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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.

23 Sep 2020

& 10 Mar 2021 , 22 Sep 2021

GBP 699
EUR 979
USD 1,090

Book now

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 and US
  • Why do we need orphan legislation?
  • What the regulations say
  • Benefits for obtaining orphan drug designation in the EU and US
  • Considerations for orphan drug designation
    • Sequence of submissions by country
    • Developing orphan versus non-orphan indications
    • Paediatric conditions
Process of orphan drug designation
  • Orphan drug designation in the EU
    • Application
    • Procedure
  • Similarities and differences with the US
    • Application, procedure and incentives
  • Strategic considerations of when to apply and to what agencies
  • Future collaborations between EU and US
Maintenance of orphan drug designation
  • Examples of recent orphan drug designation
  • Prevalence challenges
  • Changes to indication/experience of introducing a new indication

Practical case study session on obtaining orphan drug designation

Orphan drugs and ATMPs
  • The link between ATMPs and the use of orphan legislation
  • Using orphan drug designation with your ATMP strategy


Andrew Willis

Andrew Willis is an independent consultant providing expert advice and training on global regulatory solutions and pharmaceutical development. Previously, he worked for Catalent Pharma Solutions as VP Regulatory Affairs & Consulting Services, where he was head of a team of internal and external regulatory affairs consultants.

He qualified as a Chemist from the University of Glamorgan, after which he furthered his understanding of pharmaceutical development, working as a research chemist with Parke Davis. He had 10 years manufacturing and analytical experience prior to entering regulatory affairs as a Senior Executive Officer with responsibility for submission of European MAAs and project management of development programs. He has over 30 years’ pharmaceutical experience with extensive knowledge in the development and manufacture of sterile, solid oral, inhalation, topical and biotech pharmaceutical products. These experiences have allowed knowledge of many biotech products requirements with experiences of growth hormones and multiple cancer treatments, including development and clinical registration of the first genetically modified live bacterium for such treatment.
He has extensive experience of major European and US regulatory projects, in the clinical and marketing authorisation stages, and has significant experience in coordinating and managing meetings with European and US Health Authorities.

Book now

23 Sep 2020
23 Sep 2020 Rembrandt Hotel, London GBP 699.00
EUR 979.00
USD 1,090.00
+ VAT @ 20.00%
Enrol now
10 Mar 2021
10 Mar 2021 Venue not yet confirmed
+ VAT @ 20.00%
Enrol now
22 Sep 2021
22 Sep 2021 Venue not yet confirmed
+ VAT @ 20.00%
Enrol now

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Previous customers include...

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  • NDA Regulatory Science Ltd