Regulatory Affairs for Orphan Drugs

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Course Overview


Orphan drugs are vital for those people with rare diseases and this is recognised by Regulators in Europe and the US. By gaining orphan designation, pharmaceutical companies access various incentives to encourage them to develop therapeutics for patients with rare diseases. However, it is important the system is not misused and that you fully understand the regulatory requirements and roadmap.


This training course will provide you with a comprehensive understanding of the opportunities and challenges offered by orphan drug designation. Once completed, you will understand the regulations and whether they are applicable to your product. Through practical exercises you will examine multiple orphan drug examples so you have the confidence to navigate the regulations.


With the Educo Post Learning Implementation Plan (PLIP) you will have the framework to apply and implement the knowledge acquired in the training, supporting you in your development. Learn more about how we deliver live online training.


Key Learning Objectives


  • Examine the orphan drug regulations in the EU and US
  • Review multiple case study examples of orphan drugs approved by the EMA and FDA
  • Discover the regulatory processes intended to accelerate early access to innovative medicines
  • Examine the orphan drug designation process in the EU and US
  • Get first-hand insights on how to get the best from orphan drug regulations
  • Analyse the maintenance of an orphan drug designation in preparation for MAA/NDA
  • Briefly examine the orphan drug process globally
Post Learning Implementation Plan (PLIP)


  • Access to the Educo Training Portal
  • Submit your needs analysis and objectives for training by completing a simple online form

Action Plan

  • At the end of the training you will create an action plan to help you implement and develop your new skills and knowledge

Reinforcement Session

  • Delivered 3 to 4 weeks after the training
  • It is a 1 hour virtual session enabling the group to reinforce the key learnings from the training and cover and subsequent questions and challenges
Module 1

Orphan medicinal product legislation

  • Overview of the regulations in the EU, US and Japan
  • What the regulations cover and why, what they try to protect from (i.e. creation of false sub-populations of a non-orphan condition)
  • Awards for obtaining ODD
  • Considerations for Orphan Drug Designation
    • Sequence of submissions by country
    • Developing orphan versus non-orphan indications
    • Paediatric conditions including the challenges and impacts in this area, trade-off of the incentives and the ongoing evaluation of the orphan regulation by the EC


Module 2

Obtaining orphan drug designation

  • Orphan Drug Designation in the EU
    • Review the application
    • Examine the procedure
  • Similarities and differences with the US
    • Application, Procedure and Incentives
  • Rare diseases: a global issue
    • Collaboration between Agencies
  • Strategic considerations on when to apply and to what Agencies
Module 3

Maintenance of orphan drug designation

  • Examine what you need to do ahead of your dossier application
    • Marketing Authorisation Application (MAA)
    • New Drug Application (NDA)
    • Discuss policy 43 and its impact
  • Examine what you need to do during an application
    • MAA and NDA
    • Discuss the Orphan Maintenance Assessment Report (OMAR)
    • Assessment of similarity and significant benefit
Module 4

A global perspective of orphan drug designation –

  • Japan, Australia, Russia, China, Switzerland



This course is designed for regulatory affairs professionals who need to learn more about the orphan drug designation process in Europe and the US.

Could your team benefit from this course?

We can customise this course to meet the requirements of your organisation, delivered as a classroom and/or online course.

For more information you can email us at [email protected] or call +44 (0) 203 111 7357.

Or please summarise your objectives by completing the form below.

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• The training course is delivered either online or in a classroom. Please check which course format you have booked.
• For live online courses, dates and times of modules are subject to change
• Please contact us for the module dates of future deliveries.
• For live online courses, module durations are approximate.