Abstract
Background'Compassionate use' programmes allow medicinal products that are not authorised, but are in the development process, to be made available to patients with a severe disease who have no other satisfactory treatment available to them. We sought to understand how such programmes are regulated in ten European Union countries.MethodsThe European Clinical Research Infrastructures Network (ECRIN) conducted a comprehensive survey on clinical research regulatory requirements, including questions on regulations of 'compassionate use' programmes. Ten European countries, covering approximately 70% of the EU population, were included in the survey (Austria, Denmark, France, Germany, Hungary, Ireland, Italy, Spain, Sweden, and the UK).ResultsEuropean Regulation 726/2004/EC is clear on the intentions of 'compassionate use' programmes and aimed to harmonise them in the European Union. The survey reveals that different countries have adopted different requirements and that 'compassionate use' is not interpreted in the same way across Europe. Four of the ten countries surveyed have no formal regulatory system for the programmes. We discuss the need for 'compassionate use' programmes and their regulation where protection of patients is paramount.Conclusions'Compassionate use' is a misleading term and should be replaced with 'expanded access'. There is a need for expanded access programmes in order to serve the interests of seriously ill patients who have no other treatment options. To protect these patients, European legislation needs to be more explicit and informative with regard to the regulatory requirements, restrictions, and responsibilities in expanded access programmes.
Highlights
European Regulation 726/2004/EC is clear on the intentions of ‘compassionate use’ programmes and aimed to harmonise them in the European Union
It clearly states that patients must have a chronic, seriously debilitating, or life-threatening disease, that the medicinal product must be undergoing assessment in a clinical trial or be the subject of a marketing authorisation application, and that authorisation of the ‘compassionate use’ programme itself is necessary [1]
Regulation 726/2004/EC lacks details on the authorisation procedures, and allows the implementation of ‘compassionate use’ programmes to be governed by individual member states [1]
Summary
The ECRIN working group on regulatory requirements and interaction with competent authorities designed and conducted an international survey [6]. The ECRIN working group on regulatory requirements and interaction with competent authorities was composed of two chairpersons, at least one expert from each European ECRIN country, and an ECRIN European correspondent (a person trained in clinical research, working at the national coordination on the implementation of the ECRIN project) from each of following countries: Austria, Denmark, France, Germany, Hungary, Ireland, Italy, Spain, Sweden, and the United Kingdom. The two co-chairpersons and two members of the working group on regulatory requirements and interaction with competent authorities drafted the survey. Some data are reported to the regulatory authorities Medicinal product accessed through the programme, afterwards those patients can have access before the product is licensed. Spontaneous adverse events may be reported Medicinal product available on prescription
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