Dear Friend of C-Path:
As we observe National Nonprofit Day and recognize the invaluable effort nonprofits put towards making an impact in our communities and across the globe, this seemed like a perfect opportunity to reshare some of the year’s accomplishments, championed by the talented and unshakeable team at C-Path, our consortia members and collaborators.
We are rounding the corner on another year of unpredictability and uncertainty concerning the COVID-19 pandemic. Through it all, C-Path has managed to foster the development of cutting-edge approaches to the advancement of medical innovations and regulatory science. We pride ourselves in forging ahead in new and exciting directions while maintaining steady growth and interest in our endeavors — as evidenced by the overwhelming popularity of the virtual events C-Path has hosted so far this year. In fact, since the beginning of 2021 alone, nearly 2,000 people have attended a C-Path-hosted event, most of which can now be viewed in their entirety via C-Path's YouTube channel.
In terms of integrating relevant patient-level data sources, C-Path has continued its significant progress throughout 2021, with 22 new studies encompassing 18,776 subjects:
In April, C-Path’s Critical Path for Alzheimer’s Disease (CPAD) Consortium announced the significant expansion of 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 made it possible for the CPAD team to integrate 42 high-quality clinical trials and observational studies in AD, containing more than 21,000 patient-level records. 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.
May brought the announcement of an exciting joint venture between C-Path and Tufts Medical Center to integrate the first patient-level clinical trial data to generate actionable real-world evidence for neonatal drug development, from real-world neonate data. 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.
C-Path also announced the opening of 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. 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.
As evidenced above, through the unwavering support from its consortia members, collaborators and supporters, C-Path continues to develop innovative mechanisms and forge pioneering and beneficial pathways to develop safe and efficacious therapies.
In other news thus far for the year:
This February, C-Path was proud to launch the Critical Path to Therapeutics for the Ataxias (CPTA) Consortium, a public-private partnership focused on optimizing clinical trials for inherited ataxias. CPTA is a collaborative effort between C-Path, Ataxia Global Initiative, the National Ataxia Foundation, Ataxia UK and key partners in the industry and academic research communities, including Biohaven Pharmaceuticals, Ionis Pharmaceuticals, Roche Pharmaceuticals, Servier Group, Triplet Therapeutics, uniQure and Vico Therapeutics.
CPTA creates a neutral, precompetitive space where stakeholders from across different fields and backgrounds including regulators can come together and share expertise, insights and data, which will be leveraged for the generation of actionable solutions for drug development for the ataxias.
In March, C-Path’s Patient-Reported Outcome (PRO) Consortium received FDA qualification for the Diary for Irritable Bowel Syndrome Symptoms-Constipation (DIBSS-C). The DIBSS-C was developed by the PRO Consortium’s Irritable Bowel Syndrome (IBS) Working Group to support symptom-based efficacy endpoints in clinical trials for products intended to treat constipation-predominant IBS (IBS-C) in adults. Qualification of the DIBSS-C represents a major milestone for the IBS Working Group and is the PRO Consortium’s fourth clinical outcome assessment (COA) to be qualified through the COA Qualification Program within FDA’s Center for Drug Evaluation and Research.
Cures Within Reach, a global nonprofit leading the use of repurposing research to help improve patients’ lives, in March, awarded CURE ID at the FDA and NCATS/NIH as the recipient of the 2021 Golan Christie Taglia Patient Impact Philanthropy Award. C-Path’s Cure Drug Repurposing Collaboratory (CDRC), works in partnership with the FDA-NCATS CURE ID platform, and is a dedicated initiative designed to capture real-world clinical outcome data to advance drug repurposing and inform future clinical trials for diseases of high unmet medical need.
We were also extremely excited to have welcomed some new faces to the C-Path family over the last year:
First, the appointment of two new members to our Board of Directors:
On May 19, 2021, we announced the appointment of Dr. Robert M. Califf to the Board. 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. Prior to joining C-Path’s Board of Directors, Dr. Califf served as head of Clinical Policy and Strategy for Google parent company Alphabet’s Verily Life Sciences and Google Health divisions.
