Patient involvement pilot for orphan drugs launches in Canada

Canada’s Minister of Health, Rona Ambrose, says that the government has launched a pilot project targeting patient input from Canadians with rare diseases to help inform future reviews of orphan drugs.

As announced in October of 2012, the regulatory agency Health Canada has been developing an Orphan Drug Framework to spur innovation and research into new treatments for rare diseases and also to encourage patient participation. The Pilot Project will simulate how input from patients will be gathered and incorporated into the drug submission review process once the Orphan Drug Framework is in effect.

Drug manufacturers, Roche (ROG: SIX) and Hyperion Therapeutics (Nasdaq: HPTX) have agreed to participate in the pilot with two of their drug review submissions. The review of Roche's medication obinutuzumab for the treatment of chronic lymphocytic leukemia (CLL) will be used for the pilot. Hyperion Therapeutics’ review is for Ravicti (glycerol phenylbutyrate) oral liquid which is used for the treatment of urea cycle disorders. Roche and Hyperion's participation will serve as models for how patient input may inform drug authorization decisions.