title: 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.
  
  
  METHODS
  The 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).
  
  
  RESULTS
  European 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.
authors.affiliation: Copenhagen Trial Unit, Centre for Clinical Intervention Research, Copenhagen University Hospital (DCRIN), Copenhagen, DK-2100, Denmark. kate.whitfield@ctu.rh.dk
authors.forename: Kate
authors.initials: K
authors.lastname: Whitfield
authors.forename: Karl-Heinz
authors.initials: KH
authors.lastname: Huemer
authors.forename: Diana
authors.initials: D
authors.lastname: Winter
authors.forename: Steffen
authors.initials: S
authors.lastname: Thirstrup
authors.forename: Christian
authors.initials: C
authors.lastname: Libersa
authors.forename: Béatrice
authors.initials: B
authors.lastname: Barraud
authors.forename: Christine
authors.initials: C
authors.lastname: Kubiak
authors.forename: Lea
authors.initials: L
authors.lastname: Stankovski
authors.forename: Xina
authors.initials: X
authors.lastname: Grählert
authors.forename: Gabriele
authors.initials: G
authors.lastname: Dreier
authors.forename: Sebastian
authors.initials: S
authors.lastname: Geismann
authors.forename: Wolfgang
authors.initials: W
authors.lastname: Kuchinke
authors.forename: Anke
authors.initials: A
authors.lastname: Strenge-Hesse
authors.forename: Zsuza
authors.initials: Z
authors.lastname: Temesvari
authors.forename: Gyorgy
authors.initials: G
authors.lastname: Blasko
authors.forename: Gabriella
authors.initials: G
authors.lastname: Kardos
authors.forename: Timothy
authors.initials: T
authors.lastname: O'Brien
authors.forename: Margaret
authors.initials: M
authors.lastname: Cooney
authors.forename: Siobhan
authors.initials: S
authors.lastname: Gaynor
authors.forename: Arrigo
authors.initials: A
authors.lastname: Schieppati
authors.forename: Mariantonia
authors.initials: M
authors.lastname: Serrano
authors.forename: Fernando
authors.initials: F
authors.lastname: de Andres
authors.forename: Nuria
authors.initials: N
authors.lastname: Sanz
authors.forename: Raquel
authors.initials: R
authors.lastname: Hernández
authors.forename: Germán
authors.initials: G
authors.lastname: Kreis
authors.forename: Charlotte
authors.initials: C
authors.lastname: Asker-Hagelberg
authors.forename: Hanna
authors.initials: H
authors.lastname: Johansson
authors.forename: Adeeba
authors.initials: A
authors.lastname: Asghar
authors.forename: Jean-Marc
authors.initials: JM
authors.lastname: Husson
authors.forename: Jacques
authors.initials: J
authors.lastname: Demotes
authors.forename: Christian
authors.initials: C
authors.lastname: Gluud
mesh_terms: D035843:Biomedical Research; D002986:Clinical Trials as Topic; D057176:Compassionate Use Trials / Q000331:legislation & jurisprudence; D005060:Europe; D006801:Humans