C-Path Welcomes Alphabet Clinical Policy and Strategy Head, Former FDA Commissioner to Board

TUCSON, Ariz., May 19, 2021 — Critical Path Institute today announced the appointment of Robert M. Califf, MD, MACC, head of Clinical Policy and Strategy for Google parent company Alphabet’s Verily Life Sciences and Google Health divisions, to C-Path’s Board of Directors. Califf served as the U.S. Food and Drug Administration (FDA) Commissioner under President Barack Obama’s administration from 2016-2017.

A nationally and internationally recognized expert in cardiovascular medicine, health outcomes research, health care quality and clinical research, Dr. Califf has led many landmark clinical trials and is one of the most frequently cited authors in biomedical science, with more than 1,200 publications in the peer-reviewed literature.

“It’s an honor to join C-Path’s Board, as its organizational mission and vision align with my conviction that collaborative science is a key component of translating computing and technology into better health and health care,” said Califf. “My roles at Verily and Google Health support this, and I know I will draw from my experience to help C-Path continue to innovate and accelerate the path to drug development and approval.”

Califf has a long history with Duke University and, until November 2019, was founding director of Forge, Duke’s center for actionable health data science and Vice Chancellor for Health Data Science. Included in his tenure at Duke are the Donald F. Fortin, M.D., Professor of Cardiology in the School of Medicine, Vice Chancellor for Clinical and Translational Research and founding director of the Duke Clinical Research Institute, which he helped grow into the nation’s largest academic clinical research organization.

Within the FDA, Califf served as Deputy Commissioner for Medical Products and Tobacco where he provided executive leadership to the Center for Drug Evaluation and Research, Center for Biologics Evaluation and Research, Center for Devices and Radiological Health and Center for Tobacco Products. He also oversaw the Office of Special Medical Programs and provided direction for cross-cutting clinical, scientific and regulatory initiatives, including personalized medicine, orphan drugs, pediatric science and the advisory committee system. He was appointed Commissioner in February 2016 where he was committed to strengthening programs and policies that enabled the agency to carry out its mission to protect and promote the public health.

“We are excited to welcome Dr. Califf to C-Path’s Board. For decades, Rob has been an influential voice in stressing the importance of, and driving, public-private partnerships in the health industry,” said Board Chairman Timothy R. Franson, M.D. “Partnerships and collaboration are the foundation of C-Path’s work, and we know his contributions will make a significant difference in C-Path’s aim to transform the medical product development process.”

Califf has led major initiatives aimed at improving methods and infrastructure for clinical research, including the Clinical Trials Transformation Initiative (CTTI), a public-private partnership co-founded by the FDA and Duke. He also served as the principal investigator for Duke’s Clinical and Translational Science Award and the NIH Health Care Systems Research Collaboratory – Coordinating Center and was co-PI of the Patient-Centered Outcomes Research Institute Network.

He is a member of the National Academy of Medicine (formerly known as the Institute of Medicine (IOM)), one of the highest honors in the fields of health and medicine, and has served on numerous IOM committees and as a member of the FDA Cardiorenal Advisory Panel and FDA Science Board’s Subcommittee on Science and Technology. Califf has also served on the Board of Scientific Counselors for the National Library of Medicine, as well as on advisory committees for the National Cancer Institute, the National Heart, Lung, and Blood Institute, the National Institute of Environmental Health Sciences and the Council of the National Institute on Aging. He remains an Adjunct Professor at Duke and Stanford University.

“Rob’s deep expertise and passionate commitment to breaking down research and data silos have been transformational in creating technical, scientific and regulatory alliances in life sciences and health care,” said C-Path President and COO Kristen Swingle, M.S. “The Board and I look forward to working closely with Rob as we advance C-Path’s comprehensive approach to seeking solutions for unmet needs in the treatment of various diseases and conditions.”

Califf’s Board appointment begins immediately.

Critical Path Institute is supported by the Food and Drug Administration (FDA) of the U.S. Department of Health and Human Services (HHS) and is 55% funded by FDA/HHS, totaling $14,575,306, and 45% funded by non-government source(s), totaling $11,916,747. The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement by, FDA/HHS or the U.S. Government. For more information, please visit FDA.gov.

 


 

Critical Path Institute (C-Path) is an independent, nonprofit organization established in 2005 as a public and private partnership. C-Path’s mission is to catalyze the development of new approaches that advance medical innovation and regulatory science, accelerating the path to a healthier world. An international leader in forming collaborations, C-Path has established numerous global consortia that currently include more than 1,600 scientists from government and regulatory agencies, academia, patient organizations, disease foundations, and dozens of pharmaceutical and biotech companies. C-Path U.S. is headquartered in Tucson, Arizona and C-Path, Ltd. EU is headquartered in Dublin, Ireland, with additional staff in multiple other locations. For more information, visit c-path.org and  c-path.eu.

Contact:
Kissy Black
C-Path
615.310.1894
kblack@c-path.org

C-Path Announces Accelerated Data Sharing Collaboration with Tufts Medical Center

FDA Grant Supports Use of Real-World Data to Generate Real-World Evidence in Neonates

TUCSON, Ariz., May 12, 2021 — Critical Path Institute (C-Path) and Tufts Medical Center have announced a joint venture to integrate the first patient-level clinical trial data to generate actionable real-world evidence (RWE) for neonatal drug development, from real-world neonate data (RWD). This activity, funded by a grant from the U.S. Food and Drug Administration (FDA), is being executed through a neonatal pilot project within C-Path’s International Neonatal Consortium (INC). The data sharing collaboration and transfer also marks the fastest on record for C-Path — starting with the Data Contribution Agreement negotiations and receipt of the data in three months’ time this March.

