Introduction

"Compassionate Use" was introduced into the German legislation with the 14th amendment of the German Medicinal Product Act (AMG) and modified by the amendment of the German Medicinal Product Act in July 2009. Section 21 sub-section 2 No 6 of the German Medicinal Product Act provides (Tanslation):

"(2) A marketing authorisation (Zulassung) shall not be required for medicinal products which: [...]
6. are made available free of charge under the conditions specified to in Article 83 of Regulation (EC) No. 726/2004 for administration to patients with a seriously debilitating disease or whose disease is life-threatening, and who cannot be treated satisfactorily with an authorised medicinal product; this applies equally to medicinal products which do not fall under the categories stipulated in Article 3 first or second paragraph of Council Regulation (EC) No. 726/2004; rules of procedure shall be specified in an ordinance pursuant to Section 80. [...]"

The Ordinance on Medicinal Products for Compassionate Use (AMHV) pursuant to Section 80 of the German Medicinal Product Act was published on 21 July 2010 in the Federal Law Gazette 2010 part I No 37 and came into force on 22 July 2010. A non-offical translation is available for download.

Procedure

The Ordinance on Medicinal Products for Compassionate Use (AMHV) came into force on 22 July 2010. The Ordinance on Medicinal Products for Compassionate Use introduces the confirmed notification procedure for compassionate use programmes. BfArM and PEI are now the competent authorities for the confirmed notification procedure of compassionate use programmes according to their responsibilities as regulated by Section 77 of the German Medicinal Product Act. Since 23 July 2010 the commencement of any new compassionate use programme requires a confirmation of notification by the higher federal competent authority (in this case, BfArM).

BfArM has published a guide on submitting compassionate use programmes and a notification form

Guideline for Notification of a Compassionate Use Programme

Compassionate use notifications should be submitted in paper and CD-ROM/DVD to the following address:

Federal Institute for Drugs and Medical Devices (BfArM)
Clinical Trials/GCP
Kurt-Georg-Kiesinger Allee 3
D-53175 Bonn
Germany

For more information feel free to contact the BfArM at ct@bfarm.de

We would like to take this opportunity to inform you that all submissions on Medicinal Products for Compassionate Use (e.g. new notifications, notifications of changes, subsequent deliveries, etc.) can now be submitted electronically via the Common European Submission Platform (CESP). Detailed instructions for this can be found on our homepage. Submission via CESP replaces the sending of a notification in paper form as well as the sending of CDs or DVDs.

We would like to point out that as of the entry into force of the AMHV, all new medicinal product hardship programmes pursuant to Section 4 AMHV may only be started by the responsible person after receipt of a confirmation of the notification by the competent higher federal authority.

Important Notice

The Ordinance on Medicinal Products for Compassionate Use is only applicable for compassionate use programmes, i.e. cohort programmes which are intended for a group of patients (cohort compassionate use). A 'named patient compassionate use' is not in the scope of the notification procedure as laid down in the Ordinance on Medicinal Products for Compassionate Use. Please refer to the following flow chart for the decision on compassionate use:

Current Compassionate Use Programmes confirmed by BfArM

A list of currently confirmed and active compassionate use programmes is available here:

List of confirmed compassionate use programmes in Germany (list in German)

Electronic reporting of suspected cases of serious adverse reactions

In Section 6 (1) No. 1 AMHV, the AMHV requires the electronic reporting of suspected cases of serious adverse drug reactions to the competent higher federal authority. The obligation to notify the BfArM is deemed to be fulfilled if the adverse reaction is reported to the EMA's EudraVigilance database.

Information provided by the EMA

The EMA has published CHMP opinions on "compassionate use" programmes as well as a "Question and Answer" document on "compassionate use" programmes:

Information on "compassionate use" and list of CHMP opinions on "compassionate use" programmes

"Question and Answer" document on "compassionate use" programmes

Guideline on Compassionate Use of Medicinal Products, Pursuant to Article 83 of Regulation (EC) No 726/2004