Compassionate use of interventions: results of a European Clinical Research Infrastructures Network (ECRIN) survey of ten European countries

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.

An overview of Compassionate Use Programs in the European Union member states

“…Besides, a few gaps exist in the literature, due to changes and revisions of regulations and pathways that happen over time (13). Hence, there is a dearth of information in the published literature on existing CUP and current trends in all EU member states.…”

“…Previously, the information available publicly on CUP, especially on legislation was limited and in many cases outdated when compared with the available grey literature (6,12,13). However, the current systematic search addresses these gaps by including data from grey literature and peer-reviewed journal articles.…”

“…First, CUPs benefit patients unable to participate in clinical trials due to mobility issues or who fail to fulfill the eligibility criteria (13). They are also preferred when no treatment options are available or access to investigational drug/biologics/ medical devices is the last resort for patients suffering from serious diseases/disorders (13). Through early access programs, patients have access to promising drugs at an earlier stage during the life cycle, for instance, post phase II.…”

“…Otherwise, patients have to wait for a considerable amount of time until the drug In Slovenia, in line with the EU regulation, CUP is: -The administration of a new active ingredient, constituting a significant therapeutic, scientific and technical innovation -For drugs obtaining marketing authorization or clinical testing of medicines -For a group of patients with a chronically or seriously debilitating disease that cannot be satisfactorily treated with medicines that have marketing authorization -The sponsor/manufacturer of a CUP must provide the product free of charge, which includes the costs of supply and wholesale distribution. is authorized and is on the market, if not for the early access programs (13). Second, pharmaceutical and biotechnology companies provide a fast and efficient response to patient demand outside of traditional access routes due to such programs (87,90).…”

Ethical Aspects of Vulnerable Group of Patients in Clinical Trials

“…It states that patients must have a chronic or life-threatening disease, and the medicinal product must be undergoing assessment in a clinical trial or be in the marketing authorisation stage. However, details around authorisation procedures are still missing and ultimately the programmes are governed by individual member states [7].…”