“I’m extremely proud of both team’s efforts to move swiftly to integrate data, increase our understanding of neonates and improve drug development for this underserved population,” said INC Executive Director Kanwaljit Singh, M.D., M.P.H. “Dr. Jonathan Davis was key in this collaboration and we thank him for all he’s done to move this project forward quickly.”

This effort allows a unique set of clinical trial data to be integrated and managed by C-Path’s Data Collaboration Center (DCC) to support analyses run by its Quantitative Medicine Program. “This will significantly add to the goal of defining actionable reference ranges of commonly used laboratory values in neonates and the development of a natural history model of bronchopulmonary dysplasia,” indicated C-Path’s Chief Science Officer and project Co-Principal Investigator Klaus Romero, M.D., M.S., F.C.P.

The clinical trial data sets shared by Tufts MC will be integrated with other patient-level RWD from electronic health records and data from national registries and networks. Each year in the U.S., 10 percent of neonates are born preterm, creating an urgent need to improve survival and outcome. However, there is minimal new drug development and most existing drugs have insufficient evidence to support safety, efficacy and dosage in this high-risk population.

“I am privileged to work with this exceptional team of clinical and data scientists at C-Path that will make real advances in neonatology,” said Jonathan Davis, M.D., Chief of Newborn Medicine, Associate Director of the Tufts Clinical and Translational Science Institute at Tufts Children’s Hospital, and Co-Principal Investigator. “This collaboration is a perfect marriage. A balance between clinical care, clinical trials, and data science that has never been done before in the neonatology space.”

For more than 15 years, C-Path has engaged in numerous successful partnerships with the FDA and industry to act as a catalyst for drug development. The public-private partnership with FDA, industry, the academic community and families of preterm neonates is unprecedented. The overall goal is to collect, aggregate, standardize and analyze RWD and generate RWE to accelerate drug development efforts. “This style of partnership holds great promise to benefit the unmet needs of the neonatal population and will likely be generalizable to other populations and therapeutic areas as well,” said Singh.

C-Path’s DCC will develop the Real-World Data and Analytics Platform (RW-DAP) as an integrated database and analytics hub designed to be used in generating actionable RWE that can be leveraged to inform drug development in areas where RWD can play a relevant role. It will promote the sharing of relevant RWD and facilitate the optimization of future RWD collection. By integrating relevant RWD in a format suitable for analytics, the RW-DAP will accelerate the understanding of disease progression (including causes for variance in disease progression), clinical outcome measures and biomarkers, and facilitate the development of mathematical models of disease and innovative clinical trial designs. RW-DAP will be positioned to generate solutions to drug development, which can be made publicly available to qualified researchers in industry, government, regulatory agencies and academia.

“C-Path’s INC is well-equipped to lead this project. Real-world data and real-world evidence are crucial to understanding how medical product is performing, and more importantly, how we can make adjustments and explore its potential benefits for other therapeutic areas,” Romero said. “The development of the RW-DAP fits right within DCC’s impressive body of work.”

C-Path’s INC will continue to meet and work with collaborators and new data contributors to integrate additional patient-level datasets. For more information on collaborating with INC, and how to contribute data, please email Adriana Burns at aburns@c-path.org.

Critical Path Institute is supported by the Food and Drug Administration (FDA) of the U.S. Department of Health and Human Services (HHS) and is 55% funded by FDA/HHS, totaling $14,575,306, and 45% funded by non-government source(s), totaling $11,916,747. The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement by, FDA/HHS or the U.S. Government. For more information, please visit FDA.gov.


Critical Path Institute (C-Path) is an independent, nonprofit organization established in 2005 as a public and private partnership. C-Path’s mission is to catalyze the development of new approaches that advance medical innovation and regulatory science, accelerating the path to a healthier world. An international leader in forming collaborations, C-Path has established numerous global consortia that currently include more than 1,600 scientists from government and regulatory agencies, academia, patient organizations, disease foundations, and dozens of pharmaceutical and biotech companies. C-Path U.S. is headquartered in Tucson, Arizona and C-Path, Ltd. EU is headquartered in Dublin, Ireland, with additional staff in multiple other locations. For more information, visit c-path.org and  c-path.eu.

Contact:
Kissy Black
C-Path
615.310.1894
kblack@c-path.org

C-Path Appoints Quantitative Sciences Expert as New Sr. Vice President

Dr. Jeff Barrett will lead C-Path’s RDCA-DAP initiative focusing on rare disease data sharing and accelerating therapy development.

TUCSON, Ariz. May 6, 2021 — Critical Path Institute (C-Path) today announced the appointment of Jeff Barrett, Ph.D., F.C.P., as Senior Vice President and lead for its Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP®) initiative.

Dr. Barrett comes to the role internally, from his pervious role as Senior Advisor to the organization, while working closely with C-Path’s Quantitative Medicine Program. He was previously head of quantitative sciences at the Bill & Melinda Gates Medical Research Institute (MRI) where he was responsible for implementing model-based drug development, employing PK/PD modeling, statistics and clinical trial simulations to advance the discovery and development of new medicines and vaccines.

“With more than 30 years of diverse experience in quantitative science, we are honored to add Jeff to our executive leadership team and have him step in to lead the RDCA-DAP initiative focused on advancing therapies for rare diseases,” said C-Path Interim President and COO Kristen Swingle, M.S.

Prior to MRI, Jeff was Vice President of Translational Informatics at Sanofi Pharmaceuticals. He led various aspects of model-based decision-making and provided leadership for Sanofi’s cloud-based, high-performance computing and “big data” initiatives.

As SVP, and in partnership with senior leadership, Dr. Barrett will guide all operational and scientific activities for RDCA-DAP. These activities will include continuing outreach and collaboration with the rare disease community to optimize data usability and availability, as well as transforming such data into actionable knowledge to advance drug development for rare and orphan conditions. He will also spearhead the public launch of the platform in September 2021.

