title: Haematological anticancer drugs in Europe: any added value at the time of approval?
abstract: OBJECTIVE
  Current European regulations only require drugs to be safe and effective, although there is heavy demand for comparative efficacy data to demonstrate the added value of new drugs. The objective of the analysis reported here was to assess the added value of new anticancer drugs for haematological malignancies that have been approved by the European Medicines Agency (EMEA) based on the clinical data provided at the time of submission.
  
  
  METHODS
  Information on the evidence supporting the approval was extracted from the European Public Assessment Reports (EPARs). Documents were surveyed for new applications and for subsequent extensions between January 1995, when the EMEA was set up, and May 2006. The added value of newly approved drugs was assessed by an algorithm that evaluates the strength of evidence based on methodological appropriateness (randomised comparison) and the importance of clinical advantage (in terms of the magnitude of benefit, hardness of outcome measures, adequacy of comparator).
  
  
  RESULTS
  Eleven anticancer drugs were analysed. Of 17 indications, nine (53%) were approved on the basis of single-arm trials (SATs), and eight (47%) were approved on the basis of randomised controlled (clinical) trials (RCTs). The most frequently used endpoint was response rate (12 of 17 indications, 70%). On the basis of our criteria, only four of the 11 drugs show a consistent added value.
  
  
  CONCLUSIONS
  We were unable to establish an added value for about two thirds of the drugs evaluated in this study, primarily due to methodological aspects related to study design and endpoint robustness.
authors.affiliation: Laboratory of Regulatory Policies, Mario Negri Institute for Pharmacological Research, Via Eritrea 62, 20157 Milan, Italy. bertele@marionegri.it
authors.forename: Vittorio
authors.initials: V
authors.lastname: Bertele'
authors.forename: Rita
authors.initials: R
authors.lastname: Banzi
authors.forename: Filippo
authors.initials: F
authors.lastname: Capasso
authors.forename: Giovanni
authors.initials: G
authors.lastname: Tafuri
authors.forename: Francesco
authors.initials: F
authors.lastname: Trotta
authors.forename: Giovanni
authors.initials: G
authors.lastname: Apolone
authors.forename: Silvio
authors.initials: S
authors.lastname: Garattini
mesh_terms: D000465:Algorithms; D000970:Antineoplastic Agents / Q000592:standards* / Q000627:therapeutic use; D017277:Drug Approval / Q000331:legislation & jurisprudence*; D005062:European Union; D019317:Evidence-Based Medicine; D019337:Hematologic Neoplasms / Q000188:drug therapy*; D006801:Humans
chemical_list: D000970:Antineoplastic Agents