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

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

 

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

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

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

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

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

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

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

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

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

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

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

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

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


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

Media Contact:

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

 

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

 

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

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

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

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

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


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

 

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

Rare Disease Cures Accelerator-Data and Analytics Platform First Year Milestones Highlighted at Virtual Workshop

 

 

C-Path, NORD, FDA and more come together to showcase the importance of data sharing in rare disease drug development

 

TUCSON, Ariz. and WASHINGTON, D.C. October 21, 2020 — The Critical Path Institute (C-Path) and the National Organization for Rare Disorders (NORD®) hosted the Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP) 2020 Virtual Workshop on Monday, October 19. The platform, funded by a cooperative agreement through the U.S. Food and Drug Administration (FDA), serves as a centralized and standardized infrastructure to support data sharing and data analysis to aid in the understanding of rare diseases.

Theresa Mullin, Ph.D., Associate Director for Strategic Initiatives, FDA, Center for Drug Evaluation and Research (CDER) opened the workshop and explained how RDCA-DAP is part of the FDA’s aggressive plan to advance drug development for rare diseases. “The concept of RDCA-DAP has been discussed by C-Path and NORD with hundreds of stakeholders through public meetings, webinars, focus groups and one on one interviews to understand what the community would like to see in an analytics platform,” Mullin said. “These learnings are being used now to make the platform as useful as possible to as many people as possible.”

The virtual workshop, attended by more than 400 individuals from patient groups, academia, industry and regulatory agencies informed the rare disease community of the initiative’s first-year progress. Topics included pilot analyses and the development of tools facilitated by RDCA-DAP to advance characterization and accelerate drug development in rare diseases. Additional updates were provided on the platform’s data sharing infrastructure and recent enhancements, including the ability to search, sort and analyze data.

C-Path RDCA-DAP Executive Director Jane Larkindale, D.Phil., detailed milestones the effort achieved in its first year and outlined plans for the future. In year one, the RDCA-DAP team worked with the community to develop a data governance structure that answers the needs of both data contributors and data users, accessed initial datasets and built actionable prototypes to demonstrate the utility of the platform.

“Our priority is not to compete, but to collaborate with other groups doing the same or similar work in rare disease drug development,” said Larkindale. “Stakeholders are open to collaboration and data sharing and we have been meeting with different groups about what is already being done and how we can interoperate with other existing projects.”

Several case studies were presented during the workshop to demonstrate the power of the platform including a demonstration of the value of the integration of data and analysis of combined datasets. In one case, Pravin Jadhav, Ph.D., global team leader at Otsuka America Pharmaceutical, Inc. shared how patient-level data aided in the acceptance of a biomarker for an accelerated approval of a new therapy for polycystic kidney disease.

C-Path’s Chief Science Officer Klaus Romero, M.D., M.S., F.C.P. shared examples of prototypes built from RDCA-DAP patient-level data that demonstrated the ability to integrate data from multiple disease areas to generate advanced models of disease progression and to optimize the characterization of rare diseases, as a means to accelerate drug development for these conditions.

Similarly, NORD’s Director of Research Vanessa Boulanger, M.Sc. used the example of the NPKUA phenylketonuria patient registry from NORD’s IAMRARE™ platform to show how patient registry data can inform drug development programs.

“The PKU case study is one example of the insights that patient registry and natural history study data can bring to RDCA-DAP,” said Boulanger. “Patient-driven data gives us an opportunity to arrive at a more comprehensive understanding of rare diseases, to learn what matters to patients, and then to apply that knowledge to the design of studies that may result in treatments that are truly meaningful to the community.”

RDCA-DAP is positioned to generate solutions to drug development bottlenecks by maximizing the utility of patient-level data and making that data available to a larger community. C-Path President and CEO Joseph Scheeren, Pharm.D. and NORD President and CEO Peter L. Saltonstall both thanked the community for coming together around the concept of RDCA-DAP, acknowledging that data contribution is key to its success. FDA/CDER Office of Neuroscience Acting Director Billy Dunn, M.D. closed the workshop congratulating the teams on the work accomplished since RDCA-DAP’s launch in September 2019 and discussing the three main areas of the FDA’s rare disease drug development efforts, including RDCA-DAP, standard core clinical outcome assessments and clinical trials network.

“The reason RDCA-DAP is so critical, is that it focuses on the characterization of the disease and if we don’t have a good characterization of the disease, we really can’t do our job,” Dunn said. “Natural history, the variability of symptoms and experiences and the opportunities for clinical trial design are all addressed by a more robust characterization of disease.”