In July, the Board of Directors announced the appointment of health care industry leader and active civic involvement pioneer Mara G. Aspinall, MBA. 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 has served as an advisor to The Rockefeller Foundation on COVID testing.
We were also excited to announce a host of new additions to the
In December, C-Path welcomed Alexandre Bétourné, Ph.D., Pharm.D., as its new Scientific Director for the RDCA-DAP initiative. Dr. Bétourné will work with the RDCA-DAP team to expand its reach into new diseases areas accessing new data and enhancing C-Path’s relationships within the rare disease community. Before joining C-Path, he led a team of senior U.S. scientists, CMC and regulatory consultants at a small company developing therapies for amyotrophic lateral sclerosis (ALS).
In January, C-Path welcomed Martijn Muller, Ph.D., as Senior Scientific Director for the Critical Path for Parkinson's Consortium. Prior to joining C-Path, Martijn was Research Associate Professor of Radiology and Co-Director of the Clinical and Neuroimaging Resource Cores of the Udall Center of Excellence for Parkinson’s disease Research at the University of Michigan.
In April, Ramona Belfiore Oshan, Ph.D., was announced as the new Associate Director for D-RSC. The consortium is currently engaged in several modeling projects of biomarkers and outcome measures, all at different stages in development and regulatory review, as well as development of a platform trial protocol for Duchenne and considering a new patient-reported outcome instrument. Dr. Oshan will be working in a hybrid scientific director and project manager role to keep the consortium momentum moving forward.
Joining the C-Path team in May as both the Executive Director of the Electronic Patient-Reported Outcome (ePRO) Consortium and Associate Director of the Patient-Reported Outcome (PRO) Consortium was Scottie Kern. Kern is an expert in patient-based clinical technologies, with more than 25 years of pharmaceutical sector experience. For ePRO, his primary role, he will provide operational and scientific leadership that guides development and implementation of strategic priorities aimed at achieving the consortium’s mission.
Also in May, Terina N. Martínez, Ph.D., joined the team as Executive Director of both D-RSC and CPTA. 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. She will lead and define the future strategic directions and objectives of the D-RSC and CPTA consortia.
Additionally, C-Path's Critical Path to Sickle Cell Disease (CP-SCD) Consortium named its first industry co-director appointing Dr. David Readett of Pfizer to the role in April. Dr. Readett is a pediatrician/pediatric hematologist/oncologist who, prior to transitioning to clinical research in the pharmaceutical industry, spent 18 years in clinical practice, with a focus on clinical care and research for pediatric patients with oncologic or hematologic disease, including sickle cell disease.
And lastly, CPTA announced the appointment of Irina Antonijevic, M.D., Ph.D., in April, as the consortium’s industry co-director. Dr. Antonijevic has worked in drug development for over 18 years, with a focus on translational medicine and early development in neuroscience, but also ophthalmology and immunology. She has extensive experience in the development of small molecules, biologics as well as nucleic acid therapeutics, in a number of global pharma and biotech organizations in the U.S. and Europe.
We are incredibly grateful to have added these talented experts and are thankful they have joined us to build on the brilliant future ahead.
It is incredibly humbling to be working with the great minds and talents that make possible the critically important work at the heart of C-Path’s mission. In the coming months, we will continue our march unabated, as we enter into a heavy fall meeting season, starting with the RDCA-DAP 2021 Workshop and the Neuroscience Program Workshop in September, and the INC Annual Workshop in October.
I look forward to sharing more exciting news in the months to come. In the meantime, please do not hesitate to reach out with any questions, suggestions or well wishes. As we have seen — in bad times and in good — the way forward is together.
Sincerely, humbly, and with immense gratitude,
Kristen Swingle, M.S.
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.
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