OBJECTIVES: With growing interest in capturing patient experience during clinical trials, the use of PRO measures (PROMs) has been recommended by regulatory authorities. This study aimed to evaluate the use of PROMs in company evidence submitted for NICE STA in the last 5 years.

METHODS: STAs published on the NICE website between April 1st 2018 and April 1st 2023 were reviewed. Information on disease indication, key trials design, and types of PROMs were collected and analysed.

RESULTS: In total, 352 STAs were identified. After excluding appraisals which were terminated, replaced by newer guidance, or in development, 257 STAs were included . Of these 257 STAs , 140 (54.5%) were published in oncology, 20 (7.8%) in musculoskeletal, and 19 (7.4%) in neurological indications. PROMs were reported in 227 STAs (88.3%) and were limited to health-related quality of life (HRQoL) outcomes. 131 different PROMs were identified, including: (1) indication-specific QoL measures (n=76; 58.0%), (2) generic QoL measures for symptoms (e.g., pain), function (e.g., visual), or safety (e.g., adverse events) (n=30; 22.9%), and (3) generic QoL measures (n=25; 19.1%). The EuroQol-5 Dimensions (EQ-5D) and the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-core questionnaire (EORTC QLC-C30) were the most commonly used PROMs (n=144; 63.4% and n=81, 35.7%, respectively).

We further analysed PROMs in oncology STAs. Across 140 STAs, 125 (89.3%) conducted PROM assessments, the majority of which used generic QoL measures (n=124; 99.2%), indication-specific measures were used in 63 STAs (50.4%), and generic measures for symptoms, function, or safety were reported in 15 STAs (12.0%). Most oncology STAs used a combination of generic and indication-specific PROMs (46.4%) or generic PROMs only (41.6%).

CONCLUSIONS: The frequent use of PROMs in evidence submitted by company highlights the importance of QoL data in NICE STAs to support health technology assessment decision making.