This side looks at the legal aspects of off-label drugs. For this we have a look at the handling in Europe and the United States of America.
Europe
In the European Union (EU), the handling of off-label drugs is done by national laws. However efforts are made, to work on unified regulations for the use of off-label drugs in all countries of the EU. In countries with no specific regulations, the prescriber is responsible for the off-label use and there are no specific reimbursements. Countries with regulations handle off-label use differently. In some countries, the prescribers have to ask for permission to use the drug as off-label (France and Hungary). In other countries (e.g. the Netherlands) it's only allowed to prescribe off-label drugs if professionals develope a protocol for the specific off-label use of a drug in a therapy. Regarding reimbursement there are different regulations as well. In France and Italy for example, costs for off-label use can be reimbursed even if therapy alternatives exist. [5]
Germany
In Germany, expert commissions for the off-label use of drugs have been formed in the national medicines agency. These evaluate the current knowledge about off-label drug use in different medical areas. The Federal Joint Committee (GBA) can then decide about reimbursement with recommendations from the expert groups and forward them to the statutory health insurance. A ruling by the Bundesverfassungsgericht in 2005 made reimbursement possible for off-label drugs that show only small efficiency for patients with life-threatening conditions and no alternative available therapy. [5]
Figure 1: regulations of off-label use (dark blue: existing regulations; light blue: no regulations; grey: not part of the study) [5]
USA
In the United States (US), the FDA is responsible for approving drugs. Their system distinguishes between physicians and drug providers. Physicians can use off-label drugs whenever they see a medical benefit for the patient. They should, but do not have to, provide reports about the acutal use and benefit of the off-label drug then. In contrast drug providers are not allowed to promote off-label use of their drugs. However they are allowed to distribute peer-reviewed articles dealing with the off-label use of specific drugs. A new ruling by the FDA might also force drug providers to label the off-label drugs if the providers intend to promote the use of these drugs for treatments that they are not approved for. Reimbursement by health care providers/insurance companies happens rarely. It depends on whether the off-label use is stated in a drug compendia or in the local Medicare carrier center policy. These compendias are said to be not up-to-date, so some physicians don't apply off-label drugs due to the risk that there might be no reimbursement. [1] [2] [3] [4] [6]