Patients may assess the side effects of therapies or the burdens of participating in a clinical study differently from the physicians who treat them. Their opinion or that of their caregivers on an expert panel evaluating side effects of new treatments may therefore be decisive.

Patient organisations like Progena (Swiss Duchenne Foundation), which support families and people affected by Duchenne muscular dystrophy with information about the disease and its course, often also make important contributions to research. This is also the case in the study "TAMoxifen in Duchenne Muscular Dystrophy (TAMDMD)" led by Prof. Dirk Fischer at the University Children's Hospital of Basel.

Lessons learned

Thanks to the mediation by Progena, the mother of a boy affected by Duchenne disease became a member of the Data Safety Monitoring Board (DSMB) for the TAMDMD study. During the course of the clinical trial, this board meets regularly and evaluates the side effects that have occurred. The mother describes her experience as follows: "When I was asked for the Safety Board of the Duchenne study by Prof. Fischer, I was uncertain at first. How can I participate, I have no clinical knowledge! Now I see it differently. I am convinced that it is very important to bring in the point of view of the patient and the affected family. For them, study participation involves a lot of effort, sacrifice and emotion."

Tips

The mother describes the value of her contribution as a person affected by this serious disease as follows: "We experience possible side effects differently than the doctors and it is important to weigh them well in relation to the expected effect, because living with Duchenne muscular dystrophy is hard enough. It is a sign of trust when a patient organisation is allowed to actively participate in a study and the experience gained helps further in the work for and with families and patients."

September 2020