Town Hall on Rare Diseases: Developing a Strategy for Rare Disease Treatments

Town Hall on Rare Diseases: Developing a Strategy for Rare Disease Treatments

The Global Reporting Centre co-hosted a town hall to wrap up six years of work carried out by the CIHR New Emerging Team for Rare Diseases and Million Dollar Meds, investigating the impact of rare diseases on families and exploring options to improve access to effective therapies.

About one in 15 people around the world suffers from a rare disease. Many of these conditions are life threatening, and the few diseases that do have treatments available often come with a steep price tag that most patients cannot afford.

So-called “orphan drugs” comprise a growing proportion of the pharmaceutical market, with the global market worth around USD $100 billion. Canada, with a universal healthcare system, is struggling to find ways to provide these critically-important medicines to its citizens. While provincial governments are usually willing to pay the high price for proven treatments, provincial and federal governments face an emerging dilemma of whether and how to cover these burgeoning drug costs.

“These issues are relevant to all Canadians,” says Peter Klein, who oversaw the multimedia portion of the CIHR project. “Even if you don’t have a rare disease, the money spent on these expensive drugs is coming from all of our pockets, and there are lots of questions about why these medicines are so costly, and how provinces ultimately decide who gets covered.”

The evening event was moderated by CBC host Paul Kennedy and recorded for an episode of CBC Ideas. Panelists included Darren Bidulka, a patient with Fabry disease, Dr. Sandra Sirrs, a clinician and medical director of the Adult Metabolic Diseases Clinic at VGH, Eric Lun of the BC Ministry of Health’s Pharmaceutical Services Division, and Millan Patel, a clinician-scientist and co-founder of the Rare Disease Foundation.

The panel engaged in a nuanced discussion about the challenges patients experience in accessing and affording expensive medications, the difficulties governments face when deciding which drugs to fund with limited resources, and the lack of transparency around prices set by pharmaceutical companies. The audience posed pertinent and thought-provoking questions looking for solutions-oriented answers from the panelists. The audience Q&A highlighted the need for greater transparency from government and manufacturers, cooperation between the key players, and support for new, innovative approaches to drug development and funding.