activities · 13
what they do
advocacy · 1
- Hosts scientific conferences to advance rare disease researchOrganized the first conference on Rare and Orphan Diseases in September 2023 to promote research collaboration and data harmonization across the rare disease community.
research · 8
- Developing Clinical Outcome AssessmentsDevelops clinical outcome assessments that incorporate the patient’s voice to evaluate treatment efficacy for conditions such as asthma, depression, irritable bowel syndrome, myelofibrosis, and non-small cell lung cancer.
- Developing Disease Progression Models and Clinical Trial Simulation Tools Development of Standardized Research Tools and Data PlatformsDevelops disease progression models and clinical trial simulation tools for various conditions, including Progressive Supranuclear Palsy, Friedreich’s Ataxia, Alzheimer’s disease, Duchenne Muscular Dystrophy, and Parkinson’s Disease, often receiving regulatory endorsement.
- Developing and Endorsing Biomarkers Development of Standardized Research Tools and Data PlatformsDevelops and secures regulatory endorsement for viable biomarkers for various diseases, including Alzheimer’s, Parkinson’s, type 1 diabetes, kidney disease, and tuberculosis, and for detecting drug-induced pancreatic injury.
- Develops and maintains integrated data platforms for rare and orphan diseases Development of Standardized Research Tools and Data PlatformsOperates the RDCA-DAP® platform and other disease-specific databases to standardize, integrate, and enable analytics on rare disease data, supporting biomarker development, trial design, and regulatory decision-making. Hosts over 100 datasets across rare diseases and maintains similar platforms for tuberculosis, Alzheimer’s, Parkinson’s, Duchenne Muscular Dystrophy, and polycystic kidney disease.
- Develops regulatory-endorsed drug development tools and biomarkers Development of Standardized Research Tools and Data PlatformsCreates and validates biomarkers, clinical trial simulation tools, and clinical outcome assessments that have received formal regulatory support from the FDA and EMA. The organization has achieved more global regulatory endorsements for such tools than any other since 2008.
- Establishing and Maintaining Integrated Data Platforms and Databases Development of Standardized Research Tools and Data PlatformsEstablishes and maintains integrated data and analytics platforms and databases for various diseases, including rare diseases (RDCA-DAP), Alzheimer’s, Duchenne Muscular Dystrophy, Parkinson’s, Multiple Sclerosis, Polycystic Kidney Disease, and Tuberculosis (TB-APEX, TB-PACTS, PAN-TB), standardizing and integrating patient records and study data for public access or qualified researchers.
- Manages collaborative research programs to accelerate drug development Accelerating Medical Product DevelopmentConvenes stakeholders from academia, industry, regulatory agencies, and patient advocacy groups to manage large-scale research initiatives that accelerate medical product development and regulatory decision-making.
- Managing Collaborative Research Programs Accelerating Medical Product DevelopmentManages large-scale collaborative research programs involving stakeholders from academia, industry, patient advocacy groups, and regulatory agencies to accelerate drug development, particularly for rare and orphan diseases.
capacity building · 4
- Advancing Research for Specific DiseasesEstablishes initiatives, such as the Critical Path for Alpha-1 Antitrypsin Deficiency, and hosts conferences to advance research collaboration and data harmonization for specific diseases, including rare and orphan diseases.
- Facilitating Medical Product Development and Regulatory Decision-Making Accelerating Medical Product DevelopmentConvenes global stakeholders to develop tools and processes that accelerate medical product development and regulatory decision-making, and supports communication and training for efficient use of real-world data in Europe.
- Leading Data Sharing and Infrastructure Development Projects Development of Standardized Research Tools and Data PlatformsLeads work packages in projects like Unite4TB and ERA4TB to establish processes and infrastructure for secure sharing of standardized data and images in tuberculosis research.
- Leads capacity-building initiatives for data sharing and real-world evidence Accelerating Medical Product DevelopmentSupports infrastructure development, secure data sharing, and training programs in Europe and global tuberculosis consortia to advance the use of real-world data in regulatory science and drug development.
strategies · 11
how they think
Theories of action extracted from this org's own source material. Click any to see the full field of orgs running the same approach.
