title: Concepts of 'personalization' in personalized medicine: implications for economic evaluation.
abstract: BACKGROUND
  This study assesses if, and how, existing methods for economic evaluation are applicable to the evaluation of personalized medicine (PM) and, if not, where extension to methods may be required.
  
  
  METHODS
  A structured workshop was held with a predefined group of experts (n = 47), and was run using a modified nominal group technique. Workshop findings were recorded using extensive note taking, and summarized using thematic data analysis. The workshop was complemented by structured literature searches.
  
  
  RESULTS
  The key finding emerging from the workshop, using an economic perspective, was that two distinct, but linked, interpretations of the concept of PM exist (personalization by 'physiology' or 'preferences'). These interpretations involve specific challenges for the design and conduct of economic evaluations. Existing evaluative (extra-welfarist) frameworks were generally considered appropriate for evaluating PM. When 'personalization' is viewed as using physiological biomarkers, challenges include representing complex care pathways; representing spillover effects; meeting data requirements such as evidence on heterogeneity; and choosing appropriate time horizons for the value of further research in uncertainty analysis. When viewed as tailoring medicine to patient preferences, further work is needed regarding revealed preferences, e.g. treatment (non)adherence; stated preferences, e.g. risk interpretation and attitude; consideration of heterogeneity in preferences; and the appropriate framework (welfarism vs. extra-welfarism) to incorporate non-health benefits.
  
  
  CONCLUSIONS
  Ideally, economic evaluations should take account of both interpretations of PM and consider physiology and preferences. It is important for decision makers to be cognizant of the issues involved with the economic evaluation of PM to appropriately interpret the evidence and target future research funding.
authors.affiliation: Helmholtz Zentrum München, Institute of Health Economics and Health Care Management, Ingolstädter Landstr. 1, 85764, Neuherberg, Germany, rogowski@helmholtz-muenchen.de.
authors.forename: Wolf
authors.initials: W
authors.lastname: Rogowski
authors.forename: Katherine
authors.initials: K
authors.lastname: Payne
authors.forename: Petra
authors.initials: P
authors.lastname: Schnell-Inderst
authors.forename: Andrea
authors.initials: A
authors.lastname: Manca
authors.forename: Ursula
authors.initials: U
authors.lastname: Rochau
authors.forename: Beate
authors.initials: B
authors.lastname: Jahn
authors.forename: Oguzhan
authors.initials: O
authors.lastname: Alagoz
authors.forename: Reiner
authors.initials: R
authors.lastname: Leidl
authors.forename: Uwe
authors.initials: U
authors.lastname: Siebert
mesh_terms: D003362:Cost-Benefit Analysis; D003657:Decision Making; D003695:Delivery of Health Care / Q000191:economics*; D006801:Humans; D057285:Precision Medicine / Q000191:economics / Q000379:methods*