Register a Patient
The PFIC Network Patient Registry is your chance to take the power back from this rare disease. By participating you can help to emphasize the patient perspective in PFIC research.
As of October 2022 PFIC Network is collecting data twice a year. Collecting data for each patient every six months helps us observe change over time: when did the patient’s treatments change and why, when did their disease symptoms get better, or worse, or were stable. Multiple waves of data are necessary to identify and better understand trends in the disease.
Everyone’s participation is important. By joining the patient registry for you or your child, you will help to document the needs of the PFIC patient community.
How do I join?
If you are ready to participate, please click the button below.
PLEASE NOTE: If you have already enrolled, you will receive a special invitation link via email six months from your initial enrollment to complete the second wave of data collection. You do not need to register again.
Patients/caretakers enroll through the REDCap portal, https://www.pfic.org/registry. Upon signing the informed consent to participate, participants can then enter their information, complete surveys, and upload health records.
As an alternative to electronic consent, prospective participants can download a paper version of the consent and return the signed consent to PN via regular mail or email. Please email the registry coordinator if you request a paper copy of the informed consent.
Read more about the patient registry below and how it puts the power back in your hands, then sign up as a patient or patient guardian.
What is the Registry?
The PFIC Network Patient Registry is a secure data collection where patients or their caretakers provide information about their experience with the disease. The information you provide is essential for furthering research efforts for better care of PFIC patients and families. All those diagnosed with PFIC are encouraged to participate. Parents can join the registry to enroll their child.
The patient registry collects information about diagnosis, itch, quality of life, medications, surgeries, sleep quality, and patient demographics. It takes about 30 minutes to complete the patient survey. The registry coordinator is happy to help you with questions about the survey.
When completing the informed consent you can choose to be contacted by us if you or your child appears to be a match for a study or a clinical trial. It would still be your choice to connect with the researcher(s) to let them know if you are interested.
The patient registry was initiated in response to the difficulty of locating PFIC patients and PFIC data to conduct research on our rare disease. Our aim with the patient registry has been to add the patient’s voice to every facet of PFIC research and to facilitate and speed up PFIC research towards better treatment and a cure.
The purpose of the registry is to track PFIC disease status and symptoms, assist in clinical trial recruitment, and allow patient participation at each level of research. This puts the power back in your hands.
The registry can also assist with our goal to improve the lives of patients and families worldwide. The PFIC Network can work with registry information to identify knowledge and resource gaps so that we can work on creating solutions for the global patient community.
The registry is vital in our search for a cure, because PFIC is rare and poorly understood. This makes it difficult to gather information about patients and develop treatments for PFIC. As more PFIC patients participate in the registry, researchers can conduct larger clinical trials for PFIC disease and symptom treatments, and eventually discover a cure.
The registry is patient driven. This means that the data in the registry is reported by patients and stewarded by patients. The PFIC Network believes that no one is as passionate as patients and their families in wanting new treatments and a cure. Additionally, no one describes symptoms and quality of life better than patients.
The Patient Voice:
The PFIC Network Patient Registry participants represent an important voice of the PFIC community by:
- Engaging in research through patient surveys
- Joining clinical trials
- Advocating by expressing their unmet needs
- Tracking effectiveness of new trial drugs
- Speeding up new drug approval
Researchers, approved by PFIC Network, can request registry data for their research studies. The data they can receive from the registry are de-identified, which means that all personal information such as names, date of birth, and addresses have been removed. Participants are always in charge of their data. Data is used in accordance with the signed Informed Consent, and participants can have their data removed from the registry at any time.
Privacy & Confidentiality
The PFIC Network established the registry in REDCap, a technology platform that was developed by Vanderbilt University. REDCap is a secure, web-based, HIPAA-compliant, data collection platform with a user management system allowing project owners such as PFIC Network to grant and control varying levels of access to data collection instruments and data (e.g. read only, de-identified-only data views) for other users. The registry policies are reviewed annually by an Institutional Review Board (IRB).
Join the Patient Registry
How You Will Help
See how your participation today helps the entire PFIC community
The first PFIC Patient Registry was created
In 2020 we launched the first PFIC patient registry. The registry was maintained by Coordination of Rare Diseases at Sanford (CoRDS)
Data was collected through 2021
Early registry data was presented to the FDA during our FDA listening session in March, 2021.
New Registry: Patient Reported Outcomes (PRO)
In April 2022, we had the capacity to relaunch the registry under our own governance. Besides diagnosis, symptoms, medications, and surgeries our emphasis is on collecting high quality patient reported outcomes on itch, sleep quality, impact on family, and general health using validated questionnaires.
Data to be used by researchers
Researchers and industry partners can submit a formal research plan to get access to de-identified registry data. Your data can help to improve knowledge and accelerate treatment developments!
We hit our Wave 1 Goal!
60 participants joined the registry during our first recruitment effort on October 5, 2022. This was a remarkable effort, and shows the interest of the PFIC community in advancing science and research.
Launch of Wave 2
In October 2022 we started to collect follow-up data. These data are important to understand change over time: when did the patient’s treatments change and why, when did their disease symptoms get better, or worse, or were stable. Every enrolled participant is automatically invited via email 6 months after they first filled out the registry. The email provides a personal link to fill out the second wave.
Our Goal over time
The goal for our patient registry is to hit 200 patients registered by 2024! Help us reach this goal by enrolling in the PFIC Network Patient Registry!
Register Online Today
We need your participation in this patient-generated and patient-driven registry. Your voice is crucial to take back power from PFIC yourself and the whole patient community! You can help researchers worldwide by taking 30 minutes of your time to complete your or your child’s profile. Be a part of the community who is helping to advance PFIC research towards treatments and a cure!
For Researchers – Using PNPR Data
Please complete the form below if you are a researcher who wishes to submit a request to utilize de-identified data from the PNPR, or to get permission to reach out to current registry participants who have opted in to be informed about research opportunities. You will need to provide your IRB approved research plan along with the form submission. Our team will review all requests through our governance process.