Dear DMD community, I would like to have Health Canada make its own decision, independent of the FDA, about Drisapersen and Eteplirsen and any upcoming duchenne drugs. We need all countries taking action on this, so let it be known that we want these drugs NOW. Please write to Health Canada, Rona Ambrose, our federal health minister, write to your provincial health ministers, ask your physicians to do it too, write or phone:
Genevieve Moore
Senior Advisor
Food and Drugs Act Liaison Office
Bureau de liaison pour la Loi sur les aliments et drogues
200 Eglantine Driveway - C1579 
Ottawa, ON K1A 0K9
Tel: (613) 960-1202 
Fax: (613) 957-6231
genevieve.moore@hc-sc.gc.ca

They allow us deflazacort under special access while the FDA does not, so why not one of these exon 51 skipping drugs? We have to try. Approval would be the best, but even if we could get the drugs while further studies go on and data is collected while they monitor for safety and benefits is better than the alternative: natural history, right?

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Comment by Andrew Kerr on March 4, 2014 at 9:10am

Late last year I wrote to ask about an update on the Orphan Drug Framework updates the Federal Government was promising over a year ago.  The response I got wasn't very informative:

Dear Mr. Kerr:

I am writing in response to your letter of November 20, 2013, in which you requested information about the timelines associated with the implementation of the new Orphan Drug Framework.

I would like to assure you that the Government of Canada remains committed to advancing a new Orphan Drug Framework.  As officials examine the scientific and regulatory challenges we need to address, we know that, at the end of the day, any new regulations have to be designed to make a difference in the lives of Canadians who live with rare diseases.

In your letter, you mentioned a drug for which the drug manufacturer had difficulties recruiting sufficient numbers for their Phase 3 clinical trials.  Health Canada recognizes this to be a challenge faced by many manufacturers of orphan drugs, and requirements within the new framework will account for this and other challenges specific to studying rare disease populations.  Ongoing discussions have been held with clinical researchers in an attempt to address these issues, with a goal of bringing forward a set of rules that will work for all stakeholders, especially the patient population.

Work on the new orphan drug regulations continues at Health Canada. With the Government’s introduction of Bill C-17 (Protecting Canadians from Unsafe Drugs Act) on December 6, 2012, there may be opportunities to draw upon the post-market authorities that were proposed in that legislation.  These draft patient safety provisions would tie in with the need for on-market studies for orphan drugs.  The proposed regulations for orphan drugs are targeted for pre-publication in Canada Gazette, Part I, in 2014.

I thank you for your letter and your valuable input into our regulatory development process.

Sincerely,


_____________________

Kendal Weber

Director General / Directrice générale
Policy, Planning and International Affairs Directorate (PPIAD) / Direction des politiques, de la planification et des affaires internationales (DPPAI)
Health Products and Food Branch (HPFB) / Direction générale des produits de santé et des aliments (DGPSA)
Health Canada / Santé Canada
Édifice Graham Spry Building, Room/pièce 451 (AL: 2004A)
250, avenue Lanark Avenue   Ottawa, Ontario  K1A 0K9
Tel.: 613.952.8149  BB: 613.240.8086 Fax: 613.954.9981
email: kendal.weber@hc-sc.gc.ca

 

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