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Regulatory Strategies for Orphan Drugs Training Course

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

22 Nov 2021

& 16 Mar 2022 , 27 Jun 2022 , 26 Sep 2022

Book now

Details

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.

Programme

Module 1 - 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

Module 2– 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

Module 3 - Maintenance of Orphan Drug Designation

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

Module 4 – Orphan Drug Designation

  • Global perspective for Russia, China, etc

Presenter

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).

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Book now

22 Nov 2021
Live webinar
22 Nov 2021
Live webinar
GBP 599.00
EUR 859.00
USD 970.00
+ VAT @ 20.00%
Enrol now
to attend
Live webinar
16 Mar 2022
Live webinar
16 Mar 2022
Live webinar
GBP 599 499
EUR 859 719
USD 970 814
Until 9 Feb 22*
Enrol now
to attend
Live webinar
27 Jun 2022
Face-to-face, (venue not yet confirmed)
27 Jun 2022
Face-to-face
(venue not yet confirmed)
GBP 699 599
EUR 979 839
USD 1,090 934
Until 23 May 22*
Enrol now
to attend
Face-to-face
(venue not yet confirmed)
  • 1 day classroom-based training
  • Meet presenters and fellow attendees in person
  • Lunch and refreshments provided
  • Download documentation and certification of completion
  • Fair transfer and cancellation policy
26 Sep 2022
Live webinar
26 Sep 2022
Live webinar
GBP 599 499
EUR 859 719
USD 970 814
Until 22 Aug 22*
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

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
  • CureVac AG
  • Institut De Recherches Internationales Servier Iris
  • Kyowa Kirin International
  • NDA Regulatory Science Ltd
  • Takeda

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