Training and education
A key goal of NMD4C is to train and educate the next generation of NMD stakeholders.
Genomics training (Leads: James Dowling, Jodi Warman Chardon)
Canadian geneticists are world leaders and trailblazers in using genomics to identify the genetic causes of NMDs and other rare diseases. For neurology, where many of our patients have inherited NMDs, it is time to incorporate genomics into standard care. This is especially true since, unlike most rare diseases, some NMDs are amenable to targeted therapies, so accurate diagnosis is of critical importance.
To date, genomics training for NMD clinicians and scientists has been scarce and not well coordinated. To ensure patients benefit from these new diagnostic and therapeutic opportunities, training of NMD clinical specialists has been identified as a particular need, and also one that Canada is well placed to meet through excellent Canadian genomic initiatives such as the Care4Rare Canada Consortium (co-located with NMD4C at CHEO Research Institute). NMD4C will collaborate with Care4Rare to deliver ‘virtual NMD rounds’ every three months where neuromuscular clinicians and scientists will learn how to use and interpret genomic data in the context of clinical, electrophysiological, imaging and myopathological findings, and solve complex, diagnostic cases. At virtual NMD rounds a multidisciplinary team (e.g., the responsible physician, geneticist, genetic counsellor, and genomic scientist) will present and discuss cases and consider a variety of genomic and laboratory data. NMD4C will partner with Can-GARD to deliver complex NMD case rounds and develop an NMD genomics curriculum for neuromuscular fellowship programs.
- Genomics virtual/remote NMD rounds
- Genomics training at Can-GARD National Grand Rounds
- NMD genomics curriculum
Trial capacity building and educational summits (Leads: Lawrence Korngut, Craig Campbell)
Canadian hospitals and clinical researchers are involved in many cutting-edge NMD trials, but training of NMD clinicians and trial personnel, including study coordinators and physiotherapists, has been led by industry sponsors on an ad hoc basis. Coordinated training, standardized across trials and indications is needed to obtain the high-quality data needed for efficacy trials and post-marketing surveillance of very expensive NMD treatments. NMD4C will provide training opportunities for NMD clinicians and trial personnel in the collection, interpretation, and reporting of outcome measures, biomarkers, and other clinical research data. Building on the work already done by CAN-NMD and CPNG, we will work with industry partners and MDC to develop an NMD clinical research curriculum. We will develop and integrate a clinical research curriculum for neuromuscular fellowship programs. This curriculum will also be made available to other personnel involved in NMD trials through e-learning tools. NMD4C will partner with industry to launch NMD summits as a demonstration project, where NMD4C could bring together Canada’s NMD community and facilitate industry-sponsored training for natural history studies, clinical trials, standards of care and post-marketing surveillance such as the SMA summits successfully launched by Biogen, CAN-NMD and MDC in 2018 and 2019.
- Clinical research curriculum for neuromuscular fellowship programs
Expert patient capacity building (Leads: Cynthia Gagnon, Kathryn Selby, MDC)
Canadians with NMD, their families, and advocates are increasingly asked to take an active role in research following the SPOR Patient Engagement Framework developed by CIHR and give input and support decisions for clinical research, marketing authorizations and payer decisions. Research and advocacy training empowers patients and ensures they have the confidence and knowledge needed to bring their expertise forward to guide discussions and inform decisions regarding health care, research, and therapy development with policy makers, industry and scientists. International RD advocacy organizations such as EURORDIS (Rare Diseases Europe) provide highly effective training for families with RD via their ‘Open Academy’, but a module providing Canadian and NMD context is lacking. With co-leadership from MDC and our knowledge users, NMD4C will establish expert patient capacity building for Canadian NMD patients. Working with EURORDIS, we will leverage their Open Academy model to develop an easy-to-use and accessible Canadian NMD patient advocacy module with electronic and written material.
- Canadian NMD patient research and advocacy module
Clinical and translational empowerment of young scientists (Leads: Rashmi Kothary, Jodi Warman Chardon)
Canadian early-career scientists are more likely to develop a full understanding of the translational needs of NMD and make a long-term commitment to NMD if they are provided with opportunities to meet patients in clinic. Moreover, young NMD scientists are keen to exchange ideas and support each other if provided with a forum or community. Thus, NMD4C will provide NMD clinical placement opportunities (1 week each) to early career researchers through online dissemination and the forum through its clinical members as well as host regular early career webinars where senior faculty present on a topic of interest such as an overview of the recent trial results in a disease area and a biannual forum where they can discuss their research and experiences with their peers.