Incorporating patient preferences into clinical trial design: Results of the Opinions of Patients on Treatment Implications of New Studies (OPTIONS) project

Stafinski, Tania; Menon, Devidas; Nardelli, Alex; Bakal, Jeffrey A.; Ezekowitz, Justin A.; Tymchak, Wayne; Welsh, Robert C.; Gyenes, Gábor; Armstrong, Paul W.

doi:10.1016/j.ahj.2014.10.002

Cited by 25 publications

(17 citation statements)

References 21 publications

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“…The current study and previous research in this field suggest that this is not the case . In accordance with previous recommendations, we therefore recommend using “weighted” CE, in which individual CEs a valued relative to one another . Prior efforts to weigh these CE often assumed that patients, doctors and other experts would assign similar values to individual CEs; however, the study of Stolker et al .…”

Section: Discussionsupporting

confidence: 75%

“…The attributes included in this study were determined in a stepwise manner, which subsequently included a literature review, expert review and individual interviews with patients. First, a list of attributes that describe possible unfavourable outcomes of revascularization was compiled based on previously published literature . Second, this list of unfavourable outcomes was discussed within the steering committee of the research team (including 2 cardiologists with expertise in these interventions, and 2 senior health scientists for specialist methodological input).…”

Section: Methodsmentioning

confidence: 99%

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Are component endpoints equal? A preference study into the practice of composite endpoints in clinical trials

Vaanholt

Kok

Birgelen

et al. 2018

Health Expectations

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ObjectivesTo examine patients’ perspectives regarding composite endpoints and the utility patients put on possible adverse outcomes of revascularization procedures.DesignIn the PRECORE study, a stated preference elicitation method Best‐Worst Scaling (BWS) was used to determine patient preference for 8 component endpoints (CEs): need for redo percutaneous coronary intervention (PCI) within 1 year, minor stroke with symptoms <24 hours, minor myocardial infarction (MI) with symptoms <3 months, recurrent angina pectoris, need for redo coronary artery bypass grafting (CABG) within 1 year, major MI causing permanent disability, major stroke causing permanent disability and death within 24 hours.SettingA tertiary PCI/CABG centre.ParticipantsOne hundred and sixty patients with coronary artery disease who underwent PCI or CABG.Main outcome measuresImportance weights (IWs).ResultsPatients considered need for redo PCI within 1 year (IW: 0.008), minor stroke with symptoms <24 hours (IW: 0.017), minor MI with symptoms <3 months (IW: 0.027), need for redo CABG within 1 year (IW: 0.119), recurrent angina pectoris (IW: 0.300) and major MI causing permanent disability (IW: 0.726) less severe than death within 24 hours (IW: 1.000). Major stroke causing permanent disability was considered worse than death within 24 hours (IW: 1.209). Ranking of CEs and the relative values attributed to the CEs differed among subgroups based on gender, age and educational level.ConclusionPatients attribute different weight to individual CEs. This has significant implications for the interpretation of clinical trial data.

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Section: Discussionsupporting

confidence: 75%

Section: Methodsmentioning

confidence: 99%

Are component endpoints equal? A preference study into the practice of composite endpoints in clinical trials

Vaanholt

Kok

Birgelen

et al. 2018

Health Expectations

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“…We included all except death in the survey. Based on another study [19], we assumed that death would almost always be considered the most worrisome outcome. We described symptomatic outcomes in lay language with expected severities based on input from clinicians and our patient and caregiver co-investigators.…”

Section: Methodsmentioning

confidence: 99%

Outcome preferences of older people with multiple chronic conditions and hypertension: a cross-sectional survey using best-worst scaling

