National Institute for Health and Care Excellence [NICE]: 8; Scottish Medicines Consortium [SMC]: 2) in a pilot study. The framework was then refined and applied to a larger dataset.

PCO evidence had ‘substantial impact’ in 3/8 NICE and 1/2 SMC appraisals, and ‘some impact’ in those remaining. PCO evidence informed the cost-effectiveness model in 2/8 NICE and 1/2 SMC submissions, and was considered superior to EQ-5D evidence in one NICE and one SMC submission. The ERG considered PCO evidence relevant to decision-making in 5/8 NICE appraisals. PCO evidence was mentioned in guidance for 7/10 appraisals (deemed relevant in 5/10). In one assessment, committee comments were notably more favorable than ERG comments. Larger dataset analysis results provided further insights to the pilot study.

The framework allows a systematic approach to evaluating the impact of PCO evidence on HTA appraisals.

BL, AP, DGB and NY are employees of Symmetron Ltd, which received funding from Pfizer UK in connection with the development of this manuscript. KH, HT, SLC and JB are employees of Pfizer UK. This study was sponsored by Pfizer UK.