Submitting annual reports on medicine development

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This page provides information on the report that sponsors must submit to the European Medicines Agency (EMA) every year after their medicine receives an orphan designation, until they submit an application for marketing authorisation. 

If a sponsor subsequently withdraws a marketing authorisation application, or the application receives a negative opinion from the Committee for Medicinal Products for Human Use (CHMP), they need to resume submitting annual reports on development. 

These annual reports on development provide information on the status of the development of the medicine, including:

  • a review of ongoing clinical studies;
  • a description of the investigation plan for the coming year;
  • any anticipated or current problems in the process, difficulties in testing and potential changes that may have an impact on the medicine’s orphan designation.

A template for the report is available below, together with a guideline providing advice on the preparation of these reports.

Update: On 19 June 2018, EMA launched a new secure online portal for sponsors to submit applications for orphan designation and to manage post-designation activities. For more information, see Applying for orphan designation.

Single report with United States Food and Drug Administration

Sponsors of medicines with orphan designation both in the European Union and the United States (US) can submit a single report using the template below to the EMA (via Eudralink) and to the US Food and Drug Administration (FDA) at opdar@fda.hhs.gov.

The submission of a single report to the two agencies is voluntary. The two agencies carry out independent reviews and assessments of the report's contents.

Documents

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Document(s) Language Status First published Last updated Effective Date
Annual report on a designated orphan-medicinal-product (English only)   2011-11-24 2017-09-26  
Note for guidance on the format and content of the annual report on the state of development of an orphan medicinal product (English only) adopted 2009-02-19 2014-09-10