C-Path Receives DIA 2022 Americas Inspire Award for Outstanding Contribution to Health

TUCSON, Ariz., June 22, 2022 — Critical Path Institute (C-Path) is pleased to announce it has received the DIA 2022 Americas Inspire Award for Outstanding Contribution to Health. This prestigious honor is conferred upon an individual, group or organization that has made significant and innovative contributions to advancing global health.

“The work that Critical Path Institute has done through its programs has led to the development of tools and approaches to solve some of the most critical challenges in the product development pathway, including the need for aggregated data, and a platform for communication and collaboration,” wrote DIA representatives Anna McDermott-Vitak, Senior Vice President and Managing Director, Americas; and Stephanie Crawford, Ph.D., Chair, Americas Regional Advisory Council.

As also articulated by DIA, “One C-Path program, the eCOA Consortium, for example, has advanced the science of trial endpoints, particularly through a focus on patient reported outcomes through its ‘[eCOA:] Getting Better Together Initiative.’ Further, the eCOA Consortium has been a trusted and invaluable partner in the development of the DIA Digital Technology in Clinical Trials Conference that was founded in 2020.” Scottie Kern, Executive Director of the eCOA Consortium, accepted the award on behalf of C-Path at the Awards Celebration hosted by DIA’s Board of Directors in Chicago, Illinois on June 20, 2022.

“C-Path is honored to receive this prestigious award, affirming we are accomplishing our mission to accelerate the path to a healthier world,” said Kristen Swingle, M.S., C-Path Interim President and COO. “The eCOA Consortium team and everyone at C-Path would like to thank the DIA for this recognition.”



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 hundreds of pharmaceutical and biotech companies. C-Path U.S. is headquartered in Tucson, Arizona, C-Path in Europe is headquartered in Amsterdam, Netherlands and C-Path Ltd. operates from Dublin, Ireland with additional staff in multiple other locations. For more information, visit 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 54.2% funded by the FDA/HHS, totaling $13,239,950, and 45.8% funded by non-government source(s), totaling $11,196,634. 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 C-Path’s Electronic Clinical Outcome Assessment (eCOA) Consortium
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.

For more information on C-Path’s eCOA Consortium, visit:
https://c-path.org/programs/ecoac

Media Contact:

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

C-Path Launches Unique Translational Therapeutics Accelerator with Support from Cottrell Foundation

The global accelerator will transform academic discoveries to drug development opportunities

TUCSON, Ariz., June 9, 2022 — Critical Path Institute (C-Path) today announced it has been awarded a grant from the Frederick Gardner Cottrell Foundation, a non-profit organization established by Research Corporation Technologies, Inc. (RCT) that provides financial support for scientific research and educational programs, to create the C-Path Translational Therapeutics Accelerator (TRxA). The accelerator is a unique, global effort focused on supporting academic scientists in defining optimal strategies for advancing new, cutting-edge therapeutics from the lab to patients.

Advancing an idea from academic drug discovery research to human clinical trials is time and resource intensive, and, too frequently, unsuccessful. This transition from preclinical to clinical studies is therefore often referred to as the drug development ‘valley of death.’ Many aspects contribute to this valley of death, but key opportunities exist for TRxA to have a positive impact to bridge this gap.

One such opportunity is to leverage C-Path’s proficiencies in translational and regulatory science. In keeping with C-Path’s role as a unique, neutral convener between academia, industry, regulatory agencies and patients, TRxA will provide university-based researchers with advice to define optimal regulatory strategies to advance promising new drug candidates. Additionally, TRxA will have funding available to complete critical studies needed to validate the solidity of the drug product for further development.

By harnessing C-Path’s expertise in accelerating the medical product development process, grant awardees will receive guidance and mentoring to enhance the efficiency and effectiveness of advancing potential therapies from early discovery to clinical trials — starting with a clear end in mind.

Leading TRxA is newly hired Executive Director Maaike Everts, Ph.D., a pharmaceutical scientist with broad experience in drug delivery, discovery and development. Everts previously led a drug discovery program at the University of Alabama at Birmingham, and also has expertise in a CDMO environment for drug formulation and manufacturing. She will apply her firsthand experience to provide pivotal leadership, as TRxA begins accepting applications and issuing awards.

