Worldwide Orphan Drug Designation Applications and Requirements March 2012

Mar 6, 2012
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Course Description

Globally, there is a need for orphan drug research, development, and approval for underserved patient populations who have diseases that affect very few individuals. While this patient population has been largely ignored, some pharmaceutical companies have built their research pipeline around these patients. Receiving orphan drug designation and approval confers many benefits to the developer to compensate for the development costs of the drug. This web seminar will explore which countries allow orphan drug designations, the application requirements, population limits and how to support this number, how the applications are the same, who to submit the application to, if and when the application can be changed, and how it needs to be supported over the development process.


Learning Objectives

  • Identify the countries that have orphan drug designations
  • Identify, on a global basis, the differences and similarities of these applications (and how to re-use the information), including differences in population requirements
  • Discuss the importance of the indication chosen for designation, and how this ultimately affects the final label and how this can differ among countries
  • Examine the most difficult sections of the applications and mitigate agency concerns
  • Discuss the benefits of orphan designation
  • Describe how orphan drug indications and applications can be modified
  • Describe the timing of the application in the drug development process

Who Should Attend

  • Individuals who want an introduction to the orphan drug designation on a global scale
  • Regulatory Affairs Professionals
  • Clinical Research Professionals
  • Research and Development Professionals
  • Project Managers


Meredith Brown-Tuttle, R.A.C. 

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 For more information, contact Naila Ganatra at (215) 413-2471.