Our vision

To improve health care for children with rare genetic diseases by generating real-world evidence to support decision-making about new and existing therapies.

Towards improved patient and system outcomes for children with rare diseases

INFORM RARE is a Canadian research network that is co-designed by patients and families, health care providers, policy-makers, methodologists, and research ethicists. Working together, we will evaluate new and existing therapies and make recommendations to improve care for children with rare genetic diseases.

INFORM RARE's research activities build on registries and other studies established by the Canadian Neuromuscular Disease Registry (CNDR) and the Canadian Inherited Metabolic Disease Research Network (CIMDRN) to generate the evidence needed to improve clinical care, outcomes, and health policy for children with rare diseases.

Patient partnership and engagement

Patients and family member partners are involved as co-investigators and advisors in co-designing our research. They are actively involved in key decisions, for example:

• Selecting and refining research questions and priorities
• Developing strategies for consent and data sharing
• Selecting trial outcomes