- Why is the NIH/NCATS GRDR program needed?
- What are common data elements (CDEs), and why are they important?
- Can a patient organization interested in starting a registry collaborate with NCATS through the GRDR program? If so, what are the steps involved?
- Does NCATS provide institutional review board (IRB) services through its GRDR program?
- Can a patient organization that already has a patient registry underway or in development adapt its program to the GRDR program?
- Why should an organization participate in the GRDR program?
- Why is the informed consent document essential to the GRDR program?
- Who owns the data once these are part of the GRDR program?
- Who can access the data to conduct research?
- What type of research projects can be conducted?
- How are patients’ identities protected?
Why is the NIH/NCATS GRDR program needed?
Because many rare diseases share biological pathways, analyses across diseases can speed the development of new therapeutics. To facilitate cross-disease analyses, the GRDR program enables the integration of patient data from multiple rare disease registries in a standardized manner, and the data are mapped to GRDR program common data elements and other national standards. These efforts are designed to advance research for rare diseases and, through application of scientific insights gained, to further research for common diseases.
What are common data elements (CDEs), and why are they important?
A CDE consists of both a precisely defined question and a specified format or set of permissible responses. Using CDEs ensures data are defined in the same way, using the same standards and with the same vocabulary, so that the data can be used across diseases or registries.
Can a patient organization interested in starting a registry collaborate with NCATS through the GRDR program? If so, what are the steps involved?
Although establishing new registries is not part of the GRDR program,NCATS staff have developed a number of tools and services to help patient organizations establish registries and participate in GRDR program. Examples of such tools include:
- A set of CDEs.
- A template informed-consent form (Word - 40KB) for participating in patient registries, which can be modified according to the specific needs of each group.
- Global unique identifier (GUID) software.
Does NCATS provide institutional review board (IRB) services through its GRDR program?
Yes, GRDR staff can provide complimentary IRB services to participating organizations through the NIH IRB.
Although not specifically endorsed by NCATS, organizations participating in the GRDR program can use external IRBs, for example:
Can a patient organization that already has a patient registry underway or in development adapt its program to the GRDR program?
Any organization with a registry under development can incorporate the GRDR program CDEs. The CDEs should include all the elements needed to generate the GRDR program GUID. The informed consent forms of participating organizations must include patients’ permission to share their de-identified information with the GRDR program for broad use (i.e., for any disease). Also, the registry protocol must be IRB-approved.
Why should an organization participate in the GRDR program?
Patients and their families:
- Increase awareness of their specific rare disease.
Physicians and nurses:
- Facilitate contact with others who are dealing with the same issues and compare notes.
- Provide access to outcomes data.
- Provide awareness about novel treatment regimens.
- Foster collaboration between all stakeholders.
Rare disease organizations:
- Provide resources and tools and map data to national standards to facilitate interoperability with other databases.
For investigators and industry:
- Allow free access to curated and standardized clinical and genomic data.
- Facilitate the recruitment of patients to multicenter clinical trials for rare diseases.
Why is the informed consent document essential to the GRDR program?
The GRDR program is designed for interaction only with the registry representing the patient (as opposed to individual patients). Participating organizations sharing de-identified patient data must first obtain permission from their patients to share data with the program.
Informed consent includes two main steps:
- Patients receive an explanation of the nature of their involvement in the registry, study or procedure. They are given information about the risks and benefits as well as the option to change their minds and opt out of participation.
- Once patients have a full understanding of their involvement, they are required to sign a form to document their understanding and agreement.
More information about the informed consent process can be found at the following links:
- Informed consent and patient registry for the rare disease community: Editorial. Contemp Clin Trials. 2012;33(1):3–4.
- Informed consent template for patient participation in rare disease registries linked to biorepositories. Rare Dis Orphan Drug. 2014;1(2):69–74.
Who owns the data once these are part of the GRDR program?
The GRDR program does not control or own any registry or its data. The contributing organization retains ownership and control of its registries. De-identified patient data shared by the registry owner will be mapped to national standards, integrated and stored in the GRDR data repository. De-identified data that have been shared and integrated into the GRDR program belong to the community, under the NIH auspices, which protects the data and the confidentiality of the patients according to federal regulations and NIH policies.
Who can access the data to conduct research?
The GRDR program data is accessible to all users who agree to the policies for its use. There are two levels of access:
- Open Access, which provides summaries, statistics, trends and general information. There is no process of approval for this access.
- Controlled Access, with which investigators can access detailed information about patients to enable the design of new research studies. In this case, the investigator must submit an IRB-approved project and sign a confidentiality form. The GRDR program has no information about, nor access to, the identity of the patient. Investigators can contact the patient only through the contributing registry.
What type of research projects can be conducted?
Once the GRDR data are accessible, the data can be used for various types of studies such as epidemiological, basic science, natural history, comparative effectiveness, and clinical studies and trials across many diseases. As more patient registries are added to the program over time, the range of studies that could be conducted may increase.
How are patients’ identities protected?
Each registry, before submitting data, must remove all patient identifiers, according to the Department of Health and Human Services regulations at 45 C.F.R. Part 46. Data submitted with patients’ identifiers will be discarded and will not be processed.
In addition, to tracking each set of patients’ data over time and across diseases, registries, studies and countries, a GRDR program GUID will be assigned to each data set using an encrypted one way hash code. The code for the patient is saved only within the contributing registries and will never be shared with the GRDR program. Patient recruitment for subsequent studies can be facilitated only through the contributing registry.