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Patient preferences are critical for the successful development, regulation, and reimbursement of medical products and creating patient centric decision-making within the medical product lifecycle (MPLC). This thesis identified multiple systemic challenges preventing the systematic integration of patient preference studies into the MPLC. Greater methodological clarity is needed by key stakeholders before the information can be successfully incorporated into decision-making. Patient preferences can be measured through a variety of different preference elicitation and exploration methods and decision-makers are eager to discover which methods would be the most acceptable for market authorisation submissions or HTA/payer reimbursement decisions. This thesis appraised several methods and conducted a head-to-head empirical study. There is no ‘gold-standard’ patient preference elicitation or exploration method, but some methods have more promising features, and are more likely to meet decision-makers needs across the MPLC, such as the discrete-choice experiment (DCE). The development of methodologically sound patient preference studies is an important field of scientific research that will benefit the lives of patients.

Background: Patient preference (PP)information is not effectively integrated in decision-making throughout the medical product lifecycle (MPLC), despite having the potential to improve patients’ healthcare options. A first step requires an understanding of existing processes and decision-points to know how to incorporate PP information in order to improve patient-centric decision-making. Objectives: The aims were to: 1) identify the decision-making processes and decision-points throughout the MPLC for industry, regulatory authorities, and reimbursement/HTA, and 2) determine which decisionpoints can potentially include PP information. Methods:Ascoping literature review was conducted usingfive scientific databases. Semi-structured interviews were conducted withrepresentatives fromsevenEuropeancountries andthe US,including industry (n = 24), regulatory authorities (n = 23), reimbursement/HTA (n = 23). Finally, validation meetings with key stakeholders (n = 11) were conducted. Results: Six critical decision-points were identified for industry decision-making, three for regulatory decision-making, and six for reimbursement/HTA decision-making. Stakeholder groups agreed that PP information is not systematically integrated, either as obligatory information or pre-set criteria, but would benefit all the listed decision-points in the future. Conclusion: Currently, PP information is not considered as obligatory information to submit for any of the MPLC decision-points. However, PP information is considered an important component by most stakeholders to inform future decision-making across the MPLC. The integration of PP information into 15 identified decision-points needs continued discussion and collaboration between stakeholders

Background: Incorporating patient preference (PP) information into decision-making has become increasingly important to many stakeholders. However, there is little guidance on which patient preference assessment methods, including preference exploration (qualitative) and elicitation (quantitative) methods, are most suitable for decision-making at different stages in the medical product lifecycle (MPLC). This study aimed to use an empirical approach to assess which attributes of PP assessment methods are most important, and to identify which methods are most suitable, for decision

BACKGROUND: Incorporating patient preference (PP) information into decision-making has become increasingly important to many stakeholders. However, there is little guidance on which patient preference assessment methods, including preference exploration (qualitative) and elicitation (quantitative) methods, are most suitable for decision-making at different stages in the medical product lifecycle (MPLC). This study aimed to use an empirical approach to assess which attributes of PP assessment methods are most important, and to identify which methods are most suitable, for decision-makers' needs during different stages in the MPLC. METHODS: A four-step cumulative approach was taken: 1) Identify important criteria to appraise methods through a Q-methodology exercise, 2) Determine numerical weights to ascertain the relative importance of each criterion through an analytical hierarchy process, 3) Assess the performance of 33 PP methods by applying these weights, consulting international health preference research experts and review of literature, and 4) Compare and rank the methods within taxonomy groups reflecting their similar techniques to identify the most promising methods. RESULTS: The Q-methodology exercise was completed by 54 stakeholders with PP study experience, and the analytical hierarchy process was completed by 85 stakeholders with PP study experience. Additionally, 17 health preference research experts were consulted to assess the performance of the PP methods. Thirteen promising preference exploration and elicitation methods were identified as likely to meet decision-makers' needs. Additionally, eight other methods that decision

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 diffe

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 representat

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 a

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

Background: Integrating patient preferences in Health Technology Assessment (HTA) is argued to improve uptake, adherence, and patient satisfaction. However, how to elicit and incorporate these preferences in HTA in a systematic and scientifically valid manner is subject to debate. Objective: This article provides a systematic review of the challenges to integrating patient preferences in HTA that have been raised in the literature about patient preferences in HTA. Methods: A systematic review of articles published between 2013 and 2017 addressing challenges to the integration of patient preferences in HTA was conducted in 7 databases. All issues with respect to the integration of patient preferences in HTA were extracted and divided into 5 categories: conceptual, normative, procedural, methodological, and practical issues. The issues were ranked according to how often they were mentioned. Results: Of 2147 retrieved articles, 67 were included in the analysis. Thirty-seven unique research issues were identified. In the majority of the articles, methodological issues were posed (82%), followed by procedural (73%), normative (51%), practical (24%), and conceptual (9%) issues. Frequently posed methodological issues concerned preference heterogeneity and choice of method. Common procedural issues concerned how to evaluate the impact of preference studies and their degree of being evidence based. Conclusions: This article provides an overview of issues with respect to the integration of patient preferences in HTA procedures. Most issues were of a methodological or procedural nature; yet, the large number of different issues points to the overall importance of further researching the different aspects concerned with patient preferences in HTA. Through its ranking of how many articles mention particular issues, this article proposes an implicit research agenda.

Objectives: Patient preference information (PPI) is gaining recognition among the pharmaceutical industry, regulatory authorities, and health techn

Background: Integrating patient preferences in Health Technology Assessment (HTA) is argued to improve uptake, adherence, and patient satisfaction. However, how to elicit and incorporate these preferences in HTA in a systematic and scientifically valid manner is subject to debate. Objective: This article provides a systematic review of the challenges to integrating patient preferences in HTA that have been raised in the literature about patient preferences in HTA. Methods: A systematic review of articles published between 2013 and 2017 addressing challenges to the integration of patient preferences in HTA was conducted in 7 databases. All issues with respect to the integration of patient preferences in HTA were extracted and divided into 5 categories: conceptual, norma

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.

Industry, regulators, health technology assessment (HTA) bodies, and payers are exploring the use of patient preferences in their decision-making processes. In general, experience in conducting and assessing patient preference studies is limited. Here, we performed a systematic literature search and review to identify factors and situations influencing the value of patient preference studies, as well as applications throughout the medical product lifecyle. Factors and situations identified in 113 publications related to the organization, design, and conduct of studies, and to communication and use of results. Although current use of patient preferences is limited, we identified possible applications in discovery, clinical development, marketing authorization, HTA, and postmarketing phases.