They may be rare, but they affect a large number of us. One in 12 Canadians suffer from one of 7,000 different rare diseases. “One rare disease only affects a small portion of the population, but as a category these diseases affect a huge number,” says Larry Lynd, associate professor in the Faculty of Pharmaceutical Sciences at UBC.
Lynd is leading a team of researchers that has just received a $1.5 million grant from the Canadian Institutes of Health Research (CIHR) to investigate potential funding policies for treating rare diseases.
For the past several years, he has been part of the BC Ministry of Health Expensive Drugs for Rare Diseases Advisory Committee—the group that approves funding for treatment on a patient-by-patient basis. “In determining treatment for rare diseases, we can’t use the same decision-making structure that we use for common diseases,” Lynd says.
Treatment options for rare diseases are limited and can cost more than $850,000 per year for just one patient —far above average drug costs for common diseases. When reviewing cases, the committee is often faced with tough decisions, especially in cases when the drugs offer little certainty of patient benefit or improvement.
With their project, Lynd and his team of bioethicists, health policy specialists and other experts are asking the most provocative question: How much public funding should be spent on drugs for rare diseases? “Nobody has ever tried to look at what society’s willingness to pay is, but I think we’re willing to pay a premium on rarity,” Lynd says.
In addition to examining the budget that should be allocated to these drugs, the research team is working on sustainable drug payment policies and incentives for pharmaceutical firms to develop lower cost alternatives. “Ultimately, we’re doing this to create a structure within Canada that will allow us to support treatments for these diseases,” Lynd says.
Lynd is convinced of the relevance of this research, not only for provincial and national policy makers, but also at the international level. “Everyone will be watching to see how we can create cost-effective and sustainable policies to get these drugs to the people that need them,” Lynd says.
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