Developing Patient-Reported and Relevant Outcome Measures

This chapter will examine good practice guidance for patient-centred approaches towards PROM development. During the last decade, we have witnessed a paradigm shift in how outcomes are measured from a more clinical, physician-oriented perspective to a more patient-focused perspective, which has led to the emergence of the notion of patient-reported outcome (PRO). The concept of PRO seeks to understand how patients feel, function and live their lives in relation to health challenges and associated healthcare and is more encompassing than earlier terms, such as patient global assessment, health status, quality of life or symptom checklists. In this chapter, we argue that well-developed questionnaires, or PRO measures (PROMs), which reflect patients’ perspectives, have the potential to provide valuable patient-based evidence in HTA. PROM development should engage with patients as participants (US Food and Drug 2009) and increasingly as research partners (Staniszewska et al. 2012; de Wit et al. 2013; Chap. 8) through all stages of development. This promotes patients as the determinants of the key constructs underpinning the PROM. This approach will support a transparent and auditable approach towards capturing patients’ contributions to the measurement of relevant outcomes, thereby enhancing the face and content validity, relevance and acceptability of measures. In this chapter, we describe eight key stages in PROM development and reflect on how patients can participate in this process.