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

Abstract

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.

Keywords: Genetic therapy; biomedical; duchenne; muscular atrophy; neuromuscular diseases; spinal; muscular dystrophy; technology assessment.

Fig. 1

Interventional clinical trials of gene therapies for neuromuscular diseases. Note: The data shown was retrieved from ClinicalTrials.gov on June 1, 2022, based on a search of [(gene therapy) OR (gene treatment) OR (DNA therapy) OR (DNA treatment) OR (gene transfer)] AND (neuromuscular diseases), manually curated to exclude conditions other than neuromuscular diseases and interventions other than gene therapies. Only interventional trials with a specified phase were included for analysis. Becker Muscular Dystrophy (BMD). Duchenne Muscular Dystrophy (DMD). Limb Girdle Muscular Dystrophy (LGMD). Spinal Muscular Atrophy (SMA). X-Linked Myotubular Myopathy (XLMTM).