C-Path ePRO Consortium and PRO Consortium Announce COVID-19 Risk Assessment and Mitigation Strategies

Recommendations provided for sponsors and eCOA providers to facilitate the continued collection of PRO data in clinical trials.

 

TUCSON, Ariz., April 29, 2020 — In collaboration with Critical Path Institute’s (C-Path) Patient-Reported Outcome (PRO) Consortium, the Electronic Patient-Reported Outcome (ePRO) Consortium announces “Coronavirus Disease 2019 (COVID-19): Risk Assessment and Mitigation Strategies for the Collection of Patient-Reported Outcome Data through Clinical Sites.” The presentation focuses on the current challenges of capturing PRO data originally intended to be collected electronically (i.e., ePRO) from study participants during in-person visits to clinical trial sites. Recommended risk assessment and mitigation strategies are provided for consideration by trial sponsors and electronic clinical outcome assessment (eCOA) providers to facilitate the continued collection of PRO data in clinical trials.

Due to concerns surrounding COVID-19, many patients are either unable or unwilling to travel to sites for scheduled visits or sites have had to close due to social distancing measures. Representatives of ePRO Consortium and PRO Consortium member firms were invited by C-Path to collaborate on the development of recommendations aimed at lessening the impact of the disruption on PRO data collection at clinical trial sites. Over a one-month period, member representatives participated in a series of teleconferences to discuss and debate approaches to assessing the situation and effectively responding to it. The objective of the resulting presentation is to provide recommended risk assessment and mitigation strategies for consideration by sponsors and eCOA providers to facilitate the continued collection of PRO data in clinical trials. Topics include regulatory considerations, licensing considerations when altering the mode of administration of a PRO measure, and reporting protocol changes to the institutional review board.

“These mitigation strategies have led us to rethink traditional clinical trial data collection approaches and to recognize that technology, which has become increasingly important in clinical research, is available to support decentralized trials now and in the future,” said Paul O’Donohoe, M.Sc., Scientific Lead, eCOA and Mobile Health at Medidata Solutions and Industry Vice Director of the ePRO Consortium.

“COVID-19 has impacted clinical trials in significant ways, and C-Path is proud to have supported the crucial conversations among eCOA providers and clinical trial sponsors that led to the roll out of strategies aimed to facilitate the continued collection of PRO data in clinical trials,” said Sonya Eremenco, M.A., Acting Director, ePRO Consortium. “These strategies reflect the collective effort of both consortia to work together for the greater good, ensuring patient safety in these uncertain times.”

*Funding for this press release was made possible, in part, by the Food and Drug Administration through grant U18 FD 005320. 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 C-Path’s Electronic Patient-Reported Outcome Consortium

The Electronic Patient-Reported Outcome (ePRO) Consortium was established by Critical Path Institute in 2011. Along with C-Path, the members of the ePRO Consortium are firms that provide electronic data collection technologies and services for capturing patient-reported outcome (PRO) and other clinical outcome assessment (COA) data in clinical trials. The mission of the ePRO Consortium is to advance the science of clinical trial endpoint assessment by collaboratively supporting and conducting research, designing and delivering educational opportunities, and developing and disseminating best practice recommendations for electronic collection of clinical outcome data.

 

About C-Path’s 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 Receives Letter of Support from EMA on Type 1 Diabetes Biomarker Initiative

 

 

TUCSON, Ariz., April 28, 2020 — The Critical Path Institute (C-Path) today announced that its Type 1 Diabetes (T1D) Consortium has received a letter of support from the European Medicines Agency (EMA) to facilitate the development and validation of the proposed regulatory qualification of pancreatic islet autoantibodies commonly used in clinical practice to diagnose T1D: insulin autoantibodies, glutamic acid decarboxylase 65, and insulinoma antigen-2 autoantibodies as enrichment biomarkers for T1D clinical trials.

