In Canada, 14,000 children under the age of 15 die each year from a rare disease. With more than 7,000 known rare diseases, it is essential for researchers from across the country to work together to improve the health outcomes of all children and teens living with a rare disease

“The creation of this network supports our government’s commitment to improving the lives of all patients living with a rare disease. By bringing together rare disease pediatric community members from across Canada, modernized diagnosis and treatments will be made possible. Our government will support Canadian researchers as they continue to make remarkable contributions to health research to benefit not only Canadians, but rare disease patients around the world.”

The Honourable Mark Holland
Minister of Health

“There are researchers and health care workers across our country working tirelessly to help children living with a rare disease and their families. This network will allow for streamlined collaboration amongst these researchers and clinicians, as well as with industry, policy, and regulatory bodies. Most importantly the people who know these diseases best—the patients and their families— will be an integral part of this network.

Dr. Christopher McMaster
Scientific Director of the CIHR Institute of Genetics

“This initiative represents a significant step forward in addressing the complex and unmet needs of Canada's rare disease community, particularly in the realm of pediatric clinical trials. By assembling a diverse and highly skilled team of researchers, patient advocates, patient partners, research networks, equity champions, industry partners, international partners, and 17 child health academic centres from across Canada, the network is poised to make a profound impact on the lives of patients and families affected by rare diseases.”

Dr. Thierry Lacaze-Masmonteil
Scientific Director of the Maternal Infant Child and Youth Research Network

“As a parent of two children who live with a rare diagnosis, I'm encouraged by the work & development of a Canadian rare disease clinical trial research network. Our family, like many with lived experience in the rare disease community, has faced uncommon challenges. I am hopeful this network will create partnership and engagement between families and researchers, as we work together to weave hope amidst uncertainty.”

Sara Pot
Patient partner

• Dr. Lacaze-Masmonteil’s team consists of members of the pediatric rare disease community from across the country, including patients, family members, advocates, and researchers from the fields of pediatrics, epidemiology, pharmacology, genetics, and immunology.

• In March 2023, Canada launched its first National Strategy for Drugs for Rare Diseases to increase access and affordability of effective drugs for rare diseases. It included $32 million in funding to CIHR to advance rare disease research in Canada.

• This is the first of four CIHR Rare Disease Research Initiative funding opportunities derived from the National Strategy for Drugs for Rare Diseases that were created to increase collaboration across the rare disease community and improve the lives of patients living with rare diseases, and their families. 

Associated links

• What are clinical trials?
• CIHR Rare Disease Research Initiative
• Team Grant: Pediatric Rare Disease Clinical Network
• News release: National Strategy for Drugs and Rare Diseases

Christopher Aoun
Press Secretary
Office of the Honourable Mark Holland
Minister of Health
613-957-0200

Media Relations
Canadian Institutes of Health Research
mediarelations@cihr-irsc.gc.ca

At the Canadian Institutes of Health Research (CIHR) we know that research has the power to change lives. As Canada's health research investment agency, we collaborate with partners and researchers to support the discoveries and innovations that improve our health and strengthen our health care system.