compassionate use programme

Medicinal products that have not been registered may be prescribed in highly exceptional cases.

There are two possibilities:

  1. The provision of a medicinal product on the basis of a physician’s statement (named patient). ‘Named patient’ is prescribed to an individual patient and falls under the jurisdiction of the Health Care Inspectorate (see www.igz.nl for more information);
  2. If the case involves a severe condition for which no alternative medicinal product is marketed and the as-yet unauthorised medicinal product will be registered (given a marketing authorisation) in the near future, the medicinal product manufacturer can submit a request to the MEB to come under consideration for the ‘compassionate use programme’.

The compassionate use programme concerns a specific situation in which the MEB considers the availability of new medicinal products for multiple patients (cohort) to be necessary prior to the awarding of the definitive marketing authorisation.

The legal context

The Regulation 726/2004/EC gives the outline of the European framework, however as a result of the transnational character of such programmes the assignment of compassionate use programmes falls under national jurisdiction. Thus the national competent authority should award permission for the availability of the medicinal product without market authorisation.

At the national level, the legal conditions have been included in the Medicines Act and the MEB has the authority to approve a compassionate use programme.

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