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A systematic review of economic evaluations for RPE65-mediated inherited retinal disease including HTA assessment of broader value

Published online by Cambridge University Press:  14 June 2023

Maria Farris Open the ORCID record for Maria Farris [Opens in a new window] ,
Stephen Goodall  and
Richard De Abreu Lourenco
Maria Farris*
Affiliation:
Market Access Department, Novartis Pharmaceuticals Australia, Macquarie Park, Australia
Stephen Goodall
Affiliation:
Centre for Health Economic Research and Evaluation, University of Technology Sydney, Ultimo, Australia
Richard De Abreu Lourenco
Affiliation:
Centre for Health Economic Research and Evaluation, University of Technology Sydney, Ultimo, Australia
*
Corresponding author: Maria Farris; Email: Maria.H.Kokoszka@alumni.uts.edu.au
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Abstract

Objective

To summarize the key methodological challenges identified by health technology assessment (HTA) agencies assessing gene therapy (GT) and consideration of broad elements of value.

Method

Economic evaluations (EEs) of voretigene neparvovec (VN) in RPE65-mediated inherited retinal disease (IRD) published in English were selected. HTA evaluations from Australia, Canada, Ireland, Scotland, England, and the United States were reviewed. An existing methodological framework was used to identify the challenges and considerations.

Results

Eight unique EEs were identified of which six were evaluated by HTA agencies. Incremental cost-effectiveness ratios ranged from $68,951 to $643,813 per quality-adjusted life-years (QALY) gained (healthcare perspective) and dominant to $480,130 per QALY gained (societal perspective). The key challenges were the lack of validated surrogate outcome, utility values and indirect costs from IRD patients, and limited evidence of the long-term treatment effect. Two HTA agencies reviewed a range of novel broader elements of value and whether they were associated with VN while other agencies discussed some elements of broader value. Caregiver disutility was included in some, but not all, evaluations.

Conclusion

The methodological challenges were consistent with innovative interventions for rare diseases and managed using standard methods. Broader value was important to decision-makers but inconsistently applied across agencies. Possible reasons are limitations in the evidence available of the broader benefits that VN offers and how to incorporate these within an EE. A need exists for greater guidance and consistency across jurisdictions regarding the consideration of broader value that considers latest best practice.

Keywords

Technology assessmentbiomedicaldecision makingretinal diseasegenetic therapy
Type
Assessment
Information
International Journal of Technology Assessment in Health Care , Volume 39 , Issue 1 , 2023 , e38
Copyright
© The Author(s), 2023. Published by Cambridge University Press

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