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

A full recording of the workshop can be accessed here.

*Funding for this program was made possible, in part, by the Food and Drug Administration through grant (U18 FD 005320). Views expressed in written materials or publications and by speakers and moderators do not necessarily reflect the official policies of the Department of Health and Human Services; nor does any mention of trade names, commercial practices, or organization imply endorsement by the United States Government.

 


 

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

 

About 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. 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, NORD has led the way in voicing the needs of the rare disease community, driving supportive policies and education, advancing medical research and providing patient and family services for those who need them most. NORD is made strong together with over 325 disease-specific member organizations and their communities and collaborates with many other organizations on specific causes of importance to the rare disease patient community. Visit rarediseases.org.

 

Media Contacts:

Kissy Black
C-Path
615.310.1894
kissyblack@lotosnile.com

Laura Mullen
NORD
203.304.7258
lmullen@rarediseases.org

C-Path Appoints Two New Members to Board of Directors

 

TUCSON, Ariz., April 30, 2020 — Tucson-based Critical Path Institute (C-Path) today announced the appointment of two new members to its Board of Directors: James W. Newman, CPA, former Executive Chairman of Victory Pharma, Inc. and Tomas Salmonson, Ph.D., M.Sc., former Chair of the Committee for Medicinal Products for Human Use (CHMP), European Medicines Agency (EMA).

“We are pleased to welcome both Jim and Tomas to the C-Path Board of Directors,” said Timothy R. Franson, chairman of the C-Path Board. “Their wealth of knowledge and experience in the pharmaceutical industry and regulatory sector, respectively, will be invaluable as we look toward the future and how we can continue to evolve and improve drug development across the globe.”

Newman is a longtime pharmaceutical executive with more than 30 years’ experience in pharmaceutical company senior management, finance, information systems, facilities operations and strategic planning. He founded Victory Pharma, Inc. in 2001 and served as Executive Chairman from 2001 – 2013. During his tenure at Victory, Newman was responsible for the acquisition and development of several proprietary products for the treatment of chronic pain. The company grew to approximately $100 million in annual sales prior to its sale to Shionogi in 2011. In 1998, Newman founded DJ Pharma, Inc. and served as President and CEO. The company expanded to 400 employees and $65 million in sales in two years. DJ Pharma sold at a 500 percent ROI for investors in 2001.

As Senior Vice President of Finance and Administration for Dura Pharmaceuticals, Inc., Newman redirected the company from a biotech to a pharmaceutical sales and marketing organization. He was responsible for organizing and completing its IPO and for four secondary and three private offerings to expand the market capitalization to more than $2 billion in six years. During his active retirement, Newman serves on the board of several charitable organizations and on the Executive Committee of the Pacific Crest Trail Association.

Dr. Tomas Salmonson has deep expertise in pharmacokinetics, bioequivalence, clinical trial methodology, clinical drug development and regulatory affairs and is currently partner at Consilium Salmonson & Hemmings. He left the Medical Products Agency in February 2019 after more than 30 years at the Swedish agency and the European regulatory network. He chaired the CHMP at the EMA between 2012 and 2018. Before that he was a member of CHMP representing Sweden between 1999 and 2012. During his last 10 years there, he also represented the EU at the ICH Steering /Management Committee and the ICH Assembly. Salmonson obtained his Ph.D. from Faculty of Medicine Uppsala University, Uppsala, Sweden and an M.Sc. from Uppsala University. He did postgraduate research at the University of California in San Francisco in the mid-80s.

Salmonson has published over 60 publications in different areas including pharmacokinetics and regulatory affairs. In 2016, he received the DIA Outstanding Contribution to Health Award, the Pharmacist of the Year in Sweden in 2017 and the TOPRA Lifetime Achievement Award in 2018.

“We’re fortunate to be able to leverage Jim’s previous operational and senior management experience at various, highly successful pharmaceutical companies and Tomas’ extensive background in drug development and the regulatory process through complex and challenging environments is of tremendous value, especially during these unprecedented times,” said C-Path President and CEO Joseph Scheeren, Pharm.D.

Newman and Salmonson’s Board appointments will begin June 1, 2020.