“I am honored and excited to have the opportunity to shepherd TRxA through its growth into a world-class drug accelerator program,” said Everts. “Helping academic researchers move their innovative ideas for new drugs from concept to reality is a personal passion, and a multidisciplinary team approach is critical to bringing new therapies to patients as quickly as possible.”

“C-Path is exceptionally well-suited to offer a novel translational accelerator program that will support a comprehensive strategic approach for academic groups facing the valley of death,” explained Klaus Romero, M.D., M.S., F.C.P., C-Path’s Chief Science Officer and Executive Director of Clinical Pharmacology. “In this accelerator model, C-Path will leverage our core competencies to provide strategic and tactical regulatory science guidance, engage relevant stakeholders when needed and bring diverse expertise to pivotal early-stage study design and implementation.”

“Our Cottrell Foundation has been a supporter of C-Path from its inception, and we have watched it develop into a world-class organization that catalyzes the development process of new medicines working with the FDA, EMA, industry, academia and patients. TRxA represents a unique opportunity to harness the strengths of C-Path to optimize the advancement of new therapies originating at universities and research institutions worldwide, and we’re proud to partner with C-Path on this innovative venture,” said Shaun Kirkpatrick, President, Biotechnologies, at RCT. “TRxA will provide resources and expertise to support academic scientists in developing a robust research plan and validating comprehensive data packages for potential drug candidates, a key to garnering interest from biotechnology and pharmaceutical companies who would further work with the academic scientist to bring promising drug candidates to the health care setting.”

To learn more about TRxA, be informed when the award application period opens and how to apply for an award, visit c-path.org/programs/trxa or contact C-Path at TRxA@c-path.org.


About C-Path
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 hundreds of pharmaceutical and biotech companies. C-Path U.S. is headquartered in Tucson, Arizona, C-Path in Europe is headquartered in Amsterdam, Netherlands and C-Path Ltd. operates from Dublin, Ireland with additional staff in multiple other locations. For more information, visit c-path.org.

About the Frederick Gardner Cottrell Foundation
Research Corporation Technologies, Inc. (RCT) established the Frederick Gardner Cottrell Foundation in 1998 to provide financial support for scientific research and educational programs at qualified nonprofit organizations. RCT named the foundation in honor of the university professor and inventor who championed the transfer of academic innovation to public use. The Cottrell Foundation receives its support from donations made by RCT and is a private, non-operating entity.

Media Contact:

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

Biochemist, Biotechnology Expert to Lead C-Path’s Type 1 Diabetes Consortium

TUCSON, Ariz., June 2, 2022 — Critical Path Institute (C-Path) has named Elnaz Atabakhsh, Ph.D., as Executive Director of its Type 1 Diabetes Consortium (T1DC).

Atabakhsh joined C-Path this May with extensive experience in the biotechnology and biomarker development industry. Her previous work focused on biomarker discovery and screening, cancer genetics and cell therapies for autoimmune disorders, aimed at supporting patients unable to be served by traditional therapeutics. Atabakhsh has extensive experience in and a passion for leading patient-oriented, mission-driven collaborations that include diverse representation from the scientific and medical communities.

C-Path’s type 1 diabetes portfolio consists of several projects targeting relevant stages of the type 1 diabetes disease continuum, before and after diagnosis. T1DC was established in 2017 to advance medical product development for therapies intended to prevent or delay the diagnosis of T1D. The Trial Outcome Markers Initiative in Type 1 Diabetes (TOMI-T1D) is an international partnership with a mission to optimize the design of immune intervention trials in new-onset type 1 diabetes. Co-funded by JDRF and Diabetes UK, TOMI-T1D is a partnership between academic institutions, pharmaceutical industry, and independent nonprofit organizations. Collectively, through these and other efforts, C-Path’s T1D portfolio engages the type 1 diabetes community to identify challenges to the development of new therapeutics and prioritize the development of data-driven solutions that optimize T1D drug development.

“Elnaz embodies the unique combination of scientific knowledge, business development and partnership excellence, as well as the passion and experience required to successfully lead this C-Path consortium,” said C-Path Chief Science Officer Klaus Romero, M.D., M.S., F.C.P. “The challenges faced in T1D drug development, including significant patient heterogeneity across disease stages represent a tangible unmet need. Now led by Elnaz, C-Path’s type 1 diabetes work is poised to reshape and reinvigorate the T1D development landscape.”

After receiving her Ph.D. in Biochemistry from the University of Western Ontario in London, Canada, Elnaz completed postdoctoral training at the Massachusetts General Hospital Cancer Center and Harvard Medical School.

