EMEA - Guideline on Compassionate Use of Medicinal Products

The grounds for triggering a CHMP assessment are based on the principle laid down in Recital 33 of Regulation (EC) No 726/2004, where a common approach should be followed whenever possible, as regards the criteria and the conditions for compassionate use. In this context, to consider assessing, the CHMP will check the following:

- The criteria listed in paragraphs 1 and 2 of Article 83 are met,

- A MS has notified the EMEA and requested a CHMP opinion, or,

- If more than one MS have notified the EMEA of their use of Article 83 for the same compassionate use programme, without an explicit request for a CHMP opinion, the CHMP will consider, in the interest of patients, whether there is a need to give an opinion.

When they notify the EMEA, MS(s) may have already collected and assessed data following a request for compassionate use. In other situations, MS(s) may have notified the EMEA and asked for a CHMP opinion without having yet collected and assessed data.

If a CHMP opinion is to be adopted, the CHMP should use any available data and the existing MS(s)’ assessment(s) if available, on the quality, the safety and the efficacy of the medicinal product, the comprehensiveness of which will depend of the stage of development of the product. The CHMP may request additional data to the applicant.

Detailed justifications should be provided to support the claim that the medicinal product meets the criteria listed in Article 83(2), in accordance with the definitions provided in this guideline.

The scientific data submitted should allow the evaluation of the conditions of use of the medicinal product, for the intended target population, in the context of compassionate use. In terms of efficacy, the assumptions for compassionate use may be based on mature randomised phase III trials (e.g. in case of parallel assessment of compassionate use and application for marketing authorisation). However, acceptable assumptions may rely on promising early data observed in exploratory trials (e.g. uncontrolled phase II trials, exploratory subgroup analysis from phase III trials). In terms of safety, submission of all available data, which may contribute to refine the conditions of use defined in the opinion, is encouraged.