New Registry Launches For Patients With FOP
August 17, 2015
The International FOP Association (IFOPA) announced today that it has launched the FOP Connection Registry, a new tool to support and accelerate development of treatments for the rare genetic disease fibrodysplasia ossificans progressiva (FOP).
“A registry is essential for understanding any rare disease and developing potential therapies”
The FOP Connection Registry is a global, voluntary, research database that will collect and report demographic, disease, and quality-of-life information directly from individuals with FOP twice a year via a secure, web-based tool. The Registry is owned and administered by the IFOPA. Data are de-identified and may be shared with individuals or institutions conducting research or clinical trials, with guidance from Registry medical, scientific and patient advisors. The IFOPA will add a medical portal to the Registry later to allow clinicians to enter data for FOP patients under their care. Over time, and with the active participation of the FOP community, the FOP Registry promises to provide valuable insights into the prevalence, diagnosis, symptoms, impact, and treatment of FOP.
“A registry is essential for understanding any rare disease and developing potential therapies,” said Betsy Bogard, IFOPA Director of Global Research Development. “One of the biggest challenges in very rare diseases such as FOP is limited patient data. The FOP Connection Registry puts the power of data collection directly into the hands of the patient community and will allow us to bring together a much larger volume of data,” said Ms. Bogard.
The Registry will be open to the FOP community worldwide. The IFOPA’s goal is to include as many patients as possible. In order to reach this goal, the Registry will be translated into multiple languages for use by patients around the world. “The Registry is a tremendous opportunity for our whole community to play an active role in achieving our vision of a treatment and ultimately a cure for this devastating disease. We encourage all FOP patients to participate in the Registry,” said Marilyn Hair, Chair, IFOPA Board of Directors.
“The international FOP community is small but mighty and speaks with one voice in one language understandable to all: we want a cure and we need one international FOP patient registry, owned by the FOP patients, to help make that happen. It’s one critical goal we can accomplish together and one critical way we can change our world,” said FOP expert Frederick Kaplan, MD, Chief, Division of Molecular Orthopaedic Medicine, University of Pennsylvania.
The IFOPA engaged Neal Mantick, a pharmaceutical industry veteran with over 20 years of experience in disease registries and outcomes research, to support the Registry.
The FOP Connection Registry software is implemented using Digital Infuzion’s N of 1 platform. Digital Infuzion, Inc., is a provider of cloud-based solutions for health research in the life sciences. Digital Infuzion’s N of 1 Software as a Service (SaaS) Registry Platform allows the IFOPA to capture not only patient-entered data, but also data from FOP physicians, including prospective clinical data, product outcomes data, and data aggregated from past studies.
The Registry is intended to meet the needs of all stakeholders in FOP drug development: patients, doctors, and researchers. “Our unified, global, and coordinated approach to the Registry will allow for the best, most comprehensive data on FOP, ultimately enabling better and faster development of therapies,” said Ms. Bogard.
For more information, visit www.ifopa.org/registry.
About Fibrodysplasia Ossificans Progressiva (FOP)
FOP is a rare genetic disease in which soft tissue transforms permanently into bone, ultimately imprisoning those afflicted in a second skeleton of bone. Bridges of extra bone develop across the joints, progressively restricting movement and leading to eventual immobility. The disease is caused by a point mutation in the gene encoding the ALK2 receptor, rendering it overactive. Researchers estimate that there are approximately 3,000 individuals living with FOP worldwide.
About the IFOPA
Founded by Jeannie Peeper in 1988 to end the isolation formerly associated with FOP, the IFOPA is a registered 501(c)(3) organization that has evolved into the world’s leading non-profit resource for FOP and today has members from over 50 countries. The IFOPA’s programs and services are focused in four primary areas, including research, education, support programs for FOP members/families and global advocacy. The organization celebrated its 25th anniversary in 2013 and has raised more than $18 million in resources for its objectives. Find out more at www.ifopa.org.
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