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

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...

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Main Authors: Jesper Jørgensen, Spiros Servos, Panos Kefalas
Format: Article
Language:English
Published: MDPI AG 2018-01-01
Series:Journal of Market Access & Health Policy
Subjects:
Online Access:http://dx.doi.org/10.1080/20016689.2018.1500419
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author Jesper Jørgensen
Spiros Servos
Panos Kefalas
author_facet Jesper Jørgensen
Spiros Servos
Panos Kefalas
author_sort Jesper Jørgensen
collection DOAJ
description 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.
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spelling doaj-art-c17a65ddee2144eda3fc43b9cb4bfe772025-08-20T03:23:29ZengMDPI AGJournal of Market Access & Health Policy2001-66892018-01-016110.1080/20016689.2018.15004191500419The 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 diseaseJesper Jørgensen0Spiros Servos1Panos Kefalas2Cell and Gene Therapy CatapultOxford BioMedica (UK)Cell and Gene Therapy CatapultBackground: 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.http://dx.doi.org/10.1080/20016689.2018.1500419Gene therapyParkinson’s diseasecost-utility analysisbudget impact analysispricing and reimbursementpatient accessEnglandUnited Kingdom (UK)
spellingShingle Jesper Jørgensen
Spiros Servos
Panos Kefalas
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
Gene therapy
Parkinson’s disease
cost-utility analysis
budget impact analysis
pricing and reimbursement
patient access
England
United Kingdom (UK)
title 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
title_full 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
title_fullStr 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
title_full_unstemmed 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
title_short 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
title_sort 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
topic Gene therapy
Parkinson’s disease
cost-utility analysis
budget impact analysis
pricing and reimbursement
patient access
England
United Kingdom (UK)
url http://dx.doi.org/10.1080/20016689.2018.1500419
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