Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP)

Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP) is sponsored by National Organization for Rare Disorders (NORD). An integrated database and analytics hub designed to build tools accelerating drug development across rare diseases.

Rare Disease Cures Accelerator-Data and Analytics Platform Rare Disease Cures Accelerator-Data and Analytics Platform RDCA-DAP is an FDA-sponsored initiative that provides a centralized and standardized infrastructure to support and accelerate rare disease characterization with the goal of accelerating therapy development. You can access the platform here: https://portal. rdca.

c-path. org/ Over 350 million people in the world have been diagnosed with a rare disease. In the United States, a rare disease is defined as affecting fewer than 200,000 people, and there are over 10,000 classified rare diseases.

Nonetheless, 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.

Critical Path Institute’s Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP®) is an FDA-funded initiative that provides a centralized and standardized infrastructure to support and accelerate rare disease characterization, with the goal of accelerating the development of treatments and cures for rare diseases.

RDCA-DAP creates the collaborative, non-competitive space to share existing patient-level data and encourages the standardization of new data collection.

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.

RDCA-DAP is positioned to generate solutions to drug development bottlenecks through program led solutions and facilitation of C-Path Rare and Orphan diseases consortia activities. Access the platform page here: https://portal. rdca.

c-path. org/ The RDCA-DAP platform continues to expand since going live September 2021, and now contains data for 42+ different rare diseases, including Polycystic Kidney Disease, Duchenne Muscular Dystrophy, Mitochondrial Diseases, Neurodevelopmental Disorders and Rare Epilepsies and Rare Neurodegenerative disorders including Friedreich ataxia. More data will be added and made accessible as outreach efforts continue.

Since the platform’s launch, we’ve seen engagement from 325 approved platform requests and 35 approved workspaces for external users and research. For any additional 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/ . Data Use, Task Forces, and Additional Resources Data contribution, access, and use on the RDCA-DAP platform are governed by processes and agreements that protect patient privacy, ensure data security, and respect contributors’ conditions.

The Data Governance framework explains how users request datasets, how access decisions are made, and what users must complete before access. Approved users must sign a Data Use Agreement outlining terms, limitations, and obligations. Data contributors remain owners of the data they share with the platform.

RDCA-DAP encourages data sharing after review by its five-member Data Use Committee, which includes representatives from Critical Path Institute, National Organization for Rare Disorders, and other rare disease stakeholders. The committee evaluates requests based on scientific merit, educational purpose, or public interest and approves or rejects them by majority vote.

Data Contribution Agreement, GDPR SCCs According to the General Data Protection Regulation (GDPR), contractual clauses ensuring appropriate data protection safeguards can be used as a grounds for data transfers from the EU to third countries. This includes model contract clauses – so-called standard contractual clauses (SCCs) – that have been “pre-approved” by the European Commission.

Alexandre Bétourné, PhD, PharmD, Data Use Committee Edward Neilan, MD, PhD, Data Use Committee Steve Roberds, PhD, Data Use Committee James R. Rusche, PhD, Data Use Committee Theresa V. Strong, PhD, Data Use Committee Friedreich’s Ataxia Integrated Clinical Database (FA-ICD) Friedreich’s ataxia (FA) is a debilitating, life-shortening, degenerative neuromuscular disorder.

It is the most common form of hereditary ataxia, affecting approximately 1 in every 50,000 people in the United States and Europe (FA is primarily found in white, Hispanic, and Southeast Asian populations; incidence is very rare in other racial groups). FA is an autosomal recessive, single gene disorder, caused by mutations in the FXN gene.

Loss of balance and coordination is the most common presenting symptom typically beginning between the ages of 5 and 15 years with progression of symptoms leading to loss of ambulation and independence of all activities of daily living. Adult or late onset FA is less common, affecting More information can be found at https://curefa.

org Launched in February 2018, the Friedreich’s Ataxia Integrated Clinical Database (FA-ICD) is designed to catalyze and accelerate Friedreich’s ataxia (FA) research and drug development by curating and standardizing FA clinical trial and natural history data into CDISC format and making this data publicly available to qualified researchers.

These researchers can access and analyze data in aggregate, or filter and view individual de-identified patient-level data from four clinical trials and a large FA natural history study. Additional data may be available in the future.

This initiative represents a collaborative partnership between the Friedreich’s Ataxia Research Alliance (FARA) and the Rare Diseases Cures Accelerator Data and Analytic platform (RDCA-DAP) of the Critical Path Institute (C-Path), with a goal of expanding the FA-ICD platform by engaging with other data contributors to secure additional datasets.

FA-ICD Access and Data Contribution FA-ICD catalogs completed FA clinical trials and natural history data and makes it available to qualified researchers. Access to the patient level data is by request only and subject to review and approval by the FA-ICD Steering Committee.

To request access, you must agree to the Terms and Conditions for Use and submit a Request for Access application detailing how the data will be used, who will access the data and any plans for publishing work informed by the data. FA-ICD encourages data contributions from interventional and non-interventional studies and is always willing to discuss how companies or other researchers can engage with the initiative.

The FA-ICD is currently being utilized to develop nonlinear mixed effects-based models of disease progression in FA to investigate and compare available outcome measures collected in interventional and non-interventional studies. Additionally, a placebo effect model may be incorporated to quantify the magnitude, onset, and offset of the placebo response for control arm subjects included in the FA-ICD.

These models are intended to provide the foundation for a downstream clinical trial simulation tool. Limb Girdle Muscular Dystrophies Mitochondrial and Inherited Metabolic Diseases Task Force Read about this recently-developed task force here .

Progressive Supranuclear Palsy Rare Mitochondrial and Inherited Metabolic Diseases Launched January 2024, the Rare Mitochondrial and Inherited Metabolic Diseases task force includes a registry database and digital app PRO The task force has launched a data aggregation to inform data standardization and collection across diseases.

Spastic Paraplegia and Primary Lateral Sclerosis The Spastic Paraplegia and Primary Lateral Sclerosis task force was launched February of 2025. A database is currently under production, to be launched soon. The task force delivered an integrated clinical database and advancing tools and solutions to facilitate drug development.

For additional resources, such as a list of diseases’ data areas housed in the platform, past webinars, workshops, videos, FAQs and the FA-ICD Database, click here . To view RDCA-DAP’s Data Contributors, Founding Members, and additional Collaborators, visit the programs’ one sheeter, here .

Klaus Romero, MD, MS, FCP Chief Executive Officer, Chief Science Officer, Principal Investigator, RDCA-DAP Collin Hovinga, PharmD, MS FCCP, Senior Vice President, Rare and Orphan Disease Programs Heidi Grabenstatter, PhD, MS Scientific Director, RDCA-DAP Director, Rare and Orphan Disease Programs Senior Project Manager, RDCA-DAP Project Coordinator, RDCA-DAP Chief Medical and Scientific Officer Vice President, Information Technology Director of Education Programs For questions or additional information about participating in RDCA-DAP, please email rdcadap@c-path.

org . How RDCA-DAP Delivers Impact and Support for Rare Disease Treatment Innovation Across Healthcare Matrix Partners with C-Path to Expand Rare Disease Data Platform Through Direct Integration TUCSON, Ariz. , July 21, 2026 — Critical Path Institute (C-Path), a nonprofit that builds data and analytics tools to speed drug.

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