Table 4 Concerns related to PP in MPLC

1. General concerns related to PP in industry, BRA/MA and HTA/reimbursement

 • Lack of clarity and (regulatory) guidance about:

  ○ Definition of PP, hampering communication between stakeholders [1, 62]

  ○ Under what conditions to measure/use PP [1, 19]

  ○ For which medical product to collect PP [19, 27, 37]

  ○ When to conduct a PP study: before, during or after clinical development [19, 27, 37]

  ○ What preference method to use [19, 40, 72]

  ○ Which attributes to select in a PP study [19, 30, 50]

  ○ How to assure validity in a PP study [19, 38]

  ○ Whose preferences to measure (e.g. required disease experience) [19, 27, 44, 54, 73]

  ○ How to deal with preference heterogeneity [54]

  ○ Which stakeholder should collect PP [38]

  ○ Who is responsible for PP results and potential biases in results [38]

 •Lack of familiarity among stakeholders with preference methods [16, 19, 24, 34]

 •Lack of patients’ knowledge and capability of expressing preferences [62]

2. Methodological concerns related to PP in industry, BRA/MA and HTA/reimbursement

 • Low validity and reliability of preference methods [19, 25, 43]

 • Overlap in interpretation of attributes and interacting/overlapping attributes [30, 35, 50]

 • Tension between methodologically strong methods and their cognitive burden [18, 48]

 • Risk of neglecting of patient heterogeneity in PP studies [40, 52, 58]

 • Elicited PP are constructed and shaped by how information is presented [62]

 • Elicited PP are influenced by external factors [62]

 • Heuristics, inert or flexible preferences and measurement errors [19, 24, 27, 38, 48]

 • Challenge of communicating the quantitative health information to patients [14]

 • Innumeracy of the participants [38, 43]

 • Respondents not taking time to complete the survey of the PP study [35]

 • Lack of understanding among respondents [35]

 • Question framing in preference surveys [55]

 • Difficulty of balancing between understandability and accuracy of questions [55]

 • Ensuring representativeness of the sample [27, 50, 55]

3. Concerns specifically related to PP in BRA/MA and HTA/reimbursement

 • Lack of clarity about:

  ○ How PP will be used and reviewed by decision-makers [19, 24, 38]

  ○ How to submit PP for BRA/MA and HTA/reimbursement [24, 53]

  ○ Standards for measuring PP for informing BRA/MA and HTA/reimbursement [24, 72]

4. Concerns specifically related to PP in HTA/reimbursement

 • Lack of clarity about:

  ○ Measuring patient preferences versus public preferences [54, 59, 62]

  ○ Measuring PP for health aspects or also for non-health aspects [1]

  ○ Incorporating PP in economic evaluations or not [1]

  ○ Using quantitative and/or qualitative PP in reimbursement decisions [1, 59]

  ○ Where and how to incorporate PP in current procedures [1, 18, 62]

  ○ How to align PP with the traditional QALY calculation [62]

  ○ How to conduct a systematic review on PP studies for informing HTA [60]

  ○ What weight PP should receive versus other decision criteria [1, 62]

 • Current recommendation of HTA agencies (e.g. the UK, the Netherlands) to use generic measures, whereas PP elicited via PP studies are often condition-specific [59]

 • Current use of cost-utility analysis, which does not require quantitative PP beyond health state utilities [59]

 • Low generalizability of PP study results when characteristics of healthcare system are being valued as these characteristics are often system-, country- or culture-specific [55, 62]

 • Time, funding and staff required for incorporating PP in HTA/reimbursement [1]