Objectives: The aim of this study was to examine the empirical and methodological cost-effectiveness evidence of surgical interventions for breast, colorectal, or prostate cancer.

Methods: A systematic search of seven databases including MEDLINE, EMBASE, and NHSEED, research registers, the NICE Web site and conference proceedings was conducted in April 2012. Study quality was assessed in terms of meeting essential, preferred and UK NICE specific requirements for economic evaluations.

Results: The seventeen (breast = 3, colorectal = 7, prostate = 7) included studies covered a broad range of settings (nine European; eight non-European) and six were published over 10 years ago. The populations, interventions and comparators were generally well defined. Very few studies were informed by literature reviews and few used synthesized clinical evidence. Although the interventions had potential differential effects on recurrence and mortality rates, some studies used relatively short time horizons. Univariate sensitivity analyses were reported in all studies but less than a third characterized all uncertainty with a probabilistic sensitivity analysis. Although a third of studies incorporated patients’ health-related quality of life data, only four studies used social tariff values.

Conclusions: There is a dearth of recent robust evidence describing the cost-effectiveness of surgical interventions in the management of breast, colorectal and prostate cancers. Many of the recent publications did not satisfy essential methodological requirements such as using clinical evidence informed by a systematic review and synthesis. Given the ratio of potential benefit and harms associated with cancer surgery and the volume of resources consumed by these, there is an urgent need to increase economic evaluations of these technologies.

Background: Relative effectiveness has become a key concern of health policy. In Europe, this is because of the need for early information to guide reimbursement and funding decisions about new medical technologies. However, ways that effectiveness (does it work?) and efficacy (can it work?) might differ across health systems are poorly understood.

Methods: This study proposes an analytical framework, drawing on production function theory, to systematically identify and quantify the determinants of relative effectiveness and sources of variation between populations and healthcare systems. We consider how methods such as stochastic frontier analysis and data envelopment analysis using a Malmquist productivity index could in principle be used to generate evidence on, and improve understanding about, the sources of variation in relative effectiveness between countries and over time.

Results: Better evidence on factors driving relative effectiveness could: inform decisions on how to best use a new technology to maximum effectiveness; establish the need if any for follow-up post-launch studies, and provide evidence of the impact of new health technologies on outcomes in different healthcare systems.

Conclusions: The health production function approach for assessment of relative effectiveness is complementary to traditional experimental and observational studies, focusing on identifying, collecting, and analyzing data at the national level, enabling comparisons to take place. There is a strong case for exploring the use of this approach to better understand the impact of new medicines and devices for improvements in health outcomes.

We reviewed the Canadian literature on cost-effectiveness from 1980–1995 with regard to costing methodologies. We abstracted each study using a list of data elements that describe costing methodology, and evaluated costing methodologies in the light of biases in costing methods, site, and case-mix selection.

Objectives: Health systems frequently make decisions regarding acquisition and use of new technologies. It is desirable to base these decisions on clinical evidence, but often these technologies are used for multiple indications and evidence of effectiveness for one indication does not prove effectiveness for all. Here, we describe two examples of evidence inventory reports that were performed for the purposes of identifying how much and what type of published clinical evidence was available for a given technology, and the contexts in which those technologies were studied.

Methods: The evidence inventory reports included literature searches for systematic reviews and health technology assessment (HTA) reports, and systematic searches of the primary literature intended to count and categorize published clinical studies. The reports did not include analysis of the primary literature.

Results: The inventory reports were completed in 3 to 4 days each and were approximately ten pages in length, including references. Reports included tables listing the number of reported studies by specific indication for use, and whether or not there were randomized trials. Reports also summarized findings of existing systematic reviews and HTA reports, when available. Committees used the inventory reports to decide for which indications they wanted a full HTA report.

Conclusions: Evidence inventory reports are a form of rapid HTA that can give decision makers a timely understanding of the available evidence upon which they can base a decision. They can help HTA providers focus subsequent reports on topics that will have the most influence on healthcare decision making.

Health technology assessment conducted to inform decisions during technology development (development-focused or DF-HTA) has a number of distinct features compared with HTA conducted to inform reimbursement and usage decisions. In particular, there are a broad range of decisions to be informed related to the development of a technology; multiple markets and decision makers to be considered; a limited (and developing) evidence base; and constrained resources for analysis. These features impact upon methods adopted by analysts. In this paper, we (i) set out methods of DF-HTA against a timeline of technology development; (ii) provide examples of the methods’ use; and (iii) explain how they have been adapted as a result of the features of DF-HTA. We present a toolkit of methods for analysts working with developers of medical technologies. Three categories of methods are described: literature review, stakeholder consultation, and decision analytic modeling. Literature review and stakeholder consultation are often used to fill evidence gaps. Decision analytic modeling is used to synthesize available evidence alongside plausible assumptions to inform developers about price or performance requirements. Methods increase in formality and complexity as the development and evidence base progresses and more resources are available for assessment. We hope this toolkit will be used in conjunction with the framework of features of DF-HTA presented in our earlier article in order to improve the clarity and appropriateness of methods of HTA used in DF-HTA. We also seek to contribute to a continuing dialogue about the nature of, and the best approach to, DF-HTA.

