RESUMO
BACKGROUND: The biopharmaceutical industry is challenged with efficiently delivering medicines that patients truly value. This can be addressed by engaging patients and caregivers throughout a medicine's life cycle, ensuring that products meet the needs and expectations of those who take them. While isolated best practice examples of patient engagement exist, they remain relatively ad hoc and not fully embedded within Research & Development (R&D) practices. To encourage more patient engagement, the 'impact' of patient engagement projects (PEP) must be objectively measured and demonstrated. Some frameworks have been proposed; however, there is no evidence of widespread adoption, nor have patients' perspectives been robustly explored. The objective of this qualitative study was therefore to understand patients' perspectives of impact measurement that can be systematically applied within a biopharmaceutical company. METHODS: Semi-structured interviews were conducted with 13 patient organisation (PO) representatives exploring their experiences of engagement and reflections on 23 candidate patient engagement impact measures categorised into five groups: Medicines R&D Priorities; Clinical Trial Design; Regulatory & Market Access Submissions; Product Support & Information; and Disease Support & Information. Thematic analysis was undertaken and impact measures revised in line with interview participant feedback. Emerging themes and revisions to impact measures were validated at a joint workshop with 4 patient advisors representing 4 POs. RESULTS: The study revealed that PO representatives feel a deep sense of accomplishment and ownership when collaborating on PEPs with biopharmaceutical companies. They largely conceptualise 'impact' as positive, tangible and useful outcomes. The revisions made to the pre-defined patient engagement impact measures fell into three broad categories: (1) a requirement for greater context; (2) capturing the nature of patient influence; and (3) terminology changes. The greatest number of revisions concerned 'requiring greater context', for example, including additional descriptions, patient quotes, and satisfaction. CONCLUSIONS: This study sheds light on how patient advocates view 'impact'. Typically this means delivering 'value' important for them. Therefore, the authors of this paper created the term 'value-impact' to comprehensively characterise this conceptualisation, and propose a value-impact measurement plan, incorporating longitudinal data. Through this understanding and in light of other recently published work, wide-scale adoption and implementation of the measurement of value-impact across the biopharmaceutical industry can be realised.
Developing medicines involves multiple stages, all essential for pharmaceutical companies. However, this approach is becoming increasingly costly and patients are rarely involved in defining treatment outcomes. Patient insight is important for everyone involved in medicinal development and required by authorities reviewing new treatments. To identify best practices, the difference ('impact') made by engaging with patients must be measured continuously throughout the treatment development process. Possible impact measures have been proposed (for example, the PARADIGM initiative), but problems remain.This study aimed to work with patients and adapt standard measuring approaches that can be used to provide ongoing insight into the impact of patient engagement activities for companies and patients. Researchers interviewed 13 patient organisation (PO) representatives to explore their experiences of impact measures. Thematic analysis was conducted, and data used to adapt 23 potential impact measures in collaboration with patients.This study identified that PO representatives view impact as being positive, tangible, and useful outcomes, achieving a deep sense of accomplishment and ownership for those patients and representatives involved. The authors created the term 'value-impact' to describe how patients perceive such achievements. Furthermore, measures should be adapted to: 1) include greater context such as descriptions, patients quotes and satisfaction; 2) capture patient influence; and 3) use language that is understandable for all parties. Examples and suggestions for engagement at different stages and revised impact measures are presented in this report. It is our hope that this study will encourage more frequent and effective use of patient engagement strategies as medicines are developed.