Richard Edlin

Is it really possible to build a bridge between cost-benefit analysis and cost-effectiveness analysis?

Cost-benefit analysis (CBA) is a recognised as the economic evaluation technique that accords most with the underlying principles of standard welfare economic theory. However, due to problems associated with the technique, economists evaluating resources allocation decisions in health care have most often used cost-effective analysis (CEA), in which health benefits are expressed in non-monetary units. As a result, attempts have been made to build a welfare economic bridge between cost-benefit analysis (CBA) and cost-effectiveness analysis (CEA). In this paper, we develops these attempts and finds that, while assumptions can be made to facilitate a constant willingness-to-pay per unit of health outcome, these restrictions are highly unrealistic. We develop an impossibility theorem that shows it is not possible to link CBA and CEA if: (i) the axioms of expected utility theory hold; (ii) the quality-adjusted life-year (QALY) model is valid in a welfare economic sense; and (iii) illness affects the ability to enjoy consumption. We conclude that, within a welfare economic framework, it would be unwise to rely on a link between CBA and CEA in economic evaluations.

Access with evidence development schemes: a framework for description and evaluation.

There is an inevitable tension between robust reimbursement processes and providing speedy access to new and novel technologies, given uncertainties about key pieces of evidence and subsequent concerns regarding their overall efficiency. The public perception of these treatments as ‘breakthrough’, combined with substantial clinical pressure, has led to healthcare payers looking for schemes that allow the new technology to be made available to (some) patients, while (at least partially) protecting the principles of their reimbursement decision-making processes. Current literature on these schemes is almost completely descriptive and provides little help in planning future schemes. We propose a framework for evaluating current schemes and informing the design of future schemes. We examine the value of the framework using the UK Multiple Sclerosis Risk-Sharing Scheme as a case study.

Effectiveness of emergency care practitioners working within existing emergency service models of care

Background: An emergency care practitioner (ECP) is a generic practitioner drawn mainly from paramedic and nursing backgrounds. ECPs receive formal training and extended clinical skills to equip them to work as an integral part of the healthcare team working within and across traditional boundaries of emergency and unplanned care. Currently, ECPs are working in different healthcare settings in the UK. Objectives: (1) To evaluate appropriateness, satisfaction and cost of ECPs compared with the usual service available in the same healthcare setting, (2) to increase understanding of what effect, if any, ECPs are having on delivery of health services locally and (3) to evaluate whether ECP working yields cost savings. Methods: Using a mixed-methods approach, data were collected quantitatively and qualitatively from three different types of health provider setting where ECPs are operational, in three areas of England. Data were collected by sending two questionnaires to each patient eligible to be seen by an ECP, at 3 and 28 days after presentation; telephone interviews were conducted with a sample of staff that included ECPs, other health professionals and stakeholders (eg, managers) in each of the three settings; and routine data were analysed to provide a perspective on costs. Results: After adjusting for age, sex, presenting complaint and service model, some differences in the processes of care between the ECPs and the usual providers in the three settings were observed. Overall, ECPs carried out fewer investigations, provided more treatments and were more likely to discharge patients home than the usual providers. Patients were satisfied with the care received from ECPs, and this was consistent across the three different settings. It was found that ECPs are working in different settings across traditional professional boundaries and are having an impact on reconfiguring how those services are delivered locally. Costs information (based on one site only) indicated that ECP care may be cost effective in that model of ECP working. Conclusion: Care provided by ECPs appears to reduce the need for subsequent referral to other emergency and unscheduled care services in a large proportion of cases. We found no evidence that the care provided by an ECP was less appropriate than the care by the usual providers for the same type of health problem.

