Frequently Asked Questions
IPPF Natural History Study
Frequently Asked Questions
1. What is a Patient Registry?
A patient registry is a collection of standardized information about a group of patients who share a condition and is used for a variety of purposes such as conducting natural history studies and supporting disease-specific clinical trial recruitment.
2. What is a Natural History Study?
A natural history study is designed to track the course of a disease over time, including those who have the condition or are at risk of developing it. This research method explores the disease comprehensively, identifying demographic, genetic, environmental, and other variables that correlate with the disease and its outcomes.
3. What is a Research Study Sponsor?
An individual, company, institution, or organization that takes responsibility for the initiation, management, and/or financing of a clinical trial. They ensure the study is conducted properly and complies with applicable regulations.
4. Who is the International Pemphigus and Pemphigoid Foundation (IPPF)?
The IPPF aims to provide information and support to patients and doctors worldwide about pemphigus and pemphigoid, helping patients and their caregivers lead active, productive lives.
5. Who is NORD® – the National Organization for Rare Disorders Inc.?
NORD®, an independent nonprofit, leads efforts to improve the lives of rare disease patients by supporting the community, accelerating research, raising awareness, and influencing public policy. Learn more about NORD.
6. What is a Principal Investigator?
The Principal Investigator is the leader of the research group or the person primarily responsible for the design and conduct of the research project.
7. What is an Institutional Review Board (IRB)?
An IRB, also known as the Ethics Committee, is a group formally designated to review, approve, and periodically oversee research involving human subjects to ensure their rights and welfare are protected.
8. What is the purpose of the IPPF Natural History Registry Study?
The study aims to gather data from the pemphigus and pemphigoid community to develop therapeutics and improve patient quality of life, among other goals. It includes conducting a detailed natural history study and assisting in the development of care standards and clinical trials.
9. What types of data will be collected in the IPPF Natural History Registry Study?
Data collected includes socio-demographics, medical diagnostics, treatment progression, care management, and quality of life information.
10. How is the data collected?
Data is collected through a secure web-based system developed by NORD®, involving a series of surveys filled out by study participants.
11. Who is a study participant?
Study participants are individuals who consent to share their personal data for research purposes.
12. Who is a reporter/respondent?
A reporter/respondent is someone who completes the surveys on behalf of a study participant who is unable to do so themselves.
13. What is a legally authorized representative (LAR)?
A LAR is an individual legally authorized to consent on behalf of a study participant, such as a parent, spouse, or caregiver.
14. What is an Informed Consent?
Informed consent is a critical communication link between a researcher and a potential study participant, ensuring the participant understands and voluntarily agrees to partake in the research.
15. Who can join the study?
The study is open to anyone diagnosed with Pemphigus or Pemphigoid.
16. Is there a cost to participate?
There is no cost for patients to join the study; costs are absorbed by the IPPF.
17. What is a longitudinal study?
Longitudinal studies are observational studies that repeatedly measure variables over a long period to assess the outcomes and developments of conditions and treatments.
18. How often will I need to provide my data for the study?
Data collection frequency varies from one-time to annually, depending on the survey and data needed.
19. How long will this study last?
The registry is planned to be open for at least five years, with renewal options available.
20. Can data be collected worldwide?
Yes, the online platform allows for worldwide data contribution, though participants should be aware of differing data and privacy laws.
21. Where is the data stored?
Data is stored on NORD’s secure registry platform, adhering to strict security standards.
22. Is the data safe?
The registry follows stringent guidelines to protect patient information, using encrypted communications to ensure data security.
23. Who owns the data?
The data is owned by the IPPF, which decides on data sharing and usage. Participants can withdraw anytime, but data already used in research remains utilizable.
24. What is a registry Advisory Board?
The Advisory Board, including scientists, doctors, and patient advocates, oversees the study’s conduct, reviewing data use and ensuring protocol compliance.
25. How is the Patient Registry maintained?
The registry is maintained by NORD, hosting it on its cloud platform, with the IPPF managing daily operations and adherence to study procedures.