Health Technology Assessment Recommendations and Managed Entry Agreements Related to Optimizing the Treatment for Pediatric Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is a rare neuromuscular disease that causes progressive muscle wasting and is associated with a heavy burden for patients and their families. Recent advances have led to the development of 3 new therapies. However, unanswered questions remain regarding the long-term effectiveness of these therapies on disease progression, respiratory and bulbar function, optimal monitoring of patients, and optimal use of available interventions.

The objective of this project is to explore, on an international level, health technology assessment recommendations for use of the 3 new therapies, as well as identified uncertainties and gaps in evidence. If a managed entry agreement for any or all of the drugs has been established, the various components of the agreement will also be described.

Projects listed as “in progress” are at various stages and points of completion. These products have different processes and timelines; therefore, the timing of posting of the final reports varies and expected completion dates may change. Find out more about Projects in Progress.

The “Projects in Progress” page on the CADTH website is updated on a seven- to eight-day cycle. Please be advised that there may be overlap between when a new report is posted online and when the section is updated. View our current Projects in Progress.