Critical Path Institute

When rare disease data are collected across patient populations, the question that matters most is: how do we maximize the value of such data?

On Sept. 16 at the C-Path Global Impact Conference in Washington, D.C., a session titled "Leveraging Rare Disease Data to Inform Drug Development Tools for Rare Neurodegenerative Diseases" will address exactly that, from theory to regulatory action.

Presenters Alexandre Betourne and Mingyuan Wang, joined by Mark Gordon of Arvinas and Mohamed Hamdani of Larimar Therapeutics, will walk through how C-Path's Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP®) is helping reshape how clinical trials for rare neurodegenerative diseases are designed and measured.

Session highlights include:
• Advanced analytics: An update on a pioneering computational model to optimize trial design for Friedreich’s ataxia, currently under FDA review
• Patient-focused drug development: Analysis of how different versions of the progressive supranuclear palsy rating scale predict survival and track disease progression
• Data sharing and integration: A look at how integrated rare disease datasets can address longstanding challenges in drug development
• Actionable impact: Discussion of how these approaches could extend to rare dementias and similar diseases where gaps in trial design and outcome measurement persist
This is evidence-generation work with direct implications for patients, researchers and regulators alike, and a concrete example of the paradigm shift C-Path is driving across the drug development landscape.

Room Block closing August 15! Register today: https://f.mtr.cool/jeiudpqaoe

Be part of the global conversation shaping the future of drug development. The Critical Path Institute’s Global Impact Conference 2026 brings together regulators, industry leaders, scientists, clinicians, those with lived experience, and advocates for three days of high-impact dialogue and collaboration, all focused on accelerating better treatments for patients worldwide.

This is more than a conference — it’s a working forum where ideas turn into action. Join us as we redefine the evidence-generation enterprise for drug development in the 21st century. Through dynamic plenaries and interactive sessions, you’ll engage directly with leaders across the ecosystem on the most pressing challenges and opportunities, including:
- Innovative approaches to patient identification, enrichment, and stratification
- Bridging the gap between drug development and clinical care
- Advancing truly patient-centric clinical trials
- Leveraging real-world data to generate decision-ready evidence
- Integrating novel methodologies to support faster, more effective regulatory decisions

Each day is designed to move beyond discussion, culminating in clear priorities and actionable next steps that continue long after the meeting concludes.

If you are committed to advancing therapies and improving outcomes for patients, CGIC 2026 is where you need to be. Don’t miss your opportunity to connect, collaborate, and help shape what comes next.

Room Block closing August 15!Register now: https://f.mtr.cool/qgwellyqnd

Newborns and premature infants represent some of the most vulnerable patients in medicine. They also remain among the most underserved in drug development.

On September 15 at the C-Path Global Impact Conference in Washington, D.C., a breakout session titled "Reimagining Neonatal Drug Development: Challenges and Opportunities" will move beyond restating what is broken and focus on what is actually possible. Panelists will examine near-term, practical opportunities to make neonatal therapeutic development more feasible, efficient and impactful, including improved trial design, meaningful endpoints, real-world data, biomarker development and the policy and investment levers that shape what gets developed and why.

Panelists include:
• Ralph Bax, Head of the Paediatric Medicines Office, European Medicines Agency
• Robert Nelson, Executive Director, Pediatric Drug Development, Johnson & Johnson
• Carolyn Shore, Director, Forum on Drug Discovery, Development, and Translation, National Academies
• Kanwaljit Singh, Executive Director, International Neonatal Consortium, and Director of Pediatric Programs, Critical Path Institute
• Lynne Yao, Director, Division of Pediatric and Maternal Health, U.S. Food and Drug Administration

The conversation will draw on perspectives from regulators, industry and cross-sector collaborative initiatives, reflecting the kind of partnership-driven approach to innovation in drug development that C-Path has built. This is an exemplar expression of C-Path's broader mission to transform how evidence of efficacy and safety is generated, interpreted and acted on, and why that transformation is especially urgent for patients who have waited the longest for solutions.

This session is one of many reasons CGIC 2026 is the meeting drug developers, regulators, researchers and patient advocates cannot afford to miss.

Room Block closing August 15!
Register now: https://f.mtr.cool/fxogpaqkxr

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/cmpcnkgtgm

Hope on the Horizon for ADTKD Patients Autosomal Dominant Tubulointerstitial Kidney Disease, or ADTKD, is ...

ICYMI | The Critical Path Institute Podcast: Connecting the Dots in Pediatrics

Critical Path Institute CEO Klaus Romero is joined by International Neonatal Consortium Executive Director Kanwaljit Singh to discuss the unique challenges and innovations in neonatal drug development. Neonates, particularly premature babies, are not simply smaller versions of adults or older children; their rapidly evolving physiology and complex medical conditions require specialized approaches in drug development. Singh explains how the INC fosters collaboration across pharmaceutical companies, academic researchers, healthcare professionals, parents, and global regulatory agencies to accelerate neonatal drug development.

A key achievement of the consortium has been the development of the Neonatal Adverse Event Severity Scale (NAESS), which standardizes the assessment of adverse events in neonates, a critical need since traditional adult or pediatric adverse event scales are inadequate for this population. The consortium is now working on NAESS 2.0, expanding the scale and creating a digital tool to facilitate its use in clinical trials.

Another major focus is the integration of real-world data and clinical trial data, creating one of the world’s largest neonatal databases with over 380,000 patient records from hospitals across the U.S., Canada, UK, and Japan. This database supports the development of disease progression models, such as for bronchopulmonary dysplasia (BPD), a common and severe lung condition in premature neonates, and the establishment of standardized reference ranges for lab values in neonatal intensive care units (NICUs).

INC is also initiating efforts around neonatal brain injury, collecting data to validate biomarkers as surrogate endpoints, which could accelerate clinical trials by providing earlier indicators of treatment efficacy.

🔗 Listen & subscribe now:
Apple: https://f.mtr.cool/cwmyswrzyv
Spotify: https://f.mtr.cool/xrznddnszy
YouTube: https://f.mtr.cool/lkrjhclfgu

The Critical Path Institute Podcast: Connecting the Dots in Pediatrics Critical Path Institute (C-Path CEO Klaus Romero is joined by Inter...

Be part of the global conversation shaping the future of drug development. The Critical Path Institute’s Global Impact Conference 2026 brings together regulators, industry leaders, scientists, clinicians, those with lived experience, and advocates for three days of high-impact dialogue and collaboration, all focused on accelerating better treatments for patients worldwide.

This is more than a conference — it’s a working forum where ideas turn into action. Join us as we redefine the evidence-generation enterprise for drug development in the 21st century. Through dynamic plenaries and interactive sessions, you’ll engage directly with leaders across the ecosystem on the most pressing challenges and opportunities, including:
- Innovative approaches to patient identification, enrichment, and stratification
- Bridging the gap between drug development and clinical care
- Advancing truly patient-centric clinical trials
- Leveraging real-world data to generate decision-ready evidence
- Integrating novel methodologies to support faster, more effective regulatory decisions

Each day is designed to move beyond discussion, culminating in clear priorities and actionable next steps that continue long after the meeting concludes.

If you are committed to advancing therapies and improving outcomes for patients, CGIC 2026 is where you need to be. Don’t miss your opportunity to connect, collaborate, and help shape what comes next.

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

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