Attitudes towards and participation in randomised clinical trials in oncology: A review of the literature
TL;DR: There is a paucity of research examining the association between knowledge about clinical trials, anxiety associated with a new cancer diagnosis and willingness to participate in randomised clinical trials.
About: This article is published in Annals of Oncology.The article was published on 2000-08-01 and is currently open access. It has received 265 citations till now. The article focuses on the topics: Clinical study design & Clinical trial.
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TL;DR: The reasons why study participation has been declining are considered, what is known about who does participate in epidemiologic studies is summarized, and methods that may help improve study participation rates are discussed.
1,653 citations
Cites background from "Attitudes towards and participation..."
...More recent studies have suggested that more complex consent procedures discourage study participation (54, 57, 64)....
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TL;DR: Elderly were under-represented in the registration trials of new cancer therapies and various strategies may be needed to evaluate cancer therapies for the elderly in prospective clinical trials and to improve cancer care in the elderly population.
Abstract: Purpose To analyze the age-related enrollment of cancer patients onto registration trials of new drugs or new indications approved by the US Food and Drug Administration from 1995 to 2002. Patients and Methods This study involved retrospective analyses of demographic data of cancer patients enrolled onto registration trials. The data on 28,766 cancer patients from 55 registration trials were analyzed according to age distributions of ≥ 65, ≥ 70, and ≥ 75 years. The rates of enrollment in each age group for each cancer were compared with the corresponding rates in the US cancer population. The age distributions of the US cancer population were derived from the Surveillance, Epidemiology, and End Results Program of the National Cancer Institute for the period 1995 to 1999 based on the 2000 US Census. Results The proportions of the overall patient populations aged ≥ 65, ≥ 70, and ≥ 75 years were 36%, 20%, and 9% compared with 60%, 46%, and 31%, respectively, in the US cancer population. Statistically signifi...
630 citations
TL;DR: Specific clinical trials confined to older patients should be conducted to evaluate tumor biology, treatment tolerability, and the effect of comorbid conditions, and protocol designs need to stratify for age and be less restrictive with respect to exclusions on functional status,Comorbidity, and previous cancers, such that results are generalizable to Older patients.
Abstract: Purpose Older patients are significantly underrepresented in cancer clinical trials. A literature review was undertaken to identify the barriers that impede the accrual of this vulnerable population onto clinical trials and to determine what specific strategies are needed to improve the representation of older patients in research studies. Methods A systematic literature search was undertaken using several different strategies to identify relevant articles. Results Nine of 31 relevant papers from 159 citations were included. Age is a significant barrier to recruitment; only a quarter to one third of potentially eligible older patients are enrolled onto trials. Physicians' perceptions, protocol eligibility criteria with restrictions on comorbid conditions, and functional status to optimize treatment tolerability are the most important reasons resulting in the exclusion of older patients. Other barriers include the lack of social support and the need for extra time and resources to enroll these patients. Co...
538 citations
TL;DR: Age and stage were the only predictors of whether a patient was offered a trial in a multivariate analysis including comorbid conditions, and the physicians' perceptions about age and tolerance of toxicity were the greatest impediment to enrolling older women onto trials.
Abstract: Purpose: Although 48% of breast cancer patients are 65 years old or older, these older patients are severely underrepresented in breast cancer clinical trials. This study tested whether older patients were offered trials significantly less often than younger patients and whether older patients who were offered trials were more likely to refuse participation than younger patients. Patients and Methods: In 10 Cancer and Leukemia Group B institutions, using a retrospective case-control design, breast cancer patients eligible for an open treatment trial were paired: less than 65 years old and ≥ 65 years old. Each of the 77 pairs were matched by disease stage and treating physician. Patients were interviewed as to their reasons for participating or refusing to participate in a trial. The treating physicians were also given questionnaires about their reasons for offering or not offering a trial. Results: Sixty-eight percent of younger stage II patients were offered a trial compared with 34% of the older patient...
436 citations
TL;DR: It is suggested that future trials should consider the different needs at different phases in the life of trials, and place greater emphasis on 'conduct' (the process of actually doing trials).