In their response to the T1D Consortium Letter of Intent (LOI) and Briefing Package, the EMA stated, “[Therapies that preserve endogenous β-cell function and can prevent, halt or slow T1D disease progression in a clinically meaningful way would constitute a significant advancement in T1D care. If successful, the quantitative tools proposed by this Consortium have the potential to facilitate the streamlined design, execution, and review of clinical trials targeting this goal.].”

By the year 2050, the number of people diagnosed with T1D in the U.S. is projected to quadruple from an estimated 1.6 million in 2020 to 5 million in 2050. The ability to screen for subjects with early stages of T1D prior to the appearance of clinical symptoms is a valuable opportunity to potentially delay, and ultimately prevent, symptomatic T1D. The islet autoantibodies provide a means to identify patients at risk of progressing to a clinical diagnosis of T1D.

C-Path’s T1D Consortium will achieve the regulatory qualification of the islet autoantibodies currently used in clinical practice to diagnose T1D by employing the resources of all its members and engaging with regulatory agencies at each step of the process with funding and input from The Leona M. and Harry B. Helmsley Charitable Trust, Janssen Research & Development, LLC, JDRF International, Novo Nordisk A/S, and Provention Bio.

“JDRF is pleased the T1DC received a letter of support from EMA,” says Jessica Dunne, Ph.D., Senior Director, JDRF and Co-Director of the T1DC. “This is an example of collaboration between regulators and researchers, in the public and private sectors, working together to accelerate delivery of therapies into the hands of patients.”

This model-based qualification, will provide a tool endorsed by both the EMA and U.S. Food and Drug Administration (FDA) that utilizes islet autoantibody status, along with other relevant patient features, to identify and select patients with a likelihood of progressing to a T1D clinical diagnosis. This regulatory endorsement will provide sponsors with the confidence to use islet autoantibodies in the optimization of clinical trials evaluating novel therapies focused on the delay and/or prevent T1D.

“We are delighted that the EMA is strongly supporting the development of quantitative tools that can accelerate drug development in T1D,” said C-Path’s Executive Director of the T1D Consortium Inish O’Doherty, PhD. “This work has been enabled through the collaboration of the T1D community and the sharing of patient level data. We’re excited to move forward with this important project which will help pave the way for forthcoming therapies with the ability to treat early stages of T1D and delay or prevent the clinical, currently irreversible, stage of the disease.”

The consortium is currently working on the next regulatory milestones: the execution of the modeling analysis plan to inform the full Briefing Package for submission to the EMA and development of a Qualification Plan for submission to the FDA.

The Letter of Support can be found on the EMA website here or on the T1D Consortium website here.

 

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.

 


 

The Type 1 Diabetes (T1D) Consortium is a public-private partnership initiated in March 2017. Currently membership is composed of the following Industry and Foundation members: The Leona M. and Harry B. Helmsley Charitable Trust; Janssen Research & Development, LLC; JDRF International; Novo Nordisk; and Provention Bio. Other consortium members, participants, and advisors include individuals from the following organizations: Benaroya Research Institute at Virginia Mason; Lund University, Sweden; Helmholtz Zentrum München; University of Bristol; University of Colorado Denver; University of Florida; University of Helsinki; University of Leuven; University of Munich; University of Oulu; University of Tampere; University of Turku; Critical Path Institute; FDA; and NIH.

 


 

The Leona M. and Harry B. Helmsley Charitable Trust aspires to improve lives by supporting exceptional efforts in the U.S. and around the world in health and select place-based initiatives. Since beginning active grantmaking in 2008, Helmsley has committed more than $2 billion for a wide range of charitable purposes. Learn more about Helmsley at helmsleytrust.org.

 


 

About the Janssen Pharmaceutical Companies of Johnson & Johnson. At Janssen, we’re creating a future where disease is a thing of the past. We’re the Pharmaceutical Companies of Johnson & Johnson, working tirelessly to make that future a reality for patients everywhere by fighting sickness with science, improving access with ingenuity, and healing hopelessness with heart. We focus on areas of medicine where we can make the biggest difference: Cardiovascular & Metabolism, Immunology, Infectious Diseases & Vaccines, Neuroscience, Oncology, and Pulmonary Hypertension.

