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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.


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.


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 (More...)

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).

Book now

Book or reserve now

NEW higher discounts for booking multiple places - bring your colleagues to make your training budget go further:
30% off the 2nd delegate; 40% off the 3rd delegate; 50% off the 4th delegate

Choose your date below - or reserve your place with no obligation for 7 days at Reserve now
26 Sep 2022
Live webinar
UK (London)
26 Sep 2022
Live webinar
UK (London)
GBP 599 499
EUR 859 719
USD 970 814
Until 22 Aug*
Enrol now
to attend Live webinar

Learn more about our face-to-face and webinar training formats.

* Note the early booking discount cannot be combined with any other offers or promotional code. As such, discounts for the 2nd/3rd/4th attendees are based on the full price of the course. Please contact us for pricing if you wish to send more than 4 delegates.

Continuing professional development

This course qualifies for 6 hours for your CPD records.
More about CPD

Customised in-house training

We can customise this course to meet the requirements of your organisation and deliver it at your location and/or online.

Contact us

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Previous customers include...

  • Angelini Pharma s.p.a.
  • Blue Reg Pharma Consult
  • Brightwater Research
  • CANbridge Pharmaceuticals
  • CureVac AG
  • Faes Farma
  • Fujifilm Kyowa Kirin Biologics
  • Galderma (UK) Ltd
  • Institut De Recherches Internationales Servier Iris
  • JensonR+
  • Kyowa Kirin International
  • NDA Regulatory Science Ltd
  • Orphan Reach Ltd
  • Parexel Ireland USD
  • Sifi S.p.A.
  • Takeda

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

John McIntyre, Senior Consultant, Parexel Ireland USD, Jun 22

Excellent would attend other similar webinar/trainings

Cheryl Dicks, Sr. Mgr. Regulatory, CANbridge Pharmaceuticals, Mar 22

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 21

Very dynamic and interesting speaker, interactive webinar.

Anna Sarao, Regulatory Affairs Project Manager, Servier, Mar 21

Very dynamic and interesting speaker, interactive webinar.

Anna Sarao, Regulatory Affairs Project Manager, Servier, Mar 21