Orphan Drug Designation in the EU Driving Innovation for Rare Diseases

In the European Union (EU), rare diseases contribute to approximately 6-8% of total deaths across all age groups. The death percentage is about 35% among the pediatric population, especially in the first year of life, and thereby significantly impacts the later stages of life. Why is the mortality rate high? Because there are very few drugs or no drugs to treat the rare disease. It clearly underlines the need for innovation to meet unmet medical needs in the region. To meet market demand for innovative drug products as quickly as possible, the EU has established an orphan drug designation system. 

What is EU Orphan Drug Designation?

Orphan Drug Designation (ODD) is a dedicated EU pathway that encourages and incentivizes the development of medicines for rare and underappreciated diseases. To obtain an Orphan Drug Designation, a drug should be made for the rarest of the rare conditions that prevail in no more than five among 10000 people. 

Who Regulates Orphan Drug Designation in the EU?

The EU has established ODD through EC No 141/2000. In addition, a new legislation has been introduced that supports the region’s innovation capacity and aims to position the EU as a global leader in ODD and to capture 20% of the $1.6 trillion global drug market by 2030. The ODD applications are processed and reviewed by the European Medicines Agency’s Committee for Orphan Medicinal Products (COMP). It usually takes 90 days for the review, and sponsors are expected to receive a decision. 

How Different is Orphan Drug Designation in the EU?

• EMA’s Orphan Drug Designation has an exclusivity of 10 years post the approval
• For pediatric drug products, it may extend up to 12 years
• Similar to Priority Review Vouchers (PRVs) of the FDA for rare paediatric diseases, the EMA is planning to implement European Accelerated Assessment Vouchers (EAAVs). However, it is still under analysis, and the cost of such vouchers still needs to be determined. 

The Orphan Drug Designation has reshaped the EU pharmaceutical landscape and offers a promising landscape for research, analysis, and discoveries. On the other hand, the new EU pharmaceutical legislation makes the European landscape more competitive by offering incentives, which are indeed fueling the transformation. The next step for the innovators is to deep-dive into regulatory procedures and crack the European market with breakthrough therapies for rare diseases.