Learn more at www.janssen.com. Follow us at www.twitter.com/JanssenGlobal. Janssen Research & Development, LLC is one of the Janssen Pharmaceutical Companies of Johnson & Johnson.

 


 

JDRF is the leading global organization funding type 1 diabetes (T1D) research. Our mission is to accelerate life-changing breakthroughs to cure, prevent and treat T1D and its complications. To accomplish this, JDRF has invested more than $2.2 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 six 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.

 


 

Novo Nordisk is a global healthcare company with more than 95 years of innovation and leadership in diabetes care. This heritage has given us experience and capabilities that also enable us to help people defeat obesity, haemophilia, growth disorders and other serious chronic diseases. Headquartered in Denmark, Novo Nordisk employs approximately 42,700 people in 80 countries and markets its products in around 170 countries. For more information, visit novonordisk.com, Facebook, Twitter, LinkedIn, YouTube.

 


 

Provention Bio, Inc. (Nasdaq: PRVB) is a clinical-stage biopharmaceutical company leveraging a transformational drug development strategy that is focused on the prevention or interception of immune-mediated disease. Provention’s mission is to in-license, transform and develop therapeutic candidates targeting the high morbidity, mortality and escalating costs of autoimmune and inflammatory diseases including: type 1 diabetes (T1D), celiac disease and lupus. Provention’s diversified portfolio includes advanced-stage product development candidates that have undergone clinical testing by other companies. For more information, please visit proventionbio.com.

 


 

Benaroya Research Institute at Virginia Mason (BRI) is a world-renowned, non-profit research institute committed to advancing the science that will predict, prevent, reverse and cure diseases of the immune system. BRI researchers uniquely study the immune system in both health and disease, with the ultimate goal of achieving a healthy immune system for each individual. Diseases we study include type 1 diabetes, rheumatoid arthritis, lupus, multiple sclerosis, Crohn’s and colitis as well as allergies and cancer. BRI accelerates discovery through laboratory breakthroughs in immunology that can be translated to clinical therapies. A leader of collaborative initiatives such as the Immune Tolerance Network and Type 1 Diabetes TrialNet, BRI frequently partners with global research institutes, pharmaceutical and biotech companies. Visit BenaroyaResearch.org or follow us on the Autoimmune Life blog, Facebook, Instagram, YouTube, LinkedIn or Twitter.

 

C-Path 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 Receives FDA Grant to Establish Rare Disease Clinical Outcome Assessment Consortium

 

 

TUCSON, Ariz., March 19, 2020 — The U.S. Food and Drug Administration’s (FDA) Center for Drug Evaluation and Research (CDER) has funded a cooperative agreement to establish a Rare Disease Clinical Outcome Assessment (COA) Consortium. The grant (U01FD006882)* was awarded to the Critical Path Institute (C-Path) with the National Organization for Rare Disorders (NORD) as a sub-awardee. The first step taken toward the establishment of the new consortium has been the creation of the Rare Disease Subcommittee within C-Path’s Patient-Reported Outcome (PRO) Consortium. The PRO Consortium will serve as an incubator for the maturation of a pre-competitive, multi-stakeholder consortium within C-Path’s COA Program.

The PRO Consortium’s Rare Disease Subcommittee includes representatives from C-Path, NORD, FDA, the Patient-Centered Outcomes Research Institute, the National Center for Advancing Translational Sciences, and biopharmaceutical firms within the PRO Consortium that are developing treatments for rare diseases. In addition, plans are underway to enable rare disease-focused biotech firms not currently members of the PRO Consortium to be included in the strategic planning for the new consortium. Once established, the Rare Disease COA Consortium’s activities will be aimed at accelerating the development of new medical products intended to safely and effectively treat people with rare diseases by creating and curating a resource of information on publicly available COAs identified as potentially fit-for-purpose endpoint measures in treatment trials for rare diseases. The premise is that existing COAs may be able to be used or modified for use across multiple diseases sharing common characteristics.

