Influence of patient-reported outcomes on regulatory, HTA, and market access decisions

Abstract

OBJECTIVES
To identify key drivers for the successful integration of patient-reported outcomes (PROs) in clinical programs designed to support regulatory label claims, health technology assessment (HTA) and market access (MA) in diabetes and obesity.

METHODS
Reviews of published literature, regulatory, HTA and third-party websites were conducted to elicit key components of PRO strategies employed for two case examples: exenatide (diabetes) and lorcaserin (obesity). Qualitative one-on-one interviews were also conducted with payer decision-makers in key European (EU) and United States (US) markets to determine their perceptions of successful PROs strategies.

RESULTS
Although there is an FDA draft guidance on PROs to support labeling claims, no disease-specific guidance on PRO-endpoints for obesity/diabetes are issued by FDA or EMA. Registration-trials included assessments of; symptoms/health-related-quality-of-life/depression/psychological-well-being. Exenatide: no PRO US/EU label claims sought. Lorcaserin: PRO-data supported US-approval (no label-claim). PRO-data supported MA for the UK and US. Key findings from payer interviews (n=6 EU/n=4 US) revealed that healthcare-system differences influence payer-preferences for PRO-type (e.g., generic/disease-specific), influenced primarily by need for cost-effectiveness model. All payers stated that PRO-data provide unique perspective on condition/treatment but considered reimbursement difficult to influence; although stated that PRO-data can positively impact prescribing/MA. Payers reported that in EU, PROs are part of the clinical-evidence considered by HTA reviewing committees. Utility data derived from generic instruments are essential for key EU-markets (excluding Germany). US health plans are interested in PRO measures that are tied to “actionable” endpoints (e.g., adherence/persistence, tolerability, reduction in costs or resource utilisation).

CONCLUSIONS
Key drivers for a successful PRO strategy include the development of robust PRO and communication strategy tailored to the needs/requirements of the end-users (regulators, HTA and MA). Sponsors must continue to bring data based on robust PROs to regulators, HTA and MA and thus bringing the patient, the most affected stakeholder, to the forefront in decision making.