This Collaborative Platform Aims to Speed up Rare Disease Drug Development

In a recent webinar hosted by the National Organization for Rare Disorders (NORD), a number of speakers discussed an up and coming project spearheaded by the organization: the Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP). This project is being developed in partnership with the Critical Path Institute (C-Path) and the US Food and Drug Administration (FDA). The goal of this platform is to improve and speed up the process of developing therapies for rare diseases.

Michelle Campbell, PhD, of the FDA’s Center for Drug Evaluation and Research, kicked off the webinar by discussing some of the challenges of rare disease drug development:

  1. The general lack of information for many rare diseases means that making effective therapies is inherently more time-consuming and less successful.
  2. Standard protocols for research studies and trials are often not-well suited to rare disease.
  3. Research (if present) is often scattered and poorly coordinated.

The RDCA-DAP aims to help rectify some of these difficulties by standardizing and centralizing the infrastructure and processes for developing therapies, as well as optimizing the characteristics of research studies.

Jane Larkindale, DPhil, of C-Path, says that while rare disease research is often disjointed, a wealth of research studies already exist and there is a lot of data out there that could be utilized more effectively. Gathering and centralizing all of this information will be critical to the platform’s success. Since data is often owned by a variety of different stakeholders, a ‘data custodian’ will help negotiate with the various institutions to come to an agreement about how the data can be used.

This data will be consolidated and integrated with the ultimate goal of improving research and speeding up drug development.

NORD’s Director of Research Vanessa Boulanger, MSc, says that one of the most valuable resources for the RDCA-DAP will be patient data registries. Some of the most organized rare disease communities have registries of patient data and records, which have become critical tools for scientific research and will be one of the most effective data resources.

There were also a few questions from the audience:

  • How will data from different sources (but the same patients) be reconciled? How can data remain de-identified?

Larkindale says that while all data will be de-identified, the partners are still working on how to account for potential repeated data input. The platform will also aim to have differing levels of anonymity based on each disease and the nature of the data (patient genomes, etc).

  • How can patient organizations contribute and help speed up development?

Input and feedback from patient organizations will be crucial because they are among the most trusted voices in the rare disease community. Contributing to patient registries and natural history studies can play a major role as these sources contain some of the most important data.

To learn more about the RDCA-DAP, click here.

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