A York University researcher is advancing a drug initiative to give hope to millions of people around the world suffering from rare diseases for which drug therapies have not yet been developed.
Professor Conor Douglas is leading a global initiative to uncover the economic, political and regulatory barriers that prevent costly research into drugs that could treat rare diseases. The goal is to advance new approaches to drug development that could open the door to a broader range of treatments for rare diseases, not just those for more common ailments.
“The majority of rare disease patients have no kind of pharmaceutical or biopharmaceutical treatment option,” said Douglas, an associate professor in York’s Department of Science, Technology & Society. “The science is in prime time; it's ready to go. But you can have the fanciest science and technology and it's not going anywhere unless these other issues are being addressed.”
Through the Social Pharmaceutical Innovation project, Douglas is working with researchers around the world to find and advocate for changes he hopes will diversify a system that has been driven in part by profit motive and regulation.
“As a society, we're choosing not to develop certain kinds of treatments because they might not be profitable enough,” Douglas said. “Our notion of social pharmaceutical innovation is trying to understand initiatives that are tackling these problems of availability, accessibility and affordability in a way that ensures health system sustainability, supports a private insurance system that can afford to cover cutting-edge treatments and where people don’t have to pay for them out of pocket.”
Although individually they are uncommon, rare diseases as a whole are quite common, with more than 7,000 identified today, and more being discovered as science progresses. It’s estimated that up to six per cent of people around the world suffer from rare diseases. There are up to two million sufferers in Canada alone, contributing to five to 10 per cent of the country’s hospitalization costs, according to the Canadian Institutes of Health Research.
Last year, Canada announced a National Strategy for Drugs for Rare Diseases and an investment of $1.5 billion to increase access to and affordability of drugs. As part of the strategy, provinces and territories are expected to cost share through their bilateral purchasing agreements for rare disease treatments with Health Canada.
“There's definitely a policy window right now,” said Douglas, whose innovation project is looking at more ways to ensure drugs for rare diseases are brought to market.
Ideas include involving patients in research and development, teaming them with clinicians to collect data on how drugs are working outside of clinical trials in an effort to move drugs through trials more quickly. In another example, companies trying to bring a treatment to market and provincial health-care systems could enter into risk-sharing agreements as a drug is tested. One such collaboration has already resulted in approval by Health Canada for enzyme replacement therapies to treat Fabry disease, a rare genetic condition caused by a missing enzyme that can attack vital organs.
Douglas said he hopes the examples of winning approaches he and his colleagues are uncovering will offer hope to those who suffer from rare diseases, many of whom struggle to get a diagnosis and then feel hopeless when they learn there is no treatment.
“Almost all of the cases that we come up against are full of often painful stories of suffering,” he said, adding that the solution doesn’t lie only with the medical scientists.
“They need help from people looking at regulatory changes. We need the patients’ input. We need collaboration from industry, and certainly we need buy-in from Health Canada and the provincial health-care systems and pharmaceutical plans. Change is underway; a different way of doing things is possible.”