Patient Registries

INFORM RARE is contributing to the co-design, enhancement, and implementation of Canadian patient registries.

 

Registry Values

 
This image shows the patient registry values. They are: sustainability; accessibility; scalability; public benefit; benefit to patients and their families; building research capacity.

Canadian Organization for Rare Disorders. Rare Disease Patient Registries: Key to Drug Development and Access. Canadian Metabolic Patient Registries. May 2022; https://www.youtube.com/watch?v=pTGM2XifptY&t=1423s.

 

Metabolic Patient Registries 

Registries currently in development

The Canadian MPS Registry, in partnership with the Canadian MPS Society

The Canadian PKU Registry, in partnership with Canadian PKU & Allied Disorders Inc.

Objectives

  1. Furthering the understanding of the natural history of these rare inherited metabolic diseases;

  2. Evaluating intervention effectiveness (e.g., through registry-based randomized trials);

  3. Providing a clinical contact database for academic- and industry-sponsored research; and

  4. Allowing patient organizations and their partners to promote and support patient-oriented research (research that focuses on the priorities of patients and their caregivers).

Governance

 
This image shows the governance structure for the metabolic patient registries. It includes all partners and organizations contributing to the registries, as well as the contracts and agreements between them.
 

SMA Registry

Please visit the INFORM SMA webpage for more information.