Pharmaceutical Regulatory Affairs: Orphan Drug Applications in the EU and USA

  • ID: 2863525
  • Training
  • Region: Europe, United States
  • Leopink
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Orphan drugs are those for relatively rare indications. Special procedures and incentives apply to their registration, to encourage pharmaceutical companies to develop them. This module provides information necessary to enable the preparation of applications for orphan drug status in the EU and USA. The procedures for making an application are detailed, including the collection of supporting data and the acquisition of protocol assistance. In Europe the Centralised Procedure is now the mandatory route to gain marketing authorisation for orphan products. This module will serve as a useful reference tool to keep abreast of the impact of the new regulatory requirements.


Define orphan drugs in the USA and EU.

Describe the procedures for gaining orphan product designation in the USA and EU.

Outline the advantages and legal ramifications of gaining orphan product designation in the USA and EU.
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This brief session introduces the concept of orphan drugs, what they are and why they exist. It also identifies the criteria for orphan designation and gives a high level overview of the process of orphan designation and development.

US designation
This session provides an understanding of the characteristics of orphan drug designation and the commercial advantages associated with the development of orphan drugs in the USA.

US procedure
An application for orphan product designation in the USA has to be made to the Office of Orphan Products Development. Although there is no formal structure to the application, the FDA has a nine-point checklist of requirements which must be addressed by the sponsor. This session will provide information on each item of the list, the duration of the review, what happens after successful designation, and what to do if your application is refused.

EU designation
This session will explain the definition of an orphan product in the European Union and describe the role of the European Medicines Agency and the Committee for Orphan Medicinal Products.

EU procedure
Applications for orphan status within the EU are made to the EMA, and the Committee for Orphan Medicinal Products assesses the application. This session will describe how to make an application for orphan medicinal product designation to the EMA, what to do after approval is received, and how to make an appeal if you receive a negative opinion.

Multiple-choice mastery assessment.
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