Aschmann

Puhan

Robbins

et al. 2019

Health Qual Life Outcomes

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BackgroundOlder people with hypertension and multiple chronic conditions (MCC) receive complex treatments and face challenging trade-offs. Patients’ preferences for different health outcomes can impact multiple treatment decisions. Since evidence about outcome preferences is especially scarce among people with MCC our aim was to elicit preferences of people with MCC for outcomes related to hypertension, and to determine how these outcomes should be weighed when benefits and harms are assessed for patient-centered clinical practice guidelines and health economic assessments.MethodsWe sent a best-worst scaling preference survey to a random sample identified from a primary care network of Kaiser Permanente (Colorado, USA). The sample included individuals age 60 or greater with hypertension and at least two other chronic conditions. We assessed average ranking of patient-important outcomes using conditional logit regression (stroke, heart attack, heart failure, dialysis, cognitive impairment, chronic kidney disease, acute kidney injury, fainting, injurious falls, low blood pressure with dizziness, treatment burden) and studied variation across individuals.ResultsOf 450 invited participants, 217 (48%) completed the survey, and we excluded 10 respondents who had more than two missing choices, resulting in a final sample of 207 respondents. Participants ranked stroke as the most worrisome outcome and treatment burden as the least worrisome outcome (conditional logit parameters: 3.19 (standard error 0.09) for stroke, 0 for treatment burden). None of the outcomes were always chosen as the most or least worrisome by more than 25% of respondents, indicating that all outcomes were somewhat worrisome to respondents. Predefined subgroup analyses according to age, self-reported life-expectancy, degree of comorbidity, number of medications and antihypertensive treatment did not reveal meaningful differences.ConclusionsAlthough some outcomes were more worrisome to patients than others, our results indicate that none of the outcomes should be disregarded for clinical practice guidelines and health economic assessments.

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“…Patient-reported endpoints are also necessary for health technology assessments and can provide clinicians and patients with useful information to support patient education and decision-making. 31 Research agenda to advance clinical trial science and improve patient outcomes in chronic kidney disease…”

Section: Competing Risksmentioning

confidence: 99%

Cardiovascular outcome trials in patients with chronic kidney disease: challenges associated with selection of patients and endpoints

Rossignol

Agarwal

Canaud

et al. 2017

European Heart Journal

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Although cardiovascular disease is a major health burden for patients with chronic kidney disease, most cardiovascular outcome trials have excluded patients with advanced chronic kidney disease. Moreover, the major cardiovascular outcome trials that have been conducted in patients with end-stage renal disease have not demonstrated a treatment benefit. Thus, clinicians have limited evidence to guide the management of cardiovascular disease in patients with chronic kidney disease, particularly those on dialysis. Several factors contribute to both the paucity of trials and the apparent lack of observed treatment effect in completed studies. Challenges associated with conducting trials in this population include patient heterogeneity, complexity of renal pathophysiology and its interaction with cardiovascular disease, and competing risks for death. The Investigator Network Initiative Cardiovascular and Renal Clinical Trialists (INI-CRCT), an international organization of academic cardiovascular and renal clinical trialists, held a meeting of regulators and experts in nephrology, cardiology, and clinical trial methodology. The group identified several research priorities, summarized in this paper, that should be pursued to advance the field towards achieving improved cardiovascular outcomes for these patients. Cardiovascular and renal clinical trialists must partner to address the uncertainties in the field through collaborative research and design clinical trials that reflect the specific needs of the chronic and end-stage kidney disease populations, with the shared goal of generating robust evidence to guide the management of cardiovascular disease in patients with kidney disease.

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Incorporating patient preferences into clinical trial design: Results of the Opinions of Patients on Treatment Implications of New Studies (OPTIONS) project

Cited by 25 publications

References 21 publications

Are component endpoints equal? A preference study into the practice of composite endpoints in clinical trials

Are component endpoints equal? A preference study into the practice of composite endpoints in clinical trials

Outcome preferences of older people with multiple chronic conditions and hypertension: a cross-sectional survey using best-worst scaling

Cardiovascular outcome trials in patients with chronic kidney disease: challenges associated with selection of patients and endpoints

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