Dataset Information

Patient familiarity with, understanding of, and preferences for clinical trial endpoints and terminology.

ABSTRACT: BACKGROUND:Although there is increased attention to designing and explaining clinical trials in ways that are clinically meaningful for patients, there is limited information on patient preferences, understanding, and perceptions of this content. METHODS:Maximum difference scaling (MaxDiff) methodology was used to develop a survey for assessing patients' understanding of 19 clinical terms and perceived importance of 9 endpoint surrogate phrases used in clinical trials and consent forms. The survey was administered electronically to individuals with metastatic breast cancer affiliated with the Metastatic Breast Cancer Alliance. Analyses were performed using Bayesian P values with statistical software. RESULTS:Among 503 respondents, 77% had a college degree, 70% were diagnosed with metastatic disease ?2 years before survey completion, and 77% had received ?2 lines of systemic therapy. Less than 35% of respondents reported understanding "fairly well" the terms symptomatic progression, duration of disease control, time to treatment cessation, and endpoints. Income level and time since onset of metastatic disease correlated with comprehension. Patients who had received ?6 lines of therapy perceived that time until serious side effects (P < .001) and time on therapy (P < .001) were more important compared with those who had received only 1 line of therapy. Positively phrased parameters were associated with increased perceived importance. CONCLUSIONS:Even among educated, heavily pretreated patients, many commonly used clinical research terms are poorly understood. Comprehension and the perceived importance of trial endpoints vary over the course of disease. These observations may inform the design, discussion, and reporting of clinical trials.

SUBMITTER: von Itzstein MS

PROVIDER: S-EPMC7276207 | biostudies-literature | 2020 Apr

REPOSITORIES: biostudies-literature

ACCESS DATA

Publications

Patient familiarity with, understanding of, and preferences for clinical trial endpoints and terminology.

von Itzstein Mitchell S MS Railey Elda E Smith Mary L ML White Carol B CB Sledge George W GW Howell John R JR Lawton Wendy W Marinucci Donna M DM Unni Nisha N Gerber David E DE

Cancer 20200122 8

<h4>Background</h4>Although there is increased attention to designing and explaining clinical trials in ways that are clinically meaningful for patients, there is limited information on patient preferences, understanding, and perceptions of this content.<h4>Methods</h4>Maximum difference scaling (MaxDiff) methodology was used to develop a survey for assessing patients' understanding of 19 clinical terms and perceived importance of 9 endpoint surrogate phrases used in clinical trials and consent ...[more]

PMID: 31967687

Similar Datasets

Project description:ObjectivesCommunicating the concept of 'overdiagnosis' to lay individuals is challenging, partly because the term itself is confusing. This study tested whether alternative descriptive labels may be more appropriate.DesignQuestionnaire preceded by a description of overdiagnosis.SettingHome-based, computer-assisted face-to-face survey.Participants2111 adults aged 18-70 years in England recruited using random location sampling by a survey company. Data from 1888 participants were analysed after exclusions due to missing data.InterventionsParticipants were given one of two pieces of text describing overdiagnosis, allocated at random, adapted from National Health Service breast and prostate cancer screening leaflets.Primary and secondary outcome measuresMain outcomes were which of several available terms (eg, 'overdetection') participants had previously encountered and which they endorsed as applicable labels for the concept described. Demographics and previous exposure to screening information were also measured. Main outcomes were summarised with descriptive statistics. Predictors of previously encountering at least one term, or endorsing at least one as making sense, were assessed using binary logistic regression.Results58.0% of participants had not encountered any suggested term; 44.0% did not endorse any as applicable labels. No term was notably familiar; the proportion of participants who had previously encountered each term ranged from 15.9% to 28.3%. Each term was only endorsed as applicable by a minority (range: 27.6% to 40.4%). Notable predictors of familiarity included education, age and ethnicity; participants were less likely to have encountered terms if they were older, not white British or had less education. Findings were similar for both pieces of information.ConclusionsFamiliarity with suggested terms for overdiagnosis and levels of endorsement were low, and no clear alternative labels for the concept were identified, suggesting that changing terminology alone would do little to improve understanding, particularly for some population groups. Explicit descriptions may be more effective.

Project description:BackgroundPatients and their families often have preferences for medical care that relate to wider considerations beyond the clinical effectiveness of the proposed interventions. Traditionally, these preferences have not been adequately considered in research. Research questions where patients and families have strong preferences may not be appropriate for traditional randomized controlled trials (RCTs) due to threats to internal and external validity, as there may be high levels of drop-out and non-adherence or recruitment of a sample that is not representative of the treatment population. Several preference-informed designs have been developed to address problems with traditional RCTs, but these designs have their own limitations and may not be suitable for many research questions where strong preferences and opinions are present.MethodsIn this paper, we propose a novel and innovative preference-informed complementary trial (PICT) design which addresses key weaknesses with both traditional RCTs and available preference-informed designs. In the PICT design, complementary trials would be operated within a single study, and patients and/or families would be given the opportunity to choose between a trial with all treatment options available and a trial with treatment options that exclude the option which is subject to strong preferences. This approach would allow those with strong preferences to take part in research and would improve external validity through recruiting more representative populations and internal validity. Here we discuss the strengths and limitations of the PICT design and considerations for analysis and present a motivating example for the design based on the use of opioids for pain management for children with musculoskeletal injuries.ConclusionsPICTs provide a novel and innovative design for clinical trials with more than two arms, which can address problems with existing preference-informed trial designs and enhance the ability of researchers to reflect shared decision-making in research as well as improving the validity of trials of topics with strong preferences.

Dataset Information

Patient familiarity with, understanding of, and preferences for clinical trial endpoints and terminology.

Publications

Patient familiarity with, understanding of, and preferences for clinical trial endpoints and terminology.

Similar Datasets

OmicsDI is part of the ELIXIR infrastructure

Tweets