 


 

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

 

Contact:

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

C-Path Awarded FDA Contract to Enhance the Assessment of Clinical Outcomes in Pediatric Asthma Treatment Trials

Resulting novel drug development tools will support patient-focused drug development for children with asthma

 

 

TUCSON, Ariz., April 21, 2020 — The Critical Path Institute (C-Path) announced today it has been awarded a U.S. Food and Drug Administration (FDA) contract in support of ongoing development of novel clinical outcome assessments for pediatric asthma. C-Path’s Patient-Reported Outcome (PRO) Consortium will carry out this work through its Pediatric Asthma Working Group. Specifically, these assessments are intended to facilitate innovative patient-focused drug development and aid regulatory decision making by filling an unmet measurement gap.

The 5-year partnership has two aims. The first is to conduct qualitative research to confirm the content validity of a patient-reported outcome (PRO) measure for children from 8 through 11 years old with asthma and an observer-reported outcome (ObsRO) measure for caregivers of children from 4 through 11 years old with asthma. The second aim is to conduct quantitative research to generate evidence of cross-sectional measurement properties of the PRO and ObsRO measures to support their qualification under FDA’s Clinical Outcome Assessment Qualification Program.

“We are honored to receive FDA support of, and collaboration on, this new project,” said C-Path President and CEO Joseph Scheeren, Pharm.D. “FDA funding, in combination with the expertise of the PRO Consortium, will enable development of much-needed tools for the scientific community and, most importantly, will ultimately lead to improved medical outcomes for pediatric asthma patients.”

Pediatric asthma is a chronic inflammatory disease of the airways and is the most common childhood condition worldwide. In the United States, asthma is twice as common in children as adults and is a leading cause of school absenteeism as well as the third ranking cause of hospitalization of children.

“Pediatric asthma treatment trials face challenges in the reliable assessment of lung function in young children leading to the need to assess self-reportable or observable signs and symptoms of asthma to evaluate the benefit of new therapies,” said Stephen Joel Coons, Ph.D., Executive Director of the PRO Consortium. “These fit-for-purpose measures will significantly enhance the assessment of clinical benefit for children with asthma.”

*Funding for this press release was made possible, in part, by the Food and Drug Administration through contract number 75F40119C10135. Views expressed here do not necessarily reflect the official policies of the Department of Health and Human Services nor does any mention of an organization imply endorsement by the United States Government.
 


 

About Patient-Reported Outcome Consortium

The Patient-Reported Outcome (PRO) Consortium was formed in 2008 by Critical Path Institute in cooperation with the US Food and Drug Administration’s Center for Drug Evaluation and Research and the pharmaceutical industry. The mission of the PRO Consortium is to establish and maintain a collaborative framework with appropriate stakeholders for the qualification of PRO measures and other clinical outcome assessment (COA) tools that will be publicly available for use in clinical trials where COA-based endpoints are used to support product labeling claims.

 

About Critical Path Institute

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

 

Contact:

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

C-Path and Lundbeck Announce a Data Sharing Collaboration to Enable Development of Advanced Drug Development Tools in Alzheimer’s Disease

 

 

TUCSON, Ariz., March 18, 2020 — The Critical Path Institute (C-Path) and H. Lundbeck A/S (Lundbeck) are proud to announce that they will work together to significantly improve the scientific community’s insight in Alzheimer’s disease (AD) through Lundbeck’s contribution of a unique set of clinical trial data from more than 2,500 AD patients to the Critical Path for Alzheimer’s Disease (CPAD) consortium’s integrated database for qualified researchers across the globe to access.

The Data Contribution Agreement (DCA) between Lundbeck and C-Path, with support from Gates Ventures, will allow for these clinical data to be integrated and managed by CPAD, with support from C-Path’s Data Collaboration Center (DCC) adding to the CPAD patient-level database in AD, which presently contains data from more than 14,500 patients. CPAD is currently exploring acquisition of contemporary datasets from other sources with an expected growth of the CPAD database to include more than 40,000 patient-level records in the coming months.

This contribution of patient-level data from Lundbeck embodies Lundbeck’s continued support to increase pre-competitive collaborations between pharmaceutical companies and academia, and to advance effective public-private partnerships. Generous contributions such as these ensure that CPAD can continue its mission to develop innovative quantitative tools and methods to de-risk and speed up the drug development process in AD.

These data stem from Lundbeck’s Phase II and III studies that evaluated the compound idalopirdine in AD and include relevant information about disease progression, drug effects and clinical trial design. The shared data are highly valuable and the contribution of this type of data is vitally important to CPAD’s work in developing a disease progression model across the entire continuum of AD, from the earliest to late stages of the disease. CPAD aims to provide advanced drug development tools that will aid in optimizing clinical trial designs and execution and facilitating the regulatory review process.

