In 2020, members of the Canadian Life and Health Insurance Association spent $650 million on drugs for rare diseases for more than 13,000 patients. Rare diseases affect one in 12 Canadians and between 6,000 and 8,000 rare diseases are known globally. In addition, roughly 80 per cent of these diseases occur in children and only about 10 per cent currently have treatments.
Many challenges exist around drugs for rare diseases, such as small patient populations with few or no treatment options, said Michelle Boudreau, executive director in the office of pharmaceutical management strategies at Health Canada, during a session at Benefits Canada’s Face to Face in Drug Plan Management Forum in December.
Drug development is uncertain with limited evidence, she noted, “which goes together, to some extent, with the small population.”
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In addition, there’s inconsistent patient access to treatments across the country, said Boudreau, and many of these drugs are very expensive, which can limit access.
A variety of health system partners are already working “to improve access and affordability for drugs for rare diseases,” she added. As well, the 2019 federal budget proposed $500 million to help Canadians with rare diseases access the drugs they need.
Health Canada carried out online engagement sessions and heard from more than 650 stakeholders, such as clinicians, patients, caregivers, the pharmaceutical industry and payers. Through the consultation process, said Boudreau, “we began to see some consensus from stakeholders.”
A consistent definition for rare disease is required, as well as an approach or framework to address the existing access challenges, she added, which can vary from plan to plan, province to province. There’s also a desire for a coordinating body for centralized, evidence-informed decision-making.
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All of the consultation’s stakeholders were concerned about the sustainability of the health-care system, said Boudreau. “We heard overwhelmingly that everybody has to play a part.”
One suggestion was a shared responsibility for costs and pooling of risk for private and public payers. As well, the consultation found some interest in innovative reimbursement models, such as managed entry agreements, where a certain amount of money is paid upfront, with a performance-based agreement going forward.
Life and health insurers indicated an interest in being part of this strategy and sharing in the sustainability, she said, noting they support a single framework to ensure consistency and predictability for payers and plan sponsors’ and would like to be included in the decision-making to inform the design and management of a potential standard formulary.
“Introduction of infrastructure would play a critical role in improving the collection of real-world evidence” to inform funding decisions and models, said Boudreau.
Read: Rare disease drugs strategy, standard coverage in CLHIA’s 2020 budget wish list
The key objectives for the rare diseases strategy that emerged from the consultation process included ensuring national consistency, collecting and using evidence, supporting sustainability and investing in innovation.
There was also considerable stakeholder support for a staged approach, with the scope of the strategy evolving over time. Boudreau said Health Canada wants to build on what it heard during the consultation and fill some gaps with stakeholder feedback as the implementation plan begins to unfold.
“Our aim is to continue to move forward with the development of a national strategy and launch it in 2022.”
Read more coverage of the 2021 Face to Face in Drug Plan Management Forum.