The PFIC Self Report Registry is your chance to help speed up PFIC research. Everyone’s participation is important. The larger the PFIC Patient Registry is, the more meaningful it can be for researchers from around the world. Read more about the registry below, then sign up as a patient or patient guardian.
What is the Registry?
The PFIC Network Patient Registry is a secure place to store information about those affected by PFIC. All information entered into the registry is de-identified. This means that when a researcher requests information from the registry, no identifying information will ever leave the registry, including name, date of birth, address, or phone number.
All those diagnosed with PFIC are encouraged to participate. Parents can join the Registry to enroll their child. A spouse can participate on behalf of their partner.
The individual’s data collected includes information about diagnosis, family history, quality of life, medications, surgeries, other diseases, and patient demographics. It takes about 30 minutes to complete the patient survey. The registry coordinator is happy to help you complete your survey.
If you or your child appears to be a match for a study or a clinical trial, the registry coordinator will contact you. It would be your choice to connect with the researcher(s) to let them know if you are interested.
Email Registry Coordinator: email@example.com
Why Was It Created?
The PFIC Network 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 Registry has been to add the patient’s voice to every facet of PFIC research and to facilitate and speed up PFIC research towards a cure.
The purpose of the registry is to track PFIC disease status and symptoms, assist in clinical trial recruitment, facilitate drug safety monitoring for new drugs, and allow patient participation at each level of research. The questionnaire is short and simple. The goal is to identify groups of patients for further follow up.
The registry can also assist with our goal to improve the lives of patient 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.
How is Your Data Used?
Researchers, approved by the PFIC Network Patient Registry Team in collaboration with CoRDS, request de-identified registry data for their research, for patient recruitment in clinical trials, and for developing clinical study protocols.
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
- Participating in real-world monitoring for new drug tolerance and effectiveness
How Does It Work?
Patient Driven: The registry is patient-driven. This means that the patient is at the helm. PFIC Network believes that there can be no one 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.
Privacy and Confidentiality: PFIC Network established the registry in collaboration with the Coordination of Rare Diseases at Sanford (CoRDS). The registry is housed on a platform that abides by National Institutes of Health standards for privacy and confidentiality and is reviewed annually by an Institutional Review Board (IRB). No identifying information ever leaves the registry.
If you are a match for a clinical trial, the registry coordinator contacts you, and you are the one to decide whether you would want to participate in the clinical trial. Again, our registry is patient-driven. Patients can choose if they want to participate in a survey, telephone interview, clinical trial, etc.
Register Today: We need your participation in this patient-generated and patient-driven registry. Your voice is crucial for this small and dispersed PFIC community! You can help researchers worldwide by taking less than an hour of your time to complete your, your child’s, or your spouse’s profile. Join the hundreds of men, women, and children who are helping to advance PFIC research towards treatments and a cure!
Click the link at the bottom of this page to receive more information and to register.
We hope you will register today!
Alone we are Rare, Together we are Mighty!
Data Access for Researchers
For Researchers: Access the CoRDs Registry
If you’re a researcher with IRB approval and are interested in accessing the CoRDS Registry, complete the CoRDS Researcher Access Request Form and return it by email.