- Biomarker Qualification Framework Development biomarker_qualification_framework_developmentThe organization develops evidentiary frameworks and best practices to standardize the validation of biomarker assays, which is crucial for their effective use in drug development.
- Core Competency-Driven Tool Development core-competency-driven_tool_developmentThe organization leverages its core competencies in areas such as data management, biomarkers, modeling, regulatory science, and clinical outcome assessments to develop targeted tools that address specific challenges in drug development.
- Core-competency-driven development of regulatory tools and frameworks core-competency-driven tool developmentLeverages expertise in data management, biomarkers, modeling, clinical outcome assessments, and regulatory science to create targeted tools, frameworks, and best practices that address specific gaps in drug development.
- Data Standardization and Integration for Accelerated Drug Development data_standardization_and_sharingThe organization creates shared data repositories, standardizes endpoints, and curates clinical and natural history data into formats like CDISC. This standardization and integration of patient-level data enable more efficient and predictive drug development pathways, facilitate cross-study analysis, and support the modeling of disease progression, biomarkers, and innovative clinical trial designs, particularly for rare diseases.
- Data standardization and integration to enhance drug development efficiency data standardization and sharingStandardizes clinical research data—particularly in rare diseases—using frameworks like CDISC SDTM and curates shared repositories to enable cross-study analysis, modeling of disease progression, biomarker identification, and optimized clinical trial design.
- Multi-stakeholder and Pre-competitive Collaboration multi-stakeholder_collaborationThe organization acts as a neutral third party to convene diverse stakeholders, including regulators, industry, academia, and patient groups, in pre-competitive collaborations. This approach facilitates the sharing of scientific data, identifies drug development barriers, and co-develops tools and frameworks to improve clinical trial design and regulatory decision-making, ultimately accelerating drug development for diseases with unmet medical needs.
- Multi-stakeholder collaboration to accelerate drug development multi-stakeholder collaborationConvenes diverse stakeholders—including regulators, industry, academia, patient advocates, and scientists—in neutral, pre-competitive collaborations to identify barriers, co-develop tools, and accelerate drug development for diseases with unmet medical needs, particularly rare and neurodegenerative conditions.
- Parallel Pathway Drug Development parallel_pathway_drug_developmentThe organization employs a parallel pathway approach to simultaneously evaluate multiple drug candidates, which helps to reduce the overall development time for new treatment regimens.
- Patient-Centered Drug Development patient_centered_developmentThe organization integrates patient and caregiver insights and advocate perspectives into regulatory science and drug development processes. This ensures that medical product development is more efficient, relevant, and focused on the needs of patients.
- Patient-centered regulatory science and development patient-centered regulatory scienceIntegrates patient and caregiver perspectives into regulatory science, biomarker qualification, and drug development processes to ensure therapies are relevant, efficient, and aligned with patient needs.
- Pre-competitive collaboration for safe data sharing and consensus building pre-competitive collaborationActs as an independent, neutral third party to enable pre-competitive collaboration, allowing industry and other stakeholders to safely share scientific data, align on best practices, and build consensus through iterative feedback, without compromising competitive interests.
named programs · 11
what they call their work
CURE Drug Repurposing Collaboratory (CDRC)
Accelerates identification of new uses for existing drugs to treat infectious diseases, including COVID-19, with FDA support for EHR data extraction
Critical Path for Parkinson’s (CPP)
Develops tools and biomarkers to optimize clinical trial design in Parkinson’s disease, with EMA support for imaging biomarkers and simulation platforms
Duchenne Regulatory Science Consortium (D-RSC)
Advances clinical trial design and biomarker qualification for Duchenne Muscular Dystrophy, including a model-based clinical trial simulation platform endorsed by EMA
Friedreich’s Ataxia Integrated Clinical Database (FA-ICD)
Develops models of disease progression in Friedreich’s Ataxia to optimize treatment evaluation.
