Limb Girdle Muscular Dystrophy Community Advisory Board
Aleksandra Leijenhorst, Kate Adcock, Donavon Decker
Now more than ever, patients and the patient perspective are seen as a way to make medical research and clinical studies more impactful, possibly quicker and certainly more efficient. Patient Community Advisory Boards (CABs) comprise people living with the specific condition, a close family member or carer, and/or a member of a patient organisation that works with the disease. With the advent of new therapeutic opportunities such as gene therapy for people living with forms of LGMD, it is timely for investigators to engage with patient experts as early as possible when designing their studies.
CABs meet with the sponsors and investigators of clinical research (such as pharmaceutical or biotechnology companies, or a hospital or university research team) to discuss plans for clinical studies. CAB patient advocates can offer their expertise by being involved before a clinical study starts to ensure that studies are designed taking into account the real needs of patients, resulting in higher quality research. This could mean the CAB members, for example, offer their expertise in how the clinical trial is run, how the patients will be able to best adhere to protocols, or how to ensure continued access to the medication after the study ends.
The Limb Girdle Muscular Dystrophy CAB has 12 patient advocate members from Norway, Finland, UK, Netherlands, France, Spain, Italy, India, the USA and South Africa. Thanks to its breadth of expertise, the group can provide advice on a wide variety of topics, such as: clinical studies and their design; criteria for participation; retention of participants; engaging patients and families; and reporting on results. Several members of the CAB have personal experience of participating in clinical trials either directly or as parents.
The group is facilitated by EUPATI Spain.