Jennifer Adams
Family Physician in Ottawa
Facing rare disease challenges, a family calls for change to ensure affordable, life-saving treatments for all.
I am Jennifer Adams, a family physician in Ottawa, and this is my family’s story. Our journey began when my daughter, now five, was diagnosed at 18 months with primary hyperoxaluria type 1 (PH1), a rare and life-threatening genetic condition. It felt like we were handed a rickety boat and told to cross the ocean. Without treatment, our daughter faced a terrifying future of excruciating kidney stones, irreversible kidney damage and the looming need for dialysis and kidney and liver transplants or even death.
A beacon of hope
Then came lumasiran (Oxlumo). This life-changing medication became our luxury liner, offering safe passage across treacherous waters. My daughter’s quality of life improved, reduced hospital visits and no doubt has delayed and more likely eliminated the need for costly dialysis or transplants in the future. My husband was able to return to work and we began to live a “normal” life again. It’s a journey only available to a fortunate 5 per cent of rare disease patients, as 95 per cent of rare diseases have no effective treatments. We are eternally grateful this miracle drug exists.
This security, however, is threatened by the field of icebergs of drug affordability. With a list price of $204,626 per dose, every three months, lumasiran is a passage most cannot afford. We are among the fortunate few that have private insurance that covers lumasrian, but our passage is precarious. While the drug developer has verbally agreed to cover our insurance’s 10 per cent co-pay, approximately $20,000 per dose, this agreement is not legally binding or enforceable. At any moment, this massive wave could crash down on our family, leaving us struggling to stay afloat.
Trapped by the system
We are trapped in a state of fear that we will be thrown overboard, and our child’s life-saving medication out of reach. Our private insurance will likely reach its lifetime limit in 2025 and we will be forced to rely on the ill-equipped life rafts of the public system. But we won’t be allowed on board. Although our private insurance won’t cover the cost of lumasiran, our daughter is ineligible for the lifeboat of OHIP+ and its crucial zero-deductible coverage. Instead, she can only access the Ontario Trillium Drug Plan, a poorly designed life vest, squeezing us with an additional 4 per cent deductible of our household income on top of our private insurance deductible and no funds left to access medications on our private drug plan. Our family can weather this storm, but what about other families not as fortunate? What happens when the there is a “cost put on our child’s head” by the province and it pulls funding of a rare disease drug like the recent case of Charleigh Pollock in BC.
Ensuring equitable access
The recent announcement of the signed bilateral agreement between Ontario and Health Canada on the National Strategy on Drugs for Rare Diseases offers a glimmer of hope on the horizon, but the details remain shrouded in darkness. We implore both the Federal and Ontario governments to chart a new course. Implement a comprehensive National Rare Disease Strategy that recognizes the unique burdens faced by rare disease patients and families and join all other developed countries in having a national rare strategy. Ontario must accept its obligation to protect its most vulnerable citizens and eliminate the financial barriers of premiums and deductibles for rare disease treatments. As a taxpayer, I expect Ontario to negotiate a substantial discount from the list price of lumasiran. Something I can’t do as an individual.
Our daughter, and all rare disease patients, deserve a reliable, sturdy lifeboat, not stranded to face the turbulent waters alone. We urge both the Federal and Ontario governments to act before another family is left to drown.
