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Blog Post - Towards a Canadian National Rare Disease Policy?
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Joel Lexchin
Nov 16, 2020
I think that we need to be careful when we talk about a Canadian policy for drugs for rare diseases. These types of policies can have one or more of three aims: encourage research into drugs for rare diseases, improve the regulatory environment for potential drugs for rare diseases and help people afford drugs for rare diseases. Given Canada’s position with respect to pharmaceutical R&D in general, I don’t think that it’s realistic to expect that we will be doing a great deal of R&D into rare diseases unless there is a drastic increase in the money made available through CIHR or other funding bodies and I don’t see that happening. In terms of improving the regulatory environment we can compare Canada and Australia – two countries that are relatively similar (except for the weather). Australia has had a policy for drugs for rare diseases since the 1990s. Using the US as the “gold standard” for approving drugs for rare diseases and looking at what percent of drugs approved there were approved in Canada and Australia, what we find is that both countries approved the same percent of drugs and that approval in Canada was not delayed compared to Australia. In-other-words, the lack of an orphan drug policy in Canada did not affect what drugs made it onto the market. (See my article: Lexchin J, Moroz N. Does an orphan drug policy make a difference in access? A comparison of Canada and Australia. International Journal of Health Services 2019;50:166-172.) So an Australian type policy doesn’t seem like it would make much of a difference in getting drugs for rare diseases approved in Canada. Of course, that doesn’t say anything about developing a different type of policy. It’s also interesting that of the drugs that Canada did approve, only one in five were significant therapeutic advances compared to drugs already on the market. Finally, there is the issue of helping people afford drugs for rare diseases that truly make a difference in their lives. Here the solution is not to accept the price being charged by the drug companies and then try and find the public money to pay those prices but to collectively bargain as a country to get lower prices, i.e., use monopsony buying power. Australia did that for drugs for hepatitis C and reduced the expected price from US $55,000 per patient to about US $7352 per patient, a price that meant that everyone in Australia with the disease could be treated (https://healthpolicy-watch.news/netflix-pricing-model-eases-australians-access-to-expensive-hepatitis-c-drugs/). If Canada is going to develop a policy for drugs for rare diseases it needs to be one that will benefit patients not the drug companies. Joel Lexchin MD Professor Emeritus York University
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