Critical Path Institute
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The Critical Path Institute (C-Path) is an independent, non-profit organization established in 2005.
Critical Path Institute (C-Path) is a nonprofit, public-private partnership with the Food and Drug Administration (FDA) created under the auspices of the FDA’s Critical Path Initiative program in 2005. C-Path’s aim is to accelerate the pace and reduce the costs of medical product development through the creation of new data standards, measurement standards, and methods standards that aid in the scientific evaluation of the efficacy and safety of new therapies. These pre-competitive standards and approaches have been termed “drug development tools” (DDTs) by the FDA, which established a process for official review and confirmation of their validity for a given context of use. C-Path orchestrates the development of DDTs through an innovative, collaborative approach to the sharing of data and expertise. We build consensus among participating scientists from industry and academia with FDA participation and iterative feedback. The process culminates in a formal application to FDA for official “qualification” of the DDT for a given use in product development. Qualified DDTs then become open standards for the scientific community which, in turn, may be assured both of the scientific rigor under which they were developed and of the FDA’s understanding and acceptance of their validity.
ICYMI | Meeting Patients Where They Are: Validating Remote Digital Tools for Multi-Indication NMD Research
As clinical research continues to evolve, so must the tools used to measure outcomes particularly in neuromuscular disease studies, where accessibility and patient burden remain important considerations.
During the 2026 C-Path Rare and Orphan Disease Program webinar, "Meeting Patients Where They Are: Validating Remote Digital Tools for Multi-Indication NMD Research", Dr. Tina Duong joined a panel of experts from industry and regulatory science to discuss the future of digital measurement in neuromuscular disease research.
The webinar explored the scientific, clinical, and regulatory considerations involved in validating video-based assessments and other digital tools across multiple NMD indications. Through presentations and panel discussion, speakers examined how these technologies can help make clinical research more accessible to patients regardless of geographic location, reduce the burden of trial participation, and generate reliable, regulatory-grade data.
A recurring theme throughout the discussion was the importance of balancing innovation with patient-centered research principles. As digital tools become increasingly integrated into clinical trials, ensuring they are meaningful, accessible, and fit for purpose will be critical to advancing drug development and improving outcomes for people living with neuromuscular diseases.
The session highlighted the growing potential of remote digital assessments to expand participation in clinical research while maintaining the rigor needed to support regulatory decision-making and therapeutic development.
Watch the recording now: https://f.mtr.cool/9l9wy3ygvc
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09/27/2026
ICYMI | The Critical Path Institute Podcast: Connecting the Dots in Rare and Pediatric Disease
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.
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09/27/2026
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!
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09/27/2026
WEBINAR | Making Sense of Small Samples: Mixed Method Approaches to Outcome Interpretation in Rare Disease Trials
Rare disease endpoint interpretation can be particularly challenging when small sample sizes limit the utility of traditional statistical and psychometric approaches. Mixed-methods and qualitative approaches can provide complementary evidence to support the interpretation of treatment effects when conventional methods alone may be insufficient.
This 1-hour session will explore practical approaches to addressing these challenges, followed by an interactive panel discussion where attendees will have the opportunity to pose questions directly to experts and discuss challenges and considerations relevant to their own rare disease development programs.
Register now: https://f.mtr.cool/qgbmer7gvf
Lindsey Murray, Dana DiBenedetti, Shayna Egan, MPH, Daphney C. Jean, Ph.D., Naomi Knoble, PhD, Dorothee Oberdhan, Nan Rothrock, Weimeng Wang
09/26/2026
Interested in C-Path updates? Be sure to subscribe at https://f.mtr.cool/zec3dlsmkf 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.
2026 Clinical Outcome Assessment Program Annual Meeting Recordings are now live on YouTube!
At the 2026 COA Annual Meeting, experts explored how patient experience data can help inform value assessment and support access decision-making.
Moderated by Ebony Dasheill-Aje, Executive Director & Head of Patient Centered Outcomes Science at BioMarin, the session featured insights from Jessica Abel, MPH (AbbVie), Iyar Mazar, PhD (Pfizer), Leah Howard, JD (National Psoriasis Foundation), and Dana McCormick (AMCP).
