Focus on Accelerated Access to Drugs for Rare Diseases
April 8, 2026
For people with rare diseases, timely access to medications remains a challenge. Patient advocacy groups and individual advocates like Beth Vanstone are helping to ensure accelerated access initiatives include rare disease drugs
The “rare disease” category is a bit of an oxymoron. While each individual disease under the umbrella occurs infrequently, collectively these disorders impact one in 12 Canadians, making the designation all but rare.22 Fortunately, the category is gaining prominence in the political and public sphere – including in the new Ontario FAST program. “We’ve just seen the first rare disease drug, Voranigo, funded by Ontario’s FAST program,” says Durhane Wong-Rieger, President & CEO of the Canadian Organization for Rare Disorders (CORD). The drug, she explains, is “for a rare type of brain cancer, so this is amazing. It fits into the cancer bucket, but we feel other ‘rare non-cancer’ drugs could also qualify for this type of access as well.” 15
With that in mind, and with new accelerated access pathways now in place in Canada for oncology drugs, we check in with Beth Vanstone, who has been advocating for patients with cystic fibrosis (CF) since her daughter Madi was born with the disease more than two decades ago. After successfully lobbying for funding of the life-changing medication Kalydeco, Beth continues to speak for rare disease patients facing treatment access barriers. In this Q&A interview, Beth offers an advocate’s perspective on new developments in rare-disease drug access and underlines the need to keep the momentum going.
How did your personal experience inform your work in access advocacy?
When Madi enrolled in a clinical trial for Kalydeco, we knew within 24 hours that she was in the treatment group – that’s how dramatic the change was. We assumed we how had a solution for her illness, but we soon discovered that we couldn’t access the drug beyond the clinical trial period. So I began to lobby for access and discovered that other patients and their families were facing similar challenges. This inspired me to advocate for policy changes to support better access to treatment for people impacted by rare diseases.
Has anything significantly changed in the past five years for rare disease patients?
I do see things changing, albeit very slowly – and as we all know, time is often not a luxury for patients with rare diseases. The introduction of a national rare disease strategy and signing of bilateral agreements with the provinces and territories were wonderful first steps, but their long-term impact on the system is not clear yet. While we are seeing some tweaks in processes that may save a couple of months here and there, our overall approval and reimbursement system was not designed to deal with therapies for rare-disease populations. These populations are too small to provide the data required by the current system. Looking around the globe, we can see other countries doing it better. Instead of simply looking to speed up access within existing pathways, we need to take action on the reimbursement pathways that are working in other jurisdictions.
What is your vision for the national rare disease strategy?
Ideally, it should include expanded newborn screening, expedited pathways for therapies, and centres of excellence for care. Such measures will help reduce the impact of disease through early diagnosis and treatment, keep patients out of hospital emergency rooms, and improve outcomes on an individual and health-systems level. With policies that recognize and support their unique needs, rare disease patients and their families can live much better lives. Science and innovation have moved forward in leaps and bounds, and by investing in it we can ensure better outcomes for patients and their families, and a positive impact on government budgets. These investments will serve to assist in quicker diagnostics, ensuring patients receive the care and innovative therapies for their condition in a timely manner.
What are your thoughts on Ontario’s new FAST program, which enables earlier public funding of some cancer drugs?
It’s a great start and wonderful for oncology. There is no reason we can’t use this same pathway for rare disease drugs, which share many of the same features as cancer drugs – rare disease patients and cancer patients both have an urgent need to access these therapies. In my work, I see the fear, frustration and often devastation while patients with rare diseases and their families wait for life-changing drugs to grind through a system not designed for them. Even the drugs that get approved often don’t make it to reimbursement, which is effectively a death sentence for many Canadians living with these diseases. I know we can do better.
What message might you have for the architects of the FAST program?
With FAST under way, we have an opportunity to apply that momentum beyond oncology. The question we should be asking is: Which areas of unmet need could benefit most from a FAST-style pathway, and what will it take to get there?
“We have an opportunity to apply the FAST strategy beyond oncology. The question we should be asking is: Which areas of unmet need could benefit most from a FAST-style pathway?”
You recently co-founded Ontario Rare Action Group. What is it and why did you start it now?
Alice Williams, the mother of two adult children living with Wilson’s disease, and I are working on this initiative together. After attending many amazing CORD [Canadian Organization for Rare Diseases] conferences, I learned that patients wanted to play an active role in promoting change. We felt it was important to unite our “rare voices” and convey how patients and their families would benefit from a rare disease strategy, in Ontario specifically.
Although rare disease symptoms vary widely, we all face the same systemic barriers, which include a lengthy journey to diagnosis, the challenge of entering drug trials, and the long and sometimes deadly wait to access treatment. From a health-economic perspective, faster diagnosis and quicker access to therapies could reduce unnecessary testing, repeated specialist appointments, and time in hospital. We are inviting patients in the rare disease community to lobby their MPPs and to have them all join us for a breakfast at Queen’s Park on May 13, to show that “rare is not rare when united.”
“Although rare disease symptoms vary widely, we all face the same barriers to diagnosis, entering drug trials, and accessing treatment.”
Can you identify one key item that would have a significant impact for rare disease patients in Canada?
I would love to see our patient community unite our voices. If we can get patients to make the call to their MPP, get that letter sent, attend that meeting, we can explain what we need and how it will benefit our community. Advocacy is what moves barriers.
References
15. Rare Disease Day 2026 webinar. Canadian Organization for Rare Diseases. February 27, 2026. https://www.youtube.com/watch?v=u5pOZKJGI7U
22. Strategy & Access. Canadian Organization for Rare Disorders. https://www.raredisorders.ca/work/strategy-access