“Lundbeck and C-Path recognize the hard work to generate these data and contributions of the many patients with Alzheimer disease, their relatives and caregivers, and the investigators who took part in these trials,” said C-Path President and CEO Joseph Scheeren, Pharm.D. “We look forward to continuing to work together to advance and expedite the development of new therapies.”

“We are delighted with this opportunity to share data with CPAD, ensuring that researchers can get the most out of them in advancing the understanding of Alzheimer’s and how to best develop new treatments. We are proud to contribute to this work, not only through our own drug development activities, but also in this unique collaboration,” said Mads Dalsgaard, Senior Vice President, Experimental Medicine & Clinical Development at Lundbeck.

 


 

About Critical Path Institute
C-Path is an independent, nonprofit organization established in 2005 as a public-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’s US headquarters is located 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 H. Lundbeck A/S
H. Lundbeck A/S (LUN.CO, LUN DC, HLUYY) is a global pharmaceutical company specialized in brain diseases. For more than 70 years, we have been at the forefront of neuroscience research. We are tirelessly dedicated to restoring brain health, so every person can be their best.

An estimated 700 million people worldwide are living with brain diseases and far too many suffer due to inadequate treatment, discrimination, a reduced number of working days, early retirement and other unnecessary consequences. Every day, we strive for improved treatment and a better life for people living with brain diseases – we call this Progress in Mind.

Read more at www.lundbeck.com/global/about-us/progress-in-mind.

Our approximately 5,500 employees in more than 50 countries are engaged in the entire value chain throughout research, development, production, marketing and sales. Our pipeline consists of several R&D programs and our products are available in more than 100 countries. We have research centres in Denmark and California and our production facilities are located in Denmark, France and Italy. Lundbeck generated revenue of DKK 18,1 billion in 2018 (EUR 2,4 billion; USD 2,8 billion).
For additional information, we encourage you to visit our corporate site www.lundbeck.com and connect with us on Twitter at @Lundbeck and via LinkedIn.

 

Contact:

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

C-Path to Lead Multi-Stakeholder Engagement on FDA’s Proposed Novel Framework to Enhance the Pediatric Medical Device Ecosystem

 

TUCSON, Ariz., March 17, 2020 — The Critical Path Institute (C-Path) today announced it has been awarded a grant to conduct stakeholder engagement to garner insights, feedback and refinement of the U.S. Food and Drug Administration (FDA) Center for Devices and Radiological Health’s (CDRH) proposed framework to enhance the pediatric medical device ecosystem and to develop a strategic plan for implementation. Funded by a cooperative agreement through the FDA, C-Path will work in collaboration with CDRH and other stakeholders to organize a workshop to brainstorm on the framework and strategic plan for building a viable ecosystem.

“We are thrilled to be part of this effort and honored to have been selected to work with CDRH to optimize the ecosystem,” said C-Path President and CEO Joseph Scheeren, Pharm.D. “This initiative has our full support and fits in well with our ongoing efforts in the pediatric and neonatal space.”

Currently, there are challenges to advancing promising new pediatric devices from ideation to clinical studies, to regulatory approval, to use for pediatric patients, including:

  • The small numbers of children with any one condition, and their dispersion across the world, making it difficult to conduct clinical trials;
  • The need to consider children’s growth and variably influenced disease mechanisms;
  • The lengthy development timeline and associated high costs;
  • Lack of funding for small inventors and companies;
  • Difficulty in identifying expert resources across the development-to-commercialization continuum.

Modernized regulatory programs and processes developed by CDRH during the past few years have supported a growing number of novel technologies available to improve patient care. In the pediatric area, CDRH has led a public meeting where challenges to innovation were verified and aspects of a supportive ecosystem were discussed. Additionally, CDRH has finalized guidance documents clarifying regulatory issues relevant to pediatric populations and provided millions of dollars to fund pediatric device consortia across the U.S. However, over the past decade, fewer than 10% of high-risk and high-benefit medical devices have been designed, evaluated and labelled for children below the age of 18.

“Today’s health care system has yet to engender a solution to the complex public health issue of medical device development for children and small populations,” said CDRH Chief Medical Officer and Director for Pediatrics and Special Populations Vasum Peiris, M.D., M.P.H. “Novel regulatory options and public-private sector collaboration informed by patient and caregiver perspectives are necessary components to create a sustainable national environment for safe technology innovation that serves the unique needs of children. We welcome input from all stakeholders as we clarify and build on the framework CDRH has proposed to transform traditional thinking around engaging, sustaining and innovating in the pediatric medical device ecosystem.”

