Conference Coverage

C-Path and NORD team up to speed development of treatments for rare disorders


Those suffering from rare diseases could see treatments being developed at a faster pace thanks to a research tool developed by the National Organization for Rare Disorders (NORD) and the Critical Path Institute (C-Path). Developed with grant money from the Food and Drug Administration, the platform, currently named the Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP), is designed to “promote the sharing of existing patient level data and encourage the standardization of new data collection,” according to information provided at a launch event held Sept. 18, 2019.

By integrating data in a regulatory-grade format suitable for analytics, the RDCA-DAP hopes to accelerate the understanding of disease progression – including source of variability to optimize the characterization of subpopulations – develop clinical outcome measures and biomarkers, facilitate the development of mathematical models of disease, and promote innovative clinical trial designs.

The RDCA-DAP works as a database that will house patient-level data from a variety of sources, including clinical trials, longitudinal observational studies, patient registries, and other sources, such as real-world data collected from electronic health records across a wide range of rare diseases from all over the world. Data will then be made available to researchers to help speed the development of new treatments.

“The database and analytics we are creating will enable us to obtain new insight into these rare diseases,” C-Path President and CEO Joseph Scheeren, PharmD, said at the launch event. “Not only within specific diseases, but also we hope across related diseases.”

Dr. Joseph Scheeren, president and CEO, Critical Path Institute Gregory Twachtman/MDedge News

Dr. Joseph Scheeren

The key to the platform’s success will be data. “We need access to clinical data from the industry, patient groups, and academia,” Dr. Scheeren said. “Even more so, the RDCA-DAP will need to incorporate data from many sources. In addition to data from clinical trials conducted by industry and academia, we will want to access data from patients, hospitals, and any organization that can provide data.”

Dr. Scheeren said the data will take many forms, including numeric data, images, genomic information, and other forms of clinical information.

“The database will be able to handle these diverse datasets,” he said, adding that C-Path is preparing to be able to analyze the data sets “with the most sophisticated tools available.”

Dr. Scheeren made a call for all interested stakeholders with rare disease data to contribute to the platform.

Peter Saltonstall, president and CEO, NORD Gregory Twachtman/MDedge News

Peter Saltonstall

NORD President and CEO Peter Saltonstall echoed that call. “We need data. We are accepting it immediately.”

Janet Woodcock, MD, director of the FDA Center for Drug Evaluation and Research, applauded the new platform. “I think foundations and patient advocacy groups and others that have been trying to help in this space have realized that simply funding basic research, although it is necessary and really important, it is not enough to get those therapies in the hands of doctors and patients,” she said. “You have to enable translation of research for that disease.”

Dr. Janet Woodcock

Dr. Woodcock noted that the cures may not even come from that basic research, but rather from “left field” using research into cancer or another disease state, something that will be enabled by the disease-agnostic platform being created by C-Path and NORD. She said that the platform will not only put all the data in one spot, but will help to create a standardized set of disease definitions to help make the data useful across all research.

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