Along with planning the membership, governance and organizational structure of the new consortium, the Rare Disease Subcommittee has launched a multi-pronged effort aimed at tackling challenges in the assessment of clinical benefit in rare disease treatment trials. The first pilot project under this initiative is to identify COAs that can be used in children to assess activities of daily living, which is a meaningful aspect of life impacted by many rare diseases. This will be the first in a series of reviews to identify COAs aimed at symptom and functional domains that reflect important aspects of patients’ lives that are impacted by rare diseases. Concurrently, the Rare Disease Subcommittee has initiated a second pilot project that involves a literature review to explore ways in which researchers have handled heterogeneity in clinical trials including an examination of the advantages and disadvantages of the approaches to personalizing endpoints. Development of best practice recommendations for assessing clinical benefit in rare disease trials will be subsequently explored.

*Funding for this press release was made possible, in part, by the Food and Drug Administration through grant U01FD006882. 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

Funded by FDA, C-Path and NORD to Launch Rare Disease Data and Analytics Platform

 

 

The collaborative project between the organizations will kick off at a launch meeting in September and will aim to reduce barriers for the development of new treatments and cures for rare diseases

 

TUCSON, Ariz. and WASHINGTON, August 7, 2019 — The Critical Path Institute (C-Path) and the National Organization for Rare Disorders® (NORD) will host a meeting on Tuesday, September 17 in Bethesda, MD to formally launch development of a new rare disease data and analytics platform. Funded by a cooperative agreement through the Food and Drug Administration, [Critical Path Public-Private Partnerships Grant Number U18 FD005320  from the US Food and Drug Administration] the goal of the platform is to accelerate the movement of therapies from bench to bedside for rare diseases. The platform will provide the infrastructure for a sustainable, cooperative scientific approach to clinical trial readiness in rare diseases by addressing vast knowledge gaps about the natural course of disease, the clinical evaluation of new treatments, and patients’ perspective on disease and treatment.

The Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP) will provide a centralized and standardized infrastructure to support and accelerate rare disease characterization with the goal of accelerating therapy development. The robust integrated platform will include integrated rare disease data from various sources such as clinical trials, observational studies, real world data and patient registries — including those within NORD’s IAMRARE registry platform — and an analytics platform that will allow efficient and effective interrogation of that data to generate solutions to inform clinical trial design and regulatory review.

“For people living with rare diseases, time is of the essence,” said Joseph Scheeren, PharmD, C-Path President and Chief Executive Officer. “By leveraging the rare disease community access and data of NORD and the data curation, aggregation, governance and advanced analytics expertise of C-Path, we are poised to make a significant impact on rare disease drug development by providing quality data that will inform clinical trial design and accelerate the development of therapies.”

“Currently, more than 25 million people in the United States are affected by one or more of the over 7,000 rare diseases,” stated Peter L. Saltonstall, NORD President and Chief Executive Officer. “Drug development for these diseases is often impeded due to the low affected patient numbers and a limited understanding of how rare diseases progress or how to measure clinical improvements. FDA-approved treatments exist for only 10% of rare diseases; with this collaboration we can change that statistic for the better for our rare community.”

Meeting attendees will include representatives from across the community including regulators, patient organizations, clinicians, researchers and pharmaceutical companies interested in rare disease drug development. Attendees will have the opportunity to learn about the goals of the RDCA-DAP and how to engage in the early stages of this effort.

Register for the meeting here; https://bit.ly/2YOj3jl

 


 

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 is committed to the identification, treatment and cure of the more than 7,000 rare diseases, of which approximately 90% are still without an FDA-approved treatment or therapy. 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. For more than 35 years, 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 275 disease-specific member organizations and their communities and collaborates with many other organizations on specific causes of importance to the rare disease community. For more information, visit rarediseases.org.

 

Media Contacts:

Kissy Black
C-Path
615.310.1894
kissyblack@lotosnile.com

Laura Mullen
NORD
203.304.7258
lmullen@rarediseases.org