Management Forum Logo

Presented by
Management Forum

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.

★★★★★ "Excellent training."

10 February 2025
+ 23 June 2025, 7 October 2025 »
from £549

Need help?  Enrol or reserve

Download Print Enrol or reserve

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, which are often developed for the treatment of rare diseases.

The interactive course will cover all the key aspects of orphan drug legislation in both the US and EU, including a review of changes proposed in the revised EU orphan drug legislation, and the regulatory processes designed to expidite development and thereby enable earlier access to innovative medicines. The programme will explain in detail the key concepts critical for a thorough understanding of the orphan legislation in the EU and US and how it is applied. The two-stage process of first obtaining the orphan designation and then submitting the MAA/NDA and maintaining the designation as an orphan drug in order to benefit from incentives will be described. We will also cover clinical development considerations and flexibilities that are relevant for orphan drugs.

Case studies will consolidate and illustrate the learning points with real world examples 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.

This course is part of our Regulatory Affairs Training course collection, which features updates on the latest regulations to registration procedures and strategies.

Benefits of attending

  • Gain a detailed 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 how orphan status can influence drug development programmes
  • Observe how the theory is applied to real world examples

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 or reserve

This course will cover:

Orphan Drug Legislation - background and overview

  • Origin and history of orphan drug legislation
  • EU and US legislation overview and comparison
    • Brief discussion of other countries/regions
  • Key concepts and definitions
  • Orphan incentives
  • Overlaps with paediatric legislation
  • FDA and EMA databases

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

Enrol or reserve

Shaun Stapleton
Regulatory Consultant

Shaun Stapleton is an independent consultant providing regulatory strategy advice and support to biopharma clients globally.

Shaun held positions of increasing responsibility in regulatory affairs at Sterling Winthrop, Eli Lilly, Boehringer Ingelheim and Ipsen, where he managed regulatory input into development programmes globally, securing new product approvals in the neurology, endocrinology and oncology therapeutic areas.

More recently, Shaun spent 8 years in regulatory consultancy at RRG (a Voisin Consulting Life Sciences Company) where he was a Director and Vice President of Regulatory Science working with global clients on a wide range of regulatory projects. From VCLS he moved to ReNeuron Ltd where he led regulatory, pharmacovigilance and latterly quality aspects of cell therapy and exosome development programmes.

Shaun has extensive experience in orphan drug development from first in man clinical trials through to commercialisation globally. He has been involved in discussions with regulators relating to specific orphan exclusivity issues and chaired the Alliance for Regenerative Medicine working group on “sameness” in the context of market exclusivity for orphan ATMPs (cell therapy). Most recently he was VP and Head of Global Regulatory Affairs at Amryt Pharma, a company specialising in the development and commercialisation of orphan medicines globally, where he contributed to the approval and life cycle management of several orphan drugs in areas of high unmet medical need.

More details

NEW higher discounts for multiple bookings - 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*

Please contact us for pricing if you are interested in booking 5 or more delegates

10 February 2025

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 14507

  • GBP 549 649
  • EUR 789 929
  • USD 893 1,049

Until 06 Jan

View basket 

 
Not ready to book yet?

for 7 days, no obligation

23 June 2025

Live online

09:00-17:00 UK (London) (UTC+01)
10:00-18:00 Paris (UTC+02)
04:00-12:00 New York (UTC-04)
Course code 14770

  • GBP 549 649
  • EUR 789 929
  • USD 893 1,049

Until 19 May

View basket 

 
Not ready to book yet?

for 7 days, no obligation

7 October 2025

Live online

09:00-17:00 UK (London) (UTC+01)
10:00-18:00 Paris (UTC+02)
04:00-12:00 New York (UTC-04)
Course code 14982

  • GBP 549 649
  • EUR 789 929
  • USD 893 1,049

Until 02 Sep

View basket 

 
Not ready to book yet?

for 7 days, no obligation

* Early booking discounts may not be combined with other discounts or offers. As such, the discounts for 2nd/3rd/4th delegates are based on the full price; and apply only when booking multiple delegates on the same date.

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


Excellent training.

Jun 24 2024

Thiago Loureiro
Sr. manager , Regulatory Strategy, Vertex

Jun 24 2024

Very instructive webinar. Although packed, the day went by quickly.

Ariane Teyssou
Regulatory Affairs Specialist, Enterome

Jun 24 2024

Well organised and very experienced presenter

Maria Liljevald
Head of Global Regulatory Affairs, Ribocure Pharmaceuticals AB

Sep 26 2022

I believe as a starting point into the world of ODD this was very comprehensive and informative. Very clear and concise.

Khayyam Darr
Senior Regulatory Affairs Executive, Chiesi Ltd

Jun 20 2022

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

John McIntyre
Senior Consultant, Parexel Ireland USD

Mar 16 2022

Excellent would attend other similar webinar/trainings

Cheryl Dicks
Sr. Mgr. Regulatory, CANbridge Pharmaceuticals

Sep 26 2022

Very interactive session (nicely done even if remote) Expertise sharing and specific examples shared during the training to emphasize and help to understand key topics

Christine Monteiro
Senior Regulatory Project Manager , IPSEN

Sep 26 2022

Very instructive training with a very nice speaker. The speaker knew her subject very well and she was able to share some of her experiences with ODD which made the training more interactive.

Laetitia Misslin
Regulatory Affairs Director, Laboratoires DELBERT

Sep 26 2022

Everything was OK

Sarmite Liberte
Head of Regulatory Affairs, AS Kalceks

Sep 26 2022

Very instructive training with a very nice speaker. Speaker knew her subject very well and she was able to share some of her experiences with ODD which made the training more interactive.

Laetitia Misslin
Regulatory Affairs Director, Laboratoires DELBERT

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
  • Lupin Healthcare UK Ltd.
  • NDA Regulatory Science
  • NDA Regulatory Science Ltd
  • Orphan Reach Ltd
  • Sobi
  • Vertex Pharmaceuticals (Europe) Limited

France

  • Bluereg
  • Enterome
  • IPSEN
  • Ipsen Pharma
  • Laboratoires DELBERT
  • Laboratoires Thea Sas
  • SERB SAS
  • Servier

Italy

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

Ireland

  • Parexel
  • Parexel Ireland USD

Spain

  • FAES FARMA SA
  • GAT Therapeutics

Belgium

  • Imcyse

Brazil

  • Vertex

Denmark

  • Ascendis Pharma A/S

Germany

  • CureVac AG

Latvia

  • AS Kalceks

Sweden

  • Ribocure Pharmaceuticals AB

Switzerland

  • Takeda

United States of America

  • CANbridge Pharmaceuticals

Enrol or reserve

Run Regulatory Strategies for Orphan Drugs Live online for your team

1 day

Typical duration

Pricing from:

  • GBP 450
  • Per attendee, based on 10 attendees
  • Course tailored to your requirements
  • At your choice of location, or online
Contact us 

 

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 experts Aleksandra Beer and Yesim Nurko 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

Aleksandra BEER
Training expert

Yesim Nurko

Yesim NURKO
Training expert

+44 (0)20 7749 4749

inhouse@ipi.academy