Advertise on podcast: The Critical Path Institute Podcast

The newest episode of the Critical Path Institute Podcast features C-Path CEO and host Klaus Romero, as well as C-Path Scientific Director and New Approach Methodologies (NAMs) industry expert Graham Marsh. The duo engages in a discussion centered around the evolution of the translational science that bridges preclinical research and human trials. Graham emphasizes the importance of defining the “context of use” for each NAM—specifying the unmet need in regulatory science or drug development that it addresses. Recent C-Path endeavors include the formation of the NAM Developer Coalition, a pre-competitive forum to harmonize evidentiary standards and promote adoption. Additional partnerships highlighted include a collaboration involving Advanced Research Projects Agency for Health and FDA to integrate NAMs into regulatory decision-making from early development stages, and the recently announced creation of the National Center for Accelerating Cures (NCAC) to provide critical external validation and regulatory expertise for NAM developers. Looking ahead, Graham envisions NAMs becoming standardized, mainstream methods that improve drug development efficiency, reduce animal use, and enhance predictive accuracy. Data sharing and ecosystem integration remain key challenges but also immense opportunities for the field’s growth. Key Insights NAMs redefine preclinical testing beyond traditional assays. By integrating complex 3D tissue models and computational simulations, NAMs enhance biological relevance compared to 2D cultures or animal models, which often poorly predict human responses. The NCAC represents an ecosystem approach to NAM validation and scaling. By offering regulatory expertise, laboratory capabilities, and venture ecosystem support, NCAC enables NAM developers to build robust evidence for regulatory applications, accelerating these innovative tools’ transition to standard use. Mimicking human organ function with microphysiological systems is a game changer for translational science. Technologies like organ-on-chip can replicate organ-specific physiological conditions (e.g., blood flow, shear stress), recapitulating complex cell interactions and functions that animal or standard cell culture models cannot capture. The concept of “context of use” bridges scientific innovation with regulatory acceptance. A NAM must be tied to a clearly defined unmet need or regulatory question to build the right evidence package. This triad of developers, pharmaceutical scientists, and regulators facilitates qualification, ensuring NAMs are fit-for-purpose and not misapplied. Pre-competitive collaboration accelerates NAM qualification and adoption. C-Path's NAMs Developer Coalition exemplifies how companies can share standards, validation data, and advocacy efforts, overcoming the regulatory unfamiliarity of NAMs and driving widespread uptake in drug development pipelines. SUBSCRIBE to C-Path's YouTube Channel Now ►► https://www.youtube.com/c/CriticalPathInstitute FOLLOW C-Path: ►LinkedIn: https://www.linkedin.com/company/c-path ►Facebook: https://www.facebook.com/CPathInstitute/ ►Twitter: https://twitter.com/CPathInstitute?re About Critical Path Institute C-Path is an independent, nonprofit established in 2005 as a public-private partnership, in response to the FDA’s Critical Path Initiative. C-Path’s mission is to lead collaborations that advance better treatments for people worldwide. Globally recognized as a pioneer in accelerating drug development, C-Path has established numerous international consortia, programs and initiatives that currently include more than 1,600 scientists and representatives from government and regulatory agencies, academia, patient organizations, disease foundations and pharmaceutical and biotech companies. With dedicated team members located throughout the world, C-Path’s global headquarters is located in Tucson, Arizona and C-Path’s Europe subsidiary is headquartered in

The newest episode of the Critical Path Institute Podcast explores the essential collaboration between patients, researchers, regulators and drug developers working to accelerate drug development for neuromuscular diseases, particularly Duchenne muscular dystrophy (DMD) and other muscular dystrophies. In honor of World Duchenne Day, this timely discussion features Ramona Belfiore-Oshan, Executive Director of C-Path's Duchenne Regulatory Science Consortium, and Pat Moeshen, a patient advocate with Parent Project Muscular Dystrophy who is living with Limb Girdle muscular dystrophy (LGMD). They join C-Path CEO and podcast host Klaus Romero to highlight how patient voices have evolved from passive observers to active partners in clinical trial design, endpoint selection and drug approval processes.