International Neonatal Consortium (INC)
Improves clinical trial design and data collection for neonatal populations, supported by data transfers from hospitals in the UK and Japan
Predictive Safety Testing Consortium (PSTC)
Develops and qualifies safety biomarkers for use in drug development, with regulatory support from FDA and EMA
Quantitative Medicine Program
Develops modeling and simulation tools for neuroscience diseases under FDA contract
Rare Disease Cures Accelerator - Data and Analytics Platform (RDCA-DAP)
Provides infrastructure for data sharing and analytics to accelerate rare disease drug development
Transplant Therapeutics Consortium (TTC)
Focuses on improving clinical trials for transplant therapies, including qualification of the iBox Scoring System as a secondary efficacy endpoint by EMA
Type 1 Diabetes Consortium (T1DC)
Qualifies biomarkers for type 1 diabetes prevention trials, including pancreatic islet autoantibodies as enrichment biomarkers with EMA qualification
eCOA Consortium
Develops and qualifies electronic clinical outcome assessments (eCOAs) for use in regulatory submissions, formerly known as ePRO Consortium
relationships · 36
who they work with
- Bill & Melinda Gates Foundation Funder — Provides grant funding to support C-Path’s initiatives.
- British Medical Research Council Partner — Collaborative partner in the TB-PACTS data platform initiative.
- Case Western University Partner — Collaborative partner in the TB-PACTS data platform initiative.
- Duke-Margolis Center for Health Policy Partner — Convened a 2017 public workshop to gather input on C-Path’s biomarker assay validation framework
- European Joint Programme on Rare Diseases Partner — Engaged in collaboration to advance rare disease research and drug development.
- European Medicines Agency Government — Endorsed C-Path's computerized clinical trial design tool for Alzheimer’s disease.
- European Medicines Agency Government — Regulatory agency that has issued letters of support and qualification opinions for C-Path consortium platforms and biomarkers.
- European Medicines Agency Partner — Collaborates on regulatory science initiatives and has issued letters of support and qualification opinions for C-Path-developed tools.
- European Medicines Agency (EMA) Government — Partners with C-Path on regulatory science projects to support drug development in Europe.
- FDA Government — Collaborates with FDA on regulatory science, clinical policy issues, and drug development tools.
- Food and Drug Administration Government — Endorsed C-Path's computerized clinical trial design tool for Alzheimer’s disease.
- Food and Drug Administration Government — FDA-sponsored initiative supporting the RDCA-DAP platform to accelerate rare disease therapy development.
- Food and Drug Administration Government — Primary government affiliate and funder; established a Memorandum of Understanding with C-Path in 2005.
- Frederick Gardner Cottrell Foundation Funder — Provided grant funding to establish the C-Path Translational Therapeutics Accelerator (TRxA).
- Friedreich’s Ataxia Research Alliance Partner — Collaborative partner in the FA-ICD initiative to accelerate Friedreich’s ataxia research and drug development.
- Horizon Europe Government — Funds C-Path’s participation in multiple projects including ERDERA and More-EUROPA.
- Innovative Health Initiative Partner — Partner in the Unite4TB project funded under the Innovative Medicines Initiative 2 Joint Undertaking.
- Janet Woodcock, MD Government — Senior Advisor to the Board
- Michael J. Fox Foundation Funder — Provides grant funding to support C-Path’s initiatives.
- NIH Government — Collaborates with NIH on advancing research and data harmonization for rare neurodegenerative diseases.
- National Institutes of Health (NIH) Government — Funds and collaborates with C-Path on research initiatives to advance regulatory science and drug development.
- National Neonatal Research Database Partner — Provided anonymized electronic patient record data from the UK to support research through the International Neonatal Consortium.
- National Organization for Rare Diseases Partner — Co-founding partner of RDCA-DAP, collaborating on data governance and rare disease research initiatives.
- Neonatal Research Network of Japan Partner — Provided anonymized electronic patient record data from three hospitals to support research through the International Neonatal Consortium.
- Peter B. Corr, MD, PhD Partner — Senior Advisor to the Board
- Polycystic Kidney Disease Foundation Funder — Provides grant funding to support C-Path’s initiatives.
- ShaAvhrée Buckman-Garner, MD, PhD Partner — Advisor to the Board
- Special Progamme for Research and Training in Tropical Diseases (TDR) Partner — Collaborative partner in the TB-PACTS data platform initiative.
- St. George’s University of London Partner — Collaborative partner in the TB-PACTS data platform initiative.
- TB Alliance Partner — Collaborative partner in the TB-PACTS data platform initiative.
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