C-Path Announces Health Care Leader Appointment to Board of Directors

TUCSON, Ariz., July 8, 2021 — Critical Path Institute (C-Path) today announced the appointment of health care industry leader and active civic involvement pioneer Mara G. Aspinall, MBA, to its Board of Directors.

Aspinall is Managing Director and Co-Founder of BlueStone Venture Partners, LLC, a venture fund investing in life sciences technology companies in the U.S. Southwest. She also serves as CEO of Health Catalysts Group, a consulting firm dedicated to the growth of health information technology and diagnostics firms and publisher of the popular Health Catalysts Diagnostics Year in Review. During the pandemic, Aspinall served as an advisor to The Rockefeller Foundation on COVID testing.

“Mara is a long-time supporter of C-Path’s work and we’re thrilled to have her join the Board at this exciting and important new phase of evolution for C-Path,” said Board Chairman Wainwright Fishburn. “The addition of Mara complements our Board’s skills and experiences, and we are confident she will provide valuable industry perspective. We look forward to her engagement in guiding our strategic initiatives and being the catalyst for innovation that accelerates the path to healthier world.”

Aspinall has extensive experience in the risk-based capital industry. In addition to BlueStone, she has served as advisor on life sciences transactions for private equity firms. At Blue Cross Blue Shield Arizona, Aspinall chaired the Board for its strategic corporate venture fund, Trinnovate Ventures.

As President and CEO of Tucson-based Ventana Medical Systems, now Roche Tissue Diagnostics, Aspinall led her world-class team to new financial success, more than two dozen major instrument and assay launches as well as global leadership in companion diagnostics. Previously, she served as President of Genzyme Genetics and Genzyme Pharmaceuticals. Genzyme Genetics grew into one of the country’s leading diagnostic testing companies and was sold to LabCorp for $1 billion.

“I believe C-Path’s leading-edge approach to medical product development creates profound contributions that impact patients’ lives,” said Aspinall. “Their development of actionable solutions through an innovative, collaborative approach in the sharing of data and expertise is a major driver of change. I look forward to working with the Board to create value, drive consensus and generate the confidence necessary to support the adoption of solutions by sponsors and regulators.”

A leader in educating payers and policymakers on personalized medicine, Aspinall served on the Health and Human Services Secretary’s Advisory Council on Genetics, Health & Society (SACGHS) in the Bush and Obama administrations. With a passion for education about the importance of diagnostics in health care, Aspinall co-founded the School of Biomedical Diagnostics at Arizona State University, the first and only program dedicated to diagnostics as an independent discipline.

Aspinall was named Arizona Bioscience Leader of the Year by the Arizona Biotechnology Association in 2016, one of “100 Most Inspiring People in Life Sciences” by PharmaVOICE magazine and one of Women Inc’s Most Influential Corporate Board Directors. She started her business career at the strategic consulting firm Bain & Company. Aspinall holds an MBA from Harvard Business School and a BA from Tufts University.

Aspinall’s Board appointment begins immediately. She is also a Board of Directors member on Abcam, Allscripts, Castle Biosciences, OraSure, AZBio and Blue Cross Blue Shield Arizona.

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 56.5% funded by FDA/HHS, totaling $16,749,891, and 43.5% funded by non-government source(s), totaling $12,895,366. 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

Clinical Technology Expert and Experienced Neuroscientist Join C-Path in Executive Director Roles

Scottie Kern and Dr. Terina N. Martínez Will Both Serve in Dual Positions

TUCSON, Ariz., June 24, 2021 — Critical Path Institute (C-Path) today announced it has named Scottie Kern, as both Executive Director of the Electronic Patient-Reported Outcome (ePRO) Consortium and Associate Director of the Patient-Reported Outcome (PRO) Consortium and Terina N. Martínez, Ph.D., as Executive Director of both the Duchenne Regulatory Science Consortium (D-RSC) and Critical Path to Therapeutics for the Ataxias (CPTA).

Kern is an expert in patient-based clinical technologies, with more than 25 years of pharmaceutical sector experience. Dr. Martínez is a neuroscientist and an expert in science communication, research program management and leadership, as well as biomarker and drug development in neurodegenerative diseases.

“We are thrilled to have Scottie join our Clinical Outcome Assessment Program to help us advance C-Path’s mission and vision,” said Senior Vice President of the Clinical Outcome Assessment (COA) Program Stephen Joel Coons, Ph.D. “His unique experience and commitment to leveraging existing and emerging data collection technologies to enhance patient-focused drug development make him the perfect fit to lead the ePRO Consortium and substantively contribute to the PRO Consortium’s COA qualification efforts.”

As Executive Director of C-Path’s ePRO Consortium, his primary role, Kern will provide operational and scientific leadership that guides development and implementation of strategic priorities aimed at achieving the consortium’s mission. As Associate Director of the PRO Consortium, he will provide technical, operational, and scientific input to all working groups and subcommittees using or considering technology-based COA data collection.

Initially operating in clinical data management roles at several contract research organizations and pharma companies, Kern has been working with ePRO technology for nearly two decades. Enthused by the potential of this technology and its direct interaction with patients, Kern took a role as the functional lead for ePRO at Wyeth’s Vaccine Research Unit in 2004 and subsequently established himself as an industry subject matter expert and thought leader on ePRO and other electronic clinical outcome assessments (eCOAs), serving as the global ePRO lead at Wyeth and the Head of Patient Technologies/Global Head of ePRO at Pfizer until 2011.

Seeing an unmet need for experience-based, objective eCOA consultancy, Kern launched Sacaja Consulting Limited in 2013, which supported several top pharmaceutical companies with eCOA strategy and implementation, as well as advising several small to mid-sized companies, contract research organizations (CROs), and eCOA/mHealth technology vendors on a range of eCOA/mHealth-related projects.

