C-Path Promotes Nick King and Emily Turner to Executive Directors

TUCSON, Ariz., November 10, 2021 — Critical Path Institute (C-Path) today announced it has promoted Nicholas King, M.S., as Executive Director of its Predictive Safety Testing Consortium (PSTC) and Emily Turner, Ph.D., as Executive Director of the Huntington’s Disease Regulatory Science Consortium (HD-RSC).

“Nick and Emily have been incredible assets to our organization and will be great additions to our leadership team,” said C-Path Interim President and COO Kristen Swingle, M.S. “Their unique experience and commitment to PSTC and HD-RSC, respectively, will certainly elevate these initiatives and enhance C-Path’s mission to accelerate drug development.”

King has made significant operational and scientific contributions to PSTC over his 11 years at C-Path, and has held several roles during his tenure, most recently in a dual role as PSTC Scientific Program Manager and Associate Director of the Translational and Safety Sciences Program. He is an expert in project management, with more than a decade of experience in managing several working group efforts for PSTC, including those for hepatotoxicity, pancreatic injury, skeletal muscle injury and vascular injury. As scientific program manager and associate director, King provided input on regulatory science and analytical considerations for drug development tools, and supported operations and logistics across the program.

Since joining C-Path, King has been instrumental in leading efforts that have resulted in more than 10 regulatory milestone decisions for safety biomarkers and published seminal peer-reviewed scientific papers on the topic. He has played a key role as session co-chair at the PSTC Annual Workshop, the Society of Toxicology Annual Meeting and the PSTC Japan Safety Biomarker Conference. King received his undergraduate degree in biochemistry with a focus in molecular biophysics from Washington State University. He graduated from the University of Arizona College of Medicine’s Cell Biology program with a master’s degree in 2009. King’s graduate studies comprised research on cardiac and skeletal muscle physiology and mechanics. Prior to graduate school, he served as a Peace Corps volunteer in the West African country Cape Verde.

As Executive Director of PSTC, King will provide operational and scientific leadership that guides the development and implementation of strategic priorities aimed at achieving the consortium’s mission.

“Working at C-Path has been a privilege,” said King. We bring together a unique group of scientists to address unmet needs in drug development and patient safety with a collaborative approach and I look forward to advancing novel tools for safety assessment with C-Path consortia members and participants.”

Since joining C-Path in July 2019 as the Associate Director of HD-RSC, Turner has been instrumental in various successes within the consortium, including data acquisition, modeling and analysis, regulatory submissions and more recently, the optimization of the consortium to an industry-funded model to mirror the organization of other consortia at C-Path.

HD-RSC brings together diverse stakeholders in the Huntington’s disease community to define and address regulatory needs to accelerate HD therapeutic development. As Executive Director, Turner will focus the consortium’s efforts on developing and maintaining strong relationships with internal and external stakeholders to ensure the consortium’s continued success and progress in advancing clinical outcome assessments, biomarkers and other quantitative tools for HD.

“I am honored to be a part of the C-Path team and lead HD-RSC,” said Turner. “This is an important effort to the HD community because families affected by this disease are in desperate need of therapies to delay or halt the onset of symptoms, and I look forward to leading the consortium and facilitating meaningful contributions for HD patients.”

Turner’s specific areas of technical expertise include neuroanatomy, neuropathology and animal models of neurodegenerative diseases. While in academia, she focused her research on drug discovery for Alzheimer’s disease and other related neurodegenerative conditions and has numerous publications in the neuroscience area. Turner received her undergraduate degree in psychology and art history from Northwestern University and earned a Ph.D. in neuroscience from Vanderbilt University, where she studied systems neuroscience in a non-human primate laboratory. She completed her postdoctoral training at the ASU-Banner Neurodegenerative Disease Research Center. She has served as an ad hoc reviewer in peer-reviewed scientific journals and enjoys serving as a professional mentor for women in STEM careers.

For more information on PSTC, visit: https://c-path.org/pstc
For more information on HD-RSC, visit: https://c-path.org/hdrsc

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

C-Path and Cure Mito Announce Data Sharing Agreement to Support Treatment Development For Leigh Syndrome and Mitochondrial Disease

TUCSON, Ariz., November 9, 2021 — Critical Path Institute (C-Path) and Cure Mito Foundation (Cure Mito) today announced a joint collaboration to significantly promote data sharing and accelerate Leigh syndrome and other rare mitochondrial disease data incorporation into C-Path’s Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP®).

Leigh syndrome, also known as Leigh disease, is a Mitochondrial disorder that affects about 1 out of 77,000 live births, with symptoms often appearing within the first year of life. It is a debilitating neurological disorder that can affect the development of mobility, posture and mental capacities in children, with these capabilities occasionally being lost after a period of seemingly normal growth. Though unusual, symptoms may not show until the early adult years, while others may show gradual signs throughout infancy.