This latest episode of the Critical Path Institute Podcast features Julia Vitarello, founder of Mila’s Miracle Foundation, and mother to Mila. Mila was diagnosed with Batten’s disease, a rare, fatal, inherited disorder of the nervous system. Mila eventually succumbed to this disease, but not before a custom treatment, Milasen, extender her life. Julia shares this personal journey from caregiver to advocate and innovator in the rare disease space. Mila became the first patient in the world to receive a highly personalized antisense oligonucleotide (ASO) treatment targeting her unique mutation—a breakthrough in precision medicine. What followed was Mila’s Miracle Foundation and, eventually, C-Path's One to Millions initiative. Julia joins C-Path CEO and podcast host Klaus Romero to discuss the challenges of rare disease drug development, emphasizing how the current regulatory and development systems are ill-suited for the thousands of rare conditions affecting millions globally. While revolutionary science exists, scaling these individualized treatments sustainably demands transformational shifts in regulatory frameworks, clinical standards, data sharing, safety monitoring, and reimbursement systems. The conversation highlights the promising regulatory changes underway, like process-based approvals allowing grouping of medicines by mechanism rather than disease, and initiatives like One to Millions, which aim to aggregate data and standardize approaches. Julia and host Klaus underscore the critical need for collaborations across academia, industry, regulators, and patient groups to build infrastructures enabling these therapies at scale. The goal is clear: drastically accelerate personalized medicine accessibility for millions suffering from rare genetic diseases without compromising safety or scientific rigor. FOLLOW C-Path: ►LinkedIn: https://www.linkedin.com/company/c-path ►Facebook: https://www.facebook.com/CPathInstitute/ ►Twitter: https://twitter.com/CPathInstitute?re About Critical Path Institute C-Path is an independent, nonprofit established in 2005 as a public-private partnership, in response to the FDA’s Critical Path Initiative. C-Path’s mission is to lead collaborations that advance better treatments for people worldwide. Globally recognized as a pioneer in accelerating drug development, C-Path has established numerous international consortia, programs and initiatives that currently include more than 1,600 scientists and representatives from government and regulatory agencies, academia, patient organizations, disease foundations and pharmaceutical and biotech companies. With dedicated team members located throughout the world, C-Path’s global headquarters is located in Tucson, Arizona and C-Path’s Europe subsidiary is headquartered in Amsterdam, Netherlands. For more information, visit c-path.org. #CPath #drugdiscovery #researchgrants #globalhealth #drugdevelopment #datasharing #researchfunding #raredisease #academicresearch #regulatoryscience #globalhealth #consortia #collaboration

This episode of the Critical Path Institute podcast features Maaike Everts, Executive Director of C-Path's Translational Therapeutics Accelerator. Maaike joins C-Path's CEO and podcast host Klaus Romero to explain how the accelerator supports the translation of academic drug discoveries into viable therapies for patients. The discussion highlights the unique challenge of bridging what is often called the drug development "valley of death" — the gap between academic research and drug development by pharmaceutical companies. The Accelerator offers not only funding but essential mentorship, education, and coordination among drug development research scientists, regulatory experts, and industry to help academic investigators design experiments and navigate regulatory and business landscapes.

This latest Critical Path Institute® (C-Path) podcast episode centers on the global strategy and impact of C-Path’s regulatory science efforts. C-Path CEO and podcast host Klaus Romero welcomes C-Path Vice President of Global Affairs Cécile Ollivier. Cécile leads C-Path's global regulatory science initiatives from the organization’s European headquarters in Amsterdam. The discussion highlights the importance of a worldwide presence to navigate diverse regulatory ecosystems, drive alignment between major agencies like the FDA and EMA and develop innovative drug development tools.

