From the Editor
The PRP community needs a PRP Patient Registry. The following is a broad overview of issues related to Patient Registries and Natural History Studies.
WHAT IS A PATIENT REGISTRY
✽ A registry is a collection of information about individuals, usually focused around a specific diagnosis or condition. – NIH
✽ Contact registries collect basic emographic and contact data
– Not the same as registering for an email list
– It’s important to collect data which may be used as inclusion/exclusion criteria (birth date, geographic location, etc.)
WHAT IS A NATURAL HISTORY STUDIES
✽ Natural history is the natural course of the disease from the time immediately prior to its inception, progressing through its pre-symptomatic phase and different clinical stages to the point where the disease has ended without external intervention – FDA R01 Natural History Study RFA
– Focuses on progression
– Foundation of drug development/clinical trials
PATIENT REGISTRY VERSUS NATURAL HISTORY STUDIES
✽ Developing a patient registry around one rare disease does not mean that the natural history of the disease is being captured
✽ Natural history studies are the foundation of drug discovery/clinical trials; however patient registries are the foundation of natural history studies
✽ Both natural history studies and registries are important for rare diseases
– Collect information to engage community, educate public and researchers
IMPORTANCE OF PATIENT REGISTRIES AND A NATURAL HISTORY STUDIES
✽ Define your population
✽ Identify questions in which the answers would be important to researchers, physicians or patients and families
✽ Creating a patient registry is a great way to foster community (support group)
– A means to develop new relationships – Gives participants a voice
– Remove sense of isolation
– Identity
✽ A registry and natural history study creates hope for the community
✽ Patient registries and natural history studies create awareness
– Drive research
✽ Information collected will hopefully lead to publication of materials more accessible to physicians
✽ If physicians have more resources available they can make better decisions and better diagnose patients
RESEARCH
✽ A rare disease diagnosis often means the patients are the experts
✽ Patient registries and natural history studies can help your organization attract the attention of researchers
✽ Researchers are often introduced to rare disease research by chance
✽ It’s best to be prepared for when an expert is found
OPERATIONAL CHALLENGES
✽ Patients are often motivated, but it is difficult to identify them
✽ Establishing partnerships with organizations • Trust is a key issue
✽ May need to recruit globally in order to accrue adequate patient populations (especially in ultra-rare cases)
✽ Data entry can be time consuming
✽ Critical to ensure patients aren’t lost to follow-up
SUMMARY
Patient registries and natural history studies present unique opportunities to foster community, educate, promote awareness and research, Patient registries are the phonebook while natural history studies are the encyclopedia.
Source: EveryLife Foundation, Community Congress, Webinar, Sanford Research Clinical Development for Rare Diseases: A Primer for Rare Disease Patients and Advocates (March 29, 2017)