Gene Therapy for Neuromuscular Diseases: Health Economic Challenges and Future Perspectives

Highly efficacious, potentially curative gene therapies holds immense clinical promise, but also present complex challenges. At the time of regulatory approval and health technology assessment (HTA), evidence of efficacy and safety of gene therapies is often uncertain. In addition, research, development, and manufacturing costs, small pools of eligible patients, and the fact that many gene therapies are administered only once means that they frequently are associated with very high "one-off" price points. Although only a limited number of products have been brought to market globally, hundreds of clinical trials of gene therapies, including several of monogenetic neuromuscular diseases, are currently ongoing. Over time, as more and more conditions become amendable to gene therapy, the number of transformative, high-cost treatments is likely to increase considerably. For these reasons, concerns have been raised regarding the suitability of current health policy systems, including HTA frameworks, in ensuring appropriate access to these therapeutic innovations while simultaneously safeguarding value for taxpayers' money, as well as affordability and sustainability. This review provides a summary overview of current challenges and future perspectives of gene therapies for neuromuscular diseases from a health economic point of view.