Chapter 16: ResultsIn this section, we present the results of the SIS. We begin with a description of the demographic characteristics of the patients we interviewed, as well as the basic characteristics of the research projects in which some of these patients were or had been participating. We then review what we learned about patients' general attitudes toward, and beliefs about, research and their understanding of some of the terms commonly used to describe research to potential subjects. This is followed by our results concerning patients' perceptions of whether they are, or are not, participants in research and the extent to which we were able to compare these perceptions with documents and other sources. We then discuss what patients said about the distinctions between research and treatment, and their reasons for deciding to participate. We also describe the characteristics of patients who reported that they declined to be research participants. Our discussion of results closes with what we learned from the SIS about the consent process and issues of voluntariness of participating in research.
A total of 1,882 patients completed the Brief Survey. The overall response rate was 95 percent. Patients predominantly were Caucasian (80%), more than sixty years old (53%), and male (59%). Other relevant demographic features are found in table 2.
A total of 103 patients, representing fourteen of the sixteen institutions included in the overall study sample, were interviewed. This sample also was predominantly Caucasian (74%) and male (54%) (see table 2). Due to technical or administrative difficulties with four interviews, only ninety-nine transcripts were available for analysis.
Characteristics of the Research Projects
The characteristics of the projects in which patients participated are described in table 3. The expert panel categorized the disease burden associated with the projects reviewed as low (11%), medium (38%), and high (51%). Approximately half (48%) involved minimal incremental risk from research.
General Attitudes Toward and Beliefs About Biomedical Research
In the Brief Survey, patients were asked a series of questions concerning their attitudes toward, and beliefs about, "medical research." Almost all the patients had positive impressions of medical research. Specifically, 52 percent reported a "very favorable" attitude toward research and 37 percent a "somewhat favorable" attitude. Only 5 percent of patients described themselves as having an unfavorable attitude. Controlling for multiple factors, the characteristics associated with more favorable general attitudes toward research included being older (age greater than sixty), being male, being a patient in radiation oncology rather than cardiology, and having reported currently being or having been a participant in research.
More than two-thirds of the patients reported that they believed medical research usually or always advances science. More than 80 percent of the patients agreed that medical research does not involve unreasonable risks (86%). Nevertheless, some patients (9%) believed that research usually or always poses unreasonable risks to people. Controlling for multiple factors, the characteristics associated with holding this belief included being younger, being African-American, not having a college education, being in fair or poor general health, and not having any experience as a research participant. Seven percent of patients believed that participants in medical research are usually or always pressured into participating. Patients more likely to believe that people are pressured into research were African-American and had an annual income of less than $25,000.
Thirty-seven percent of patients believed that patients who participate in medical research are usually or always better off, medically, than similar patients who are not in medical research. Patients with a more positive view about research tended to be older, have incomes of less than $50,000 a year, and have had some experience as a research participant.
In the In-Depth Interviews, patients' general attitudes about research often seemed to be shaped by what their own research experiences had been, and patients generally had very positive things to say about their own experiences. Typically, they believed that the projects in which they were or had been participants had been explained thoroughly, that they had been treated kindly, and that they had received at least as much benefit as could have been expected. Moreover, the more experience people had with research, the more positive was their attitude. In addition, a few patients admitted that they had held a rather negative impression of research until they themselves had participated, at which time their impression changed. One respondent said, "I didn't know what to expect. In the beginning I was worried, you know. I was a little upset, a little frighten[ed] and everything. Once I got here, I found that the people were very nice, very professional, and they care about their patients . . . [Y]ou think that you are going to be a number, that they just may be cold and calculating, they['ll] just be thinking about just the data itself and you are just a number or something. But once I got here I found that . . . the doctors and nurses and everybody are very concerned about the individual and you find that out because they take the time to know your name." These findings are consistent with those from the Brief Survey, in which patients who currently were, or once had been, research participants had significantly more positive attitudes about research than those who had never participated.