Elnaz believes that her knowledge of various aspects of the biomarker development process, building collaborative teams and interacting with various industry and academic organizations will be beneficial in leading the consortium.

“It is my privilege to join C-Path and to lead our projects aimed at improving the lives of patients living with T1D, a disease that brings significant daily burden to millions around the world,” said Atabakhsh. “The T1D community has shown a remarkable passion and willingness to collaborate at a global scale, and I look forward to joining the community as we take on this important work.”

For more information on C-Path’s T1D efforts, visit: https://c-path.org/programs/t1d/ and https://c-path.org/programs/tomi-t1d/.


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 hundreds of pharmaceutical and biotech companies. C-Path U.S. is headquartered in Tucson, Arizona, is headquartered in Amsterdam, Netherlands and C-Path Ltd. operates from Dublin, Ireland with additional staff in multiple other locations. For more information, visit 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 54.2% funded by the FDA/HHS, totaling $13,239,950, and 45.8% funded by non-government source(s), totaling $11,196,634. 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.

Contact:

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

C-Path Receives Largest Data Transfer to Date from UK’s National Neonatal Research Database

The EPR data transfer is the first from the United Kingdom and includes data from nearly 200 hospitals


TUCSON, Ariz., June 1, 2022 — Critical Path Institute (C-Path) has received fully anonymized electronic patient record (EPR) data from the National Neonatal Research Database (NNRD). This is part of efforts to better understand and find treatments for a chronic lung disease which frequently affects premature infants. This initiative, 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). It is the first time C-Path has received EPR data from the United Kingdom and includes data from a network of nearly 200 hospitals in England.

The NNRD data transfer received ethics approval from the UK Health Research Authority, and none of the data can be traced back to individual patients.

“We have been working with the NNRD team for more than five years and are very excited that extensive discussions have brought us to this significant milestone in data sharing,” said Mark Turner, Ph.D., Professor of Neonatology and Research Delivery, University of Liverpool, UK and INC Co-director. “This EPR data, coupled with previous clinical trial data transfers from other international centers, will shed new light on the causes and treatments of a major cause of death and disability among babies born very prematurely.”

The data transfer is the largest ever for C-Path to date, with nearly 200,000 fully anonymized patient-level data points to be used to understand how the lung condition bronchopulmonary dysplasia (BPD) develops in premature infants. The aim is also to identify stages when doctors can intervene to reduce the risk of BPD, to help find treatments, and to help understand the long-term effects of BPD.

The generation of these real-world evidence solutions will be led in partnership between INC and C-Path’s Quantitative Medicine Program with support from its Data Collaboration Center. C-Path’s INC Executive Director Kanwaljit Singh, M.D., MPH, added, “C-Path has extensive experience in real-world data project collaborations and we’re ecstatic to welcome the NNRD’s data into this initiative. We can’t thank Professor Neena Modi enough for recognizing the unmet need in neonatal drug development and accelerating this important collaboration.”

The EPR data shared by the NNRD will be integrated with other patient-level real world data national registries and networks, as well as clinical trial datasets contributed by INC industry members.

Neena Modi, FMedSci, who established the NNRD and is a Professor of Neonatal Medicine at Imperial College London, said, “The NNRD is a one-stop source of clinical data to improve newborn care, developed with wide stakeholder and parent involvement. We are exceptionally grateful to the families who agree to share data, and to the healthcare staff who carefully record this. By combining the NNRD data with data from other international registries and clinical trials, C-Path has created a novel resource for researchers to study BPD in detail and hopefully reduce the risk of a preterm baby developing the condition. I hope that in time this will improve treatments and the outlook for infants with BPD.”

C-Path’s INC will continue to partner with collaborators and new data contributors to integrate additional patient-level datasets.

Learn more about Imperial College of London’s Neonatal Data Analysis Unit, which supports the management and development of the NNRD, here:
https://web.www.healthdatagateway.org/search?search=NNRD&tab=Datasets.

For more information on collaborating with INC, and how to contribute data, please email Christine Barry at
cbarry@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 54.2% funded by the FDA/HHS, totaling $13,239,950, and 45.8% funded by non-government source(s), totaling $11,196,634. 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.


