Jenny Hewison

The ethics of randomised controlled trials from the perspectives of patients, the public, and healthcare professionals

Since the introduction of randomised controlled trials, professionals and lay people alike have worried over whether doing this sort of experiment in humans is ethical. It has been argued that participants may be called to sacrifice their own best interests for the benefit of future patients.1 The scientific rationale for conducting a trial rests in collective equipoise, which means that the medical community as a whole is genuinely uncertain over which treatment is best. The key point, however, is that future patients benefit at no cost to participants, provided that participants are in personal equipoise and give informed consent on this basis. In these circumstances, the trial arms are an equally good bet prospectively.2 ### Summary points To find out what patients, the general public, and healthcare professionals thought about trials, we undertook a review of the ethics of randomised controlled trials from these perspectives as part of a broader review relating to the ethics of designing and conducting clinical trials.1 We searched BIDS, Medline, and Psychlit (for strategy see the BMJ website). There were 61 studies on attitudes …

The impact of head and neck cancer and facial disfigurement on the quality of life of patients and their partners

Psychological and physical stresses from head and neck cancer can be substantial for patients and partners. There is minimal research exploring treatment impact, particularly facial disfigurement after surgery.

The Diabetes Health Profile (DHP): a new instrument for assessing the psychosocial profile of insulin requiring patients--development and psychometric evaluation.

The aim of the studies was to evaluate the psychometric properties and construct validity of the Diabetes Health Profile (DHP-1). Content for the DHP-1 was derived following in-depth interviews with 25 insulin dependent and insulin requiring patients, a review of the literature and discussions with health care professionals. Initial analysis of the factor structure of the DHP-1 was carried out on the responses of 239 insulin dependent and insulin requiring patients, with a mean age of 40.85 years (SD=13.0), resulting in a 43 item three factor solution. The 43 item version of the DHP-1 was completed by 2,239 insulin dependent/requiring patients (mean age = 39.8, SD=10) years. Fifty-one per cent were men. A forced three factor Principal Factoring Analysis with varimax rotation was carried out. Eleven items were excluded with item factor cross loadings >0.30 or item factor loadings <0.30. PAF analysis of the 32 items resulted in a three factor solution accounting for 33% of the total explained variance. The three factors were interpreted as Psychological Distress, Barriers to Activity and Disnhibited Eating. Factor congruence between subsamples were: Psychological distress (0.93), Barriers to Activity (0.93) and Disinhibited Eating (0.99). Coefficients of congruence between men and women were 0.94, 0.92 and 0.99 for Psychological Distress, Barriers to Activity and Disinhibited Eating respectively. Internal consistency of the three factors (Cronbachs α) were: Psychological Distress (0.86), Barriers to Activity (0.82), and Disinhibited Eating (0.77). Construct-convergent validity was investigated on a sample of 233 insulin dependent and insulin requiring patients (mean age = 51.46 years). Psychological Distress and Barriers to Activity subscales correlated with the Hospital Depression and Anxiety Scale = 0.50 to 0.62, p<0.01 and subscales of the SF-36 (range: r=−0.17 to -0.62, p<0.01). These findings lend support to the construct validity and reliability of the DHP-1 and that it is suitable for further development.

Informed consent for clinical trials: in search of the "best" method

OBJECTIVE To review the literature on comparisons between different methods of obtaining informed consent for clinical trials. DESIGN Eight hundred and twelve articles were traced, in the process of conducting a systematic review of the ethics of clinical trials, by searching a number of sources: bibliographic databases (Medline, Psychlit and BIDS science and social science indices), hand searches, personal contacts, an original collection and a systematic follow-up of reference lists. Fourteen research reports were found which provided comparative data on different methods of obtaining informed consent. Eleven of these used a randomised design. Studies were classified according to three outcome measures (anxiety, consent rate and understanding). RESULTS The results of the various studies suggest that giving people more information and more time to reflect tends to be associated with a lower consent rate. There seems to be an optimal level of information about side-effects such that patients are not overburdened by detail, while grasping the most important risks. More information in general is associated with greater awareness of the research nature of the trial, voluntariness of participation, right to withdraw and (available) alternative treatments. This result does not, however, extend to explanations of the concept of randomisation on which the literature is contradictory--sometimes more information is associated with increased understanding of the concept and sometimes it is not. Although divulging less information seems to be associated with less anxiety, there is evidence of an interaction with knowledge--high levels of knowledge are significantly associated with less anxiety, irrespective of consent method. The more that patients know before they are invited to participate in a trial, the better equipped they are to cope with the informed consent procedure. CONCLUSION There is some evidence to suggest that there is an optimal amount of information which enhances patient understanding and which might, in turn, reduce anxiety. However. the studies were not altogether conclusive. More work needs to be carried out, especially on public understanding of science and on how different ways of explaining scientific concepts affect that understanding.

Development of a symptom based outcome measure for asthma.

Measuring symptom specific health outcome is complex, but the methodologies now exist to develop measures with the appropriate properties. As one element of a major programme to develop multidomain health outcome measures for chronic disease, a symptom based measure for asthma care has been developed for use in general practice and outpatient departments. This article outlines the development process, which used a framework recently described in the theoretical literature to show the constraints that scientific criteria place on the development of outcome measures and the means of overcoming such limiting factors. Although substantial effort is required to undertake a rigorous process of development, useful tools are the result. Two five item, symptom based outcome measures for adult asthma are described.

