Funded by FDA, C-Path and NORD to Launch Rare Disease Data Analytics Platform

The goal of the FDA-funded platform is to inform clinical trial design and accelerate therapy development for rare diseases. The project will kick off at a launch meeting in September.

Aug 7, 2019 | C-Path, NORD | PR Newswire

PRESS RELEASE – The Critical Path Institute (C-Path) and the National Organization for Rare Disorders® (NORD) will host a meeting onTuesday, September 17 in Bethesda, MD to formally launch development of a new rare disease data and analytics platform. Funded by a cooperative agreement through the Food and Drug Administration, [Critical Path Public-Private Partnerships Grant Number U18 FD005320 from the US Food and Drug Administration] the goal of the platform is to accelerate the movement of therapies from bench to bedside for rare diseases. The platform will provide the infrastructure for a sustainable, cooperative scientific approach to clinical trial readiness in rare diseases by addressing vast knowledge gaps about the natural course of disease, the clinical evaluation of new treatments, and patients’ perspective on disease and treatment.

The Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP) will provide a centralized and standardized infrastructure to support and accelerate rare disease characterization with the goal of accelerating therapy development. The robust integrated platform will include integrated rare disease data from various sources such as clinical trials, observational studies, real world data and patient registries — including those within NORD’s IAMRARETM registry platform — and an analytics platform that will allow efficient and effective interrogation of that data to generate solutions to inform clinical trial design and regulatory review.

“For people living with rare diseases, time is of the essence,” said Joseph Scheeren, PharmD, C-Path President and Chief Executive Officer. “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.” 

“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, NORD President and Chief Executive Officer. “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.”

Meeting attendees will include representatives from across the community including regulators, patient organizations, clinicians, researchers and pharmaceutical companies interested in rare disease drug development. Attendees will have the opportunity to learn about the goals of the RDCA-DAP and how to engage in the early stages of this effort.

Register for the meeting here: https://bit.ly/2YOj3jl

TRNDS 2019

University of Rochester
Saunders Research Building
Helen Wood Hall Auditorium
255 Crittenden Blvd
Rochester, NY 14642

Date: Friday, September 13
Time: 7:30 am – 4:00pm

TRNDS News

Sign-up for TRNDS updates and rare disease news

  • New Grants will Accelerate Clinical Trials in Rare Neurological Disorders
    READ MORE
  • Neurogene Offers Access to Genetic Testing for Lysosomal Storage Disease
    READ MORE
  • NIH launches 5-year, $10 million study on acute flaccid myelitis
    READ MORE
  • Study refines ALS risk among first-degree relatives of patients with disease
    READ MORE
  • NCATS Director Warns of 'Poorly Understood Public Health Implications of Rare Diseases'
    READ MORE
  • Speeding Up Drug Discovery for Brain Diseases
    READ MORE
  • NIH-Funded Project Aims to Build a ‘Google’ for Biomedical Data
    READ MORE
  • Rare Disease Groups Seek Public Support to Renew Newborn Screening Act in Senate
    READ MORE
  • Jsyne Gershkowitz, Amicus Therapeutics, TRNDS 2019
    (Podcast) Get to know Jayne Gershkowitz, Chief Patient Advocate at Amicus Therapeutics
    READ MORE
  • PJ Brooks, NCATS, NIH, TRNDS
    Meet TRNDS 2019 Speaker PJ Brooks
    READ MORE
  • Rare Pediatric Disease PRVs: FDA Updates Guidance
    READ MORE
  • Batten Disease Study
    Neurogene Opens Natural History Study of CLN7, CLN5 Diseases at UT Southwestern
    READ MORE
  • C-Path and NORD Collaborate to Launch Rare Disease Data Platform
    READ MORE
  • TRNDS 2019 speaker Tauna Batiste, BDSRA
    Meet TRNDS Speaker Tauna Batiste
    READ MORE
  • HD Patients Prefer Less Invasive, No-Placebo Gene Therapy Trials
    READ MORE
  • Evidation & Eli Lilly Study Uses Apple Devices, Apps to Predict Cognitive Impairment
    READ MORE
  • 'Dr. Google' Helps Some Patients Diagnose a Rare Disease
    READ MORE
  • Antisense Drugs for HD, ALS & Prion Could Meet the Need for Brain Treatments
    READ MORE
  • Ultragenyx Partners with GeneTx to Advance Treatment for Angelman Syndrome
    READ MORE
  • Netflix's Medical Investigation Docuseries 'Diagnosis'
    READ MORE
  • NIH All of Us Project Tops 270,000 Sign-ups
    READ MORE
  • FDA Cancer Office Taps Syapse for Real-world Evidence Development
    READ MORE

Latest Tweets

TRNDS Speakers

Room Block

Hilton Garden Inn
Rochester/University
Medical Center

30 Celebration Drive
Rochester, NY 14620

Phone: 585-424-4404

TRNDS Rate: $149/night