OBJECTIVES: Neurodegenerative diseases (NDs) have lagged behind in terms of therapeutic innovation compared to other chronic diseases. However, recent successful launches have allowed the commercialization of few disease-modifying therapies (DMTs) targeting NDs. Considering that promising therapies (e.g., sodium phenylbutyrate and taurursodiol, lecanemab) are launching in North America, this study aims to review Health Technology Assessments (HTA) on previous DMTs on NDs in Canada and provide learnings for upcoming DMTs.

METHODS: We followed the National Institutes of Health´s (NIH) definition of NDs. Subsequently, we retrieved all HTA reports evaluating DMTs on NDs published by the Canadian Agency for Drugs and Technologies in Health (CADTH) in the past ten years. Finally, we conducted descriptive analysis on the reports to identify areas of transferable learning.

RESULTS: We identified five diseases classified as NDs by the NIH. Among these, only amyotrophic lateral sclerosis (ALS) was associated with DMTs. Within ALS, only two DMTs were identified. For both DMTs, reimbursement was granted only for early stages of the disease and tied to a 95% price reduction to meet the willingness-to-pay thresholds (WTPT) for cost-effectiveness. In the HTA reports, both DMTs + standard of care (SoC) were compared against placebo + SoC, as no DMTs were available. The efficacy of both DMTs was assessed using a clinically validated disease-specific instrument to measure disease progression and neurologic functionality. The relatively short time in the clinical trials length and follow-up was aligned with the fast disease progression.

CONCLUSIONS: Manufacturers can expect similar methodology and requirements for the assessment of new DMTs in NDs as in other therapeutic areas since the HTA decision-making process appears to be aligned with that one seen in other chronic diseases. In contrast, the appropriateness of short clinical trial and follow-up times in NDs is related to fast disease progression and expected short survival time.