In times of situational therapeutic impasse health care professionals (HCPs) are under
pressure to conduct off-label unlicensed and compassionate drug use - generally summarized
under the term non-licensed drug use (NDU). Liability contractual and penal risks pose a
problem when treating a patient in a non-licensed way. There is a knowledge gap about
institutional and governmental methods to resolve these problems. Different countries have
developed strategies to manage NDU. In her book Vanessa Platé compares the effects of NDU on
pharmaceutical legislation in selected industrial countries and determines strategies brought
forth by NDU. Furthermore the development of a general regulatory approach to the management
of NDU is sought. Semi-structured qualitative interviews comparison of laws and literature
research are part of this study which includes Canada the U.S. the U.K. Japan France
Germany Switzerland Austria and the transnational E.U. The comparison of the impact of NDU
focuses on terminology for NDU supply and necessity of NDU pharmaceutical promotion of NDU
legal responsibility for NDU and public policies related to NDU. The results of the study
suggest there is indeed a circumstantial need for off-label unlicensed and compassionate use
but also showed isolated evidence suggesting intermittent non-rational NDU. Furthermore the
legal comparison showed evidence of legal obligations for physicians to perform off-label
unlicensed and compassionate drug use. Vanessa Platé also found proof of inappropriate
off-label marketing on the part of market authorization holders (MAH). On the other hand a
demand for information on NDU on behalf of HCPs was present. Obtained results illustrate
cross-liability for HCPs and MAH. Finally results demonstrated regulatory strategies of
different efficiencies: In the E.U. for instance incentives for new indications were (a)
limited to one year or (b) restricted to (i) pediatrics or (ii) rare disorders. The absence of
statutory terminology causes incoherent interpretation of NDU across the researched nations
harmonization is crucial for an effective concept development. There is a situational need for
early access to yet unapproved treatments. Denial of (non-licensed) treatment is considered
unethical. Proposed solutions for an enduring management of NDU are firstly the amendments of
templates for patient information leaflets by competent authorities to include well-recognised
off-label use secondly full development research and duly authorized marketing of medicinal
products used compassionately and thirdly modelled on German procedure a modified standard
market authorization for essential unlicensed drugs.
