Design, Conduct, and Use of Patient Preference Studies in the Medical Product Life Cycle: A Multi-Method Study

Abstract

Objectives: To investigate stakeholder perspectives on how patient preference studies (PPS) should be designed and conducted to allow for inclusion of patient preferences in decision-making along the medical product life cycle (MPLC), and how patient preferences can be used in such decision-making. Methods: Two literature reviews and semi-structured interviews (n = 143) with healthcare stakeholders in Europe and the US were conducted; results of these informed the design of focus group guides. Eight focus groups were conducted with European patients, industry representatives and regulators, and with US regulators and European/Canadian health technology assessment (HTA) representatives. Focus groups were analyzed thematically using NVivo. Results: Stakeholder perspectives on how PPS should be designed and conducted were as follows: 1) study design should be informed by the research questions and patient population; 2) preferred treatment attributes and levels, as well as trade-offs among attributes and levels should be investigated; 3) the patient sample and method should match the MPLC phase; 4) different stakeholders should collaborate; and 5) results from PPS should be shared with relevant stakeholders. The value of patient preferences in decision-making was found to increase with the level of patient preference sensitivity of decisions on medical products. Stakeholders mentioned that patient preferences are hardly used in current decision-making. Potential applications for patient preferences across industry, regulatory and HTA processes were identified. Four applications seemed most promising for systematic integration of patient preferences: 1) benefit-risk assessment by industry and regulators at the marketing-authorization phase; 2) assessment of major contribution to patient care by European regulators; 3) cost-effectiveness analysis; and 4) multi criteria decision analysis in HTA. Conclusions: The value of patient preferences for decision-making depends on the level of collaboration across stakeholders; the match between the research question, MPLC phase, sample, and preference method used in PPS; and the sensitivity of the decision regarding a medical product to patient preferences. Promising applications for patient preferences should be further explored with stakeholders to optimize their inclusion in decision-making.

Keywords: decision-making; health technology assessment; marketing authorization; medical products; patient preferences.

Figure 1

Methods used in the multi-method design. First, two literature reviews were performed to identify 1) factors and situations that influence the value of patient preference studies (PPS) in decision-making along the MPLC (van Overbeeke et al., 2019), and 2) the potential roles, expectations, concerns and requirements associated with using patient preferences (Janssens et al., 2019). Second, interviews (n = 143) with different healthcare stakeholders were held to address the same research questions (Whichello et al., 2019; Janssens et al., 2019). Lastly, focus groups (n = 8) were conducted to discuss topics related to the design, conduct and use of PPS for which opinions of interviewees differed or deeper understanding was needed. Abbreviations: HTA, Health Technology Assessment; N, number of interviews/focus groups; n, number of participants.

Figure 2

Graphical summary of the main results from the different steps in the multi-method study. Main learnings to consider in the design and conduct of patient preference studies are shown in orange. Barriers to the use of patient preferences in the medical product life cycle are indicated in the red box. In green, the situations sensitive to patient preferences are highlighted as facilitators toward to use of patient preferences. In blue, the applications of patient preferences are indicated throughout the medical product life cycle. Abbreviations: HTA, health technology assessment; MCDA, multi criteria decision analysis; MPLC, medical product life cycle; PPS, patient preference studies.