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Regulatory Strategies for Orphan Drugs Training Course

This must-attend event will provide an essential overview of the opportunities and challenges presented by orphan drug regulation.

★★★★★ "Superb, excellent delivery and very knowledgeable [...] the best training course I attended in a log... more"

Need help?  Customise

  • Format: Bespoke training
  • CPD: 6 hours for your records (depending on your requirements)
  • Certificate of completion

Course overview

The market for orphan drug products continues to expand and developing new treatments for rare diseases is becoming an increasingly important growth area for many pharmaceutical companies. This interest in rare and orphan diseases is linked not only to the incentives offered by the different regulatory authorities but also to the recent development of advanced therapies.

The interactive course will cover all the key aspects of orphan drug legislation in both the US and EU and the regulatory processes designed to enable early access to innovative medicines. The programme will help you to assess the strategic considerations of when to apply for orphan designation and to which agency and look at the development of advanced therapies and using orphan drug designation with your ATMP strategy.

A case study session on how to prepare and manage an orphan drug submission will consolidate learning and ensure that you leave the event with practical skills and knowledge that can be put to use in the workplace. The course will allow you not only to learn from our expert trainer but also to share the experiences of other delegates.

Benefits of attending:
  • Gain an overview of the orphan medicinal product legislation in the EU and the US
  • Receive practical advice on how to prepare and manage orphan submissions
  • Assess strategic considerations of when to apply for orphan designation and to what agencies
  • Discuss the use of orphan drug designation with your ATMP strategy

Who should attend?

This event will be particularly relevant to regulatory affairs professionals who are involved in orphan drug submissions. It will also be of interest to those working in rare diseases who would benefit from an overview of orphan drug designation.

The Regulatory Strategies for Orphan Drugs course will cover:

Orphan Medicinal Product Legislation

  • Overview of the legislation in the EU, US and Japan
  • What the Regulations cover and why, what they try to protect
  • Benefits of Orphan Drug Designation
  • Key Considerations
    • Country Submissions
    • Differences of Orphan versus non-orphan
    • Paediatric Conditions

Obtaining Orphan Drug Designation

  • Orphan drug designation in the EU
    • Application
    • Procedure
  • Similarities and differences with the US
    • Application, procedure and incentives
  • Global Issues around Rare Disease
  • Strategic Considerations
  • Case Study

Maintenance of Orphan Drug Designation

  • How to maintain
  • Maintenance during MAA and NDA
  • Assessment and Case Study

Global Environment

Marloes van der Geer

Marloes van der Geer works as senior regulatory affairs scientist at Qdossier. Marloes holds a master in Life Sciences (Drug Innovation) from the University of Utrecht, the Netherlands. For nine years she held several positions at Hoffmann-la Roche in Switzerland, including the role of regulatory intelligence manager and regulatory policy lead for the EMEA region. Marloes has been actively involved in several industry associations like EFPIA and IFPMA. Also, she contributed to the ongoing IMI PREFER project in Europe, focused on the generation and use of patient preference data. In 2019, Marloes joined Qdossier. Marloes’ areas of expertise are: eCTD, lean authoring, publishing, biopharmaceuticals (incl. biosimilars), clinical development, regulatory convergence & harmonization and facilitated registration pathways (incl. orphan drugs), local RA activities (e.g. product information management).

More details

Reviews of IPI's Regulatory Strategies for Orphan Drugs training course

Superb, excellent delivery and very knowledgeable [...] the best training course I attended in a log time!

Jun 20 2022

John McIntyre
Senior Consultant, Parexel Ireland USD

Mar 16 2022

Excellent would attend other similar webinar/trainings

Cheryl Dicks
Sr. Mgr. Regulatory, CANbridge Pharmaceuticals

Mar 10 2021

I liked the webinar very much, because it provided a comprehensive and detailed overview of the ODD process in the EU and other countries. Andrew presented himself as an excellent speaker who answered all questions of the participants in a very detailed and knowledgeable manner.

Burkhard Siefert

Mar 10 2021

Very dynamic and interesting speaker, interactive webinar.

Anna Sarao
Regulatory Affairs Project Manager, Servier

Mar 10 2021

Very dynamic and interesting speaker, interactive webinar.

Anna Sarao
Regulatory Affairs Project Manager, Servier

United Kingdom

  • Blue Reg Pharma Consulting
  • Brightwater Research
  • Chiesi Ltd
  • Fujifilm Kyowa Kirin Biologics
  • Galderma (UK) Ltd
  • JensonR+ Ltd
  • Kyowa Kirin
  • NDA Regulatory Science Ltd
  • Orphan Reach Ltd
  • Vertex Pharmaceuticals (Europe) Limited


  • Bluereg
  • Laboratoires DELBERT
  • Servier


  • Angelini Pharma
  • Angelini Pharma s.p.a.
  • Sifi S.p.A.


  • GAT Therapeutics


  • Imcyse


  • CureVac AG


  • Parexel Ireland USD


  • AS Kalceks


  • Takeda

United States of America

  • CANbridge Pharmaceuticals

Run Regulatory Strategies for Orphan Drugs Bespoke training for your team

1 day

Typical duration

Pricing from:

  • GBP 450
  • Per attendee
  • Course tailored to your requirements
  • At your choice of location, or online


We can customise this course to your requirements and deliver it on an in-house basis for any number of your staff or colleagues.

Contact our in-house training expert Aleksandra Beer to discuss your requirements:

Multiple colleagues? See above for details of our discounts for 2, 3, or 4 delegates. For more, talk to one of our training experts to discuss how to:

Run this course conveniently and cost-effectively in-house for your staff and colleagues

Aleksandra BEER
Training expert

+44 (0)20 7749 4749