Compassionate use of medicinal products: a comparison between the European and the United States of America regulatory frameworks

The treatment of patients suffering from chronic, potentially lethal diseases is a lengthy and disheartening process, especially in cases where patients cannot be satisfactorily treated with authorized medications. In these cases, the only therapeutic option may be experimental drugs. However, in some cases, not all patients meet the inclusion criteria for enrollment in a trial. This problem can be addressed through a “compassionate use”. Because inadequate information can be a barrier it is essential to clarify the regulatory framework. Despite the availability of reviews, information available on this topic is limited also due to changes in regulations that occur over time. Moreover, the different definitions between states and the lack of a common legislative approach makes the process confusing. The purpose of this article is to compare the legislative frameworks currently in place in European Union and Usa pharmaceutical markets, in order to highlight their strengths, analogies and differences, in a global context that goes beyond the regulatory realities of individual countries.