EMA orphan drug designation
Gaining orphan drug designation from the European Medicines Agency (EMA) is essential for any company seeking to market a new treatment for a rare disease in Europe. Launching a new orphan drug in Europe can be a daunting prospect. The 27 nations which make up the European Union (EU) bring 24 different languages, and almost as many diverse cultures, healthcare systems, reimbursement approaches, and regulatory landscapes.
For any biotech company looking to gain a foothold in the European market, understanding these differences, particularly in the European rare disease medicine regulatory system, is crucial.
What does orphan drug designation mean?
Orphan drug designation is a special status granted by the European Commission to encourage the development of treatments for rare diseases, which typically affect small patient populations.
Due to the limited commercial potential of these treatments, many pharma companies can be reluctant to invest in their development. With a range of incentives, orphan designation helps ensure that companies are motivated to develop these vital therapies. This support is crucial in addressing the unmet medical needs of rare disease patients, who might otherwise have few or no treatment options available to them.
Benefits of achieving orphan drug designation
Orphan drug designation offers significant benefits that make developing treatments for rare diseases more feasible. One of the key advantages is 10 years of market exclusivity in the EU, meaning that no other similar drugs can compete on the market during this period, even after the patent expires. This exclusivity is a major incentive for companies, especially when targeting small patient populations.
In addition to market protection, orphan designation provides significant fee reductions, especially for small to medium-sized enterprises (SMEs), reducing the financial burden of regulatory procedures. There can also be discounts on application fees for marketing authorization, as well as reduced costs for scientific advice and inspections.
Another crucial benefit is protocol assistance from the EMA. This includes guidance on the best ways to design clinical trials and other strategies, which can save time and resources. Access to expedited development programs such as priority review and accelerated assessments can also streamline the regulatory process, helping treatments reach patients faster.
Together, these benefits significantly lower the financial and regulatory hurdles, making orphan designation a valuable tool in bringing life-saving rare disease treatments to market.
EMA and orphan drug designation: How does it work?
The EMA is responsible for reviewing applications for orphan designation. To qualify for orphan designation in the EU, a medicine must meet several criteria:
- It must be intended for the treatment, prevention, or diagnosis of a disease that is life-threatening or chronically debilitating, and
- The condition must affect fewer than five in 10,000 people in the EU or it must be unlikely that marketing of the medicine would generate sufficient returns to justify its development, and
- No satisfactory method of diagnosis, prevention, or treatment of the condition can be authorized, or, if a method exists, the medicine must be of significant benefit to those affected by the condition.
All applications for orphan designation are reviewed by the EMA’s Committee for Orphan Medicinal Products (COMP). The evaluation process takes up to 90 days and the EMA then sends its recommendation to the European Commission, which is responsible for granting orphan designation.
Crucially, applicants need to provide clinical and non-clinical data to support their claims. In the case of rare diseases, with clinical trial data and expert knowledge in short supply, real-world data (RWD) is the most effective way to prove that a product deserves orphan designation. Developing RWD to support your application can help to:
- Provide insight into disease prevalence – a critical step in proving eligibility for orphan designation
- Confirm if a disease meets the rarity criteria
- Complement clinical data to show real-world effectiveness and safety.
Find out more about how Sciensus can support every step of your product launch, including orphan designation.
