Will the US$5 million onasemnogene abeparvosec treatment for spinal muscular atrophy represent ‘value for money’ for the NHS? A rapid inquiry into suggestions that it may be cost-effective

ABSTRACT

Objectives

Nusinersen (Spinraza®, Biogen) and onasemnogene abeparvosec (Zolgensma®, Novartis) are novel gene-based therapies for the orphan disease Spinal Muscular Atrophy. Onasemnogene abeparvosec has been allocated an acquisition cost of up to US$5 million per patient. We undertook a rapid inquiry to evaluate if onasemnogene abeparvosec is likely to be cost-effective for the UK NHS.

Methods

We used publicly available cost-effectiveness data and recommended methodology to perform cost-utility evaluation of onasemnogene abeparvosec versus best supportive care and nusinersen.

Results

Our evaluations highlight wide variations in cost and benefit estimates of nusinersen and indicate that onasemnogene abeparvosec is unlikely to represent value for money according to current standards of reimbursement. Results are discussed in the context of reimbursement decisions for orphan diseases.

Conclusion

Commonly implemented commercial confidentiality practices combined with uncertain data obscure scrutiny and justification of past and present reimbursement decisions for orphan drugs. Future cutting edge expensive therapies will be numerous, they will entail very substantial economic strains. We conclude that there is an urgent and increasing need for the development of improved procedures that can lead to equitable, consistent, and transparent decision-making.

KEYWORDS:

• Cost-effectiveness
• onasemnogene abeparvosec
• spinal muscular atrophy
• Zolgensma

Article highlights

• Onasemnogene abeparvosec and nusinersen are expensive new gene-based therapies for the orphan disease SMA1, they represent harbingers for the development of gene therapies for many rare diseases in the near future.

• The treatment cost of these therapies is in the multiple million £ range per individual patient.

• Utilizing data from industry sponsored cost-utility analyses we evaluated their cost-effectiveness from the perspective of the UK NHS.

• Our evaluations indicate that these therapies are unlikely to represent value for money for the NHS but that accurate assessment is difficult due to discrepant estimates from different industry sources and the commercially confidential cost arrangements established between industry and decision makers.

• Reimbursement decisions for orphan drugs need to be equitable, consistent, and transparent; improved policies are required.

Author contributions

XA and MC conceived and designed the study; LA and PA undertook the reported cost-effectiveness analyses. All authors contributed to data interpretation and manuscript preparation. All authors approve the final version and agree to be accountable for all aspects of the work. No funding was received for this study.

Declaration of interest

The authors have no 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. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Reviewer disclosures

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

Additional information

Funding

This paper was not funded.