Critical Path Institute

09/06/2026

ICYMI | What does it mean to dedicate your career to accelerating drug development, while also living with the very diseases you're working to fight?

This Critical Path Institute® podcast episode explores exactly that. In this deeply personal and inspiring conversation, C-Path CEO Dr. Klaus Romero sits down with two members of our own team: Hailey Davenport, Type 1 Diabetes Consortium Project Manager, and Alex Diegel, Communications Assistant Director. Hailey lives with Type 1 diabetes (T1D), and Alex was recently diagnosed with IgA nephropathy, a form of chronic kidney disease (CKD). Together, they open up about their diagnoses, the daily challenges of managing chronic conditions, and how their lived experience fuels their work at C-Path.

🔗 Listen & subscribe now:
Apple: https://f.mtr.cool/wss2i0mvbr
Spotify: https://f.mtr.cool/kvqy4urfwr
YouTube: https://f.mtr.cool/ugbfvopemy

09/06/2026

Imaging the Invisible: A CGIC 2026 Session Spotlight

For decades, drug development in neurodegenerative disease has relied on classifying patients by symptoms that often surface only after significant, irreversible damage has occurred. Neuroimaging biomarkers are transforming how we conduct clinical trials across brain disorders.

On Sept. 16 at the C-Path Global Impact Conference in Washington, D.C., a session titled “Imaging the Invisible: Neuroimaging Biomarkers as Catalysts for Early-Stage Drug Development” will examine how PET, MRI and other imaging modalities are giving researchers in vivo insight into proteinopathies, neurodegeneration and circuit-level dysfunction across Alzheimer’s disease, Parkinson’s disease and rare neurodegenerative disorders. Panelists will discuss how these tools support biological staging frameworks that can identify disease well before clinical symptoms appear. Such a paradigm shift that aligns with the emerging paradigm of precision medicine in neurology, where clinical trial participants are stratified by underlying pathophysiology rather than by heterogeneous symptoms.

Register today: https://f.mtr.cool/azz4w7s233

09/06/2026

Dive into the world of solutions for Drug Development with C-Path's YouTube channel! Subscribe now for access to on-demand content including meetings, webinars, interviews, and more. Stay informed, stay inspired!

SUBSCRIBE to C-Path's YouTube Channel Now ►► https://f.mtr.cool/uh6a7pu3t1

09/05/2026

Interested in C-Path updates? Be sure to subscribe at https://f.mtr.cool/0h0irjmer3 For 20 years, C-Path has been providing vital infrastructure to generate a neutral environment for everyone working in drug development to collaborate, not compete. Let's improve lives, together.

09/05/2026

2026 Clinical Outcome Assessment Program Annual Meeting Recordings are now live on YouTube!

One of the standout discussions at the 2026 COA Annual Meeting explored a critical question: How can we move beyond simply "checking the box" and meaningfully incorporate the patient voice into clinical trial design?

Moderated by Nicki Bush (OPEN Health), the session brought together experts from industry, regulatory agencies, and patient advocacy organizations, including representatives from Sanofi, Takeda, the U.S. Food and Drug Administration, Italfarmaco, the International Rare Disease Research Consortium, and the International Rett Syndrome Foundation.

Panelists shared insights on how patient engagement can strengthen patient-focused medical product development, improve clinical research practices, and help ensure that studies are designed around outcomes that matter most to patients. The discussion highlighted both strategic and practical approaches for engaging patients throughout the clinical development process, as well as regulatory perspectives on integrating patient input into trial design.

A key theme throughout the session was that meaningful patient engagement requires ongoing collaboration among sponsors, regulators, researchers, and patient communities not as a one-time activity, but as a foundational component of clinical development.

Access the full collection of session recordings here: https://f.mtr.cool/m1gk7ovlee

09/05/2026

ICYMI | Public Private Partnerships for Rare Neurodegenerative Disease Webinar.

In a recent webinar, Critical Path Institute's Rare Neurodegenerative Diseases Program brought together representatives from the collaboration among federal partners, researchers, advocates, and members of the ALS lived-experience community to discuss progress achieved through Public Private Partnerships for Rare Neurodegenerative Disease, including those established by Act for ALS.

The discussion highlighted how collaboration among regulators, researchers, patient advocates, and people living with Rare Neurodegenerative Disease is helping advance the tools, data, and scientific approaches needed to support more efficient drug development and improve outcomes for patients.

