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Beyond study participants: a framework for engaging patients in the selection or development of clinical outcome assessments for evaluating the benefits of treatment in medical product development

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Abstract

Background

Patients are participating more actively in health care decision-making with regard to their health, as well as in the broader realm of assessing the value of medical products and influencing decisions about their registration and reimbursement. There is an increasing trend to include patients’ perspectives throughout the stages of medical product development by broadening the traditional study-participant role to that of an active partner throughout the process. Including patients in the selection and development of clinical outcome assessments (COAs) to evaluate the benefit of treatment is particularly important. Still, despite widespread enthusiasm, there is substantial uncertainty regarding how and when to engage patients in this process.

Purpose

This manuscript proposes a methodological framework for engaging patients at varying levels in the selection and development of COAs for medical product development.

Framework

The framework builds on the Food and Drug Administration’s roadmap for patient-focused COA. Methods for engaging patients across each stage in this roadmap are summarized by levels of engagement. Opportunities and examples of patient engagement (PE) in the selection and/or development of COAs are summarized, together with best practices and practical considerations.

Conclusion

This paper offers a framework for understanding, planning, and implementing methods to advance PE in the selection and/or development of COAs for evaluating the benefit of medical products. The intent is to further this important discussion and enhance the process and outcome of PE in this context.

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Acknowledgements

H. Wilson, M. Anatchkova, A. Hareendran, K. Coyne, C. McHorney, and N.K. Leidy are employed by Evidera, a health care research firm that provides outcome consulting and other research services to pharmaceutical, device, government, and non-government organizations. K. Wyrwich was employed by Evidera at the time the manuscript was developed, but is now currently employed by Eli Lilly and Company. E. Dashiell-Aje was employed by Evidera during the time the manuscript was developed, but is now currently employed at the U.S. Food and Drug Administration (FDA). This publication reflects the views of the authors and should not be construed to represent the views, policies, or guidance of the FDA. This paper does not represent any new FDA guidance or policy.

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  1. Evidera, Bethesda, MD, USA

    Hilary Wilson, Ebony Dashiell-Aje, Milena Anatchkova, Karin Coyne, Asha Hareendran, Nancy Kline Leidy, Colleen A. McHorney & Kathy Wyrwich

  2. Evidera, Outcomes Research, 1417 Fourth Avenue, Suite 510, Seattle, WA, 98101, USA

    Hilary Wilson

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  1. Hilary Wilson
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Wilson, H., Dashiell-Aje, E., Anatchkova, M. et al. Beyond study participants: a framework for engaging patients in the selection or development of clinical outcome assessments for evaluating the benefits of treatment in medical product development. Qual Life Res 27, 5–16 (2018). https://doi.org/10.1007/s11136-017-1577-6

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