Stakeholder groups including the Advanced Medical Technology Association (AdvaMed), the American Academy of Pediatrics (AAP), the Milken Institute, and clinicians, researchers, innovators, payors and patient groups will review and refine the high-level proposed framework developed by CDRH. The framework lays out a vision for an ecosystem that would facilitate the design, development and commercialization of medical devices for children. Through stakeholder input and feedback the framework will be iterated and refined, and goals for the yet to-be-formed multi-stakeholder workgroup – whose objective would be to optimize and complete the design of the ecosystem – will be established.

“There is an opportunity for the FDA, patient advocacy groups, academia and industry to collaborate and optimize design — with the guidance and support of C-Path — of this much needed ecosystem,” said AdvaMed Vice President for Technology and Regulatory Affairs Tara Federici. “Stakeholders will review and evaluate the proposed framework to ensure it could be effective in de-risking and accelerating pediatric medical device development while maintaining existing safety and effectiveness requirements.”

“Children are not small adults — their biology is different. Even so, every day across the nation, the care of children requires the use of many medical devices that were not specifically designed for them,” said Kurt Newman, M.D., President and CEO of Children’s National Hospital and immediate past Chair of the Board of Trustees of Children’s Hospital Association. “Children should be a priority when it comes to healthcare innovation because entire lifetimes are at stake. We commend and support the FDA’s leadership in this effort to create a system that supports innovation for children.”

“Pediatricians often find themselves without the medical or surgical devices they need to care for their patients because medical devices for children can lag five to ten years behind those for adults,” said American Academy of Pediatrics CEO and Executive Vice President Mark Del Monte, J.D. “That time frame is most of an entire childhood. We must do more to get children the devices they need sooner, and innovation is essential to enhance safe and effective options. We applaud those who have come together with that goal in mind and look forward to working with the FDA, C-Path, and other stakeholders to advance device development for children.”

“It is essential to shift the balance of device innovation toward fulfilling the vast unmet needs of children,” said Christopher Lee, former Director of the Milken Institute Center for Financial Markets and co-chair of the FasterCures BRIDGE Initiative, a recently launched program that develops novel models for driving biomedical innovation. “This proposed framework seeks to take this challenge head on by realigning incentives across the system, and we look forward to contributing to that process.”

The project’s initial deliverable, to bring together interested stakeholders to share insights and further refine and develop the proposed framework at the workshop, will occur in the latter part of this year. Specific dates, location, and registration information for the meeting will be released soon. Meeting proceedings and a strategic development plan to enhance the pediatric medical device ecosystem will be developed within six months of the workshop.

This grant is funded by a cooperative agreement through the FDA [Critical Path to Public Private Partnerships Grant Number U18 FD005320]; the grant is administered by the Center for Drug Evaluation and Research (CDER); this project is funded by the Center for Devices and Radiological Health (CDRH). Views expressed in written materials or publications do not necessarily reflect the official policies of the U.S. Department of Health and Human Services; nor does any mention of trade names, commercial practices, or organization imply endorsement by the United States Government.


 

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


 

About AdvaMed
AdvaMed member companies produce the medical devices, diagnostic products and health information systems that are transforming health care through earlier disease detection, less invasive procedures and more effective treatments. AdvaMed members range from the largest to the smallest medical technology innovators and companies. For more information, visit advamed.org.

 


 

About the American Academy of Pediatrics
The American Academy of Pediatrics (AAP) is an organization of 67,000 primary care pediatricians, pediatric medical subspecialists and pediatric surgical specialists dedicated to the health, safety and well-being of infants, children, adolescents and young adults. For more information, visit aap.org and follow us on Twitter @AmerAcadPeds

 


 

About Milken Institute
The Milken Institute is a nonprofit, nonpartisan think tank that helps people build meaningful lives, in which they can experience health and well-being, pursue effective education and gainful employment, and access the resources required to create ever-expanding opportunities for themselves and their broader communities. FasterCures is a center of the Milken Institute, with a mission to put patients in the center of the healthcare system, and break down the unnecessary barriers to innovation. For more information, visit milkeninstitute.org.


