Critical Path Institute (C-Path)

Interviews from the 2024 C-Path Global Impact Conference are now live on our YouTube! In this insightful interview, Allison Moore, CEO and Founder of HNF, discusses HER participation in the 2024 Bio Conference and the CMT Clinical Readiness Summit, emphasizing the collaborative efforts made in addressing challenges faced by patients with Charcot-Marie-Tooth (CMT) disease and other rare conditions. She highlights the importance of patient registries, digital outcome measures, and innovative endpoints in clinical trials. She also expresses optimism about the growing partnerships and the necessity of advocacy groups in driving research and development. With a focus on creating a collaborative environment, the speaker believes that these efforts will foster impactful changes in the healthcare landscape, particularly in the commercialization of novel therapies. Watch now: https://lnkd.in/gd8VXvqp Save the date for CGIC 2025 which will take place September 9-11, in downtown D.C. at the Washington Marriott at Metro Center.  #CPath20Years #CGIC #DrugDevelopment #RareDiseases #Collaboration #DataSharing #GlobalHealth

Interviews from the 2024 C-Path Global Impact Conference are now live on our YouTube! Listen to Jill Platko, an expert from Suvoda, share her experiences and perspectives on the evolving landscape of electronic clinical outcome assessments (eCOA). Having been with Suvoda for nearly three years, she played a pivotal role in the establishment of their eCOA platform and the company’s membership in the eCOA Consortium. Platko emphasizes the significance of being part of this consortium to influence and develop best practices for eCOA implementation, rather than merely adhering to existing standards. Watch now: https://lnkd.in/gu3FXUHA Save the date for CGIC 2025 which will take place September 9-11, in downtown D.C. at the Washington Marriott at Metro Center.  #CPath20Years #CGIC #DrugDevelopment #eCOA 

📆 Register Now!    On April 9-10, 2025, the 2025 Clinical Outcome Assessment Program Annual Meeting will be held at the Bethesda North Marriott & Conference Center, Rockville, MD. Topics to be covered during this year’s event include:    ▶️ The potential of AI for COA development and deployment in clinical trials  ▶️ Regulatory pathways for using patient experience data pre- and post-approval: navigating benefit-risk assessment and communications consistent with labeling  ▶️ Unique considerations for collection of clinical trial endpoints in rare diseases  ▶️ Generating patient-centered evidence that addresses regulatory, access, patient, and other stakeholder expectations: navigating a varied landscape  ▶️ Current thinking on the accessibility and burden of eCOA & DHT system use  ▶️ High-level summaries of the ongoing activities and recent accomplishments of the COA Program including updates from the PRO Consortium, Rare Disease COA Consortium, and eCOA Consortium.    Register here: https://lnkd.in/gz68mxaz   #CPath20Years #COA #DrugDevelopment #Collaboration #PRO #RDCOA #eCOA #datasharing #GlobalHealth

Interested in C-Path updates? Be sure to subscribe at https://lnkd.in/gsQT98Rb 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. #CPath #drugdevelopment #neutral #datasharing #globalhealth #collaboration #regulatoryscience

SAVE THE DATE | We are thrilled to invite you to the 2025 Critical Path Institute Global Impact Conference (CGIC), scheduled for September 9-11, in downtown D.C. at the Washington Marriott at Metro Center. This can’t-miss event will build upon the monumental success of CGIC 2024 and provide a unique opportunity to connect the dots across key initiatives dedicated to accelerating drug development for conditions in neurology, rare diseases, pediatrics, and more. This year’s event takes on special meaning, as C-Path celebrates 20 years of impact. Be sure to join us to discover how C-Path can assist you in addressing challenges in drug development.  Engage with colleagues and advocates from diverse sectors, including industry, regulatory agencies, academia, patient organizations, and our vibrant community of those with living experiences. CGIC 2025 promises a dynamic agenda, bringing together a multitude of voices and perspectives to foster collaboration and open dialogue. Stay tuned for forthcoming communications, including the full conference agenda, featured speakers, and more. Your participation is key to making this event a resounding success! Date: September 9-11, 2025 Location: Washington Marriott at Metro Center, Washington, D.C. Registration: Coming soon, with Early Bird pricing! We look forward to seeing you there! #CPath20Years #Collaboration #DrugDevelopment #DataSharing #GlobalHealth #CGIC

Interviews from the 2024 C-Path Global Impact Conference are now live on our YouTube! Listen to Jonathan Davis, MD, Vice-Chair of Pediatrics and Chief of the Division of Newborn Medicine at Tufts Children's Hospital at Tufts Medical Center, discuss the significant efforts and achievements of C-Path in collaboration with the International Neonatal Consortium to improve drug development for preterm infants, a population that has been historically underserved. He outlines the challenges faced in acquiring and analyzing extensive data on neonatal drug exposure and outcomes, emphasizing the urgency of addressing the high rates of prematurity and the lack of therapeutic options. With a successful data collection effort that amassed 400,000 patient records, the initiative aims to optimize clinical trials by identifying high-risk infants and using existing data as simulated controls. He also highlights the need for standardized definitions of conditions like bronchopulmonary dysplasia (BPD) and stresses the importance of collaboration among various stakeholders, including parent groups, nursing organizations, and regulatory agencies, to enhance outcomes for vulnerable neonates. Watch now: https://lnkd.in/gTe5baQx Save the date for CGIC 2025 which will take place September 9-11, in downtown D.C. at the Washington Marriott at Metro Center.  #CPath20Years #CGIC #DrugDevelopment #INC 

