title: Six-year activity on approval of compassionate use of medicines by the Ethics Committee of the University Hospital of Bologna (Italy): time to update rules and recommendations.
abstract: OBJECTIVE
  Compassionate use of forthcoming drugs has become an increasing pathway through which patients can take advantage of promising medicines. We aimed to analyse the main features of the requests of compassionate use submitted to the Independent Ethics Committee (IEC) of the University Hospital of Bologna in the period 2010-2015.
  
  
  METHODS
  The present analysis concerns the requests of compassionate use received by the IEC in the period 2010-2015. For each requested drug, we paired the date of the first request to our IEC with the date(s) of (a) submission to EMA, (b) CHMP positive opinion, and (c) European marketing authorization (if issued).
  
  
  RESULTS
  In the period 2010-2015, our IEC received compassionate use requests for 610 patients. Most of the requests concerned patients suffering from solid or haematological cancers not responsive to first or second line of treatment. Sixty-five couples of medicine/clinical condition (corresponding to 56 individual medicines) were submitted to our IEC, and 62 of them regarded products following the centralised procedure at the EMA. Twenty-one out of the latter (34%) had already obtained CHMP positive opinion.
  
  
  CONCLUSIONS
  Our results indicate that compassionate use of forthcoming medicines represents a not negligible portion of the therapies utilized in hospital care. The observed large resort to medicines still on trial may suggest that doctors are more aware with the potential benefits of the new drugs. However, this trend may also indicate an increasing marketing activity of the pharmaceutical industry, addressing to get the clinicians used to the upcoming medicines.
authors.affiliation: University of Bologna, University Hospital St. Orsola-Malpighi,, Via Albertoni 15, 40138, Bologna, Italy. nicola.montanaro@unibo.it.
authors.forename: Nicola
authors.identifier: http://orcid.org/0000-0002-5710-8077
authors.initials: N
authors.lastname: Montanaro
authors.affiliation: Unit of Pharmacology, Department of Medical and Surgical Sciences, University of Bologna, via Irnerio 48, 40126, Bologna, Italy.
authors.forename: Mauro
authors.initials: M
authors.lastname: Melis
authors.affiliation: University Hospital St. Orsola-Malpighi, Via Albertoni 15, 40138, Bologna, Italy.
authors.forename: Stefania
authors.initials: S
authors.lastname: Proni
authors.affiliation: University Hospital St. Orsola-Malpighi, Via Albertoni 15, 40138, Bologna, Italy.
authors.forename: Giacomo
authors.initials: G
authors.lastname: Chiabrando
authors.affiliation: Unit of Pharmacology, Department of Medical and Surgical Sciences, University of Bologna, via Irnerio 48, 40126, Bologna, Italy.
authors.forename: Domenico
authors.initials: D
authors.lastname: Motola
mesh_terms: D057176:Compassionate Use Trials; D004345:Drug Industry; D017041:Ethics Committees; D006801:Humans; D007558:Italy
keywords: Compassionate use; EMA; Ethics Committee; New drugs