What is an Orphan Drug Designation?


Orphan Drug Designation in the European Union (EU) is a regulatory status granted to pharmaceutical products developed to treat rare diseases. This designation provides various incentives to encourage the development of drugs for these rare diseases, including market exclusivity, fee reductions, and access to scientific advice. It aims to promote the development of therapies for conditions that might not otherwise receive attention due to their limited patient population, ultimately benefiting patients with rare diseases by facilitating the development and availability of treatments.


How to Apply for an Orphan Drug Designation?


To apply for Orphan Drug Designation (ODD) in the European Union (EU), specific procedures should be followed and certain criteria should be met. Here’s a general overview of the application process:


Eligibility Assessment: Ensure that your drug product or medical device meets the criteria for orphan designation. In the EU, a condition is considered rare if it affects no more than 5 in 10,000 people. Your product must also demonstrate the potential to provide a significant benefit to patients with the rare condition.


Compile the Application: Prepare a comprehensive application dossier, including detailed information about the drug or medical device, the rare disease it aims to treat, preclinical and clinical data, as well as any relevant literature. 


Submit the Application: Submit the application to the EMA. The EMA’s Committee for Orphan Medicinal Products (COMP) reviews ODD applications. 


Payment of Fees: There are fees associated with the application, but these fees can be reduced or waived for small and medium-sized enterprises (SMEs) and academic sponsors. It’s important to review the latest fee schedule and policies.


Orphan Designation Decision: The COMP will evaluate your application and decide whether to grant orphan designation. 


ECONiX Research is accustomed to these steps and assists Pharmaceutical Companies to get the ODD in the EU. 


For more information please contact us at: hello@econix.net