Overview

Critical Path Institute’s Rare and Orphan Disease Program is dedicated to addressing the unmet needs of rare and orphan diseases, including Duchenne muscular dystrophy, rare neurodegenerative conditions such as ALS, Huntington's disease, ataxias and more. Utilizing C-Path's core competencies, the program endeavors to devise drug development tools and other solutions endorsed by regulatory bodies like the FDA and EMA. These resources are accessible to developers, bolstering the progress of treatments and potential cures. Moreover, the program ensures the inclusion of individuals and families with lived experience at every stage.

Workspaces Overview

Rare Disease Drug Development

Over 350 million people in the world have one of more than 10,000 rare diseases. However, only about 600 or 10% of rare diseases have an FDA-approved treatment available, and drug development is frequently slowed by the low numbers of patients and limited understanding of the variability and progression of each disease. This makes evaluating the efficacy and safety of clinical trial designs challenging. Developing a clear understanding of how each disease progresses would allow for the development of clinical trial protocols that could better evaluate new therapeutics. This would accelerate clinical development, make it less expensive, and encourage new companies to develop rare disease drugs.

A Leading Data and Analytics Platform

The Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP®) is central to the success of the Rare and Orphan Disease Program. RDCA-DAP is an FDA-sponsored initiative that provides a centralized and standardized infrastructure to support and expedite rare disease characterization, with the goal of advancing drug development solutions to accelerate the development of treatments for rare and orphan diseases. RDCA-DAP promotes the sharing of existing patient-level data and encourages the standardization of new data collection. By integrating data in a format suitable for analytics, RDCA-DAP accelerates the understanding of disease progression (including sources of variability to optimize the characterization of subpopulations), clinical outcome measures and biomarkers, and facilitates the development of mathematical models of disease and innovative clinical trial designs.

You can find more information about RDCA-DAP here.

How RDCA-DAP Works

RDCA-DAP houses integrated patient-level data from diverse sources across a multitude of rare diseases. Data are contributed from different organizations and companies around the world. C-Path has extensive experience in building such integrated databases for many diseases, including existing rare disease databases (e.g. Duchenne muscular dystrophy, Huntington’s disease, Friedreich’s ataxia and polycystic kidney disease). Together with other rare and orphan disease consortia within C-Path, RDCA-DAP has grown to host a variety of diseases areas with more than 40 rare and orphan diseases currently represented.

C-Path has partnered with the National Organization for Rare Disorders (NORD) to leverage its IAMRARE™ registry platform and extensive expertise to help identify data contributors and establish contacts with the contributing organizations. C-Path negotiates data contribution agreements, standardizes and integrates data, and makes rare disease data more accessible.

For questions or additional information about participating in RDCA-DAP, please email rdcadap@c-path.org.

If you’re a patient or a patient organization looking to start a registry, visit https://rarediseases.org/rdca-dap/.

FDA Acknowledgement
FAIR Data Collections

We've highlighted a couple of our collections below, to view more you can browse all collections here with an active platform user account.

Data Collection A

Ataxias

This is a collection of Ataxia datasets currently available, including the largest Friedreich’s Ataxia aggregate and multiple Spinocerebellar ataxia types.

Data Collection B

Muscular disease

This is a collection of Muscular disease datasets currently available.

Data Collection C

Neurodevelopment disease

This is a collection of Neurodevelopment disease datasets currently available.

View all FAIR Data Collections

Quick Start

New users: please sign up here before visiting Workspaces and FAIR. Platform access will be reviewed within 3-5 business days. More info here.

Use these Quick Start steps to explore the platform and available data. If you are interested in using data for research or study, please request a research workspace at the link below before submitting a Data Access Request in FAIR. A member of the C-Path team will address workspace requests within 3-5 business days.

1. Discover Data
  1. Go to FAIR Data ServicesFAIR logoData Services
  2. Review the existing datasets: Click 'Datasets' and select ‘All datasets’ in the dropdown.Datasets
  3. Click on an individual dataset to review its metadata.
2. Request Access
  1. When you have found a dataset of interest: Request a Research Workspace
  2. Once approved, you will be invited to join a workspace.
  3. To request data access, go to FAIR and click the Request Icon  in the menubar.
  4. Fill out the form and submit – once approved, you can transfer the dataset to your workspace.
3. Conduct Research
  1. Go to WorkspacesWorkspaces logoWorkspaces
  2. From the Workspaces home page, you can view your workspaces and invitations.
  3. Accept any pending invites and click to enter your workspace.

Contribute Data

Contributing to RDCA-DAP
Interested in contributing research, study, or clinical trials data to RDCA-DAP?

  1. Share your interest in contributing data to C-Path and provide supporting information.Connect
  2. C-Path will review and reach out with more information within 10 business days.

If you are a patient or patient advocate seeking a place for single patient data, or help on establishing data collection efforts, please reach out to NORD (US based) or EURORDIS (EU based) to identify available databases.

Additional Resources

Using RDCA-DAP

The Data and Analytics Platform includes FAIR Data Services and Workspaces.

  • FAIR includes integrated rare disease data from clinical trials, observational studies, real-world data, patient registries and other sources.
  • In Workspaces, advanced analytics capabilities allow efficient and effective interrogation of data to generate insights, analyses and solutions.

When using the RDCA-DAP, please refer to the current list of supported browsers.

Workspaces Fundamentals
FAIR Standard
Using FAIR Data Services
Workspaces Advanced
Workspaces Advanced Learning

Useful Information

Data Security

Data can be highly sensitive: it can be at risk for re-identification even when explicit personally identifiable information is removed. Find out more about the measures that we use to keep data safe.

Find out more

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