Critical Path Institute

03/27/2026

ICYMI | C-Path's team hosted a webinar bringing together drug development leaders for a practical, collaborative discussion on evidence needs, data strategies, platform opportunities, and approaches to regulatory engagement for individualized and small-population therapies.

The “Transforming Drug Development for Precision Medicines” webinar highlighted how precision medicine continues to evolve and explored shared challenges and opportunities in creating more predictable, aligned pathways from development through reimbursement.

This expert-led discussion covered:
- Evolving evidence needs for individualized and small-population therapies
- Data strategies that support regulatory and payer decision-making
- Framework and infrastructure opportunities to improve predictability and efficiency
- Practical approaches to early and ongoing regulatory engagement
- Clear next steps for organizations navigating precision medicine development today

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

03/27/2026

As precision medicine continues to evolve, so too must the frameworks we use to evaluate benefit–risk, especially for patients facing severe, rare conditions with limited or no treatment options.

Join us on April 8 at noon (Eastern) for a timely and important discussion: Scalable Approaches to Patient-Centered Benefit–Risk: Defining Patient-Focused Drug Development in Precision Medicine

This session will feature patients and families sharing real-world experiences that are shaping how benefit–risk is understood in the context of individualized medicines. Their perspectives will provide critical insight into how decisions are made when stakes are high and options are limited.

Together, we will explore:
- 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
- As traditional population-based models are increasingly challenged, this conversation will highlight how incorporating patient experience can help inform more responsive and scalable approaches to drug development.

Register now to be part of this important discussion: https://f.mtr.cool/uzakkksyco

03/26/2026

Now Available on Demand! Catch up on every moment of the 2025 C-Path Global Impact Conference! All sessions are now streaming on our YouTube channel — watch, learn, and share.

This engaging discussion from Critical Path Institute’s (C-Path) Global Impact Conference (CGIC) 2025, focuses on the future of regulatory science, drug development, and the role of collaboration between public and private sectors to accelerate biomedical innovation. This session, led by Consilium Salmonson & Hemmings Partners and C-Path Board Members Tomas Salmonson and Robert Hemmings, C-Path Senior Scientific Director Smith Heavner, FDA Senior Advisor Marie Bradley, EMA Head of Regulatory Science and Academia Ralf Herold, and Senior Medical Director at Biogen, Angela Paradis, emphasizes the importance of speeding up drug development to benefit patients faster and at lower costs.

The conversation highlighted the need for better tools, frameworks, and guidance to facilitate the appropriate use of external control arms. Key points include the current regulatory uncertainty, the evolving nature of methodological standards, and the necessity of early engagement between sponsors and regulators to improve study designs and regulatory acceptability.

Watch the video on C-Path's YouTube channel here: https://f.mtr.cool/jsaxrkmqee

This engaging discussion from Critical Path Institute’s (C-Path) Global Impact Conference (CGIC) 2025, focuses on the future of regulatory science, drug deve...

03/26/2026

Now enrolling!

Advance your expertise at the intersection of science, policy, and innovation at the University of Arizona.

The Graduate Certificates in Health Law & Policy at the University of Arizona offer professionals the opportunity to deepen their understanding of the legal and regulatory frameworks that shape healthcare and drug development—while earning a recognized credential.

Courses are taught by leading experts, including contributors from Critical Path Institute, bringing real-world perspective to topics like regulatory science, clinical research, and data-driven decision-making.

Whether you’re in industry, research, or policy, these certificates are designed to help you navigate an evolving healthcare landscape and drive meaningful impact.

Learn more and apply: https://f.mtr.cool/xcnadohipz

03/26/2026

Registration is now open for the second Biomarker Data Repository virtual workshop: Moving Biomarkers from Research to Better Real-World Care for Patients! Join us virtually on March 30, 2026, at 2:00 PM ET to explore how emerging kidney biomarkers and integrated data approaches can improve the detection, interpretation, and management of kidney disease.

The workshop will be organized into three focused acts followed by a live Q&A session:
Act 1 – Patient Perspectives: Understanding Biomarkers in Kidney Care
This session will focus on how biomarker information can be presented and understood from a patient perspective. Using examples of simulated biomarker results in patient portals, speakers will explore strategies for communicating results clearly and meaningfully to patients, including improved patient-facing reports and educational resources.
Act 2 – Expert Perspectives: Clinical Interpretation and Adjudication
Nephrologists and subject matter experts will discuss clinical interpretation and adjudication of biomarker data, including lessons from biomarker qualification efforts and the potential for interactive “sandbox” tools to support education and broader clinical adoption.
Act 3 – The Future is Now: Integrating Biomarkers and Advanced Data
The final session will highlight how pooled datasets and advanced molecular insights—such as single-cell and molecular mapping data—are helping deepen understanding of kidney disease and may support more precise diagnosis, prognosis, and treatment decisions.

