Frequently Asked Questions
ITP Natural History Study Registry
FAQ for ITP Natural History Study Registry
Thank you for your interest in ITP Natural History Study Registry. Please see below some frequently asked questions. If you have additional questions that are not listed, please contact us!
What is the purpose of the ITP Natural History Study Registry?
The purpose of the ITP Natural History Study Registry is to bring the immune thrombocytopenia (ITP) community together and collect data.
Some of the goals of the ITP Natural History Study Registry are:
- To describe the people who have immune thrombocytopenia (ITP) and better understand the stages of the disease and the different ways the disease affects people, including diagnosis, treatment, medical history, social and economic environment, and treatment outcomes.
- To understand how immune thrombocytopenia (ITP) changes over a person’s lifetime and to learn about clinical practice patterns and variations over the course of treatment.
- To help develop best practices, management guidelines, and recommendations so clinicians can provide the best care to improve quality of life and outcomes for people with ITP.
- To identify people with immune thrombocytopenia (ITP) who may be willing to take part in other research studies or clinical trials. Participants may choose whether they want to hear about these opportunities.
What is a Patient Registry?
A patient registry is a collection of standardized information about a group of patients who share a condition. The information may be used for a variety of purposes such as conducting natural history studies and supporting disease-specific clinical trial recruitment.
What is a Natural History Study?
A natural history study is designed to track the course of a disease over time. It includes people who have a specific medical condition or those who may be at risk of developing the condition. This type of research identifies demographic, genetic, environmental, and other information common within a disease and its outcomes.
Natural history studies can also show differences in symptoms and disease progression among individuals with the same condition. They support patient care best-practice development and clinical trial recruitment and are often conducted through patient registries.
How is the data collected?
Data is collected through a secure, web-based application developed by the National Organization for Rare Disorders (NORD®). The platform can be accessed by computer, tablet, or phone. Study participants respond to questions grouped within a series of surveys developed according to study standards and in collaboration with disease-specific experts.
What types of data will be collected in the ITP Natural History Study Registry?
Data collected includes, but is not limited to:
- Socio-demographics
- Medical history and diagnostics
- Treatment and disease progression
- Management of care
- Quality of life
What is a Research Study Sponsor?
A Research Study Sponsor is an individual, company, institution, or organization responsible for choosing qualified researchers, initiating and managing the study, funding study activities, and ensuring the study is conducted ethically and in compliance with regulations.
The sponsor of this registry is the Platelet Disorder Support Association (PDSA).
Who is the Platelet Disorder Support Association (PDSA)?
The Platelet Disorder Support Association is dedicated to enhancing the lives of people with immune thrombocytopenia (ITP) and other platelet disorders through education, advocacy, research, and support.
What is a Principal Investigator?
The Principal Investigator (PI) has primary responsibility for the design and conduct of the research study and oversees all aspects of the Registry, its staff, and the research conducted using registry data.
Who is a Study Participant?
A Study Participant is the individual about whom information is entered into the registry. If the individual is of legal age and able, they will consent and enter their own information. If not, a caregiver or legally authorized representative (LAR) may provide consent and enter data on their behalf.
What is a Legally Authorized Representative (LAR)?
A Legally Authorized Representative (LAR) is someone authorized under applicable law to consent and enter data in the registry on behalf of another individual. This may include a parent, grandparent, spouse, caregiver, or guardian with legal authority.
What is a Designated Representative?
A Designated Representative is a legal adult who was the caretaker of an individual who passed away from immune thrombocytopenia (ITP). This person must have participated in and had knowledge of the deceased individual’s medical care and may enter retrospective data on their behalf.
What is an Informed Consent Form (ICF)?
An Informed Consent Form provides potential participants with key information about the registry, including risks, benefits, data use, and privacy. Participants must electronically sign the ICF before entering data or completing surveys.
After consenting, can a participant withdraw from the study?
Yes. Participants may withdraw from the study at any time. However, data already collected and shared with researchers prior to withdrawal cannot be retrieved or removed.
What is an Institutional Review Board (IRB)?
An IRB is a board designated to review, approve, and periodically review research involving people. Its primary purpose is to protect the rights and welfare of study participants. It may also be called an Ethics Committee (EC) or Research Ethics Board (REB) in Canada.
What is a Registry Advisory Board?
A Registry Advisory Board may include scientists, physicians, and patient advocates. The board oversees study conduct, advises on survey development, reviews use of registry data, evaluates research requests, and ensures protocol or confidentiality issues are reported to the IRB.
Who can join the study?
This study is open to anyone with an immune thrombocytopenia (ITP) diagnosis who meets the study’s inclusion criteria.
Is there a cost to participate?
There is no cost to participate in this study.
Is there payment for participating?
Participants are not paid for the information they provide.
How long will this study last?
Registries on the IAMRARE® platform typically remain open for at least five years. Participants will be asked to return periodically to update their information.
Can data be collected worldwide?
Yes. The online platform allows participants from anywhere in the world to contribute data. Data and privacy protections follow U.S. requirements, which may differ from laws in other countries.
What are the GDPR considerations?
Participants outside the United States receive the same privacy protections. Residents of the European Union and Switzerland have additional rights, including access, correction, deletion, portability of personal data, restriction of processing, and the right to lodge complaints with supervisory authorities.
Where is the data stored?
NORD stores registry data on encrypted servers and/or encrypted third-party servers hosted in Canada. Regular backups are performed, and systems comply with U.S. and international regulations, including GDPR.
Is the data safe?
Yes. The registry follows strict government guidelines to protect patient information. Data is encrypted in transit and at rest, and communications between application servers and databases are encrypted.
Who owns the data?
The study data are owned by the Platelet Disorder Support Association (PDSA). PDSA determines how and with whom data are shared. NORD staff may access data for platform support and participation metrics as outlined in the informed consent.
Who will have access to Protected Health Information (PHI)?
Access to PHI is limited to approved research team members, NORD technical staff (when necessary), and IRB-approved cross-disease research activities with sponsor approval. Researchers receive only the minimum data necessary and must sign confidentiality agreements.
How is the registry maintained?
NORD hosts and maintains the registry platform and provides technical support. The Platelet Disorder Support Association (PDSA) manages day-to-day registry operations.
Who is NORD – the National Organization for Rare Disorders, Inc.?
NORD is an independent nonprofit organization working to improve the lives of rare disease patients and families by supporting research, education, advocacy, and public policy. Learn more at rarediseases.org.