C-Path Opens Access to Duchenne Regulatory Science Consortium Database

Database will allow sharing of individual-level data with the goal of accelerating therapy development for Duchenne muscular dystrophy

TUCSON, Ariz., April 22, 2021 — Critical Path Institute (C-Path) announced today that it will open access to the Duchenne Regulatory Science Consortium (D-RSC) database to qualified researchers, through its Rare Disease Cures Accelerator, Data and Analytics Platform (RDCA-DAP®). The D-RSC database includes data from Duchenne muscular dystrophy (DMD) clinical trials, natural history studies and clinical data collections. The contributors of these datasets have given permission for this data to be shared externally to accelerate therapy development for DMD.

DMD is a rare, fatal, genetic neuromuscular disorder that is diagnosed in childhood and primarily affects males. It causes progressive muscle loss, resulting in the loss of the ability to walk, loss of upper body strength, progressive breathing issues, cardiomyopathy and premature death. Although there are medical treatments that may help slow its progression, there is currently no cure for DMD.

C-Path’s RDCA-DAP provides a centralized and standardized infrastructure to support and accelerate rare disease characterization, with the goal of accelerating therapy development across all rare diseases. By sharing this valuable data resource through RDCA-DAP, D-RSC aims to extend and amplify the availability of data to accelerate therapy development for DMD, foster the development of tools to accelerate clinical trials for DMD and help develop a deeper understanding of DMD natural history.

“Individuals living with Duchenne are in urgent need of effective treatments,” explained C-Path’s Chief Science Officer Klaus Romero, M.D., M.S., F.C.P. “With the strength in numbers of data available in the D-RSC database, we believe that opening this platform and making data available to qualified researchers will have a profound effect on expediting the drug development process.”

All data in the D-RSC database have been mapped to current Clinical Data Interchange Standards Consortium (CDISC) Standard Data Tabulation Model (SDTM) structures. Data contained in the D-RSC database are fully de-identified and datasets are integrated, so researchers will not be able to identify participants of the studies and data can be used in aggregate.

D-RSC was formed through collaboration between the Critical Path Institute and Parent Project Muscular Dystrophy. Individuals and companies from industry and academia are members of the consortium, and representatives of the U.S. Food and Drug Administration, European Medicines Agency and the National Institutes of Health have joined the consortium coordinating committee as observers.

Researchers interested in contributing data to D-RSC and/or utilizing the database to advance DMD-related research initiatives can learn more at: https://c-path.org/programs/d-rsc/overview/database-access/ or contact D-RSC Associate Director Ramona Belfioire-Oshan, Ph.D., at rbelfioreoshan@c-path.org.

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


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

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

C-Path’s Alzheimer’s Disease Consortium Expands Data Repository

New patient-level data will facilitate more efficient clinical trial design

TUCSON, Ariz., April 12, 2021 — The Critical Path Institute’s (C-Path) Critical Path for Alzheimer’s Disease (CPAD) Consortium today announced that it is significantly expanding its Alzheimer’s disease (AD) patient-level data repository with high-quality contemporary industry clinical trial datasets, focusing on early stages of the disease.

Generous data contributions and support from the pharmaceutical industry have made it possible for the CPAD team to integrate 42 high-quality clinical trial and observational studies in AD, containing more than 21,000 patient-level records.

Since July 2019, CPAD has signed Data Contribution Agreements (DCAs) with data contributors, for five highly relevant datasets, representing over 4,000 patient-level records, which have been integrated into the CPAD repository. CPAD also expects the transfer of two additional datasets with another 4,000 patient-level records. In addition, CPAD is currently negotiating DCAs with five CPAD member pharmaceutical companies, with an expected addition of nine datasets, bringing in more than 10,000 additional patient-level records to the CPAD repository.

“Serving as a neutral third party, CPAD provides a safe harbor for individual organizations to share data in a pre-competitive environment,” said Sudhir Sivakumaran, Ph.D., Vice President, Neuroscience at C-Path and Executive Director of CPAD. “By facilitating cross-functional, pre-competitive collaboration and active engagement between the regulatory agencies and drug development professionals, we can make a significant difference in the bench to bedside timeline for patients.”

