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EURORDIS conducts pilot survey to explore off-label use of medicines for rare diseases

October 2012
Know more about off-label drug use for rare diseasesWhen doctors prescribe a medicine for a use different from what is authorised on the label, this is called “off label” use. For example when the drug is prescribed for a different disease or when the dosage differs from the one stated on the label. Patients with rare diseases and their families are often familiar with this practice, or may not even realise that they are taking products that are prescribed “off-label”.

In order to explore and better understand “off-label” drug use, EURORDIS’ DITA (Drug Information, Transparency and Access) task force conducted a pilot survey from May to July 2012. For example, one parent, who responded to the pilot survey, described using a product to treat his 6-month old’s epilepsy, whereas the label indicated that the drug is not recommended for children between the ages of six months and three years.

The enquiry received over 250 responses, describing experiences with over 100 different medications for 90 different rare diseases. The preliminary results were presented to the European Medicines Agency in London last September.

The good news is that 86% of the respondents were satisfied by their experience using prescribed medicines “off-label”. The bad news is that 3 out of 4 people answering the survey would have liked more information about the benefits and risks of taking the drug “off-label”, and 1 out of 4 were not even told that the drug prescribed was “off-label” and therefore not recommended. In addition, one-third of the respondents experienced side effects.

EURORDIS has decided to continue its research into rare disease “off-label” use and to expand it to more patients in more countries. Future plans are to:

  • Build a database on off-label use in rare diseases;
  • Engage a dialogue with regulators on how to obtain more data on the benefits and risks of off-label uses, in particular with experts of the European Medicines Agency who are best placed to explore the data when available;
  • Discuss the utility of a consent form;
  • Explore other sources of information on off-label use in rare diseases, in particular by collaborating to healthcare professionals.



This activity is funded by an Operating Grant from the European Commission, Public Health Programme – DG Sante.
The responsibility of the content of this report lies with the project partners. The European Commission is not responsible for any use that may be made of the information contained therein.