Patient Experience in Health Technology Assessment (HTA): Landscape Assessment and Comparison of HTA Guidance on Patient Reported Outcome Measures (PROMs), With a Review of HTA Appraisals in Multiple Myeloma (MM)

OBJECTIVES: Measuring patients' treatment experience with patient-reported outcome measures (PROMs) during drug development is necessary in decision-making. We conducted a landscape assessment and comparison of HTA guidance on PROMs and summarized their use in multiple myeloma (MM) HTAs as a case study.

METHODS: Targeted review across 15 HTA organizations (12 countries) was conducted in December 2023 to identify HTA methods guidance and technology appraisals including PROMs for MM. Findings were synthesized by study design, fit-for-purpose data, study conduct and reporting.

RESULTS: Twenty-two HTA guidance documents (12 organizations) and 116 MM HTA appraisals (10 organizations) mentioning PROMs were identified. Germany had the most guidance/appraisal documents (n=50). Guidance review: Organizations from 10 countries/regions provided PROM recommendations; most recommended methods for PROM analysis (n=11), use of validated tools (n=7) and acceptable trial designs (n=7); European Union (EU) HTA regulation and German guidance discussed the use of minimally-important-differences for interpretation of PROMs. HTA appraisals review: Most documents included disease-specific PROMs (EORTC-QLQ-C30, n=109; EORTC-QLQ-MY20, n=52) but preferences for generic (e.g., EQ-5D) versus disease-specific PROMs varied across countries. All data were from pivotal clinical trials; Where reported, HTA critique focused on open-label trial design (n=30), lack of comparator data (n=5), incomplete data (n=2), and unclear definitions (n=4); Psychometrics were rarely discussed (4 German, 4 Canadian submissions). Additional benefit was granted only to two drugs (4 German appraisals) based on PROM improvements despite the absence of significant survival.

CONCLUSIONS: Guidance on PROMs differed across countries/regions, limiting the transparency in acceptable criteria for PROMs in decision-making, as evident in our review of MM HTA appraisals. To improve consideration of PROMs and integration of the patient experience in decision-making, clarity, such as efforts by EU Member State Coordination Group on HTA and GBA to standardize PROM data collection and interpretation, and harmonization of requirements across HTA bodies is needed.