Rare Disease Trials: The Beginning, the Middle and the End

Life Sciences, Clinical Trials, Pharmaceutical Regulation, Pharmaceutical,
  • Monday, May 12, 2014

This session includes a regulatory overview of rare diseases and orphan drug status as well as practical considerations for varying stages of the development process.

Viewers will learn more about conducting successful Natural History studies and the presenters will review how to avoid common challenges through lessons learned. Additionally, there will be a discussion of best practices for running a program of studies in parallel. Helpful hints for preparing for the final regulatory submission will also be provided (including key factors for when studies are ongoing at the time of the submission).


Angi Robinson, Executive Director, Pediatrics and Rare Diseases, 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.

Message Presenter

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 Partner

Premier Research

Premier Research is a leading global contract research organization serving highly innovative biotech, Pharmaceutical and medical technology companies.The company has a wealth of experience in rare disease and pediatric research having managed about 100 projects in each area in the last five years alone. Its services include clinical research and regulatory outsourcing in the areas of analgesia, neurology, cardiovascular, dermatology, oncology and medical devices.

Premier Research has 24 offices across the globe. It employs over 1,000 clinical professionals. This includes a strong international network of monitors and project management professionals combined with regulatory, data management, statistical, scientific, and medical experts, and staff at its well-established network of dedicated clinical sites.

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