Together, these sessions will foster dialogue and identify opportunities to advance the use of biomarkers and integrated data to support more informed, personalized kidney care.

📅 Date: March 30, 2026
⏰ Time: 2:00 – 3:15 PM ET
💻 Format: Virtual
👉 Register here: https://f.mtr.cool/hfyybhhowq

We encourage you to share this invitation with colleagues and networks who may be interested in participating.

We look forward to your participation.

03/26/2026

Critical Path Institute® today announced the launch of One to Millions, a global, multi-stakeholder public-private initiative to enable scalable development of advanced therapies for highly individualized conditions. Rapid advances in technologies such as antisense oligonucleotides, genome editing, gene therapies, and RNA-based treatments are making it possible to design precisely targeted interventions for very small patient populations, even individual patients. However, existing regulatory and reimbursement frameworks, built for population-based medicines and linear development models, are not equipped to keep pace, creating a growing gap that delays patient access.

To help close this gap, One to Millions advances the U.S. Food and Drug Administration Plausible Mechanism Framework and Rare Disease Evidence Principles by leveraging C-Path’s centralized, regulatory-grade data platform to support evidence generation, regulatory decision-making, and scalable development pathways.

Read the full story here: https://f.mtr.cool/ybccchthuz

Klaus Romero, Kristen Swingle, Paul Edmeier, Chris Lunt, Cécile Ollivier, Diane Stephenson, Collin Hovinga, Cheryl C**n, Jennifer Kendra, Ramona Walls, Julia Vitarello, Sarah Glass, PhD, Tim Yu, janet woodcock, Karen Bernstein

Mila's Miracle Foundation, N=1 Collaborative, n-Lorem Foundation, Boston Children's Hospital, BioCentury Inc.

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This link will take you to a page that’s not on LinkedIn

03/26/2026

The future of individualized medicine is within reach, but scaling it requires more than scientific breakthroughs.

A new feature from BioCentury Inc. highlights the challenge: while frameworks like FDA’s plausible mechanism approach are opening the door, the infrastructure needed to bring these therapies to patients at scale is still taking shape.

That’s exactly where C-Path's One to Millions comes in.

C-Path’s new global initiative is designed to help operationalize these frameworks, building the shared data, tools, and pathways needed to move from one patient to many, and ultimately to millions.

🔗 Read the full article and explore what’s next for scalable, individualized therapies: https://f.mtr.cool/snntocqdmq

Klaus Romero Kristen Swingle Jennifer Kendra Collin Hovinga Diane Stephenson Cheryl C**n Chris Lunt Ramona Walls Julia Vitarello janet woodcock Tim Yu Sarah Glass, PhD Karen Bernstein

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This link will take you to a page that’s not on LinkedIn

03/26/2026

Expectations around submissions of eCOA-related materials at trial start up are a persistent challenge globally. In this Educational Series session, regulatory experts from the Electronic Clinical Outcome Assessment Consortium’s membership will explore the expectations of the EU’s Clinical Trial Regulation and the use of the EU’s Clinical Trial Information System from an eCOA trials perspective. Are we over-preparing for EU-CTR submissions? Are eCOA source screen reports and translations required for this regulatory approval? Join us to hear the experts weigh in, and bring your own queries for further discussion.

eCOA Exchange #6

eCOA and the EU’s Clinical Trial Regulation

Host: Scottie Kern

Participants:

Pierre Mermet-Bouvier, Senior Manager, eClinical Strategy & Delivery, ICON

Pierre-Frederic Omnes, Executive Director, Life Sciences, TransPerfect

Date/Time:

Thursday, April 2nd, 2026

10-11 AM ET


Registration Link here: https://f.mtr.cool/foxjaavyhq

03/25/2026

Save the Date: C-Path’s 2026 Global Impact Conference

Critical Path Institute’s (C-Path) flagship event is set to return to downtown Washington, D.C. this September 15-16 at the Washington Marriott at Metro Center. Building on momentum from last year’s successful conference, the C-Path Global Impact Conference (CGIC) will host regulators, researchers, industry experts, and patient advocates for two days of thought-provoking discussions, keynote speakers, networking events and more as we set the stage for the next wave of medical and regulatory science innovation. You won’t want to miss this exclusive event as we redefine the evidence-generation enterprise for drug development in the 21st Century with topics focused on innovation in accelerating patient identification, enrichment, and stratification, closing the gap between drug development and clinical care, new approaches to patient centric clinical trials, and more.

