Patient and researcher meetings are important because they allow engaged members of a rare disease community to organize, strategize, and take action. This year, patient and family meetings were held in Maryland in the United States, Leiden in the Netherlands, and Birmingham in the UK. FDF hosted the United States meeting and attended both European meetings including the 3rd annual FDMAS International Consortium.
Netherlands Meeting – 3rd Annual International FD/MAS Consortium Meeting, and Patientenvereniging Fibreuze Dysplasie Patient’s Day! November 15-17th, 2017
Leiden University Medical Center was the site of a large, three-day meeting in mid-November. The meeting was organized by Leiden University Medical Center researchers, including Dr. Natasha Appelman-Dijkstra and Neveen Hamdy, and patient advocates from the patient group Patientenvereniging Fibreuze Dysplasie, including Martine Dekker. The meeting was supported by the European Society of Endocrinology, and the Endo-ERN and ERN BOND of the European Reference Networks.
The first two days of the meeting were attended by dozens of leading FD/MAS scientists, and leaders from four FD/MAS patient groups. The goals of the first two days of meeting were to review and update the clinical management of FD/MAS, and to encourage interaction between basic scientists with clinicians of various specialties and patient advocacy groups. In both respects, the meeting was very successful.
“For the third year in a row, FD/MAS researchers have come together at an annual scientific meeting specifically focused on FD/MAS. These types of meetings are just as crucial to progress as research grants. They offer a venue where researchers can exchange cutting-edge information, and find new areas for collaboration,” said FDF executive director Deanna Portero, who attended the meeting.
“We were so grateful that the researchers welcomed the Fibrous Dysplasia Foundation to the meeting, along with patient community leaders from the UK, Italy, the Netherlands and Romania. The researcher community recognizes that the FD/MAS patient community is a full partner in the path to a cure, and that we need to be at the table where challenges and opportunities are being discussed.”
The meeting truly was international. In addition to the international delegation of patient advocates, presenting researchers at the scientific meeting came from Dutch, Italian, American, Danish, French and British research institutions. Many of the research topics–from the development of new treatments, to clinical trials of existing therapeutic options–require international collaborations, either because the top experts in an area are distributed across counties, or because many research questions would require so many patients to enroll in a study that an international collaboration is the most realistic option to conducting a study.
One of the major points of discussion was finalization of a new “clinical pathway,” a comprehensive guide on how to manage FD/MAS based on the consensus opinions of top FD/MAS clinical researchers. The hard work of achieving consensus is mainly complete, so now the conversation has turned toward distributing this resource once it is complete. Part of that solution, of course, involves working with patient groups like the Fibrous Dysplasia Foundation. However, it’s also just as important, and much more challenging, to reach clinicians directly, and the researchers and patient groups discussed strategies to accomplish that goal.
The third day was the “Patients Day,” where families from all over Holland came to Leiden to learn directly from the researchers. Topics included bisphosphonates, denosumab, pediatric management, orthopedic management, and the role of patients and families in advancing care. The third day ended triumphantly, with the presentation of a check for 55,000 euros to the Leiden University Medical Center from FD/MAS patient advocates, for the purpose of FD/MAS research.
For more information about Patientenvereniging Fibreuze Dysplasie, visit fibreuzedysplasie.eu.