A 91亚色 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.

鈥淭he majority of rare disease patients have no kind of pharmaceutical or biopharmaceutical treatment option,鈥� said Douglas, an associate professor in 91亚色鈥檚 Department of Science, Technology & Society. 鈥淭he science is in prime time; it鈥檚 ready to go. But you can have the fanciest science and technology and it鈥檚 not going anywhere unless these other issues are being addressed.鈥�

Through the  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.

鈥淎s a society, we鈥檙e choosing not to develop certain kinds of treatments because they might not be profitable enough,鈥� Douglas said. 鈥淥ur 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鈥檛 have to pay for them out of pocket.鈥�

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鈥檚 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鈥檚 hospitalization costs, according to the Canadian Institutes of Health Research.

Last year, Canada announced a  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.

鈥淭here鈥檚 definitely a policy window right now,鈥� 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.

鈥淎lmost all of the cases that we come up against are full of often painful stories of suffering,鈥� he said, adding that the solution doesn鈥檛 lie only with the medical scientists.

鈥淭hey need help from people looking at regulatory changes. We need the patients鈥� 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.鈥�

Courtesy of YFile

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Areeba Chaudhry - Gold Medal

Chaudhry, who majored in biomedical science, received the prestigious regional McCall MacBain and Life Science Ontario scholarships for her leadership and research experiences during her time at 91亚色. She also received three NSERC Undergraduate Summer Research Awards, and in 2022, received second prize for best oral presentation at the Faculty鈥檚 Undergraduate Summer Research Conference. On campus, Chaudhry represented the student voice as an elected member of the Science Student Caucus, served as a Peer Mentor, and was hired as a PASS Leader to create supplementary lessons for first-year biology students. In fall 2023, she will be starting medical school at the University of Toronto to pursue her dream of becoming a physician.

Sarah Pullano 鈥� Gold Medal

Pullano, who majored in biology, received the 91亚色 President's Scholarship, the Toronto Transportation Club Scholarship, and the LiUNA! Local 183 Scholarship over her four years at 91亚色. In her last year of study, she became more involved by joining the Bethune College Council, which she says, 鈥渢aught me the importance of building a sense of community during my undergraduate career.鈥� During her final semester, she began volunteering in a lab, which ignited her interest in research and inspired to pursue an MSc at 91亚色 under the supervision of Professor Nikola Kovinich in the Department of Biology.

Nicole Frias - Silver Medal

Frias, who majored in biochemistry, was the top student graduating from any program in the Department of Chemistry this year. The letters from her research mentors, with whom she held NSERC Undergraduate Summer Research Awards, highlighted how her exceptional intellect and natural curiosity enabled her to make significant research contributions as an undergraduate researcher, including co-authoring a . She also received the 2023 Canadian Society for Chemistry Silver Medal for her high academic standing.

Frias received the 2019 91亚色 award for her contributions to the campus life of undergraduate students. She also served as a Science Ambassador, which involved meeting with prospective students and parents and working on social media campaigns, and as an Orientation Leader at Bethune College. As co-president of 鈥淏reaking Stigmas,鈥� she supported her fellow students by raising awareness around mental health.

She will be continuing her research career by pursuing graduate studies in the Department of Pharmacology & Toxicology at the University of Toronto.

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