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Rare Disease

Clinical Trials Are Vital for Rare Disease Treatments

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Dr. John Wallenburg, Cystic Fibrosis Canada

Dr. John Wallenburg

Chief Scientific Officer, Cystic Fibrosis Canada

Whether you realize it or not, you likely know someone affected by a rare disease. According to the World Health Organization (WHO) and a publication by Carlos Ferreira ,as many as one person in 16 is affected by a rare disease — or about 2.4 million Canadians.

Rare diseases are serious chronic diseases, often genetic, and many, such as cystic fibrosis (CF), are life-threatening. Unfortunately, like CF, many are invisible, so you don’t necessarily know which friend, colleague, neighbour, or acquaintance is impacted. 

You might ask, “So what?” Well, compared to more common conditions, there are specific challenges when it comes to diagnosing and treating rare diseases. Individually, rare diseases attract less attention and so the basic discovery science is often lacking. Without the science, therapeutic development is delayed or even non-existent. When development does proceed, complications like small patient groups, few expert centres to run clinical trials, and limited clinical expertise — including within regulatory bodies — hinder the task of clinical development.

Clinical trials are critical for both patients and researchers

One way that patients can help themselves, help their communities, and gain access to drugs or treatments not yet approved is through clinical trials. Clinical trials are an important and essential step in the development of new treatments and an integral part of the process that brings new therapeutics and better care to Canadians who are living with rare diseases like CF. Clinical trials test potential new drugs, treatments, and devices to assess how well they work, to ensure they’re safe, and to determine whether they offer benefits over currently-used drugs, treatments, or therapies. 

Clinical trials provide participants the opportunity to play a role in advancing research and impacting the future of the disease. This is why Cystic Fibrosis Canada established the CF Canada Accelerating Clinical Trials program, a network that helps to facilitate the development of new treatments for CF, as well as increase capacity and enhance participation of people with CF in clinical trials. The network is now directly accessible to 60% of the CF population in Canada. 

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