Presentation (CTI)

OBJECTIVES: Outcomes-based agreements (OBAs), and coverage with evidence development (CED), are often positioned as mechanisms for managing HTA uncertainty. This study assessed whether OBA-CED mechanisms are used routinely in response to clinical uncertainty, or selectively within a narrower subset of European HTA and reimbursement decisions.
METHODS: We reviewed published HTA and reimbursement decisions for oncology, rare disease, and advanced therapy medicinal products (ATMPs), from 2023 to 2025. Decisions were coded for publicly identifiable OBA-CED use: performance-linked payment, CED, registry-based conditional reimbursement, or other outcomes-linked access mechanisms. Evidence limitations, endpoint suitability, and access context were coded using pragmatic descriptive indices. Access context captured orphan or rare disease status, ATMP designation, limited treatment alternatives, unmet need, and high budget impact. Analyses mapped evidence profiles, high-plausibility OBA-CED scenarios, and observed use.
RESULTS: Overall, 93 decisions across 35 products, and 6 countries were included. OBA-CED mechanisms were identified in 11 decisions (11.8%): 5 CED, 5 performance-linked payment, and 1 registry-based conditional reimbursement. Use varied by country, from none in France, to 27.8% in the UK (n=5/18), and 33.3% in Italy (n=2/6). Use was concentrated among ATMPs (n=8/14; 57.1%), and orphan-designated products (n=8/29; 27.6%), representing 11 products (31.4%). High clinical uncertainty was common but not sufficient, with OBA-CED used in 21.6% of high-uncertainty decisions (n=8/37). Access context was more discriminating, with OBA-CED used in 57.1% of high access-context decisions (n=8/14). Case review suggested clustering around very high-cost therapies, with high budget impact reported in 8 OBA-CED decisions (72.7%). Among 11 high-plausibility scenarios, only 6 included OBA-CED (54.5%).
CONCLUSIONS: Publicly identifiable OBA-CED mechanisms were uncommon and concentrated in selected access contexts and jurisdictions. Clinical uncertainty alone did not appear to drive uptake. OBA-CED should be viewed less as a routine solution to uncertainty, and more as a selective, institutionally dependent access mechanism requiring measurable endpoints, implementation infrastructure, and policy readiness.