“Jeff’s experience and current work embodies C-Path’s vision of being a partner of excellence in transforming the medical product development process worldwide,” said C-Path Chief Science Officer Klaus Romero, M.D., M.S., F.C.P. “We are looking forward to the impact Jeff will have on RDCA-DAP and the rare disease drug development ecosystem.”

RDCA-DAP is a collaboration between C-Path and the National Organization for Rare Disorders (NORD) created to provide a centralized and standardized infrastructure to accelerate and optimize the quantitative characterization of rare diseases, with the goal of accelerating therapy development. Dr. Barrett succeeds Jane Larkindale, D.Phil., Executive Director of RDCA-DAP since its inception in 2019.

Critical Path Institute is supported by the Food and Drug Administration (FDA) of the U.S. Department of Health and Human Services (HHS) and is 55% funded by FDA/HHS, totaling $14,575,306, and 45% funded by non-government source(s), totaling $11,916,747. The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement by, FDA/HHS or the U.S. Government. For more information, please visit FDA.gov.


Critical Path Institute (C-Path) is an independent, nonprofit organization established in 2005 as a public and private partnership. C-Path’s mission is to catalyze the development of new approaches that advance medical innovation and regulatory science, accelerating the path to a healthier world. An international leader in forming collaborations, C-Path has established numerous global consortia that currently include more than 1,600 scientists from government and regulatory agencies, academia, patient organizations, disease foundations, and dozens of pharmaceutical and biotech companies. C-Path U.S. is headquartered in Tucson, Arizona and C-Path, Ltd. EU is headquartered in Dublin, Ireland, with additional staff in multiple other locations. For more information, visit c-path.org and c-path.eu.

Media Contact: Kissy Black
C-Path
615.310.1894
kblack@c-path.org

C-Path Selects New Leaders of Data Science and Quantitative Medicine

TUCSON, Ariz., April 26, 2021 — Critical Path Institute (C-Path) has named Amanda J. Borens, M.S., as Executive Director of Data Science within C-Path’s Data Collaboration Center (DCC), and Jackson Burton, Ph.D., as Executive Director of C-Path’s Quantitative Medicine Program.

Amanda Borens, MSc
Executive Director of Data Science

Borens has been with C-Path for nearly five years and has more than 20 years of development, analytics and scientific experience in academia, clinical settings, health care informatics and biotech companies. She brings a wealth of practical experience and leadership to C-Path’s Data Collaboration Center.

“Time and again, Amanda has demonstrated her technical skills and experience in leading key C Path data collaboration projects to success,” said C-Path Chief Technology Officer and Data Collaboration Center Director Rick Liwski. “Every day, C Path relies on Amanda’s knowledge and experience to drive critical DCC programs and guide our data science strategy.”

Borens’ experience in the emerging field of data science led her to work in medical diagnostics, where she was part of a device development team that successfully achieved U.S. Food and Drug Administration clearance and CE Marking in Europe. Throughout her career, there has been a common thread of providing Big Data solutions to solve scientific problems and acting as the interface between life scientists and technology innovation.

As Executive Director of Data Science, Borens is responsible for leading the Data Science team, which aims to increase the FAIRness (findable, accessible, inoperable and reusable) of data by developing and integrating semantic standards, tools for consumption and sharing of data, performing data transformations that increase data accessibility, and by performing analyses that transform data into information — all core components of C-Path’s expertise. In addition to her management responsibilities, Borens also has the technical responsibility for the architecture and development of C-Path’s overall data and analytics platform strategy. The initial focus of her efforts has been on the Rare Disease Cure Accelerator–Data and Analytics Platform (RDCA-DAP®) and the evolution of its data efforts moving forward.

Jackson Burton, PhD
Executive Director,
Quantitative Medicine Program

Burton obtained his doctoral degree in applied mathematics at the University of Arizona, where he focused on quantitative modeling of drug transport in solid tumors. Prior to joining C-Path, Burton worked in industry settings conducting modeling and statistical analyses for a variety of mission-driven quantitative solutions in oncology and business decision science. He has been with C-Path for four years.

As leader of C-Path’s Quantitative Medicine Program, Burton oversees the organization’s efforts in the development of innovative quantitative solutions to accelerate medical product development across therapeutic areas (including neurological disorders, diabetes, solid organ transplantation, rare/orphan diseases and pediatrics). Such solutions include clinical trial simulation tools, model-based biomarker tools, machine learning and AI for remote monitoring technologies, and evidence generation from real-world data. Additionally, he leads C-Path’s postdoctoral fellowship program in model-informed drug development, where the next generation of cutting-edge scientists who will further transform medical product development paradigms are being trained.

“Jackson embodies the type of scientist needed to truly accelerate medical product development in the 21st century,” said C-Path Chief Science Officer Klaus Romero, M.D., M.S., F.C.P. “With his expertise in integrating mathematics and medicine, and his ability to lead the team that transforms data into actionable solutions to accelerate and de-risk the R&D and regulatory process, the lives of countless patients will benefit from his leadership at C-Path.”

For more information on C-Path’s Data Collaboration Center, visit https://c-path.org/programs/dcc. For more information on C-Path’s Quantitative Medicine Program, visit: https://c-path.org/programs/quantmed.

Critical Path Institute is supported by the Food and Drug Administration (FDA) of the U.S. Department of Health and Human Services (HHS) and is 55% funded by FDA/HHS, totaling $14,575,306, and 45% funded by non-government source(s), totaling $11,916,747. The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement by, FDA/HHS or the U.S. Government. For more information, please visit FDA.gov.


 

Critical Path Institute (C-Path) is an independent, nonprofit organization established in 2005 as a public and private partnership. C-Path’s mission is to catalyze the development of new approaches that advance medical innovation and regulatory science, accelerating the path to a healthier world. An international leader in forming collaborations, C-Path has established numerous global consortia that currently include more than 1,600 scientists from government and regulatory agencies, academia, patient organizations, disease foundations, and dozens of pharmaceutical and biotech companies. C-Path U.S. is headquartered in Tucson, Arizona and C-Path, Ltd. EU is headquartered in Dublin, Ireland, with additional staff in multiple other locations. For more information, visit
www.c-path.org and  c-path.eu.

