It’s important for private payers and employers to be nimble and innovative to support plan members seeking access to novel drugs and treatments by addressing common hurdles within their own drug plans, said John Adams, chair of the board of directors for patient advocacy group Best Medicines Coalition, during a session at Benefits Canada’s 2023 Face to Face Drug Plan Management Forum in December.

There are currently 791 medicines in development for the treatment of a host of rare diseases — including genetic disorders like cystic fibrosis, neurological disorders like amyotrophic lateral sclerosis, autoimmune diseases and rare cancers — that could transform the lives of plan members with those conditions.

Read: Highest-cost drugs reimbursed by Canadian public plans in 2021/22 were rare disease treatments: report

Adams’ advocacy work was motivated by his son, who was born with a rare disorder called phenylketonuria. If not treated early in life, the condition can lead to intellectual disabilities and other major health problems. After entering a clinical trial in 2007, the biomarker in his son’s blood dropped 90 per cent and for 13 years, he was able to receive medication for free through an orphan drug development company, then later through his workplace benefits.

Adams also became a caregiver for his late wife when she began experiencing neurological symptoms and was also diagnosed with metastasized breast cancer. After her death, she was diagnosed with amyotrophic lateral sclerosis and frontotemporal dementia, two conditions for which there are no disease-altering medications.

He said members of private plans commonly experience process delays for coverage of new drugs, with plans requiring “every ‘T’ crossed and every ‘I’ dotted” before a new medicine is allowed to be accessed, as well as restrictive criteria for what’s covered.

Read: Feds name advisory group for national strategy for drugs for rare diseases

One solution for private plans is to adopt a policy similar to Quebec’s independent advisory committee for exceptional circumstances: when a patient needs a particular drug that’s not on the provincial formulary or not yet approved by Health Canada, the committee reviews the circumstances and makes a binding decision. 

He said weight management therapies are among plan members’ unmet needs. While weight management drugs have historically been categorized as lifestyle drugs and not covered by plans, Adams pointed to research that has found both Ozempic and Wegovy have demonstrated not only weight loss but “huge cardiovascular benefits as well.”

He called on private plans not to base their formularies on Canadian Agency for Drugs and Technologies in Health recommendations, as the agency has been using the “shaky, untested premise” that a quality adjusted life year is worth $50,000 for more than 20 years, without adjustment for inflation or rarity or severity of conditions. He also argued the agency’s mandate is to serve the needs of provincial drug plans rather than plan sponsors.

Read: Sounding Board: How employers can better support employees with rare diseases

“It’s not about serving the needs of employers and their employees and dependants who want access to better health care and to keep those valuable, talented employees on the job and highly productive.”

With drugs coming down the pipeline that, while highly expensive, would deliver a long-term benefit for the plan member in a single course of treatment, Adams said private plans should be thinking about how to pay for these medications. He likened paying for these high-cost medications to taking out a mortgage. “When we’re talking about an intervention that’s going to have multi-year benefits, we need to put our heads together about how to pay for it over multiple years.”

Read more coverage of the 2023 Face to Face Drug Plan Management Forum.