The evolving role of patient preference studies in health-care decision-making, from clinical drug development to clinical care management

ABSTRACT

Introduction: There is a growing trend of using patient preference studies to help incorporate the patient perspective into clinical drug development, care management, and health-care decision-making. Collecting and interpreting patient preference data is integral to multi-stakeholder engagement, patient-centric drug development, and clinical care management. Operationally, challenges exist in understanding ‘when’ and ‘how’ to embark on patient preference studies. This review will provide a brief overview of stated-preference methods, discuss applications throughout the clinical drug development and care management, and highlight how preference studies serve as a powerful tool for quantifying patient experiences for better outcomes.

Areas covered: We present case studies to complement the different applications of stated-preference methods in clinical drug development and care management. We discuss the applications of preference data to help inform evidence-based patient advocacy, clinical development strategy, operational feasibility, regulator benefit-risk assessments, health technology assessments, and clinical decision-making.

Expert commentary: Patient preference studies can serve as a powerful tool to engage patients and their communities as well as quantify the patient voice across different stages of clinical drug development and care management to support patient-centric health-care decision-making. It is expected that the application of these strategies will quickly advance in the coming years.

KEYWORDS:

• Stated-preference methods
• patient-centered
• patient preferences
• benefit-risk assessment
• health technology assessment
• drug development

Article highlights

• Stated-preference studies provide robust, quantitative data about the patient perspective from a large sample of patients. When combined with qualitative methods, stated-preference studies can provide unique insights into the patient perspective and effectively capture and articulate the broader patient voice and experience.

• Patient preference data can support patient-centric decision-making by informing evidence-based patient advocacy, clinical development strategy and operational feasibility, benefit-risk assessments, health technology assessments, and clinical decision-making.

• A wide variety of stakeholders – patients and caregivers, academic researchers, industry researchers, clinicians, health economists, health services researchers and policymakers – can conduct patient preference studies, and studies generally benefit from multi-stakeholder engagement.

• The direct involvement and leadership of patients and caregivers in the patient preference field has been invaluable and should continue for patient preference methods to remain a viable option to capture meaningful data on the patient experience.

• Despite advancements in patient preference initiatives, it is not always clear how and when to implement patient preferences studies across the stages of clinical drug development and in clinical care management. Open and continued dialogue and publication efforts surrounding the role of preference studies in health-care decision-making are critical for the continued evolution of the patient preference field.

Acknowledgments

The authors would like to thank Vivien Ong, an employee of ICON Clinical Research Limited, for her editorial contributions to the manuscript. We also thank Andreas Pleil, a retired Pfizer Inc. employee, for his conceptual contribution to this manuscript.

Declaration of interest

YJackson, J Loftus, K Beaverson, K Betteridge and M Lundie are employees of Pfizer. E Flood, S Rhoten, and E Janssen are employees of ICON Clinical Research Limited who were paid consultants to Pfizer in connection with the development of this manuscript. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Reviewer Disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Supplementary material

Supplemental data can be accessed here.

Additional information

Funding

This paper was sponsored by Pfizer.