Be sure to check in as we publish more details, including speakers, panelists, session titles and the conference agenda. Don’t wait! Save the date!

Learn more here: https://f.mtr.cool/qxrjnlqvev

03/25/2026

We are pleased to invite you to register now for Critical Path Institute's 2026 Clinical Outcome Assessment Program Annual Meeting, taking place April 16–17, 2026, at the Washington Marriott at Metro Center in Washington, D.C.

This year’s meeting will once again bring together leaders from across industry, regulatory agencies, academia, patient organizations, and technology and allied service partners to advance the science and application of COAs in medical product development. Building on the strong engagement and momentum from last year, the 2026 program will feature deep-dive sessions, case studies, and multi-stakeholder discussions on pressing issues in the field, including:
- Using patient experience data to inform value assessment for access decision making
- Pulling back the curtain: Multi-stakeholder insights on electronic clinical outcome assessment implementation
- Incorporating the patient voice into clinical trial design: More than just checking the box
- From meaningful aspect of health to metric to endpoint: How multidisciplinary teams form a comprehensive approach to digitally-derived endpoints
- Revisiting meaningful change: Current thinking on establishing and applying benchmarks
- Addressing challenges in rare disease clinical trials: Methodological case studies

Register Now: https://f.mtr.cool/kvrxdwttzf

We look forward to sharing a more detailed agenda as it becomes available and welcoming you in April for another engaging and collaborative Annual Meeting.

03/25/2026

As precision medicine continues to evolve, so too must the frameworks we use to evaluate benefit–risk, especially for patients facing severe, rare conditions with limited or no treatment options.

Join us on April 8 at noon (Eastern) for a timely and important discussion: Scalable Approaches to Patient-Centered Benefit–Risk: Defining Patient-Focused Drug Development in Precision Medicine

This session will feature patients and families sharing real-world experiences that are shaping how benefit–risk is understood in the context of individualized medicines. Their perspectives will provide critical insight into how decisions are made when stakes are high and options are limited.

Together, we will explore:
- 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
- As traditional population-based models are increasingly challenged, this conversation will highlight how incorporating patient experience can help inform more responsive and scalable approaches to drug development.

Register now to be part of this important discussion: https://f.mtr.cool/mxnehuucqz

03/24/2026

Registration is now open for the second Biomarker Data Repository virtual workshop: Moving Biomarkers from Research to Better Real-World Care for Patients! Join us virtually on March 30, 2026, at 2:00 PM ET to explore how emerging kidney biomarkers and integrated data approaches can improve the detection, interpretation, and management of kidney disease.

The workshop will be organized into three focused acts followed by a live Q&A session:
Act 1 – Patient Perspectives: Understanding Biomarkers in Kidney Care
This session will focus on how biomarker information can be presented and understood from a patient perspective. Using examples of simulated biomarker results in patient portals, speakers will explore strategies for communicating results clearly and meaningfully to patients, including improved patient-facing reports and educational resources.
Act 2 – Expert Perspectives: Clinical Interpretation and Adjudication
Nephrologists and subject matter experts will discuss clinical interpretation and adjudication of biomarker data, including lessons from biomarker qualification efforts and the potential for interactive “sandbox” tools to support education and broader clinical adoption.
Act 3 – The Future is Now: Integrating Biomarkers and Advanced Data
The final session will highlight how pooled datasets and advanced molecular insights—such as single-cell and molecular mapping data—are helping deepen understanding of kidney disease and may support more precise diagnosis, prognosis, and treatment decisions.

Together, these sessions will foster dialogue and identify opportunities to advance the use of biomarkers and integrated data to support more informed, personalized kidney care.

📅 Date: March 30, 2026
⏰ Time: 2:00 – 3:15 PM ET
💻 Format: Virtual
👉 Register here: https://f.mtr.cool/xkahqrtxgh

We encourage you to share this invitation with colleagues and networks who may be interested in participating.

We look forward to your participation.