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opinion: a better future for 1 in 12 canadians starts with rare disease reform
4 minute read
jori fales with daughter charleigh pollock, 9 in this photo, who has a rare neurodegenerative condition known as batten disease.
jori fales
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for families impacted by rare diseases, “devastating experience” is an understatement. from diagnosis shock to heartbreaking knowledge of an existing treatment not yet available in canada, every step is fraught with uncertainty.
charleigh pollock’s family lived this reality when the british columbia government initially decided not to reimburse her therapy for batten disease, a rare disorder robbing children of sight, mobility and, ultimately, life. following relentless patient and medical community advocacy, the decision was reversed, granting her access to life-sustaining therapy. pollock’s story is proof progress is possible, but for too many, treatment access delays are time they will never get back.
rare diseases are defined as affecting fewer than one in 2,000 individuals; but with over 7,000 such conditions, one in 12 canadians live with one. while some are treatable, accessing treatments requires negotiating convoluted processes with the knowledge that delays may cause irreversible damage.
getting diagnosed typically involves almost four years of misdiagnoses, multiple clinic visits and unnecessary procedures. but diagnosis does not guarantee treatment access. according to the canadian organization for rare disorders, only 60 per cent of treatments for rare disorders are available in canada; most are approved up to six years later than in the u.s.
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pricing and reimbursement discussions can last years, partly because the traditional evidence standards of canada’s drug agency don’t fit realities of rare disease trials. another persistent challenge is time taken by public jurisdictions to list drugs for rare disorders, even after pan-canadian pharmaceutical alliance (pcpa) negotiations have successfully concluded.
although federal government implementation of a $1.5-billion national strategy for drugs for rare diseases alongside a $33-million boost to the non-insured health benefits program in 2023 improved support for rare disease therapies, each province is only required to reimburse one drug in the first two years. this risks creating an inequitable patchwork of provincial access. however, ontario’s funding accelerated for specific treatments program for oncology provides a blueprint for rapid implementation of a canadian solution—more proof progress is possible.
inspiration from global benchmarks
throughout our careers, in positions in canada and abroad, we’ve seen what is possible when governments, clinicians and industry collaborate to ensure innovation reaches patients faster.
in 1983, the u.s. government passed the orphan drug act, introducing powerful incentives such as tax credits for clinical research, grant funding, assistance in clinical research design and seven years of market exclusivity. the eu passed similar legislation in 2000, leading to high reimbursement rates. australia’s life saving drugs program broadens access to high-cost drugs intended for treating rare diseases. brazil’s rare diseases strategy allows for priority regulatory and pricing reviews, and a timeline of approximately 450 days following regulatory approval to public reimbursement, compared with an average of 730 days in canada.
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and let’s be clear: these initiatives work. in a 2022 analysis, germany (84 per cent) had the highest proportion of orphan drugs approved by the european medicines agency that were reimbursed, with median time from approval to reimbursement being 4.1 months. in that analysis, ontario was the province with the highest proportion of publicly reimbursed drugs (32 per cent) and british columbia had the shortest median time from regulatory approval to reimbursement (17.3 months). this disparity does not reflect well on canada; we can and must do better.
vision 2030: from patchwork to progress
potential roadmaps toward a rare disease framework in canada that achieves universal, equitable healthcare for all are ready to implement, like the canadian rare disease network strategic plan. steps outlined in that plan could help make rare disease care in 2030 look much different than it does today, including:
- diagnostic transformation: leverage existing $1.5-billion funding to deploy ai-driven tools to shorten the “diagnostic odyssey,” and build on current registry initiatives.
- faster reimbursement: streamline approval-to-reimbursement processes to fit within 12 months.
- legislative reform: adopt eu definition of rare diseases and develop a national rare disease drug framework including incentives aligned with u.s. and eu strategies, ensuring at least 90 per cent of globally approved therapies are submitted in canada.
- equity by design: create provincial rare disease hubs by 2029, with mental health and financial supports built in, eliminating the “postal code lottery” via standardized reimbursement criteria.
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stories like charleigh pollock’s show what becomes possible when families fight for what should have been done from the start. but that shouldn’t be necessary. for one in 12 canadians living with a rare disease, waiting is not an option. canada must commit to and act on a framework that delivers equitable, timely access to lifesaving treatments for canadians with rare diseases.
rodrigo reis is the general manager of ucb canada inc. with more than 25 years of international experience in the pharmaceutical industry, having led teams across brazil, argentina, the u.s. and now canada, he brings a global perspective to advance practical and innovative solutions in rare diseases.
chander sehgal is the head of access, sustainability and external engagement at ucb canada inc. an oncologist with more than 20 years of experience in both private and public biopharmaceutical sectors, he has held leadership roles within major pharmaceutical companies and national health agencies, including cadth (now cda-acm) and innovative medicines canada.
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