The FDA has qualified the DIBSS-C, a patient-reported outcome measure, to assess symptom-based efficacy endpoints in IBS-C clinical trials. This qualification reflects FDA's commitment to patient-focused drug development and is a major milestone for the IBS Working Group.
The Critical Path to Therapeutics for the Ataxias (CPTA) Consortium aims to optimize clinical trials for inherited ataxias through a public-private partnership. CPTA will create a neutral space for stakeholders to share expertise and data, leveraging this input to guide the development of actionable solutions.
The System of Hospitals for Innovation in Pediatrics - Medical Devices (SHIP-MD) Virtual Workshop will focus on accelerating medical device development for children. The workshop aims to enhance safe and effective pediatric care options by addressing challenges such as clinical trials, funding, and regulatory approval.
The FDA has awarded a grant to Critical Path Institute (C-Path) to develop real-world evidence for treatments in neonates using real-world data. This project will create a platform to standardize and integrate data from NICUs worldwide, enabling the creation of actionable reference ranges and natural history models.
The Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP) has achieved significant milestones in its first year, thanks to data sharing and collaboration among stakeholders. The platform serves as a centralized infrastructure to support data analysis and aid in the understanding of rare diseases.
The FDA has issued a positive response to C-Path's biomarker panel for detecting acute drug-induced skeletal muscle injury in phase 1 clinical trials. The panel, developed by C-Path's Predictive Safety Testing Consortium and Duchenne Regulatory Science Consortium, uses four molecular biomarkers to aid in the detection of this condition.
The Predictive Safety Testing Consortium and Duchenne Regulatory Science Consortium have developed a glutamate dehydrogenase (GLDH) biomarker to detect drug-induced liver injury in patients with inherited muscle disorders. The FDA has agreed to accept the Qualification Plan for GLDH as an important and accurate measurement of DILI.
The CURE Drug Repurposing Collaboratory aims to accelerate the identification of potentially effective drugs for patients with unmet treatment options. The initiative uses data collected through the FDA-NCATS CURE ID platform to identify existing drugs that demonstrate possible treatment approaches.
The Critical Path Institute (C-Path) has received a letter of support from the European Medicines Agency (EMA) to facilitate the development and validation of pancreatic islet autoantibodies as enrichment biomarkers for type 1 diabetes clinical trials. This collaboration aims to accelerate drug development in T1D by identifying patient...
Critical Path Institute (C-Path) has been awarded a U.S. Food and Drug Administration (FDA) contract to develop novel clinical outcome assessments for pediatric asthma. The project aims to create patient-focused drug development tools for children with asthma.
The Critical Path Institute (C-Path) has established a Rare Disease Clinical Outcome Assessment Consortium with the FDA's support. The consortium aims to accelerate the development of new medical products for rare diseases by creating and curating a resource of information on publicly available clinical outcome assessments.
Lundbeck contributes clinical trial data from over 2,500 patients to CPAD consortium's integrated database. C-Path and Lundbeck aim to expedite the development of new therapies for Alzheimer's disease through public-private partnerships.
The Critical Path Institute (C-Path) has been awarded a grant to conduct stakeholder engagement on the FDA's proposed framework to enhance the pediatric medical device ecosystem. The framework aims to facilitate design, development, and commercialization of medical devices for children.
A global Foundational Standard describes how to represent data for natural history and efficacy studies in animals submitted under the FDA's Animal Rule. The standard aims to streamline data reporting and accelerate therapies for humans.
The Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP) aims to accelerate the development of treatments for rare diseases by providing data and analytics. The platform, launched by C-Path and NORD, will facilitate collaboration among key stakeholders, including patients, industry, and regulatory agencies.
Dr. Martha A. Brumfield, a renowned leader in pharmaceutical development and regulatory science, has been awarded the Jon W. McGarity Arizona Bioscience Leader of the Year honor. Her contributions to accelerating medical product development and building collaborative solutions have significantly benefited patients and their families.
The Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP) aims to reduce barriers for rare disease treatment development. The platform will provide a centralized infrastructure to support accelerated therapy development, leveraging integrated data from various sources and advanced analytics expertise.
A global Therapeutic Area Standard has been released to represent data in research studies pertaining to CDAD, driven by the need for a major medical and infection control problem. The standard aims to accelerate clinical research and improve public health outcomes.
The new standard will facilitate collaboration and cross-study analyses to ensure the highest return on research investments. By structuring data in a common format, researchers can focus on discoveries that will have invaluable impact on clinical research and global public health.
A collaborative project between C-Path and Japan's PMDA aims to generate critical data for safety decisions in early-stage clinical trials. The study will compare biomarkers in healthy Japanese volunteers with similar Western subjects, informing regulatory submissions.
The Doris Duke Charitable Foundation has awarded a grant to the Critical Path Institute (C-Path) to accelerate the development of therapies for sickle cell disease. C-Path will plan a consortium of stakeholders to identify and work collaboratively on jointly beneficial drug development tools and novel methodologies.
The Critical Path Institute has received a $10 million grant from the Leona M. and Harry B. Helmsley Charitable Trust to identify unmet needs for biomarker development in Crohn's disease. The pre-consortium will focus on laying the groundwork for future regulatory endorsement and accelerate the development of new treatments.
The Critical Path Institute and Translational Genomics Research Institute will sequence at least 12,000 tuberculosis bacteria isolates from around the world to better understand drug resistance. The partnership aims to develop personalized medicine options for patients with drug-resistant TB.
The Critical Path Institute has launched a consortium to qualify islet autoimmunity antibodies as prognostic biomarkers for Type 1 Diabetes (T1D) research. The goal is to identify individuals at risk of disease progression and develop interventions to prevent or delay the onset of symptomatic T1D.
The US FDA has awarded Critical Path Institute three grants to develop data standards for HIV treatment, Clostridium difficile-associated diarrhea, and improved animal efficacy studies. C-Path will collaborate with CDISC to create therapeutic area data standards to accelerate clinical research and medical product development.
The Critical Path Institute's CPTR initiative and the WHO Global TB Programme partnered to develop quantitative analyses of TB-PACTS database data. The collaboration, called TB-ReFLECT, will extract key lessons and package them as tools for future trial design.
The Innovative Medicines Initiative (IMI) SAFE-T and C-Path PSTC have obtained regulatory support from the US FDA and EMA for four new liver safety biomarkers. These biomarkers, including cytokeratin 18, HMGB1, osteopontin, and MCSFR1, show promise in predicting drug-induced liver injury and may improve early prediction of liver toxici...