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Course Overview
Orphan drugs are vital for those people with rare diseases, and this is recognised by Regulators globally. By gaining orphan drug designation, pharmaceutical companies access various incentives to encourage therapeutic development. However, it is important the system is not misused and that you fully understand the regulatory requirements and roadmap.
This training course will provide you with a comprehensive understanding of the opportunities and challenges offered by orphan drug designation. Once completed, you will understand the regulations and whether they are applicable to your product. You will also have a good understanding of the application process and procedure to obtain orphan drug designation. You will examine strategic considerations to ensure the most efficient regulatory path for your product. Learn more about how we deliver live online training.
Key Learning Objectives
Examine the orphan drug regulations in the EU, US and Japan
Gain a deep understanding of the regulations; what they cover and why
Discover if your drug can be considered an orphan drug under the current regulations
Review multiple considerations for orphan drug designation and the awards this brings
Examine the orphan drug designation process in the EU and US
Get first-hand insights on how to get the best from orphan drug regulations
Analyse the maintenance of an orphan drug designation in preparation for MAA/NDA
What is Included?
Direct access to an expert trainer
Interactive and engaging sessions
Access to the Educo Life Sciences Training Portal
Unlimited access to the recordings for 2 weeks after the session
Download the Agenda
You can view the agenda below or if you would like to receive a pdf version with all the course information, please complete the form below.
Learn more about the course by toggling through the tabs below (trainer, agenda, who should attend?)
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
28 April 2023 | 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
28 April 2023 | 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
Could your team benefit from this course?
We can customise this course to meet the requirements of your organisation, delivered as a classroom and/or online course.
For more information you can email us at [email protected] or call +44 (0) 203 111 7357.
Complete our contact form by following the link. Get in touch.
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• This course is delivered online
• For live online courses, module durations are approximate
• You will have full access to the training portal and the recordings for 2 weeks after the training
