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Mitochondrial disease patient motivations and barriers to participate in clinical trials.

ABSTRACT: BACKGROUND:Clinical treatment trials are increasingly being designed in primary mitochondrial disease (PMD), a phenotypically and genetically heterogeneous collection of inherited multi- system energy deficiency disorders that lack effective therapy. We sought to identify motivating factors and barriers to clinical trial participation in PMD. METHODS:A survey study was conducted in two independent mitochondrial disease subject cohorts. A discovery cohort invited subjects with well-defined biochemical or molecularly- confirmed PMD followed at a single medical center (CHOP, n = 30/67 (45%) respondents). A replication cohort included self-identified PMD subjects in the Rare Disease Clinical Research Network (RDCRN) national contact registry (n = 290/1119 (26%) respondents). Five-point Likert scale responses were analyzed using descriptive and quantitative statistics. Experienced and prioritized symptoms for trial participation, and patient attitudes toward detailed aspects of clinical trial drug features and study design. RESULTS:PMD subjects experienced an average of 16 symptoms. Muscle weakness, chronic fatigue, and exercise intolerance were the lead symptoms encouraging trial participation. Motivating trial design factors included a self-administered study drug; vitamin, antioxidant, natural or plant-derivative; pills; daily treatment; guaranteed treatment access during and after study; short travel distances; and late-stage (phase 3) participation. Relative trial participation barriers included a new study drug; discontinuation of current medications; disease progression; daily phlebotomy; and requiring participant payment. Treatment trial type or design preferences were not influenced by population age (pediatric versus adult), prior research trial experience, or disease severity. CONCLUSIONS:These data are the first to convey clear PMD subject preferences and priorities to enable improved clinical treatment trial design that cuts across the complex diversity of disease. Partnering with rare disease patient communities is essential to effectively design robust clinical trials that engage patients and enable meaningful evaluation of emerging treatment interventions.

SUBMITTER: Zolkipli-Cunningham Z

PROVIDER: S-EPMC5957366 | biostudies-literature | 2018

REPOSITORIES: biostudies-literature

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Mitochondrial disease patient motivations and barriers to participate in clinical trials.

Zolkipli-Cunningham Zarazuela Z Xiao Rui R Stoddart Amy A McCormick Elizabeth M EM Holberts Amy A Burrill Natalie N McCormack Shana S Williams Lauren L Wang Xiaoyan X Thompson John L P JLP Falk Marni J MJ

PloS one 20180517 5

<h4>Background</h4>Clinical treatment trials are increasingly being designed in primary mitochondrial disease (PMD), a phenotypically and genetically heterogeneous collection of inherited multi- system energy deficiency disorders that lack effective therapy. We sought to identify motivating factors and barriers to clinical trial participation in PMD.<h4>Methods</h4>A survey study was conducted in two independent mitochondrial disease subject cohorts. A discovery cohort invited subjects with well ...[more]

PMID: 29771953

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Project description:BACKGROUND:Clinical trials are essential to improve knowledge of anesthesia and perioperative medicine. Unfortunately, many studies face participant-recruitment issues and fail to include the planned number of participants. There is limited published data about how information delivered about the study or how the experiences and attitudes of prospective participants influence willingness to participate. The purpose of this study was to identify such factors in the domain of anesthesia care. METHODS:We performed a cross-sectional study at the Geneva University Hospitals (Switzerland) using a newly developed paper-based questionnaire on a sample of outpatients with a recent hospital stay and that were aged over 18?years, confident speaking French and free of any disease that could hinder participation. We explored patient personal factors, such as current health, past exposure to clinical research and anesthesia, as well as study-related factors. Six different scenarios for clinical studies were assessed. Linear regression modeling was used to assess the specific association between personal and study-related factors and willingness to participate in the studies described in the scenarios. RESULTS:On the 1318 eligible patients, 398 fully completed the questionnaire. Multivariable adjustment revealed that factors related to altruistic values (?, 9.6, 95% CI 3.4 to 15.7, P?=?0.002), to the feeling of benefiting from a more effective treatment (?, 4.7, 95% CI 0.2 to 9.2, P?=?0.041) and to the absence of fear about double blinding (?, 5.7, 95% CI 1.3 to 10.2, P?=?0.012) were positively associated with willingness to participate. Conversely, concerns about drug-related adverse effects (?, -?11.7, 95% CI -?16.9 to -?6.5, P?<?0.001) and anxiety about surgery (?, -?5.2, 95% CI -?10.0 to -?0.5, P?=?0.031) were negatively associated with willingness to participate. CONCLUSION:Our study was based on vignettes illustrating typical scenarios of clinical trials performed in anesthesia. However, their similarities with real studies still remains hypothetical and our results should be interpreted as such. Nevertheless, the study contributes to improve understanding of factors that may act as incentives or barriers to participation in clinical trials. It highlights the importance of providing appropriate information and reassurance to patients.

