Orphan drug designation (status)

Regulatory affairs services – Orphan Drug Designation

In the European Union (EU) and in the United States (US) the respective government agencies provide incentives to companies developing treatments for rare diseases. Treatments meeting the criteria are often referred to as ‘orphan drugs’.

The criteria for orphan designation (or orphan ‘status’) in the European Union (EU) and United States (US) differ.

The EU criteria can be summarised as:

  • The medicinal product is intended for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition affecting no more than five in 10,000 persons in the EU.
  • The medicinal product it is intended for the diagnosis, prevention or treatment of a life-threatening, seriously debilitating or serious and chronic condition and that the revenue generated after marketing of the product would not cover cost of development.
  • No satisfactory method of diagnosis, prevention or treatment of the condition is authorised, or, the medicinal product will be of significant benefit to those affected by the condition.

The US criteria can be summarised as:

  • The disease or condition for which the drug is intended affects fewer than 200,000 people in the US or, if the drug is a vaccine, diagnostic drug, or preventative drug, the persons to whom the drug will be administered in the US number fewer than 200,000 per year,


  • Where the condition affects 200,000 or more people, or for a vaccine, diagnostic drug, or preventive drug to be administered to 200,000 or more persons per year in the US, the costs of development of the drug for the indication would not be recovered by sales of the drug in the US

Companies with an orphan designation for a medicinal product benefit from incentives such as:

  • Protocol assistance (scientific advice for orphan medicines during the product-development phase)
  • Direct access to centralised marketing authorisation and 10-year marketing exclusivity
  • Fee reductions or exemptions
  • Provision of grants to fund clinical research

Companies developing products which they consider meet the relevant criteria and wishing to benefit from these incentives may make a common application to the FDA/EMA or submit separate applications.

vector pharma supports company’s regulatory affairs plans by helping to assess a product’s eligibility, advise on the procedure to follow and make the best case when compiling the relevant application(s).  Preparation of a well-researched and cogently presented application will reduce the possibility of delays and failure to obtain the important orphan designation.

We frequently hear from companies who have encountered difficulties after failing to make the best possible application.  The consequences can be serious not only in financial terms but for the patient’s who may not benefit from important new treatment if the development programme is delayed or cancelled.

We are always willing to help in the above situation but prefer to help our clients avoid finding themselves in the predicament.