Meeting the needs of the global FOP community
“The IFOPA and the FOP community have been aware of the value of initiating a registry program for FOP for some time,” shares Neal Mantick, FOP Registry Study Manager, as he explains the importance of the FOP patient registry, which was launched in July 2015.
“Betsy Bogard, Director of Global Research Development, led the charge on this project with strong support from the IFOPA board,” he continues. “And it’s thanks to Betsy’s leadership the project was carried on after some initial attempts in partnership with a larger group of rare disease organizations and the National Institutes of Health (NIH) didn’t materialize.”
One challenge in creating the patient registry is that while the IFOPA is based in the United States, the needs of the FOP community are global. The registry must be multi-lingual and include other countries with a large enough concentration of FOP patients and physicians working in the field. The initial languages into which the patient registry is being translated are French, German, Italian, Spanish, Portuguese and Russian. Evaluation is currently underway to explore further translations into Japanese and Chinese.
To ensure consistency and accuracy, a formal forward and backward translation process is being used for each language. FOP parents, families and caregivers then participated in beta testing the translated registry websites that covered all the bases.
“Not only did we have to review the patient surveys, we also had to test all of the system’s error messages and prompts to get the kinks out and we’re very grateful to the individuals and families who helped us address all those small, but critical details,” Neal says.
The registry process begins by the person with FOP (or a parent if they are under 18) submitting a registration form and setting up their profile with their contact details and other administrative information. Initial health information is collected on an enrollment survey, then follow-up surveys are sent every six months to collect updates on their health. Participants answer questions about physical symptoms, their use of doctor and dentist visits, hospitalizations and overall quality of life.
Neal explains that the registry is a formal research study with specific study objectives to:
- Organize the community for clinical studies
- Empower both individual patients and the community as a whole
- Improve the collective understanding of FOP
- Advance the understanding of treatments (when available)
Ultimately, the plan is for the FOP Registry to have two portals: one for patients and one for the medical community to capture data from clinicians treating FOP patients. The two portals will be linked in the common registry database so that a patient’s reported data will be linked with the physician’s reported data to create a more complete picture of that particular patient’s FOP status.
Neal notes that Eurodis, a rare disease organization in Europe, has stated that rare disease communities with well-implemented registries and active patient organizations have a higher likelihood of developing a treatment.
“I commend the IFOPA for taking this on,” he concludes. “The processes are the key. We’re documenting everything to ensure the best practices for all stakeholders and to foster an environment of sharing and collaboration.”
Learn more about the FOP Registry and how you can participate.