#NationalKidneyMonth | The Rare Inherited Kidney Disease Research Team at Wake Forest University School of Medicine led by Anthony Bleyer, MD, MS is excited to join C-Path’s Polycystic Kidney Disease Outcomes Consortium led by Sorin Fedeles, PhD, MBA on a new collaboration to analyze clinical and laboratory data that will help evaluate prognosis in autosomal dominant tubulointerstitial kidney disease and help set the stage for future clinical trials. Wake Forest’s team works closely with families who suffer from this rare disease. ADTKD is different from other kidney diseases because the only clinical feature seen in families is inheritance and slowly progressing kidney disease often leading to kidney failure. This is devastating for families, as the age of kidney failure for their loved ones ranges from as young as 20 years old through over 80 years old, and what causes this variation is still unknown. Dr. Bleyer has been working with families since 1996 to identify the cause of their disease. At that time less than 50 families were described in the literature. In 2002, our team identified the first cause of ADTKD as pathogenic variants in the gene UMOD. Followed by REN (2009), MUC1 (2012), SEC61A1 (2016) and most recently APOA4 (2023).  We now have over 400 families identified with ADTKD and over 1000 individuals in our registry. Our team follows the mission statement of “Helping one patient, one family, at a time.” We believe that every patient is important, and we know that we wouldn’t be part of PKDOC today without the help of families who work with us to understand how this disease progresses and hope for finding a treatment in the near future. In honor of National Kidney Month, we wanted to reach out to a family that has been part of our ADTKD study since 2004. We asked how participating in research impacts their family: “When a silent genetic disease has claimed your mother, tormented your youngest brother, and threatens the lives of your children, hope becomes essential. ADTKD research has given us that hope. With the dedication of brilliant doctors and their teams of scientists, we will see this hope transform into a plan—and ultimately, a life-changing treatment for generations to come.” - Aleta V Hickey, ADTKD-UMOD. Please read through to her family’s journey shared with their local newspaper, Plymouth Independent on February 26, 2025: https://lnkd.in/e8baCf9s #CPath20Years #Collaboration #DrugDevelopment #DataSharing #GlobalHealth #AreYourKidneysOK #WorldKidneyDay 

Interviews from the 2024 C-Path Global Impact Conference are now live on our YouTube! Listen to C-Path's VP of Global Affairs, Cécile Ollivier, discuss the evolution and current activities of C-Path's two-decade existence, particularly emphasizing its efforts in the United States and Europe. Initially, C-Path was informally involved in regulatory science and collaboration with regulatory authorities but has since expanded its influence significantly. With offices in Amsterdam and a focus on regulatory science, C-Path aims to harmonize drug development efforts across regions, particularly in the context of high unmet medical needs such as rare diseases and tuberculosis. The organization collaborates with various stakeholders, including the European Medicines Agency (EMA) and the World Health Organization (WHO), to facilitate data sharing and regulatory processes effectively. As C-Path continues to grow, it seeks to extend its reach to other regions globally, ensuring that innovative solutions address healthcare challenges worldwide. Watch now: https://lnkd.in/gQpDnm6s Save the date for CGIC 2025 which will take place September 9-11, in downtown D.C. at the Washington Marriott at Metro Center.  #CPath20Years #CGIC #DrugDevelopment #EU #RegulatoryScience #GlobalHealth #Collaboration

In 2025, C-Path proudly celebrates 20 years of impact. Take a look back at the highlights and the incredible individuals who made it all possible. Here's to the next chapter of innovation! For the past 20 years, Critical Path Institute has led collaborations that accelerate drug development and advance better treatments for people worldwide. C-Path orchestrates the development of these actionable solutions through an innovative, collaborative approach to the sharing of data and expertise. C-Path builds consensus among participating scientists from industry and academia with regulatory participation and iterative feedback. Such consensus provides the mechanism to generate the necessary confidence to assure the adoption of the medical product development solutions by sponsors and regulators. Examples of pathways through which this confidence is achieved include informal and formal regulatory pathways. Through these various mechanisms, sponsors can confidently adopt the solutions generated through C-Path’s collaborative approach, thus ensuring the continuous optimization of the medical product development process.  Check out our 20 milestones for 20 Years here: https://lnkd.in/dn8jDgDR #CPath20Years #Collaboration #DrugDevelopment #DataSharing #GlobalHealth

REGISTER NOW for our April Webinar: ACE Inhibitors as a Cardioprotective Treatment in Dystrophinopathies. ACE inhibitors have played a groundbreaking role in heart failure treatment—not only in the general population but also in Duchenne and Becker muscular dystrophies cardiomyopathy. Despite strong evidence supporting their benefits, these treatments remain underutilized in DMD patients, even those with significant cardiac dysfunction. Join us on April 17, noon ET, for an insightful presentation by Dr. Karim Wahbi, Professor of Cardiology at the University of Paris and head of the Reference Center for Neuromuscular Cardiomyopathies. Dr. Wahbi specializes in the clinical management of cardiomyopathies, with a focus on neuromuscular diseases like Duchenne muscular dystrophy. This webinar will explore: ▪️ How ACE inhibitors slow the progression of myocardial dysfunction in DMD and BMD ▪️ The research-backed rationale for their use in expert guidelines ▪️ How they’ve paved the way for broader heart failure treatments, including MRAs, beta-blockers, sacubitril/valsartan, and SGLT2 inhibitors This webinar will summarize key findings and encourage conversation on this critical topic. 🔗 Register now: https://lnkd.in/gEGg9f3x Collin Hovinga Ramona Belfiore-Oshan Alexandre Bétourné, PhD, PharmD, PMP Laura Hopkins, MS, MLS #CPath20Years #globalhealth #raredisease #datasharing #drugdevelopment #Duchenne #Becker #Cardiomyopathy #HeartFailure #MedicalResearch #Webinar #collaboration