Drugs for Rare Diseases: National Pharmacare or Separate Program?
FORMAT: Panel Debate with Discussion
Drugs for Rare Diseases or other “targeted” small patient populations should be assessed using the same processes and “cost-effectiveness” thresholds as drugs for more common conditions. Moreover, small and short clinical trials mean many of these drugs are approved with limited evidence of safety and effectiveness; access should be restricted until better data are available (from extended clinical trials or real world evidence).
Canada should expand upon the learning from its own experiences as well as programs of other countries to develop specific expedited pathways for approval and access to drugs for small patient populations with severe, progressive, and/or life-threatening diseases for which there are very few or no other viable treatments.
Some rare disease patients in Canada have had their lives extended or vastly improved thanks to various “managed access” programs for conditions, such as Fabry’s Disease, Cystic
Fibrosis, and Pulmonary Arterial Hypertension. A National Pharmacare program should use the learning from these specialized programs to develop a “managed access” scheme with registries and post-market monitoring, which would immediately be applied to most rare disease drugs as soon as they are approved by Health Canada. Patients with rare disease are willing to take an active role in assuring appropriate access, adherence to usage as prescribed, and participation in registries and monitoring.
Information on how to register for the webinar and other details will be announced shortly.
This is one of six webinars in a National Pharmacare Debate series hosted by CORD.
For the complete list of webinars in this series, click here.