Effect of Robotic-Assisted vs Conventional Laparoscopic Surgery on Risk of Conversion to Open Laparotomy Among Patients Undergoing Resection for Rectal Cancer: The ROLARR Randomized Clinical Trial

Importance Robotic rectal cancer surgery is gaining popularity, but limited data are available regarding safety and efficacy. Objective To compare robotic-assisted vs conventional laparoscopic surgery for risk of conversion to open laparotomy among patients undergoing resection for rectal cancer. Design, Setting, and Participants Randomized clinical trial comparing robotic-assisted vs conventional laparoscopic surgery among 471 patients with rectal adenocarcinoma suitable for curative resection conducted at 29 sites across 10 countries, including 40 surgeons. Recruitment of patients was from January 7, 2011, to September 30, 2014, follow-up was conducted at 30 days and 6 months, and final follow-up was on June 16, 2015. Interventions Patients were randomized to robotic-assisted (n = 237) or conventional (n = 234) laparoscopic rectal cancer resection, performed by either high (upper rectum) or low (total rectum) anterior resection or abdominoperineal resection (rectum and perineum). Main Outcomes and Measures The primary outcome was conversion to open laparotomy. Secondary end points included intraoperative and postoperative complications, circumferential resection margin positivity (CRM+) and other pathological outcomes, quality of life (36-Item Short Form Survey and 20-item Multidimensional Fatigue Inventory), bladder and sexual dysfunction (International Prostate Symptom Score, International Index of Erectile Function, and Female Sexual Function Index), and oncological outcomes. Results Among 471 randomized patients (mean [SD] age, 64.9 [11.0] years; 320 [67.9%] men), 466 (98.9%) completed the study. The overall rate of conversion to open laparotomy was 10.1%: 19 of 236 patients (8.1%) in the robotic-assisted laparoscopic group and 28 of 230 patients (12.2%) in the conventional laparoscopic group (unadjusted risk difference = 4.1% [95% CI, −1.4% to 9.6%]; adjusted odds ratio = 0.61 [95% CI, 0.31 to 1.21]; P = .16). The overall CRM+ rate was 5.7%; CRM+ occurred in 14 (6.3%) of 224 patients in the conventional laparoscopic group and 12 (5.1%) of 235 patients in the robotic-assisted laparoscopic group (unadjusted risk difference = 1.1% [95% CI, −3.1% to 5.4%]; adjusted odds ratio = 0.78 [95% CI, 0.35 to 1.76]; P = .56). Of the other 8 reported prespecified secondary end points, including intraoperative complications, postoperative complications, plane of surgery, 30-day mortality, bladder dysfunction, and sexual dysfunction, none showed a statistically significant difference between groups. Conclusions and Relevance Among patients with rectal adenocarcinoma suitable for curative resection, robotic-assisted laparoscopic surgery, as compared with conventional laparoscopic surgery, did not significantly reduce the risk of conversion to open laparotomy. These findings suggest that robotic-assisted laparoscopic surgery, when performed by surgeons with varying experience with robotic surgery, does not confer an advantage in rectal cancer resection. Trial Registration isrctn.org Identifier: ISRCTN80500123

Total hip arthroplasty versus resurfacing arthroplasty in the treatment of patients with arthritis of the hip joint: single centre, parallel group, assessor blinded, randomised controlled trial

Objectives To compare the clinical and cost effectiveness of total hip arthroplasty with resurfacing arthroplasty in patients with severe arthritis of the hip. Design Single centre, two arm, parallel group, assessor blinded, randomised controlled trial with 1:1 treatment allocation. Setting One large teaching hospital in the United Kingdom. Participants 126 patients older than 18 years with severe arthritis of the hip joint, suitable for resurfacing arthroplasty of the hip. Patients were excluded if they were considered to be unable to adhere to trial procedures or complete questionnaires. Interventions Total hip arthroplasty (replacement of entire femoral head and neck); hip resurfacing arthroplasty (replacement of the articular surface of femoral head only, femoral neck remains intact). Both procedures replaced the articular surface of the acetabulum. Main outcome measures Hip function at 12 months after surgery, assessed using the Oxford hip score and Harris hip score. Secondary outcomes were quality of life, disability rating, physical activity level, complications, and cost effectiveness. Results 60 patients were randomly assigned to hip resurfacing arthroplasty and 66 to total hip arthroplasty. Intention to treat analysis showed no evidence for a difference in hip function between treatment groups at 12 months (t test, P=0.242 and P=0.070 for Oxford hip score and Harris hip score, respectively); 95% of follow-up data was available for analysis. Mean Oxford hip score was 40.4 (95% confidence interval 37.9 to 42.9) in the resurfacing group and 38.2 (35.3 to 41.0) in the total arthroplasty group (estimated treatment effect size 2.23 (−1.52 to 5.98)). Mean Harris hip score was 88.4 (84.4 to 92.4) in the resurfacing group and 82.3 (77.2 to 87.5) in the total arthroplasty group (6.04 (−0.51 to 12.58)). Although we saw no evidence of a difference, we cannot definitively exclude clinically meaningful differences in hip function in the short term. Overall complication rates did not differ between treatment groups (P=0.291). However, we saw more wound complications in the total arthroplasty group (P=0.056) and more thromboembolic events in the resurfacing group (P=0.049). Conclusions No evidence of a difference in hip function was seen in patients with severe arthritis of the hip, one year after receiving a total hip arthroplasty versus resurfacing arthroplasty. The long term effects of these interventions remain uncertain. Trial registration Current Controlled Trials ISRCTN33354155, UKCRN 4093.

