Overcoming the Challenges of Conducting Clinical Studies in Rare Pediatric Populations

Drawing from their own experiences designing, planning and executing rare and pediatric clinical studies, PRA experts use relevant examples and case studies to discuss the unique challenges of conducting clinical studies of rare diseases in pediatric populations.

By their very nature, clinical studies in rare disease populations pose unique scientific, medical, ethical, and logistical challenges. Innovative solutions to overcome these hurdles are desperately needed so that clinical studies can be completed in a timely way to bring these new therapies to rare disease patients. Compounding this issue are the unique challenges of conducting studies in the pediatric population. Physiological and psychological diversity of pediatric age groups can affect study designs and increase timelines and costs.

In this webinar, our experts will highlight some of the key challenges associated with conducting research within rare pediatric populations that are few in number to begin with, are geographically dispersed and are often not located near a research center. Factors that have to be taken into account include, regional and local regulatory requirements, legal differences that impact consent/assent, local infrastructure, availability of specialist centers for multidisciplinary approaches and more. The speakers will describe patient-centric approaches to support engagement, recruitment, and retention, while recognizing and accounting for cultural diversity. They will describe strategies to ensure patients, parents and caregivers have a seat at the drug development table. They will discuss how technology is being used to address some of these challenges and look ahead to how virtual studies may drive the future of bringing new therapies to rare disease patients.

Speakers

Scott Schliebner, Vice President, Rare Disease - Scientific Affairs, PRA Health Sciences

Scott Schliebner, MPH, Vice President, Rare Disease – Scientific Affairs, is a clinical strategist with a 20+ year background in clinical development specializing in rare diseases and orphan drug development. His breadth of experience encompasses all trial phases; a broad variety of study designs; interventional and observational studies; involving small molecules, biologics, gene therapies, and enzyme replacement therapies. His therapeutic expertise focuses on rare tumors, rare hematologic disorders, neuromuscular diseases, inborn errors of metabolism, and lysosomal storage disorders.  Mr. Schliebner provides strategic and scientific consultation aimed at accelerating the development of orphan drugs and bringing new therapies to rare disease patients faster.

 Mr. Schliebner has served in leadership roles across the biotech, non-profit, and CRO settings, with a focus on innovative approaches to accelerate rare disease clinical development.  He has developed close working relationships with several rare disease research consortia, patient advocacy organizations, study research groups, and key opinion leaders. He is regularly asked to speak on patient-centric approaches, leveraging medical informatics, and new strategies to develop rare diseases therapies in emerging markets.  He is a member of the DIA Rare Disease Planning Committee, holds an MPH degree from the University of Utah, School of Medicine, and completed a Graduate Research Fellowship at the NIH.

Message Presenter

Mark Sorrentino, MD, Vice President, Center for Pediatric Clinical Development, PRA Health Sciences

Mark Sorrentino, MD, Vice President, Scientific Affairs – Pediatric Solutions, has 20 years of experience in the pharmaceutical and biotech industries and currently leads PRA’s global pediatric center of excellence. Prior to joining PRA, Dr Sorrentino spent 5 years as the global chair and founder of the pediatric practice area at PPD overseeing all aspects of strategy and pediatric clinical development, as well as the global therapeutic head of critical care. Before joining PPD, Dr Sorrentino served as the global chief medical officer at ADMA Biologics, where he oversaw all clinical development operations, and as director of medical science at MedImmune, where he was responsible for development and oversight of numerous Phase 4/post-marketing studies across several therapeutic areas, including infectious diseases, respiratory diseases, and transplant medicine. As a former consultant to biotechnology firms, Dr Sorrentino has also overseen implementation of medical information systems. Having earned his medical degree and master’s degree in clinical nutrition from the Chicago Medical School, Dr Sorrentino completed both a residency in pediatrics and a fellowship in pediatric critical care at Children’s National Medical Center (CNMC) in Washington, DC. Currently, Dr Sorrentino holds an appointment as an assistant clinical professor at the George Washington University School of Medicine.

Prior to joining the industry, Dr. Sorrentino was an attending physician in the CNMC pediatric intensive care unit for 8 years and participated in numerous industry-sponsored critical care trials. He holds active medical licenses in Washington, DC and Maryland and serves as the medical director for all Susan G Komen events in the DC area. An active medical team member of Team Rubicon, Dr Sorrentino sits on the Maryland Responds advisory board, and he has also volunteered on several international medical missions. In addition, he is on the board of directors for the non-profit life sciences education company, the CMR Institute.

Message Presenter