 

About FDA
The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

 

Contact:

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

C-Path Appoints Dr. Klaus Romero as Chief Science Officer

 

 

TUCSON, Ariz., March 12, 2020 — The Critical Path Institute (C-Path) today announced the appointment of Klaus Romero, M.D., M.S., F.C.P., as its new Chief Science Officer (CSO) effective April 1, 2020. Dr. Romero, a clinical pharmacologist and epidemiologist with more than 17 years combined experience in academic and pharmaceutical clinical research, translational sciences, pharmacometrics, modeling and simulation and pharmacoepidemiology, has been with C-Path since December 2007. His most recent role has been as Executive Director of Clinical Pharmacology and Quantitative Medicine. He will continue in dual roles as CSO and head of the Quantitative Medicine Program.

As CSO, Dr. Romero will spearhead innovations in research and science by delivering strategic and operational leadership of C-Path’s data-driven nationally and internationally recognized programs in medical product development solutions. In collaboration with senior leadership, he will set the vision for C-Path’s scientific endeavors and ensure that the focus of C-Path’s public-private partnerships stay closely aligned with its mission to accelerate the pace and reduce the costs of medical product development, through innovation in data science, model-informed drug development, patient-centric drug development and precision medicine to optimize the scientific evaluation of the efficacy and safety of new therapies. This will be achieved through strengthening C-Path’s pillars in quantitative medicine, data science, biomarkers, clinical outcome assessment tools and regulatory science.

“With more than 15 years of diverse experience in the drug development space, including demonstrated leadership abilities in heading large programs and cross-functional teams, there are few people who would be equally suited to lead C-Path’s scientific initiatives than Klaus,” said C-Path President and CEO Joseph Scheeren, Pharm. D. “His combination of clinical pharmacology, epidemiology and model and simulation experience, plus his business development expertise and history of fostering meaningful interactions between regulatory agencies, industry, academia and patient groups will be key in enhancing signature C-Path consortia initiatives, while bringing strategic focus to data analytics to drive research and create efficiencies across all stages of drug development.”

During his tenure with C-Path, he has helped lead clinical pharmacology, pharmacoepidemiology and modeling and simulation projects in Alzheimer’s disease, polycystic kidney disease (PKD), tuberculosis, type 1 diabetes, Parkinson’s disease, Duchenne muscular dystrophy, kidney transplantation, Huntington’s disease and cardiovascular drug safety. His work has helped to achieve major milestones, including the first regulatory endorsement by the U.S. Food and Drug Administration and European Medicines Agency of a clinical trial simulation tool for mild and moderate Alzheimer’s disease and the regulatory qualification of the first imaging biomarker for PKD. Dr. Romero’s scientific production with C-Path has resulted in more than 60 peer-reviewed publications related to his work with the Institute.

Reporting directly to Scheeren, Dr. Romero will serve as the Institute’s spokesperson on all scientific matters.

“I am incredibly excited and honored to have been given the opportunity to further serve C-Path in this role. I am proud of our 15-year history, as we have grown from a small group of pioneers into a mature organization of innovators in solutions to expedite medical product development,” said Dr. Romero. “I look forward to advancing and growing our pipeline of collaborative pre-competitive efforts. The era of knowledge management, decision science and personalized medicine represents the right time for C-Path to grow its leadership in transforming smart data into actionable cutting-edge knowledge to de-risk and expedite medical product development.”

Dr. Romero is a fellow of the American College of Clinical Pharmacology, a founding member of the International Society of Pharmacometrics, as well as a member of the American Society for Clinical Pharmacology and Therapeutics, and the International Society for Pharmacoepidemiology. In addition to his duties at C-Path, he serves as Chairman of the Board of Directors for CredibleMeds®. He is fluent in English, Spanish, German and Portuguese, and has a passion for music, designing and playing innovative electric guitars. Dr. Romero is also a Research Associate Professor at the University of Arizona College of Medicine, Adjunct Professor at the College of Health Solutions at Arizona State University, Adjunct Professor at the University of Southern California’s School of Pharmacy and serves on the Scientific Board of Pharos Dx. Dr. Romero received his medical degree from Pontifical Xavierian University, completed his training in Clinical Pharmacology at Columbia National University and holds an MS degree in Epidemiology from the Columbia School of Medicine.

Dr. Romero succeeds C-Path CSO Lynn Hudson, Ph.D., who will retire at the end of March; a position she has held since 2011. Under Hudson’s leadership, C-Path’s scientific consortia and programs have more than doubled.