Media Contact:
Kissy Black
C-Path
615.310.1894
kblack@c-path.org

C-Path Opens Access to Duchenne Regulatory Science Consortium Database

Database will allow sharing of individual-level data with the goal of accelerating therapy development for Duchenne muscular dystrophy

TUCSON, Ariz., April 22, 2021 — Critical Path Institute (C-Path) announced today that it will open access to the Duchenne Regulatory Science Consortium (D-RSC) database to qualified researchers, through its Rare Disease Cures Accelerator, Data and Analytics Platform (RDCA-DAP®). The D-RSC database includes data from Duchenne muscular dystrophy (DMD) clinical trials, natural history studies and clinical data collections. The contributors of these datasets have given permission for this data to be shared externally to accelerate therapy development for DMD.

DMD is a rare, fatal, genetic neuromuscular disorder that is diagnosed in childhood and primarily affects males. It causes progressive muscle loss, resulting in the loss of the ability to walk, loss of upper body strength, progressive breathing issues, cardiomyopathy and premature death. Although there are medical treatments that may help slow its progression, there is currently no cure for DMD.

C-Path’s RDCA-DAP provides a centralized and standardized infrastructure to support and accelerate rare disease characterization, with the goal of accelerating therapy development across all rare diseases. By sharing this valuable data resource through RDCA-DAP, D-RSC aims to extend and amplify the availability of data to accelerate therapy development for DMD, foster the development of tools to accelerate clinical trials for DMD and help develop a deeper understanding of DMD natural history.

“Individuals living with Duchenne are in urgent need of effective treatments,” explained C-Path’s Chief Science Officer Klaus Romero, M.D., M.S., F.C.P. “With the strength in numbers of data available in the D-RSC database, we believe that opening this platform and making data available to qualified researchers will have a profound effect on expediting the drug development process.”

All data in the D-RSC database have been mapped to current Clinical Data Interchange Standards Consortium (CDISC) Standard Data Tabulation Model (SDTM) structures. Data contained in the D-RSC database are fully de-identified and datasets are integrated, so researchers will not be able to identify participants of the studies and data can be used in aggregate.

D-RSC was formed through collaboration between the Critical Path Institute and Parent Project Muscular Dystrophy. Individuals and companies from industry and academia are members of the consortium, and representatives of the U.S. Food and Drug Administration, European Medicines Agency and the National Institutes of Health have joined the consortium coordinating committee as observers.

Researchers interested in contributing data to D-RSC and/or utilizing the database to advance DMD-related research initiatives can learn more at: https://c-path.org/programs/d-rsc/overview/database-access/ or contact D-RSC Associate Director Ramona Belfioire-Oshan, Ph.D., at rbelfioreoshan@c-path.org.

Critical Path Institute is supported by the Food and Drug Administration (FDA) of the U.S. Department of Health and Human Services (HHS) and is 55% funded by FDA/HHS, totaling $14,575,306, and 45% funded by non-government source(s), totaling $11,916,747. The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement by, FDA/HHS or the U.S. Government. For more information, please visit FDA.gov.


Critical Path Institute (C-Path) is an independent, nonprofit organization established in 2005 as a public and private partnership. C-Path’s mission is to catalyze the development of new approaches that advance medical innovation and regulatory science, accelerating the path to a healthier world. An international leader in forming collaborations, C-Path has established numerous global consortia that currently include more than 1,600 scientists from government and regulatory agencies, academia, patient organizations, disease foundations, and dozens of pharmaceutical and biotech companies. C-Path US is headquartered in Tucson, Arizona and C-Path, Ltd. EU is headquartered in Dublin, Ireland, with additional staff in multiple other locations. For more information, visit www.c-path.org and  c-path.eu.

Media Contact:
Kissy Black
C-Path
615.310.1894
kblack@c-path.org

C-Path’s Alzheimer’s Disease Consortium Expands Data Repository

New patient-level data will facilitate more efficient clinical trial design

TUCSON, Ariz., April 12, 2021 — The Critical Path Institute’s (C-Path) Critical Path for Alzheimer’s Disease (CPAD) Consortium today announced that it is significantly expanding its Alzheimer’s disease (AD) patient-level data repository with high-quality contemporary industry clinical trial datasets, focusing on early stages of the disease.

Generous data contributions and support from the pharmaceutical industry have made it possible for the CPAD team to integrate 42 high-quality clinical trial and observational studies in AD, containing more than 21,000 patient-level records.

Since July 2019, CPAD has signed Data Contribution Agreements (DCAs) with data contributors, for five highly relevant datasets, representing over 4,000 patient-level records, which have been integrated into the CPAD repository. CPAD also expects the transfer of two additional datasets with another 4,000 patient-level records. In addition, CPAD is currently negotiating DCAs with five CPAD member pharmaceutical companies, with an expected addition of nine datasets, bringing in more than 10,000 additional patient-level records to the CPAD repository.

“Serving as a neutral third party, CPAD provides a safe harbor for individual organizations to share data in a pre-competitive environment,” said Sudhir Sivakumaran, Ph.D., Vice President, Neuroscience at C-Path and Executive Director of CPAD. “By facilitating cross-functional, pre-competitive collaboration and active engagement between the regulatory agencies and drug development professionals, we can make a significant difference in the bench to bedside timeline for patients.”