The expanded patient-level data will allow generation of novel drug development tools (DDTs) and solutions such as disease progression models across the entire continuum of AD, incorporating various fluid and imaging biomarkers, in addition to genotype, cognitive and functional outcome assessments and demographics. The novel tools and solutions will enable a more efficient design of clinical studies with better informed inclusion criteria, endpoint selection and patient enrichment strategies. C-Path’s quantitative approach allows for a comprehensive integration of relevant patient characteristics for informed and efficient decision‐making in drug development.

Data sharing is fundamental to enable the development of novel DDTs, which require integration and aggregation of patient-level data from multiple independent sources and studies. Successful data sharing for the purpose of seeking regulatory endorsement of a DDT relies on extensive partnerships between industry, academia, research institutes and societies, patient-advocacy groups, and governmental regulatory agencies. “CPAD’s successes and accomplishments in the last decade is testimony to the extensive collaboration, partnership, and support of the industry members,” Sivakumaran continued. “The consortium seeks to continue accelerating drug development by increased data sharing and ultimately, be able to successfully address the key unmet needs and unanswered questions including the voice of the patients.“

“CPAD provides the AD field with a unique opportunity to transform patient-level data into actionable solutions, which can in turn transform the drug development process for Alzheimer’s disease,” said C-Path Chief Science Officer and Executive Director for Clinical Pharmacology Klaus Romero, M.D., M.S., F.C.P. “The unique way in which CPAD presents such solutions for formal regulatory review generates the necessary confidence for the adoption of such solutions by our industry partners.”

Data acquisition and integration is a critical prerequisite for the development of novel regulatory-grade quantitative drug development solutions. For more than a decade, CPAD has led the space with regulatory successes and expertise in data aggregation and generation of novel drug development tools. The consortium continues to pursue efforts to expand its datasets, which will significantly add to the increased understanding of disease progression and pathology in AD.

For more information, visit https://c-path.org/programs/cpad/. To learn more about data contribution to the repository, email info@c-path.org. To access the Critical Path Institute (C-Path) Online Data Repository (CODR) go to https://codr.c-path.org/main/applyDatabaseSelection.html

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

 


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

 

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

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

 

Watch now: RDCA-DAP and EATRIS Present Rare Disease Drug Develoment Webinar

January 21, 2021 | 5:00 p.m. – 6:30 p.m. CET
Data Sharing and Analysis to Support Rare Disease Drug Development

The European infrastructure for translational medicine, EATRIS, has invited the Rare Disease Cures Accelerator-Data and Analytics Platform team to present as part of its webinar series, EATx, to introduce RDCA-DAP to the European academic and industry audience. The webinar will discuss how RDCA-DAP works with European entities and how data sharing within Europe, and between Europe and other countries, can be achieved with RDCA-DAP.

RDCA-DAP EATRIS Live Webinar Q&A

C-Path and Diamyd Medical Announce Data Sharing Collaboration to Develop Advanced Drug Development Tools in Type 1 Diabetes

 

TUCSON, Ariz., and STOCKHOLM, Sweden. December 16, 2020 — The Critical Path Institute (C-Path) and Diamyd Medical (DMYD B; Nasdaq First North Growth Market) are proud to announce their collaboration to significantly improve the scientific community’s insight into type 1 diabetes (T1D) through Diamyd Medical’s contribution of fully anonymized data from a European Phase III trial to the Trial Outcome Markers Initiative (TOMI-T1D) integrated database. The Phase III trial evaluated the use of the diabetes vaccine Diamyd®, an antigen-specific immunotherapy based on the auto-antigen GAD (glutamic acid decarboxylase), to induce immunological tolerance and stop the autoimmune destruction of insulin producing cells. The Data Contribution Agreement (DCA) between Diamyd Medical and C-Path will allow for this unique set of fully anonymized clinical trial data to be integrated into an ever-growing list of committed trial data sets within the TOMI-T1D project.