“Progress towards the establishment of approved therapies for people with Leigh syndrome and other rare mitochondrial diseases is reliant on the participation of organizations like the Cure Mito Foundation,” said RDCA-DAP Scientific Director Alexandre Betourne, Ph.D., Pharm.D. “This is a laudable step in that direction, and we are thrilled to be partnering with Cure Mito to promote data sharing, standardization and develop tools that will accelerate the development of treatments for Leigh syndrome and other mitochondrial disorders.”

C-Path’s RDCA-DAP provides a centralized and standardized infrastructure to support and accelerate rare disease characterization targeted for clinical development. Additionally, the platform includes a framework that supports the rigorous conduct of natural history studies, with attention to established data quality standards, in order to be most useful to clinical trial design and regulatory review. It includes a robust, integrated database and analytics hub that allows for the aggregation of rare disease data from various sources and the efficient and effective interrogation of that data.

Cure Mito was founded in 2018 as the Cure SURF1 Foundation by a group of families determined to fight for their children’s lives with the primary focus of advancing research towards a cure for Leigh syndrome and eventually for mitochondrial disease as a whole.

“Patients enroll in registries and research studies hoping that their participation will matter,” said Sophia Zilber, Cure Mito board member. “Cure Mito Foundation recognizes this and is committed to advancing the way data is collected, accessed and used, and empowering the patient community to be informed and active contributors of their data to advance research, treatments and eventually cures. We are proud to partner with C-Path in these important and much needed efforts.”

The collaboration between C-Path and Cure Mito establishes the path for future integration of longitudinal data collected by the foundation, as available, along with other clinical outcome assessment data, to be used for the development of tools and solutions that ultimately lead to the development of new treatments for rare disease patients.

Groups interested in contributing data to RDCA-DAP, may visit, c-path.org/rdca-dap or email rdcadap@c-path.org. The platform is now OPEN and accepting applications for use; visit https://portal.rdca.c-path.org to apply and learn more. To access the 2021 RDCA-DAP Workshop held on Tuesday, Sept. 14, click here; registration is not required.

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.

 

About Cure Mito Foundation

Cure Mito Foundation is a parent-led 501(c)(3) nonprofit organization that was founded in 2018 as a Cure SURF1 Foundation by a group of families determined to fight for their children’s lives. In 2021, after successfully blazing the trail for SURF1 gene therapy at the University of Texas Southwestern Medical Center, the mission of the organization expanded and the name was updated.  Cure Mito’s primary focus is advancing research towards a cure for Leigh syndrome and eventually for Mitochondrial disease as a whole.  Cure Mito is a volunteer organization with 100% of funds used towards research. Patients are at the heart of everything Cure Mito does. For more information please visit: curemito.org.

Media Contact:

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

Academic Innovation Leader and Experienced Scientist to Join C-Path’s PKD Consortium

Sorin Fedeles, Ph.D., MBA to serve as new Executive Director

TUCSON, Ariz., October 14, 2021 – Critical Path Institute (C-Path) today announced it has named Sorin Fedeles, Ph.D., MBA as Executive Director of its Polycystic Kidney Disease Outcomes Consortium (PKDOC), effective October 1, 2021.

Dr. Fedeles comes to C-Path after 12-plus years as a scientist and faculty at Yale University School of Medicine, where he led a program focused on identifying new targets and pharmacological avenues for the treatment of autosomal dominant polycystic kidney disease, which represents the leading genetic cause of kidney failure in the U.S.  With expertise in genetics, biomedical discovery/translational research and inter-disciplinary collaborations, his primary role will be providing operational and scientific leadership that guides development and implementation of strategic priorities aimed at achieving PKDOC’s mission.

Established in 2010, PKDOC serves as a convener, curator, and innovator in the PKD community to generate solutions that accelerate drug development to bring new therapies to patients in need. “We are delighted to have Sorin join our PKDOC team to advance the consortium’s important work,” said Senior Vice President of the Translational and Safety Sciences Program John-Michael Sauer, Ph.D. “His extensive and unique combination of experience will be pivotal in continuing momentum toward ensuring PKD patients have access to safe and effective drugs as quickly as possible.”

PKDOC operates with support from the U.S Food and Drug Administration and the Polycystic Kidney Disease Foundation. “As the only organization in the U.S. solely dedicated to finding treatments and a cure for polycystic kidney disease, we are thrilled to have Sorin join the PKDOC team as Executive Director,” said Chris Rusconi, Ph.D., Chief Research Officer of the Foundation and Co-director of PKDOC. “With his breadth of professional experience and long-standing commitment to PKD research, the consortium is positioned to make a profound impact in the field.”

Originally from Romania, Dr. Fedeles received his undergraduate degree in biochemistry and cell biology from Hampshire College (Massachusetts), and earned three graduate degrees from Yale University, including a Master of Science in biological & biomedical sciences, a Ph.D. in genetics, and an MBA in healthcare management.

