Orphan Drug Designation
We specialise in rare diseases and orphan drug designation, helping developers through the regulatory deliverables from proof of concept to marketing authorisation.
If you are developing a medicine for a rare disease, orphan drug designation can be critical to commercial success.
Address Challenges
We can help you respond to questions often faced by orphan drug developers.
Navigate Development
We have the scientific expertise and regulatory experience to help you navigate your development to medicine approval.
Maximize Return on Investment
We will help you maximise product exclusivity and commercial return.
An approved orphan designation doesn’t mean your medicine is approved, and at the time of a marketing authorisation application, you also need to apply for maintenance of the orphan designation to be eligible for the ten-year market exclusivity incentive.
You are also required to identify any medicinal products designated as orphan drugs and protected by market exclusivity. If any orphan-designated medicine has been approved in the European Union and has market exclusivity protection, you are required to include an orphan similarity report in your MAA explaining the possible similarity between your medicinal product and the orphan medicinal products that have been granted approval.
Download our free guide
This guide explains when to submit the orphan application and how to plan for the maintenance report submission at the time of the MAA.
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Our monthly newsletter gives you the latest European and UK regulatory intelligence. We bring you new and interesting approvals; fast-to-market applications, orphan designations; paediatric applications and therapy area-specific news.
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Key takeaways for orphan designation
The overall orphan registration and maintenance strategy, in addition to recommendations for protocol assistance, to gauge acceptance of the proposed strategy.
How Somerville Development Partners can help you
Gaining approval for orphan medicines targeting rare diseases takes a specialist understanding of the therapeutic and regulatory landscapes of rare diseases. Oftentimes, a sponsor developing an orphan medicine will face specific challenges such as a small patient sample, a lack of a comparator arm and dependence on natural history comparators. All of which will elicit specific questions and require specific justifications to regulatory authorities during development.
You need a regulatory affairs agency that has the scientific expertise and regulatory experience to help you navigate your development to medicine approval.
Somerville Development Partners specialises in rare diseases and orphan designation, helping developers through the regulatory deliverables from proof of concept to marketing authorisation.
You may be interested in reading
How will the revised pharmaceutical legislation change orphan-designated medicines for rare diseases?
In this blog, we explore the current EU orphan designation framework, examine the proposed legislative changes, and discuss the potential impact on medicine developers pursuing orphan medicinal product designation and approval in Europe.
Comparing EU, UK and US Regulatory Approaches to Orphan Drugs
EMA, MHRA, and FDA: A Comparative Guide to Orphan Drug Regulations and how they affect medicine developers.
The EMA’s new fees: What does the new fee regulation mean for medicine developers?
The fees payable to the EMA will follow the current legal framework until December 31st, 2024. Then, as of January 1st, 2025, the new fee regulation (EU 2024/568) will be implemented.
Subscribe to our Monthly Regulatory Intelligence Scan
Our monthly newsletter gives you the latest European and UK regulatory intelligence. We bring you new and interesting approvals; fast-to-market applications, orphan designations; paediatric applications and therapy area-specific news.
You can unsubscribe at any time from the link in the newsletter footer. Read our Privacy Policy here.
FAQs
Does orphan designation mean a medicine with be approved quickly?
No. The European Medicines Agency (EMA) offers incentives for orphan designation, including specialised scientific advice (protocol assistance), expanded market exclusivity and fee reductions for orphan designated medicines, but this does not equate to an expedited review to approval.
What are the fee incentives for orphan designated medicinal products?
75% fee reduction for protocol assistance (scientific advice specific to orphan-designated medicinal products)
10% fee reduction for initial marketing authorisation applications
100% fee reduction for pre-authorisation inspections
100% fee reduction for protocol assistance for small and medium-sized enterprises (SMEs)
100% fee reduction for initial marketing authorisation applications for SMEs
100% fee reduction for the post-authorisation activities for SMEs
In the US, a 25% tax credit is awarded to qualified clinical trial costs for orphan drugs
What is the market exclusivity for orphan-designated medicinal products?
The EU/EEA and UK grant a longer exclusivity (10 years) than the US (7 years). In the EU/UK, this can be extended to 12 years for paediatric development compliance, whereas in the US, a separate 6-month paediatric extension is available (but that extends other exclusivities, not the orphan exclusivity itself).
years of regulatory expertise
clients supported
agency interactions
orphan applications
marketing applications
We have supported 30+ orphan applications