Programs

C-Path’s Predictive Safety Testing Consortium (PSTC) has launched the Biomarker Data Repository (BmDR): a repository for data on novel translational safety biomarkers from drug development programs. Masked, de-identified data from multiple sponsors will be collected and stored in a secured repository. The data will then be available to C-Path and FDA staff to support research that leads to the submission of documents to worldwide regulatory agencies to qualify novel safety biomarkers for new Contexts of Use (CoUs), to modify and expand existing CoUs, and to identify appropriate exploratory biomarkers to advance drug development in the future. The initial pilot focuses on kidney safety biomarkers.

The main goal of the BmDR is to provide industry with new drug development tools (kidney safety biomarkers). Existing biomarker data could be used to significantly advance and accelerate understanding of the utility of novel biomarkers as drug development tools.

As a sponsor, you can join with other innovative companies simply by sharing non-proprietary data. Since only summary, non-proprietary data will be available, each participating organization’s intellectual property is protected. C-Path has set up a straightforward governance process that protects all submitted data through sponsor-directed data use agreements. Contributors to BMDR will have access to aggregated biomarker data and summary analyses—enabling collaboration and support of their drug development efforts, reference ranges, medically relevant thresholds, and other outputs from accumulated data on both conventional and novel biomarkers.

Mission

CPAD has a mission to develop new technologies and methods to accelerate the development and review of medical products for neurodegenerative diseases.

CPAD is focused on accelerating therapeutic treatment development for patients with chronic neurodegenerative disease, namely, Alzheimer’s disease (AD), the most prevalent and devastating dementia, by advancing drug development tools (DDTs) and when appropriate, medical device development tools (MDDTs) for evaluating drug efficacy, conducting clinical trials, and streamlining the process of regulatory review. The consortium focuses on sharing precompetitive patient-level data from the control arms of legacy clinical trials, developing new tools to be submitted to the regulatory agencies, and developing consensus data standards.

CPAD has the following areas of focus: (1) qualification of objective biomarkers, including both biochemical and observational digital biosensor measures of health, (2) development of common data standards, (3) creation of integrated databases for clinical trials data, and (4) development of quantitative model-based tools for therapeutics development.

Regulatory milestones for CPAD include a qualification opinion with EMA for the use of low baseline hippocampal volume for patient enrichment in pre-dementia trials, and, more recently, positive regulatory decisions from the FDA and EMA for the use of a clinical trial simulation tool to aid in trials for mild to moderate stages of AD.

Until December 2015, CPAD focused on projects that spanned AD and Parkinson’s disease. Starting in January 2016, a newly formed consortium, Critical Path for Parkinson’s (CPP) consortium, assumed responsibilities for the projects initiated by CPAD. CPP was enabled by a generous donation by the Parkinson’s UK Foundation and is led by Dr. Diane Stephenson as Executive Director.

Traditionally, Parkinson’s has been viewed as a disorder in which individuals don’t have enough of a chemical called dopamine because specific nerve cells inside their brain have died. Current research indicates that the processes that lead to dopamine deficit start much earlier (decades), making it a pressing need to increase the understanding of Parkinson’s progression. CPP brings together pharmaceutical companies and academic partners working toward a common goal of establishing best practices and more efficient protocols for planning and designing clinical trials in early Parkinson’s.

“It’s our goal that the partnership with the Critical Path Institute will improve the clinical trial process and deliver new and better treatments, faster, to the people who urgently need them,” says Parkinson’s UK Chief Executive Steve Ford.

The Critical Path to TB Drug Regimens (CPTR Initiative) is a public‐private partnership initiated in March 2010 by Critical Path Institute (C-Path), the Bill & Melinda Gates Foundation (BMGF) and the Global Alliance for TB Drug Development (TB Alliance). C-Path leads the CPTR Regulatory Science Consortium and the CPTR Rapid Drug Susceptibility Testing (RDST) Consortium, with participation from the pharmaceutical industry, academia, as well as, national and global government agencies, in order to: develop and integrate data standards, qualify biomarkers through the FDA/EMA, develop quantitative disease progression (natural history) models, create disease response metrics, develop target product profiles and supporting assays, and develop new pharmacokinetic/dynamic measures of drug interactions.