Dr. Fedeles brings his understanding of the drug development process, including preclinical research and clinical trials, cross-functional leadership and expert knowledge of genetic kidney disorders to C-Path and PKDOC. “As an academic member of PKDOC for several years I have seen first-hand the transformative impact of the consortium and C-Path as a whole,” said Fedeles. “I am honored to have the opportunity to lead PKDOC as it cultivates collaborations and advances innovative projects to improve the lives of those living with PKD.”

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

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.


 

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.

 

Media Contact:

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

C-Path Appoints Tufts’ Jonathan M. Davis as Senior Advisor in Pediatrics

TUCSON, Ariz., September 16, 2021 — Critical Path Institute (C-Path) today announced the appointment of Jonathan M. Davis, M.D., as Senior Advisor in Pediatrics working with its International Neonatal Consortium (INC) and advising on drug and device development in infants and children, effective immediately.

Davis is Vice-Chair of Pediatrics and Chief of Newborn Medicine at Tufts Children’s Hospital and Professor of Pediatrics at Tufts University School of Medicine. His research has focused on neonatal drug development, including optimizing approaches to treat neonatal abstinence syndrome.

“On behalf of everyone at C-Path and our INC partnership, we celebrate the appointment of Dr. Davis to the leadership team working in pediatrics,” said Klaus Romero, M.D., F.C.P., C-Path Chief Science Officer. “Jon has the experience, expertise and dedication to help advance C-Path’s goal as a global collaborator to forge an optimized regulatory path for evaluating the efficacy and safety of therapies not just for neonates, but the broader pediatric population as well.”

Davis has a long history of working with C-Path on various projects, including being INC’s co-director since its inception six years ago and as a co-principal investigator of its FDA-funded Real-World Data, Real-World Evidence project. “Jon is an outstanding colleague and true supporter of the C-Path model,” said Kanwaljit Singh, M.D., MPH, C-Path’s Director of Pediatric Programs. “We are very grateful for the opportunity to have Jon work with us on future pediatric initiatives at C-Path.”

Davis is currently funded by National Institutes of Health (NIH) and the U.S. Food and Drug Administration (FDA) to develop better biomarkers and outcome measures for clinical trials and new and existing therapeutics to improve neonatal outcomes. He serves as Chair of the Neonatal Advisory Committee in the Office of the Commissioner at FDA and Associate Director of the Tufts Clinical and Translational Science Institute. He is also the Co-PI of a recent $8 million NIH award to Tufts Medical Center to fund a national clinical trial to study the integration of targeted genomic sequencing into neonatal diagnosis and care.

Davis is currently funded by National Institutes of Health (NIH) and the U.S. Food and Drug Administration (FDA) to develop better biomarkers and outcome measures for clinical trials and new and existing therapeutics to improve neonatal outcomes. He serves as Chair of the Neonatal Advisory Committee in the Office of the Commissioner at FDA and Associate Director of the Tufts Clinical and Translational Science Institute. He is also the Co-PI of a recent $8 million NIH award to Tufts Medical Center to fund a national clinical trial to study the integration of targeted genomic sequencing into neonatal diagnosis and care.

“I am honored by this appointment and see it as an outstanding opportunity to grow and strengthen pre-competitive sciences, ultimately benefiting pediatric populations,” said Davis. “As an innovator in research and development of treatments for preterm infants, I’m excited to work with C-Path in this expanded partnership with the goal of accelerating medical product development in pediatrics.”

INC will hold its annual scientific meeting October 19 – 20, 2021. Those interested in attending may register for free here.

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.


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 US is headquartered in Tucson, Arizona and C-Path, Ltd. EU is headquartered in Dublin, Ireland, with additional staff in multiple other locations. For more information, visit c-path.org and c-path.eu.

Media Contact:

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

Rare Disease Innovation and Data Sharing Accelerated by New RDCA-DAP Program

C-Path, NORD and FDA host annual meeting, demonstration and launch of new rare disease data and analytics platform.

TUCSON, Ariz. and WASHINGTON, September 15, 2021 — The Rare Disease Cures Accelerator-Data and Analytics Platform initiative (RDCA-DAP®) officially launched its next phase on Tuesday establishing itself as the leading platform to accelerate rare disease treatment innovation. The launch was part of an all-day virtual workshop and annual meeting of rare disease stakeholders, featuring a platform demonstration, industry expert presentations and attendance by over 400 patients, providers, researchers, clinicians, biopharmaceutical companies, regulatory reviewers and scientists.

RDCA-DAP is an FDA-funded initiative that will serve as a centralized and standardized base to host and share de-identified rare diseases data and to support treatment innovation. It was created through a partnership between Critical Path Institute (C-Path), the National Organization for Rare Disorders (NORD®) and the U.S. Food and Drug Administration (FDA) and has grown to include many others throughout the rare disease community.

