How the patient perspective can improve clinical trial protocols in neuromuscular diseases
AFM-Telethon wanted to formalise the objectives that clinical study protocols should pursue and the points of attention they call for concerning organisational and methodological aspects. This formalisation should help AFM members in their participation in protocols.
In addition to the sponsors, investigators and clinical experts (who focus on the medical aspects and the evaluation of the product efficacy and safety), the associations can propose protocol adjustments in order to facilitate patient adherence, which improves their inclusion and limits the risk of premature trial drop-out. They also promote more patient-centered assessment criteria, which can smoothe the regulatory treatment pathway (reimbursement stage).
Download our paper "Clinical study protocols in neuromuscular diseases (NMD): objectives and points of attention for AFM-Telethon"