Strategies for Rare Disease Studies and Orphan Drug Development in the US

Angi Robinson, Executive Director, Scientific Account Leader, Premier Research

Angi Robinson has been with Premier Research for over 12 years and has provided executive oversight and full management support for numerous studies, including global studies with a focus in paediatrics and rare diseases. Ms. Robinson has supported FDA Pre-IND meetings, IND submissions and BLA & NDA directorship. Ms. Robinson’s experience includes multiple study designs including PK/PD, adaptive design, FDA Fast Track designations and she has directed several studies requiring the oversight of Data Safety Monitoring Boards and Data Monitoring Committees. She has supported NDA submissions, including a pivotal studies resulting in product approval. Ms. Robinson is the Global Project Director for Good Clinical Practice Journal’s (GCPj) 2008 Clinical Research Team of the Year for a pivotal clinical trial in a rare disease.

Charlene Sanders, Vice President, Global Regulatory Affairs & Pediatric Strategic Consulting, Premier Research

Dr. Sanders’ brings extensive regulatory expertise including more than 18 years of Pharmaceutical management experience to Premier Research. Her experience in drug development and regulatory affairs includes interactions with both regulators and major Pharmaceutical companies. Dr. Sander’s therapeutic experience in oncology, neuroscience, pain and pediatrics is well aligned with Premier Research’s therapeutic focus and prowess in executing clinical trials targeting rare diseases and orphan drug status . She trained in pediatrics and has had post-doctoral specialty fellowships. She has held teaching faculty appointments at the Children’s Hospital of Los Angeles, Morgan Stanley Children’s Hospital (Columbia University Medical Center), Children’s Hospital of Boston (Harvard University) and Yale University. She received her Medical Degree from the University of Pennsylvania School of Medicine and has practiced clinical and research medicine for three decades.

Dr. Sanders is able to leverage her career experiences, both in global regulatory strategy and medical practice, by bringing her extensive knowledge to the Premier Research team. Dr. Sanders has recurrent success in providing clinical design input and direct protocol review that overcome regulatory hurdles associated with research trials, as well as broad regulatory, clinical and translational research expertise in US regulatory healthcare policy environments. She is an expert in pediatric regulatory requirements and orphan drug regulatory pathways. She has strong familiarity with non-US healthcare delivery and regulatory research models used by European and Australian pharma and biotech industry organizations, as well as experience in Pan-Pacific regulatory and business environments.