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