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 hundreds of pharmaceutical and biotech companies. C-Path U.S. is headquartered in Tucson, Arizona, is headquartered in Amsterdam, Netherlands and C-Path Ltd. operates from Dublin, Ireland with additional staff in multiple other locations. For more information, visit c-path.org.

 

Contact:

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

C-Path Receives Qualification Opinion from EMA on Type 1 Diabetes Biomarker Initiative

 

TUCSON, Ariz., April 5, 2022 Critical Path Institute’s (C-Path) Type 1 Diabetes Consortium (T1DC) today announced that the European Medicines Agency (EMA) has issued a positive qualification opinion for pancreatic islet autoantibodies as enrichment biomarkers for type 1 diabetes (T1D) prevention trials. The purpose of this model-based qualification is to make publicly available tools to assist in the identification and selection of patients with a likelihood of progressing to a T1D clinical diagnosis, in trials of reasonable duration.

This regulatory endorsement will provide sponsors with the confidence to use islet autoantibodies in the optimization of clinical trials evaluating novel therapies focused on the delay and/or prevention of T1D. The models used to generate the underlying evidence for this EMA qualification opinion utilize islet autoantibody status with other patient features to identify a patient’s risk of progressing to a T1D diagnosis. Positivity for two or more of the autoantibodies, together with other patient features, will be used for enrichment of clinical trials focusing on the delay or prevention of the clinical diagnosis of T1D.

In their qualification opinion, EMA reiterated the unmet drug development need that T1DC targeted with this work, stating, “There is clearly an unmet need for biomarkers to aid development in T1DM prevention, a field with a long history of failed trials.” EMA’s qualification opinion statement says, “Positivity to at least 2 of the following islet AAs; IAA, GAD65, IA-2, and ZnT8 is qualified for use as enrichment biomarker, in combination with clinical parameters (sex, baseline age, blood glucose measurements from the 120-minute timepoints of oral glucose tolerance test (OGTT), and hemoglobin A1c (HbA1c) levels) in T1D prevention trials targeting individuals at risk of developing T1D.”

The incidence of T1D is on the rise worldwide, particularly in children. In Europe, incidence rates are between 0.2 and 0.5%, with steep rises in the number of children and young people being diagnosed in certain European countries. The ability to identify individuals at risk of progressing to a clinical diagnosis of T1D is a valuable opportunity to enrich clinical trials testing interventions that can potentially delay and ultimately prevent T1D.

“This qualification from the EMA would not have been possible without the tireless dedication and collaboration of the T1D research community,” said Marjana Marinac, Pharm.D., JDRF Senior Director of Regulatory Affairs and T1DC Co-Director. “JDRF applauds EMA’s decision to support and accelerate more innovation in therapies to delay and prevent T1D.”

“This endorsement from EMA is a result of many years of extensive work and extraordinary collaboration among clinical researchers, patient advocacy groups, nonprofit organizations, and members of the biopharmaceutical industry facilitated by T1DC at C-Path,” said Klaus Romero, M.D., M.S., F.C.P., C-Path Chief Scientific Officer. “This success was only possible through collaborative patient-level data sharing across stakeholders, and we are grateful to our many collaborators for their continued support. We look forward to the impact this tool will make in the development of novel therapies to treat the early stages of T1D with the goal of delaying or preventing the clinical onset of this disease.”

The Qualification Opinion can be found on the EMA website here, or on the T1D Consortium website here.

The consortium is also working on the next regulatory milestone: the endorsement of these islet autoantibodies by the U.S. Food and Drug Administration. C-Path’s T1D Consortium will achieve the regulatory endorsement of the islet autoantibodies currently used in clinical practice to diagnose T1D by employing the resources of all its members and engaging with regulatory agencies at each step of the process with funding and input from The Leona M. and Harry B. Helmsley Charitable Trust, Janssen Research & Development, LLC, JDRF International, Novo Nordisk A/S and Provention Bio.


About C-Path

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 hundreds of pharmaceutical and biotech companies. C-Path U.S. is headquartered in Tucson, Arizona, C-Path in Europe is headquartered in Amsterdam, Netherlands and C-Path Ltd. operates from Dublin, Ireland with additional staff in multiple other locations. For more information, visit c-path.org.

In the U.S., Critical Path Institute is supported by the Food and Drug Administration (FDA) of the Department of Health and Human Services (HHS) and is 54.2% funded by the FDA/HHS, totaling $13,239,950, and 45.8% funded by non-government source(s), totaling $11,196,634. 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.

