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Patient Advocacy: Canada’s Patient Involvement Pilot Program To Help Orphan Drug Reviews

August 8, 2014

This is the sixth Blog Post in a series that examines the influence and power of patients and patient advocacy groups  in helping and influencing  the different stages of orphan drug developement.

Since October of 2012 Health Canada has been in the process of implementing orphan drug regulations. On August 6th, Rona Ambrose, Canada’s Minister of Health, announces the launch of a pilot project “ … targeting patient input from Canadians with rare diseases to help inform future reviews of orphan drugs …. 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.”

Two drug manufacturers agree to participate in the Pilot Project with their drug review submissions :

•   Hoffmann-La Roche Limited’s drug Gazyva (Obinutuzumab) for Chronic Lymphocytic Leukemia (CLL)

•   Hyperion Therapeutics’ drug Ravicti (Glycerol Phenylbutyrate) for Urea Cycle Disorders (UCDs).

Patients are asked for input on the following topics about their rare diseases:

•   How does it affect their daily lives

•   What are the current available treatments

•   What therapeutic benefits are most important

•   What is their risk tolerance for new treatments.

Patient advocacy groups, like CORD (Canadian Organization for Rare Disorders), will assist Health Canada in engaging Canadian patients with CLL or UCDs to participate in the Pilot Project. The feedback from patients involved in the Pilot Project will help Health Canada evaluate how to best collect patient input, which will ensure that their opinions and feedback are considered in future orphan drug authorizations.

Rona Ambrose concludes that:

“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.”

Orphan drug Ravicti is approved by the FDA in February 2013 and is currently marketed in the US. In June 2014, the European Medicines Agency (EMA) accepts for review the Marketing Authorization Application (MAA) and Health Canada accepts a New Drug Submission (NDS) with Priority Review for Ravicti.

Orphan Drug Gazyva (Obinutuzumab) is the first medicine approved (November 2013) with the FDA’s coveted Breakthrough Therapy Designation (BTD). Just last month, the European Commission approves Obinutuzumab in combination with Chlorambucil chemotherapy for CLL.

Please Note : “Kid Hugging a Rainbow” by Marendo Müller, artwork (Own work) [Public domain] | Wikimedia Commons.

Copyright © 2012-2014, Orphan Druganaut Blog. All rights reserved.

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