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Original Research ARTICLE Provisionally accepted The full-text will be published soon. Notify me

Front. Pharmacol. | doi: 10.3389/fphar.2019.01009

Factors and situations affecting the value of patient preference studies: semi-structured interviews in Europe and the US

 Chiara Whichello1*,  Eline van Overbeeke2,  Rosanne Janssens2, Karin Schölin Bywall3, Selena Russo4, Jorien Veldwijk1, Irina Cleemput5,  Juhaeri Juhaeri6, Bennett Levitan7, Jürgen Kübler8, Meredith Smith9, Richard Hermann10, Matthias Englbrecht11, Axel Hueber11, Alina Comaneuscu12, Sarah Harding13, Steven Simoens2,  Isabelle Huys2 and Esther W. de Bekker-Grob1
  • 1Erasmus University Rotterdam, Netherlands
  • 2KU Leuven, Belgium
  • 3Uppsala University, Sweden
  • 4Istituto Europeo di Oncologia s.r.l., Italy
  • 5Belgian Health Care Knowledge Centre, Belgium
  • 6Sanofi (United States), United States
  • 7Janssen Research and Development, United States
  • 8CSL Behring (Germany), Germany
  • 9Amgen (United States), United States
  • 10AstraZeneca (United States), United States
  • 11Department of Internal Medicine 3, Rheumatology and Immunology, University Hospital Erlangen, Germany
  • 12Other, Romania
  • 13Takeda (United Kingdom), United Kingdom

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 organisation (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.

Keywords: Patient Preference, drugs, Drug life cycle, Medical products, benefit-risk assessment, medical devices, Patient perception, Decision - making, Health technolgy assessment, market authorisation, patient preference (MeSH)

Received: 14 May 2019; Accepted: 08 Aug 2019.

Copyright: © 2019 Whichello, van Overbeeke, Janssens, Schölin Bywall, Russo, Veldwijk, Cleemput, Juhaeri, Levitan, Kübler, Smith, Hermann, Englbrecht, Hueber, Comaneuscu, Harding, Simoens, Huys and de Bekker-Grob. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.

* Correspondence: Mx. Chiara Whichello, Erasmus University Rotterdam, Rotterdam, Netherlands, whichello@eshpm.eur.nl