C-Path’s Type 1 Diabetes Consortium (T1DC) is a public-private partnership initiated in March 2017. Currently membership is composed of the following Industry and Foundation members: The Leona M. and Harry B. Helmsley Charitable Trust; Janssen Research & Development, LLC; JDRF International; Novo Nordisk; and Provention Bio. Other consortium members, participants and advisors include individuals from the following organizations: Benaroya Research Institute at Virginia Mason; Lund University, Sweden; Helmholtz Zentrum München; University of Bristol; University of Colorado Denver; University of Florida; University of Helsinki; University of Leuven; University of Munich; University of Oulu; University of Tampere; University of Turku; Critical Path Institute; FDA and NIH.


The Leona M. and Harry B. Helmsley Charitable Trust aspires to improve lives by supporting exceptional efforts in the U.S. and around the world in health and select place-based initiatives. Since beginning active grantmaking in 2008, Helmsley has committed more than $3 billion for a wide range of charitable purposes. The Helmsley Type 1 Diabetes Program is one of the largest private foundation funders of T1D in the nation focused on understanding the disease, developing better treatments, and improving care and access in the U.S and low- and middle- income countries. For more information on Helmsley and its programs, visit helmsleytrust.org

About the Janssen Pharmaceutical Companies of Johnson & Johnson. 

At Janssen, we’re creating a future where disease is a thing of the past. We’re the Pharmaceutical Companies of Johnson & Johnson, working tirelessly to make that future a reality for patients everywhere by fighting sickness with science, improving access with ingenuity, and healing hopelessness with heart. We focus on areas of medicine where we can make the biggest difference: Cardiovascular & Metabolism, Immunology, Infectious Diseases & Vaccines, Neuroscience, Oncology, and Pulmonary Hypertension.

Learn more at www.janssen.com. Follow us at www.twitter.com/JanssenGlobal. Janssen Research & Development, LLC is one of the Janssen Pharmaceutical Companies of Johnson & Johnson.

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.

Novo Nordisk is a leading global healthcare company, founded in 1923 and headquartered in Denmark. Our purpose is to drive change to defeat diabetes and other serious chronic diseases such as obesity and rare blood and endocrine disorders. We do so by pioneering scientific breakthroughs, expanding access to our medicines, and working to prevent and ultimately cure disease. Novo Nordisk employs about 47,800 people in 80 countries and markets its products in around 170 countries. Novo Nordisk’s B shares are listed on Nasdaq Copenhagen (Novo-B). Its ADRs are listed on the New York Stock Exchange (NVO). For more information, visit novonordisk.com, Facebook, Twitter, LinkedIn, YouTube.

Provention Bio, Inc. (Nasdaq: PRVB) is a biopharmaceutical company focused on advancing the development of investigational therapies that may intercept and prevent debilitating and life-threatening immune-mediated disease. The Biologics License Application (BLA) for teplizumab, its lead investigational drug candidate, for the delay or prevention of clinical type 1 diabetes in at-risk individuals, has been filed by the U.S. Food and Drug Administration (FDA). The Company’s pipeline includes additional clinical-stage product candidates that have demonstrated in pre-clinical or clinical studies proof-of-mechanism and/or proof-of-concept in other autoimmune diseases, including celiac disease and lupus. Visit www.Proventionbio.com for more information and follow us on Twitter: @ProventionBio.

Contact:

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

C-Path European Nonprofit Established in Amsterdam

C-Path in Europe works seamlessly with its U.S. counterpart to advance the development of novel regulatory-endorsed methodologies to accelerate medical product development

AMSTERDAM and TUCSON, Ariz., March 23, 2022 — In support of Critical Path Institute’s (C-Path) mission to catalyze innovation that accelerates the path to a healthier world, C-Path is excited to announce the opening of its EU nonprofit headquarters in the Netherlands, which will focus on the development of existing and future activities in Europe.

C-Path is recognized globally as a leader in regulatory science, with extensive knowledge and experience in seeking and obtaining regulatory endorsement of drug development tools (DDTs) or novel methodologies in cooperation with the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA) and Japan’s Pharmaceuticals and Medical Devices Agency (PMDA). All C-Path regulatory-endorsed tools and methodologies are freely available to the broader scientific community.

