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.

Strengthening health systems for access to gene therapy in rare genetic disorders

“…With several gene therapies now approved by health regulators globally and with more expected to be approved over the coming years, 1 focus must now be placed on how individuals with rare genetic disorders can achieve timely access to these potentially transformative therapies and what health systems need to due to enable this. Patient centricity is key and is essential to co-create solutions with patients.…”

“…After decades of innovation, the promise of gene therapy is becoming a reality for patients with certain rare genetic disorders. Several cell and gene therapies have already been approved by regulators worldwide, 1 with many more expected at a rate of up to 10%–20% annually by 2025. 2 These therapies have the potential to be transformative, changing the course of a disease and reducing the long-term patient and health system burdens associated with chronic disease management ( Figure 1 ).…”

“…Following regulatory approval, evaluation of comparative effectiveness for gene therapies is often complicated by uncertainties due to small clinical trial sizes, lack of appropriate direct comparators, and the need for long-term follow-up. 1 …”

“…As such, the development of methodologies with greater flexibility that can appropriately incorporate estimates of future benefits is required to support reimbursement decisions, for example incorporating a lifetime perspective in modeling and utilizing appropriate discount rates in budget sensitivity. 1 …”

“…As such, novel payment models that spread payment over time and link to real-world outcomes could help payers to manage the budget impact of gene therapies while recognizing the innovative one-time approach. 1 , 11 Piloting innovative outcomes-based agreements, updating legal and regulatory frameworks, and sharing best practices around innovative financing would be useful to facilitate access to gene therapies. 11 To enable this, registries need to be identified or established to collect patient outcome data to evaluate the safety and durability of gene therapies over time.…”