In deciding whether to use a particular treatment for conditions such as depression,
arthritis, or heart disease, clinicians and patients must balance the benefits against
the side effects and burden. To make this trade-off, they must understand the likely
degree of benefit in patients’ symptoms and perceived wellbeing, best undertaken
using patient-reported outcomes (PROs). PROs are measures of any aspect of a
patients’ health status that are obtained from direct patient inquiry without
interpretation by a clinician or anyone else. PRO measures (PROMs) are
increasingly used in clinical trials and systematic reviews to evaluate health care
interventions, and information obtained from PROMs can guide clinical decisions
and inform shared-decision making. The use of PROMs, however, involves
challenges, the most important of which is deciding if a particular treatment effect
is trivial, small but important, moderate or large. One way to make this judgment is
to consider the minimal important difference (MID), the smallest change in a
PROM score that is important enough that patients would consider a change in
treatment to achieve that benefit. The number of published studies providing
anchor-based MIDs for PROMs has grown rapidly over the last three decades, and
researchers have proposed several anchor-based methods to derive MID estimates,
each with its own merits and limitations. This thesis begins with the development
of a framework to determine the extent to which the design and conduct of studies
measuring anchor-based MIDs are likely to have protected against misleading
estimates. Subsequently, this thesis presents a comprehensive inventory of
empirically estimated anchor-based MIDs and their associated credibility for all
PROMs published in the medical literature. Further, this thesis highlights critical
issues that key stakeholders should consider, and demonstrates how the use of
credible MIDs may inform the development of a clinical practice guideline in which
PROs were identified as critically important. Finally, this thesis concludes with
insights to improve the methodological quality and transparency for researchers in
the PRO and MID field. / Thesis / Doctor of Philosophy (PhD)
| Identifer | oai:union.ndltd.org:mcmaster.ca/oai:macsphere.mcmaster.ca:11375/23923 |
| Date | 23 May 2019 |
| Creators | Devji, Tahira |
| Contributors | Guyatt, Gordon, Health Research Methodology |
| Source Sets | McMaster University |
| Language | English |
| Detected Language | English |
| Type | Thesis |
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