Critical Path Institute

10/15/2026

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/yl71164jlj

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

10/15/2026

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/9ia7c7i5q3

10/14/2026

Now Available: New Evidence Frontier with Mike Davis and Klaus Romero from day one of the 2026 C-Path Global Impact Conference!

Critical Path Institute CEO, Klaus Romero, welcomes attendees and sets the stage for C-Path's Global Impact Conference 2026. Romero is then joined by FDA's CDER Director Michael Davis for the conference's first session, "A New Evidence Frontier.

A new paradigm is required to collect meaningful evidence for these rare diseases where large-scale RCTs are not feasible. Such a new paradigm is essential for converting evidence generation from a checkbox activity into a core strategic asset that drives optimal healthcare outcomes. This session covered how the rise of personalized medicine, complex treatments, and targeted therapies requires evidence that reflects real-world patient diversity, long-term safety, and economic value, rather than just short-term efficacy in highly controlled, homogenous populations.

Watch the recording here: https://f.mtr.cool/d4ig11ved4

Critical Path Institute CEO Klaus Romero welcomes attendees and set...

10/12/2026

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/3qxh9h8rcy

Autosomal Dominant Tubulointerstitial Kidney Disease, or ADTKD, is ...

10/12/2026

Now Available: Lived Experience with ALS Advocate, Jessica Morris from day two of the 2026 C-Path Global Impact Conference!

Jessica Morris, a social worker from a small town in Illinois, shares a deeply personal and inspiring journey living with a rare genetic form of ALS, specifically SOD1 ALS, a disease that has affected many members of her family across generations. Diagnosed at age 34, Jessica describes the emotional and physical challenges of facing a disease with a historically grim prognosis. However, her story diverges from what many expect due to advancements in medical research, particularly the availability of tofersen, a treatment that offers new hope.

Jessica emphasizes the critical perspective of patients living with ALS, highlighting how incremental improvements, even those clinically small, can translate into profound changes in daily life such as regaining the ability to climb stairs, attend family events, and maintain independence. Her experience underlines the importance of patient-centered approaches in drug development and clinical trials, where understanding the real-world impact on quality of life should guide measures of success beyond statistics.

Ultimately, Jessica appeals to researchers, clinicians, and policymakers to remember the human face behind the data the urgent necessity of extending life, preserving function, and allowing patients and families to cherish more moments together. Her story embodies the intersection of science, patient advocacy, hope, and innovation, illustrating why continuous investment in rare disease research matters so deeply.

Watch the recording here: https://f.mtr.cool/yv837d03ue

Stay tuned for Jessica's interview coming soon!

Jessica Morris, Care Advocate, joins C-Path's 2026 Global Impact Co...

10/11/2026

ICYMI | The Critical Path Institute Podcast: 20 Years of Impact Reflect on C-Path’s Legacy and a Look Ahead

This Critical Path Institute podcast episode celebrates C-Path's 20 years of impactful innovation, and looks at the future of 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, type 1 diabetes prevention, irritable bowel syndrome, and myelodysplasia —from stagnant or neglected fields into thriving areas with new drug approvals and robust development pipelines. Building on these successes, C-Path looks to scale to materialize more meaningful impact for people in need, 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.

🔗 Listen & subscribe now:
Apple: https://f.mtr.cool/lnmj8pjrko
Spotify: https://f.mtr.cool/2c1b8smq7o
YouTube: https://f.mtr.cool/k7toukx0qz

This Critical Path Institute (C-Path) podcast episode celebrates C-...

10/11/2026

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/eqoqa1bhis

10/11/2026

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/1ho11rwa12

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

10/10/2026

C-Path in the News | C-Path awards $1.7 million to advance five drug development projects

Turning promising science into the next generation of treatments.

Critical Path Institute’s Translational Therapeutics Accelerator has awarded $1.7 million across five BRIDGe grants to help advance promising drug development projects addressing critical unmet needs in brain health, pediatrics, and rare and orphan diseases.

The five projects span a diverse range of therapeutic approaches including small molecules, a protein-based therapeutic, and an in vivo gene therapy and are focused on conditions ranging from inherited blindness and neurological lysosomal storage diseases to pediatric cryptosporidiosis, neuropathic pain, and sepsis-related cognitive decline.

What makes the BRIDGe program unique is the combination of funding, regulatory expertise, scientific guidance, and drug development support. Awardees work toward defined milestones and decision points while engaging with experts across C-Path to help generate the evidence needed to move their innovations closer to clinical development.

As C-Path CEO Klaus Romero noted, sharing regulatory expertise early in development can help innovators anticipate evidentiary expectations and generate the right evidence at the right time.

This is what collaboration in drug development looks like: bringing together science, resources, regulatory insight, and expertise to help promising ideas become meaningful options for patients.

Read more about the five projects and the impact of C-Path’s Translational Therapeutics Accelerator in the featured story: https://f.mtr.cool/flbqusexu7

Critical Path Institute (C-Path) today announced that its Translational Therapeutics Accelerator has awarded $1,699,500 through five Bridging Research and Innovation in Drug Development Grants (BRIDGe).

10/10/2026

Interested in C-Path updates? Be sure to subscribe at https://f.mtr.cool/akte4yg2lg 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.