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

Clinical Trials, Drug Discovery and Development, Life Sciences, Pharmaceutical, Pharmaceutical Regulation ,
  • July 16, 2013 | 10am BST (UK) / 11am CEST (EU-Central) / 5am EDT (NA)
  • 0 min

Recording not available for this webinar

Tap into the knowledge of two seasoned drug developers from Premier Research for a webinar packed with practical, experience-based guidance. Learn about the hurdles to overcome when studying rare diseases and find out how to skillfully navigate the clinical and regulatory challenges on the path to successful orphan drug development.

Speakers

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.

Who Should Attend?

VPs, Directors, Managers, Department Heads, Scientists and Researchers working within:

  • Clinical Affairs
  • Clinical R&D
  • Clinical Research
  • Clinical Pharmacology
  • Clinical Operations
  • Project Management
  • Regulatory Affairs
  • Medical Affairs

for pharmaceutical, biotechnology and medical device companies

Xtalks Partners

Premier Research

At Premier Research we focus on doing what we do best, and on putting all the pieces together to take our customers from proof of concept to regulatory approval. To do that, we have developed a surprising concentration of experience for a mid-sized CRO:
Over 600 trials for virtually every major class of analgesic, including every NSAID pain medication on the market today.
Three times as many medical device studies as the world’s largest CRO.
Pediatric experience with both familiar and extremely rare childhood illnesses, including work at PICUs, NICUs, clinics, and ERs – and a hard-to-match track record of successful neonatal studies.
For the future, we plan on continuing to focus on serving the needs of the small, highly innovative Pharmaceutical and biotechnology companies whose developments are changing medical history, especially in the areas of pediatric medicine and rare diseases. We will offer them everything they need to transform brave new ideas into life-saving therapies.

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