AUTHOR=van Overbeeke Eline , Janssens Rosanne , Whichello Chiara , Schölin Bywall Karin , Sharpe Jenny , Nikolenko Nikoletta , Phillips Berkeley S. , Guiddi Paolo , Pravettoni Gabriella , Vergani Laura , Marton Giulia , Cleemput Irina , Simoens Steven , Kübler Jürgen , Juhaeri Juhaeri , Levitan Bennett , de Bekker-Grob Esther W. , Veldwijk Jorien , Huys Isabelle 

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

JOURNAL=Frontiers in Pharmacology

VOLUME=10

YEAR=2019

URL=https://www.frontiersin.org/journals/pharmacology/articles/10.3389/fphar.2019.01395

DOI=10.3389/fphar.2019.01395

ISSN=1663-9812

ABSTRACT=<p><bold>Objectives:</bold> 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.</p><p><bold>Methods:</bold> 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.</p><p><bold>Results:</bold> 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.</p><p><bold>Conclusions:</bold> 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.</p>