Methodological Questions Regarding the Elicitation of Patient Preferences and Empirical Evidence to Help Address Them

Patient preferences are qualitative or quantitative assessments of the relative desirability or acceptability to patients of specified alternatives or choices among outcomes or other attributes that differ among alternative health interventions. In simpler terms, they are assessments of what patients want or would accept in their care. This PhD thesis presents methodological questions regarding the assessment of patient preferences along with research addressing these questions. The aim of this thesis is to help stakeholders who would potentially use this information to better understand and conduct patient preference studies and support the use of patient preferences in medical product decision-making.

Chapter 1 reports on a survey conducted with members of the PREFER consortium. In this survey the stakeholders ranked a list of methodological questions regarding their priority for research to better understand and conduct patient preference studies and support their use in decision making. From this prioritized list, the following research topics are addressed in this thesis. First, the generalizability of preferences from one specific population in a disease to different populations in that or related diseases is addressed in Chapters 2 and 3. Chapter 2 compares the preferences of Diabetes patients in the Netherlands and Poland for glucose monitoring technology. Chapter 3 presents a study identifying common opinions about the most important areas of unmet medical needs in patients with two types of neuro-musculoskeletal diseases, myotonic dystrophy type 1 and mitochondrial disorders and their caregivers. Second, the impact of attribute framing on preferences is addressed in Chapter 4 using a case study in which participants were asked to complete a discrete choice experiment which varied in the way that the attributes were framed (positively, negatively, or both) when presented to participants. Third, how the results of simpler/cheaper preference elicitation methods differ from more complex/expensive methods is assessed in two case studies using Discrete Choice Experiments and Swing Weighting. In Chapter 5 these methods are compared in a case study assessing patient preferences for glucose monitoring for self-management of diabetes, and in Chapter 6 they are compared in a case study measuring treatment preferences of patients with non-small cell lung cancer. Fourth, how preferences results differ when participants are presented with information in a video-based educational tool versus traditional text-based education is explored in Chapter 7 using a case study of diabetes patients. Finally, the initial aim of Chapter 1 is revisited in Chapter 8 which reports on a second survey (conducted 4 years after the first study), asking stakeholders in the research area of patient preference assessment what methodological topics and questions for future studies they think are important to answer to increase acceptance of preference methods and the use of their results by decision makers. This thesis concludes with a summary of the findings, a general discussion of the research conducted, and the implications for future patient preference studies.