The newest Critical Path Institute (C-Path) podcast episode features a candid and inspiring conversation with host Klaus Romero and two C-Path staff members, T1DC Project Manager Hailey Davenport and Communications Assistant Director Alex Diegel. Alex and Hailey strive to accelerate drug development as members of the C-Path team, and also live with chronic health conditions —Type 1 diabetes (T1D) and chronic kidney disease (CKD). The episode explores their personal journeys with their diagnoses, the challenges they face daily, and how their work at C-Path intertwines with their lived experiences. Hailey, diagnosed with T1D at age 14, shares the complexities of managing diabetes beyond just insulin treatment, emphasizing the physical and psychological burdens of the disease. Alex recounts his recent diagnosis of IgA nephropathy, a form of CKD, the frustrations of navigating healthcare while continuing an active lifestyle, and advocating for himself to ger answers. Both discuss how C-Path's collaborative and innovative approach to drug development and patient advocacy motivates them., as well as the importance of connecting patient voices to research and early intervention strategies. The conversation underlines the significance of community, empathy, and the role of patient-centered research in transforming chronic disease management to improve quality of life. Highlights Hailey shares her experience being diagnosed with Type 1 diabetes at 14 and how it shaped her career and life. Alex describes his diagnostic journey with IgA nephropathy and the importance of self-advocacy in managing chronic illness. Hailey explains why insulin treatment is not a cure and the ongoing daily physical and psychological challenges of T1D. Alex highlights how sharing his diagnosis story improved C-Path's patient-focused communication and storytelling. Klaus connects their experiences with the goal of achieving instantaneous, easy-to-access biomarkers for CKD similar to those in T1D. Alex illustrates how C-Path's community resources helped him and his network navigate rare diseases beyond his own condition. The three emphasize the importance of not letting diagnoses define them, but using their experiences to drive change. This episode offers deep insights into the realities of living with chronic disease while working within an organization dedicated to accelerating drug development and improving patient outcomes. It underscores the critical role of patient voices in shaping research priorities and the transformative potential of collaborative, multidisciplinary approaches in regulatory science and clinical innovation.

In this episode of the Critical Path Institute© Podcast, C-Path Vice President of Rare/Orphan and Pediatric Disease Programs Collin Hovinga, joins host and CEO Klaus Romero to discuss the current landscape of rare diseases, the unique challenges and opportunities they present, and how C-Path is uniquely positioned to accelerate drug development for these conditions. This podcast highlights how rare diseases collectively represent a vast and complex group with about 10,000 known rare conditions and numerous subtypes. These differences present challenges, but also opportunities due to shared underlying disease mechanisms. C-Path’s approach to advancing drug development centers on collaboration among regulators, industry, patient advocacy groups, and researchers, ensuring that patient voices are integral to drug development. A key resource in this ecosystem is the Rare Disease Cures Accelerator-Data and Analytics Platform, a hub that aggregates, standardizes, and curates' diverse data sources such as registries, clinical trials, and natural history studies. The platform enables data reuse to avoid redundant research, supports innovative clinical trial designs, and facilitates regulatory decision-making by providing tools such as trial simulations and alternative control models. The conversation highlights examples of successful applications of these tools in diseases like Friedreich’s ataxia, polycystic kidney disease, and Duchenne muscular dystrophy, emphasizing the importance of regulatory engagement and public-private partnerships. The podcast also covers the legislative framework supporting rare neurodegenerative diseases, specifically the Act for ALS, which fosters collaboration between FDA, NIH, C-Path, patients, and industry to accelerate drug development. Highlights Introduction to the Rare Disease Cures Accelerator-Data and Analytics Platform, C-Path’s data platform that aggregates and standardizes rare disease data to accelerate drug development. Description of trial simulation tools and how they support regulatory decisions and clinical trial design. Importance of regulatory engagement and public-private partnerships in successful rare disease drug development. Discussion of the Act for ALS and how it fosters collaboration among NIH, FDA, industry, patients, and C-Path. Insight into pharmaceutical industry participation and trust in C-Path’s role as the neutral convener.