Understanding why decision aids work: linking process with outcome

Decision aids help patients make treatment choices. There is little empirical evidence to explain how they work. The results from this randomised controlled trial comparing routine with decision-aided consultations in the prenatal diagnosis for Downs syndrome context are used to describe the strategies employed during decision making, to assess the impact of a decision aid on decision processes, and to investigate decision process and outcome associations. Data were elicited from two content analyses of consultation transcripts and questionnaires assessing knowledge, anxiety, decisional conflict, reasons, and information usefulness. 68/106 women completed measures at consultation and follow-up. Decision-aided women employed more cognitive and emotional strategies during decision making. More negative evaluations during decision making were associated with better outcomes. Decision-aided consultations facilitated the employment of strategies associated with more effective choices. These consultations take longer and elicit greater expressions of negative affect, so may be less rewarding encounters for health professionals.

Ultrasound screening for chromosomal abnormality: Women's reactions to false positive results

Objective. Ultrasound scans have become a routine part of antenatal care. Recent developments have meant that ultrasound has gone beyond monitoring the pregnancy and diagnosis of major abnormalities. Its routine use now includes screening for subtle fetal abnormalities and indications of chromosomal abnormality. Unfortunately ultrasound screening for chromosomal abnormalities has a low positive predictive value (similar to maternal serum screening), and only about 2% of pregnancies identified as ‘high risk’ will be diagnosed with a chromosomal abnormality. The other 98% of ‘high risk’ results are termed ‘false positive’. The aim of this paper is to elucidate the range of subjective experiences of women in response to having these results. Method. The approach employed was interpretative phenomenological analysis. Semi-structured interviews were carried out with 24 pregnant women after ‘false positive’ ultrasound results. These were subjected to a qualitative analysis. Results. Participants perceived the ultrasound scan primarily as a social, non-medical event and were unprepared for the result the scan elicited. Two-thirds described residual feelings of anxiety even after normal diagnostic results were known, sometimes attributed to continued fears about fetal abnormality, but more often attributed to a generalized feeling that something else unexpected could happen to threaten the pregnancy. Conclusions. The paper has pointed to important discrepancies in the experiences of women undergoing ultrasound screening and the aims of informed choice. We would suggest consideration should be given to providing information on both the medical purposes of screening and on possible outcomes from it. The study also points to the possible need for counselling support for the majority of women who go on to receive confirmation of a negative diagnosis, counselling addressed at helping women to readjust to having a ‘low risk’ pregnancy.

Prenatal Diagnosis for Inherited Deafness—What is the Potential Demand?

Genetic testing for inherited deafness is now available within some genetics centres. This study used a structured questionnaire to assess the potential uptake of prenatal diagnosis (PND) for inherited deafness, and document the opinions of deaf and hearing individuals toward PND and termination of pregnancy (TOP) for hearing status. Participants were self-selected from the whole of the UK, of whom 644 were deaf, 143 were hard of hearing or deafened, and 527 were hearing individuals who had either a deaf parent or child. The results showed that 21% of deaf, 39% of hard of hearing and deafened, and 49% of hearing participants said they would consider PND for deafness. Six percent of deaf, 11% of hard of hearing and deafened, and 16% of hearing participants said they would consider a TOP if the fetus was found to be deaf. Two percent of deaf participants said they would prefer to have deaf children and would consider a TOP if the fetus was found to be hearing.

How should we measure informed choice? The case of cancer screening

Informed choice is increasingly recognised as important in supporting patient autonomy and ensuring that people are neither deceived nor coerced. In cancer screening the emphasis has shifted away from just promoting the benefits of screening to providing comprehensive information to enable people to make an informed choice. Cancer screening programmes in the UK now have policies in place which state that it is their responsibility to ensure that individuals are making an individual informed choice. There is a need to evaluate whether such policies mean that those people invited for screening are making informed choices, and how comprehensive information affects other variables such as uptake, cost effectiveness, and satisfaction. At the present time, there is no validated measure of informed choice in cancer screening. Such a measure could be used to evaluate the effectiveness of interventions to increase informed choice and levels of informed choice in a population invited for screening. It could encourage health professionals to be accountable. Factors important when measuring informed choice in cancer screening include an individual’s understanding of the limitations of screening, the ability to make an autonomous choice, and the difference between choice and behaviour.

Disagreement in Patient and Carer Assessment of Functional Abilities After Stroke

BACKGROUND AND PURPOSE The aims of this study were to describe differences between functional ability assessments made by stroke patients and their informal carers and to investigate the psychological associates of the difference in assessments. METHODS A prospective design was used, with repeated assessments of function, mood, and carer strain. Thirty hospital stroke patients and their main carer were interviewed 3 times: within 1 month of stroke, 1 month after discharge, and 6 months after discharge. RESULTS There were significant differences between patient and carer assessments at all 3 time points, with patient self-assessment less disabled than carer assessment (at least P<0.02). The disagreement in assessment was unrelated to patient or carer mood (P>0.05) but greater disagreement was associated with greater carer strain (P<0.05). The source of the disagreement in functional ability assessment remains unclear. CONCLUSIONS The method of assessment affects the rating of functional abilities after stroke. Carer strain is potentially increased when the patient or carer makes an unrealistic assessment of the patients level of independence.