The potential price and access implications of the cost-utility and budget impact methodologies applied by NICE in England and ICER in the US for a novel gene therapy in Parkinson's disease. Issue 1 (1st January 2018)

• Record Type: Journal Article
• Authors: Jørgensen, Jesper; Servos, Spiros; Kefalas, Panos
• Is Part Of: Journal of market access & health policy. Volume 6:Issue 1(2018)
• Publication Date: 2018-01-01
• Subjects: Gene therapy -- Parkinson's disease -- cost-utility analysis -- budget impact analysis -- pricing and reimbursement -- patient access -- England -- United Kingdom (UK); Medical policy -- Periodicals; Medical policy -- Economic aspects -- Periodicals; International trade -- Environmental aspects -- Periodicals; Health Policy -- economics; Technology Assessment, Biomedical; Marketing of Health Services; International trade -- Environmental aspects; Medical policy; Periodicals; Periodicals; Fulltext; Internet Resources; Periodicals; 362.1
• DOI: 10.1080/20016689.2018.1500419
• Languages: English
• ISSNs: 2001-6689

Abstract

ABSTRACT: Background : NICE in England, and ICER in the US both use cost-utility analyses (CUA) and budget impact analyses (BIA) to assess value for money and affordability, however the thresholds used differ greatly. Objective : To perform a cross-country comparison of the results of the CUA and BIA and detail the implications for reimbursed price and volumes, for a novel gene therapy for Parkinson's disease (PD). Methods : A Markov model was built to perform country-specific CUAs and BIAs Findings : The US ceiling price identified through CUA is ~ 1.8 times higher than in England (aligning to our previous US/UK price comparison analysis of high-cost drugs). However, the net budget impact corresponding to these price levels would limit number of patients treated in order not to exceed the BIA threshold. Performance-based annuity payments can increase patient access at launch without exceeding the thresholds while reducing payers' data uncertainty. Conclusion : Our cost-utility analysis in PD shows a difference in price potential between the US and England that aligns with what is observed in practice for other high-cost drugs. Furthermore, the budget impact threshold operational in England imposes a greater downwards pressure on price and/or volumes than the one applied by ICER in the US.