Lacaze-Masmonteil, Thierry

Published purpose

The specific objectives of this funding opportunity are to: • Develop a platform to support pediatric rare disease clinical trials in Canada in an accessible manner, for both the profit and not-for-profit sectors, to maximize the impact and benefit of the research for clinical practice, policy, and future research; • Increase the capacity to perform rare disease clinical trials in Canada by offering training and mentorship opportunities for researchers, patients, clinicians and other relevant stakeholders; • Attract international clinical trials, increasing investments in Canada and providing earlier access to innovative therapies to people living in Canada; • Foster diversity and inclusion in and improve access to clinical trials; • Implement processes to acquire data from clinical trials, real world registries, health economics evaluation, and post-marketing surveillance to inform assessment reviews throughout the whole drug/therapy cycle; and, • Increase the number of new rare disease drug submissions for authorization of commercialization to Health Canada.