“It has been an honor to serve as C-Path’s scientific leader for the past decade,” Hudson said. “C-Path continues to reach important milestones and achieve great progress in creating drug development efficiencies that have changed the time and expense it takes to get safe medical products to those who need them. I am incredibly proud of the work done here and I know Klaus will be a key player in C-Path’s future success.”


 

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

 

Contact:

Kissy Black
615.310.1894
kissyblack@c-path.org

C-Path Selects Experienced Neuroscientist to Lead Alzheimer’s Consortium

 

 

TUCSON, Ariz., March 12, 2020 — The Critical Path Institute (C-Path) today announced it has named Sudhir Sivakumaran, Ph.D., as Executive Director of its Critical Path for Alzheimer’s Disease (CPAD) consortium effective immediately. Dr. Sivakumaran, a neuroscientist and research and development professional, has nearly 20 years combined experience in academic, pre-clinical and early clinical research and development, phase 1 and phase 2 clinical study design, data analysis and diligence and business development. He has been with C-Path since July 2019 as Associate Director for CPAD.

As Executive Director of CPAD, Dr. Sivakumaran will guide all operational and scientific input for the CPAD Consortium and develop and implement the strategic plan and priorities for individual working groups within CPAD. He will work with key individuals at global regulatory agencies including the U.S. Food and Drug Administration, European Medicines Agency and the Pharmaceuticals and Medical Devices Agency. Dr. Sivakumaran will also provide scientific leadership and oversee projects dealing with neuroscience, biomarker qualification, clinical outcome assessment development, data sharing and aggregation related to utility of modeling and simulation in Alzheimer’s disease (AD).

“We are thrilled that Sudhir has accepted the role of Executive Director of our long-standing Alzheimer’s consortium, CPAD,” said C-Path President and CEO Joseph Scheeren, Pharm. D. “He brings a wealth of experience to the program and in the short time Sudhir has been with C-Path, he has stepped up to continue to drive CPAD’s initiatives to create new tools and methods to increase the efficiency in the development process of new treatments for AD and related neurodegenerative disorders. With Sudhir leading CPAD, I know we’ll continue to enhance our already ample collaborative efforts with our agency, industry and patient organization partners in this area.”

Dr. Sivakumaran previously held positions as Associate Director, Research Management at Aptinyx, Inc. in Greater Chicago and Senior Manager, External Partnerships & Alliances, Neuroscience at Pfizer, Inc. in Cambridge, Mass. He has published more than a dozen peer-reviewed publications and was the recipient of multiple research grants and awards including the Epilepsy Foundation & American Epilepsy Society Post-doctoral Training Fellowship, Integrated Pilot Award from BU-CTSI, Academy of Finland Research Grant and the CURE Young Investigator Travel Award among others. He speaks English, Tamil, and Italian and his interests include traveling the world, long road trips and exploring and enjoying world cuisine. Dr. Sivakumaran holds a doctorate in neuroscience from SISSA/ISAS in Trieste, Italy and an M.Sc. and B.Sc. in microbiology from Univ. of Madras in India.

“I am very excited to be at C-Path and for the opportunity to lead the CPAD consortium,” said Dr. Sivakumaran. “Alzheimer’s disease is globally one of the most devastating diseases for millions of people and their loved ones. We are at a game changing moment in finding meaningful and effective therapeutic options in AD. An organization like C-Path, and its consortium CPAD, provides a great opportunity for us to come together within a neutral, pre-competitive environment in partnership with industry experts, regulatory agencies and patient organizations and work towards finding effective solutions for the most pressing needs and de-risk the drug development process in AD. I look forward to continuing to work with everyone at C-Path and beyond, towards refining and redefining our future course of action in AD drug development.”

Reporting directly to Scheeren, Dr. Sivakumaran will serve on C-Path’s leadership team, which is responsible for the overall strategic direction of the Institute.


 

About CPAD
CPAD’s mission is to accelerate the drug development process for patients with chronic neurodegenerative disease leading to dementia. Its primary focus is on AD. CPAD works with industry, regulatory authorities, and patient advocacy organizations to advance Drug Development Tools for evaluating drug efficacy and safety, to optimize novel clinical trial designs, and aggregating anonymized patient-level data using CDISC consensus standards to facilitate the regulatory review process.

CPAD has the following areas of pre-competitive focus: (1) regulatory qualification of biomarkers (2) development of CDISC data standards for AD endpoint assessments, (3) creation of integrated databases for observational and clinical trials data, and (4) development of quantitative model-based tools for drug development.