TOMI-T1D is an international partnership between academia, the pharmaceutical industry and nonprofit organizations. It is funded by the world’s leading charities dedicated to diabetes research, JDRF, and Diabetes UK, guided by both organizations’ strong commitment to facilitate deep interrogation of consolidated community-wide trial data as a means to accelerate clinical research and therapeutic development for T1D. TOMI-T1D aims to create a clinical trial simulation tool (CTST) with large and diverse clinical datasets from the T1D community. The project also seeks to engage the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) to identify opportunities for regulatory endorsement of such drug development tools.

The Diamyd Medical data includes relevant information about disease progression, drug effects and clinical trial design. Contribution of these robust data sets from industry led trials is critical to TOMI-T1D’s work in developing innovative and quantitative tools that can facilitate clinical development efforts and be endorsed by regulators for future use by the pharmaceutical industry to optimize the design of future clinical trials.

“Progress towards the establishment of approved therapies for people with T1D is critically reliant on participation from our partners in industry with their data,” said Simi Ahmed and Elizabeth Robertson, on behalf of the charity partnership. “This is indeed a right step in that direction,” said Colin Dayan, lead PI at Cardiff University.

“We are thrilled that Diamyd Medical is taking a leading role and championing precompetitive collaborations advancing type 1 diabetes regulatory science solutions,” said C-Path Executive Director Inish O’Doherty, Ph.D. “Their data will help in the construction and evaluation of a clinical trial simulation tool to assist in the development of novel therapies for type 1 diabetes patients.”

“We are very honored to be part of this important collaboration -involving key stakeholders within the T1D landscape,” said Ulf Hannelius, President & CEO of Diamyd Medical. “As we are moving into an era of precision medicine in type 1 diabetes, we can expect to see significant therapeutic advances, and access to high quality data will be integral to maximizing these efforts.”

To learn more about the TOMI-T1D project visit: https://c-path.org/programs/tomi-t1d/

TOMI-T1D is solely funded by JDRF and Diabetes UK. Critical Path Institute is supported by the U.S. Food and Drug Administration (FDA) of the U.S. Department of Health and Human Services (HHS) and is 69% funded by FDA/HHS, totaling $19,471,171, and 31% funded by non-government source(s), totaling $8,612,313. 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 the Organizations:

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.


 

Diamyd Medical develops therapies for type 1 diabetes. The diabetes vaccine Diamyd® is an antigen-specific immunotherapy for the preservation of endogenous insulin production. Significant results have been shown in a genetically predefined patient group in a large-scale metastudy as well as in the Company’s European Phase IIb trial DIAGNODE-2, where the diabetes vaccine is administered directly into a lymph node in children and young adults with newly diagnosed type 1 diabetes. A new facility for vaccine manufacturing is being set up in Umeå for the manufacture of recombinant GAD65, the active ingredient in the therapeutic diabetes vaccine Diamyd®. Diamyd Medical also develops the GABA-based investigational drug Remygen® as a therapy for regeneration of endogenous insulin production and to improve hormonal response to hypoglycaemia. An investigator-initiated Remygen® trial in patients living with type 1 diabetes for more than five years is ongoing at Uppsala University Hospital. Diamyd Medical is one of the major shareholders in the stem cell company NextCell Pharma AB. Diamyd Medical’s B-share is traded on Nasdaq First North Growth Market under the ticker DMYD B. FNCA Sweden AB is the Company’s Certified Adviser; phone: +46 8-528 00 399, e-mail: info@fnca.se.


 

JDRF‘s mission is to accelerate life-changing breakthroughs to cure, prevent, and treat T1D and its complications. To accomplish this, JDRF has invested more than $2.5 billion in research funding since our inception. We are an organization built on a grassroots model of people connecting in their local communities, collaborating regionally for efficiency and broader fundraising impact and uniting on a national stage to pool resources, passion and energy. We collaborate with academic institutions, policymakers and corporate and industry partners to develop and deliver a pipeline of innovative therapies to people living with T1D. Our staff and volunteers throughout the United States and our five international affiliates are dedicated to advocacy, community engagement and our vision of a world without T1D. For more information, please visit jdrf.org or follow us on Twitter: @JDRF.