The nonprofit’s headquarters in Amsterdam will extend and complement C-Path’s activities to improve public health, as well as share expertise, data, risks and costs to expedite advancements in global regulatory science by facilitating public-private partnerships with members from the biopharmaceutical industry, government regulatory agencies, academic institutions and patient groups in Europe.

“C-Path already has an established and longstanding presence in Europe,” said C-Path Board Member, Tomas Salmonson, Ph.D., M.S., and former Chair of the EMA Committee for Medicinal Products for Human Use (CHMP). “We look forward to facilitating existing and new collaborations to productively support European Regulatory Science research programs and also contribute to the delivery of the EMA Regulatory Science Strategy to 2025.”

Since its inception in 2005, C-Path has worked to forge relationships and collaborations in Europe in tandem with U.S. efforts to facilitate global collaboration. Since then, C-Path has received a significant number of Qualification Opinions and Letters of Support from EMA and has a substantial track record of successful DDT and novel methodology endorsements by FDA, EMA and PMDA. These regulatory milestones have contributed to addressing different unmet needs in medical product development, which have transformed drug development in different areas (among others, tuberculosis, polycystic kidney disease, Alzheimer’s, Parkinson’s, and irritable bowel syndrome).

“Advances in drug development are rapidly playing out on a global stage, and C-Path’s extensive background in accelerating the drug development and regulatory processes will fuel innovation for the development of novel approaches that can be endorsed by regulators in many countries,” said C-Path Chief Science Officer Klaus Romero, M.D., M.S., F.C.P. “Working together with European organizations remains a key C-Path priority. We’re excited to have an office in Amsterdam, which we know will serve to enhance our collaborative efforts and speed the development of treatments for some of the world’s most complex health issues.”

An informative webinar, C-Path in Europe: Moving Global Regulatory Science Forward, is scheduled for April 20, 2022. Register here.

To learn more, visit: https://c-path.org/c-path-in-europe.


 

About C-Path

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 hundreds of pharmaceutical and biotech companies. C-Path U.S. is headquartered in Tucson, Arizona, C-Path in Europe is headquartered in Amsterdam, Netherlands and C-Path Ltd. operates from Dublin, Ireland with additional staff in multiple other locations. For more information, visit c-path.org.

In the U.S., Critical Path Institute is supported by the Food and Drug Administration (FDA) of the Department of Health and Human Services (HHS) and is 54.2% funded by the FDA/HHS, totaling $13,239,950, and 45.8% funded by non-government source(s), totaling $11,196,634. 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.

 

Contact:

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

C-Path and the European Joint Programme on Rare Diseases to Expand Global Impact and Partnership

PARIS, France and TUCSON, Ariz., U.S., February 1, 2022 — Critical Path Institute (C-Path), an independent global nonprofit organization, and the European Joint Programme on Rare Diseases (EJP RD), an initiative that has received funding from the European Union’s Horizon 2020 research and innovation programme, have announced a collaboration to advance technologies and methodologies that are fit for regulatory purposes to further global rare disease research and drug development.

C-Path has a proven track record in establishing public-private-partnerships that positively impact the process of medical product development in rare diseases. EJP RD promotes integration, efficacy, production and social impact of research in rare diseases. In this context, EJP RD and C-Path have successfully established a collaboration using a shared data model to demonstrate that key datasets from both efforts can be queried simultaneously.

“Collaboration is a foundational principle of success to address unmet needs in medical product development in rare diseases,” said C-Path Chief Science Officer Klaus Romero, M.D., M.S., F.C.P. “As novel approaches are vital to address these unmet needs, our partnership with EJP RD will expand our global capabilities in collaborating for methodologies and technologies to accelerate research with tangible impact for individuals and their families living with rare disease.”

Building on the experience from the past year , both organizations will support the creation of an interoperable global data ecosystem for rare disease to expedite effective development of new therapies. The partnership will benefit patients, regulators, advocacy stakeholders, researchers and industry.

“The cooperation between EJP RD and C-Path not only allows us to bring our unique expertise together, but also sets the foundation for future joint actions in the data, regulatory and clinical methodology space strengthening capacities of the research and development community in EU and on a global scale,” said EJP RD Coordinator Daria Julkowska, Ph.D., Assistant Director, Thematic Institute of Genetics, Genomics & Bioinformatics, INSERM.


 

About C-Path

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.

