NMD4C

Real-World Evidence for Canadian Neuromuscular Disease: Establishing a Framework for National Integration of Patient Reported Outcomes, Clinical Registry Data, Healthcare Utilization and Healthcare Associated Costs

The NMD4C is excited to share that network investigator Dr. Reshma Amin has been awarded funding from the CIHR to establish a framework for real-world evidence (RWE) for Canadian neuromuscular diseases (NMDs) – congratulations to principal applicants Drs. Reshma Amin, Victoria Hodgkinson, Hanns Lochmüller, Eyal Cohen, Gord Jewett, Homira Osman and the entire team behind this successful grant application! The grant received funding through a CIHR team grant, with contributions from Cure SMA Canada, Defeat Duchenne Canada, and Muscular Dystrophy Canada for a total of $1.9 million over a four-year period from 2024-2028. 

This grant aims to understand the burden of NMDs in the Canadian health care system including the prevalence, healthcare utilization, direct cost and health inequity, and to establish a versatile platform for RWE generation that can be used as a model for other rare diseases. Working with a network of site investigators from across the country and the Canadian Neuromuscular Disease Registry, the grant team will link patient level registry data, health administrative data and patient reported outcomes in the pursuit of RWE generation to understand the disease burden for Canadians with NMDs and their families and highlight potential inequities in access to care. 

“The integration of patient reported outcomes, clinical registry data, and health administrative data through an established data integration platform is paramount to moving the needle for NMD care and other rare diseases in Canada” says Dr. Reshma Amin, Director of Sleep Medicine and Long-term Ventilation, Respirologist and Sleep Physician, The Hospital for Sick Children | Professor, Department of Pediatrics, University of Toronto | Clinician Investigator, Senior Associate Scientist, Child Health and Evaluative Sciences, SickKids Research Institute. “Our project framework will help address existing knowledge gaps and allow for clinical and policy questions to be rigorously studied, while creating a model for future rare disease studies.” 

The NMD4C are thrilled to see many of our investigators, members and knowledge users named to this grant. Open, collaborative projects like this display the strength and unity of the Canadian neuromuscular research community, and we are excited to see further projects building on existing partnerships and collaborations. We are looking forward to working with Dr. Amin and the grant team to support the project’s work in building the RWE that is critical to understanding the experience of people affected by NMDs and their families and creating pathways to access effective NMD treatments and therapies for all Canadians.

NMD4C investigators and members named on Dr. Amin’s Team Grant: 

Nominated principal applicant: Dr. Reshma Amin 

Principal applicants: Drs. Victoria Hodgkinson, Gord Jewett and Hanns Lochmüller 

Principal knowledge user: Dr. Homira Osman 

Co-applicants: Drs. Abrahao Agessandro, Bernard Brais, Craig Campbell, Megan Crone, James Dowling, Cynthia Gagnon, Hernan Gonorazky, Aaron Izenberg, Kristina Joyal, Marta Kaminska, Alexandra King, Malcolm King, Lawrence Korngut, Jean Mah, Rami Massie, Laura McAdam, Hugh McMillan, Cam-Tu Nguyen, Colleen O’Connell, Erin O’Ferrall, Maryam Oskoui, Gerald Pfeffer, Xavier Rodrigue, Kerri Schellenberg, Kathryn Selby, Jordan Sheriko, Mark Tarnopolsky, Jodi Warman-Chardon,  and Lorne Zinman. 

Collaborators: Susi Vander Wyk, Corrine Kagan, Jonathan Mediano-Crookston and Andrew Sedmihradsky