A cluster randomised controlled trial of a web based decision aid to support parents' decisions about their child's Measles Mumps and Rubella (MMR) vaccination

Highlights • The use of decision aids for immunisation decisions is under researched and controversial.• Parents receiving a decision aid or a leaflet had reduced decisional conflict for the MMR decision.• MMR uptake in the decision aid and control arms achieved levels required for population immunity.• Leaflet arm parents were less likely to vaccinate their child.• Childhood immunisation decision aids can achieve both informed decision-making and uptake.

Examining the attitudes and preferences of health care decision-makers in relation to access, equity and cost-effectiveness: A discrete choice experiment

OBJECTIVES To describe the views of health care decision-makers and providers operating in the UK National Health Service (NHS) concerning the concepts of cost-effectiveness, equity and access through a series of attitudinal questions; to evaluate the preferences of health care providers in relation to each of these concepts using a discrete choice experiment (DCE); to assess the impact of prior completion of an attitude questionnaire on preferences elicited through a DCE. METHOD Three versions of a DCE questionnaire were developed with and without a series of attitudinal questions and randomly distributed to 1456 health care decision-makers and providers. The questionnaire sought to elicit their preferences between the competing objectives of cost-effectiveness, equity and access within the context of different hypothetical, specialist treatment programmes for cardiovascular disease. RESULTS The response rate was 26%. Female respondents exhibited a stronger preference than males for reducing health inequalities by targeting the worst off (Wald test, P<0.001). Primary Care Trusts (PCTs), Strategic Health Authorities (SHA) or Department of Health (DoH) staff were also more likely than hospital managers to favour programmes that targeted the worst off (Wald test, P<0.001 in each case). Those who were clinically trained and currently in a clinical post had a stronger preference for programmes with shorter waiting times compared to those in a managerial or non-clinical posts, who exhibited stronger preferences for equity. Completion of a series of attitudinal questions prior to completing the DCE task resulted in a lower proportion of dominant responses and an increased willingness to make trade-offs between attributes.

Principles of Design of Access with Evidence Development Approaches: A Consensus Statement from the Banff Summit

Healthcare payers are increasingly entering into innovative reimbursement agreements to manage the tension between funding new but expensive treatments, and obtaining value (measured in terms of clinical effectiveness, improved quality of care, health-related quality of life [HR-QOL], etc.) for money. These often commit substantial resources, and thus impose a significant opportunity cost on healthcare systems. Commentators and stakeholders are increasingly concerned that not all such approaches represent good value for money. However, there is little if any literature providing guidance on their design or evaluation. These reimbursement mechanisms have been given many names, such as ‘risk sharing’, ‘coverage with evidence development’, ‘field evaluations’ and ‘health impact guarantees’. We used the umbrella term ‘access with evidence development’ approaches (AEDs) to ensure discussions focussed upon the substantial issues common to all of these rather than their differences. The purpose of the Banff Summit (Alberta, Canada; 22–23 February 2009) was to bring together experts from around the world with direct experience of designing and implementing approaches to access with the aim of identifying principles to inform their design. The programme consisted of a presentation of findings from a review of the published literature and, drawing on that literature, a proposed checklist to describe and potentially evaluate such schemes. Participants from the US, the UK, Australia and Canadian provinces (British Columbia, Ontario and Alberta) gave presentations on their experience of AED approaches. Each talk was followed by an open discussion of the lessons that could be drawn from this experience. The final session considered whether it was possible to identify principles of good design in AED schemes. The Summit organizers were charged with producing a written statement of these principles for circulation to all participants. Each individual was then invited to sign up to the statement. We provide here the consensus statement from the summit. This is followed by the literature review completed in preparation for the meeting, a paper that outlines the descriptive framework (which was developed for the Summit) and an evaluation of an AED model implemented in the UK NHS using this framework. In addition, experts from the US and the UK write about the future development of AEDs in their respective healthcare systems and consider how adherence to the principles set out in the consensus statement would improve current and future AEDmodels.

