Globe and Mail
Jul. 17, 2008
In 1983, U.S. President Ronald Reagan signed into law the Orphan Drug Act, one of the most significant pieces of drug legislation ever.
Twenty-five years later, Canada remains one of the only developed countries that has not followed the lead of its neighbour to the south and foster drug development to help those with rare diseases.
As a result, millions of Canadians are unable to access potentially life-saving medications and treatments in a timely and coherent manner.
This legislative inaction beggars belief. The foot-dragging not only offends the principles of medicare, it makes no business sense.
So let’s be thankful for the dogged determination of Don Bell, member of Parliament for North Vancouver, who crafted the private member’s motion M-426 calling on the government to “respond specifically to the challenges faced by Canadians with rare diseases and disorders.”
And a tip of the hat to his fellow MPs for putting aside partisan politics and approving the motion, which, while vague, should light a fire under the collective backside of federal and provincial governments.
Rare disorders are those that affect fewer than one in every 2,000 people. There are an estimated 7,000 such diseases in Canada and three in every four touch children principally. All told, these “orphan” diseases affect close to three million Canadians to varying degrees.
While there are scores of drugs for those with cardiovascular ailments, a bevy of antidepressants and antibiotics, and more anti-impotence treatments than the world will ever need, there is a paucity of medications for pulmonary hypertension (the rare disorder that claimed the life of Mr. Bell’s grandson), cervical dystonia, hairy cell leukemia and so on.
There is little incentive for drug companies to do research on possible treatments for conditions that affect dozens or hundreds of people. Even where there is a desire, it can be virtually impossible (not to mention prohibitively expensive) to recruit enough patients to conduct a proper clinical trial to test the efficacy of a drug.
The U.S. Orphan Drug Act addressed these challenges by providing a series of financial and other incentives, including tax credits and subsidies for clinical trials involving orphan drugs, granting seven-year exclusive marketing to help companies recoup expenses and waiving drug registration fees that cost companies about $1-million (U.S.) for each product.
The results have been remarkable. In the decade before the Orphan Drug Act was adopted, only 10 drugs were developed to treat rare conditions. Since the law was passed, 326 new treatments have been introduced.
These drugs all go through the regular approval process, so they work as well as any other prescription drug, but they are essentially subsidized so they can get to consumers.
Canada, thankfully, has been able to piggyback on many of these findings.
While patients here have benefited, Canada is not pulling its weight on the research front, and governments have missed a golden opportunity to partner with industry to address a pressing social issue.
Worse yet, access to orphan drugs in this country is poor at best and horribly uneven. For example, Hunter syndrome (also known as MPS II), an enzyme disorder that affects only about 40 people nationwide, can be treated effectively with a drug called Elaprase. That drug, which is covered by drug plans is Alberta and British Columbia, is not covered in Ontario, so the six sufferers in that province are out of luck.
The drug, which needs to be infused weekly, costs about $400,000 a year.
The reality is that orphan drugs are costly.
The United States, it must be said, doesn’t do so well in helping consumers pay for orphan drugs. But Britain, with a national advisory group and funding to subsidize rare and expensive treatments, is a model.
In the 2003 federal-provincial health accord, Canadians were promised a National Pharmaceuticals Strategy, one whose cornerstone is a plan to protect Canadians against catastrophic drug costs.
Five years later, the NPS remains a good idea mired in a bureaucratic bog, and millions of Canadians are suffering unnecessarily – financially, physically and emotionally – as a result, and none more so than those with rare disorders.
The Canadian Organization for Rare Disorders has made a series of recommendations that would largely solve the problem, including:
Establish a national “Chance for Life” fund equivalent to 2 per cent of the total annual public drug spending and designated for therapies for rare disorders.
Create an advisory body to review access to treatments for life-threatening or serious rare disorders based on scientific standards and social values.
Provide incentives to researchers and manufacturers similar to those in the U.S. Orphan Drug Act, including supporting clinical trials.
This is not a difficult issue for Canada’s health ministers to solve, and it should be near the top of their agenda when they meet in September.
Twenty-five years of laggardness is quite enough