An Urgent Need for Data to Drive Decision Making: Rationale for the Canadian Registry for Amyloidosis Research

Recent developments in cardiac amyloidosis have raised awareness of the disease and have advanced diagnostic and treatment strategies. Novel therapies may vastly improve the prognosis of the disease but will be associated with significant costs. Data are needed to inform clinical decisions and to drive resource allocation within the health care system. Many aspects of disease management are unlikely to be addressed by clinical trials and are better suited to nonrandomized cohort studies, but the limited numbers of patients in any single centre present barriers to high-quality observational research. Disease registries offer opportunities to assemble large numbers of patients from multiple institutions for adequately powered observational studies, to recruit patients for randomized clinical trials; to provide real-world effectiveness and safety data, to study cost-effectiveness of novel therapies, and to engage patients in the collection of patient-reported outcome data. Existing amyloidosis registries have limitations. Canada is an ideal setting for a national amyloidosis registry, with ethnic diversity, relatively few academic centres, access to advanced diagnostic and therapeutic options, and a track record of collaboration among institutions. The Canadian Registry for Amyloidosis Research aims to capitalize on this opportunity and provide high-quality data to inform clinical practice and health care policy in Canada and beyond.