Canada seeks patient input for orphan drug reviews

Canadian Minister of Health Rona Ambrose announced the launch of a pilot project that solicits input from patients with rare diseases to guide future reviews of orphan drugs. Canada is in the process of implementing orphan regulations since a designation was authorized in 2012.

Patients will be asked how their disease affects their daily lives, what treatments are currently available, what therapeutic benefits are most important to them, and what their risk tolerance is for new treatments. The pilot project will simulate how input from patients will be gathered and incorporated into the drug review process once the Orphan Drug Framework is in effect. ...