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Volunteer Advocate
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Advocating for better access to medications

Most Canadians don’t think about or need to know how a new medicine is discovered, developed, approved and funded.

For Canadians with rare disorders, however, access to new therapies – and even medicines that are being studied in clinical trials – is sometimes the best hope for a longer and higher quality life.

The pathway from discovery of a potential medicine to a rare disorder treatment is often longer and more challenging than for drugs for a broader population. The small numbers of people with rare disorders make it difficult to recruit patients for clinical trials. Demonstrating safety and efficacy of drugs, sometimes with just a handful of participants – when the standards of evidence demand randomized controlled clinical trials – is often a major barrier to obtaining approval and funding for new therapies.

In fact, the biggest challenge for rare disorder treatments often occurs when they are reviewed for comparative clinical and cost effectiveness against other treatments. With so few potential patients, manufacturers have to charge a higher per-patient cost for the development and commercialization of medicines, compared to drugs for that will be marketed for millions, globally. It sometimes becomes unethical to continue RCT research, when manufacturers see a positive effect of their medicine in recipients, and the control group has to be switched to the treatment arm of studies. But so-called “cross-over” trials are often viewed as less compelling or convincing evidence of effectiveness, and health technology assessment bodies have questioned the clinical value of emerging rare disorder treatments for life saving medicines.

The final hurdle comes when provincial governments and private health insurers (mainly sponsored by employers) need to decide whether or not to fund new rare disorder treatments. Based on evidence reviewed by Health Canada and analyzed by health technology assessment bodies, Canadian governments collectively negotiate funding conditions for patient access with manufacturers.

Patients and clinician specialists have an increasing role to play throughout this process. This includes formal input opportunities at Health Canada and the HTA bodies (CADTH and Quebec’s INESSS, as well as provincial groups like Ontario’s Committee to Evaluate Drugs). They are also playing an important role in providing input on policy frameworks and decision-making processes, and regulations, like Ontario’s proposed children and youth pharmacare program, OHIP+.

There is so much to do in terms of advocacy throughout the regulatory, HTA process and funding decisions, that patients and caregivers need a range of tools to understand and input into the system effectively. Access to one medicine at any point in the process depends on the entire system being reformed or improved. That’s where organizations like Cystic Fibrosis Canada and umbrella groups like the Canadian Organization for Rare Disorders come in. It’s the efforts of individuals, caregivers and their advocacy groups that will collectively lead to equitable and affordable access to new medicines for rare disorders, including Cystic Fibrosis. The current system is flawed. It’s up to all Canadians to demand and create a system that supports the rare Canadians who need the most help.

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