The Complexities of Pediatric Rare Disease Clinical Trials: Regulatory and Operational Strategies for Success

Life Sciences, Clinical Trials,
  • Monday, February 28, 2022

Strategic planning and execution are crucial when implementing rare disease clinical trials, especially when factoring in the added complexities of pediatric rare disease trials.

There are many operational and regulatory challenges to consider when planning for pediatric rare disease clinical trials, including recruitment, elevated regulatory scrutiny, blood volume restrictions and supply chain logistics. Additionally, the COVID-19 pandemic has brought additional challenges that have further intensified the need for strategic planning and flexibility to best position a trial for success.

Without careful, strategic planning to tackle these challenges, trials may be fraught with operational hurdles which can delay recruitment and other study milestones. Understanding potential challenges and pitfalls and setting realistic regulatory expectations are critical when developing study timelines and strategies. Identifying potential challenges before they arise while mitigating associated risks ensures studies are best positioned for success.

Join this webinar with Medpace experts on Rare Disease Day 2022 as they share operational and regulatory challenges associated with pediatric rare disease clinical trials, along with strategies to help anticipate and overcome them.


Gregory Hale, MD, Senior Medical Director, Hematology & Oncology, Medpace

Dr. Gregory Hale is a physician with over 28 years of experience in all phases of clinical development, with expertise in hematopoietic stem cell transplantation, cellular and gene therapies and immuno-oncology. Dr. Hale has served as clinical director of the Transplant and Gene Therapy Program at St. Jude Children’s Research Hospital and medical director of the Division of Hematology/Oncology at Johns Hopkins All Children’s Hospital. Most recently he was Professor of Oncology at Johns Hopkins and has authored more than 200 peer-reviewed manuscripts, review articles and book chapters. He has held leadership positions in the National Marrow Donor Program (NMDP) and the Center for International Blood and Marrow Transplant Registry (CIBMTR).

He earned his medical degree from the Joan C. Edwards School of Medicine at Marshall University. He completed his pediatrics residency at Children’s Hospital of Pittsburgh and his pediatric hematology/oncology fellowship at St. Jude Children’s Research Hospital.

Message Presenter

Tanya Konovalenko, MPharm, RAC, Director, Regulatory Affairs, Scientific and Strategic Development, Medpace

Tanya Konovalenko has over 25 years of international experience in drug development of small molecules and biologics through all phases of clinical research, authorization and post approval. She has worked in Canada and Switzerland and has served as a principal interface for interactions with major regulatory agencies including EMA, FDA, Health Canada and various National Competent Authorities in Europe. Tanya has extensive experience with pre-submission meetings, interactions and negotiations with the agencies, and has designed and executed effective global regulatory strategies, submission review and post approvals.

Message Presenter

Kyle Haas, MBA, Associate Director, Clinical Trial Manager, Medpace

Kyle Haas has over 7 years of experience in clinical research with a strong background in healthcare. As a Clinical Trial Manager, he has collaborated with key opinion leaders and Sponsors worldwide to successfully coordinate and manage operations of Phase 1 to 3 neuromuscular trials. Kyle started at Medpace as a Clinical Research Associate monitoring and managing sites. Kyle obtained his Master of Business Administration and degree in Biology at the University of Cincinnati.

Message Presenter

Who Should Attend?

VPs, Directors, Managers and Department Heads working within:

  • Clinical Affairs
  • Clinical Research
  • Clinical Pharmacology
  • Clinical Outsourcing
  • Project Management
  • Regulatory Affairs
  • Medical Affairs

What You Will Learn

In this webinar, you will learn about the complexities faced in pediatric rare disease clinical development from Medpace experts. Topics will include:

  • Key considerations of pediatric rare disease clinical trials
  • Common operational and regulatory challenges
  • Mitigating the risks of pediatric rare disease trials

Xtalks Partner


Medpace is a scientifically-driven, global, full-service clinical contract research organization (CRO) providing Phase I-IV clinical development services to the biotechnology, pharmaceutical and medical device industries. Medpace’s mission is to accelerate the global development of safe and effective medical therapeutics through its high-science and disciplined operating approach that leverages local regulatory and deep therapeutic expertise across all major areas including oncology, cardiology, metabolic disease, endocrinology, central nervous system and anti-viral and anti-infective.

You Must Login To Register for this Free Webinar

Already have an account? LOGIN HERE. If you don’t have an account you need to create a free account.

Create Account