Project description:BackgroundWhile patient and public involvement (PPI) in clinical trials is beneficial and mandated by some funders, formal guidance on how to implement PPI is limited and challenges have been reported. We aimed to investigate how PPI is approached within a UK Clinical Trials Unit (CTU)'s portfolio of randomised controlled trials, perceived barriers to/facilitators of its successful implementation, and perspectives on the CTU's role in PPI.MethodsA mixed-methods study design, involving (1) an online survey of 26 trial managers (TMs) and (2) Interviews with Trial Management Group members and public contributors from 8 case-study trials. Quantitative survey data were summarised using descriptive statistics and interview transcripts analysed thematically. Two public contributors advised throughout and are co-authors.Results(1) 21 TMs completed the survey; (2) 19 in-depth interviews were conducted with public contributors (n=8), TMs (n=5), chief investigators (n=3), PPI coordinators (n=2) and a researcher. 15/21 TMs surveyed reported that a public contributor was on the trial team, and 5 used another PPI method. 12/21 TMs reported that public contributors were paid (range £10-50/h). 5 TMs reported that training was provided for public contributors and few staff members had received any formal PPI training. The most commonly reported tasks undertaken by public contributors were the review of participant-facing materials/study documents and advising on recruitment/retention strategies. Public contributors wanted and valued feedback on changes made due to their input, but it was not always provided. Barriers to successful PPI included recruitment challenges, group dynamics, maintaining professional boundaries, negative attitudes to PPI amongst some researchers, a lack of continuity of trial staff, and the academic environment. Successful PPI required early and explicit planning, sharing of power and ownership of the trial with public contributors, building and maintaining relationships, and joint understanding and clarity about expectations/roles. CTUs have an important role to play in supporting recruitment, signposting and coordinating PPI.ConclusionsWhile highly valuable, PPI in trials is currently variable. PPI representatives are recruited informally, may not be provided with any training and are paid inconsistently across trials. Study findings can help optimise PPI in trials and ensure researchers and public contributors are adequately supported.

Project description:Rationale & objectiveData suggest that exercise is beneficial for patients with chronic kidney disease (CKD) to reduce cardiovascular disease and the progression of CKD. Despite these benefits, the majority of patients with CKD remain sedentary. The purpose of this study was to identify attitudes, motivators, and barriers to exercise among individuals with CKD.Study designQualitative study.Setting & participants10 focus groups (41 adult participants with CKD stages 3-4 who were not currently exercising) from the Denver Metro Area.Analytical approachThematic analysis. Clinics were purposively sampled. Focus groups were recorded and transcribed.ResultsWe identified 7 themes reflecting perceptions of exercise of patients with CKD: improvement in health and quality of life; motivation from family, friends, or peers; limitations due to comorbid conditions; challenges due to environmental factors (safety, weather, and cost concerns); lack of time; family as a barrier; and lack of provider counseling on type of exercise and the benefits. Participants recognized the benefits of exercise on health, but exercise was not viewed to have a significant impact on CKD. Having a friend, family member, or group to exercise with was viewed as a significant motivator. However, particularly in Spanish-speaking participants, family was often seen as a barrier if they were not willing to exercise with the patients. Most participants expressed receiving limited information from their kidney provider regarding how exercise affected their kidney health. Spanish-speaking participants in particular expressed frustration with their nephrologist's lack of advice regarding exercise.LimitationsPotential selection bias and inclusion of only sedentary patients with CKD.ConclusionsBarriers and motivators to exercise were similar among participating men and women with CKD. Lack of advice from kidney providers regarding exercise was a significant barrier. Exercise interventions in patients with CKD should include not only increased patient self-efficacy and social support, but also counseling and prescribing of exercise by nephrologists.

