Critical Path Institute

Critical Path Institute (C-Path) is a nonprofit organization dedicated to enhancing and streamlining the process of drug development. Established in 2005 under the U.S. Food and Drug Administration’s (FDA) Critical Path Initiative program, C-Path operates as an independent, public-private partnership with the FDA. The organization has developed several quantitative clinical trial enrichment tools for diseases such as Alzheimer’s, Parkinson’s, and Type 1 Diabetes. C-Path’s Duchenne Regulatory Science Consortium (D-RSC) has created a clinical trial simulation tool to optimize clinical trial design for Duchenne Muscular Dystrophy (DMD). Additionally, the Rare Disease Clinical Outcome Assessment (COA) Consortium provides information on published COAs that can support efficacy endpoints in treatment trials for rare diseases. The Translational Therapeutics Accelerator (TRxA) focuses on advancing emerging safety biomarkers and early phase discovery research to propel drug development forward. C-Path also maintains the Integrated Parkinson’s Database, a comprehensive collection of observational and clinical studies related to Parkinson’s disease, and the Multiple Sclerosis Outcome Assessments Consortium (MSOAC) Placebo Database, which includes 2465 individual patient records from nine clinical trials. The Polycystic Kidney Disease Outcomes Consortium database consists of de-identified data from three longitudinal observational patient registries. The Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP) promotes the sharing of existing patient-level data and encourages standardization in new data collection. C-Path’s TB-Platform for the Aggregation of Preclinical Experiments Data (TB-APEX) and TB-Platform for Aggregation of Clinical TB Studies (TB-PACTS) are designed to accelerate tuberculosis research. The Critical Path for Alzheimer’s Disease Database contains demographic information, APOE4 genotype, concomitant medications, and cognitive scales. The Duchenne Regulatory Science Consortium Database includes data from DMD clinical trials, natural history studies, and clinical data collections. C-Path’s neuroscience program brings together regulators, pharmaceutical and biotech companies, academics, and patient advocates to advance drug development for Parkinson’s, Alzheimer’s, and rare neurodegenerative diseases. The pediatrics program focuses on advancing drug development for children with Type 1 Diabetes, Alpha-1 Antitrypsin Deficiency, Lysosomal Diseases, and neonatal complications. The rare and orphan disease program supports drug development for diseases such as Duchenne muscular dystrophy, Huntington’s disease, polycystic kidney disease, and rare neurodegenerative diseases. The Clinical Outcome Assessment Program provides scientific leadership and best practice recommendations based on clinically meaningful outcomes data. The Predictive Safety Testing Consortium (PSTC) achieved a significant milestone with the FDA’s support for four new pancreatic injury biomarkers. Lastly, the Critical Path for Rare Neurodegenerative Diseases (CP-RND) aims to enhance medical advances for rare neurodegenerative diseases, including ALS.