INDEX PRP Patient Registry

From the Editor
The PRP Survival Guide is a repository of experiences and insights shared by PRP patients and their caregivers. Collectively, the PRP community possesses a wealth of practical knowledge about pityriasis rubra pilaris. We need to harvest that knowledge for those in need of enlightenment.

Share what you have learned about PRP as a patient or caregiver. Share what you have been told by your dermatologist? Share articles  you feel might be worth reading or websites worth visiting. Please use “Leave a Reply” at the bottom of this webpage to answer the following question:

What do you want to know about the PRP Patient Registry?

INDEX PRP Patient Registry

One Reply to “INDEX PRP Patient Registry”

  1. 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 VERSION 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
    – Benefits outweigh the challenges
    ✽ Patientregistriesarethephonebook
    ✽ Natural history studies are the encyclopedia
    ✽ CoRDS is both

    Austin Letcher
    Sr. Research Associate, CoRDS 605.312.6423 Austin.Letcher@sanfordhealth.org http://www.sanfordresearch.org/cords

    About CoRDS
    Mission: To accelerate research into rare diseases
    Goal: To establish an international rare disease patient registry for all rare diseases
    ✽ Established by David Pearce, PhD in 2010
    ✽ Create resource of contact information and clinical data on individuals diagnosed with any rare disease to enable a comparative analysis across diseases
    ✽ Connect researchers to participants interested in participating in research
    ✽ Partner with patient advocacy groups to create disease specific registries and natural history studies
    ✽ IRB approval

    Source: EveryLife Foundation, Community Congress, Webinar, Sanford Research
    Clinical Development for Rare Diseases: A Primer for Rare Disease Patients and Advocates
    March 29, 2017

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