This panel discussion, hosted by C-Path’s Electronic Clinical Outcome Assessment Executive Director Scottie Kern, features experts from industry and academia; Bill Byrom, Principal of eCOA Science at Signant Health, Melissa Mooney, Director of eCOA Solution Engineering at IQVIA, Thomas Moll, Senior Scientific Advisor at Clario, and Devin Pipert, Professor of Health Outcomes Research and 125th Anniversary Chair at the University of Birmingham, UK. The conversation explores the evolving role and strategic integration of clinical outcome assessments, particularly those administered electronically, in oncology research. The episode highlights historical context, regulatory insights, practical implementation challenges, and future directions for clinical outcome assessment use in oncology clinical trials.

Critical Path Institute’s (C-Path) newest podcast episode picks up on the 20th Anniversary commemoration and provides an insightful outlook to what’s coming next for C-Path as we mold the future of drug development. This engaging conversation between C-Path CEO Klaus Romero and President/COO Kristen Swingle highlights how C-Path's core competencies in regulatory science, biomarkers, data, clinical outcome assessment, and modeling and analytics accelerate therapeutic innovation. As the pharmaceutical industry transitions into a new century marked by personalized medicine and advanced therapeutic modalities, such as advanced therapeutic medical products (including, but not limited to gene editing and cell therapies), and C-Path’s role in advancing modern drug development paradigms. The speakers emphasize the necessity to adopt novel scientific frameworks that maintain rigorous evidence standards while embracing personalization and innovation. Key themes discussed include the rise of decentralized clinical trials supported by digital health technologies, the transformative potential of real-world evidence from real-world data, and the promising role of artificial intelligence (AI) in drug development — though caution is urged to ensure AI outputs are scientifically sound and ethically applied. The discussion stresses the crucial involvement of patients, advocating for patient-focused drug development to place the patient voice at the center of innovation. The episode also explores C-Path’s initiatives to bridge the “valley of death” in translational research in order to empower academicians to move discoveries from the lab to practical drug development with regulatory guidance and funding support.

This Critical Path Institute (C-Path) podcast episode celebrates C-Path's 20 years of impactful innovation in accelerating drug development by connecting scientific, clinical, regulatory, and data-driven disciplines. Founded in 2005 in response to the FDA’s 2004 Critical Path Initiative, C-Path was established as a neutral, nonprofit organization to facilitate public-private partnerships involving industry, academia, patient groups, and regulators. These partnerships aim to identify bottlenecks in drug development and create actionable scientific solutions that make the process more efficient for all stakeholders. Over the past two decades, C-Path has transformed challenging therapeutic areas—such as Alzheimer’s disease, rare diseases like Duchenne muscular dystrophy and Friedreich’s ataxia, tuberculosis, polycystic kidney disease, and type 1 diabetes prevention—from stagnant or neglected fields into thriving areas with new drug approvals and robust development pipelines. This success was largely driven by integrating diverse data sources (clinical trials, observational studies, patient registries, electronic health records) into comprehensive platforms, enabling the development and qualification of biomarkers, clinical outcome assessments, and quantitative modeling tools. Join C-Path CEO Klaus Romero and President/COO Kristen Swingle Part One of this podcast for a final celebration of our 20 years of impact, as well as a year in review of all of the accomplishments C-Path made through 2025 alone.

Critical Path Institute (C-Path CEO Klaus Romero is joined by International Neonatal Consortium Executive Director Kanwaljit Singh to discuss the unique challenges and innovations in neonatal drug development. Neonates, particularly premature babies, are not simply smaller versions of adults or older children; their rapidly evolving physiology and complex medical conditions require specialized approaches in drug development. Singh explains how the INC fosters collaboration across pharmaceutical companies, academic researchers, healthcare professionals, parents, and global regulatory agencies to accelerate neonatal drug development. A key achievement of the consortium has been the development of the Neonatal Adverse Event Severity Scale (NAESS), which standardizes the assessment of adverse events in neonates, a critical need since traditional adult or pediatric adverse event scales are inadequate for this population. The consortium is now working on NAESS 2.0, expanding the scale and creating a digital tool to facilitate its use in clinical trials. Another major focus is the integration of real-world data and clinical trial data, creating one of the world’s largest neonatal databases with over 380,000 patient records from hospitals across the U.S., Canada, UK, and Japan. This database supports the development of disease progression models, such as for bronchopulmonary dysplasia (BPD), a common and severe lung condition in premature neonates, and the establishment of standardized reference ranges for lab values in neonatal intensive care units (NICUs). INC is also initiating efforts around neonatal brain injury, collecting data to validate biomarkers as surrogate endpoints, which could accelerate clinical trials by providing earlier indicators of treatment efficacy.

