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Patients and investigators prefer measures of absolute risk in subgroups for pragmatic randomized trials.

ABSTRACT: OBJECTIVES:Pragmatic randomized trials are important tools for shared decision-making, but no guidance exists on patients' preferences for types of causal information. We aimed to assess preferences of patients and investigators toward causal effects in pragmatic randomized trials. STUDY DESIGN AND SETTING:We (a) held three focus groups with patients (n = 23) in Boston, MA; (b) surveyed (n = 12) and interviewed (n = 5) investigators with experience conducting pragmatic trials; and (c) conducted a systematic literature review of pragmatic trials (n = 63). RESULTS:Patients were distrustful of new-to-market medications unless substantially more effective than existing choices, preferred stratified absolute risks, and valued adherence-adjusted analyses when they expected to adhere. Investigators wanted both intention-to-treat and per-protocol effects but felt methods for estimating per-protocol effects were lacking. When estimating per-protocol effects, many pragmatic trials used inappropriate methods to adjust for adherence and loss to follow-up. CONCLUSION:We made four recommendations for pragmatic trials to improve patient centeredness: (1) focus on superiority in effectiveness or safety, rather than noninferiority; (2) involve patients in specifying a priori subgroups; (3) report absolute measures of risk; and (4) complement intention-to-treat effect estimates with valid per-protocol effect estimates.

SUBMITTER: Murray EJ

PROVIDER: S-EPMC6175611 | biostudies-literature | 2018 Nov

REPOSITORIES: biostudies-literature

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Patients and investigators prefer measures of absolute risk in subgroups for pragmatic randomized trials.

Murray Eleanor J EJ Caniglia Ellen C EC Swanson Sonja A SA Hernández-Díaz Sonia S Hernán Miguel A MA

Journal of clinical epidemiology 20180630

<h4>Objectives</h4>Pragmatic randomized trials are important tools for shared decision-making, but no guidance exists on patients' preferences for types of causal information. We aimed to assess preferences of patients and investigators toward causal effects in pragmatic randomized trials.<h4>Study design and setting</h4>We (a) held three focus groups with patients (n = 23) in Boston, MA; (b) surveyed (n = 12) and interviewed (n = 5) investigators with experience conducting pragmatic trials; and ...[more]

PMID: 29966732

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Project description:BackgroundThe requirement to obtain written informed consent may undermine the potential of pragmatic randomized clinical trials (pRCTs) to improve evidence-based care. This requirement could compromise trials statistical power or even force it to close them down prematurely. However, recent data from the U.S. and Spain suggest that a majority of the public endorses written consent for low-risk pRCTs. The present manuscript assesses whether this view is shared by patients.MethodsThis was a cross-sectional, probability-based survey, with a 2 × 2 factorial design, assessing support for written informed consent versus verbal consent or general notification for two low-risk pRCTs in hypertension, one comparing 2 drugs with similar risk/benefit profiles and the other comparing the same drug being taken in the morning or at night. This web-based survey was conducted in May 2016. Two-thousand and eight adults who were representative of the Spanish population participated in the survey (response rate: 61%). Of these 2008 respondents, 338 indicated that they had been diagnosed with hypertension and were being treated with prescription medicines for this condition at the time of responding to the survey. The primary outcome measures were respondents' personal preference and recommendation to a research ethics committee regarding the use of written informed consent versus verbal consent or general notification.ResultsOverall, 74% of the 338 patient respondents endorsed written consent. In both scenarios, general notification received significantly more support (30.6%-44.7%) than verbal consent (13.3%-17.6%). 43% of respondents preferred and/or recommended general notification rather than written consent.ConclusionsAs in the survey of the general public, more patients endorsed written consent than the alternative option. However, two factors suggest that a different approach to written consent should be investigated for low-risk pRCTs: a) a substantial minority of respondents supported general notification, b) data from the US have shown that most patients who prefer written consent are willing to forego it if obtaining written consent makes the trial too difficult to be conducted; and c) 2016 CIOMS guidelines endorse waivers of consent when the trial fulfills specific conditions. Surveys in other EU countries are needed to assess what patients believe towards pRCTs. If similar results to that reported in this study are found, it is foreseeable that with educational efforts, general notification could be an acceptable and widespread approach to the conduct of low-risk pRCTs.

Project description: To compare the effectiveness of shared decision making with usual care in choice of admission for observation and further cardiac testing or for referral for outpatient evaluation in patients with possible acute coronary syndrome. Multicenter pragmatic parallel randomized controlled trial. Six emergency departments in the United States. 898 adults (aged >17 years) with a primary complaint of chest pain who were being considered for admission to an observation unit for cardiac testing (451 were allocated to the decision aid and 447 to usual care), and 361 emergency clinicians (emergency physicians, nurse practitioners, and physician assistants) caring for patients with chest pain. Patients were randomly assigned (1:1) by an electronic, web based system to shared decision making facilitated by a decision aid or to usual care. The primary outcome, selected by patient and caregiver advisers, was patient knowledge of their risk for acute coronary syndrome and options for care; secondary outcomes were involvement in the decision to be admitted, proportion of patients admitted for cardiac testing, and the 30 day rate of major adverse cardiac events. Compared with the usual care arm, patients in the decision aid arm had greater knowledge of their risk for acute coronary syndrome and options for care (questions correct: decision aid, 4.2 v usual care, 3.6; mean difference 0.66, 95% confidence interval 0.46 to 0.86), were more involved in the decision (observing patient involvement scores: decision aid, 18.3 v usual care, 7.9; 10.3, 9.1 to 11.5), and less frequently decided with their clinician to be admitted for cardiac testing (decision aid, 37% v usual care, 52%; absolute difference 15%; P<0.001). There were no major adverse cardiac events due to the intervention. Use of a decision aid in patients at low risk for acute coronary syndrome increased patient knowledge about their risk, increased engagement, and safely decreased the rate of admission to an observation unit for cardiac testing.Trial registration ClinicalTrials.gov NCT01969240.

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Patients and investigators prefer measures of absolute risk in subgroups for pragmatic randomized trials.

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Patients and investigators prefer measures of absolute risk in subgroups for pragmatic randomized trials.

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