Government decisions to fund expensive treatments for rare diseases shouldn't be made on an ad hoc basis that leaves countless patients out in the cold, says a new analysis published Monday by the Canadian Medical Association Journal.
Canada needs a new system that would allow policy makers to measure the effectiveness of treatments while connecting rare-disease patients with much-needed drugs, wrote Chaim Bell, co-author of the analysis and associate professor of medicine and health policy management at the University of Toronto.
The issue of rare diseases and access to treatment has been brewing for years. Canada has long been criticized by rare-disease patients and health experts for not having a formal policy that allows access to "orphan drugs," or those that are used to treat rare diseases. It's one of the few developed countries that doesn't have one.
Without a formal policy, there is too much red tape and not enough financial incentives for drug companies to obtain approval for these products in Canada because the market is relatively small. As a result, patients may have no way of getting certain drugs or have to pay exorbitant amounts for them.
In recent years, a growing number of patient advocates, as well as physicians and experts who study rare diseases, have been urging the federal government to establish an orphan-drug policy.
But it's a challenge on many fronts because the costs of orphan drugs are often so high and the evidence of benefit for patients can be difficult to assess. Unlike other treatments, such as cholesterol or cardiovascular drugs, which are tested on a wide number of people before being introduced to the population, the very nature of orphan drugs means there are few people to test them on.
As a result, decisions to fund drugs for rare diseases are often made on a patchwork basis and driven by patients with the loudest voice who take their cases to Parliament Hill or the media.
Dr. Bell argues the obstacles involved in treating rare diseases can be overcome if the federal government is willing to create a new system that takes into account the unique nature of orphan drugs. Instead of applying the same criteria that's used to evaluate conventional drugs to treat common diseases and disorders, orphan drugs require a new framework that's based on the best available evidence and consistently applied across the country.
"It's not just stepping in saying we need to fund these drugs because I think that's the simplistic approach," Dr. Bell, who is also a physician and scientist in the Keenan Research Centre of the Li Ka Shing Knowledge Institute of St. Michael's Hospital in Toronto, said in an interview.
He said there are several elements Canada should incorporate in a comprehensive orphan-drug policy, such as:
*Develop a standard measure that dictates when a disease is considered "rare" based on its prevalence in the population. Creating a standard definition would help officials identify the number of rare diseases there are and help guide decisions on drug access.
*Create patient registries that can track the incidence of rare diseases in the population, their progression over time and the effectiveness of treatment. Such a move could help identify side effects or other drug reactions, as well as be added to data on a drug's efficacy.
But one of the most important long-term moves Dr. Bell advocates is for the creation of federal legislation to reimburse patients for orphan drugs and provide incentives for the development of new drugs to help people with rare diseases.
Lorne Clarke, professor of medical genetics at the University of British Columbia and head of the genetics and health research cluster at the Child & Family Research Institute said Dr. Bell's analysis sends a strong message that many experts in the rare-disease community have been trying to get across for years.
"We need to be able to now establish a method which is going to allow for these patients to have access to therapeutics," he said.
Carly Weeks