“There is a lot of enthusiasm for seeing this platform become all it can be for patients and drug developers. It is not just about the data. This is a place to generate solutions in a highly collaborative way,” said Jeff Barrett, Ph.D., F.C.P., C-Path Senior Vice President and RDCA-DAP Lead. “We expect RDCA-DAP to be a place to promote candid dialogue, optimize patient selection and trial design, and co-develop the tools to further de-risk decision making for rare disease drug development.”

After a public premiere showcasing the functionality of the platform, presentations throughout the day emphasized how RDCA-DAP will curate data to break down silos, provided insight into the platform’s development and testing and showcased the importance of RDCA-DAP from the perspective of critical stakeholders, including academic, clinical, regulatory and patient communities.

On the importance of incorporating patients’ perspectives through RDCA-DAP, Theresa Mullin, Ph.D., Associate Director for Strategic Initiatives, Center for Drug Evaluation and Research (CDER), FDA, said, “Identifying the burdens of disease and current therapies that matter most, what can be measured, and what would constitute a meaningful change, can provide insight on the clinical context for regulatory review and potentially more direct evidence of drug benefits and risks when collected using valid and reliable measures and tools.”

A total of 31 speakers and panelists from patient organizations and regulatory, industry and academic fields participated in the workshop. Five patient profiles underscoring the importance of data sharing were also highlighted. Those interested in viewing the workshop and accessing supplemental information can do so here.

“Patient owned data collected by patient groups are less likely to get stuck in a silo,” said Ed Neilan, M.D., Ph.D., Chief Medical and Scientific Officer, NORD. “Of the over 7,000 estimated rare diseases, more 90% have no FDA approved treatment. And currently the attention of the pharmaceutical industry is on 100 or 200 of these rare diseases. RDCA-DAP may bring attention to rare diseases that might otherwise remain relatively neglected.”

To learn more and request access to RDCA-DAP, apply directly on the platform at https://portal.rdca.c-path.org. To submit critical rare disease data, contact the project team at rdcadap@c-path.org or visit c-path.org/programs/rdca-dap and rarediseases.org/rdca-dap.

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.


About Critical Path Institute
Critical Path Institute (C-Path) is an independent, nonprofit organization established in 2005 as a public and private partnership. C-Path’s mission is to catalyze the development of new approaches that advance medical innovation and regulatory science, accelerating the path to a healthier world. An international leader in forming collaborations, C-Path has established numerous global consortia that currently include more than 1,600 scientists from government and regulatory agencies, academia, patient organizations, disease foundations, and dozens of pharmaceutical and biotech companies. C-Path 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 the National Organization for Rare Disorders (NORD)®
The National Organization for Rare Disorders (NORD) is the leading independent advocacy organization representing all patients and families affected by rare diseases in the United States. NORD began as a small group of patient advocates that formed a coalition to unify and mobilize support to pass the Orphan Drug Act of 1983. Since then, the organization has led the way in voicing the needs of the rare disease community, driving supportive policies, furthering education, advancing medical research, and providing patient and family services for those who need them most. Together with over 330 disease-specific member organizations, more than 15,000 Rare Action Network advocates across all 50 states, and national and global partners, NORD delivers on its mission to improve the lives of those impacted by rare diseases. Visit rarediseases.org.

Media Contacts:

Kissy Black
C-Path
615.310.1894
kissyblack@lotosnile.com

Rohan Narayanan
NORD
571-255-0042
rnarayanan@rarediseases.org

C-Path and RARE-X Establish New Collaboration for Rare Disease Data Sharing

RARE-X and Critical Path Institute align on their mission and commitment for data sharing to improve rare disease research

TUCSON, Ariz. and ALISO VIEJO, Calif., September 13, 2021 — Critical Path Institute (C-Path), who’s aim is to catalyze the development of new approaches that advance medical innovation and regulatory science, today announced a collaboration with RARE-X to improve ways researchers can access and analyze patient data. RARE-X is a nonprofit organization dedicated to enabling patient communities to collect, manage and share their de-identified data to advance research.

C-Path, with its Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP®) initiative, and RARE-X are developing an ecosystem in which rare disease data are collected, shared and used to further research. The two groups have a similar vision to make individual-level data available and used as widely as possible by researchers and drug developers.

“Working jointly with RARE-X, we can develop models and drug development tools informed by the data collected and consented for on the RARE-X platform,” said Jeff Barrett, Ph.D., F.C.P., C-Path Senior Vice President and RDCA-DAP Lead. “Such models would be developed with open data science principles in mind and the intention to facilitate the development of new treatments for rare disease patients. For example, an initial collaborative effort with RARE-X may include the co-development of a disease progression model for a specified disease area.”