The Data Collaboration Center (DCC) of the Critical Path Institute (C-Path) was founded to provide large-scale data solutions for scientific research. The DCC’s work takes place in a neutral, noncompetitive environment, utilizing appropriate data standards (such as those of the Clinical Data Interchange Standards Consortium [CDISC]). The DCC possesses the top-tier technical, scientific, and project management expertise necessary to support advanced research efforts. DCC solutions include:

• Development of customized data-sharing platforms
• Planning and execution of multisource data aggregation and standardization
• Sustainable curation and administration of data and its storage
• Ability for teams to work together to analyze and interpret data
• Robust security policies and framework
• Application of current regulations to ensure compliance

The mission of the eCOA Consortium is to advance the science of clinical trial endpoint assessment by collaboratively supporting and conducting research, designing and delivering educational opportunities, and developing and disseminating best practice recommendations for electronic collection of clinical outcome data. The eCOA Consortium provides a pre-competitive environment in which a critical mass of experts can collaborate to generate measurement equivalence data, develop specification documents and data standards, and provide guidance on methodological considerations related to eCOA applications. All of these activities are aimed at enhancing the quality, practicality, and acceptability of electronic capture of clinical trial endpoint data. The eCOA Consortium works closely with C-Path’s PRO Consortium to make the PRO instruments emerging from its therapeutic area working groups available in multiple data collection formats. The overarching aim is to enhance public health by optimizing the value of PRO data in medical product evaluation and clinical decision making.

C-Path’s Role

C-Path’s role in the eCOA Consortium is to serve as a recognized and respected neutral third party that provides overall administrative support and oversight. C-Path provides a Director who is responsible for the overall management of the Consortium. The Director and her staff coordinate all projects and provide financial oversight, project management, and scientific oversight/consultation. Working with the eCOA Consortium participants, C-Path will facilitate the development and publication of scientific articles and supporting materials from the projects undertaken by the eCOA Consortium.

This global initiative aims to facilitate collaboration among the bio-pharmaceutical industry partners, technology industry partners, academic institutions, government agencies, and patient-advocacy associations. HD-RSC fosters consensus and data-driven research to increase efficiency, safety, and speed in developing new therapies.

Our Mission

Huntington’s Disease (HD) is a rare, inherited genetic disorder for which no cure currently exists. The degeneration of nerve cells of the brain in HD results in a loss of motor function and muscle control, a loss of cognitive ability (thinking), and mood changes including depression and irritability. Research indicates that changes in the brain occur up to 15 years prior to the onset of motor symptoms, highlighting the need for better disease progression understanding and early intervention.

The overall goal of the Huntington’s Disease Regulatory Science Consortium (HD-RSC) is to create a regulatory science strategy for HD, offering additional incentive and de-risking for HD therapeutic development by all stakeholders. HD-RSC will provide a forum and structure to bring together the necessary participants from the HD community for data contribution and tool development, leading to efficiencies in development of new therapies.

“By uniting stakeholders from research institutions, drug developers, regulatory agencies, patient advocacy and other organizations,” said Janet Woodcock, Director of the U.S. Food and Drug Administration’s Center for Drug Evaluation and Research (FDA/CDER), “INC can develop practical tools that can be incorporated into clinical trials for neonates, which will then lead to more successful, efficient trials and provide this population with better treatments.

” The scientific workshop that led to the formation of the consortium – “Roadmap for Applying Regulatory Science to Neonates” – was held at the FDA on October 28–29, 2014:  http://www.fda.gov/Drugs/NewsEvents/ucm410863.htm.

The National Multiple Sclerosis Society recognized the gap in the MS treatment pipeline and decided to employ a method that has steadily gained support within the research community — consortia science. MSOAC brings stakeholders from industry, academia, MS advocacy groups, and regulatory agencies together to spur development of drug development tools to assess the effectiveness of potential treatments for all forms of MS.

The PRO Consortium was formed in December of 2008 and formally launched in March of 2009. The PRO Consortium’s membership is comprised of pharmaceutical companies along with representatives from the FDA, EMA and NIH who provide advice to the Coordinating Committee.

Patient-Reported Outcome Instruments

PRO instruments involve measurement of one or more aspects of a patient’s health status based on information gathered directly from the patient, without interpretation by physicians or others. Patients provide information concerning the impact of an intervention or therapy from their perspective. PRO instruments offer a means for capturing how a patient feels or functions with respect to her/his health, condition, or disease.

There are many disease areas for which adequate PRO instruments are not available. PRO instrument development is expensive, resource-intensive, and time consuming due to the extensive research and testing process. Under the direction of Stephen Joel Coons, PhD, the PRO Consortium provides a critical mass of experts with different skill sets, experiences, and perspectives. The PRO Consortium consists of scientists from C-Path, pharmaceutical/biotechnology companies, FDA and National Institutes of Health (NIH).

C-Path’s Role

In the PRO Consortium, C-Path’s role is to serve as a recognized and respected neutral third party that provides overall administrative support and oversight. C-Path provides a Director who is responsible for the overall management of the Consortium and establishment of a process for identification, prioritization, and development of potential PRO instruments. The Director and his staff coordinate all projects and provide financial oversight and project implementation management. Working with other Consortium participants, C-Path will develop and publish scientific articles and support educational activities with data, expertise, and other outcomes from the projects supported under the Consortium, to benefit all stakeholders and the public health.

