Canada Adds Patient Input To Drug Submission Review Process

The Government of Canada announced in a press release that it intends to launch a pilot project to include patient input during the drug submission review process. Hoffmann-La Roche Limited and Hyperion Therapeutics have agreed to be part of the Pilot Project of the Orphan Drug Framework. Canada’s Minister of Health, Honorable Rona Ambrose, announced the initiative, saying, "No one has a better understanding of what it means to have a rare disease than the patients who are affected by them. The input of these patient volunteers is absolutely invaluable, and our government has committed to making their experiences and perspectives an important addition to our scientific approach to drug assessments for rare diseases."

Pharmaceutical Company Participation

Two pharmaceutical companies will participate in the Pilot Project. Hoffmann-La Roche will include patient feedback in its submission for obinutuzumab to treat chronic lymphocytic leukemia (CLL). Obinutuzumab is an IgG1 monoclonal antibody, which selectivity binds to antigens on malignant human B cells.

Hyperion will submit an application for Ravicti (glycerol phenylbutyrate) for the treatment of urea cycle disorders. Ravicti is a liquid taken with meals, helping to dispose of ammonia in the body. The Food and Drug Administration (FDA) approved Ravicti in 2013.

The pilot will define the role of patient input and will serve as a model for future drug authorization decisions. Lorenzo Biondi, VP of Medical and Regulatory Affairs at Hoffmann-La Roche, said, "Roche recognizes that patients and patient organizations are playing an increasingly important role in drug review and health technology assessment and bring valuable experience, perspective, and insight to the process.”

Canadian Orphan Drug Initiative

Health Canada instituted the Orphan Drug Framework in 2012 to spark Orphan Drug research and invite patient participation — now through this new review process. Patients are invited to share how the rare disease affects their lives, what treatments they have access to, which symptoms are the most important to treat, and how likely they would be to try new treatments. The government health agency will be reaching out to patient advocacy groups to invite participation. For the pilot project, patients with CLL or urea cycle disorders are asked to come forward. When the Orphan Drug Framework is finalized and information from the Pilot Project is analyzed, Health Canada’s website will be optimized to receive patient feedback for use during new drug submissions.