ADVOCACY WITH RARE DISEASES AND CORD

Advocacy efforts for a Rare Diseases (RD) Clinical Trials Infrastructure (under the premises of the National Strategy on High-Cost Drugs for Rare Diseases). Triggered by preparatory consultations that took place under the auspices of Health Canada and involved CPS, RD researchers, and several RD research-intensive institutions, in January 2022, MICYRN wrote a proposal outlining the rational for a national infrastructure with the following vision: To create the sustained national coordination, expertise, and infrastructure necessary to ensure effective and novel therapeutics and interventions are available for children and their families with rare diseases (RD) in Canada; this coalition will generate the high-quality data to inform Health Canada and facilitate the decision-making process for access and pricing in high-cost RD drugs and enhance the overall cost effectiveness in the healthcare system. The proposal was endorsed by the MICYRN BOD and the RI Directors in February 2022. It was shared with the HC Leadership involved in the rolling out of the Strategy. The pivotal role played by MICYRN in driving the cause of the RD infrastructure was recognized at the CORD conference in June 2022. MICYRN was hailed as the potential vehicle for steering this vital initiative forward. Following the impactful CORD conference in November 2022 and in anticipation of the imminent launch of the National Strategy, along with research funding opportunities, MICYRN took proactive steps to engage researchers and representatives from patient organizations. MICYRN hosted a webinar in December 2022 providing a platform to discuss potential roles and mandates of a RD Clinical Research Network. Participants envisioned various categories of services and support that would be imperative should a national research infrastructure for RD clinical research activities receive funding.