 

1.   Diabetes UK’s UK’s aim is creating a world where diabetes can do no harm. Diabetes is the most devastating and fastest growing health crisis of our time, affecting more people than any other serious health condition in the UK – more than dementia and cancer combined. There is currently no known cure for any type of diabetes. With the right treatment, knowledge and support people living with diabetes can lead a long, full and healthy life. For more information about diabetes and the charity’s work, visit www.diabetes.org.uk.
2.   Diabetes is a condition where there is too much glucose in the blood because the body cannot use it properly. If not managed well, both type 1 and type 2 diabetes can lead to devastating complications. Diabetes is one of the leading causes of preventable sight loss in people of working age in the UK and is a major cause of lower limb amputation, kidney failure and stroke.
3.   People with type 1 diabetes cannot produce insulin. About 10 per cent of people with diabetes have type 1. No one knows exactly what causes it, but it’s not to do with being overweight and it isn’t currently preventable. It’s the most common type of diabetes in children and young adults, starting suddenly and getting worse quickly. Type 1 diabetes is treated by daily insulin doses – taken either by injections or via an insulin pump. It is also recommended to follow a healthy diet and take regular physical activity.
4.   People with type 2 diabetes don’t produce enough insulin or the insulin they produce doesn’t work properly (known as insulin resistance). Around 90 per cent of people with diabetes have type 2. They might get type 2 diabetes because of their family history, age and ethnic background puts them at increased risk. They are also more likely to get type 2 diabetes if they are overweight. It starts gradually, usually later in life, and it can be years before they realise they have it. Type 2 diabetes is treated with a healthy diet and increased physical activity. In addition, tablets and/or insulin can be required.

For more information on reporting on diabetes, download our journalists’ guide: Diabetes in the News: A Guide for Journalists on Reporting on Diabetes (PDF, 3MB).

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

 

C-Path Selects Aridhia to Support Rare Disease Cures Accelerator-Data and Analytics Platform

 

TUCSON, Ariz., December 3, 2020 — The Critical Path Institute (C-Path) today announced it has selected Aridhia to support its Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP). The RDCA-DAP initiative, launched in September 2019 as a collaborative project between C-Path and the National Organization for Rare Disorders® (NORD), will provide a centralized and standardized infrastructure to accelerate and optimize the quantitative characterization of rare diseases, with the goal of accelerating therapy development. RDCA-DAP is designed to accept patient-level rare disease data from clinical trials, observational studies, real-world data, patient registries and other sources, to support the analysis and interpretation of those data.

Based in Scotland, Aridhia encompasses a multidisciplinary team of data scientists, information governance specialists, computer scientists, software developers and health care experts who are uniquely skilled to give researchers and innovators the ability to discover and understand data through dataset searching, classification and efficient metadata browsing capabilities. Aridhia will work closely with C-Path’s Data Collaboration Center (DCC) team of top-tier technical, scientific and project management experts to build a neutral, precompetitive data collaboration environment to support advanced research efforts in rare diseases.

“We look forward to working with Aridhia on this exciting collaboration,” said C-Path Executive Director of Data Science Amanda Borens, MS. “RDCA-DAP is an important resource for the entire rare disease community. Using Aridhia technology, RDCA-DAP will help the community come together and share data and knowledge that will revolutionize the development of therapies for rare diseases.”

Aridhia was chosen by C-Path based on its unparalleled expertise in providing secure cloud-enabled digital research environments for biomedical, precision medicine and health care researchers. Coupled with the fact that Aridhia has been on the forefront of the fair, lawful and transparent handling of personal data — prior to the implementation of the EU’s General Data Protection Regulations (GDPR) — and have continued to be an industry leader in the strict adherence to GDPR standards, Aridhia was the stand-out choice to assist with the development of the RDCA-DAP.

“We’re delighted to be working with colleagues at C-Path on such an important area of biomedical research,” said Aridhia CEO David Sibbald. “We know that improving therapy development for rare disease requires significant data discovery and data access, while preserving the governance requirements of individual data contributors. Our collaboration will help the rare disease scientific community gain access to richer, more comprehensive datasets to accelerate rare disease therapy development.”

RDCA-DAP already includes integrated rare disease data from a number of different sources — including those within NORD’s IAMRARE™ registry platform — and building in advanced analytics capabilities will allow efficient and effective interrogation of that data to generate solutions for clinical trial design and regulatory review.

