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Strategies for Obtaining and Maintaining Orphan Drug Designation
Register Your Interest | Short Online Course
1-day live online training | Starts 9:30 AM UK Time
Optimise Your Regulatory Strategies for Orphan Drugs – Click Here to Download the Agenda
Learn more about the course by toggling through the tabs below. Scroll down to view the agenda, trainer info and who should attend.
Learn more about the course by toggling through the tabs below (Agenda, Trainer and Who Should Attend?)
Course Information
Course Information
- The course begins at the time stated at the top of the page
- The course is broken up into modules outlined below
- There will be breaks between modules
Session 1
13 March 2025
29 October 2025
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180 mins | 9:30 AM UK
Orphan medicinal product legislation
- Overview of the regulations in the EU, US and Japan
- What the regulations cover and why, what they try to protect from (i.e. creation of false sub-populations of a non-orphan condition)
- Awards for obtaining Orphan Drug Designation in EU and US
- Considerations for Orphan Drug Designation
- Developing orphan versus non-orphan indications
- Paediatric conditions including the challenges and impacts in this area, trade-off of the incentives and the ongoing evaluation of the orphan regulation by the EC
Session 2
13 March 2025
29 October 2025
180 mins | 1:30 PM UK
Obtaining orphan drug designation
- Orphan drug designation in the EU and US
- Review the application
- Examine the procedure
- Similarities and differences with the US
- Rare diseases: a global issue
- Collaboration between Agencies
- Strategic considerations on when to apply and to what Agencies
Orphan drug maintenance & development
- Brief overview of maintenance of orphan drug designation
- Key considerations for orphan drug development
Andrew Willis
Regulatory and Development consultant with 35 years of experience. Andrew’s management roles have seen him act as global Vice President of Regulatory Affairs and Medical Affairs, creating business and marketing strategies for complex pharmaceutical development projects. Andrew has gained therapeutic experience in areas covering CNS, oncology, urology and pain management.
Experienced with both EU and US applications (NDA, 505 B 2, ANDA, BLA, MAA) for NCEs, generics and line extensions, with particular knowledge of sterile and biotech products. Experience covers multiple applications, scientific advice meetings, Clinical Trial applications (IND and IMPD compilation and submission), orphan drug applications. Significant Experience with novel drug delivery systems has been utilized in all of the above type of applications.
Andrew continues to provide training for all major companies and also a number of EU Member states agencies in CMC, variations and advanced regulatory affairs. More recently for IO Biotech, Andrew was SVP of Quality, Regulatory and CMC sitting on core C-suite providing CTO support activities – overseeing all pharmaceutical development of drug substance and drug products for peptide molecules and ensuring appropriate quality systems are in place.
He currently delivers extensive training courses available in Quality By Design, Global Module 3 Requirements, global generic development and Optimizing the Life of Your Products. Specializing in Lifecycle management, creation of new line extensions and development strategies to maximize return on investment.
This course is designed for professionals who need to learn more about the orphan drug designation process and is relevant for large organisations all the way to start-ups. The course is very much applicable to those involved in the regulatory roadmap of their products and include:
- Regulatory professionals (managers, executives)
- Senior management
- Project management
Individual Booking (verified owner) –
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AOP Health –
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