RARE-X supports the collection of structured patient-owned data, and has developed data governance and consents that support patients in sharing their de-identified data broadly and often. In addition to data collection, RARE-X is focused on connecting disparate data, while embarking on comprehensive federated data-sharing initiatives that support data sharing consortia to accelerate research and medicine development.

C-Path’s RDCA-DAP provides a centralized and standardized infrastructure to support and accelerate rare disease characterization targeted for clinical development. Additionally, the platform includes a framework that supports the rigorous conduct of natural history studies, with attention to established data quality standards, in order to be most useful to clinical trial design and regulatory review. It includes a robust, integrated database and analytics hub that allows for the aggregation of rare disease data from various sources and the efficient and effective interrogation of that data.

The C-Path and RARE-X collaboration will share learnings around standardization of data elements and data structures, consent language and other elements promoting the inter-operability of data sharing.

“We look forward to bringing forward best practices on structure and standardization of rare disease data, governance, consent and data tracking with C-Path to accelerate research,” said RARE-X CEO Charlene Son Rigby. “And by collaborating with C-Path, patients’ ability to share their data through the RARE-X platform, enabled with our unique consent and data-sharing agreements, will expand.”

A first look at RDCA-DAP’s functionality and operability will be unveiled at the 2021 RDCA-DAP Workshop Tuesday, September 14. Those interested in attending can register here.

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.


About Critical Path Institute
Critical Path Institute (C-Path) is an independent, nonprofit organization established in 2005 as a public and private partnership. C-Path’s mission is to catalyze the development of new approaches that advance medical innovation and regulatory science, accelerating the path to a healthier world. An international leader in forming collaborations, C-Path has established numerous global consortia that currently include more than 1,600 scientists from government and regulatory agencies, academia, patient organizations, disease foundations, and dozens of pharmaceutical and biotech companies. C-Path 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 RARE-X
RARE-X is a 501(c)(3) rare disease technology nonprofit focused on supporting the acceleration and development of life-altering treatments and future cures for patients impacted by a rare disease. Enabled by best-in-class technology, patients, researchers, and other technology vendors, RARE-X will gather structured, fit-for-purpose data to share broadly, benefitting from 21st-century governance, consent, and federated data-sharing technology. RARE-X is building the largest collaborative patient-driven, open-data access project for rare diseases globally. For more information, visit rare-x.org.

Media Contacts:

Kissy Black
C-Path
615.310.1894
kissyblack@lotosnile.com

Tom Hume
RARE-X
tomh@rare-x.org

Breaking Down Data Siloes: New Analytics Platform Sparks Opportunity for Rare Disease Field

C-Path, NORD and FDA have spent two years building the platform set to launch Sept. 14 at the 2021 RDCA-DAP Workshop.

TUCSON, Ariz. and WASHINGTON, September 1, 2021 — For two years, Critical Path Institute (C-Path), the National Organization for Rare Disorders (NORD) and the U.S. Food and Drug Administration (FDA) have joined with others throughout the rare disease community to create a novel, best-in-class platform to accelerate rare disease treatment innovation.

On Tuesday, September 14, 10 a.m. – 3:45 p.m. ET, the Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP®), an FDA-funded initiative, will have its public premiere during the annual meeting for the project. The platform will host, standardize and share rare diseases data as part of its functionality.

In addition to the demonstration of the platform itself, presentations throughout the day will show how RDCA-DAP is breaking down data silos, provide insight into the platform’s development and testing process, and showcase the importance of RDCA-DAP from the perspective of critical stakeholders, including academic, clinical, regulatory and patient communities.

“We invite colleagues and patients in the rare disease community to join us at the workshop to see the latest developments since launching the RDCA-DAP initiative in 2019,” said Jeff Barrett, Ph.D., F.C.P., C-Path Senior Vice President and RDCA-DAP Lead. “It will be the public’s first look at the platform’s capabilities, showcasing how it can be used to generate solutions for rare diseases.”

In collaboration with Aridhia Informatics, the team from C-Path’s Data Collaboration Center has been designing and building the platform, which will allow researchers to develop advanced models to quantitatively describe relevant aspects of disease progression within and across diseases, capturing relevant sources of variability. Researchers can interact with these sophisticated models through a user-friendly interface to potentially submit them for regulatory review and endorsement as quantitative drug development tools, or for the purpose of running simulations intended to optimize clinical trial design.

“There is tremendous promise in the RDCA-DAP platform to empower patients and families to be a driving force in rare disease innovation,” said Ed Neilan, M.D., Ph.D., Chief Medical and Scientific Officer, NORD. “The demonstration and workshop will showcase what’s possible when the community comes together to share de-identified data for broader use to expedite the development of treatments.”

NORD has worked with data custodians to contribute datasets from six different disease states with surveys housed in NORD’s IAMRARE® registry platform to RDCA-DAP and has agreements in place to contribute datasets in three additional disease areas. C-Path has secured 10 additional disease datasets, with existing datasets from C-Path’s rare disease consortia having been integrated as well. Data from many more disease states are at an intermediate stage of the contribution process.

Those interested in attending the annual meeting may register for free here.

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.


About Critical Path Institute
Critical Path Institute (C-Path) is an independent, nonprofit organization established in 2005 as a public and private partnership. C-Path’s mission is to catalyze the development of new approaches that advance medical innovation and regulatory science, accelerating the path to a healthier world. An international leader in forming collaborations, C-Path has established numerous global consortia that currently include more than 1,600 scientists from government and regulatory agencies, academia, patient organizations, disease foundations, and dozens of pharmaceutical and biotech companies. C-Path 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 the National Organization for Rare Disorders (NORD)®
The National Organization for Rare Disorders (NORD) is the leading independent advocacy organization representing all patients and families affected by rare diseases in the United States. NORD began as a small group of patient advocates that formed a coalition to unify and mobilize support to pass the Orphan Drug Act of 1983. Since then, the organization has led the way in voicing the needs of the rare disease community, driving supportive policies, furthering education, advancing medical research, and providing patient and family services for those who need them most. Together with over 330 disease-specific member organizations, more than 15,000 Rare Action Network advocates across all 50 states, and national and global partners, NORD delivers on its mission to improve the lives of those impacted by rare diseases. Visit rarediseases.org.

Media Contacts:

Kissy Black
C-Path
615.310.1894
kissyblack@lotosnile.com

Rohan Narayanan
NORD
571-255-0042
rnarayanan@rarediseases.org

C-Path Appoints Clinical Outcome Assessment Expert as New Executive Director

Sonya Eremenco will lead C-Path’s PRO Consortium

TUCSON, Ariz., August 5, 2021— Critical Path Institute (C-Path) announced it has named Sonya Eremenco, M.A., as Executive Director of its Patient-Reported Outcome (PRO) Consortium. Eremenco joined C-Path in 2016 as Associate Director of the PRO Consortium and was promoted to Director in January 2021. In addition to her leadership roles with the PRO Consortium, she also served as Acting Director of the ePRO Consortium from September 2018 to May 2021.

“Since joining C-Path five years ago, Sonya has consistently and amply demonstrated she is more than capable of taking on the role that I have had the privilege to hold for more than 12 years,” said Stephen Joel Coons, Ph.D., Senior Vice President of the Clinical Outcome Assessment (COA) Program. “Based on her extensive experience and unwavering commitment to leveraging the voice of the patient in drug development, I have every confidence that Sonya will lead the PRO Consortium to new heights and significant accomplishments.”

As Executive Director of C-Path’s PRO Consortium, Eremenco will provide operational and scientific leadership that guides development and qualification of PRO measures and other COAs that will be publicly available for use in clinical trials where COA-based endpoints are used to support labeling claims.

Eremenco has more than 25 years of experience in the development and evaluation of PRO measures and other COAs, with a focus on multicultural development, linguistic validation and electronic implementation. Prior to joining C-Path, Eremenco served as Director of ePRO New Products at Evidera.

“I am honored to be taking on the leadership of the PRO Consortium at a time when patient-focused drug development is increasingly recognized as essential to ensuring that new medical products provide clinical benefit in terms of how patients feel, function and survive,” said Eremenco. “The PRO Consortium enables sponsors to collaborate pre-competitively to shape the drug development environment by creating robust measurement tools that all stakeholders can employ in clinical trials. I look forward to continuing this important work.”

Eremenco received her undergraduate degree in cultural anthropology with a concentration in linguistics from Duke University and earned a Master of Arts in cross-cultural communication from DePaul University.
For more information on C-Path’s PRO Consortium, visit: https://c-path.org/programs/proc.

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.


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.

Media Contact:

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

Digital Health Technologies Hold Key to New Parkinson’s Treatments

Global stakeholders come together to collaborate with urgency to address unmet needs for Parkinson’s therapies.

TUCSON, Ariz., July 20, 2021 — The use of digital health technologies across health care and drug development has accelerated. A new paper titled “Digital Progression Biomarkers as Novel Endpoints in Clinical Trials: A Multistakeholder Perspective,” co-authored by experts across diverse disciplines, highlights how new remote monitoring technologies present a tremendous opportunity to advance digital medicine in health care even further, specifically in Parkinson’s disease. This perspective paper is co-authored by the academic leader of the largest funded project for digital technologies in Europe, Professor Lynn Rochester, University of Newcastle; European Medicines Agency (EMA) scientific leader, Dr. Maria Tome; young investigator and Ph.D. candidate Reham Badawy; physician and Parkinson’s patient, Dr. Soania Mathur; and Dr. Diane Stephenson, Executive Director of the Critical Path for Parkinson’s (CPP) Consortium.

Global collaborative efforts are underway with the goal of advancing the use of digital health technologies for use in Parkinson’s clinical research and therapeutic trials — yet several gaps and barriers stand in the way of success. These include data security issues, the rapidly evolving nature of the technology, lack of consensus on data standards, vast diversity of distinct studies carried out on different devices and the need for open science.

CPP’s Digital Drug Development Tool team at Critical Path Institute consists of industry members, scientific academic advisors, patient research organizations and people living with Parkinson’s all collaborating across the globe to seek advice early and often from regulatory agencies. Companies advancing innovative therapies for the treatment of Parkinson’s see the promise of digital technologies, yet they also recognize that there are gaps that are too challenging to overcome on their own. CPP’s focus on the voice of people living with Parkinson’s aligns with the U.S. Food and Drug Administration (FDA) and EMA’s vision for patient-focused drug development. Sharing costs, risks and knowledge will streamline a more efficient runway for regulatory endorsement in the future.

“We felt it was imperative to come together on this paper, at this moment, to bring attention to how existing digital health technologies can complement traditional modalities and transform and accelerate clinical research and therapeutic development,” said Rochester.

Dr. Mathur, who has lived with Parkinson’s for 22 years, inspired the team of five women leaders to work on this paper across different countries during the pandemic. “It is vital to include the patient voice to drive the sense of urgency when it comes to Parkinson’s research. As patients, we fully experience the unrelenting progression of this disease, the ongoing daily challenges that we live with. From the direction of research to identifying the tools that can estimate relevant outcome measures in the search for new therapeutics that are directed towards disease modification or improved quality of life, patient input is absolutely integral to its success. This collaboration kept that sense of urgency at the forefront.”

“EMA works with the FDA to assure that digital technologies are aligned with what is important to patients,” said Dr. Tome. “The pace of progress is going to be accelerated by applying principles of what it took the world to tackle the COVID-19 pandemic,” Stephenson added. “True collaborations amongst all stakeholders are urgently needed to make efficient progress, avoid duplication of effort, share costs and risks and advance with warp speed.”

Professor Bas Bloem, editor-in-chief of the Journal of Parkinson’s Disease and author of the book “Ending Parkinson’s Disease” said, “We are very excited to publish this very important paper in our journal, as it provides a clear and visionary glimpse into the future of better care and innovative research approaches in the field of Parkinson’s disease.”

The paper is featured in Journal of Parkinson’s dedicated issue Digital Health in Parkinson’s Disease here https://content.iospress.com/articles/journal-of-parkinsons-disease/jpd202428. For more information, visit CPP’s website or read Digital Technology Driving Tangible Advancements in Parkinson’s Disease Research and Clinical Care.

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.

The views expressed in this release are the personal views of the authors and may not reflect the position of the various groups or parties represented.


About CPP

Created in partnership with Parkinson’s UK, one of the world’s largest charity funders of Parkinson’s research, the Critical Path for Parkinson’s Consortium was launched on October 14, 2015. This is a global collaboration that promises to pave the path to new treatments for Parkinson’s. By facilitating collaboration among scientists from the bio-pharmaceutical industry, academic institutions, government agencies, and patient-advocacy associations, CPP fosters consensus and data-driven research to increase efficiency, safety, and speed in developing new therapies.

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.

Media Contact:

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

Launch of UNITE4TB Partnership Marks a New Era in Tuberculosis Treatment Development

The partnership will accelerate the development of new Tuberculosis (TB) drug regimens as part of the Innovative Medicines Initiative (IMI), a public-private European Research & Development Consortium

TUCSON, Ariz., July 15, 2021 — To advance anti-tuberculosis (TB) science and enable the progression of new, safe, and affordable treatment solutions for TB patients worldwide, a new consortium of 30 partners from 13 countries has officially launched. The 7-year, €185 million project called UNITE4TB, aims to accelerate and improve the clinical evaluation of combinations of existing and novel drugs, with the goal of developing new and highly active TB treatment regimens for drug-resistant and -sensitive TB.

UNITE4TB is the newest project of the IMI AMR Accelerator, a public-private collaboration with the shared goal of progressing the development of new medicines to treat or prevent resistant bacterial infections.

“Tuberculosis is a major threat to public health worldwide. By bringing together leading experts from the public and private sectors in Europe and beyond, UNITE4TB is well placed to deliver results that will accelerate the development of better treatment regimens to tackle this disease,” says Dr. Pierre Meulien, Executive Director of IMI.

Worldwide, TB is one of the top 10 causes of death and the leading cause from a single infectious agent (above HIV/AIDS). The growing emergence of multidrug-resistant TB is well-recognised as a public health challenge and has sparked new interest and investment in anti-TB drug development. Despite increased activity in the field, an integrated approach to TB drug development is still limited.

