Review of economic modeling evidence from NICE appraisals of rare disease treatments for spinal muscular atrophy

ABSTRACT

Introduction

The National Institute for Health and Care Excellence (NICE) in England has appraised three treatments for spinal muscular atrophy (SMA), namely, nusinersen, onasemnogene abeparvovec, and risdiplam. As rare disease treatments (RDTs) commonly face challenges in health technology assessment (HTA) processes due to their clinical and economic uncertainties, an in-depth review of these appraisals is useful to enable a deeper understanding of economic modeling considerations for SMA.

Areas covered

This review is a detailed analysis of NICE appraisals for SMA and aims to compare the economic modeling evidence from the three RDTs. This is done by examining differences and similarities and by discussing critical outstanding issues across the economic evaluations of the appraisals.

Expert opinion

This article aims to contribute to the development of evidence that can be used as guidance to inform resource allocation decisions for RDTs for SMA, but also to be a resource about approaches for the generation, analysis and interpretation of economic modeling evidence for RDTs more broadly.

KEYWORDS:

• Health technology assessment
• National Institute for Health and Care Excellence
• nusinersen
• onasemnogene abeparvovec
• risdiplam
• spinal muscular atrophy

Article highlights

• Currently, three disease-modifying treatments for spinal muscular atrophy have been appraised by NICE and are available to patients in England

• The comparative assessment of economic evaluations examining the benefits and costs of these treatments for SMA reflect six critical outstanding issues, relating to the classification of SMA health states, long-term survival, resource use data, patient utility values, caregiver utility values, and additional utility values for patients on treatment compared to best supportive care (BSC)

• A consensus on how these issues should be approached in economic evaluations of SMA is desirable to achieve more consistency across appraisals

Declaration of interest

The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Authors contributions

Both authors contributed to conceptualising and designing the study. L Wiedmann analysed the data and drafted the protocol manuscript. J Cairns revised the manuscript for important intellectual content and contributed to the methodology.

Data availability statement

The data that support the findings of this study are available in the NICE technology appraisal guidance and the NICE highly specialized technology guidance at https://www.nice.org.uk/guidance, reference number TA588, TA755, and HST15. Data was derived from the following resources available in the public domain: https://www.nice.org.uk/guidance/ta588/history, https://www.nice.org.uk/guidance/hst15/history, https://www.nice.org.uk/guidance/ta755/history.

Supplementary material

Supplemental data for this article can be accessed online at https://doi.org/10.1080/14737167.2023.2193690

Additional information

Funding

L Wiedmann has received general support through the exposé scholarship scheme funded by the German Academic Scholarship Foundation. The funder was not involved in any aspect of the study conduct or the decision to submit the paper for publication.