Grant: Emerging Team Grant: Rare Diseases - Health Services/GELS

As our understanding of diseases and how to treat them evolves, so too must our decision making procedures for providing fair and cost effective treatments for those living with an illness. Today, one area of policy and decision making in particular lags behind, that for treating rare diseases. There are approximately 7,000 known rare diseases worldwide, with 250 new ones discovered annually. An estimated 1 in 12 Canadians and 55 million people in Europe and the US have a rare disease. Available treatments for these rare diseases can cost more than $500,000 a year per patient, and treatment can be lifelong. Given the high cost of treatments, and the number of patients being diagnosed with a rare disease, the financial burden of treating these patients is rapidly increasing. However, there is a strong moral justification not to deny treatment because of cost, and government payers therefore face difficult choices about whether or not to provide coverage. Approving funding for a drug may mean that some other treatments or socially desirable programs (e.g. education) may not be funded. And while approval for funding may be encourage innovation and development of more new treatments; it may also be setting a benchmark against which other companies, who seek primarily to increase profits rather than help people, price their products. At present there is no policy framework to help decision makers navigate the complex factors involved when making decisions about paying for orphan drugs. This project brings together a multidisciplinary team of experts in matters relating to treatment for rare diseases, and will incorporate input from the public and key stakeholders to develop such a framework. It will facilitate priority setting for orphan treatment decisions constrained by a limited budget, that considers the relevant developmental, clinical and economic factors and ethical principles, as well as being consistent with the values of society at large.