Health Technology Assessment Recommendations for Pharmaceutical Drugs Submitted in Blood Disorders in CDA and INESSS: Focus on Rare Diseases

OBJECTIVES: While progress in screening and new treatments for blood disorders has been made, unique characteristics of rare diseases can pose barriers to accessing novel treatments. To gain an understanding of reimbursement decisions, we reviewed and characterized Canadian HTA submissions in blood disorders with a focus on rare diseases.

METHODS: Submissions for blood disorders 2019–2024 were retrieved from the CDA. Submissions for these products were also retrieved from the INESSS website. Information on indication, product, pivotal trial design, additional clinical evidence submitted, final decision and critique were extracted.

RESULTS: Of 123 HTAs by CDA between 2019 and 2024, 16 were for blood disorder-related therapies, including 11 for rare blood disorders. Seven of these products were also assessed by INESSS during this period. Two out of 11 products received a negative reimbursement decision by CDA, of which one also received a negative decision from INESSS. This product was resubmitted for review but both agencies’ decisions remained unchanged. Two products had differing recommendations between the agencies. Main criticisms mentioned in the negative recommendations were related to limitations in the clinical evidence submitted and uncertainty around the clinically meaningful benefit of the product. Two single-arm trial-based submissions for hemophilia B treatment received positive recommendations from CDA, which noted both therapies addressed patient important needs as gene therapies. Conversely, one of these products received a negative recommendation by INESSS due to the lack of therapeutic value demonstrated.

CONCLUSIONS: Most therapies assessed for rare blood disorders received positive recommendations from CDA, however, there was moderate agreement in CDA and INESSS decisions. Unmet needs and rarity of the conditions were frequently noted in the rationale for positive recommendations. Future studies can build on this research by assessing concordance between CDA recommendations and listing decisions of participating drug plans.