When asked for their attitudes and beliefs about medical research generally (rather than about their own experiences), patients, again, had very positive things to say. Research was thought of as a promising endeavor, something that would advance knowledge and help other people: "[Research is] the only way advancement is made in the medical field particularly. . . [I]t's gotta be done at some point in time on human beings . . . and there are people who are alive today because of the people [who] did research projects." Another respondent strongly endorsed research activities: "Overall I have to say clinical trials, medical experiments are the only way we're going to find any type of results . . . because you can . . . practice on guinea pigs, monkeys, or whatever, but the only way you're going to find out if any of these drugs are going to work is you're going to have to do it on a human being." While patients articulated the necessity of conducting research, a few reiterated the importance of looking out for the interests of the human participants: "I think that . . . research is awfully important in all fields and . . . the more it involves human life the more guarded one has to be about it."
In the Brief Survey, patients were asked to compare the term medical research with one of four alternative terms: clinical trial, clinical investigation, medical study, or medical experiment. The term medical experiment evoked the most striking and negative associations. It was the only term to be evaluated as worse than the term medical research on all of the dimensions considered. Specifically, patients who were asked to compare medical experiments with medical research reported that patients in "medical experiments" were more likely to get unproven treatments and be at greater risk than patients in "medical research" and also that they were less likely to do better medically. By comparison, patients thought those in "medical research" were more likely than those in either "clinical investigations" or "clinical trials" to get unproven treatments and to be at greater risk, but they were more likely to do better medically. The term medical study got better ratings than the term medical research in every respect; medical studies were viewed as less risky, as less likely to involve unproven treatments, and as offering a greater chance at medical benefit.
Distinctions in meaning among different terms for biomedical research also emerged from the In-Depth Interview. Elaborating on the findings of the Brief Survey, the terms experiment and experimental, for the vast majority of participants, meant that something was unproven, untested, or in the first stage of testing and was thereby riskier and perhaps scary. Some patients said they would become a participant in an "experiment" only if they were terminally ill. A few participants described quite explicit images of what experiments involved: "I envision all kinds of weird things done to the body and I assume that's not true, but also I envision a medical experiment maybe . . . done in a laboratory sealed up somewhere where no one even knows what [is] going on." Another respondent said, "Medical experiment--almost sounds like Frankenstein to me." When asked to explain the term experiment, patients often invoked the term guinea pig to convey the sentiment of being the "victim" of an experiment. For example, one respondent, when asked to define the term medical experiment, said, "That's where you get down to the human guinea pig . . . where they may be injecting medication or whatever they want to inject in someone and watching them for a reaction."
In comparison with the term experiment, clinical trial and clinical investigation were not such evocative terms. Some patients gave hesitant or stumbling definitions or said they were not familiar with these terms. On the other hand, some patients did attach meaning to these terms, defining them as endeavors that were at an intermediate stage of inquiry, where researchers already know something about the topic and they are now trying the next step.
Patients were most likely to consider "study" a benign endeavor, akin to studying something in school: "Study brings to my mind more of using documentation for analysis. . . . With a study . . . you're looking at records. You look at past histories and so forth. . . . It is mostly paperwork, documents, or the books and things."
Of the four terms offered, patients usually said they would prefer to be in a study. It was reported to be the least harmful because it was believed to be the least invasive. In comparison to experiments, which many patients believed involved "trying things out" on animals and/or humans, "studies," they felt, usually entailed gathering information and reviewing paperwork.
Personal Experience With Research
Thirty percent (570) of the 1,882 patients interviewed reported that they were or had been participants in research (see table 4). We were able to review records or consult other sources for 541 of these 570 cases. By these reviews, we were able to confirm research participation in 302 of 541 cases (56%). In another 203 of the 541 cases (38%), we were unable to find documentation to suggest whether or not the patient was participating or had participated in research. In the remaining 36 cases, the review by health professionals on the Advisory Committee staff concluded that these patients were probably in error and that they were not, indeed, research participants. In summary, 16 percent (302 of 1,882) of the total sample, consistent with their reports, were former or current research participants. Also, assuming that most of the patients for whom research participation could not be verified but, consistent with their own reports were probably truly former or current participants (11%, or 203 of 1,882), then a total of 27 percent of the Brief Survey respondents were former or current research participants. By contrast, 2 percent of the total sample (36 of 1,882), were likely incorrect in their perception of themselves as being participants in research.