Informed consent in randomised controlled trials: development and preliminary evaluation of a measure of Participatory and Informed Consent (PIC).

BACKGROUND: Informed consent (IC) is an ethical and legal prerequisite for trial participation, yet current approaches evaluating participant understanding for IC during recruitment lack consistency. No validated measure has been identified that evaluates participant understanding for IC based on their contributions during consent interactions. This paper outlines the development and formative evaluation of the Participatory and Informed Consent (PIC) measure for application to recorded recruitment appointments. The PIC allows the evaluation of recruiter information provision and evidence of participant understanding. METHODS: Published guidelines for IC were reviewed to identify potential items for inclusion. Seventeen purposively sampled trial recruitment appointments from three diverse trials were reviewed to identify the presence of items relevant to IC. A developmental version of the measure (DevPICv1) was drafted and applied to six further recruitment appointments from three further diverse trials to evaluate feasibility, validity, stability and inter-rater reliability. Findings guided revision of the measure (DevPICv2) which was applied to six further recruitment appointments as above. RESULTS: DevPICv1 assessed recruiter information provision (detail and clarity assessed separately) and participant talk (detail and understanding assessed separately) over 20 parameters (or 23 parameters for three-arm trials). Initial application of the measure to six diverse recruitment appointments demonstrated promising stability and inter-rater reliability but a need to simplify the measure to shorten time for completion. The revised measure (DevPICv2) combined assessment of detail and clarity of recruiter information and detail and evidence of participant understanding into two single scales for application to 22 parameters or 25 parameters for three-arm trials. Application of DevPICv2 to six further diverse recruitment appointments showed considerable improvements in feasibility (e.g. time to complete) with good levels of stability (i.e. test-retest reliability) and inter-rater reliability maintained. CONCLUSIONS: The DevPICv2 provides a measure for application to trial recruitment appointments to evaluate quality of recruiter information provision and evidence of patient understanding and participation during IC discussions. Initial evaluation shows promising feasibility, validity, reliability and ability to discriminate across a range of recruiter practice and evidence of participant understanding. More validation work is needed in new clinical trials to evaluate and refine the measure further.

Understanding of regional variation in the use of surgery.

The use of common surgical procedures varies widely across regions. Differences in illness burden, diagnostic practices, and patient attitudes about medical intervention explain only a small degree of regional variation in surgery rates. Evidence suggests that surgical variation results mainly from differences in physician beliefs about the indications for surgery, and the extent to which patient preferences are incorporated into treatment decisions. These two components of clinical decision making help to explain the so-called surgical signatures of specific procedures, and why some consistently vary more than others. Variation in clinical decision making is, in turn, affected by broad environmental factors, including technology diffusion, supply of specialists, local training frameworks, financial incentives, and regulatory factors, which vary across countries. Better scientific evidence about the comparative effectiveness of surgical and non-surgical interventions could help to mitigate regional variation, but broader dissemination of shared decision aids will be essential to reduce variation in preference-sensitive disorders.

Strategies to reduce variation in the use of surgery.

Provision rates for surgery vary widely in relation to identifiable need, suggesting that reduction of this variation might be appropriate. The definition of unwarranted variation is difficult because the boundaries of acceptable practice are wide, and information about patient preference is lacking. Very little direct research evidence exists on the modification of variations in surgery rates, so inferences must be drawn from research on the alteration of overall rates. The available evidence has large gaps, which suggests that some proposed strategies produce only marginal change. Micro-level interventions target decision making that affects individuals, whereas macro-level interventions target health-care systems with the use of financial, regulatory, or incentivisation strategies. Financial and regulatory changes can have major effects on provision rates, but these effects are often complex and can include unintended adverse effects. The net effects of micro-level strategies (such as improvement of evidence and dissemination of evidence, and support for shared decision making) can be smaller, but better directed. Further research is needed to identify what level of variation in surgery rates is appropriate in a specific context, and how variation can be reduced where desirable.

Evaluation and stages of surgical innovations.

Surgical innovation is an important part of surgical practice. Its assessment is complex because of idiosyncrasies related to surgical practice, but necessary so that introduction and adoption of surgical innovations can derive from evidence-based principles rather than trial and error. A regulatory framework is also desirable to protect patients against the potential harms of any novel procedure. In this first of three Series papers on surgical innovation and evaluation, we propose a five-stage paradigm to describe the development of innovative surgical procedures.

No surgical innovation without evaluation: the IDEAL recommendations.

Surgery and other invasive therapies are complex interventions, the assessment of which is challenged by factors that depend on operator, team, and setting, such as learning curves, quality variations, and perception of equipoise. We propose recommendations for the assessment of surgery based on a five-stage description of the surgical development process. We also encourage the widespread use of prospective databases and registries. Reports of new techniques should be registered as a professional duty, anonymously if necessary when outcomes are adverse. Case series studies should be replaced by prospective development studies for early technical modifications and by prospective research databases for later pre-trial evaluation. Protocols for these studies should be registered publicly. Statistical process control techniques can be useful in both early and late assessment. Randomised trials should be used whenever possible to investigate efficacy, but adequate pre-trial data are essential to allow power calculations, clarify the definition and indications of the intervention, and develop quality measures. Difficulties in doing randomised clinical trials should be addressed by measures to evaluate learning curves and alleviate equipoise problems. Alternative prospective designs, such as interrupted time series studies, should be used when randomised trials are not feasible. Established procedures should be monitored with prospective databases to analyse outcome variations and to identify late and rare events. Achievement of improved design, conduct, and reporting of surgical research will need concerted action by editors, funders of health care and research, regulatory bodies, and professional societies.

Challenges in evaluating surgical innovation.

Research on surgical interventions is associated with several methodological and practical challenges of which few, if any, apply only to surgery. However, surgical evaluation is especially demanding because many of these challenges coincide. In this report, the second of three on surgical innovation and evaluation, we discuss obstacles related to the study design of randomised controlled trials and non-randomised studies assessing surgical interventions. We also describe the issues related to the nature of surgical procedures-for example, their complexity, surgeon-related factors, and the range of outcomes. Although difficult, surgical evaluation is achievable and necessary. Solutions tailored to surgical research and a framework for generating evidence on which to base surgical practice are essential.

Preferences for endovascular (EVAR) or open surgical repair among patients with abdominal aortic aneurysms under surveillance.

OBJECTIVES: There is no evidence about patient preferences for treatment of abdominal aortic aneurysms (AAA) by endovascular aneurysm repair (EVAR) or open surgical repair (OSR). This study examined patient preferences for elective future aneurysm repair and factors that may influence such preferences. METHODS: Patients with small AAAs under ultrasound scan surveillance at two United Kingdom (UK) hospitals participated in a semi-structured telephone interview. Features of the two techniques were assessed with regard to their influence on the preferences of participants for EVAR or OSR, using a Likert scale. In addition, participants ranked the relative importance of 14 features against each other. RESULTS: Fifty-six out of 100 eligible participants (56%) completed the semi-structured telephone interview. Of those, 84% (47 patients) said they would prefer a future EVAR repair. Patients who expressed a preference for OSR were significantly younger. Risks of major organ failure and death were most commonly judged as important features in influencing patient preference (Likert scale score 5/5). Risk of death was also most frequently ranked above all other features. Postoperative morbidity and mortality were regarded by patients as more important than the need for surveillance and risk of long-term problems with EVAR. Type of incision and radiation exposure were both given low Likert scale scores of 1/5, and the risk of sexual dysfunction was most frequently ranked as the least important feature of either operation, out of 14 other features. CONCLUSION: When presented with detailed information about EVAR and OSR, most patients with small aneurysms would prefer EVAR.