Advisory Group important to make sure Canadians have their say in improving access to effective treatments and services for patients living with rare diseases
One in 12 individuals in Canada, many of whom are children, grapple with rare diseases. However, access to treatments for these conditions remains limited.
To address this issue, the Government of Canada has launched its first-ever National Strategy for Drugs for Rare Diseases in March 2023. As part of this strategy, the government pledges to create a national governance structure aimed at improving access to effective drugs for rare diseases across the country.
In a more recent development, Mark Holland, minister of health, introduced the Implementation Advisory Group (IAG) for drugs for rare diseases. Over the next three years, this advisory group will serve as a platform for patients and stakeholders to provide patient-centred advice, share information about rare diseases, and exchange best practices to guide the implementation of the National Strategy.
“Access and affordability should never be barriers to care,” says Holland. “Since consultations on a drugs for rare disease strategy began in 2021, we’ve heard from patients and families with lived experience as well as from stakeholders that a stronger patient-centred approach is needed to ensure Canadians living with rare diseases could access the drugs they need, regardless of where they live.
“The Advisory Group is an important part of our efforts to make sure patients have their say in improving access to effective treatments and services for patients living with rare diseases.”
The IAG comprises 20 members representing the rare disease community, including individuals with lived experience, healthcare professionals, the pharmaceutical industry, and researchers. Dr. Avram Denburg, chair of the drugs and therapeutics committee and staff physician at The Hospital for Sick Children, and Dr. Gail Ouellette, former founding president of Regroupement québécois des maladies orphelines, will be the initial co-chairs of the IAG, with their roles rotating annually. The group held its inaugural meeting October 26.
The National Strategy for Drugs for Rare Diseases
In March 2023, the government pledged up to $1.5 billion over three years to support the National Strategy for Drugs for Rare Diseases. The investment aims to increase access to and affordability of effective drugs for rare diseases, benefiting patients across Canada, including children.
Within this funding, up to $1.4 billion will be allocated to provinces and territories through bilateral agreements, enabling them to improve access to new and emerging drugs, enhance access to existing drugs, and advance early diagnosis and screening for rare diseases.
Health Canada had previously conducted a national engagement in July 2021 to gather insights on the National Strategy, involving over 650 individuals and organizations, including patients, family members, and caregivers.
