Consistency in Reimbursement Decisions for Pediatric Rare Disease Drugsat Canadian HTA Agencies: CDA Versus INESSS
Author(s)
Saadul Islam, Masters;
Queen's University, Department of Public Health Science, Kingston, ON, Canada
Queen's University, Department of Public Health Science, Kingston, ON, Canada
Presentation Documents
OBJECTIVES: In Canada, pharmaceutical products are subject to health technology assessment (HTA) in tandem or following market approval by Health Canada. Canada’s Drug Agency (CDA) makes reimbursement recommendations that are considered by provincial and federal plans, with the exception of Quebec, where Institute national d’excellence en santé et en services sociaux (INESSS) issues recommendations to the Regie d’assurance maladie Quebec (RAMQ). It is important to understand the differences between these two agencies with respect to reimbursement decisions, which may provide strategic insights for drug manufacturers and industry professionals.
METHODS: Using our proprietary CDA Forecaster® database, we examined the recommendations by both agencies and compared recommendation positivity and congruence for all drugs for pediatric rare diseases (age <18 years) which have been reviewed by CDA and INESSS from January 1, 2015, up to December 31, 2024.The definition of rare disease was based on the widely used definition from the Canadian Organization for Rare disease (CORD), i.e. conditions affecting less than 1 in 2,000 people [Canadian Organization for Rare Diseases. “About CORD Key Facts.” https://www.raredisorders.ca/about-cord/]. We will analyze the rationale for differences in recommendations by the two agencies, and determine if there are any commonalities.
RESULTS: A total of 917 submissions was identified from CDA during the 10-year review period and 6652 from INESSS from inception to present date. These submissions are currently being reviewed to analyze the reason for differences in reimbursement decisions/answer the research question.
CONCLUSIONS: Final results will be provided in the upcoming ISPOR conference. It is expected that the findings from this research will provide valuable insights into the key focuses of the two HTA agencies, particularly where disagreements exist in reimbursement decision.
METHODS: Using our proprietary CDA Forecaster® database, we examined the recommendations by both agencies and compared recommendation positivity and congruence for all drugs for pediatric rare diseases (age <18 years) which have been reviewed by CDA and INESSS from January 1, 2015, up to December 31, 2024.The definition of rare disease was based on the widely used definition from the Canadian Organization for Rare disease (CORD), i.e. conditions affecting less than 1 in 2,000 people [Canadian Organization for Rare Diseases. “About CORD Key Facts.” https://www.raredisorders.ca/about-cord/]. We will analyze the rationale for differences in recommendations by the two agencies, and determine if there are any commonalities.
RESULTS: A total of 917 submissions was identified from CDA during the 10-year review period and 6652 from INESSS from inception to present date. These submissions are currently being reviewed to analyze the reason for differences in reimbursement decisions/answer the research question.
CONCLUSIONS: Final results will be provided in the upcoming ISPOR conference. It is expected that the findings from this research will provide valuable insights into the key focuses of the two HTA agencies, particularly where disagreements exist in reimbursement decision.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
HTA80
Topic
Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes
Disease
SDC: Pediatrics, SDC: Rare & Orphan Diseases