Critical Path Institute

10/22/2026

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/dcv37d0bxt

Lindsey Murray, Dana DiBenedetti, Shayna Egan, MPH, Daphney C. Jean, Ph.D., Naomi Knoble, PhD, Dorothee Oberdhan, Nan Rothrock, Weimeng Wang

10/22/2026

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

At the 2026 COA Annual Meeting, attendees joined a dynamic panel discussion, Pulling Back the Curtain: Multi-stakeholder Insights on Electronic Clinical Outcome Assessment Implementation, moderated by Scottie Kern, Executive Director of Critical Path Institute's Electronic Clinical Outcome Assessment Consortium.

The session brought together experts from across the clinical trial ecosystem to examine real-world challenges that arise when implementing electronic clinical outcome assessments in clinical trials. Panelists shared perspectives from their roles across COA licensing, site operations, sponsor eCOA management, technology development and project implementation.

Through practical scenarios and interactive audience discussion, the panel explored how implementation decisions can affect different stakeholders throughout the trial process. The conversation highlighted challenges that may not always be visible across stakeholder groups and underscored the importance of transparency, collaboration and shared understanding when planning and executing eCOA strategies.

Access the full collection of session recordings here: https://f.mtr.cool/0szjalalwm

10/21/2026

What does it mean to live with muscular dystrophy, and what should researchers, regulators and drug developers understand about it? Adith Thummalapalli, president of the Parent Project Muscular Dystrophy Adult Advisory Committee, shares his personal journey: the everyday challenges, the emotional realities and his experiences with the health care system.

Recorded Sept. 15, 2026, at the C-Path Global Impact Conference in Washington, D.C.

Adith's story is a reminder of why the voices of people living with a disease belong at the center of research and therapeutic development, and of what's lost when they aren't heard.

Watch all CGIC 2026 sessions: https://f.mtr.cool/d0ua3ogqag

Save the date: The 2027 C-Path Global Impact Conference is planned for the week of Sept. 13, 2027, at the Watergate Hotel in Washington, D.C. Get updates: https://f.mtr.cool/skmzt1oysu

Learn more about Parent Project Muscular Dystrophy: https://f.mtr.cool/lqawhdlpuo

SUBSCRIBE to C-Path's YouTube channel: https://f.mtr.cool/bftvfmhnkg

10/19/2026

ICYMI | As precision medicine advances, approaches to benefit–risk must evolve beyond traditional models designed for large, repeatable populations. This webinar brought together patients, families, regulators, and industry to explore how benefit–risk is understood, weighed, and acted on in the context of highly individualized therapies. Through real-world perspectives, panelists examined what outcomes matter most, how risk is navigated under uncertainty, and what it means to make truly informed decisions.

Building on C-Path’s February webinar on transforming drug development for precision medicines, which explored evidence generation, data strategies, and regulatory pathways, this session advances the conversation by centering the patient voice and highlighting the shift toward more coordinated, patient-centered approaches in benefit–risk decision-making.

What We Covered:

- How traditional development models translate to personalized contexts
- Patient and family perspectives on real-world benefit–risk decisions
- How benefit–risk evolves as new therapies become available
- Opportunities for collaboration across patients, regulators, and industry

If you missed it, you can access the recording here: https://f.mtr.cool/tv9v1vowr4

10/18/2026

ICYMI | The Critical Path Institute Podcast: eCOA Conversations on Digital Health Technologies

This podcast episode from Critical Path Institute’s® Electronic Clinical Outcome Assessment Consortium in the eCOA Conversations series focuses on the adoption of sensor-based digital health technologies 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.

🔗 Listen & subscribe now:
Apple: https://f.mtr.cool/u2bpvlqxw9
Spotify: https://f.mtr.cool/w4cm42ni0u
YouTube: https://f.mtr.cool/lgbgry4ft7

This newest podcast episode from Critical Path Institute’s® (C-Path...

10/18/2026

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/py33g3oyw2

10/18/2026

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/aqzaybvoxg

Lindsey Murray, Dana DiBenedetti, Shayna Egan, MPH, Daphney C. Jean, Ph.D., Naomi Knoble, PhD, Dorothee Oberdhan, Nan Rothrock, Weimeng Wang

10/17/2026

C-Path in the News | GATC Health Joins Critical Path Institute’s New Approach Methodologies Developer Coalition to Advance AI-Enabled Regulatory Science

Advancing AI-enabled regulatory science through collaboration.

Critical Path Institute is pleased to welcome GATC Health as a contributing member of the New Approach Methodologies Developer Coalition.

As the coalition’s first AI-native drug discovery and development-focused collaborator, GATC Health brings expertise in AI-enabled, in silico modeling of human biology, multiomics, and predictive modeling to a growing community of NAM developers, biopharma companies, regulators, and other stakeholders.

Together, NAMs-DC members are working to advance the evaluation, validation, qualification, and broader regulatory adoption of human-relevant methodologies helping bridge the gap between innovative technologies and their use in drug development.

GATC Health’s participation adds an important computational perspective to these efforts and reflects the growing need for collaborative approaches to understanding how emerging AI and in silico tools can generate reliable evidence for drug development and regulatory decision-making.

We look forward to working with the GATC Health team and our broader NAMs-DC community to help build confidence in innovative approaches and advance the science needed to support their responsible use.

Innovation moves faster when we work together

Learn more about GATC Health joining NAMs-DC: https://f.mtr.cool/cn0nq0jfjr

GATC to contribute AI-native, in silico drug discovery and predictive modeling expertise and insights to collaborative effort advancing the valida...

10/17/2026

Interested in C-Path updates? Be sure to subscribe at https://f.mtr.cool/20etx84rh9 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.

10/17/2026

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

Attendees at the 2026 COA Annual Meeting received an update on the newly rebranded Patient-Centered Evidence Consortium, formerly known as the Patient-Reported Outcome Consortium, from Sonya Eremenco, MA, Executive Director of Critical Path Institute's Patient-Centered Evidence Consortium.

The session highlighted the consortium’s evolution and its continued commitment to advancing patient-centered outcomes and clinical outcome assessments in drug development. Participants learned about key accomplishments achieved through collaboration among industry, regulatory agencies, academia, and patient organizations, as well as the consortium’s growing impact on generating evidence that reflects what matters most to patients.

The update also provided a look ahead at current initiatives and future priorities, underscoring the C-Path's Patient-Centered Evidence Consortium’s expanding role in supporting patient-focused medical product development and advancing the science of patient-centered evidence.

As the field continues to evolve, the consortium remains focused on fostering collaboration and developing tools and methodologies that help ensure the patient perspective is meaningfully incorporated into drug development and regulatory decision-making.

Access the full collection of session recordings here: https://f.mtr.cool/dq0ou2frge