Bringing the patient’s perspectives forward in drug development and health-care evaluation

ABSTRACT

Introduction

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.

Areas covered

This paper discusses some 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.

Expert opinion

The development and use of PROMs situates them at the vertex of two very different trends in medicine: patient-centered care and standardization. Indeed, the application of PROMs – which pull in the direction of standardization – 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.

KEYWORDS:

• Clinical trials
• drug evaluation
• healthcare evaluation
• narrative evidence
• patient perspective
• patient-reported outcomes

Article highlights

• There is the need to extend the range of quantitative evidence resulting from the application of PROMs to the inclusion of narrative evidence

• The development and use of PROMs situates them at the vertex of two very different trends in medicine: patient-centered care and standardization

• Standardization calls for the nomothetic approach, which suggests that it is possible to measure behavior and experience independent of context

• This arguably results in the elimination of the features that are the hallmarks of patient-centered care

• Patient narratives contain valuable information about the patient experience that numerical ratings from PROMs do not capture and which can enhance patient-centered care

• Collection of narrative data within clinical trials through in depth interviews is rare, but are gathering momentum as a way to provide insights, which can augment and enrich quantitative findings

Declaration of interest

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership, or options, expert testimony, grants or patents received or pending, or royalties.

Reviewer disclosures

Peer reviewers in this manuscript have no relevant financial or other relationships to disclose.

Notes

1. The nomothetic approach compares individuals in terms of traits or dimensions common to everyone.

2. See Meadows [2] for a more detailed discussion of the limitations of the standardized patient-reported outcome measure.

3. One of the features of positivism is precisely its postulate that scientific knowledge is the paradigm of valid knowledge, a postulate that indeed is never proved nor intended to be proved [19].

4. Narrative is the phenomenological form in which patients experience illness is given structure. There are a number of methods for acquiring narrative data which include conversations, observation, autobiographies, transcripts of in-depth interviews, focus groups, diaries, letters etc [2].

5. https://www.ema.europa.eu/en/documents/product-information/xermelo-epar-product-information_en.pdf

Additional information

Funding

This paper was not funded.