The discussion examined how patient experience data is being integrated into health technology assessment and payer decision-making processes, including current HTA requirements, payer readiness to incorporate patient experience data into formulary and coverage decisions, and real-world examples of patient advocacy organizations engaging with ICER and payers.
Speakers also highlighted opportunities and challenges for sponsors in generating and presenting patient experience data to demonstrate value in ways that reflect what matters most to patients. The session reinforced the importance of ensuring patient perspectives are meaningfully considered not only in clinical development, but also in the decisions that shape treatment access.
Access the full collection of session recordings here: https://f.mtr.cool/v2nu2rfhxo
09/26/2026
ICYMI | Hope on the Horizon for ADTKD Patients
Did you know that Autosomal Dominant Tubulointerstitial Kidney Disease is the second most common inherited kidney disease after polycystic kidney disease? While considered a rare disease, ADTKD is estimated to affect 75,000–100,000 people in the United States and as many as two million individuals worldwide.
For generations, families affected by ADTKD have faced progressive kidney disease with few treatment options. Today, however, there is growing hope. Researchers are closer than ever to developing the first targeted treatments for ADTKD caused by UMOD and MUC1 gene variants, which account for the majority of cases. With clinical trials anticipated in the near future, the field is entering a pivotal moment that could transform care for patients and families living with this disease.
Together, researchers, clinicians, patient advocates, and families are building momentum toward a future where ADTKD can be identified earlier, managed more effectively, and ultimately prevented from impacting future generations.
Watch the recording now: https://f.mtr.cool/wvcrkun6lm
Hope on the Horizon for ADTKD Patients Autosomal Dominant Tubulointerstitial Kidney Disease, or ADTKD, is ...
2026 Clinical Outcome Assessment Program Annual Meeting Recordings are now live on YouTube!
Day 2 of the 2026 COA Annual Meeting opened with a welcome and update from Scottie Kern, Executive Director of Critical Path Institute's Electronic Clinical Outcome Assessment Consortium, who reflected on the Consortium’s achievements as it celebrated its 15th anniversary.
The session highlighted the Consortium’s significant contributions to advancing the science of electronic endpoint data collection and its role in fostering collaboration across the clinical research community. Attendees learned about ongoing initiatives in both the eCOA and digital health technologies spaces, as well as recent publications and resources developed to support the implementation of patient-centered data collection approaches in clinical trials. The update also provided insight into the Consortium’s future priorities and its continued commitment to addressing emerging challenges and opportunities in clinical outcome assessment and digital measurement.
Over the past 15 years, Critical Path Institute's Electronic Clinical Outcome Assessment Consortium has helped bring together industry, regulators, technology providers, researchers, and other stakeholders to advance best practices and improve the quality and consistency of endpoint data collection. The session served as both a celebration of that progress and a look ahead at the work still to come.
Access the full collection of session recordings here: https://f.mtr.cool/dfwo0dchwb
09/21/2026
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/cwj2hjiqc4
Public Private Partnerships for Rare Neurodegenerative Disease Webinar Critical Path Institute's Critical Path for Rare Neurodegenerative ...
09/20/2026
ICYMI | The Critical Path Institute Podcast: Accelerating Drug Development in Friedreich's Ataxia
This episode of the Critical Path Institute® (C-Path) podcast features Ron Bartek, president and co-founder of the Friedreich’s Ataxia Research Alliance (FARA), for an insightful conversation with C-Path CEO and podcast host Klaus Romero.
Ron shares the inspiring journey of how FARA was established nearly 28 years ago after his son was diagnosed with Friedreich’s ataxia (FA), a rare neurodegenerative disease with no approved treatments or substantial research at the time. Faced with a void in scientific and clinical data, Ron, his wife and a small community of parents, patients, and scientists collaborated to create a research alliance that would foster collaboration and accelerate drug development for FA.
🔗 Listen & subscribe now:
Apple: https://f.mtr.cool/by6q1tdxqd
Spotify: https://f.mtr.cool/b9et9fr3qz
YouTube: https://f.mtr.cool/modosnvf54
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