Framework for Drugs for Rare Diseases

Drugs for rare diseases (DRD) can address significant unmet therapeutic needs for patients living with seriously debilitating and life-threatening conditions; however, the high costs of these drugs can pose challenges for public drug programs and health care systems.

There are several challenges with the application of standard health technology assessment methods for the assessment of DRD. To address these challenges, agencies and public payers have established separate or modified processes and programs to review and make reimbursement recommendations for DRD.

CADTH conducted an Environmental Scan to identify, describe, and compare how health technology assessment agencies in Canada and internationally make reimbursement recommendations on DRD. The report provides a comparison of the review and decision-making processes for agencies in Canada, the UK, Australia, New Zealand, Germany, France, and the US. The report also presents information on how selected public drug programs in Canada and internationally evaluate and make funding decisions on DRD.