title: Four years of early benefit assessment of new drugs in Germany: a qualitative study on methodological requirements for quality of life data.
abstract: BACKGROUND
  Since 2011, an early benefit assessment (EBA) of new drugs constricts free price setting in Germany. According to the Pharmaceutical Market Restructuring Act (AMNOG), pharmaceutical companies are obliged to demonstrate added benefit of new drugs over comparative treatment. Benefit is usually evaluated by the Institute for Quality and Efficiency in Health Care (IQWiG). The final appraisal is made by the Federal Joint Committee, Germany's highest-ranking decision body in the health sector, triggering drug prize negotiations between companies and statutory health insurance funds. One of four evaluation criteria is quality of life (QoL). QoL outcomes have, however, only rarely been pivotal in EBAs.
  
  
  OBJECTIVE
  This study determined methodological requirements for QoL measurement and data presentation in the EBA.
  
  
  METHODS
  In a qualitative content analysis, documents of all EBAs completed by 2014 were searched for the term QoL. Relevant passages of all EBAs of 2011-2013 were independently extracted and reduced to key content by two researchers. Recurring patterns were identified and verified through comparison with EBAs of 2014.
  
  
  RESULTS
  We identified a range of requirements regarding QoL assessment, analysis, presentation, and interpretation, which go beyond official regulations. Disease-specific questionnaires are preferred and have to be validated according to certain standards and in the respective patient group. Effects must exceed the minimal important difference, which in turn must be validated in compliance with specific requirements. Often, instruments were not accepted as QoL measures, sometimes inconsistently across EBAs. Another frequent reason for non-acceptance of QoL data was that more than 30 % of randomized patients could not be analyzed due to missing data.
  
  
  CONCLUSIONS
  Non-compliance with methodological requirements for QoL evidence impairs chances for positive benefit evaluation.
authors.affiliation: German Center for Health Services Research in Dermatology (CVderm), Institute for Health Services Research in Dermatology and Nursing (IVDP), University Medical Center Hamburg-Eppendorf, Martinistraße 52, 20246, Hamburg, Germany. c.blome@uke.de.
authors.forename: Christine
authors.initials: C
authors.lastname: Blome
authors.affiliation: German Center for Health Services Research in Dermatology (CVderm), Institute for Health Services Research in Dermatology and Nursing (IVDP), University Medical Center Hamburg-Eppendorf, Martinistraße 52, 20246, Hamburg, Germany.
authors.forename: Matthias
authors.initials: M
authors.lastname: Augustin
authors.affiliation: Janssen-Cilag GmbH, Neuss, Germany.
authors.forename: Hidayet
authors.initials: H
authors.lastname: Metin
authors.affiliation: German Center for Health Services Research in Dermatology (CVderm), Institute for Health Services Research in Dermatology and Nursing (IVDP), University Medical Center Hamburg-Eppendorf, Martinistraße 52, 20246, Hamburg, Germany.
authors.forename: David
authors.initials: D
authors.lastname: Lohrberg
mesh_terms: D015982:Bias; D003362:Cost-Benefit Analysis / Q000379:methods*; D003365:Costs and Cost Analysis; D017277:Drug Approval / Q000331:legislation & jurisprudence / Q000379:methods*; D004345:Drug Industry / Q000379:methods*; D005858:Germany; D006801:Humans; D036301:Qualitative Research; D011788:Quality of Life; D015203:Reproducibility of Results; D012107:Research Design; D011795:Surveys and Questionnaires / Q000592:standards
keywords: AMNOG; Comparative effectiveness research; Early benefit assessment; Health technology assessment; Qualitative research; Quality of life
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