In the European Union, early access may be achieved through "compassionate use programs", which are contemplated by Article 83 of Regulation (EC) No 726/2004, on centrally approved medicinal products, while specific regulation of these is left to the Member States.
According to the Guideline on Compassionate Use of the European Medicines Agency, compassionate use programs should apply to group of patients and should be kept distinct from a named-patient supply (as meant in Article 5 of Directive 2001/83/EC).
In that context, "Expanded access programs" are usually defined as a subcategory of compassionate use, where access is allowed on the basis of eligibility criteria and conditions as applied for enrolling the patients in the clinical trial for the concerned product (e.g. expanded access may be used to ensure the continuation of therapy by patients formerly enrolled in a trial that has been terminated). In the United States, "expanded access" is the common terminology used for early access to medicinal products which has not yet been authorised.
Italy has a broad range of mechanisms that allow early access to the market of medicinal products that are not yet authorised and which do not perfectly match with the above definitions, even though essentially achieving the same purpose. The most important instrument is certainly represented by the Law No. 648 of 23 December 1996 (Law No. 648/96), which sets out a list of non-authorised medicinal products that are purchased by Italian hospitals for the subsequent distribution to patients free of charge.
The Ministerial Decree of 8 May 2003 was enacted in order to enable patients, who were not enrolled in clinical trials already concluded, to benefit from medicinal products for which clinical trials were still ongoing or were completed, prior to approval. The supply regime under the Ministerial Decree of 8 May 2003 (and the same applies to the new regulation) is substantially amenable to expanded access as defined at European Union level, even though it also covers instances of compassionate use and named patient supply.
From an economic standpoint, it is important to note that, as opposed to early access programs according to the Law No. 648 of 23 December 1996, where products are purchased by the National Health System from pharmaceutical companies at a market price, medicinal products supplied under Ministerial Decree of 8 May 2003 are always provided free of charge by the pharmaceutical company.