The expanded patient-level data will allow generation of novel drug development tools (DDTs) and solutions such as disease progression models across the entire continuum of AD, incorporating various fluid and imaging biomarkers, in addition to genotype, cognitive and functional outcome assessments and demographics. The novel tools and solutions will enable a more efficient design of clinical studies with better informed inclusion criteria, endpoint selection and patient enrichment strategies. C-Path’s quantitative approach allows for a comprehensive integration of relevant patient characteristics for informed and efficient decision‐making in drug development.

Data sharing is fundamental to enable the development of novel DDTs, which require integration and aggregation of patient-level data from multiple independent sources and studies. Successful data sharing for the purpose of seeking regulatory endorsement of a DDT relies on extensive partnerships between industry, academia, research institutes and societies, patient-advocacy groups, and governmental regulatory agencies. “CPAD’s successes and accomplishments in the last decade is testimony to the extensive collaboration, partnership, and support of the industry members,” Sivakumaran continued. “The consortium seeks to continue accelerating drug development by increased data sharing and ultimately, be able to successfully address the key unmet needs and unanswered questions including the voice of the patients.“

“CPAD provides the AD field with a unique opportunity to transform patient-level data into actionable solutions, which can in turn transform the drug development process for Alzheimer’s disease,” said C-Path Chief Science Officer and Executive Director for Clinical Pharmacology Klaus Romero, M.D., M.S., F.C.P. “The unique way in which CPAD presents such solutions for formal regulatory review generates the necessary confidence for the adoption of such solutions by our industry partners.”

Data acquisition and integration is a critical prerequisite for the development of novel regulatory-grade quantitative drug development solutions. For more than a decade, CPAD has led the space with regulatory successes and expertise in data aggregation and generation of novel drug development tools. The consortium continues to pursue efforts to expand its datasets, which will significantly add to the increased understanding of disease progression and pathology in AD.

For more information, visit https://c-path.org/programs/cpad/. To learn more about data contribution to the repository, email info@c-path.org. To access the Critical Path Institute (C-Path) Online Data Repository (CODR) go to https://codr.c-path.org/main/applyDatabaseSelection.html

Critical Path Institute is supported by the Food and Drug Administration (FDA) of the U.S. Department of Health and Human Services (HHS) and is 55% funded by FDA/HHS, totaling $14,575,306, and 45% funded by non-government source(s), totaling $11,916,747. The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement by, FDA/HHS or the U.S. Government. For more information, please visit FDA.gov.

 


Critical Path Institute (C-Path) is an independent, nonprofit organization established in 2005 as a public and private partnership. C-Path’s mission is to catalyze the development of new approaches that advance medical innovation and regulatory science, accelerating the path to a healthier world. An international leader in forming collaborations, C-Path has established numerous global consortia that currently include more than 1,600 scientists from government and regulatory agencies, academia, patient organizations, disease foundations, and dozens of pharmaceutical and biotech companies. C-Path US is headquartered in Tucson, Arizona and C-Path, Ltd. EU is headquartered in Dublin, Ireland, with additional staff in multiple other locations. For more information, visit www.c-path.org and  c-path.eu.

 

Media Contact:
Kissy Black
C-Path
615.310.1894
kblack@c-path.org

C-Path Program and Consortia Leaders Selected to Serve in Sr. Vice President and Vice President Roles

Inish O’Doherty and Sudhir Sivakumaran will lead new programs;
Stephen Joel Coons and John-Michael Sauer move into Sr. VP roles.

 

TUCSON, Ariz., March 10, 2021 — Critical Path Institute (C-Path) today announced it has promoted Inish O’Doherty, Ph.D., to Vice President of the organization’s newly formed Immunology and Hematology Program and Sudhir Sivakumaran, Ph.D., to Vice President for its Neuroscience Program, effective immediately. Both Drs. O’Doherty and Sivakumaran currently serve as Executive Directors of multiple C-Path consortia. Additionally, current Program Officer Stephen Joel Coons, Ph.D., will now serve as Senior Vice President of the Clinical Outcome Assessment (COA) Program and John-Michael Sauer, Ph.D., as Senior Vice President of Translational and Safety Sciences Program.

The promotions and new program groups stem from a reorganization and optimization within C-Path after a year of strategic development planning.

As Vice Presidents, and in partnership with senior leadership, Dr. O’Doherty and Dr. Sivakumaran will guide all operational and scientific activities for the Immunology and Hematology and Neuroscience Programs, respectively. These activities will include supporting and implementing strategic priorities for the various consortia in these programs, and individual working groups within them. Each will also work with key individuals at regulatory agencies worldwide, including the U.S. Food and Drug Administration (FDA), European Medicines Agency (EMA) and the Pharmaceuticals and Medical Devices Agency (PMDA).

Dr. O’Doherty, a clinical development scientist, has been with C-Path for more than four years, as Executive Director of C-Path’s Type 1 Diabetes (T1D) Consortium and Transplant Therapeutic Consortium (TTC). His work focuses on the pre-competitive development of biomarkers and quantitative tools, which aim to optimize clinical trial design and minimize risk in regulatory decision-making.

Dr. Sivakumaran, a neuroscientist and research and development professional, has nearly 20 years combined experience in academic, pre-clinical and early clinical research and development, and business development. He has been with C-Path since July 2019 and serves as Executive Director of the Critical Path for Alzheimer’s Disease (CPAD) consortium and Interim Executive Director for the Huntington’s Disease Regulatory Science Consortium (HD-RSC).

The Immunology and Hematology Program will include T1D, TTC and the Critical Path for Sickle Cell Disease (CP-SCD) Consortium. The Neuroscience Program will consist of CPAD, HD-RSC, Critical Path for Parkinson’s (CPP) Consortium, Duchenne Regulatory Science Consortium (D-RSC) and the Critical Path to Therapeutics for the Ataxias (CPTA) Consortium.

“Inish and Sudhir are natural selections for these new leadership roles within C-Path,” said C-Path President and CEO Joseph Scheeren, Pharm. D. “They bring deep and insightful experience to the programs and will continue to drive the organization’s strategic leadership and business development initiatives in support of C-Path as the premier advisor to academia, industry, regulators and other partners in the ever-evolving medical product development and regulatory ecosystem.”

The Translational and Safety Sciences Program will focus on drug safety, nephrology, inflammatory bowel disease (IBD), drug repurposing and translational therapeutics, where the COA Program will continue its work on patient-reported outcome measures and other COAs, with an added emphasis on rare diseases.

For three decades, Dr. Coons has focused on incorporating the patient’s voice into the assessment of health and health care. Prior to C-Path, he was a professor in the College of Pharmacy and the Mel & Enid Zuckerman College of Public Health at the University of Arizona. Dr. Coons is a fellow in the American Association of Pharmaceutical Scientists and Professor Emeritus at the University of Arizona.

Dr. John-Michael Sauer is a toxicologist by training with more than 15 years’ experience in drug discovery and development. He has led multiple functional areas across pharmaceutical companies and is dedicated to bringing quantitative translational science approaches to safety assessment, as well as transforming the way we use nonclinical safety data to drive clinical study design and data interpretation. Dr. Sauer is a Research Professor, Department of Pharmacology, College of Medicine, Professor of Practice, James E. Rogers College of Law and Designated Campus Colleague, University of Arizona.

“John-Michael and Stephen are accomplished experts in their respective fields, and we’re honored to have them continue to lead these areas for C-Path,” said Scheeren. “As longstanding members of our leadership team, they have made significant contributions to the growth and success of the organization.”

Reporting directly to Dr. Scheeren, O’Doherty, Sivakumaran, Coons and Sauer will continue to serve on C-Path’s leadership team, “helping to shape the innovative and regulatory pathways to accelerate delivery of therapies for patients in need,” Scheeren said.

Critical Path Institute is supported by the Food and Drug Administration (FDA) of the U.S. Department of Health and Human Services (HHS) and is 55% funded by FDA/HHS, totaling $14,575,306, and 45% funded by non-government source(s), totaling $11,916,747. The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement by, FDA/HHS or the U.S. Government. For more information, please visit FDA.gov.


Critical Path Institute (C-Path) is an independent, nonprofit organization established in 2005 as a public and private partnership. C-Path’s mission is to catalyze the development of new approaches that advance medical innovation and regulatory science, accelerating the path to a healthier world. An international leader in forming collaborations, C-Path has established numerous global consortia that currently include more than 1,600 scientists from government and regulatory agencies, academia, patient organizations, disease foundations, and dozens of pharmaceutical and biotech companies. C-Path US is headquartered in Tucson, Arizona and C-Path, Ltd. EU is headquartered in Dublin, Ireland, with additional staff in multiple other locations. For more information, visit c-path.org and c-path.eu.

Media Contact:

Kissy Black
C-Path
615.310.1894
kblack@c-path.org

 

C-Path and Diamyd Medical Announce Data Sharing Collaboration to Develop Advanced Drug Development Tools in Type 1 Diabetes

 

TUCSON, Ariz., and STOCKHOLM, Sweden. December 16, 2020 — The Critical Path Institute (C-Path) and Diamyd Medical (DMYD B; Nasdaq First North Growth Market) are proud to announce their collaboration to significantly improve the scientific community’s insight into type 1 diabetes (T1D) through Diamyd Medical’s contribution of fully anonymized data from a European Phase III trial to the Trial Outcome Markers Initiative (TOMI-T1D) integrated database. The Phase III trial evaluated the use of the diabetes vaccine Diamyd®, an antigen-specific immunotherapy based on the auto-antigen GAD (glutamic acid decarboxylase), to induce immunological tolerance and stop the autoimmune destruction of insulin producing cells. The Data Contribution Agreement (DCA) between Diamyd Medical and C-Path will allow for this unique set of fully anonymized clinical trial data to be integrated into an ever-growing list of committed trial data sets within the TOMI-T1D project.

TOMI-T1D is an international partnership between academia, the pharmaceutical industry and nonprofit organizations. It is funded by the world’s leading charities dedicated to diabetes research, JDRF, and Diabetes UK, guided by both organizations’ strong commitment to facilitate deep interrogation of consolidated community-wide trial data as a means to accelerate clinical research and therapeutic development for T1D. TOMI-T1D aims to create a clinical trial simulation tool (CTST) with large and diverse clinical datasets from the T1D community. The project also seeks to engage the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) to identify opportunities for regulatory endorsement of such drug development tools.

The Diamyd Medical data includes relevant information about disease progression, drug effects and clinical trial design. Contribution of these robust data sets from industry led trials is critical to TOMI-T1D’s work in developing innovative and quantitative tools that can facilitate clinical development efforts and be endorsed by regulators for future use by the pharmaceutical industry to optimize the design of future clinical trials.

“Progress towards the establishment of approved therapies for people with T1D is critically reliant on participation from our partners in industry with their data,” said Simi Ahmed and Elizabeth Robertson, on behalf of the charity partnership. “This is indeed a right step in that direction,” said Colin Dayan, lead PI at Cardiff University.

“We are thrilled that Diamyd Medical is taking a leading role and championing precompetitive collaborations advancing type 1 diabetes regulatory science solutions,” said C-Path Executive Director Inish O’Doherty, Ph.D. “Their data will help in the construction and evaluation of a clinical trial simulation tool to assist in the development of novel therapies for type 1 diabetes patients.”

“We are very honored to be part of this important collaboration -involving key stakeholders within the T1D landscape,” said Ulf Hannelius, President & CEO of Diamyd Medical. “As we are moving into an era of precision medicine in type 1 diabetes, we can expect to see significant therapeutic advances, and access to high quality data will be integral to maximizing these efforts.”

