Orphan Drug Designations (ODD)

The European Medicines Agency (EMA) and the United States Food and Drug Administration (FDA) provide specific incentives to companies who are granted ODD for their product.


An orphan drug is a pharmaceutical agent that has been developed specifically to treat a rare medical condition, which is generally defined as affecting fewer than 200,000 people in the United States.


Understanding Orphan Drug Status

The orphan drug designation process is important for products seeking approval for the treatment of rare diseases. The benefits of orphan drug designation can provide a major advantage to products seeking approval, and it is important for sponsors to understand both the EMA and FDA processes in order to maximize the potential benefits of orphan drug designation




What does it mean to be granted orphan drug designation?

The benefits of orphan drug designation are significant and can provide a major advantage to products seeking approval for the treatment of rare diseases. It is important to note that orphan drug designation is not a guarantee of marketing approval, but it does provide a number of benefits that can help to streamline the approval process.

The FDA process for orphan drug designation is fairly straightforward; however, the EMA’s Orphan Medicinal Product Designation (OMPD) process can be more complex and time intensive. It is important to note that having granted orphan drug designation at both the EMA and FDA does not prevent a product from also being granted marketing authorization as a breakthrough therapy.

Product eligibility for orphan drug designation

Orphan drug designation may be granted to drugs intended for the treatment, diagnosis or prevention of any rare disease/condition. Eligibility for orphan drug designation is not dependent on novelty or on the mechanism of action being known.


Rare diseases

A rare disease or condition is generally defined as one that affects fewer than 200,000 people in the United States. The National Institutes of Health (NIH) has a searchable online database that provides information on more than 7,000 rare diseases and conditions.

However, in order for a disease to be recognized as rare in Europe, it must affect no more than five people out of 10,000.

 Orphan Drug Designation Application

  1. FDA


The sponsor must provide the FDA with a submission to request orphan designation. The Center for Drug Evaluation and Research (CDER) evaluates the eligibility of products seeking orphan drug designation, which includes the following criteria:


  • The drug or biologic intended to treat, diagnose or prevent the disease/condition is intended to address an unmet medical need


  • The disease/condition is serious or life-threatening


  • The drug is intended for use in rare patients


Design Human


  1. EMA


The sponsor must provide the EMA with a submission to request orphan medicinal product designation, which includes the following:


  • The product that is the subject of the application is intended for the diagnosis, prevention or treatment of a rare disease/condition


  • The product is intended to address an unmet medical need


  • The product is expected to provide significant benefit to patients with the disease/condition


  • There is no reasonable expectation that the product will generate sufficient income to cover its development and marketing costs


  • There is no satisfactory method of diagnosis, prevention or treatment for this disease/condition known to the medical community in the country where it affects more than five people out of 10,000


  • It is likely that the product, if approved, would be granted a marketing authorization for this disease/condition

Case study

Coordination and Regulatory support across Europe for an innovative product/orphan drug

The Vice-President Global Regulatory Affairs of a US based company developing an important innovative product (orphan drug) contacted BlueReg to...

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Benefits of Orphan Drug Designation


In addition to a potential reduction in the regulatory filing fees, there are a number of benefits that may be granted to a product that has been granted orphan drug designation by both the EMA and FDA:


  • Years of marketing exclusivity in the EU following a first authorization


  • Regulatory fee reductions for clinical trials


  • Access to specialized medical expertise from the authorities


  • Free scientific advice from Committee for Medicinal Products for Human Use (CHMP)


  • Priority review by the FDA


  • Fast-track designation by the FDA

Outsourcing Regulatory Affairs projects for orphan drugs


BlueReg can determine if your product is eligible for orphan drug designation and provide the necessary support for your European Medicines Agency (EMA) / Food and Drug Administration (FDA) application.

Regulatory expert woman surrounded by orphan drugs

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