With European Federation of Pharmaceutical Industries and Associations (EFPIA) and Associated Partners on board, UNITE4TB has access to the majority of the most innovative TB compounds, currently in late pre-clinical, clinical phase 1, and early phase 2 stage. The consortium will deliver an efficient, global clinical trials network equipped to conduct phase 2 trials. State-of-the-art adaptive trial designs will be implemented, and advanced modelling, artificial intelligence and machine learning techniques will be employed. All of this will allow for the selection and testing of novel combination regimens with a high probability of success in subsequent phase 3 clinical trials.

Anja Karliczek, Germany’s Federal Minister of Education and Research, says: “Europe’s UNITE4TB project creates an important new platform for research to combat tuberculosis. Science and industry will jointly test their clinical candidates and share research results. The objective is to develop effective combinations for new, urgently needed solutions to treat tuberculosis. This public-private partnership will set a new standard in the fight against global diseases such as TB. UNITE4TB is a remarkable example of international research collaboration. I am delighted that Germany is supporting the consortium with funding of around 25 million euros to the two German partners. I am confident that UNITE4TB will contribute towards achieving the goal of ending tuberculosis by 2030 that was adopted by the G20 Heads of State and Government at the UN General Assembly.”

Critical Path Institute, Ltd. (Ireland) is a wholly owned subsidiary of Critical Path Institute, an independent, nonprofit established as a public-private partnership. C-Path, Ltd. will lead the data sharing workgroup for UNITE4TB and will be assisting with machine learning and artificial intelligence efforts. 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. C-Path, Ltd. EU is in Dublin, Ireland, and C-Path U.S. is in Tucson, Arizona. Learn more at c-path.eu and c-path.org.

UNITE4TB is the largest public-private collaboration on clinical TB drug development in the history of the EU. It will set a new standard for anti-TB regimen development, enhancing the efficiency with which new treatments are delivered to TB patients across the world.

This project has received funding from the Innovative Medicines Initiative 2 Joint Undertaking (JU) under grant agreement No 101007873. The JU receives support from the European Union’s Horizon 2020 research and innovation programme and EFPIA, Deutsches Zentrum für Infektionsforschung e. V. (DZIF), and Ludwig-Maximilians-Universität München (LMU). EFPIA/AP contribute to 50% of funding, whereas the contribution of DZIF and the LMU University Hospital Munich has been granted by the German Federal Ministry of Education and Research.

For more information and interview requests, please contact:

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



UNITE4TB is a public-private partnership with representation from academic institutions, small- and medium-sized enterprises (SMEs), public organisations, and pharmaceutical companies. Over the next 7 years, the consortium will be active in approximately 40 trial sites on four continents (Europe, Asia, Africa and South America), with the goal of delivering novel phase 2 clinical trials that will accelerate the development of new TB drugs and regimens. Achieving this goal will facilitate fulfilment of one of the main unmet needs in the TB field: better-tolerated drug regimens of shorter duration that can be deployed to tackle tuberculosis across various drug-resistance patterns and co-morbidities. For more information, visit the Consortium website: www.unite4TB.org

Consortium partners

Academic/SME partners

  • Stichting Radboud Universitair Medisch Centrum (Radboudumc) (The Netherlands)
  • London School for Hygiene and Tropical Medicine (LSHTM) (United Kingdom)
  • University of Oxford (United Kingdom)
  • Forschungszentrum Borstel, Leibniz Lungenzentrum (Germany)
  • Lygature (The Netherlands)
  • Lancaster University (United Kingdom)
  • University College London (United Kingdom)
  • TASK (South Africa)
  • Vita-Salute San Raffaele University (UniSR) (Italy)
  • Helmholtz Zentrum München (Germany)
  • KNCV Tuberculosis Foundation (KNCV) (The Netherlands)
  • Critical Path Institute, Limited (Ireland) 
  • European Lung Foundation (United Kingdom)
  • Instituto de Saude Publica da Universidade do Porto (ISPUP) (Portugal)
  • University of Liverpool (United Kingdom)
  • Institut de Recherche Pour le Developpement (France)
  • University of Hamburg (Germany)
  • University of California San Francisco (UCSF) (USA)
  • TB Alliance (USA)
  • FIND (Switzerland)
  • University of Milano (UMIL) (Italy)
  • University St Andrews (United Kingdom)
  • Uppsala University (Sweden)
  • European Respiratory Society (Switzerland)
  • TBnet (Germany)

EFPIA/Associated Partners

  • GlaxoSmithKline Investigación y Desarrollo S L (GSK) (Spain)
  • Janssen Pharmaceutical (Belgium)
  • Otsuka Novel Products GmbH (Germany)
  • Deutsches Zentrum für Infektionsforschung (Germany)
  • LMU University Hospital Munich (Germany)

View a detailed overview of all partner information on the UNITE4TB website