Overview
The orphan drug market is rapidly expanding, with the development of new treatments for rare diseases becoming a key growth area for many pharmaceutical companies. This rising interest in rare and orphan conditions is fuelled not only by attractive incentives from regulatory authorities but also by the emergence of advanced therapies, many of which target rare diseases.
This highly interactive course provides comprehensive coverage of orphan drug legislation in both the EU and US, including an overview of changes to the EU orphan drug framework being proposed as part of the ongoing comprehensive review of the EU pharmaceutical legislation. You’ll gain a clear understanding of the regulatory mechanisms designed to accelerate development and improve early access to innovative treatments.
Through expert-led sessions and detailed guidance, the course explores the core concepts essential to navigating orphan drug legislation. It walks you through the entire process - from obtaining orphan designation during development through to maintaining the designation to secure key incentives following submission and approval of the MAA/NDA.
We’ll also examine clinical development flexibilities specific to orphan drugs, offering insight into the unique regulatory considerations and strategies that support innovation in this space.
Real-world case studies bring the concepts to life, consolidating your learning and ensuring you leave with practical knowledge and actionable insights you can apply immediately. Alongside our experienced trainer, you’ll also benefit from connecting with other professionals in the field and sharing valuable perspectives.
CPD Hours: 6
Course Content
- Day 1
- Orphan Drug Legislation - background and overview
- Challenges with orphan definitions and application of the legislation
- Procedural aspects for orphan designated products
- Case Study 1: EU orphan drug process
- Development considerations for orphan drugs
- Case Study 2: US process and clinical development considerations
- Proposed changes to the EU pharmaceutical legislation relating to orphan drugs
Speakers
Shaun Stapleton
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.