To learn more about the TOMI-T1D project visit: https://c-path.org/programs/tomi-t1d/

TOMI-T1D is solely funded by JDRF and Diabetes UK. Critical Path Institute is supported by the U.S. Food and Drug Administration (FDA) of the U.S. Department of Health and Human Services (HHS) and is 69% funded by FDA/HHS, totaling $19,471,171, and 31% funded by non-government source(s), totaling $8,612,313. The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement by, FDA/HHS or the U.S. Government.


About the Organizations:

Critical Path Institute (C-Path) is an independent, nonprofit organization established in 2005 as a public and private partnership. C-Path’s mission is to catalyze the development of new approaches that advance medical innovation and regulatory science, accelerating the path to a healthier world. An international leader in forming collaborations, C-Path has established numerous global consortia that currently include more than 1,600 scientists from government and regulatory agencies, academia, patient organizations, disease foundations, and dozens of pharmaceutical and biotech companies. C-Path US is headquartered in Tucson, Arizona and C-Path, Ltd. EU is headquartered in Dublin, Ireland, with additional staff in multiple other locations. For more information, visit c-path.org and c-path.eu.


 

Diamyd Medical develops therapies for type 1 diabetes. The diabetes vaccine Diamyd® is an antigen-specific immunotherapy for the preservation of endogenous insulin production. Significant results have been shown in a genetically predefined patient group in a large-scale metastudy as well as in the Company’s European Phase IIb trial DIAGNODE-2, where the diabetes vaccine is administered directly into a lymph node in children and young adults with newly diagnosed type 1 diabetes. A new facility for vaccine manufacturing is being set up in Umeå for the manufacture of recombinant GAD65, the active ingredient in the therapeutic diabetes vaccine Diamyd®. Diamyd Medical also develops the GABA-based investigational drug Remygen® as a therapy for regeneration of endogenous insulin production and to improve hormonal response to hypoglycaemia. An investigator-initiated Remygen® trial in patients living with type 1 diabetes for more than five years is ongoing at Uppsala University Hospital. Diamyd Medical is one of the major shareholders in the stem cell company NextCell Pharma AB. Diamyd Medical’s B-share is traded on Nasdaq First North Growth Market under the ticker DMYD B. FNCA Sweden AB is the Company’s Certified Adviser; phone: +46 8-528 00 399, e-mail: info@fnca.se.


 

JDRF‘s mission is to accelerate life-changing breakthroughs to cure, prevent, and treat T1D and its complications. To accomplish this, JDRF has invested more than $2.5 billion in research funding since our inception. We are an organization built on a grassroots model of people connecting in their local communities, collaborating regionally for efficiency and broader fundraising impact and uniting on a national stage to pool resources, passion and energy. We collaborate with academic institutions, policymakers and corporate and industry partners to develop and deliver a pipeline of innovative therapies to people living with T1D. Our staff and volunteers throughout the United States and our five international affiliates are dedicated to advocacy, community engagement and our vision of a world without T1D. For more information, please visit jdrf.org or follow us on Twitter: @JDRF.


 

1.   Diabetes UK’s UK’s aim is creating a world where diabetes can do no harm. Diabetes is the most devastating and fastest growing health crisis of our time, affecting more people than any other serious health condition in the UK – more than dementia and cancer combined. There is currently no known cure for any type of diabetes. With the right treatment, knowledge and support people living with diabetes can lead a long, full and healthy life. For more information about diabetes and the charity’s work, visit www.diabetes.org.uk.
2.   Diabetes is a condition where there is too much glucose in the blood because the body cannot use it properly. If not managed well, both type 1 and type 2 diabetes can lead to devastating complications. Diabetes is one of the leading causes of preventable sight loss in people of working age in the UK and is a major cause of lower limb amputation, kidney failure and stroke.
3.   People with type 1 diabetes cannot produce insulin. About 10 per cent of people with diabetes have type 1. No one knows exactly what causes it, but it’s not to do with being overweight and it isn’t currently preventable. It’s the most common type of diabetes in children and young adults, starting suddenly and getting worse quickly. Type 1 diabetes is treated by daily insulin doses – taken either by injections or via an insulin pump. It is also recommended to follow a healthy diet and take regular physical activity.
4.   People with type 2 diabetes don’t produce enough insulin or the insulin they produce doesn’t work properly (known as insulin resistance). Around 90 per cent of people with diabetes have type 2. They might get type 2 diabetes because of their family history, age and ethnic background puts them at increased risk. They are also more likely to get type 2 diabetes if they are overweight. It starts gradually, usually later in life, and it can be years before they realise they have it. Type 2 diabetes is treated with a healthy diet and increased physical activity. In addition, tablets and/or insulin can be required.

For more information on reporting on diabetes, download our journalists’ guide: Diabetes in the News: A Guide for Journalists on Reporting on Diabetes (PDF, 3MB).

Contact:
Kissy Black
C-Path
615.310.1894
kblack@c-path.org

 

C-Path Selects Aridhia to Support Rare Disease Cures Accelerator-Data and Analytics Platform

 

TUCSON, Ariz., December 3, 2020 — The Critical Path Institute (C-Path) today announced it has selected Aridhia to support its Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP). The RDCA-DAP initiative, launched in September 2019 as a collaborative project between C-Path and the National Organization for Rare Disorders® (NORD), will provide a centralized and standardized infrastructure to accelerate and optimize the quantitative characterization of rare diseases, with the goal of accelerating therapy development. RDCA-DAP is designed to accept patient-level rare disease data from clinical trials, observational studies, real-world data, patient registries and other sources, to support the analysis and interpretation of those data.

Based in Scotland, Aridhia encompasses a multidisciplinary team of data scientists, information governance specialists, computer scientists, software developers and health care experts who are uniquely skilled to give researchers and innovators the ability to discover and understand data through dataset searching, classification and efficient metadata browsing capabilities. Aridhia will work closely with C-Path’s Data Collaboration Center (DCC) team of top-tier technical, scientific and project management experts to build a neutral, precompetitive data collaboration environment to support advanced research efforts in rare diseases.

