Patients reveal thoughts on high-cost drug strategy

Patients and their families said they want more transparency about how drug pricing for rare diseases works.

Dave Yasvinski 4 minute read July 27, 2021
drug costs rare diseases

Canadians with rare diseases are hoping for more transparency about drug costs. GETTY

Canadians coping with rare diseases said the creation of a single framework that ensures equal access to innovative and expensive new medications must be a main component of the national strategy Health Canada is in the process of building.

Patients with rare diseases, their families and other vested stakeholders received an invitation from Health Canada earlier this year to respond to a report intended to spur debate on how to better provide access to costly drugs. The report, entitled Building a National Strategy for High-Cost Drugs for Rare Diseases: A Discussion Paper for Engaging Canadians, notes that there were 93 drugs approved for rare diseases in the country as of 2019 that cost more than $100,000 per patient per year. Half of them surpassed the $200,000 mark.

After rolling out five virtual public town halls and 16 virtual meetings, and poring over the results from an online questionnaire and email submissions, Health Canada is revealing the results of its outreach. On the issue of access to medication, patients said they were frustrated by the “postal code lottery” that often governs which drugs will be covered by certain provinces or territories. Respondents favoured the creation of a transparent coordinating body that would offer patients better access and input into the country’s rare drug strategy going forward. Some patients said they experienced gaps in coverage between private and public healthcare plans based solely on where they live.

“Because of the small numbers of people with rare diseases, meeting the usual standard of evidence for drug approvals is rarely possible,” Health Canada said in a summary of the input it received. “Many participants said rare disease drugs need to be assessed differently than drugs for common diseases. There were calls for patients and their caregivers to be involved in defining what constitutes a ‘benefit’ or ‘improvement’ when treatment outcomes are assessed.”

Respondents overwhelmingly favoured innovative approaches to the approval process for new drugs, such as the implementation of a pay-for-performance system that would fund medications based on the results they deliver. They would also like to see a process in place to fast-track drugs that have been approved in other countries and to have more involvement on the panels that decide which drugs qualify for approval and coverage.

“Many people felt the emphasis on the high cost of drugs overlooked their value for patients, the health system and society as a whole,” Health Canada said. “Generally, participants felt there is a need for more transparency in how drug prices are set and suggested governments should work together to lower prices. In considering options, most people felt better cost-sharing and pooling of risks was the best approach. Some felt the federal government could reinsure drug plans by paying costs for drugs over a specified dollar threshold.”

Patients would also like to see the government invest more heavily in Canadian research and development, bringing more clinical trials to the country and offering access to the latest research and treatments.

Indigenous respondents, who often have a very different experience with the healthcare system, said the new framework needs to prioritize building trust and reducing racism throughout the network. “Indigenous participants and many other stakeholder groups want transparency in decisions on an individual patient’s eligibility for rare disease drug funding, including an appeal process where patients denied funding have an opportunity to challenge the decision,” the report said.

Health Canada said it plans to continue consultations with the public and the provinces and territories throughout the summer to inform the creation of a comprehensive model that works for all Canadians. The Canadian government intends to launch its national strategy for drugs for rare diseases in 2022.

Dave Yasvinski is a writer


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