The Polycystic Kidney Disease (PKD) Outcomes Consortium is a successful collaboration between Critical Path Institute (C-Path), the PKD Foundation, Clinical Data Interchange Standards Consortium (CDISC), four leading academic medical centers (Tufts University, University of Colorado Denver, Emory University, and
Mayo Clinic), and three pharmaceutical companies. Its mission is to develop tools and promote research that will lead to the discovery of treatments for PKD and improve the lives of all it affects. The Consortium is led by C-Path and funded through a grant from the PKD Foundation and philanthropic donations. Additionally, a representative from the U.S. Food and Drug Administration (FDA) serves as an active advisor to the Consortium.

Autosomal Dominant PKD (ADPKD) is a debilitating genetic disease affecting more than 600,000 Americans and 12 million people worldwide and for which there is currently no known cure or effective treatment. Traditional endpoints of renal function only show changes very late in the course of the disease, making it difficult to assess the effectiveness of new medications. There is critical need for a biomarker that will assess disease progression at an earlier stage when patients may be more likely to respond to new therapies.

The primary goals of the PKD Consortium are to develop CDISC data standards for PKD and to use clinical data from ADPKD patients collected over many years in patient registries and observational studies to support the FDA and EMA qualification of an imaging biomarker, Total Kidney Volume (TKV), for use in drug development trials.

Scientists will use the data collected to develop a disease progression model that will evaluate the relationship between TKV and the known complications of ADPKD, including rate of loss of kidney function, hypertension, gross hematuria, kidney stones, urinary tract infections, development of end-stage renal disease, and mortality. These analyses will be used to support the regulatory qualification of TKV as an accepted measure for assessing the progression of ADPKD in clinical trials in which new therapies are tested.

PSTC was formed and officially announced on March 16, 2006 by Health and Human Services Secretary Michael Leavitt, FDA Commissioner Dr. Andrew von Eschenbach and FDA Deputy Commissioner Dr. Janet Woodcock, who identified the consortium as “unprecedented” and a “shining example” of the type of work the FDA would like to see conducted.

Who we are

PSTC brings together pharmaceutical companies to share and validate innovative safety testing methods under advisement of the FDA (U.S. Food and Drug Administration), its European counterpart, the EMA (European Medicines Agency), and PMDA (Japanese Pharmaceutical and Medical Devices Agency). PSTC was formed and officially announced on March 16, 2006 by Health and Human Services Secretary Michael Leavitt, FDA Commissioner Dr. Andrew von Eschenbach and FDA Deputy Commissioner Dr. Janet Woodcock, who identified the consortium as “unprecedented” and a “shining example” of the type of work the FDA would like to see conducted.

The FDA, EMA and PMDA participate as advisors, along with more than 250 participating scientists across industry and academia. The mission of PSTC is to identify new and improved safety testing methods and submit them for formal regulatory qualification by the FDA, EMA and PMDA.

Our mission

The tests used to determine drug safety have not changed in decades. Although companies have developed newer safety testing methods, these are not generally accepted by the FDA or EMA as proof of safety. This is due, in part, because the methods used for testing are often different from company to company. That discrepancy leaves regulatory scientists uncertain about which methods should be preferred. Another key factor is that the tests have not, in the past, been independently validated. PSTC now fills that important role and serves as a neutral third party to assess drug safety tests.

How we do it

PSTC’s 15 corporate members have the same goal: to find improved safety testing methods. The members share their internally developed methods and test these methods developed by one another across the Consortium. Ten EMA and 28 FDA scientists serve as advisors along with more than 250 participating scientists. C-Path leads the collaborative process and collects and summarizes the data.

The testing is done with pre-clinical and clinical safety biomarkers in six working groups: kidney, liver, pancreatic injury, skeletal muscle injury, testicular toxicity, and vascular injury. All biomarker research programs have a strong translational focus to select new safety tools that are applicable across the drug development spectrum.

Researches can access and analyze data in aggregate, or filter and view individual patient-level data from the REMoxTB, RIFAQUIN and OFLUTUB clinical trials. Additional trial data may be available in the future.

TB-PACTS data platform Partnership

This initiative represents a collaborative partnership between the Special Progamme for Research and Training in Tropical Diseases (TDR), the TB Alliance, St. George’s University of London and Critical Path Institute (C-Path).

TB-PACTS data platform Content and Access

The TB–PACTS data platform will catalogue contemporary (TB) clinical trial data sets and make these data sets available to qualified researchers. Approved researchers can access patient-level data from the REMoxTB, RIFAQUIN and OFLOTUB clinical trials. Additional trial data is expected to be made available in the future. For information on how to access the TB-PACTS data platform, visit the TB-PACTS Platform page on this website.

