Reference Framework, Key Decision-Making Factors, and the Impact of Critical Parameters on Listing Recommendations for Nononcologic Drugs for Rare Diseases Issued by Canadian HTA Bodies
Author(s)
Isabelle Lejeune, BSc, MSc1, Dan Cooper, BPharm, MSc, PhD2, Michelle Savoie, MBA, MPH, PhD, LLM, IAS.A3.
1Student, University of Montreal, Montréal, QC, Canada, 2TACT Intelligence-Conseil Inc., Quebec, Qc, QC, Canada, 3Faculté de pharmacie, Université de Montréal, Montréal, QC, Canada.
1Student, University of Montreal, Montréal, QC, Canada, 2TACT Intelligence-Conseil Inc., Quebec, Qc, QC, Canada, 3Faculté de pharmacie, Université de Montréal, Montréal, QC, Canada.
OBJECTIVES: The absence of specific Health Technology Assessment (HTA) guidelines for rare diseases remains a limitation in Canada. The decision-making criteria used by Canadian HTA agencies, Institut national d’excellence en santé et en services sociaux (INESSS) and the Canadian Drug Agency (CDA), are not well defined and have been minimally studied to date. This research aimed to analyse the key decision-making parameters underlying drug listing recommendations for non-oncologic drug for rare diseases (NODRDs) issued by INESSS and CDA from 2020 to 2023, to develop a reference framework for the recommendations of drugs intended to treat rare non-oncologic diseases in Canada.
METHODS: A reimbursement review was conducted for NODRDs (defined at disease prevalence ≤ 1/2,000, derived from the rare disease registry of Orphanet) of listing recommendation issued from 2020 to 2023 inclusively. Data extraction, synthesis and characterization focused on clinical evidence, pharmacoeconomic results, key decision-making rationales reported by each HTA agency and outcome of the pricing negotiations with the pan-Canadian Pharmaceutical Alliance (pCPA) following positive listing recommendations.
RESULTS: Among the 48 recommendations analyzed for this study, CDA issued a significantly higher proportion of positive recommendations (88%, n=42) compared to INESSS (56%, n=27) respectively. In 40% (n=19) of cases, the two agencies reached divergent conclusions on the listing recommendation of the same drug. The key predominant decision-making reported in INESSS and CDA’s negative recommendations were similar, but weighted differently, except for safety and cost-effectiveness considerations as no negative recommendation were issued by CDA for economic reasons or toxicity.
CONCLUSIONS: The findings of this Canadian comparative analysis support the need for more harmonized and adaptative HTA processes that bridge clinical, economic, and policy considerations, particularly in the context of high-cost, evidence-limited therapies for rare diseases as only 71% of positive recommendations led to successful pCPA negotiations, underscoring a critical disconnect between HTA outcomes and patient access in Canada.
METHODS: A reimbursement review was conducted for NODRDs (defined at disease prevalence ≤ 1/2,000, derived from the rare disease registry of Orphanet) of listing recommendation issued from 2020 to 2023 inclusively. Data extraction, synthesis and characterization focused on clinical evidence, pharmacoeconomic results, key decision-making rationales reported by each HTA agency and outcome of the pricing negotiations with the pan-Canadian Pharmaceutical Alliance (pCPA) following positive listing recommendations.
RESULTS: Among the 48 recommendations analyzed for this study, CDA issued a significantly higher proportion of positive recommendations (88%, n=42) compared to INESSS (56%, n=27) respectively. In 40% (n=19) of cases, the two agencies reached divergent conclusions on the listing recommendation of the same drug. The key predominant decision-making reported in INESSS and CDA’s negative recommendations were similar, but weighted differently, except for safety and cost-effectiveness considerations as no negative recommendation were issued by CDA for economic reasons or toxicity.
CONCLUSIONS: The findings of this Canadian comparative analysis support the need for more harmonized and adaptative HTA processes that bridge clinical, economic, and policy considerations, particularly in the context of high-cost, evidence-limited therapies for rare diseases as only 71% of positive recommendations led to successful pCPA negotiations, underscoring a critical disconnect between HTA outcomes and patient access in Canada.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA283
Topic
Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes, Value Frameworks & Dossier Format
Disease
Rare & Orphan Diseases