MaRS

Reform hope for neglected patients

The Globe ran an excellent, high profile (Page A3!) story today on the need for improvements in orphan drug policy (to treat orphan, or rare, diseases) in this country. The articles focuses on a patient with a rather unfortunate condition who cannot afford the current treatment. While that story has been written before, the new twist comes from a private member’s bill in Federal Parliament that was introduced Monday that would define rare disorders and create a fund to improve access to rare-disease treatments and research in these areas.

Unfortunately, the current economics does not work in favour of patients with rare diseases. In order for a company to recoup the hundreds of millions of dollars spent on R&D and clinical trials, the price for treatments for such small potential markets must be inordinately high. The example in the article is a new treatment for Hunter Syndrome which costs $400,000 annually.

Orphan drug policies in other jurisdictions run the gamut, from funding high-priced drugs that provide marginal improvements in quality of life (UK), to enticing producers to serve a market through incentives (US). Here at home, the funding decision is left to the provinces through Public Drug Programs.

While pharmacoeconomic decisions on the merits of various treatments can result in heart-wrenching and ethical debates, the current mishmash of policies from province to province is far from equitable.

Jurisdictionally, provinces do and will continue to have the power to choose how health care is delivered. If I were to require treatment for one of these orphan disesases, I’d hope I was living in the right province. Federal acknowledgement of the problem is a great first step. Federal support to solve this problem would go a long way towards helping those unfortunate enough to develop these regrettable conditions, who are further marginalized and under-served by today’s health care system.