ORPHAN DRUG POLICY FOR CANADA

                                                                                                                          ~ ann gloyn, education specialist

WHAT IS AN ODP?

An orphan drug policy is a guideline which helps provide opporunties for treatments for rare disorders in Canada.  It can also be a social program, which will help with funding to enable individuals affected by rare disorders to obtain access to treatments that have been deemed safe and effective.

Families need to have the right for prompt and accurate diagnosis; access to physicians with knowledge about thier rare disorder; the benefit of a rare disorder data base; and to have the rare disorder understood by sectors of health, education and by the provincial and federal government. 

A data base needs to be supported by the federal and provincial governments so that families looking for information will not only have this accessible, but also it will give them the opportunity to be aware of research.  It will also be able to match Pharma's, Bio-techs, teaching institutions, and so on with population groups to study.  Tax incentives need to be given to those interested in the study of rare disorders.  A rare disorder registry needs to be managed and funded centrally, and contain statistical information on the rare disorder; demographics about the populations; specifics for diagnosis and treatment; listing of medications; and information updated on drug trials.

WHAT COULD BE THE EFFECTS OF ADVOCATING FOR AN ODP IN CANADA?

• earlier diagnosis
• access to research
• timely and effective treatment

WHAT COULD THIS MEAN DIRECTLY FOR FAMILIES?

It could mean a mandate to fund families for the following:

• medications
• therapueutic products
• biotech products
• medical devices
• radiopharmaceuticals
• related travel expenses

UPDATE ON ADVOCACY AND GOVERNMENT RESPONSE

In October, a media briefing was held with patient groups from across Canada before a schedualed meeting of Federal, Provincial, and Territorial Health Ministries in Toronto.  The critical issues at hand with the funding for Fabry and MPS 1 (Hurler-Scheie) patients who will die without treatment, has brought the issues for an ODP into the headlines. 

There has been an ongoing battle over access to enzyme replacment therapy (ERT). Genzyme is the company who has been distributing the drug, but there has been no promise of continued funding from government. For example, in Ontario, the clinical trials for children with MPS 1 ended, and the Ontario government refused to provide further treatment.    

The announcement from the Health Ministers after their meeting was that they were "committed to putting something in place where drugs for rare diseases would be funded while ongoing studies take place".  Genzyme agreed to continue to provide the funding for the clinical trials in Ontario until such time as the logistics of the funding mechanism could be worked out. 

HOW CAN WE ADVOCATE NOW?

There is a letter writing campaign which is ongoing.  Please help keep these issues around the development of an ODP on the front line with the Ministry of Health. Write to:

The Honourable Jjjal Dosanjh MP

Minister's Office - Health Canada

Booke Claxton Building, Tunnney's Pasture

Postal Locator: 09060

Ottawa, ON

K1A 0K9

CHARGE Syndrome Canada Newsletter Christmas 2005 <back to newsletter>