Stories Power Research through the Launch of the FD/MAS Patient Registry

After many years of planning and preparation, the FD/MAS Patient Registry opened for enrollment this week.

The FD/MAS Patient Registry is an IRB-approved research study that allows patients or their guardians to complete surveys about their symptoms, treatments and experiences. Anyone who has been diagnosed with fibrous dysplasia or McCune-Albright syndrome can join the study.

“Registries have emerged as an important research tool, particularly in rare diseases where performing large, high quality studies is challenging,” said Dr. Alison Boyce of the National Institutes of Health. “The FD/MAS registry is an exciting opportunity for patients, clinicians, and investigators to share information that can accelerate the pace of research in this disease, and hopefully lead to better treatments to improve quality of life for patients and families.”

The project wouldn’t have been possible without a very special leader. Lisa Heral, a former member of the Fibrous Dysplasia Foundation Board of Directors, and the mother of an adult son living with fibrous dysplasia, was that leader. When the Fibrous Dysplasia Foundation Board of Directors first decided to research a patient registry in 2010, Heral volunteered to learn about patient registries and come up with a plan for the FDF. Today, Heral is a member of the FD/MAS Patient Registry Steering Committee, along with Dr. Alison Boyce, Dr. Andrea Burke, Dr. Amanda Konradi and Catherine Fairchild, JD.

“The more I looked, the more reasons I found to start a registry for fibrous dysplasia and McCune-Albright syndrome,” said Heral. “I knew it would be hard, and that it would take a lot of work, but there was no doubt in my mind that we would be way better off if we made this happen.”  Patient registries can accelerate research, improve quality of medical care and, increase the likelihood of drug development.

Heral’s biggest achievement was bringing together a talented team of people and a network of partnerships to help build the registry. Her progress bringing together a dedicated group to work on the project accelerated in mid-2015, when the project received an award from the Patient-Centered Outcomes Research Institute.

“The PCORI contract was transformative, but I don’t think Lisa’s role in making this dream a reality can be overstated,” said Deanna Portero of the Fibrous Dysplasia Foundation. “I’ve never met anyone who could bring people together like Lisa. She has a real superpower to unite people behind a common goal.”

The slogan of the FD/MAS Patient Registry is “Your Story Powers Research,” because each unique patient story will be like a puzzle piece. When put together with other puzzle pieces, each patient story could help qualified researchers answer important questions.Patient registries are a unique type of research project because they are most useful when they are designed, built and run by patient organizations. Patient organizations, like the Fibrous Dysplasia Foundation, make sure that the study is designed so that the data can be used and reused by a diverse set of research projects. After identifying information about each patient is removed, the data becomes a central resource for the entire research community.

“Now that the study is open, it’s up to patients and parents to learn about the study, and decide if they will enroll. The more patients that join the registry, the more powerful and accurate the results will be,” said Heral. “Once a significant number of patients have finished the surveys, we’ll be able to share the data and the results. Until then, I’m enjoying a bit of a break!”

You can learn more about the FD/MAS Patient Registry by visiting https://www.fdmasregistry.org