Project description:ObjectivesIf patients are to reap the benefits of continued drug development, an understanding of why healthy participants take part in phase I clinical trials is imperative. The current study aimed to explore the nature of these underlying motivations which may, in turn, improve the overall participant experience and assist in the development of more effective recruitment and retention strategies.DesignThis study used a qualitative design based on the theory of planned behaviour. Specifically, it explored healthy participants' underlying behavioural, control and normative beliefs which influence their participation in phase I clinical trials.SettingThis study took place at a company that specialises in conducting phase I and phase II clinical trials in the Australian state of Queensland.ParticipantsParticipants (n=31) were either currently undergoing a phase I clinical trial or had previously taken part in a phase I clinical trial.ResultsResults showed that the motivations were varied and not solely centred on financial gains. Reported advantages of participation included altruism, while inconvenience was most often reported as a disadvantage. Friends were reported as those most likely to approve, while one's mother was reported as most likely to disapprove. Having a suitable time frame/flexible scheduling and feeling comfortable taking part in the trial were both the most commonly reported facilitators, while inflexible scheduling/time commitment was the most commonly reported barrier.ConclusionsPractical implications included the need for organisations involved in clinical trials to be mindful of inflexible scheduling and exploring the possibility of making educational materials available to family members who may be concerned about the risks associated with participation. Overall, it is anticipated that the results of this study will improve the understanding of factors that influence phase I clinical trial participation which may, ultimately, help develop new therapeutics to improve patient health.

Project description:PurposeGeneral notification offers a possible alternative to written informed consent for pragmatic randomized controlled trials (pRCTs). It involves patients being informed through brochures, posters, and letters that research is being conducted simultaneously to providing clinical care and that patients will be enrolled in pRCTs without study-specific consent. A previous survey found that a substantial minority of respondents endorsed general notification. We aimed to know who is willing to enroll in this type of trials using general notification rather than written consent.MethodsThe previous study was a cross-sectional, probability-based survey, with a 2 × 2 factorial design. Two scenarios were assessed: two low-risk pRCTs in hypertension, one comparing two drugs with similar benefit/risk ratio and the other taking the same drug in the morning or at night. Each scenario had two routes: written consent vs verbal consent and written consent vs general notification. In this study, we were interested in the latter route in both scenarios. Respondents' preferences were measured based on their recommendation to the research ethics committee and the respondent's personal preference. We aimed to investigate the characteristics of those supporting general notification in either outcome or the variables explaining consistency and inconsistency between their personal preference and their recommendation. Based on the results of the original survey, we aimed to have at least 200 inconsistent respondents; to this end, the sample size was increased accordingly in a second wave of the survey.ResultsOne thousand six hundre and ten respondents were included; 1003 from the original survey and 607 new ones belonging to the second wave. Thirty-nine percent of respondents chose general notification as personal preference and/or recommendation. Respondents with lower education levels were more prone to accept general notification than those holding a university degree [OR (95% CI)], primary school [2.959 (2.069-4.232)], secondary school [2.899 (2.09-4.021)], or high school [1.620 (1.184-2.217)]. Also unemployed [1.372 (1.064-1.770)] and retired [1.445 (1.049-1.990)], but not students, showed preference for general notification in comparison with those employed. Individuals more than 24 years old and having received high school or university (or postgraduate) education were statistically significantly more consistent in their decisions.ConclusionsThirty-nine percent of respondents is open to not to be asked for their informed consent in low-risk pRCTs; of these, those being less educated and not having current job or being retired are significantly more open to general notification. The use of this alternative method to written consent for simultaneous conduct of pRCTs and care should be considered and educational programs settled up to, in the case of public acceptance, ensure its ethical appropriateness.

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Mitochondrial disease patient motivations and barriers to participate in clinical trials.

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Mitochondrial disease patient motivations and barriers to participate in clinical trials.

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