Rare diseases, with their low prevalence and significant impact on patients, have long posed challenges for pharmaceutical advancement. In response, the European Union (EU) has established incentives to stimulate the development and accessibility of medicines for these conditions. A sponsor must understand the incentives, application, and considerations for orphan medicinal product designation in the EU to successfully advance their rare disease treatments.

Within the EU, orphan incentives help the pharmaceutical companies invest in developing treatments for rare diseases. The European Medicines Agency (EMA) provides guidance, offering protocol assistance tailored for orphan medicines. This guidance assists sponsors in navigating the studies required to demonstrate a medicine's efficacy, safety, and potential risks. Reduced application fees and multiple guidance requests allow sponsors to optimize development strategies.

Streamlined Approval Process

A central aspect is the streamlined centralized approval process. Orphan drug designation undergo an expedited marketing authorization review. Submitting a single application to the EMA rewards sponsors with a unified decision from the European Commission. This decision applies across all EU Member States, eliminating the individual country approvals. Additionally, the process allows for conditional approval, which expedites treatment availability.

Individual EU states also contribute incentives by tailoring measures to support orphan medicine development. This collective approach ensures the rare disease system can effectively address unique challenges.

Support and Market Protection

A key incentive is the over a decade of market exclusivity protecting authorized orphan medicines from competition. This can extend by two years through providing paediatric investigation plans. This extension allows the treatments to solidify their position.

The EU offers support to small and medium enterprises through the EMA office support services and fee reductions for protocol assistance, applications, inspections, and post-approval changes. These reliefs enable the SMEs to actively contribute to rare disease treatment development.

Furthermore, fee reductions across regulatory activities foster increased rare disease treatment development and underscore the EU commitment to innovation.

Format and content

The full information on the procedure for orphan medicinal products designation is available on the EMA orphan designation website. When applying for orphan designation, there are also guidelines available, offering supplementary advice on the information the sponsors must provide.

  1. Timing of Applications: A sponsor can seek orphan designation for a product at any stage before marketing authorization. However, if the same sponsor has already submitted a marketing application in the EU for the same product and indication, the orphan designation may be denied. 
  2. Pre-Submission Meeting: Sponsors are strongly encouraged to request a pre-submission meeting with the EMA, free of charge, before submitting the orphan designation application, especially if it is their first application for an orphan designation.
  3. Considering Multiple Orphan Conditions: Sponsors should submit separate applications for each orphan condition if seeking designation for more than one condition with the same product. Different diagnostic, treatment, or prevention uses for the same condition are considered separate orphan conditions requiring individual applications. 
  4. Application Information: Orphan designation applications must include the registered active substance name, details of the proposed orphan condition and its diagnosis/prevention/treatment use, and any available invented name, strength, dosage form and administration route. Sponsors must also furnish EU-based contact information and designate a representative for the EMA communication. 
  5. Scientific Documentation: The scientific section of an orphan designation application must justify the medical plausibility of using the product for the condition. It should also present the condition's prevalence, severity, and existing diagnosis/treatment methods. If satisfactory methods exist, significant benefits over these must be demonstrated 

Essential Designation Considerations  

Applications for orphan designation are examined by the EMA's Committee for Orphan Medicinal Products (COMP), using its network of experts. The Agency sends the COMP opinion to the European Commission, which is responsible for granting the orphan designation.  

Several important factors influence the success of orphan designation applications. Presenting accurate and supported prevalence data is important. Also, to show the meaningful medical benefits the potential treatment could provide to the patients. Providing details about the current development stage, regulatory status, past marketing history, and future plans strengthens the case for receiving orphan designation. Demonstrating low prevalence, benefits to patients, as well as development progress and regulatory information are factors that can impact the success of an orphan designation application.  


The EU has established incentives and processes to support the development of orphan medicines for rare diseases. The streamlined regulatory review and extended market exclusivity help companies develop treatments and the guidance and reduced fees make the process more practical for all sponsors. The Orphan Drug Designation aims to address the unique challenges of developing drugs for diseases with small patient populations. This coordinated approach addresses the challenges of rare disease drug development to ultimately benefit patients in need of novel therapeutic options. 

 As a developer, partnering with NDA ensures that you have a strategic ally to address unmet medical needs and support you through a process tailored to the special demands of this underserved patient population. With our expertise, we'll guide you through every step to meet their unique needs. Contact us by using the form below to find out more. 

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