Revised standards of care were recently published in Neuromuscular Disorders (part 1 & part 2). Coordinated by Treat-NMD, SMA Europe is been working with members of other organisations, on a version for families.
Providing the perspective of patients and families is invaluable to the development of clinically meaningful outcomes that are accepted by health authorities and reimbursement decision makers. Patient Centered Outcome Measures (PCOMs) are defined as tools to determine efficacy of a treatment and how a disease and its progression impacts the lives of patients by placing the patients at the centre of the development of what has to be measured.
SMA Europe is committed to applying its resources to designing pivotal outcome measures for SMA, shaped in a patient-centric way. We believe that the only way to achieve this goal is through a close partnership with researchers, clinicans, authorities and industry. An open dialogue should pave the way to shape PCOMs that have a broad acceptance as tools for research on the disease and on therapies. Once trials have been successfully completed, health and reimbursement authorities have the responsability of granting access to treatment. Tools that adequately reflect the impact of a therapy on a patient’s well being will support these authorities to make sound decisions, also taking into account the patients’s perspective.
Read our position statement on PCOMs
SMA Europe has initiated a structured collaboration with Cure SMA and the Industry Consortium in the US, through a pilot project which consists of mapping Europe’s current capacity to run clinical trials. The project is anticipated to take 6 months.