The use of medicines in exceptional circumstances in Spain: adding perspective to early access

Abstract

The use of medicines in exceptional circumstances refers to the use of non authorized medicines, or which are used outside the authorized conditions, and includes three different situations: (a) The use of medication in research or compassionate use; (b) The use of foreign medicines (medicines unauthorized in one country but authorized in others), and; (c) Off-label use (medicines outside the authorized conditions of use, or use out of indication). In Spain we intend to govern the period between authorization and price, with standard criteria, through Therapeutic Positioning Reports. The keys to manage this situation, in our opinion, could be: (1) A restrictive interpretation of access that can be made through the Therapeutic Positioning Reports, (2) with an agreement so that the medication is paid at the price at which finally it is financed, and (3) restricting this period of time to only a few months or less (e.g. orphan medicines). Introduction Access to medicines in exceptional circumstances (compassionate use, off-label use, and foreign drugs) is a health need. Many of these non-covered pharmacological needs are given in committed clinical situations, understanding such as chronic or severely debilitating diseases or those that they place patient's life in danger, and cannot be satisfactorily addressed with one authorized and commercialized medication. The use of medicines in exceptional circumstances refers to the use of non authorized medicines, or which are used outside the authorized conditions, and includes three situations different well delimited in the Spanish Royal Decree (RD) 1015/2009 regulating the availability of medicinal products in special situations: (a) The use of medication in research or compassionate use; (b) The use of foreign medicines (medicines unauthorized in Spain but authorized in other countries, and; (c) Off-label use (medicines outside the authorized conditions of use or use out of indication). The legislation limits the use of each one of them for those exceptional circumstances in which there is no other commercial therapeutic alternative and describe two different situations for each one, the individual use or those situations in which there may be a collective use (which the legislation states, respectively, temporary use authorization, use protocol or recommendations of use). The Spanish procedure is absolutely guarantor [1], requiring for its authorization: 1) the informed consent of the patient; 2) the request of a specialist physician; 3) the conformity of the Medical Chief Director of the healthcare center; 4) the conformity of the promoter or pharmaceutical company, and, finally; 5) the authorization by the Spanish Agency of Medicines and Medical Devices.