NORD and C-Path Announce Rare Diseases Data and Analytics Platform

Joseph Scheeren, PharmD, president and chief executive officer, C-Path

Joseph Scheeren, PharmD

This fall, a combination meeting hosted by the Critical Path Institute (C-Path) and the National Organization for Rare Disorders (NORD) will be held to initiate the launch of a new rare disease data and analytics platform aimed at accelerating the development of new therapies.

The Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP) is funded by a cooperative agreement through the FDA (Grant Number U18 FD005320), and will provide a variety of solutions, including filling the gaps in the educational groundwork for sustaining a cooperative, scientific approach to rare disease clinical trials. According to NORD, the platform will also offer patient perspectives on disease and treatment.

"For people living with rare diseases, time is of the essence," Joseph Scheeren, PharmD, president and chief executive officer, C-Path, said in a statement. "By leveraging the rare disease community access and data of NORD and the data curation, aggregation, governance and advanced analytics expertise of C-Path, we are poised to make a significant impact on rare disease drug development by providing quality data that will inform clinical trial design and accelerate the development of therapies."

The meeting is planned to be held in Bethesda, Maryland on September 17, 2019. Attendees are expected to include representatives from across the medical community, and they will be able to learn about the goals of the RDCA-DAP and how to get involved on the ground floor. Regulatory members, patient organizations, clinicians, researchers, and pharmaceutical companies are all anticipated to be in attendance.

"Currently, more than 25 million people in the United States are affected by one or more of the over 7,000 rare diseases," stated Peter L. Saltonstall, president and chief executive officer, NORD. "Drug development for these diseases is often impeded due to the low affected patient numbers and a limited understanding of how rare diseases progress or how to measure clinical improvements. FDA-approved treatments exist for only 10% of rare diseases; with this collaboration, we can change that statistic for the better for our rare community."

Earlier this year, Sika Dunyoh, director of education programs, NORD, told NeurologyLive that there is a spectrum of educational needs across the provider community, ranging from general information on these diseases to facets of diagnosis.

“There are also educational needs that are very disease-specific like with SMA, Duchenne, Huntington disease, and other diseases where there needs to be education that can quicken diagnosis and improve clinical care,” Dunyoh said. “Across the board, we're trying to identify what those needs are and develop CME programming that will help move the needle on some of these issues.”

Those interested can register for the meeting by clicking here.

REFERENCE

Funded By FDA, C-Path And NORD To Launch Rare Disease Data Analytics Platform [press release]. Tuscon, AZ, and Washington; C-Path and NORD; Published August 7, 2019. prnewswire.com/news-releases/funded-by-fda-c-path-and-nord-to-launch-rare-disease-data-analytics-platform-300898014.html. Accessed August 7, 2019.