Do incentives, reminders or reduced burden improve healthcare professional response rates in postal questionnaires? two randomised controlled trials.

BackgroundHealthcare professional response rates to postal questionnaires are declining and this may threaten the validity and generalisability of their findings. Methods to improve response rates do incur costs (resources) and increase the cost of research projects. The aim of these randomised controlled trials (RCTs) was to assess whether 1) incentives, 2) type of reminder and/or 3) reduced response burden improve response rates; and to assess the cost implications of such additional effective interventions.MethodsTwo RCTs were conducted. In RCT A general dental practitioners (dentists) in Scotland were randomised to receive either an incentive; an abridged questionnaire or a full length questionnaire. In RCT B non-responders to a postal questionnaire sent to general medical practitioners (GPs) in the UK were firstly randomised to receive a second full length questionnaire as a reminder or a postcard reminder. Continued non-responders from RCT B were then randomised within their first randomisation to receive a third full length or an abridged questionnaire reminder. The cost-effectiveness of interventions that effectively increased response rates was assessed as a secondary outcome.ResultsThere was no evidence that an incentive (52% versus 43%, Risk Difference (RD) -8.8 (95%CI −22.5, 4.8); or abridged questionnaire (46% versus 43%, RD −2.9 (95%CI −16.5, 10.7); statistically significantly improved dentist response rates compared to a full length questionnaire in RCT A. In RCT B there was no evidence that a full questionnaire reminder statistically significantly improved response rates compared to a postcard reminder (10.4% versus 7.3%, RD 3 (95%CI −0.1, 6.8). At a second reminder stage, GPs sent the abridged questionnaire responded more often (14.8% versus 7.2%, RD −7.7 (95%CI −12.8, -2.6). GPs who received a postcard reminder followed by an abridged questionnaire were most likely to respond (19.8% versus 6.3%, RD 8.1%, and 9.1% for full/postcard/full, three full or full/full/abridged questionnaire respectively). An abridged questionnaire containing fewer questions following a postcard reminder was the only cost-effective strategy for increasing the response rate (£15.99 per response).ConclusionsWhen expecting or facing a low response rate to postal questionnaires, researchers should carefully identify the most efficient way to boost their response rate. In these studies, an abridged questionnaire containing fewer questions following a postcard reminder was the only cost-effective strategy. An increase in response rates may be explained by a combination of the number and type of contacts. Increasing the sampling frame may be more cost-effective than interventions to prompt non-responders. However, this may not strengthen the validity and generalisability of the survey findings and affect the representativeness of the sample.

Sharing Risk between Payer and Provider by Leasing Health Technologies: An Affordable and Effective Reimbursement Strategy for Innovative Technologies?

The challenge of implementing high-cost innovative technologies in health care systems operating under significant budgetary pressure has led to a radical shift in the health technology reimbursement landscape. New reimbursement strategies attempt to reduce the risk of making the wrong decision, that is, paying for a technology that is not good value for the health care system, while promoting the adoption of innovative technologies into clinical practice. The remaining risk, however, is not shared between the manufacturer and the health care payer at the individual purchase level; it continues to be passed from the manufacturer to the payer at the time of purchase. In this article, we propose a health technology payment strategy-technology leasing reimbursement scheme-that allows the sharing of risk between the manufacturer and the payer: the replacing of up-front payments with a stream of payments spread over the expected duration of benefit from the technology, subject to the technology delivering the claimed health benefit. Using trastuzumab (Herceptin) in early breast cancer as an exemplar technology, we show how a technology leasing reimbursement scheme not only reduces the total budgetary impact of the innovative technology but also truly shares risk between the manufacturer and the health care system, while reducing the value of further research and thus promoting the rapid adoption of innovative technologies into clinical practice.