Orphanet: About orphan drugs
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Orphan drugs in the United States of America

As early as 1983, the Public Health authorities realised that legislation on orphan drugs was needed signing of the 'Orphan Drug Act'.

This law defines the 'orphan drug' with regard to prevalence (frequency) of the disease for which it is indicated in the American population.

In the US, the concept of 'orphan drug' does not simply cover pharmaceutical or biological products. It also covers medical devices and dietary or diet products. The OOPD (Office of Orphan Products Development) was created within the FDA (Food and Drug Administration). It is in charge of promoting the availability of safe and efficacious products for the treatment of rare diseases. The 'orphan' status allows the drug sponsor to benefit from incentives for the development of these products until the marketing approval.

These measures apply to all stages of the drug development :

For further information, you can go and see the list of the American orphan drugs.

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Public policy on orphan drugs in the USA

The legal status of orphan drugs in the USA dates back to 4 January 1983 with the passing of a law called the Orphan Drug Act :

Orphan drugs are used in diseases or circumstances which occur so infrequently in the USA, that there is no reasonable expectation that the cost of developing and making available in the USA a drug for such disease or condition will be recovered from sales in the USA for such drugs.

Several amendments were subsequently passed, aiming at defining the criteria orphan drugs must meet.

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Orphan drugs labelling and legal status

Orphan drugs are covered by a specific individual legal system endowing them with a special status. Granting orphan drug status is based upon an application dossier submitted to the Office of Orphan Products Developpement (OOPD), including :

Orphan drug status.
Orphan drug status FDA Office of Orphan Products Development.
Marketing Authorisation
  • Center for Drug Evaluation and Research
  • Center for Biologics Evaluation and Research
  • Center for Devices and Radiological Health
  • Center for Food and Safety & Applied Nutrition

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Incentives to orphan drugs providers in terms of R&D, intellectual property and marketing

Granting orphan drugs status may enable the sponsor to obtain the following advantages for the development of the product :

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