orphan drugs

Orphan Drug Designation.

What is orphan drug designation?

Orphan designation is a status that can be assigned to a medicine designed to treat rare health conditions, typically called orphan diseases. 

Many regulatory authorities around the world offer specific financial and support incentives for companies to develop medicines that can prevent or treat these rare diseases, since the market for these products is relatively small and specialized. 

Companies developing orphan medicines can apply for an orphan designation to take advantage of the relevant incentives available.

What are the criteria for a drug to qualify for an orphan designation?

European Union:

  • It must be intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating.
  • The prevalence of the condition in the EU must not be more than 5 in 10,000, or, it must be unlikely that marketing the medicine would generate sufficient returns to justify the investment needed for its development.
  • No satisfactory method of diagnosis, prevention or treatment of the condition concerned can be authorised, or, if such a method exists, the medicine must be of significant benefit to those affected by the condition.

Full information from the EMA at:

United States:

  • Drugs (includes biologics) for the prevention, diagnosis, or treatment of diseases or conditions affecting fewer than 200,000 persons in the US.
  • Drugs that will not be profitable within 7 years following approval by the FDA.

Full information from the FDA at:

What incentives are available?

European Union*

  • Fee Reduction on Protocol Assistance
  • Access to EMA centralised procedure
  • 10 years market exclusivity

United States

  • Tax credits for qualified clinical testing
  • Waiver of NDA/BLA user fees
  • Eligibility for 7-year marketing exclusivity (“orphan exclusivity”) upon marketing approval

* Additional incentives are available for companies with small and medium-sized enterprise (SME status). 

How can Arriello help?

Our Regulatory Affairs team has in-depth experience working in the orphan drug space and understand exactly how to get your product to your target markets efficiently.

Some of the ways we can support sponsors with orphan drug designation applications globally are as follows:

  • Act as the Orphan Drug Sponsor for non-EU clients or provide a US agent for filing to the FDA.
  • Agency liaison throughout the review process.
  • Pre-submission meetings (not mandatory but in some cases can be helpful).
  • Preparation or review of the application.
  • Submission through the applicable agency portal (e.g. EMA IRIS).
  • Oral explanation preparation and attendance.
  • Submission of Annual Reports.
  • Maintenance of orphan status at the time of MAA.

Whatever your requirements around orphan drug designation, you can be sure our regulatory experts have the experience and understanding to make sure you achieve your product objectives.