Bringing the patient’s perspectives forward in drug development and healthcare evaluation

By Keith Meadows and Matt Reaney

For many years psychologists and other social scientists have been pushing for the individual patient’s perspective – priorities, needs, feelings and functioning – to be incorporated into drug development. This is usually achieved through the use of patient-reported outcome measures (PROMs) in clinical trials. The development and use of PROMs situates them at the vertex of two very different trends in medicine: patient-centred care and standardization. Indeed the application of PROMs – which pull in the direction of standardisation – results in a narrow conception of evidence by overriding the subjectivity of individual experiences, beliefs and judgments. Without additional context, PROM data cannot easily support individual patient-level care. When collected systematically and with an interpretive phenomenological approach, narrative data can contain valuable information about the patient experience that numerical ratings from PRO measures do not capture.

Here we would like to discuss some of the key issues in the use of PROM data as the sole method of generating information about the patient’s perspective and outlines the relevance of narrative evidence to enhance understanding and interpretation of PROM data.

A brief overview of the development and standardisation of PROMs

Development of a PROM is a sophisticated process involving the generation of individual items through a range of methods including literature reviews, qualitative in-depth interviews, focus groups, cognitive interviews and clinical observation. Once developed, the PROM is required to undergo extensive psychometric evaluation to establish its reliability and validity [1, 2]. Most trials now include at least one PROM to understand some aspect of how patients feel or function. In some areas, such as irritable bowel syndrome [3], PROMs are used as primary indicators of treatment benefit. The most common scenario for clinical trials is that all patients complete the same ‘static’ PROM. It is then straightforward to use an index of change from baseline (e.g., mean change from baseline in each treatment group; percent of patients in each treatment group meeting a threshold for reduction or worsening of symptoms) as the trial endpoint. However, this ‘static’ approach inherently assumes that all patients have the same experiences underlying the concept of interest.

A mixed-method approach

Having PROM data from clinical trials allows for treatments to be developed and approved in acknowledgement of the patient perspective. It also enhances the clinical consultation, offering data to healthcare teams to answer the common questions from patients, such as “how will it make me feel?” and “will I be able to do x?” We are not trying to discourage the collection of PROM data; far from it. However, PROM data alone cannot easily answer the question “why does it do that?”, or “how do people cope with it”. To answer this, a narrative approach is needed. The combination of qualitative and quantitative data in a “mixed methods” approach to understanding the patient perspective (priorities, needs, preferences, outcomes) and tailoring treatment to individuals is important in trials and in clinical practice [4]. I have put forward the case for a pragmatic epistemology based on a mixed methods research paradigm encompassing a hermeneutical approach and interpretive phenomenology with a focus of the use of narrative in the application of PROMs in the understanding of the experience and behaviour of living with an illness [2].  However, collection of narrative data within clinical trials through in-depth interviews is rare, but is gathering momentum as a way to provide insights which can augment and enrich quantitative findings [5, 6, 7]. Embedded interviews are typically conducted at study entry and/or exit.

Qualitative research can also, of course, be conducted outside of a trial setting. This is common to support an understanding of general experiences of disease  [8], to understand priorities and needs of people with disease [9], explore treatment or care satisfaction and reasons for non-adherence [10] and to explore patient preferences; in particular the idiosyncratic experiences and decisions of participants [10].

This is not the first to call for researchers to make better use of mixed methods research [12], but it is necessary to stress the importance of doing it carefully and not just for the sake of it; that is the core research questions or hypotheses drive the methodological approach and the questions posed in the quantitative and qualitative components of the research. The qualitative approach can be used to supplement or complement the quantitative data, or can be used to gather in depth information about things not captured in the quantitative data. The combination of this data to offer detailed insights and clear applicability is essential [

Conclusion

We would like to encourage researchers to engage in mixed methods research; to harness the combined power of qualitative and quantitative methodologies to understand patient expectations, perspectives and experiences of treatments, trial activities, and care processes. We are not the first to call for researchers to make better use of mixed methods research [12], but we would like to stress the importance of doing it carefully and not just for the sake of it; that is the core research question or hypotheses drive the methodological approach and the questions posed in the quantitative and qualitative components of the research. Read the full article Here.

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