August 20, 2024
Martina Bebin, MD, MPA
Director, UAB Tuberous Sclerosis Clinic

With the advances in drug development and the potential for gene therapy on the horizon for a number of pediatric neurology diseases, understanding the path for drug development and FDA approval is essential. As a practicing pediatric neurologist, with a special interest in epilepsy and rare neurologic diseases, the role of precision medicine is becoming an important aspect in clinical decision-making. It is important to remain aware of new FDA approved therapeutic options and their indications and how one would incorporate these therapies into your clinical practice.  For pediatric neurologists completing their training at an academic university or who have interest in clinical trial development, involvement in clinical trials during your training or early career has tremendous value. Most importantly, you provide greater therapeutic options for your patients; develop a valuable understanding of the therapy under investigation and experience of how it would be used in your clinical decision-making once approved by the FDA. 

It not infrequent, that a parent may ask you about a new FDA approved therapy that is approved for patients 18 years of age and older and if “you would prescribe it off label”.  Having the understanding of the FDA drug approval process, information about ongoing pediatric clinical trials is of great value. You then have the ability to counsel the parent and provide guidance about potential participation in a pediatric clinical trial and facilitate the referral for screening and potential enrollment in the trial. One of the most important lessons I have learned from over 20 years of clinical trial development, is the beneficial aspects that a clinical trial provides for your patient and parent(s).  Participant safety of the utmost importance and the close serial study visit follow up provides a level of care above the standard clinical follow up. The clinical trial experience is a win-win for both the participant/parent as well as, the site investigator, which ultimately helps facilitate new therapeutic development and treatment options.  

Enroll Today

Learn more about the role of clinical trials in pediatric neurology by taking the CNF course on Clinical Trials. This course provides valuable insights from both participant and clinician perspectives and offers CME credits upon completion. Click here to Enroll today! 

As the PI of a multi-centered Phase IIb clinical trial, I was impressed that the majority of inquiries regarding potential enrollment came from interested parents and not their neurologist. This factor confirmed the power of social media, rare disease advocacy groups, parental courage, and keen interest to improve the child’s life. As a result, if our center were not involved in a clinical trial that would be of potential benefit, I facilitate referring them to the closest participating site and call the site PI for additional information.  It is essential that as pediatric neurologist we always consider clinical trial options for our patients faced with significant burden of their disease and improvement in their quality of life. 

This CNF course is focused on Clinical Trials in Pediatric Neurology and provides valuable information from both the participant/parent and clinician perspective that can be incorporated into your clinical practice. It is also an opportunity to earn CME credits for completing this CNF course. 

Clinical trials play a crucial role in advancing pediatric neurology care. By participating in or referring patients to these trials, pediatric neurologists can offer cutting-edge therapeutic options and contribute to the development of new treatments that improve patient outcomes.

Author Bio

Dr. Bebin is Professor of Neurology and Pediatrics at the University of Alabama at Birmingham. Her primary research interest is in tuberous sclerosis complex (TSC) and has been involved in several NIH funded TSC clinical research projects. She is director of the UAB TSC Center of Excellence and is actively involved in clinical epilepsy and TSC research for the past 25 years. She also serves on the TSC DOD-CDMRP panel, FDA Orphan Product Development grant review panel, ABPN MOC Child Neurology Committee, and past member of the TSC Alliance Board of Directors.

About Child Neurology Foundation

The Child Neurology Foundation (CNF) is a leading nonprofit organization dedicated to supporting children and their families living with neurologic conditions. Our vision is to create a world in which all children affected by neurologic disorders reach their full potential. We work closely with families, healthcare professionals, researchers, and advocacy groups to provide essential resources, foster connections, and drive research and innovation in the field of child neurology.

For more information, please visit our homepage at www.childneurologyfoundation.org or contact us at [email protected].

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