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Volume 6
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10.1080/20016689.2018.1500419
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Journal of Market Access & Health Policy (JMAHP) is published by MDPI from Volume 12 Issue 1 (2024). Previous articles were published by another publisher in Open Access under a CC-BY (or CC-BY-NC-ND) licence, and they are hosted by MDPI on mdpi.com as a courtesy and upon agreement with Taylor & Francis.
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Article

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

by
Jesper Jørgensen
1,
Spiros Servos
2 and
Panos Kefalas
1,*
1
Health Economics and Market Access Department, Cell and Gene Therapy Catapult, 12th Floor Tower Wing, Guys Hospital, Great Maze Pond, London SE1 9RT, UK
2
Business Development Department, Oxford BioMedica (UK), Oxford, UK
*
Author to whom correspondence should be addressed.
J. Mark. Access Health Policy 2018, 6(1), 1500419; https://doi.org/10.1080/20016689.2018.1500419
Submission received: 12 April 2018 / Revised: 4 July 2018 / Accepted: 9 July 2018 / Published: 6 August 2018

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.
Keywords: gene therapy; Parkinson’s disease; cost-utility analysis; budget impact analysis; pricing and reimbursement; patient access; England; United Kingdom (UK) gene therapy; Parkinson’s disease; cost-utility analysis; budget impact analysis; pricing and reimbursement; patient access; England; United Kingdom (UK)

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MDPI and ACS Style

Jørgensen, J.; Servos, S.; Kefalas, P. 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. J. Mark. Access Health Policy 2018, 6, 1500419. https://doi.org/10.1080/20016689.2018.1500419

AMA Style

Jørgensen J, Servos S, Kefalas P. 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. Journal of Market Access & Health Policy. 2018; 6(1):1500419. https://doi.org/10.1080/20016689.2018.1500419

Chicago/Turabian Style

Jørgensen, Jesper, Spiros Servos, and Panos Kefalas. 2018. "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" Journal of Market Access & Health Policy 6, no. 1: 1500419. https://doi.org/10.1080/20016689.2018.1500419

APA Style

Jørgensen, J., Servos, S., & Kefalas, P. (2018). 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. Journal of Market Access & Health Policy, 6(1), 1500419. https://doi.org/10.1080/20016689.2018.1500419

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