Abstract
Background
As more studies report on patient preferences, techniques are needed to identify, assess and, eventually, synthesize results from a diverse set of methodologies. Data on patient preferences are valuable to decision makers in a variety of ways. Preferences for outcomes can be used to inform decision and cost-effectiveness models, while preferences for treatments can inform patient-centered outcomes research (PCOR) and patient-centered care.
Objectives
This project sought to identify and assess the literature reporting on the treatment preferences of adult patients with type 2 diabetes. In addition to cataloging the preference elicitation methods used, we developed and assessed a novel quality assessment checklist for preference-based studies.
Data sources
PubMed, EMBASE, CINAHL, and EconLit databases were searched to identify studies examining patient preferences for medications for type 2 diabetes studies published since inception of each database.
Study eligibility criteria, participants, and interventions
The review protocol specified inclusion of studies reporting diabetes-treatment preferences among adults with type 2 diabetes, using a range of preference measurement methods. Studies were excluded if participants were not patients with type 2 diabetes and if treatments were not pharmacological therapies targeting glycemic control, or if no primary preference information was collected. Two investigators independently reviewed titles, abstracts, and articles sequentially to select studies for data abstraction based on the inclusion and exclusion criteria. Disagreements were resolved by consensus.
Study appraisal and synthesis methods
Data on study country, year, number of respondents, preference elicitation method, number of attributes, subgroup analyses, and funding source were abstracted into standardized tables. A novel checklist (PREFS) was used to assess the data quality and validity across different types of preference studies by assessing the following: purpose of the study; respondent sampling; explanation of preference assessment methods; findings reported for total sample; and significance testing. Each item was scored, and an aggregate score was then calculated (ranging from 0 to 5).
Results
Of the 2,100 unique citations, 61 met the inclusion criteria. The studies used conjoint analysis (n = 10), time trade-off (n = 6), standard gamble (n = 2), contingent valuation (n = 1), other stated preference methods (n = 39), and revealed preferences (n = 5). Sample sizes ranged from 27 to 14,033, with an average of 562 respondents, and two-thirds included a subgroup analysis. Most studies were conducted in one country, predominantly the USA (n = 27), UK (n = 14), Canada (n = 10), and Germany (n = 7), while 14 were conducted in multiple (2–18) countries across two or more countries. There was an increase in the annual rate of studies published over time from the time of the first publication in 1985 (p = < 0.01). Most (n = 52) studies were funded by pharmaceutical or device companies, with government, academic, association, and hospital sources also funding studies. One study met all five of the PREFS criteria and 12 met four; yet four studies met none of the criteria. The average was 27.
Limitations
Currently, preferences reviews are limited by the mixed quality in the reporting of studies, the publication bias inherent in the literature, a lack of guidelines to conduct various methods, and the difficulty of synthesizing results from different studies. Our study is also limited by its focus on English language articles.
Conclusions and implications of key findings
This study provides the first systematic evaluation of the methods used in the broad existing body of research into patient preferences for type 2 diabetes medications and can serve as a primary source of information for decision makers. Future work is necessary to assess the utility of the results of reviews of preference information and to develop best-practice guidelines for the reporting of, and methods of conducting, preference studies and systematic reviews of such studies.
Registration
This systematic review was registered with PROSPERO (registration number CRD42012002285).
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Acknowledgments
The analysis upon which this publication is based was performed under contract number HHSF2232010000072C, entitled, “Partnership in Applied Comparative Effectiveness Science,” sponsored by the Food and Drug Administration, Department of Health and Human Services. The funder had no role in designing and conducting the study; collection, management, analysis, or interpretation of the data; or preparation/approval of the manuscript. SJ, NM, and JB conceptualized this paper; SJ, JB, EL, and TP developed the criteria for identifying preferences studies and rating their quality; SJ, EL, and TP conducted the systematic review; NM and TP provided clinical insights; and SJ and JB led the writing of the manuscript. SJ, EL, NM, TP, and JB all contributed to the writing of the manuscript and approved the final version. SJ, EL, NM, TP, and JB report no conflicts of interest. We thank Dr Jodi Segal for her advice and encouragement.
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Appendix 1: Example of Search Terms (PubMed)
Appendix 1: Example of Search Terms (PubMed)
“Diabetes mellitus, type 2”[mh] OR diabet*[tiab] OR “non-insulin dependent”[tiab] OR type-2[tiab] OR “type II”[tiab] OR “type 2”[tiab] “ketosis-resistant diabetes mellitus”[tw] OR “non-insulin-dependent diabetes mellitus”[tw] OR “type 2 diabetes mellitus”[tw] OR “stable diabetes mellitus”[tw] OR “maturity-onset diabetes mellitus”[tw] OR “maturity onset diabetes mellitus”[tw] OR “MODY”[tw] OR “NIDDM”[tw] OR “adult-onset diabetes mellitus”[tw]
AND
Treatment[tiab] OR management[tiab] OR pharmaceutical[tiab] OR drug therapy[mesh] OR medication[tiab]
AND
“Conjoint analysis” OR “satisfaction” OR “choice model” OR “stated preference” OR “discrete choice” OR DCE OR “decision analysis” OR preferences OR “multi-criteria decision analysis” OR MCDA OR “multi-attribute utility” OR “analytic hierarchy process” OR “trade off” OR “self-explicated” OR “best-worst scaling” OR utilities OR “preference weight” OR “willingness to pay” OR WTP OR “willingness to accept” OR “contingent valuation” OR priorities[tiab] OR valuation[tiab].
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Joy, S.M., Little, E., Maruthur, N.M. et al. Patient Preferences for the Treatment of Type 2 Diabetes: A Scoping Review. PharmacoEconomics 31, 877–892 (2013). https://doi.org/10.1007/s40273-013-0089-7
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DOI: https://doi.org/10.1007/s40273-013-0089-7