Thanks to its large population and well-established healthcare system, the European Union (EU) can offer an attractive market opportunity for biotech or pharmaceutical companies looking to develop a new drug for a rare disease.
However, getting a new drug approved in the EU can be a complex and lengthy process, particularly for orphan drugs — those that are intended to treat rare diseases affecting fewer than 5 in 10,000 people.
In the EU, the regulatory authority responsible for granting marketing authorisation for drugs is the European Medicines Agency (EMA). The EMA’s Committee for Orphan Medicinal Products (COMP) is specifically tasked with assessing applications for orphan drug designation and recommending medicines for orphan designation to the European Commission.
Receiving orphan drug designation
To be eligible for orphan drug designation, a drug must meet certain criteria, such as being intended for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition — as well as affecting no more than 5 in 10,000 people in the EU. The drug must also demonstrate a significant benefit over existing treatments (or no satisfactory treatment options should exist).
Once a drug has been granted orphan designation, the sponsor can apply for EMA marketing authorisation through the centralised procedure, which is mandatory for orphan drugs.
The centralised procedure involves a single application to the European Medicines Agency, which is then evaluated by a committee of experts from all EU member states. This process can take up to 210 days for the EMA to assess the application and issue a recommendation to the European Commission.
If the drug is recommended for approval, the European Commission then grants a marketing authorisation, which allows the drug to be marketed and sold across all EU member states. This authorisation is valid for five years and can be renewed if the drug continues to meet the criteria for orphan designation.
Applying for EMA marketing authorisation
The EU provides incentives for the development of orphan drugs, such as fee reductions, scientific advice, protocol assistance and a period of market exclusivity of 10 years — during which no other similar products can be placed on the market. This exclusivity period can be extended to 12 years if the drug is authorised for use in paediatric patients.
However, it’s important to be aware that from 1 January 2025, the European Medicines Agency is implementing the EU Regulation 2024/568 on fees for regulatory submissions. This change will result in a significant increase in some of the fees payable to the EMA for scientific assessment of a medicinal product for the application of an EMA marketing authorisation, variation or authorised product.
As an established EU/EEA-based marketing authorisation holder (MAH) with SME status, TMC can support eligible companies with the European Medicines Agency’s impending fee increase for regulatory submissions — passing on significant discounts to save biotech and pharmaceutical companies thousands on their regulatory application.
TMC can be your EU/EEA representative and act as your marketing authorisation holder, taking on all the legal and regulatory accountability to ensure your product is manufactured, marketed and distributed according to the terms and conditions laid out in the marketing authorisation.
We make sure, on your behalf, that the product always meets the quality, safety and efficacy standards as approved by the regulatory authorities — helping you get your product to market faster.
Learn more about our regulatory affairs support for new drug approvals and marketing authorisations to see how we can help you ensure compliance, secure substantial discounts on EMA fees and, ultimately, bring your products to market efficiently and cost-effectively.