Rare Disease Cures Accelerator – Frequently Asked Questions

What is the Rare Disease Cures Accelerator-Data and Analytics Platform?

The Rare Disease Cures Accelerator–Data and Analytics Platform (RDCA-DAP^®) is a database containing information about many rare diseases, coupled with an analytical framework to help understand that data. RDCA-DAP helps us to understand how rare diseases progress and how to best assess patient progression to inform the effects of future therapies with the goal of accelerating drug development for rare diseases.

Why is RDCA-DAP important to the rare disease community?

RDCA-DAP is a resource through which researchers and drug developers can access rare diseases data to analyze that data and develop new insights and discoveries about the diseases and how they progress. It also provides a way to develop new tools and methodologies to improve clinical trial design and empower the rare disease community. This will result in faster, more effective clinical trials and more rapid (and cheaper) development of new drugs.

Each rare disease, and even subsets of rare diseases, is different. What is the value in aggregating data from multiple rare diseases?

Each rare disease is unique, and it is important to understand the progression of each one individually. However, there are many aspects of rare diseases that may be common to several disease states and learnings can be applied across diseases. For example, tools, such as biomarkers, endpoints, outcome measures, etc., developed for a given disease, might be adapted and applied to a different but related rare disease even with less supportive data at our disposal. This is of particular value in disease areas where there are very few patients and limited knowledge, and where there is no precedent in terms of what to measure and when.

How do I initiate sharing data with the platform?

Data may be contributed to RDCA-DAP from multiple sources, including IAMRARE® registries. Contact [email protected] if you would like to explore sharing data with the project or fill out a contact form at https://portal.rdca.c-path.org/contribute-data.

Can small disease communities use data in RDCA-DAP for their own independent research?

Researchers, including researchers from patient communities, can access data that has been approved for sharing and utilize statistical, analytical and visualization tools. Request a login and explore the database at https://portal.rdca.c-path.org/.

Does this compete with existing observational (natural history) studies, clinical data collections or patient registries?

Because RDCA-DAP aggregates data already collected but does not collect data, it works in tandem with these efforts, including through collaboration with patient groups collecting prospective data through NORD’s IAMRARE® platform. Working with such groups ensures the highest possible data quality in new prospective studies, the ability to integrate such data in the future, and to ensure common use of data standards. RDCA-DAP can also convert existing data from prospective studies into regulatory-ready formats and share that information back with those collecting the data.

Who owns the data contributed to RDCA-DAP?

The original custodian of the data (the person or institution that shares the data with RDCA-DAP) retains ownership. They dictate how RDCA-DAP may use the data through a legal agreement signed with the platform called a Data Contribution Agreement. When users of the platform are authorized to access the patient data, they need to sign and obey the terms and conditions of the Data Use Agreement.

How are data contributed to RDCA-DAP kept secure?

Before data is shared, an agreement is signed with a data custodian, or organization conducting the research study. This agreement confirms that patients have allowed their data to be shared and establishes what can be done with the data. Once this agreement is in place, de-identified data can be safely moved into RDCA-DAP. Data in RDCA-DAP are encrypted from the point that they transfer to RDCA-DAP from the original location. Access is strictly regulated and limited to those with permission to see specific data. The platform itself is hosted in a high-security cloud infrastructure, and security is checked regularly.

Can a patient affected by a rare disease participate directly in RDCA-DAP?

RDCA-DAP is currently set up to integrate existing datasets, which have been collected through clinical trials, other studies or registries. RDCA-DAP is not set up for prospective data collection from individual patients at this time. However, any patient diagnosed with a rare disease can participate in RDCA-DAP by taking part in a clinical study or registry of patients for their condition and encouraging the group collecting the data to share with RDCA-DAP. If you are interested in contributing in this way, we recommend that you contact NORD or a patient organization for your specific condition, who will be able to identify any ongoing data collections.

How will the FDA be reviewing and utilizing the data coming out of RDCA-DAP?

FDA will have input into the development of the platform to assure that the aggregated data and the interpretation of those data will have the most impact on innovating therapy development for rare diseases. FDA will have access to analyses of the data as will other researchers according to the governance procedures. FDA will also potentially be asked to review select data sets as evidence packages for the evaluation of new tools (e.g., quantitative models, biomarkers, clinical outcome assessment instruments, etc.)