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Approval and therapeutic value of gene therapies in the US and Europe



Gene therapies are a fast-growing area of innovation and hold promise for the treatment of many diseases currently with unmet medical need. To better understand the clinical importance of the current landscape of approved gene therapies, we conducted a systematic analysis of the approved gene therapies and their added therapeutic value. Through December 2022, 13 gene therapies have been approved in the US, 15 in the EU, and 9 in Switzerland. Nine gene therapies have been approved in all three jurisdictions, and 11 in both the US and EU. Among the 11 gene therapies approved in more than one jurisdiction, there were differences in the approved indications among the regulatory agencies, mostly the European drug agencies (EMA and Swissmedic) being more restrictive. Among the gene therapies with available therapeutic ratings, approximately two-thirds had high added therapeutic value, which is substantially higher than the average prevalence of high added therapeutic value ratings among new drugs and biologics (approximately one-third). However, therapies with high added therapeutic value will not be useful for patients if excessive prices limit access to them. Drug pricing reforms should address gene therapies to ensure access to new gene therapies that can offer important therapeutic value to patients.

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This study was partially funded by the Swiss National Science Foundation (SNSF, grant number 194607) and Kaiser Permanente Institute for Health Policy. KNV work is also funded by the Swiss Cancer Research Foundation (Krebsforschung Schweiz). ASK work is also funded by Arnold Ventures.

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Correspondence to Aaron S. Kesselheim.

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Vokinger, K.N., Glaus, C.E.G. & Kesselheim, A.S. Approval and therapeutic value of gene therapies in the US and Europe. Gene Ther 30, 756–760 (2023).

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