“We look forward to working with Aridhia on this exciting collaboration,” said C-Path Executive Director of Data Science Amanda Borens, MS. “RDCA-DAP is an important resource for the entire rare disease community. Using Aridhia technology, RDCA-DAP will help the community come together and share data and knowledge that will revolutionize the development of therapies for rare diseases.”

Aridhia was chosen by C-Path based on its unparalleled expertise in providing secure cloud-enabled digital research environments for biomedical, precision medicine and health care researchers. Coupled with the fact that Aridhia has been on the forefront of the fair, lawful and transparent handling of personal data — prior to the implementation of the EU’s General Data Protection Regulations (GDPR) — and have continued to be an industry leader in the strict adherence to GDPR standards, Aridhia was the stand-out choice to assist with the development of the RDCA-DAP.

“We’re delighted to be working with colleagues at C-Path on such an important area of biomedical research,” said Aridhia CEO David Sibbald. “We know that improving therapy development for rare disease requires significant data discovery and data access, while preserving the governance requirements of individual data contributors. Our collaboration will help the rare disease scientific community gain access to richer, more comprehensive datasets to accelerate rare disease therapy development.”

RDCA-DAP already includes integrated rare disease data from a number of different sources — including those within NORD’s IAMRARE™ registry platform — and building in advanced analytics capabilities will allow efficient and effective interrogation of that data to generate solutions for clinical trial design and regulatory review.

Groups interested in contributing data to the effort, collaborating on rare disease research or using the platform for their own research may visit c-path.org/rdca-dap or email rdcadap@c-path.org for more information. The platform is open to accept data immediately and the user interface and analytics will be available in early 2021, with additional analytics tools being developed concurrently.

Critical Path Institute is supported by the Food and Drug Administration (FDA) of the U.S. Department of Health and Human Services (HHS) and is 69% funded by FDA/HHS, totaling $19,471,171, and 31% percent funded by non-government source(s), totaling $8,612,313. The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement by, FDA/HHS or the U.S. Government.

 


 

About Critical Path Institute
Critical Path Institute (C-Path) is an independent, nonprofit organization established in 2005 as a public and private partnership. C-Path’s mission is to catalyze the development of new approaches that advance medical innovation and regulatory science, accelerating the path to a healthier world. An international leader in forming collaborations, C-Path has established numerous global consortia that currently include more than 1,600 scientists from government and regulatory agencies, academia, patient organizations, disease foundations, and dozens of pharmaceutical and biotech companies. C-Path US is headquartered in Tucson, Arizona and C-Path, Ltd. EU is headquartered in Dublin, Ireland, with additional staff in multiple other locations. For more information, visit c-path.org and c-path.eu.

 

About Aridhia
Aridhia is a health data science company founded in 2008 and based in Glasgow’s Queen Elizabeth University hospital and central Edinburgh. Aridhia provides a cloud based digital research platform enabling both the discovery and analysis of data to take place in a trusted, secure environment at scale. Our multidisciplinary team of data scientists, information governance specialists, computer scientists, software developers and health care experts delivers research services across the UK and Europe, working collaboratively with the NHS, research organizations, life sciences companies, IT service providers and patients to transform clinical research into clinical practice.

 

Contact:

Kissy Black
C-Path
kissyblack@c-path.org

C-Path, Ltd. Announces New Contract with Innovative Medicines Initiative

 

DUBLIN, Ireland, December 3, 2020 — Critical Path Institute, Ltd. (C-Path, Ltd.) announced today a new contract with the Innovative Medicines Initiative 2 Joint Undertaking (IMI2 JU). The new project will leverage C-Path’s global expertise in developing novel product development tools.

“The mission of IMI, to improve health by speeding up the development of, and patient access to, innovative medicines, is carried out through collaborative projects, and each project has taken large steps forward towards reaching this goal,” said Pierre Meulien, IMI2 JU’s Executive Director. “IMI’s new contract with C-Path, Ltd. will identify opportunities to expand the impact of these projects for patients across Europe.”

Novel methodologies, including biomarkers, clinical outcome assessments and disease progression models, are tools that inform regulatory decision-making during the product development process. These tools can decrease clinical trial size and/or duration, inform endpoint selection, increase the patient voice, improve safety monitoring and more, depending on the tool. As a result, product developers have a more efficient process, bringing therapies to patients faster.

“C-Path’s expertise lies beyond leading collaborations that gather consensus among its member organizations,” said Acting Chief Executive of C-Path, Ltd. Graham Higson, M.Sc. “We’ve been uniquely successful in using these collaborations to identify key product development needs, assessing available data, then using that data to develop novel drug development tools.”

Often, these tools can be submitted for endorsement at the regulatory agencies, such as the European Medicines Agency. By doing so, product developers have increased confidence in the tool and are more likely to incorporate it into their development programs. Regulatory endorsed tools are also publicly available on agency websites.


Critical Path Institute, Ltd. (C-Path, Ltd.) is a wholly owned subsidiary of Critical Path Institute (C-Path), an independent, nonprofit organization established in 2005 as a public and private partnership. C-Path’s mission is to catalyze the development of new approaches that advance medical innovation and regulatory science, accelerating the path to a healthier world. An international leader in forming collaborations, C-Path has established numerous global consortia that currently include more than 1,600 scientists from government and regulatory agencies, academia, patient organizations, disease foundations, and dozens of pharmaceutical and biotech companies. C-Path, Ltd. EU is headquartered in Dublin, Ireland, and C-Path US is headquartered in Tucson, Arizona, with additional staff in multiple other locations. For more information, visit www.c-path.org and  c-path.eu.

 

Media Contact:
Kissy Black
C-Path
615.310.1894
kblack@c-path.org