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.
★★★★★ "I believe as a starting point into the world of ODD this was very comprehensive and informative. Ver... more (10)"
24 June 2024
+ 8 October 2024 »
from £549
- Format: Live online
- CPD: 6 hours for your records
- Certificate of completion
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.
The Regulatory Strategies for Orphan Drugs 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
Challenges with orphan definitions and application of the legislation
- Condition vs indication
- “salami-slicing” of indications
- Similarity and sameness
- Extra challenges of ATMPs
- Case law
- Significant benefit and superiority
- Market exclusivity
Procedural aspects for orphan designated products
- The two-stage process
- Orphan designation process EU and US
- MAA/NDA process for orphan drugs EU and US including maintenance of orphan designation
Case Study 1
- EU orphan drug process
Development considerations for orphan drugs
- Clinical development requirements and flexibilities applied to orphan drugs
- EMA and FDA guidance documents on clinical development relevant to orphan drugs
- Applicability of expedited and early access programmes in EU and US
- New clinical indications
- Orphan/non-orphan
Case Study 2
- US process and clinical development considerations
Anticipated future developments and changes to the regulation of orphan drugs
- EU and US
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.
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
24 June 2024
Live online
09:30-17:00 UK (London) (UTC+01)
10:30-18:00 Paris (UTC+02)
04:30-12:00 New York (UTC-04)
Course code 13737
- GBP 549 649
- EUR 789 929
- USD 893 1,049
Until 20 May
- 1 day live online training
- Browser-based, no download usually required
- See presenters and interact with fellow attendees
- Download documentation and certification of completion
- Fair transfer and cancellation policy
Not ready to book yet?
for 7 days, no obligation
8 October 2024
Live online
09:30-17:00 UK (London) (UTC+01)
10:30-18:00 Paris (UTC+02)
04:30-12:00 New York (UTC-04)
Course code 13919
- GBP 549 649
- EUR 789 929
- USD 893 1,049
Until 03 Sep
- 1 day live online training
- Browser-based, no download usually required
- See presenters and interact with fellow attendees
- Download documentation and certification of completion
- Fair transfer and cancellation policy
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
I believe as a starting point into the world of ODD this was very comprehensive and informative. Very clear and concise.
Sep 26 2022
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 Ltd
- Orphan Reach Ltd
- Vertex Pharmaceuticals (Europe) Limited
France
- Bluereg
- Enterome
- IPSEN
- Ipsen Pharma
- Laboratoires DELBERT
- Laboratoires Thea Sas
- Servier
Italy
- Angelini Pharma
- Angelini Pharma s.p.a.
- Sifi S.p.A.
Ireland
- Parexel
- Parexel Ireland USD
Spain
- FAES FARMA SA
- GAT Therapeutics
Belgium
- Imcyse
Germany
- CureVac AG
Latvia
- AS Kalceks
Switzerland
- Takeda
United States of America
- CANbridge Pharmaceuticals
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
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:
