Factors and Situations Affecting the Value of Patient Preference Studies: Semi-Structured Interviews in Europe and the US

Abstract

Objectives: Patient preference information (PPI) is gaining recognition among the pharmaceutical industry, regulatory authorities, and health technology assessment (HTA) bodies/payers for use in assessments and decision-making along the medical product lifecycle (MPLC). This study aimed to identify factors and situations that influence the value of patient preference studies (PPS) in decision-making along the MPLC according to different stakeholders. Methods: Semi-structured interviews (n = 143) were conducted with six different stakeholder groups (physicians, academics, industry representatives, regulators, HTA/payer representatives, and a combined group of patients, caregivers, and patient representatives) from seven European countries (the United Kingdom, Sweden, Italy, Romania, Germany, France, and the Netherlands) and the United States. Framework analysis was performed using NVivo 11 software. Results: Fifteen factors affecting the value of PPS in the MPLC were identified. These are related to: study organization (expertise, financial resources, study duration, ethics and good practices, patient centeredness), study design (examining patient and/or other preferences, ensuring representativeness, matching method to research question, matching method to MPLC stage, validity and reliability, cognitive burden, patient education, attribute development), and study conduct (patients’ ability/willingness to participate and preference heterogeneity). Three types of situations affecting the use of PPS results were identified (stakeholder acceptance, market situations, and clinical situations). Conclusion: The factors and situation types affecting the value of PPS, as identified in this study, need to be considered when designing and conducting PPS in order to promote the integration of PPI into decision-making along the MPLC.

Use of Patient Preferences in Health Technology Assessment: Perspectives of Canadian, Belgian and German HTA Representatives

“…The design of the focus group guide was informed by research conducted previously within the PREFER project [19,[22][23][24][25] and the study of Huls et al [27]. The interpretation of results was validated by participants.…”

“…This was also highlighted by the Patient Preferences in Benefit-Risk Assessments during the Drug Life Cycle (PREFER) project that aims "to strengthen patient-centric decision-making throughout the life cycle of medicinal treatments by developing expert and evidencebased recommendations on how patient preferences should be assessed and inform decision-making" [21]. While two literature reviews, 143 semi-structured interviews, and eight focus groups were previously conducted within PREFER on the design, conduct, and use of PPS [19,[22][23][24][25], some questions regarding the integration of PP in HTA and differences between healthcare systems remained unanswered.…”

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

“…In our previous literature review and interview papers on factors to consider in PPS design and conduct (van Overbeeke et al, 2019; Whichello et al, 2019), we identified 15 main factors that can influence the value of PPS and can occur along design and conduct of the studies: expertise, financial resources, study duration, ethics and good practices, patient centeredness, examining patient and/or other preferences, ensuring representativeness, matching method to research question, matching method to MPLC stage, validity and reliability of the method, cognitive burden, patient education, attribute development, patients' ability/willingness to participate, and preference heterogeneity. The focus group results confirm the necessity of different expertise, as also described by Wolka et al (van Til and Ijzerman, 2014), the Medical Device Innovation Consortium (MDIC) report (MDIC, 2015), van Til et al (2014), Selig (2016), and Ho et al (2016).…”

“…Two literature reviews and 143 interviews with healthcare stakeholders in Europe and the US were conducted ( Figure 1 ) to identify stakeholders' needs, factors influencing the value of PPS for decision-making, and potential applications of patient preferences along the MPLC. Methods and results of literature reviews have been published elsewhere (Janssens et al, 2019; van Overbeeke et al, 2019), as well as those of the interviews (Janssens et al, 2019; Whichello et al, 2019). Several topics remained unanswered and group discussions were deemed necessary to provide more insight and to give further direction on the potential implementation of patient preferences in the MPLC.…”

“…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.…”