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

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 69% funded by FDA/HHS, totaling $19,471,171, and 31% percent funded by non-government source(s), totaling $8,612,313. 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 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 Aridhia
Aridhia is a health data science company founded in 2008 and based in Glasgow’s Queen Elizabeth University hospital and central Edinburgh. Aridhia provides a cloud based digital research platform enabling both the discovery and analysis of data to take place in a trusted, secure environment at scale. Our multidisciplinary team of data scientists, information governance specialists, computer scientists, software developers and health care experts delivers research services across the UK and Europe, working collaboratively with the NHS, research organizations, life sciences companies, IT service providers and patients to transform clinical research into clinical practice.

 

Contact:

Kissy Black
C-Path
kissyblack@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

Alzheimer’s Disease Data Initiative Launches, Critical Path for Alzheimer’s Disease Consortium to Partner

 

CPAD will partner with ADDI to build better tools for researchers around the world to access, use and share dementia-related data to accelerate the development of new and more effective treatments for Alzheimer’s disease

TUCSON, Ariz., November 17, 2020 — The Critical Path Institute’s (C-Path) Critical Path for Alzheimer’s Disease (CPAD) Consortium today announced its participation as a Partner with the Alzheimer’s Disease Data Initiative (ADDI), a 501(c)(3) medical research organization which launched today. ADDI is dedicated to advancing scientific breakthroughs in the treatment of Alzheimer’s disease (AD) and related dementias.

“ADDI will be a game-changing collaboration to provide the necessary data accessibility to advance data-driven solutions to accelerate Alzheimer’s disease drug development,” said C-Path’s Chief Science Officer Klaus Romero, M.D., M.S., F.C.P. “C-Path is honored to be a key Partner with ADDI, through CPAD, and contribute our data science, model-informed drug development and regulatory science expertise to ensure the success of ADDI.”

CPAD’s integrated and standardized patient-level database comprised of randomized clinical trials from industry, which is currently available to qualified researchers across the globe, will be a relevant and crucial component of ADDI’s Alzheimer’s Disease Workbench. This will enable researchers in AD and related dementias around the world to interact with relevant data resources more efficiently and accelerate the generation of actionable scientific insights leading to innovative solutions, diagnostic tools and therapies in AD.

Emphasizing the expected impact of ADDI, CPAD’s Executive Director Sudhir Sivakumaran, Ph.D., said, “ADDI is uniquely positioned to maximize the value of each data point contributed by individuals living with AD, who participate in clinical trials, observational studies, patient registries and other research efforts.”

CPAD’s Industry Co-Director and Vice President, Clinical Research, Neurology Business Group, Eisai, Inc. Michael C. Irizarry, M.D., M.P.H., added, “In this important initiative, the CPAD partnership with ADDI aims to facilitate increased aggregation and robust analysis of high-quality AD clinical research data, to model disease progression and help accelerate the development of biomarkers and therapeutics.”

For more information, visit: https://c-path.org/programs/cpad/; https://codr.c-path.org/main/applyDatabaseSelection.html; https://www.alzheimersdata.org/; https://www.alzheimersdata.org/ad-workbench.

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 62% funded by FDA/HHS, totaling $14,448,917, and 38% percent funded by non-government source(s), totaling $8,669,646. The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement by, FDA/HHS or the U.S. Government.

 


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

 

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

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

Biomarkers of Crohn’s Disease to Support the Development of New Therapeutic Interventions

C-Path’s Crohn’s Disease Biomarkers Pre-Consortium funded by The Helmsley Charitable Trust is proud to announce its first publication in the journal Inflammatory Bowel Diseases titled, “Biomarkers of Crohn’s Disease to Support the Development of New Therapeutic Interventions.” The result of collaboration among experts in Crohn’s disease, including academia, health care institutes, foundations, public advocacy groups, industry and regulatory bodies, this work has identified biomarkers that can be used as drug development tools to advance clinical trials in IBD and potentially improve patient care.

Free access: https://academic.oup.com/ibdjournal/advance-article/doi/10.1093/ibd/izaa215/5896885?guestAccessKey=70d1e3c0-2aaf-4d65-b1bb-292f7df3b625