Presented by
Management Forum

Regulatory Strategies for Orphan Drugs

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

22 Nov 2021 + 3 more dates

Enrol now

  • Format: Live online, Classroom
  • CPD: 6 hours for your records
  • 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.

Enrol now

The Regulatory Strategies for Orphan Drugs course will cover:

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

Enrol now

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

Book Regulatory Strategies for Orphan Drugs Live online/Classroom training

22 Nov 2021

Live online

09:00-17:00 UK (London) (UTC+00)
10:00-18:00 Paris (UTC+01)
04:00-12:00 New York (UTC-05)
Course code 11063

  • GBP 599
  • EUR 859
  • USD 970

In your basket 

16 Mar 2022

Live online

09:00-17:00 UK (London) (UTC+00)
10:00-18:00 Paris (UTC+01)
05:00-13:00 New York (UTC-04)
Course code 11571

  • GBP 499 599
  • EUR 719 859
  • USD 814 970

Until 09 Feb

In your basket 

27 Jun 2022

Classroom
London venue TBC

00:00-00:00 UK (London)
Course code 11376

  • GBP 599 699
  • EUR 839 979
  • USD 934 1,090

Until 23 May

  • 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

In your basket 

26 Sep 2022

Live online

00:00-00:00 UK (London) (UTC+01)
01:00-01:00 Paris (UTC+02)
19:00-19:00 New York (UTC-04)
Course code 11695

  • GBP 499 599
  • EUR 719 859
  • USD 814 970

Until 22 Aug

In your basket 

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


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.

Mar 10 2021

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

UK

  • Brightwater Research
  • Kyowa Kirin International plc
  • NDA Regulatory Science Ltd
  • Orphan Reach Ltd

France

  • Bluereg
  • Servier

Italy

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

Germany

  • CureVac AG

Switzerland

  • Takeda

Enrol now

Multiple colleagues?
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

inhouse@ipi.academy