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.
Highlights
Patient preference information is defined by the United States, (US) Food and Drug Administration (FDA) as “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” (FDA, 2016)
Stakeholder perspectives on how patient preference studies (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 medical product life cycle (MPLC) phase; 4) different stakeholders should collaborate; and 5) results from PPS should be shared with relevant stakeholders
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
Summary
Patient preference information is defined by the United States, (US) Food and Drug Administration (FDA) as “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” (FDA, 2016). Preference exploration methods can be defined as “qualitative methods that collect descriptive data through participant or phenomenon observation, and examine the subjective experiences and decisions made by participants” (Soekhai et al, 2019). Preference elicitation methods can be defined as “quantitative methods collecting quantifiable data that can be reported through statistical inferences or analysis” (Soekhai et al, 2019). Examples of preference elicitation methods include discrete choice experiments (DCE), analytical hierarchy process (AHP), and standard gamble. While health preference research has existed for over 40 years, the use of PPS in decision-making along the lifecycles of drugs and medical devices (called the medical product lifecycle; MPLC) is limited but gaining attention (Craig et al, 2017)
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