Patients' satisfaction in clinical trials

Authors


L. Terenius, Department of Clinical Neuroscience, Experimental Alcohol and Drug Addiction Research Section, Karolinska Institutet, S-171 76 Stockholm, Sweden (fax: +46 8 341939; e-mail: lars.terenius@cmm.ki.se).

Medical progress is dependent on new treatment or diagnostic protocols, new drugs or drug regimens. There is hope that the soon completed sequence of nucleotides in the human genome will generate a large number of potential new drug targets. This wealth of new information, as well as the sensitive nature of animal experimentation, may increase the need for clinical trials. The conduct of clinical trials requires adequate time to devote to the patients participating in the trial. However, clinical research is increasingly pressured by the demands on doctors interested in and capable of conducting clinical trials. It has even been debated whether there are enough clinical scientists and whether those who technically might be available are adequately trained. Although it is hard to define the ideal proportions between laboratory science and clinical science, the factors mentioned all work in the direction of potentially limiting clinical research [1].

Ethical considerations of clinical trials weigh the benefits and hazards the patients are subjected to. The trial should be motivated from a treatment or diagnostic perspective, but also scientifically, i.e. the increase of new knowledge. Patients are protected in the protocols approved by the ethical committees. Still, a clinical trial may induce discomfort or risks, particularly when the treatment principle is new and not yet investigated. There is an obvious risk that changes in the biomedical perspective as a result of the new developments in genomics and functional genomics will increase the demands on the competence of the clinical scientist. He/she may be enthralled by the enthusiasms expressed by the laboratory investigator, who is anxious to see his ideas tested, and not be aware of potential hazards in terms of side-effects. Likewise, competence in the ethical committees, which is essential for the responsible development of clinical science, needs continued attention.

The recruitment of eligible patients for a clinical trial must obviously be based on a trustful relationship with the clinician. It is important that patients participating in a trial are representative of the patient population at large. It is therefore essential that the patients are adequately informed about the purpose of the trial and its predicted outcome in terms of new knowledge. Patient recruitment in clinical trials has been studied by several authors and reasons for declining to participate may vary from ‘no time’ to ‘not interested’.

A paper in this journal issue [2] analyses the level of satisfaction in patients who participated in two different clinical trials, a trial of interferon-2α versus a standard treatment with predinsolone in ulcerative colitis, and a study of MRI, a noninvasive imaging technique for the evaluation of inflammatory disease. The two studies typify a therapeutic and a diagnostic trial, respectively. The level of discomfort was significantly greater in the therapeutic study. Interestingly, the level of satisfaction was similar in the two groups. When asked what made them accept to participate in the trial the patients explained that their participation would increase medical knowledge and eventually lead to better treatment (either for personal benefit or for the help of others). Although most patients expressed unrealistic reasons for participation, all expressed satisfaction with becoming more informed and being better cared for. They also valued continuity (seeing one doctor). This is interesting information, which suggests that in a clinical trial a control or comparison group may not be comparable to the average treated patient. This bias is perhaps unavoidable, but certainly a caveat for clinical trials where the psychic condition of the patient will play a marked role for the treatment outcome. Attitudes to blinding and randomization were mixed with about an equal number of participating patients accepting and not accepting. The study has limitations in that it is not known what proportion of eligible patients participated in the two studies. The patients who actually participated might be a subgroup who had positive attributes or expectancies from participating. It would have been interesting to know how many and why some patients declined to participate, particularly whether there was low motivation. It has been observed, but not well documented, that the willingness to participate in clinical trials has been falling in recent years.

In general, the present results are in line with previous similar studies. In a paper by Cassileth et al. in 1982 [3] over 70% of patients in two clinical studies and a group of healthy volunteers responded to a questionnaire about their attitude to clinical trials. The general contention was that those who participate in clinical studies make an important contribution to society. Patients asked what their main reason for participating in a clinical study would be: over 50% responded better medical care, whereas about 25% responded ‘to contribute to medical knowledge’.

Verheggen et al. [4] used criteria to evaluate medicotechnical aspects of satisfaction in 21 clinical studies and in addition the interrelational and organizational aspects. An elaborate statistical analysis indicated high satisfaction with the medical treatment, but as many as 70% indicated that they had had even greater expectations and only 30% were positively surprised.

Patient satisfaction cannot be calculated beforehand. The importance of having a high satisfaction rate is many-fold. A satisfied patient is the best advocate for future clinical studies. Patients should be given a realistic view of what the clinical trial could mean for their own health. A follow-up, as reported by these three studies, is in line with other initiatives for treatment quality control. It may also serve the purpose of showing that the doctor cares.

Received 11 September 2000; accepted 13 September 2000.

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