Abstract: Objectives To identify factors associated with good and poor recruitment to multicentre trials. Data sources Part A: database of trials started in or after 1994 and were due to end before 2003 held by the Medical Research Council and Health Technology Assessment Programmes. Part B: interviews with people playing a wide range of roles within four trials that their funders identified as 'exemplars'. Part C: a large multicentre trial (the CRASH trial) of treatment for head injury. Review methods The study used a number of different perspectives ('multiple lenses'), and three components. Part A: an epidemiological review of a cohort of trials. Part B: case studies of trials that appeared to have particularly interesting lessons for recruitment. Part C: a single, in-depth case study to examine the feasibility of applying a business-orientated analytical framework as a reference model in future trials. Results In the 114 trials found in Part A, less than one-third recruited their original target within the time originally specified, and around one-third had extensions. Factors observed more often in trials that recruited successfully were: having a dedicated trial manager, being a cancer or drug trial, and having interventions only available inside the trial. The most commonly reported strategies to improve recruitment were newsletters and mailshots, but it was not possible to assess whether they were causally linked to changes in recruitment. The analyses in Part B suggested that successful trials were those addressing clinically important questions at a timely point. The investigators were held in high esteem by the interviewees, and the trials were firmly grounded in existing clinical practices, so that the trial processes were not alien to clinical collaborators, and the results could be easily applicable to future practice. The interviewees considered that the needs of patients were well served by participation in the trials. Clinical collaborators particularly appreciated clear delineation of roles, which released them from much of the workload associated with trial participation. There was a strong feeling from interviewees that they were proud to be part of a successful team. This pride fed into further success. Good groundwork and excellent communications across many levels of complex trial structures were considered to be extremely important, including training components for learning about trial interventions and processes, and team building. All four trials had faced recruitment problems, and extra insights into the working of trials were afforded by strategies invoked to address them. The process of the case study in Part C was able to draw attention to a body of research and practice in a different discipline (academic business studies). It generated a reference model derived from a combination of business theory and work within CRASH. This enabled identification of weaker managerial components within CRASH, and initiatives to strengthen them. Although it is not clear, even within CRASH, whether the initiatives that follow from developing and applying the model will be effective in increasing recruitment or other aspects of the success of the trial, the reference model could provide a template, with potential for those managing other trials to use or adapt it, especially at foundation stages. The model derived from this project could also be used as a diagnostic tool if trials have difficulties and hence as a basis for deciding what type of remedial action to take. It may also be useful for auditing the progress of trials, such as during external review. Conclusions While not producing sufficiently definitive results to make strong recommendations, the work here suggests that future trials should consider the different needs at different phases in the life of trials, and place greater emphasis on 'conduct' (the process of actually doing trials). This implies learning lessons from successful trialists and trial managers, with better training for issues relating to trial conduct. The complexity of large trials means that unanticipated difficulties are highly likely at some time in every trial. Part B suggested that successful trials were those flexible and robust enough to adapt to unexpected issues. Arguably, the trialists should also expect agility from funders within a proactive approach to monitoring ongoing trials. Further research into different recruitment patterns (including 'failures') may help to clarify whether the patterns seen in the 'exemplar' trials differ or are similar. The reference model from Part C needs to be further considered in other similar and different trials to assess its robustness. These and other strategies aimed at increasing recruitment and making trials more successful need to be formally evaluated for their effectiveness in a range of trials.
418 citations
References
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TL;DR: Evidence Based Medicine (IBM) as discussed by the authors is the conscientious, explicit, and judicious use of current best evidence in making decisions about the care of individual patients, which is a hot topic for clinicians, public health practitioners, purchasers, planners and the public.
Abstract: It's about integrating individual clinical expertise and the best external evidence
Evidence based medicine, whose philosophical origins extend back to mid-19th century Paris and earlier, remains a hot topic for clinicians, public health practitioners, purchasers, planners, and the public. There are now frequent workshops in how to practice and teach it (one sponsored by the BMJ will be held in London on 24 April); undergraduate1 and postgraduate2 training programmes are incorporating it3 (or pondering how to do so); British centres for evidence based practice have been established or planned in adult medicine, child health, surgery, pathology, pharmacotherapy, nursing, general practice, and dentistry; the Cochrane Collaboration and Britain's Centre for Review and Dissemination in York are providing systematic reviews of the effects of health care; new evidence based practice journals are being launched; and it has become a common topic in the lay media. But enthusiasm has been mixed with some negative reaction.4 5 6 Criticism has ranged from evidence based medicine being old hat to it being a dangerous innovation, perpetrated by the arrogant to serve cost cutters and suppress clinical freedom. As evidence based medicine continues to evolve and adapt, now is a useful time to refine the discussion of what it is and what it is not.
Evidence based medicine is the conscientious, explicit, and judicious use of current best evidence in making decisions about the care of individual patients. The …
12,134 citations
TL;DR: An important goal of the medical residency program is to educate physicians in the practice of evidence-based medicine, and strategies include a weekly, formal academic half-day for residents devoted to learning the necessary skills.
