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"
26 June 2023
+ 27 October 2023
from £549
- Format: Live online
- 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
This course is part of our Regulatory Affairs Training course collection, which features updates on the latest regulations to registration procedures and strategies.
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 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
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
26 Jun 2023
Andrew Willis
San Salvatore Training Ltd
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.
27 Oct 2023
Joanne Roza
Qdossier
Joanne Roza works as a senior regulatory affairs consultant at Qdossier. She is involved in regulatory affairs consultancy and has also expertise in regulatory operations for big- and small-sized pharmaceutical companies. Joanne holds a bachelor degree in Health Sciences from Maastricht University and a master degree in Health Economics, Policy and Law from the Erasmus University in Rotterdam. She started her career at Qdossier in 2016 and has been involved in a variety of regulatory operations and affairs projects. Joanne’s areas of expertise are: regulatory affairs (strategy and planning), preparation/creation and dispatch of different types of electronic submission formats (e.g. US FDA, CA, EMA, CH), lean/structured authoring and the creation of baseline (CMC) submissions.
Book Regulatory Strategies for Orphan Drugs Live online training
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
26 June 2023
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 12729
- GBP 649
- EUR 929
- USD 1,049
- 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
27 October 2023
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 12337
- GBP 549 649
- EUR 789 929
- USD 893 1,049
Until 22 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.
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
- NDA Regulatory Science Ltd
- Orphan Reach Ltd
- Vertex Pharmaceuticals (Europe) Limited
France
- Bluereg
- IPSEN
- Laboratoires DELBERT
- 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
- 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:
