who runs it
organizations running this strategy · 1
what it looks like in practice
activities of orgs running this strategy
A sample of programmatic activities from the orgs above. These are what the strategy looks like on the ground.
- Advancing Research for Specific Diseases CRITICAL PATH INSTITUTEcapacity buildingEstablishes 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.
- Developing Clinical Outcome Assessments CRITICAL PATH INSTITUTEresearchDevelops 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 CRITICAL PATH INSTITUTEresearchDevelops 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 CRITICAL PATH INSTITUTEresearchDevelops 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 CRITICAL PATH INSTITUTEresearchOperates 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 CRITICAL PATH INSTITUTEresearchCreates 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 CRITICAL PATH INSTITUTEresearchEstablishes 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.
- Facilitating Medical Product Development and Regulatory Decision-Making CRITICAL PATH INSTITUTEcapacity buildingConvenes 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.
- Hosts scientific conferences to advance rare disease research CRITICAL PATH INSTITUTEadvocacyOrganized the first conference on Rare and Orphan Diseases in September 2023 to promote research collaboration and data harmonization across the rare disease community.
- Leading Data Sharing and Infrastructure Development Projects CRITICAL PATH INSTITUTEcapacity buildingLeads 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 CRITICAL PATH INSTITUTEcapacity buildingSupports 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.
- Manages collaborative research programs to accelerate drug development CRITICAL PATH INSTITUTEresearchConvenes 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 CRITICAL PATH INSTITUTEresearchManages 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.