Abstract: A NEW paradigm for medical practice is emerging. Evidence-based medicine de-emphasizes intuition, unsystematic clinical experience, and pathophysiologic rationale as sufficient grounds for clinical decision making and stresses the examination of evidence from clinical research. Evidence-based medicine requires new skills of the physician, including efficient literature searching and the application of formal rules of evidence evaluating the clinical literature. An important goal of our medical residency program is to educate physicians in the practice of evidence-based medicine. Strategies include a weekly, formal academic half-day for residents, devoted to learning the necessary skills; recruitment into teaching roles of physicians who practice evidence-based medicine; sharing among faculty of approaches to teaching evidence-based medicine; and providing faculty with feedback on their performance as role models and teachers of evidence-based medicine. The influence of evidencebased medicine on clinical practice and medical education is increasing. CLINICAL SCENARIO A junior medical resident working in a teaching hospital
3,906 citations
TL;DR: An alternative concept of equipoise is suggested, which would be based on present or imminent controversy in the clinical community over the preferred treatment, which is satisfied if there is genuine uncertainty within the expert medical community--not necessarily on the part of the individual investigator--about the preferredreatment.
Abstract: The ethics of clinical research requires equipoise--a state of genuine uncertainty on the part of the clinical investigator regarding the comparative therapeutic merits of each arm in a trial. Should the investigator discover that one treatment is of superior therapeutic merit, he or she is ethically obliged to offer that treatment. The current understanding of this requirement, which entails that the investigator have no "treatment preference" throughout the course of the trial, presents nearly insuperable obstacles to the ethical commencement or completion of a controlled trial and may also contribute to the termination of trials because of the failure to enroll enough patients. I suggest an alternative concept of equipoise, which would be based on present or imminent controversy in the clinical community over the preferred treatment. According to this concept of "clinical equipoise," the requirement is satisfied if there is genuine uncertainty within the expert medical community--not necessarily on the part of the individual investigator--about the preferred treatment.
2,088 citations
"Attitudes towards and participation..." refers background in this paper
...It has been argued that clinical trials should address questions where there is collective uncertainty within the expert medical community [8], However, an individual clinician's decision to recruit patients to a randomised trial is more likely to be influenced by their individual uncertainty than collective uncertainty....
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TL;DR: Most patients in each age group displayed high levels of hope, preferences for open communication about their illness, and a desire for maximum amounts of information.
Abstract: The present study explored the degree to which patients prefer to become informed about and to participate in their medical care. A total of 256 cancer patients completed an Information Styles Questionnaire and the Beck Hopelessness Scale. Patients' behavior and beliefs were found to incorporate the contemporary standard of informed and active involvement. Significant age trends were found: The younger the patients, the more closely they conformed to the well-informed participant standard of patient behavior; the older the patients, the more likely they were to prefer the older, nonparticipatory patient role. Patients who wanted to be involved in treatment decisions were significantly more hopeful than others. Most patients in each age group displayed high levels of hope, preferences for open communication about their illness, and a desire for maximum amounts of information.
1,145 citations
"Attitudes towards and participation..." refers background in this paper
...However, many patients choose to participate in clinical trials in the hope of receiving better care or treatment [37-39]....
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TL;DR: Priorities for information identified in this study provide an empirical basis to guide communication with women seeking care for breast cancer and suggest systematic approaches to assess and respond to women's desired level of participation in treatment decision making need to be evaluated.
Abstract: Objective. —To determine the degree of involvement women with breast cancer wanted in medical decision making, extent to which they believed they had achieved their preferred level of involvement, and types of information they judged to be most important. Design and Setting. —Cross-sectional survey at 2 tertiary oncology referral clinics and 2 community hospital oncology clinics in Winnipeg, Manitoba. Patients. —Consecutive sample of 1012 women with a confirmed diagnosis of breast cancer who were scheduled for a visit at 1 of 4 hospital oncology clinics. Main Outcome Measures. —The following measures were used: (1) Preferences about various levels of participation in treatment decision making; (2) the extent to which subjects believed they had achieved their preferred levels of involvement in decision making; and (3) priority needs for information and how these needs differed by selected sociodemographic, disease, and treatment variables. Results. —A total of 22% of women wanted to select their own cancer treatment, 44% wanted to select their treatment collaboratively with their physicians, and 34% wanted to delegate this responsibility to their physicians. Only 42% of women believed they had achieved their preferred level of control in decision making. The 2 most highly ranked types of information were related to knowing about chances of cure and spread of disease. Women younger than 50 years rated information about physical and sexual attractiveness as more important than did older women ( P P =.002); and women who had a positive family history of breast cancer rated information about family risk as more important than did other women ( P =.03). Conclusions. —The substantial discrepancy between women's preferred and attained levels of involvement in treatment decision making suggests that systematic approaches to assess and respond to women's desired level of participation in treatment decision making need to be evaluated. Priorities for information identified in this study provide an empirical basis to guide communication with women seeking care for breast cancer.
1,124 citations
"Attitudes towards and participation..." refers background in this paper
...These patient groups may also prefer to receive less information about their illness and are more likely to leave treatment decision-making to their doctor [44, 45]....
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