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One Programme, Four Stakeholders: An Overview of the Utilisation of Patient-Reported Outcomes in Intervention Development to Meet the Needs of Regulators, Payers, Healthcare Professionals and Patients

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Abstract

For a new intervention to achieve commercial success, regulators (to provide marketing authorisation), payers (for reimbursement and formulary placement), healthcare professionals (HCPs; to prescribe) and patients (to adhere and persist) must all consider the intervention to be valuable. These stakeholders are increasingly using the patient’s perspective on a condition and its treatment—measured through patient-reported outcome (PRO) instruments—to define the value of a treatment. However, there is common confusion about the most appropriate way to incorporate the patient’s perspective into the clinical development of pharmaceutical interventions in a way that will resonate with these four key stakeholder groups. This article briefly summarises the perspectives of regulators, payers, HCPs and patients regarding PRO endpoints, and examines how a robust, comprehensive and systematic PRO endpoint strategy can be developed to meet the needs of all stakeholders in a single development programme. Such an endpoint strategy may include PRO assessments of first-order signs and symptoms using a disease-specific measure, health-related quality of life using both a disease-specific measure and generic utility measure, and general quality of life. Other PRO endpoints (e.g. preference/satisfaction, ease of use, HCP contact time, absenteeism) may be useful with some stakeholders to provide further differentiation between interventions.

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Acknowledgments

The authors would like to thank Dr. Lee Bowman for detailed review and guidance in the early stages of this work. No funding was received for the preparation of this article.

Conflict of interest

Matthew Reaney, Peter Black and Chad Gwaltney are employees of ERT, which provides services related to the development and implementation of patient-reported outcomes in clinical research. Elizabeth Bush, Kate van Brunt and Bradley Curtis are employees of Eli Lilly and Company, which manufactures pharmaceutical products. Chad Gwaltney, Elizabeth Bush and Bradley Curtis are also stockholders in the respective companies they are employed by.

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Authors and Affiliations

  1. ERT, Peterborough Business Park, Lynch Wood, Peterborough, PE2 6FZ, UK

    Matthew Reaney

  2. Eli Lilly and Company, Indianapolis, IN, USA

    Elizabeth N. Bush & Bradley Curtis

  3. ERT, Pittsburgh, PA, USA

    Peter Black & Chad Gwaltney

  4. Eli Lilly and Company, Windlesham, Surrey, UK

    Kate van Brunt

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  1. Matthew Reaney
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Correspondence to Matthew Reaney.

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Reaney, M., Bush, E.N., Curtis, B. et al. One Programme, Four Stakeholders: An Overview of the Utilisation of Patient-Reported Outcomes in Intervention Development to Meet the Needs of Regulators, Payers, Healthcare Professionals and Patients. Pharm Med 29, 69–78 (2015). https://doi.org/10.1007/s40290-015-0088-4

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