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Design, conduct, and use of patient preference studies in the medical product life cycle: A multi-method study

Lookup NU author(s): Dr Nikoletta Nikolenko

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This work is licensed under a Creative Commons Attribution 4.0 International License (CC BY 4.0).


Abstract

© 2019 Georg Thieme Verlag. All rights reserved.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.


Publication metadata

Author(s): Van Overbeeke E, Janssens R, Whichello C, Bywall KS, Sharpe J, Nikolenko N, Phillips BS, Guiddi P, Pravettoni G, Vergani L, Marton G, Cleemput I, Simoens S, Kubler J, Juhaeri J, Levitan B, De Bekker-Grob EW, Veldwijk J, Huys I

Publication type: Article

Publication status: Published

Journal: Frontiers in Pharmacology

Year: 2019

Volume: 10

Online publication date: 03/12/2019

Acceptance date: 31/10/2019

Date deposited: 10/01/2020

ISSN (electronic): 1663-9812

Publisher: Frontiers Research Foundation

URL: https://doi.org/10.3389/fphar.2019.01395

DOI: 10.3389/fphar.2019.01395


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