A Rare Look into New Guidances: The Future State of Orphan Drug Development

Adam Bloomfield, MD, FAAP, Vice President, Internal Medicine, Rare Disease and Pediatrics, Medical Affairs, Premier Research

Dr. Adam Bloomfield oversees the Rare Disease and Pediatrics Medical Affairs team at Premier Research. He brings to Premier extensive US and global experience in medical affairs leadership and clinical development strategy from his previous work at AstraZeneca, Sobi and Moderna. In his current role, he provides medical monitoring oversight, therapeutic knowledge to study programs and supports business development and consulting activities in the fields of Rare Disease and Pediatrics. A pediatrician by training, Dr. Bloomfield has over fifteen years of clinical experience treating thousands of patients and brings a patient-centric perspective to research programs.

Dr. Bloomfield completed his pediatric residency training at New York University Medical Center. He holds a Doctor of Medicine from Rutgers New Jersey Medical School and a Bachelor of Science in Mathematics from the University of Michigan – Ann Arbor. He is a fellow of the American Academy of Pediatrics and board certified by the American Board of Pediatrics.

Leslie Wetherell, Executive Director, Program Strategy, Rare Disease and Pediatrics, Premier Research

Leslie Wetherell, Executive Director, Program Strategy, Rare Disease and Pediatrics, is a clinical research professional with over 25 years of global drug development experience, with a focus in rare disease. In her role, Leslie’s therapeutic and operational expertise supports biotech and pharmaceutical companies to achieve key results in rare disease and pediatric studies. Her focus is producing strategic efficiencies that support patient-centric and timely program delivery.

She has extensive phase I-III experience in rare disease, oncology, hematology and general medicine and is accomplished in gaining market/FDA approvals, having led seven phase III FDA-approved products over the course of her career.

Leslie joined the Premier team in her current role in 2023. Prior to her tenure with Premier, her past roles have included data management, project management, procurement and global strategy development positions.

Paulla Dennis, Executive Director, Program Strategy, Rare Disease, Pediatrics, Cell and Gene Therapy, Premier Research

Paulla Dennis has been dedicated to rare disease and pediatric clinical research for more than 25 years, specializing in gene therapy for the last 10+ years. She is highly experienced in operational delivery after spending 17 years at a pediatric hospital running the Clinical Research Services group, where she delivered pivotal studies in several rare pediatric disorders inclusive of gene therapies. Paulla has an additional 8 years’ experience in the CRO setting in startup project management as well as in program strategy, delivering rare, pediatric disease and advanced therapies studies.

As Executive Director, Program Strategy, Paulla advises, consults, plans and directs the design and strategy for prospective and ongoing rare, pediatric and gene and cell therapy studies. In her current role, she provides strategies and solutions for operational excellence in this complex clinical research setting with a focus on patient and family pathway planning and comprehensive site assessment and educational support.

Paulla has extensive experience in phase I-IV studies in a global setting, including autosomal, neurological, metabolic and endocrine diseases. Her key experience areas include rare/ultra-rare disease, pediatrics, gene therapies and neurological (neuropsychological, neurodegenerative and neuromuscular) indications.