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Unlocking patient access to gene therapy: five key practices

Gene Therapy volume 32, pages 75–77 (2025)Cite this article

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Cost-effectiveness models typically assess short-term affordability and long-term value of chronic therapies [11]. One-time therapies like onasemnogene abeparvovec require a different framework for cost-effectiveness analysis that combines various factors: innovative analogues for other lifelong transformative impact (e.g., transplant surgery), comparison with treatment costs over an extended period, cost-effectiveness comparisons with other available disease-targeting treatments, statistical valuation via systematic review and quantitative analysis, and the welfare economics theory application.

Because of clinical trial limitations, one-time therapies face evidence challenges that RWE studies can address to reinforce confidence in long-term efficacy and safety. By investing in natural history studies, developing disease registries (e.g., RESTORE for spinal muscular atrophy [12]), and/or partnering with other patient registries, organizations, and healthcare stakeholders, companies can improve disease understanding, support clinical trial design and label discussions, and uncover unmet needs and real-world clinical efficacy profiles. RWE can also expand contracting options (e.g., outcomes-based agreements [OBAs] with endpoints beyond clinical trials) and support policy and practices. In some countries (e.g., Germany), RWE is mandated for certain therapies [13].

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Fig. 1
Fig. 2: Availability of rare disease therapies by country type and income with World Bank classification [25].

Data availability

Data sharing is not applicable to this article as no new data were created or analyzed in this study.

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Acknowledgements

Editorial support was provided by Caryne Craige, PhD, of Kay Square Scientific, Inc., Newtown Square, PA, USA.

Funding

Article development support was funded by Novartis Gene Therapies, Inc.

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Authors and Affiliations

  1. Novartis Gene Therapies Inc., Bannockburn, IL, USA

    Tay Salimullah & Burcu Kazazoglu Taylor

  2. Novartis Gene Therapies, London, United Kingdom

    Madeleine Zerbato

Authors
  1. Tay Salimullah
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  2. Burcu Kazazoglu Taylor
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  3. Madeleine Zerbato
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Contributions

TS, BKT, and MZ organized the supportive data presented and prepared the manuscript. All authors reviewed and approved the final manuscript for submission.

Corresponding author

Correspondence to Madeleine Zerbato.

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Competing interests

TS, BKT, and MZ were employees of Novartis Gene Therapies, Inc., at the time of the study.

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Salimullah, T., Kazazoglu Taylor, B. & Zerbato, M. Unlocking patient access to gene therapy: five key practices. Gene Ther 32, 75–77 (2025). https://doi.org/10.1038/s41434-024-00509-2

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