“Throughout my career, I’ve derived the greatest satisfaction from any work I’ve contributed to that required collaboration with my industry contemporaries to generate substantial and meaningful change,” said Kern. “I am a passionate believer in enhancing the clinical trial participant experience with mobile technology, and I’ve been a long-term consumer of the outputs from the ePRO Consortium as well as the PRO Consortium. The chance to help drive the ePRO Consortium’s mission forward and to work with accomplished people I have known and respected for many years was simply too good an opportunity to miss.”

Prior to joining C-Path, Martínez was a Senior Associate Director, Research Programs at The Michael J. Fox Foundation for Parkinson’s Research, in New York, where she led the Foundation’s programs for preclinical tools and animal models, emerging targets and inflammation. Thereafter, Martínez was a field application and collaboration scientist with Taconic Biosciences based out of Cambridge, MA, where she provided expert technical and scientific consultation across all research sectors for preclinical model selection, application, translational and IND-enabling study design, encompassing diverse disease and therapeutic areas.

“Terina is a highly motivated scientist who has worked and collaborated with academia, industry, nonprofit foundations and patient advocacy groups, to name a few,” said Vice President of C-Path’s Neuroscience Program, Sudhir Sivakumaran, Ph.D. “Her expertise, experience and commitment to addressing patient needs is perfectly aligned with our core competencies and we look forward to our Duchenne and ataxia consortia thriving under her leadership.”

As Executive Director of D-RSC and CPTA, Martínez will lead and define the future strategic directions and objectives of the D-RSC and CPTA consortia. She will convene diverse stakeholders and worldwide experts across the Duchenne muscular dystrophy and ataxia fields, to accelerate therapeutic innovation through data acquisition, regulatory science, generation of patient-level data driven drug development tools and solutions, and projects to develop novel biomarkers and refine outcome measures.

Martínez received her undergraduate degree in biology from the University of Dallas and earned a Ph.D. in integrative biology from the University of Texas Southwestern Medical Center at Dallas, where she studied cellular and molecular neuroscience. She completed her postdoctoral training at the University of Pittsburgh.

“I am passionate about improving the lives of people living with devastating neurodegenerative diseases and am thrilled to have joined C-Path to lead the D-RSC and CPTA teams in executing C-Path’s mission to develop new approaches to advance medical innovation and regulatory science,” said Martínez.

For more information on C-Path’s ePRO Consortium, visit: https://c-path.org/programs/eproc
For more information on C-Path’s PRO Consortium, visit: https://c-path.org/programs/proc
For more information on C-Path’s D-RSC, visit: https://c-path.org/programs/d-rsc
For more information on C-Path’s CPTA, visit: https://c-path.org/programs/cpta

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 Virtual Workshop to Feature Latest Advances in Clinical Trials for T1D

More than 300 Attendees and 20 Speakers Are Expected to Participate in the International Workshop, June 15-16

TUCSON, Ariz., May 25, 2021 — Critical Path Institute (C-Path) today announced its schedule for the Design of Clinical Trials in New-Onset Type 1 Diabetes: Regulatory Considerations for Drug Development Workshop, to be held virtually June 15-16. Together with the U.S. Food and Drug Administration (FDA), European Medicines Agency (EMA), and in collaboration with Benaroya Research Institute, INNODIA and JDRF, this 2-day public workshop will focus on the implementation of endpoints and outcome measures for clinical trials in new onset type 1 diabetes (T1D). More than 300 researchers, pharmaceutical representatives, academia members, investigators, T1D patients and regulatory experts from throughout the United States and Europe are expected to gather virtually to hear 15 presentations about T1D research and medical product development.

“An important element of C-Path’s work is to bring together leading researchers from academia and industry, alongside clinicians and patient advocates, to gather consensus on the latest scientific developments that can improve the lives of members of the T1D community,” said C-Path’s T1D Consortium Executive Director Inish O’Doherty, Ph.D. “By providing a forum for the community and regulators to publicly engage, our workshop seeks to bridge the gap between scientific understanding of outcome measures, such as C-peptide, and their implementation in registration studies for new onset T1D.”

As one main objective of the workshop, attendees can expect discussion on existing evidence regarding the role of C-peptide in clinical trials intended to support regulatory decision making, regulatory considerations from the FDA and EMA, and perspectives from the T1D drug development community.

“This meeting comes at a critical time in the development of disease-modifying therapies to address the significant unmet needs for people with type 1 diabetes,” says JDRF Vice President of Research Sanjoy Dutta, Ph.D. “We are excited to participate in this important discussion and thank the FDA, EMA, meeting organizers and presenters for bringing the community together to accelerate pathways to cures for T1D.”

The conference will include scheduled live-broadcast sessions, live panel discussions and Q&A.

The preliminary agenda is as follows:

  • Welcome and Introductory Remarks
  • Session I: Regulatory Framework for Clinical Investigations in New/Recent Onset T1D
  • Session II: Scientific Framework: The rationale for C-peptide preservation and use as a clinical trial endpoint
  • Session III: Establishing/confirming clinical benefit
  • Session IV: Overall Issues of Study Design: Considerations and panel discussion/open comment

Registration is free and open to the public. For more information and to receive additional updates leading up the event, visit the workshop event page.

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.

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), Facebook (@myjdrf), and Instagram (@jdrfhq).

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

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

ERA4TB Update

NEWSLETTER: The ERA4TB (European Regimen Accelerator for Tuberculosis) project, a public-private initiative devoted to accelerating the development of new treatment regimens for tuberculosis, issued its first newsletter this month which includes updates on the project thus far. Read the full communication and subscribe, here: https://era4tb.org/newsletter.

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