 

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

 

Contact:

Kissy Black
615.310.1894
kissyblack@c-path.org

ERA4TB, an International Consortium to Accelerate the Development of Comprehensive Treatments Against Tuberculosis

C-Path, Ltd. and more than 30 partners from 13 countries participate in the ERA4TB project.

The ERA4TB (European Regimen Accelerator for Tuberculosis) project is a public-private initiative dedicated to the development of drugs against tuberculosis. With a team of more than thirty organizations and a budget of over 200 million euros ERA4TB will focus on developing a new, improved tuberculosis treatment. The partners will share their expertise, knowledge and resources to rapidly progress new candidate drugs into clinical trials.

Tuberculosis is the leading cause of death by an infectious disease worldwide. According to the World Health Organization (WHO), an estimated 10 million people became ill with tuberculosis in 2018, and 1.6 million died. Even though the incidence of tuberculosis is declining, the drug-resistant form constitutes a growing threat to the safety of the world population. It is in this spirit that the UN has pledged to end the tuberculosis epidemic by 2030 through joint action of its member states.

David Barros-Aguirre, Project Lead for ERA4TB and VP and Head of Tuberculosis Research, Global Health R&D, GSK said: “On behalf of the ERA4TB partners, we are very excited about the start of this unique collaborative project in the field of tuberculosis where the expertise from public partners and a pipeline of promising preclinical candidates from pharmaceutical companies merge to accelerate the development of novel clinical candidates. The goal is to deliver an innovative and differentiated combination regimen for the treatment of TB, which can play a key role in the elimination agenda.”

Standard tuberculosis treatment is based on a combination regimen of four drugs that were all developed more than 60 years ago. Treatment lasts for at least six months and, in the case of resistance to the standard drugs, can be as long as two years. The current drugs are inefficient by today’s standards and a new, faster-acting and safer treatment is required to reduce the length of therapy and to overcome the menace of drug-resistant strains. Until now, the development of new drugs has been slow and their incorporation into tuberculosis treatment regimens conducted in a sequential manner.

Stewart Cole, Scientific leader of ERA4TB and President of the Institut Pasteur, said “ERA4TB has assembled an impressive array of resources to seamlessly harness the agility and innovation of academia with the pragmatism and scientific expertise of pharma. I am confident that this powerful European initiative will speed the path to TB elimination.”

ERA4TB is set to change the paradigm of tuberculosis treatment development by abandoning the sequential approach in favor of a parallel pathway, which will allow the simultaneous investigation of more than a dozen drug candidates. By implementing a standardized approach to tuberculosis drug development, that is well coordinated with the collaborations outside Europe, ERA4TB has the potential to optimize, and, more importantly, greatly reduce the development times of the new regimens needed to eliminate this epidemic.

According to Juan José Vaquero, project coordinator from UC3M (University Carlos III Madrid) and leader of the imaging team, “ERA4TB will change the paradigm for the development of new TB drugs by adopting a flexible and modular collaboration scheme in which research efforts can be rapidly mobilized to where they are most needed allowing several molecules to be investigated simultaneously. This will save both time and money.”

ERA4TB is sponsored by the European Union’s Horizon 2020 research and innovation programme and the European pharmaceutical industry (via EFPIA) under the auspices of the Innovative Medicines Initiative 2 Joint Undertaking and part of the AMR Accelerator umbrella.

About the Innovative Medicines Initiative

The Innovative Medicines Initiative (IMI) is Europe’s largest public-private initiative aiming to speed up the development of better and safer medicines for patients. IMI supports collaborative research projects and builds networks of industrial and academic experts in order to boost pharmaceutical innovation in Europe. IMI is a joint undertaking between the European Union and the European Federation of Pharmaceutical Industries and Associations, EFPIA. More information can be found at www.imi.europa.eu.

Acknowledgement

This project has received funding from the Innovative Medicines Initiative 2 Joint Undertaking (JU) under grant agreement No 853989. The JU receives support from the European Union’s Horizon 2020 research and innovation programme, and in-kind support from EFPIA, TB Alliance, Bill & Melinda Gates Foundation and University Of Dundee.

 

Disclaimer

This communication reflects the views of the ERA4TB Consortium and neither IMI nor the European Union and EFPIA are liable for any use that may be made of the information contained herein.

 

CONTACTS:

Project Leader: David Barros-Aguirre
Project Coordinator: Juan Jose Vaquero Lopez
Scientific Leader: Stewart Cole