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Recommendations Framework for Drugs for Rare Diseases: A Review of National and International Health Technology Assessment Agencies and Public Payers’ Decision-Making Processes

Published on: June 22, 2018
Project Number: ES0326-000
Product Line: Environmental Scans
Result type: Report

The number of drug for rare diseases (DRD) in the pipeline and the number of marketed DRDs are expanding. As such, policy-makers and clinicians have struggled on how to make DRDs available to patients while ensuring that only clinical- and cost-effective treatments are reimbursed.

This Environmental Scan identifies, describes, and compares how health technology assessment (HTA) agencies in Canada and internationally make funding recommendations on DRDs. The Environmental Scan also presents information on how funding agencies — that is public payers — evaluate and make funding decisions on DRDs.

The Environmental Scan answers the following questions:

  • How do HTA agencies review and make reimbursement recommendations for DRDs?
  • How do public payers make funding decisions for DRDs?
  • Do any of Canada’s publicly funded drug plans use a DRD-specific evaluation framework to evaluate DRD funding?

Tags

comparative effectiveness research, drug approval, formularies as topic, orphan drug production, rare diseases, technology assessment, other miscellaneous topics, Biomedical, Health technology assessments, Orphan drugs, formularies; Evaluation, reimbursement