A Relentless Advocate: Fighting for Rare Disease Coverage
For families living with rare disease, access to treatment often comes down to a single word: no.
In Canada, one in every 12 people is affected by a rare disorder, most of them children, yet limited research investment and complex regulations frequently delay access to treatment. When therapies finally reach the market, families often face another harsh reality: cost. Without coverage, rare disease treatments can range from $100,000 to more than $2 million per patient, per year.
With stakes this high, families can’t afford to accept a drug coverage denial, yet they hear it far too often. In those moments, they need a knowledgeable, persistent advocate. That’s where BioScript Solutions thrives. For our reimbursement specialists, a denial is never an end point; it’s the moment advocacy begins.
“Rare disease drugs are expensive, and insurers apply strict eligibility criteria,” says Rihab El-Ali, Patient Support Program Manager. “The initial submission only paves the way. We expect appeals, and we’re prepared for them.”
BioScript operates seven dedicated rare disease Patient Support Programs, navigating one of the most complex coverage landscapes in healthcare. “Our specialists become subject matter experts in [the reimbursement landscapes of] each province, so we can move quickly and avoid unnecessary back and forth,” El-Ali explains.
BioScript’s rare disease programs track patient-reported outcomes and quality of life improvements to demonstrate the real-world impact insurers look for. Our teams also built a centralized knowledge hub with proven templates, appeal letters, and best practices, helping cut timelines from eight weeks to as little as two, depending on the insurer.
Beyond drug coverage, our rare disease specialists want families to know they can—and should—ask for more. Through research and collaboration with patient advocacy groups, we secure funding for mental health services, mobility aids, physical therapy, employment services, and more. When private coverage falls short, we leverage provincial grants, tax credits, and community programs.
“We know every patient is different, so we customize our services to individual needs,” says Rihab.
In rare disease coverage, “no” doesn’t have to be the final answer. Every denial represents an opportunity to advocate, apply evidence, and push access forward. When a patient’s health is at stake, persistence is essential.
Learn more about BioScript’s patient support services and how we can help families with reimbursement navigation.