OBJECTIVES : Patient-reported outcomes (PROs) focus on the patient experience beyond strictly clinical outcomes and are increasingly used in clinical trials and real-world evidence to elucidate multidimensional impact to patients’ health and daily life. However, it is unclear how PROs inform regulatory and reimbursement processes. This review assessed research related to PRO impact on regulatory, health technology assessment (HTA) and market access (MA) milestones.

METHODS : A literature review was undertaken to identify research on the impact of PROs on the three milestones. Searches were conducted in Embase, Medline and REF2014 from 1 January 2009 to 18 August 2020; results were supplemented through hand searching. Information on study design, impact metrics and barriers and facilitators to impact was extracted. EQ-5D data were excluded from the analysis.

RESULTS : Electronic searches identified 809 records; 77 studies (81 publications) were included. Regulatory impact (37 studies), HTA impact (19 studies) and MA impact (47 studies) were assessed. The most common regulatory impact category was “support drug labelling and approval” (30/37) and number of PRO labelling claims was a key impact metric (16/37). Nine studies reported that the EMA is more accepting of PRO claims than the FDA. For HTA, 12/19 studies reported the impact of PROs in “supporting reimbursement decisions”, but only five quantified impact using a metric (number of HTA appraisals including PROs). Several PRO impact categories were identified for MA; however, studies were heterogeneous in terms of design and overall study quality was low (approximately 70% were case studies/made unsubstantiated claims). Across all milestones, the most common barriers to impact were inadequate clinical trial design, insufficient data analysis and a lack of evidence of PRO validity.

CONCLUSIONS : This review shows that most quantitative research on PRO impact has focused on regulatory approval; HTA impact is underexplored and MA impact has been analysed more qualitatively.