Participation in Data Safety Monitoring Boards

Patients may rate the side effects of therapies or the stress of participating in a study differently than the doctors or other staff who is in charge of the study. The involvement of patients in Data Safety Monitoring Boards (DSMB) can therefore be of crucial importance.

Patient organizations 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.

The mother of a boy affected by Duchenne disease has become a member of the Data Safety Monitoring Board (DSMB) of the TAMDMD study through Progena. She describes her experience as follows:

"When I was asked by Prof. Fischer to serve on the Safety Board of the Duchenne study, I was a little uncertain at first. How can I participate, I have no clinical knowledge! Now I look at it differently. I am convinced that it is very important to bring in the perspective of the patient and the affected family. For them, participation in a study involves a lot of effort, sacrifice and emotions. We experience possible side effects differently than the physicians and it is important to weigh them up 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 in the work for and with families and patients."

TAMDMD

TAMoxifen in Duchenne Muscular Dystrophy

Principal Investigator
Prof. Dirk Fischer, University Chisdren's Hospital Basel

Study centres
11 in 8 european country

Planned patients
99

Duration of the project
2018-2022

DKF support
Methodological Consulting, Project Management, Statistics, Data Management, On Site Management

September 2020