 

About EJP RD
The European Joint Programme on Rare Diseases (EJP RD) is a programme aiming to create an effective rare diseases research ecosystem for progress, innovation and for the benefit of everyone with a rare disease. EJP RD supports rare diseases stakeholders by funding research, bringing together data resources & tools, providing dedicated training courses, and translating high quality research into effective treatments.

For more information, please contact info@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 54.2% funded by the FDA/HHS, totaling $13,239,950, and 45.8% funded by non-government source(s), totaling $11,196,634. 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.

The European Joint Programme on Rare Diseases is an initiative that has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement N°825575.

 

Contact:

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

C-Path Appoints Health Outcomes Expert as Executive Director of the Rare Disease COA Consortium

Dr. Lindsey Murray will lead C-Path’s New Rare Disease COA Consortium

TUCSON, Ariz., January 13, 2022Critical Path Institute (C-Path) today announced it has named Lindsey Murray, Ph.D., MPH, as Executive Director of its Rare Disease Clinical Outcome Assessment (COA) Consortium, effective immediately. Murray joined C-Path in 2019 as Associate Director for rare diseases within C-Path’s Patient-Reported Outcome Consortium.

“Lindsey is a tremendous asset to C-Path and I’m grateful she joined the team to help establish the Rare Disease COA Consortium and, ultimately, become its founding Executive Director,” said Stephen Joel Coons, Ph.D., Senior Vice President for C-Path’s Clinical Outcome Assessment Program. “Her efforts, underwritten by grant funding from FDA, enabled us to launch a viable and sustainable multi-stakeholder consortium on January 1st aimed at facilitating the evaluation of new drugs and biologics for the treatment or cure of rare diseases. She has my full support in this exceptionally worthwhile endeavor.”

As executive director, Murray will provide scientific and operational leadership for this pre-competitive public-private partnership among C-Path, FDA, the National Organization for Rare Disorders (NORD), other governmental and non-governmental organizations, and key partners in the biopharma, clinical research and patient communities that are seeking and/or developing treatments for rare diseases.

Murray’s experience extends for over 15 years in health outcomes research, specializing in quantifying the patient’s perspective of health, illness and treatment through COA development, psychometric testing and the design and analysis of clinical trials involving COAs. Prior to joining C-Path, Murray served as a research scientist and Director of the EXacerbations of Chronic Pulmonary Disease Tool (EXACT®) PROgram at Evidera.

“I’m honored to take on leadership of the Rare Disease COA Consortium,” said Murray. “ We’ve brought together an amazing group of collaborators, including regulatory, clinical, outcomes research and patient advocacy experts. I’m looking forward to continuing to build out the Rare Disease COA Resource that will help guide patient-centered COA selection in rare disease medical product development, as well as collectively tackling the pressing methodological challenges faced in rare disease research programs.”

Murray holds a Ph.D. in epidemiology from George Washington University, where she also received her MPH in epidemiology with a certificate in health promotion. She has a BA from the University of Virginia in Charlottesville, Virginia, where she double majored in anthropology and biology with a minor in German literature and language studies.

For more information on C-Path’s Rare Disease COA Consortium, visit: c-path.org/programs/rd-coac.

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 54.2% funded by the FDA/HHS, totaling $13,239,950, and 45.8% funded by non-government source(s), totaling $11,196,634. 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.

Specific funding for the establishment of the Rare Disease COA Consortium was provided by FDA grant U01FD006882.


About C-Path

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 Launches Rare Disease Clinical Outcome Assessment Consortium

The Rare Disease COA Consortium aims to advance patient-focused assessment of clinical benefit in treatment trials for rare diseases

TUCSON, Ariz., January 13, 2022Critical Path Institute (C-Path) today announced the launch of the Rare Disease Clinical Outcome Assessment (COA) Consortium, a public-private partnership focused on optimizing COA selection during medical product development for rare diseases. The Rare Disease COA Consortium is a collaborative effort among C-Path, the U.S. Food and Drug Administration (FDA), the National Organization for Rare Disorders (NORD), other government agencies, as well as key partners in the biopharma, clinical research and patient communities that are seeking treatments for rare diseases.

