Presentation (CTI)

OBJECTIVES: High budget impact therapies often face reimbursement delays or access restrictions despite substantial unmet medical need. These barriers are driven by payer uncertainty regarding long-term effectiveness, real-world adherence, and population-level affordability. This research examines how innovative contracts have improved access to high budget impact therapies in other disease areas and what implications these analogues have for obesity pharmacotherapy. Given the increasing prevalence and health system burden of obesity, alongside limited reimbursed access to effective anti-obesity medications, a assessment of analogue experience is timely.
METHODS: A structured literature review (2010-2024) was conducted using PubMed, EMBASE, HTA agency publications, national reimbursement databases, and manufacturer-payer agreement reports. Four analogue categories were selected based on high budget impact and use of innovative contracts: CAR-T cell therapies (oncology); hepatitis C antivirals (population-level cure models); spinal muscular atrophy gene therapies (ultra-high cost one-time treatments); and PCSK9 inhibitors (targeting large prevalent populations). Data were extracted on contract structures, endpoints, data requirements, affordability mechanisms, and impacts on access decisions.
RESULTS: Across all analogue categories, innovative contracts improved reimbursement feasibility. Outcomes-based arrangements for CAR-T therapies linked payment to remission or survival milestones, reducing payer uncertainty and supporting approvals in Germany, Italy, and the UK. Subscription-based models for hepatitis C antivirals enabled population-level treatment while capping expenditure in Australia and several U.S. states. For spinal muscular atrophy gene therapies, annuity-based and outcomes-verified payments mitigated upfront cost risk. Performance-linked pricing and real-world adherence monitoring for PCSK9 inhibitors facilitated reassessment and broader reimbursed use. Key transferable elements included measurable endpoints, early data infrastructure planning, shared governance, prospective data collection, and financial guardrails.
CONCLUSIONS: Experience from multiple high budget impact therapeutic areas demonstrates that innovative contracting can reduce uncertainty, align stakeholders, and expand access while preserving financial sustainability. These mechanisms are directly applicable to obesity pharmacotherapy and offer a pathway to address the current access gap.