Whitepaper

How to Successfully Apply for Orphan Designation in the EU and How to Maintain It

Published 26th September 2024

How to Successfully Apply for Orphan Drug Designation in the EU and How to Maintain It

Successfully applying for and maintaining orphan designation in the European Union (EU) is a critical pathway for sponsors developing treatments for rare diseases. The process involves navigating regulatory frameworks set by the European Medicines Agency (EMA) under Regulation (EC) No 141/2000.

To obtain an orphan designation, the applicant must demonstrate that:
  • the condition affects no more than 5 in 10,000 individuals in the EU,
  • the product offers significant therapeutic benefit over existing treatments, or
  • no satisfactory method of diagnosis, prevention, or treatment exists.

 

Once granted, maintaining this status requires ongoing compliance with regulatory obligations, including regular reporting on the development status, marketing exclusivity updates, and adherence to timelines.

In this whitepaper we provide key reflections for navigating potential challenges in the preparation of an orphan designation application, such as classification of the orphan condition, prevalence estimation, evidence of significant benefit, and medical plausibility. We emphasise the importance of a pre‑submission meeting for obtaining an orphan designation, and about generating evidence during the marketing authorisation (MA) process to maintain the designation. We also review the benefits and incentives of orphan designations such as protocol assistance, market exclusivity, access to EU grants, and fee reductions, which are vital for the commercial viability of orphan drugs in the EU. Finally, we highlight how the new EU pharmaceutical reforms are going to impact orphan designations.

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    Meet Our Experts

    Praveen Meka
    Dr. Praveen Meka

    Regulatory Executive, DLRC

    Praveen Meka is a Regulatory Executive, joined DLRC in 2023. He did his PhD in Neuroscience and has more than 10 years of non-clinical academic research experience in neuronal development and degeneration. As a Regulatory Executive, he supported a number of clients with orphan designation applications, CHMP and National Scientific Advice procedures, CTIS and Policy 70 redactions and non-clinical gap analysis.

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    Michelle Blake
    Michelle Blake

    Senior Regulatory Consultant, DLRC

    Michelle is a Senior Regulatory Consultant and Team Leader and has been working at DLRC for over 13 years. Her expertise includes extensive clinical development background including paediatrics, rare disease and oncology indications. She has worked with a large range of clients including small biotech companies, big pharma and CROs. Michelle has led national and CHMP scientific advice procedures in the EU as well as leading Oral Explanations with various EMA committees and due to her expertise, is the DLRC subject-matter expert for paediatric development.

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