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Choosing a patient-reported outcome measure

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Abstract

There has been much philosophical interest regarding the ‘hierarchy of evidence’ used to determine which study designs are of most value for reporting on questions of effectiveness, prognosis, and so on. There has been much less philosophical interest in the choice of outcome measures with which the results of, say, an RCT or a cohort study are presented. In this paper, we examine the FDA’s recently published guidelines for assessing the psychometric adequacy of patient-reported outcome measures. We focus on their recommendations for demonstrating content validity and also for how researchers should weigh up the sum of psychometric evidence when choosing these measures. We argue that questions regarding judgment and understanding meaning of these measures should play a more central role in determining their adequacy.

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Notes

  1. See, for instance, John Worrall [1], Maya J. Goldenberg [2], and Nancy Cartwright [3].

  2. The potential for new insight into a text does not suggest that just any interpretation of a text is acceptable. For Gadamer two further conditions delimit understanding, namely, that a text is understood as coherent and taken as something from which we might learn.

  3. This difference is not insignificant, but for our purposes here, it is what they share that distinguishes them.

  4. True criterion validation of PROMs is rarely possible owing to the absence of accepted gold standards, and we do not consider it further in this paper.

  5. Reliability was assessed in terms of internal consistency and test–retest reliability; validity was assessed in terms of content validity, criterion validity, construct validity (within scale analyses, convergent and discriminant, known groups), and other hypothesis testing.

  6. Although it is beyond the scope of this paper, it is worth noting that the suggestion that measures of study quality are a sound pointer to the presence of bias is controversial. For instance, there is an ongoing debate within the evidence-based medicine movement about the extent to which measures of the quality of studies of clinical effectiveness can discriminate between biased and nonbiased studies. One example of this is the controversy around the quality of randomized controlled trials of screening for breast cancer with mammography; see Freedman et al. [22]. Indeed, we might also see John Worrall’s philosophical work critiquing randomization as an extension of this debate insofar as he questions the importance of randomization for ensuring high quality studies; see [1].

  7. Alternative practices are used by, for example, health economists who would argue that PROMs designed for use in economic evaluation (e.g., the EQ-5D instrument) do not need to meet minimum psychometric standards, but must simply be ‘better’ than any alternative on offer [19].

  8. It is arguable that this construct validity problem is caused by poor content validity in the VF-14 for the range of the construct in question. This example is illustrative of the iterative nature of construct and content validation. The potential flaw in content validity only emerges after construct validation. Moreover, the problem with the VF-14 can only be solved after further rounds of content and construct validation.

  9. Our suggestion that the choice of a PROM may need to be determined on a case by case basis is not meant to suggest that expert judgment is infallible, but rather that attempts to codify such judgments does not overcome problems of fallibility.

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Authors and Affiliations

  1. Department of Philosophy, University of South Carolina, Columbia, SC, 29208, USA

    Leah M. McClimans

  2. Department of Epidemiology and Public Health, Second Floor, Brookfield Health Sciences Complex, University College Cork, College Road, Cork, Ireland

    John Browne

Authors
  1. Leah M. McClimans
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  2. John Browne
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Correspondence to Leah M. McClimans.

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McClimans, L.M., Browne, J. Choosing a patient-reported outcome measure. Theor Med Bioeth 32, 47–60 (2011). https://doi.org/10.1007/s11017-010-9163-8

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