Among initiatives being piloted and implemented are the following: Patients look to their physicians and nurses as the most trusted and primary source for education and information about clinical trials and for advice on whether to enroll in a clinical trial. Effective, continuous improvement in patient engagement depends on an intimate understanding of public and patient attitudes and perceptions and experiences in clinical research.
It has been more than 12 years since public knowledge and perceptions of clinical research were formally discussed in JAMA , 10 and at that time, only the opinions of Americans were summarized. In this article, we present key findings of a , multicontinent country survey examining attitudes, perceptions, and experiences of clinical research to provide updated benchmarks that inform patient engagement strategies and tactics implemented by clinical research and clinical care professionals.
This study examined the views of clinical trial participants and nonparticipants over time to provide insight into gaps in public awareness and knowledge about clinical research, identify opportunities to reduce unnecessary participation burden, and reveal where current efforts are or are not resonating with the public and patients.
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Continuous assessment of public and patient attitudes, perceptions, and experiences in clinical research may inform patient engagement strategies and tactics that may ultimately accelerate the pace at which useful medicines are currently getting to market and help mitigate increasing drug-development costs and decreasing success rates of new drug and biologic agent approvals.
The Center for Information and Study on Clinical Research Participation CISCRP , an independent nonprofit organization in Boston, Massachusetts, began administering a global, online study on a biennial basis in to evaluate public and patient perceptions about clinical research and the motivations and experiences of clinical trial participants. Implied consent was used in the study. Participants were informed of the study objectives and risks and benefits of the survey and assured of anonymity.
All data were deidentified. These organizations disseminated an online link to the survey to their respective networks. These networks were composed of individuals who have opted to receive health-related information. The findings are organized into 3 distinct groupings: The survey instrument was developed by a cross-functional work group composed of patients and representatives from biopharmaceutical companies, contract research organizations, and investigative sites.
To allow for longitudinal analyses, many of the questions on the original survey were posed in subsequent surveys in and Additional, new topic areas were incorporated in the latest survey to provide baseline figures for future comparisons about issues of heightened recent interest, including the association between health care professionals and the clinical research experience and initiatives that enhance study volunteer convenience, enrollment, and retention. The overall structure of the survey has remained constant, starting with questions that assess general clinical research impressions among the public and patients using conditional branching to further probe those who had actively searched for trials, expressed an interest in doing so, or participated in a clinical trial.
The survey was programmed in and administered via an online survey tool. The data set was subsequently exported from the online survey tool and imported into MarketSight data analysis and visualization software MarketSight LLC. Data sets for the and studies were also imported into this software. Comparisons among subgroups in the study and with the and studies were performed using z tests, and comparisons of means were performed using t tests.
The data for this article were specifically chosen to reveal multiyear trends in public and patient perceptions about the value of clinical research, factors that influenced their decision whether to participate in a study, and the clinical trial experience. Table 1 summarizes the survey respondent characteristics and compares them with those from the and surveys.
The concentrations of respondents in were Although interest in participating in a clinical trial remains high overall, a smaller proportion of individuals [ Both of these figures were significantly higher for those who had participated in a clinical research study in the past and were more adept at using online clinical trial registries to locate a trial general willingness to participate: In the study, of respondents An even higher number of [ The time to bring a drug to market a mean of 12 years also continued to be underestimated.
In the survey, nearly respondents A total of respondents Among the respondents The top perceived risks and benefits associated with clinical research remained largely unchanged over time Table 2 , with adverse effects [ Altruistic variables, such as helping to advance science [ A well-informed health care professional was also found to positively affect patient perceptions of clinical research and participation experiences.
A high comfort level among patients in using the health information to identify clinical trials is in contrast with a recent study 22 that found patient distrust in medical leaders and the health system as a whole. In each of the 3 CISCRP studies, patients consistently reported a preference to learn about clinical research through their regular physician or specialist more than any other source.
This preference has increased [ In the study, the highest number of the study volunteers [ Only of respondents Integrating routine and clinical study visits appealed disproportionately more to women than to men. The choice to participate in a clinical trial is not straightforward but involves numerous considerations. More practical aspects were also identified as important, including the physical location of the study center [ Among younger respondents, greater importance was placed on taking time off from work and having study visits conducted at their home or office when deciding whether to participate.
For older respondents, the greater concern was having access to a study drug after their participation ended. The study also found that of those considering participation in a clinical study, Consulting with peers in an online patient community was identified as another key source of information, with The highest interest in using this information channel was observed among younger respondents aged years.
The survey results included 2 indicators that clinical study participation experiences remained positive. A total of of the respondents A total of In the survey, the figures were similarly high [ However, in the survey, respondents For the respondents who once qualified for a study, 94 Overall, adoption of new convenience-enhancing technologies and services appear to be in early stages. Keeping clinicians in clinical research: A systematic review highlights threats to validity in studies of barriers to cancer trial participation.
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Clinical Trials - General Internal Medicine - Mayo Clinic
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Introduction
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Knowledge and attitudes about health research amongst a group of Pakistani medical students. For endoscopic endpoints, reproducibility has proven problematic, leading many studies to move to grading of endoscopic appearance by central reviewers in order to improve consistency. Analyses of multicenter trials should always consider site as a covariate, as a few outlier sites can skew the outcome. Over time, clinicians will need to determine whether there is an improved, long-term, beneft-to-risk ratio that results from achieving biologic or endoscopic remission in patients with IBD.
In an example from another field of medicine, very tight control of blood pressure showed no survival benefit over usual control; to avoid similar missteps, clinicians need prospective evidence that tight control in IBD will improve outcomes. One of the challenges of all trials is how to deal with missing data. Subjects often drop out of a study before the primary endpoint date is reached, fail to show up for appointments, or change their mind and refuse to undergo the scheduled endoscopy at the final evaluation.
There are 2 common methods for handling missing data.
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The most rigorous method is to consider any subject who has missing data as one who would have failed to meet the endpoint. This method is often called nonresponder imputation. It lowers success rates and placebo rates , thus making results look less impressive. Another approach is to use the last observation at which the subject was measured in place of the missing data; this approach is especially common in maintenance studies with repeated measurements.
Called the last-observation-carried-forward approach, this method is reasonable, but it may inflate the success rates of both the primary intervention and the control arm, as a common reason patients do not return for visits is that they feel the treatment does not provide a clinical benefit. Finally, a third approach for addressing missing data is called imputation, in which other data are used to estimate what the missing data would have been.
This approach is generally considered appealing but questionable, as it is impossible to check the accuracy of these estimates. A number of clinical journals and funding sources expect clinical trials to be registered on the ClinicalTrials. This website contains a large amount of information about the trial, including endpoints, date of first enrollment, inclusion and exclusion criteria, and a description of the intervention.
An interested reader can view the registration page for the trial to ensure that the endpoints did not change during the study and that the trial was truly prospective. Readers may also discover that some of the outcomes mentioned on the initial registration page are not reported in the published paper.
In these instances, the reader could presume that the outcome results were disappointing.
Understanding Medical Research
Many readers rely on P -values to determine if a clinically important effect is present. The P -value is based on the assumption that the null hypothesis is true that the treatment arms are equally effective , and the P -value gives the probability that the difference observed between the 2 treatment arms is due to chance alone. For example, if testing the difference in blood pressures between 2 populations that truly had the same blood pressure resulted in a P -value of. Statistically, a P -value serves as a combination of 2 measurements: These 2 values should be displayed in a paper and should be examined separately.
A very large study will have more precision, which will generally lead to a smaller P -value without a meaningful change in effect size. A very small study will inherently have less precision, in which case a clinically meaningful effect size may be missed due to a higher P -value. To illustrate how a large sample size can make a small effect significant, consider the 2 examples in Figure 1. A large sample size, as shown in Figure 1 A, can make a blood pressure difference of 2. When the difference between the 2 groups is larger 12 mmHg , a sample size of 50 patients per group is only barely sufficient to yield a statistically significant difference between the 2 groups Figure 1 B.
The smaller study may have a larger and more clinically relevant effect, but this effect is difficult to detect with a small sample size. If the smaller study had failed to reach its recruiting goal and had stopped at 40 patients per arm, the P -value would not have been significant. Figure 1 A shows overlaid mountain plots of the systolic blood pressures of two subject arms of a hypothetical study. The difference between the 2 means is 2.
How to Read a Clinical Trial Paper
Because of the large sample size, this small difference achieves a statistically significant P -value. Figure 1 B shows overlaid mountain plots of the systolic blood pressures of two subject arms of a much smaller hypothetical study, which has a difference between the means of 12 mmHg.
Due to the smaller sample size, this study barely achieves the same P -value, despite having a larger, clinically significant effect. As mentioned earlier, the dropout rate due to lack of benefit can be compared between the different arms of a study, and this rate should be lowest in the arm with the most effective treatment. A meta-analysis measuring this outcome compared the clinical response in ulcerative colitis patients who were treated with 5-aminosalicylic acid drugs versus placebo, and it confirmed that the dropout rate was a powerful and intuitive way to confirm clinical response from the point of view of the patient, no matter how clinical response was measured in the study.
Data from Colombel J F, et al. This number represents the reciprocal of the absolute risk reduction and gives the reader an understanding of how many patients a clinician would have to treat to see the desired outcome. For example, in a study assessing the benefit of high-dose proton pump inhibitor PPI therapy prior to endoscopy, high-dose therapy was shown to reduce the need for endoscopic therapy in patients with gastrointestinal bleeding. The absolute risk difference was.
There-fore, a clinician would have to treat 11 patients presenting with upper gastrointestinal bleeding with high-dose PPI therapy prior to endoscopy in order to reduce the need for endoscopic therapy by 1 patient. Finally, readers should consider a few points when reviewing figures. The most basic and easily overlooked errors involve axis manipulation: Having an axis that covers the full range of possible results for a measurement is generally best. Another consideration with graphs is that, while plots of means without an indication of variability may be more aesthetically pleasing, these figures do not give the reader an understanding of the variation in the data.
While researchers have learned a great deal from randomized controlled trials, some hypotheses cannot be tested with prospective randomized controlled trials due to cost, duration, or ethical constraints. For example, a recent cross-sectional study of the relationship between fiber intake and diverticular disease is a reminder that other study designs serve an important function in medicine.
With this point in mind, we emphasize the importance of considering study designs beyond clinical trials, as other types of studies can address important questions that are not suited to a prospective randomized controlled trial design. National Center for Biotechnology Information , U. Gastroenterol Hepatol N Y. Author information Copyright and License information Disclaimer. Govani is a Fellow and Dr. Abstract While the number of clinical trials performed yearly is increasing, the application of these results to individual patients is quite difficult. Statistics, clinical trials, randomized controlled trials, outcomes, clinical research.
Open in a separate window. What Happened to the Subjects? Did They Drop Out? Is the Study Design Biased? Is This a Test of Superiority? Does the Measurement Matter? Surrogate Outcomes Ideally, the outcome being studied should be the one in which clinicians are most interested. Dichotomous Outcomes, Continuous Outcomes, Correlations, and Time-to-Event Endpoints Some studies focus on a dichotomous outcome endpoint like clinical remission or response. Multicenter Trials Multicenter trials offer a chance to expand the sample size in studies of rare diseases and to ensure some population diversity.
How Are Missing Data Addressed?