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Will rising prices and budget constraints prevent patients from accessing novel gene therapies?

Gene Therapy volume 24, pages 542–543 (2017)Cite this article

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The breadth of potential of gene therapy to combat genetic disorders that are beyond conventional cure is unparalleled. The support afforded to gene therapy by patient organisations is significant and the field is indebted to the thousands of patient volunteers who have taken part in clinical trials over the decades. What ultimately matters is whether gene therapies are accessible and affordable. To date, companies have sought extraordinarily high prices for gene therapy products reaching market. The readiness and ability of healthcare systems to afford these products will determine whether or not the potential of gene therapies will be fully realised. By the same token, it is essential to question whether the prices asked by companies are truly justified. We examine the social, regulatory and economic factors that will determine whether or not gene therapy will deliver on its promise and propose that significant innovation in pricing and health technology will be the key determinants in the future of these innovative treatments.

Of the 2409 gene therapy trials initiated to date, a little over 10% have been in monogenic diseases (248).1 The pace of innovation in gene therapy and the growth of the field have been phenomenal; take for example Spinraza the first disease modifying therapy for Spinal Muscular Atrophy (SMA), taken from its first dose in humans in 2011 to its first regulatory approval in five years.2, 3

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  1. Genetic Alliance UK, London, UK

    A Kent & J Spink

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  1. A Kent
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  2. J Spink
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Correspondence to A Kent.

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Kent, A., Spink, J. Will rising prices and budget constraints prevent patients from accessing novel gene therapies?. Gene Ther 24, 542–543 (2017). https://doi.org/10.1038/gt.2017.66

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