More than 350 million people worldwide are impacted by one of more than 7,000 rare diseases. These conditions result in substantial burden on patients, families, communities and global health care systems. Nearly 50% of rare diseases impact children, of whom 30% will die by age 5. It is currently estimated that less than 10% of rare diseases have approved treatments. COAs to measure clinical benefit of treatment do not exist or remain unidentified for the majority of rare diseases. With no approved therapies for most rare diseases, and considerable uncertainty around appropriate treatment trial endpoints, a significant unmet public health need remains unfulfilled. The Rare Disease COA Consortium seeks to address this need with the creation of the Rare Disease COA Resource that will identify and describe potentially fit-for-purpose publicly available COAs for use in treatment development programs across multiple rare diseases.

“With the increased emphasis on patient centeredness in clinical trials, selection of COAs that assess outcomes that are meaningful is critical,” said Lindsey Murray, Ph.D., Executive Director of the Rare Disease COA Consortium. “The vision of the Rare Disease COA Consortium is to catalyze medical product development by measuring what truly matters to people with rare diseases and their families.”

The underlying premise for the Rare Disease COA Resource is that, for rare diseases that share common characteristics, existing COAs may be used or modified for use as endpoint measures for treatment trials. By identifying existing measures that are potentially fit-for-purpose, the considerable time and cost associated with the development of new COAs may be reduced. With the support of funding provided by FDA, work toward developing the Rare Disease COA Resource has been underway since 2019. The first iteration of the resource focuses on the assessment of daily function in pediatric, non-oncologic rare disease populations.

Additionally, work to identify and prioritize other challenges related to the assessment of clinical treatment benefit in rare disease therapeutic trials has been initiated, and will continue to be a focus of the new consortium. Initial efforts included identifying methods for assessing clinical treatment benefit in conditions with heterogeneous manifestations and impact profiles, and a webinar on strategies to mitigate the impact of COVID-19 in pediatric rare disease clinical trials.

“We are exceptionally grateful to FDA for the vision and funding that enabled the Rare Disease COA Consortium to be established,” said Stephen Joel Coons, Ph.D., Senior Vice President for C-Path’s Clinical Outcome Assessment Program. “It will now be supported by membership fees from biopharmaceutical firms and other stakeholders advocating for or developing new drugs or biologics for the treatment or cure of rare diseases. We are very pleased this new consortium will join other C-Path consortia and programs aimed at enhancing the health and well-being of individuals with rare disorders.”

To learn more, visit c-path.org/programs/rd-coac.

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 54.2% funded by the FDA/HHS, totaling $13,239,950, and 45.8% funded by non-government source(s), totaling $11,196,634. 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.

Specific funding for the establishment of the Rare Disease COA Consortium was provided by FDA grant U01FD006882.


About C-Path

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’s ePRO Consortium Announces Rebranding, Changes Name to eCOA Consortium

 

TUCSON, Ariz., January 11, 2022 — Critical Path Institute (C-Path) is pleased to announce the Electronic Patient-Reported Outcome (ePRO) Consortium is now the Electronic Clinical Outcome Assessment (eCOA) Consortium. This rebranding more accurately reflects the expanded portfolio range of work performed by the consortium, the actual capabilities of member firms and the scope and significance of the consortium’s role. The new name was chosen to more authentically characterize the mission and aims of the consortium, which have evolved significantly since its inception in 2011.

“This name change more effectively communicates the breadth of the activities of the consortium and its members and enhances the consortium’s visibility across not just the eCOA ecosystem, but the clinical research community more broadly,” said Senior Vice President of C-Path’s COA Program, Stephen Joel Coons, Ph.D. “Rebranding as the eCOA Consortium signals an important step in the evolution of the consortium and will further facilitate multi-stakeholder discussions to identify and address the root causes of issues with eCOA implementation in clinical trials. The eCOA Consortium will continue its close collaboration with the Patient-Reported Outcome Consortium on the joint eCOA–Getting Better Together Initiative.”

Coordinated by C-Path, members of the eCOA Consortium are firms that provide electronic data collection technologies and services for capturing COA data in clinical trials. The eCOA Consortium’s mission 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.

“This is a very necessary change that emphasizes both the substance and scope of the consortium and its output,” said Executive Director of the eCOA Consortium, Scottie Kern. “This consortium boasts not just unrivalled knowledge and experience of eCOA implementation, it has at its heart a drive to progress the science of eCOA and its criticality to modern medical product development.”

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.

For more information on C-Path’s eCOA Consortium, visit: https://c-path.org/programs/ecoac

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 54.2% funded by the FDA/HHS, totaling $13,239,950, and 45.8% funded by non-government source(s), totaling $11,196,634. 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 C-Path

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