3 Key Trends Transforming the Future of Rare Disease Clinical Development

Rare disease clinical development is undergoing rapid transformation, driven by scientific innovation, evolving regulatory frameworks and the need for more flexible, patient-centric trial designs. This webinar will explore the current rare disease clinical development landscape and the key trends shaping its future.

The webinar will highlight three major forces redefining how rare disease studies are designed and executed. First, the featured speaker will examine the rise of customizable platform trials and how adaptable trials can accelerate timelines while maintaining scientific rigor. Second, panelists will explore emerging technologies in rare disease therapeutics, including the growing role of ex vivo clinical trials and practical insights from cell therapy studies initiated with effective study start-up and patient recruitment strategies. Finally, the session will address the impact of new global regulatory laws and expedited pathways, such as RDEP and China’s Fast Track IND, with special attention to regulatory preparedness for ultra-rare indications.

Register for this upcoming webinar in recognition of Rare Disease Day 2026. Learn how to align innovative science with global regulatory expectations to successfully advance rare disease clinical development and programs to drive meaningful change for patients.

Speakers

Ann Woolfrey, MD, Vice President, Medical Department, Medpace

Dr. Ann Woolfrey is board certified in internal medicine and pediatric hematology/oncology and provides clinical and scientific expertise in cellular therapies, transplantation immunology, hematology/oncology and gene therapy. Dr. Woolfrey joined Medpace nine years ago, after a 25-year career in translational and clinical research in cell and gene therapies at the Fred Hutchinson Cancer Research Center (FHCRC)/University of Washington (UW). In addition to her role as the Medical Director of the Unrelated Donor Program at FHCRC/Seattle Cancer Care Alliance, she was Principal Investigator on numerous clinical trials and has more than 150 scientific publications in the fields of stem cell transplantation, transplant immunology, HIV and autoimmune diseases. Since joining Medpace, Dr. Woolfrey has focused on supporting biotech in the development of cellular and gene therapies.

Message Presenter

Tanya Konovalenko, MPharm, RAC, Director, Regulatory Affairs, 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

Jackie Widmer, Director, Clinical Trial Management, Medpace

Jackie Widmer has over 18 years of global industry experience leading Phase I-IV studies across multiple therapeutic areas, including over 13 years of Medpace global project management in progressive leadership roles. She has over five years of pharmaceutical experience leading global rare disease and gene therapy clinical development programs.

Message Presenter