Evaluating the Evolution of Patient-Reported Outcome Measures (PROMs) in Health Technology Assessments: A Focus on Neurological Diseases in NICE Technology Appraisals

OBJECTIVES: The utilization of Patient-Reported Outcome Measures (PROMs) has amplified in recent years in response to the increased need to incorporate patients' Quality-of-Life (QoL) experiences in the health technology assessments (HTA). The objective of this study is to assess the use of PROMs in the evidence submitted by the drug manufacturers for National Institute for Health and Care Excellence (NICE) technology appraisals related to neurological diseases.

METHODS: We have reviewed published NICE technology appraisals for neurological diseases between January 1, 2001, and December 15, 2023. This includes company submissions, final appraisal documents, and committee papers. Information regarding PROMs, their specific purposes, and the mapping between PROMs were extracted.

RESULTS: After excluding terminated appraisals, 50 technology appraisals (TA) were identified. In 94% of the submissions, manufacturers used PROMs in the pivotal trials. We classified PROMs into three distinct categories: generic QoL (GQoL), disease-specific QoL (DQoL), and symptom-specific QoL (SQoL) measures. Out of 39 distinct PROMs 23% were GQoL (n=9), 62% were DQoL (n=24) and 15% were SQoL (n=6) measures. The predominant measures used were: EuroQol-5 Dimensions (EQ-5D) for GQoL in 76% of the TAs (n=38), Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) for SQoL in 8% of the TAs (n=4), and Migraine-Specific Quality of Life Questionnaire (MSQ) for DQoL in 12% TAs (n=6). Mapping between PROMS was observed in 6 TAs. Among them, MSQ was mapped to EQ-5D in 4 TAs related to headaches. Out of 2 TAs for epilepsy, the Quality of Life in Epilepsy-31-P scale was mapped to SF36 in 1 TA, while EQ-5D-5L was mapped to EQ-5D-3L in the other TA.

CONCLUSIONS: Over time, there has been a shift towards using DQoL measures alongside GQoL measures for a more focused evaluation. The growing trend in the manufacturers’ regular use of PROMs highlights the necessity of QoL data in HTA for supporting decision-making.