As a trusted neutral convener, C-Path continues to play a key role in bringing stakeholders together to address complex challenges in neurodegenerative disease research, fostering data-driven solutions and patient-centered approaches that can help accelerate access to new therapies. The webinar underscored the power of public-private partnerships to drive innovation and advance a shared goal: delivering meaningful treatments to people affected by ALS and other rare neurodegenerative diseases.

Watch the webinar recording now: https://f.mtr.cool/2erkzh2uzm

Critical Path Institute's Critical Path for Rare Neurodegenerative ...

09/05/2026

CGIC 2026: Evidence-Driven. Patient-Focused. Future-Ready.

As you've come to expect from previous years, CGIC 2026 will offer the chance to collaborate on the next wave of innovative therapies, discuss the latest initiatives, network with colleagues, and drive progress for those who need it most — people living with unmet medical needs.
If you're part of the movement to improve drug development with accelerated timelines and patient-centered therapies, this is the event you can't afford to miss. You'll have two full days to engage in inspiring conversations, discover the latest developments in neurology, rare diseases, and pediatrics, and explore the newest tools and collaborations C-Path has to offer.

This year's program gets right to work on Day One with a session that goes to the heart of one of drug development's most persistent challenges: Fixing the Front End of Drug Development: Why Target Selection Is Foundational to Evidence Generation

Despite significant advances in clinical trial design, real-world evidence, and regulatory science, late-stage attrition remains stubbornly high, often due to insufficient biological confidence in therapeutic targets. This session tackles that challenge head-on, examining target selection as a critical but underrecognized component of the evidence-generation continuum.

Panelists will explore how weak or siloed target validation can cascade uncertainty through every stage of development, increasing patient burden and system-wide inefficiency. The discussion will spotlight emerging pre-competitive, collaborative approaches to building target confidence before first-in-human studies, grounded in shared learning, disease-agnostic principles, and patient relevance.

Featured Speakers:
• Chris Chen, VP of Research Programs, Polycystic Kidney Disease Foundation
• Rita Cowell, Professor, University of Alabama at Birmingham
• Maaike Everts, Executive Director, Translational Therapeutics Accelerator, Critical Path Institute
• Alexis Hoffher, Head of Cardiorenal Development, AstraZeneca
• Robert Schuck, Deputy Director, Division of Translational and Precision Medicine, Office of Clinical Pharmacology, CDER, FDA

This session is just one example of the cross-sector, pre-competitive collaboration that has defined C-Path's approach for the past 20 years, and a preview of the conversations that will shape the future of drug development for the next 20.

Register today to secure your place at CGIC 2026: https://f.mtr.cool/3rena5hm7b

09/04/2026

One Biomarker, Multiple Uses: Connecting Safety and Disease Applications | Wednesday, Sept. 16. | 9-10 a.m. ET | Grand Ballroom

Every biomarker provides information on a biological process. But how relevant such information is to inform specific types of decisions in the evidence generation process for new drugs varies. A tool built to flag drug-induced organ injury can also help characterize disease progression. A safety signal in one program can become a disease marker in another. The question is whether the field is set up to make those connections, or whether each program is left to rebuild the case from scratch.

The session opens with an update on C-Path's Biomarker Data Repository, followed by a multi-stakeholder panel of industry, clinical, bioanalytical, modeling and regulatory experts. The discussion will cover real-world use cases, measurement considerations in clinical trials and opportunities to align biomarker efforts across C-Path consortia to maximize impact.

Register today: https://f.mtr.cool/8avqzwz1k7

09/04/2026

📢 New panelist announced for CGIC 2026's "Got Data?" workshop
We're pleased to welcome Wei Hua, Director of Division of Epidemiology with Center for Drug Evaluation and Research FDA, to the panel for "Got Data? Evaluating Real-World Data Suitability to Inform Regulatory Decision-Making."

Join us Monday, Sept. 14 (9 a.m. to 12:30 p.m. ET) for this free, in-person workshop ahead of C-Path's Global Impact Conference in Washington, D.C. Attendees will work through a structured framework for assessing whether real-world data is fit for regulatory use, covering relevance, observability, comparability and reliability, with a focus on rare diseases.
This session is free, but space is limited.

Register now: https://f.mtr.cool/oqh00uq7pk

Alexandre Bétourné, PhD, PharmD, PMP, Collin Hovinga

09/03/2026

It is with great sadness that we share the passing of Peter Barton Hutt, a cherished member of the C-Path Board of Directors since 2015. His decades of leadership in food and drug law shaped the landscape we work in today. We honor his legacy and send our deepest condolences to his family.

Read the full statement here: https://f.mtr.cool/mvqi8wtmfx