In the latest episode of the Critical Path Institute (C-Path) Podcast, C-Path CEO and podcast moderator Klaus Romero sits down the Vice President of our Quantitative Medicine Program Vice President Dr. Shu Chin Ma. Shu Chin and Klaus discuss how C-Path integrates mathematics, statistics, artificial intelligence, and digital technologies to accelerate drug development. She clarifies how the QuantMed program uses mathematical models to understand how drugs move through the body and how the body reacts, enabling optimized clinical trial designs to increase success rates, reduce risks, and improve patient safety. By simulating various clinical trial scenarios, these models help tailor treatments to individual patient characteristics, addressing variability in responses and disease progression. Shu Chin also emphasizes that clinical trials are complex endeavors requiring detailed knowledge about patient populations, disease stages, and other treatments. Modeling helps differentiate natural disease progression from drug effects, which is crucial to accurately assessing a drug’s efficacy and safety. Even unsuccessful trials provide valuable data that can refine future trial designs. The approach also has significant economic benefits by reducing trial time and patient burden.

This newest podcast episode from Critical Path Institute’s® (C-Path) Electronic Clinical Outcome Assessment (eCOA) Consortium in the eCOA Conversations series focuses on the adoption of sensor-based digital health technologies (DHTs) in clinical research, with concentrations on the current landscape, challenges, and future opportunities. The expert panel of Dr. Elena Izmailova, Former Chief Scientific Officer at Koneksa Health, Dr. Christine Guo, Chief Scientific Officer at Ametris, and Managing Partner of Patient Cloud at Medidata, Mark Matson, join eCOA Executive Director, Scottie Kern, to share their diverse journeys in digital health and insights on how these technologies can transform clinical trials. The panelists agree that while sensor-based DHTs are gaining traction, the field is still in its early stages and not yet commoditized like biomarker assays. Key barriers include limited familiarity within biopharma sponsors, lack of technical expertise, the complexity of data collection and interpretation, and regulatory uncertainties. They underscore the critical need for education across clinical, regulatory, and operational teams, as well as the importance of multidisciplinary collaboration, including data scientists and biomedical engineers.

This newest Critical Path Institute (C-Path) podcast episode provides an in-depth exploration of one of C-Path's core competencies: the Data Collaboration Center (DCC). Led by Ramona Walsh, Director of the DCC, and Rick Liwski, Chief Technology Officer, Rick and Ramona join C-Path CEO Klaus Romero to discuss how data drives C-Path’s efforts to accelerate drug development. The podcast explains the types of data integrated by =DCC — including clinical trial data, electronic health records (EHR), imaging, digital device data, and preclinical findings—and how the team manages complex challenges of data acquisition, standardization, privacy, and accessibility. DCC emphasizes Findable, Accessible, Interoperable, and Reusable (FAIR) data principles and implements rigorous curation to ensure data quality and regulatory compliance. The episode highlights DCC’s pivotal role in assembling large, integrated datasets crucial for developing drug development tools, especially in challenging areas such as rare and orphan diseases where patient populations are limited. The conversation also outlines the distinctions and interplay between data management, data engineering, and data science within C-Path, as well as the evolution of the Data and Analytics Platform (DAP), which securely hosts and facilitates collaborative research on over 700,000 patient datasets spanning 450+ studies. Looking to the future, the leaders share a vision emphasizing federated data access across multiple platforms and the integration of artificial intelligence and machine learning to enhance data processing, curation, and analytical efficiencies. The episode wraps up by reinforcing the DCC’s critical mission to convert complex data into actionable insights that move drug development forward and facilitate regulatory review.