Table 5.
Requirements related to PP in the MPLC
| 1. General requirements | |
| • Recognition of the value of PP among stakeholders [24, 25, 39, 51, 59, 64] | |
| • Consensus on the role of PP in decision-making [1, 62] | |
| • More familiarity among stakeholders with PP studies [19, 34, 48, 66, 70, 72] | |
| • More educated researchers in preference research [53] | |
| • Resources to evaluate PP [1, 48] | |
| • Taxonomic work for PP research [1, 60] | |
| • Guidance on: | |
| ○ When during development to measure PP [1, 34, 51] | |
| ○ Which preference method to use in which circumstance [1, 40, 44, 56, 72] | |
| ○ Whose preferences to measure (e.g. required disease experience) [1, 19, 44] | |
| ○ Sample size [37] | |
| ○ Good research practice and quality criteria for PP studies [1, 19, 38, 43, 44, 62] | |
| ○ How to ensure validity of a PP study [75] | |
| ○ How to report about PP studies [44] | |
| • Further research to: | |
| ○ Validate and test preference methods [37, 44, 46, 66, 76] | |
| ○ Identify methods for integrating clinical evidence in PP study analysis [50, 56] | |
| ○ Investigate methodological issues (e.g. hindsight bias) [62] | |
| ○ Compare the performance of different methods in a given situation [37] | |
| ○ Determine impact of changing list of attributes with any given method [37] | |
| ○ Explore statistical methods to detect preference heterogeneity [77] | |
| ○ Guide the development of newer methods for eliciting PP [76] | |
| ○ Assess comprehension differences by participants between methods [76] | |
| ○ Assess impact of the level of previous education on PP [33] | |
| ○ Quantify the effect of the attribute descriptions on elicited PP [78] | |
| 2. Operational requirements | |
| • Requirements related to timing of PP study: | |
| ○ Decision depends on level of information of the treatments’ key risks [19] | |
| ○ Timing needs to be decided by sponsor [19] | |
| ○ During marketing phase to assess long-term side effects and burden [1] | |
| • Requirements related to dealing with PP study results: | |
| ○ Stakeholders should be prepared for disappointing PP study results [24] | |
| ○ PP study results should be provided to patient community and public [24] | |
| ○ Presentation of PP study results should be tailored to the audience [79] | |
| ○ PP study results should be described transparently [56, 75] | |
| 3. Quality requirements | |
| • General requirements regarding design, set-up and conduct of PP studies: | |
| ○ Selected research question should be answerable with PP study [75] | |
| ○ Study objectivity throughout PP study [24] | |
| ○ Independent design as design can influence analysis outcomes [25] | |
| ○ Extensive and forward planning [19, 27, 48] | |
| ○ Determination of objectives and attributes before design [24] | |
| ○ Design based on prior literature and preference information [19] | |
| ○ Clear definition of the patient sample and characteristics [19, 24, 49] | |
| ○ Training partners on methodology, objectives and expectations of study [79] | |
| ○ Good communication and documentation of changes to study plans [79] | |
| ○ Methodological expertise when designing and executing a PP study [24, 70] | |
| ○ Multi-stakeholder partnerships (patients, academics, industry) [24, 37, 79] | |
| ○ Interaction between decision-makers and industry in design [14, 19, 24] | |
| ○ Involvement of patients, caregivers and patient organizations [24, 42, 49, 51] | |
| ○ Application of ‘good science’ principles [1, 19, 24, 51] | |
| ○ Consideration of patient heterogeneity and cognitive burden [14, 40, 58, 75] | |
| ○ Consideration of internal and external validity [75] | |
| ○ Administration of survey by trained researchers [14] | |
| ○ Provision of tutorial for participants if self-administered survey is used [14] | |
| ○ Training of participants in elicitation tasks [40] | |
| ○ Ensuring participants’ understanding of aim and how results will be used [40] | |
| ○ Consideration of low level of health numeracy in general population [43] | |
| • Sample requirements: | |
| ○ Sample should be heterogeneous (large samples, setting quotas) [19, 49, 75] | |
| ○ Sample should be representative of population of interest [14, 19] | |
| ○ If not possible to elicit from patients, include proxies [19, 34] | |
| ○ Sample ideally is clinical trial population [71] | |
| ○ Sample ideally is broader population than clinical trial population [41] | |
| ○ Patient should be the focus, not health care professional [14] | |
| ○ Sample should be representative of affected patients [56] | |
| ○ Sample should be representative of target population [75] | |
| ○ Sample that can yield reliable results should be drawn [24] | |
| ○ PP should come from the same population as data of effectiveness [1] | |
| ○ Both patients in remission as well as patients in recovery should be included [50] | |
| ○ Sampling should consider sociodemographic and disease characteristics [50, 61] | |
| • Sample size requirements: | |
| ○ Adequate size so that results are generalizable to population of interest [14] | |
| ○ Sufficient size to generate acceptably robust results [24] | |
| ○ If subgroups: sufficient number in each subgroup [14] | |
| • PP results requirements: | |
| ○ Type of PP (qualitative vs quantitative) depends on stage and decision-making context of MPLC [1, 14, 16, 19, 60] | |
| ○ Type of PP should be determined by research question [19] | |
| ○ Clinical data should be collected and used to augment PP data [42, 43] | |
| ○ Patient’s willingness and unwillingness to accept risks should be measured [14] | |
| • Preference method requirements: | |
| ○ Method should be selected based on factors [1, 19, 40, 44, 76, 80] | |
| ○ Method should adhere to utility theory [18, 76] | |
| ○ Method should account for patient-relevant attributes/outcome measures [18] | |
| ○ Methods should be easy and simple for patients to understand [18] | |
| • Requirements regarding attribute selection: | |
| ○ Research question should guide attribute and level selection [75] | |
| ○ Attributes should be broader than clinical attributes to elicit meaningful trade-offs [41] | |
| ○ Attributes should be patient-centered to investigate meaningful attributes [49] | |
| ○ Attributes should come from existing clinical trials [50, 81] | |
| ○ Selection by literature, qualitative study, asking group of medical experts or decision-makers [19] | |
| ○ Patient representatives, patients and experts should inform selection [50, 62] | |
| ○ Attributes should not overlap [30, 35, 50] | |
| • Requirements regarding survey instrument: | |
| ○ Survey should be developed with input from multiple stakeholders [24] | |
| ○ Survey should be piloted [24, 40] | |
| ○ Survey should include screening questions, informed consent provisions, background information, training and definitions, testing, survey questions, follow-up survey questions [24] | |
| ○ Benefit descriptions and effectiveness measures should be carefully defined [78] | |
| ○ Patients should understand objective of the elicitation tasks and how data will be used [40] | |
| ○ Questions have to be asked in an open and understandable way [18, 56, 75] | |
| ○ For choice-based preference measures, options should: | |
| ■ Be clearly described [56] | |
| ■ Have realistic advantages and disadvantages [56] | |
| ■ Be communicated to patients together with their characteristics [80] | |
| • Requirements regarding the analysis: | |
| ○ Interpretation of results should consider the mode of sampling [68] | |
| ○ Interpretation of study results should be validated with patients [40] | |
| ○ Results should be considered with preferences from other stakeholders (clinicians, decision-makers) [68] | |
| ○ Appropriate stakeholders should interpret analysis [79] | |
| ○ Sources of uncertainty should be reported through confidence interval and/or standard error [14] | |
| ○ Written agreements about intellectual property and data use are needed [24] |
Requirements related to using PP in the MPLC grouped according to their type and nature: general requirements, operational requirements and quality requirements (bold and underlined font). PP patient preferences, MPLC medical product life cycle