One of the most important purposes of the IAMRARE platform is to bring rare disease communities together and collect data which could be used to inform future clinical trials to create therapeutics and improve the quality of life for patients. Individual patient advocacy groups (sponsors) can establish patient registries specific to their rare disease, to be hosted on the IAMRARE platform. Some other goals of a patient registry may be to:
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.
A natural history study is a study designed to track the course of a disease over time and includes people who have a specific medical condition or disease and those who are at risk of developing such. This method of research explores the disease in a comprehensive way and identifies demographic, genetic, environmental, and other variables that correlate with the disease and its outcomes. Natural history studies have many potential uses such as patient care best practice developments and clinical trial recruitment.
The data collected includes but is not limited to:
Data is collected through a secure web-based system developed by the National Organization for Rare Disorders, Inc. (NORD®), an independent non-profit committed to the identification, treatment, and cure of all 7,000 rare diseases. Study participants respond to questions grouped within a series of surveys developed per study standards and in collaboration with disease specific experts.
An individual, company, institution, or organization that takes responsibility for choosing appropriately trained and experienced researchers as well as the initiation, management, and/or financing of a research study. The study sponsor ensures that the study is conducted in a reputable manner and upholds regulations as they apply to the study. For IAMRARE registries, many of the registries are sponsored by patient organizations.
The Principal Investigator is the research group leader or, the person with the primary responsibility for the design and conduct of the research project or study.
A study participant is the individual who takes part in a research study and whose information is collected for that research. Study participants may consent to enter and share their own personal data.
A reporter/respondent is an individual who completes the surveys on behalf of the patient/study participant, when they are unable to do so on their own behalf.
An individual who is authorized under applicable law to consent, on behalf of a prospective subject, to the subject’s participation in the clinical trial. The LAR may be a parent, grandparent, spouse, caregiver, or guardian who has the legal authority to grant consent on behalf of another who is eligible to participate in research. When a LAR acts on behalf of a study
participant, they are considered to be the reporter/respondent in the research.
The Office for Human Research Protections (OHRP) states that, “… the informed consent process is the critical communication link between the prospective human subject and an investigator beginning with the initial approach of an investigator to the potential subject (e.g., through a flyer, brochure, or any advertisement regarding the research study) and continuing until the completion of the research study. […] The informed consent process involves three key features:
Simply put, Informed Consent is the process by which a study sponsor informs a potential research participant about they study and gives them the information they need to make a decision about participating.
Patient natural history studies hosted on the IAMRARE platform are open to open to anyone who has the specific diagnosis and meets the individual study inclusion criteria for a particular study.
There is no cost to the patient to join this study. The Sponsor absorbs the cost of the registry for its members.
A registry on the NORD platform will typically be open for at least five years. Participants may be asked to return to the registry periodically to update their information.
The patient registry uses an online platform which allows participants to contribute data from anywhere in the world. Individuals from other countries who enter data into the registry
1 Informed Consent FAQs: https://www.hhs.gov/ohrp/regulations-and-policy/guidance/faq/informed-consent/index.html.
Accessed Feb. 9, 2021. should be aware that data and privacy laws are different in the U.S. from other countries. This U.S. based registry will protect data and privacy according to U.S. requirements.
The data is stored on NORD’s secure registry platform system which adheres to industry standards regarding security protections.
The registry follows strict government guidelines to assure patient information is protected. The platform is served over HTTPS, which provides traffic encryptions so as to prevent
eavesdropping and man-in-the-middle attacks. Communications between the registry platform application server and the database are also encrypted. As with any information you provide electronically, there is a risk that your privacy could be compromised, however the registry is designed to minimize the chance of this occurring.
The identifiable and de-identifiable data are owned by the study sponsor. The Sponsor decides how and with whom to share the data. A subset of the pseudonymized data collected across the NORD Registry Platform is available to NORD to support cross disease analysis and advocacy activities to members of the rare disease community as a whole. Participants are able to withdraw from the study at any time, however, the researchers may still use the information that they have collected prior to changing your mind in order to complete the research that has already started. Information that has already been shared with the RDCA-DAP or sent to a researcher for a specific study prior to your request for removal cannot be retrieved or removed.
A Registry Advisory Board committee, that may include scientists, doctors, and patient advocates, will be assembled to oversee the conduct of the study. The Advisory Board will review aggregate registry data and the use of this registry, ensure proper evaluation of protocols requesting to use registry data and/or contact registry participants, and will review any protocol or confidentiality deviations on a case by case basis and ensure that any such deviations are reported to the IRB.
Any board, committee, or other group formally designated by an institution or investigator to review, approve the initiation of, and to conduct periodic review of research involving people. The primary purpose of such review is to assure the protection of the rights and welfare of the participants in the study. Also known as Ethics Committee (EC).
The registry is maintained by NORD who hosts the registry on its cloud-based Platform and provides oversight and ongoing support of the system. The Sponsor provides the day-to-day management of their patient registry, including the development and adherence to the study procedures.
IAMRARE is designed to be easy-to-use, scalable, and accessible from any internet connection around the world. Individuals living with a rare disease, or their caregiver, may answer the survey questions once enrolled.
Participant data is made anonymous and aggregated. Study data may be shared with individuals or institutions conducting research, as approved by the study’s governing body known as the Institutional Review Board that includes scientists, doctors and patient advocates
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