The long-term goal of the TTC is to accelerate the medical product development process for transplantation, identifying 1) areas that have hindered product development, 2) potential biomarkers, endpoints, or process improvements to address those areas, and 3) appropriate regulatory pathways to achieve endorsement for the proposed solutions. The consortium is first focusing on kidney transplant but may expand to other solid organ transplants in the future

Key regulatory science areas have been identified as critical to accomplish the mission of TTC and have resulted in the following workgroups:

• Workgroup I. Endpoints and Biomarkers
• Workgroup II. Drug Safety Profile Characterization and Labeling

TOMI-T1D is a JDRF and Diabetes UK funded international partnership between researchers from academic institutions, pharmaceutical industry and independent non-profit organizations. TOMI’s mission is to accelerate drug development and optimize immune intervention trials in T1D through the development of a composite outcome measure which 1) improves clinical interpretability and patient acceptability 2) shortens the time to primary outcome and 2) minimizes the number of participants required in trials.

To accomplish this, we are developing an open access Clinical Trial Simulation Tool (CTST) which will model anticipated drug and placebo effects based on variability that is specific and relevant to selected patient populations and that will also identify clinical endpoints for optimal clinical trial design. Our goal is to seek regulatory agency (FDA and EMA) endorsement of this CTST which may help facilitate 1) identification of appropriate clinical endpoints and 2) a clearer path for drug development T1D.

Our first step is to assemble a worldwide inventory of completed clinical trials and observational studies with the goal of identifying as many datasets as possible. This will ensure that we build a CTST based on robust data that can be endorsed by regulators and be used in confidence by the biopharmaceutical industry to optimize clinical trial designs. By combining patient level data across studies, the power and interpretability of individual studies are increased and importantly, the CTST will include data from studies where treatment effect may not have been demonstrated, but which are valuable in characterizing disease progression and heterogeneity in T1D.

The Type 1 Diabetes (T1D) Consortium is a public-private partnership initiated in March 2017 by Critical Path Institute (C-Path), The Leona M. and Harry B. Helmsley Charitable Trust; Janssen Research & Development, LLC; JDRF International; Novo Nordisk; and Sanofi. Membership also comprises academic partners and advisors from the NIH.

By 2050, the number of individuals diagnosed with T1D in the US alone is projected to more than triple. The ability to screen for risk and stage of T1D prior to the appearance of symptoms presents a valuable opportunity to delay, and ultimately prevent, symptomatic T1D. Islet autoantibodies are a series of blood-based biomarkers that are tied to the progression of T1D and have the potential to identify those patients who will likely progress to symptomatic T1D. By employing the resources of all its members, and engaging with regulatory agencies at each step of the process, C-Path’s T1D Consortium will work to qualify islet autoimmunity antibodies as prognostic biomarkers to be used in the development of therapies to ultimately the prevent T1D.

The initial goal of the T1D Consortium is to achieve the regulatory qualification (from both the US Food and Drug Administration and the European Medicines Agency) of the islet autoantibodies as prognostic biomarkers for T1D disease progression in pre-symptomatic T1D patients. These autoantibodies may also be used as an enrichment factor in clinical trials to identify subjects in early stages of the disease with a high risk of disease progression to symptomatic.

CFAST was initiated as a partnership between CDISC and the Critical Path Institute (C-Path).  Since launching CFAST, CDISC and C-Path have worked to expand the Therapeutic Area Program Steering Committee (TAPSC) to ensure input from US FDA, the National Cancer Institute Enterprise Vocabulary Services (NCI EVS) and TransCelerate BioPharma. CDISC also initiated the CFAST Scientific Advisory Committee (SAC), which brings advice from the TAPSC organizations and also Innovative Medicines Initiative (IMI) and the Association of Clinical Research Organizations (ACRO).

Mission

To accelerate clinical research and medical product development by creating and maintaining data standards, tools and methods for conducting research in therapeutic areas that are important to public health.

PTC is committed to enabling the creation of a sustainable solution that assures the timely and efficient evaluation of innovative drugs, biologics and devices for children by delivering the regulatory-quality data needed for product labeling. PTC Activities It will provide the support and guidance necessary for C-Path to create a new freestanding non-profit organization (“the Non-Profit”) that will work independently, but with input across relevant sectors. More about PTC Launched in October 2015, PTC is:

• Overseen by a cross-stakeholder Coordinating Committee
• Supported by 5 work streams, project support and communications
• Slated to complete work in 12–18 months

PTC will seek guidance and advice from academia, patient advocacy groups, government scientific and regulatory agencies, biopharmaceutical companies, foundations and other relevant stakeholders.