Objectives: Inappropriate hospital admissions are commonly believed to represent a potential for significant cost reductions. However, this presumes that these patients can be identified before the hospital stay. The present study aimed to investigate to what extent this is possible.

Methods: Consecutive admissions to a department of internal medicine were assessed by two expert panels. One panel predicted the appropriateness of the stays from the information available at admission, while final judgments of appropriateness were made after discharge by the other.

Results: The panels correctly classified 88% of the appropriate and 27% of the inappropriate admissions. If the elective admissions predicted to be inappropriate had been excluded, 9% of the costs would have been saved, and 5% of the gain in quality-adjusted life-years lost. The corresponding results for emergency admissions were 14% and 18%.

Conclusions: The savings obtained by excluding admissions predicted to be inappropriate were small relative to the health losses. Programs for reducing inappropriate health care should not be implemented without investigating their effects on both health outcomes and costs.

Objectives: To compare cost effectiveness models for the first-line treatment of multiple myeloma, and explore the differences between the models’ structure, parameters, assumptions and results.

Methods: Three cost effectiveness models for the treatment of multiple myeloma, were compared that had been developed to inform resource allocation in the UK for the chemotherapy regimens bortezomib, melphalan and prednisolone (BMP); and melphalan, prednisolone and thalidomide (MPT) versus melphalan and prednisolone (MP). The models used alternative approaches and assumptions to estimate the overall survival and progression-free survival for each of the interventions. Through the use of sensitivity analyses, the most influential parameters and assumptions of each of the models were identified.

Results: The models developed by the manufacturers gave conflicting results, with each manufacturer favouring their drug. The differences between the model results were determined by two parameters: the hazard ratio for overall survival for MPT vs. MP and the cost of bortezomib.

Conclusions: Using models developed for assessing treatments for multiple myeloma we demonstrated that it was feasible to compare models, which then aided decision makers in making reimbursement decisions.

Objectives: The overall aim of this study is to discern whether and to what degree vaccination sites exhibit constant returns to scale.

Methods: Data Envelopment Analysis is used to compare all the facilities in the sample in terms of input costs used to produce multiple outputs. The application considers the Expanded Program on Immunization (EPI), which operated in Dhaka City, Bangladesh, during 1999.

Results: A preponderance of EPI sites were determined to be operating at increasing returns to scale.

Conclusions: Our findings question the applicability of cost-effectiveness analyses that assume constant returns to scale.

Objectives and Methods: To review Switzerland's mixed public and private healthcare system with regard to health technology assessment (HTA).

Results: In the past, remarkable work in HTA was done. Accomplishments include the following: (i) Switzerland became an early member of the International Network of Agencies for Health Technology Assessment. (ii) HTA has its legal bases in terms of effectiveness, appropriateness, and efficiency. (iii) The federal law allows the introduction of new technologies for a limited time for evaluation. (iv) A Swiss Network for Health Technology Assessment was established. In 2004, federal HTA activities moved from the Swiss Federal Office “of Social Security” to the one for “Public Health.” The Office mainly mandates, manages, and coordinates evaluations attached to its prevention and intervention sections in the fields of AIDS, illegal drugs, and legal drugs.

Conclusions: Because of the absence of a governmental institution assessing and reporting on new health technologies, private and for profit organizations became more important for the decision-making processes. In a regulated market, the implications may be crucial for the public health.

Objectives: To assess whether sentinel lymph node biopsy (SLNB), an alternative to axillary lymph node dissection in treating female breast cancer, affords any cost savings.

Methods: We profile cumulative treatment costs of 811 breast cancer patients, 555 of whom received SLNB. Univariate and multivariate statistical tests are used to appraise whether these cost profiles differ between SLNB and other patients.

Results: The statistical results are mixed. However, none supports the conjecture that SLNB necessarily lowers the cost of treating the average breast patient.

Conclusions: SLNB may be cost-effective, but longer term costs and outcomes must be estimated before firm conclusions can be reached.

Background: Health technology assessment (HTA) examines the consequences of the application of health technologies and is aimed at better informing decision-makers. Over the past 30 years, different countries have implemented HTA organizations. Colombia established by law its own HTA agency (IETS) in 2011 which started operations in November 2012. The aim of this study was to assess the feasibility of conducting and using HTA to inform decision-making in this context. Through a qualitative approach, ten “drivers” emerged with the ability to help or hinder HTA development in this context: availability and quality of data, implementation strategy, cultural aspects, local capacity, financial support, policy/political support, globalization, stakeholder pressure, health system context, and usefulness perception.

Methods: Semi-structured interviews were conducted with key HTA researchers, after following rigorous transcription, and thematic content analysis, those aspects that may be barriers or facilitator for HTA development and use in Colombia were identified.

Results: Although HTA has become a tool to inform decision-making around the world, its use may vary according to setting. Determining those aspects which may enable or interfere with HTA development and use in Colombia may be useful for other countries when considering the establishment of HTA systems.

Conclusions: The conceptual transferability of concepts like “drivers” with caveats may be of interest for similar settings trying to incorporate HTA processes and institutions into systematic decision-making.

Objectives: The aim of this study was to develop a guide to identifying and managing risks for health technology assessment (HTA) programs and to obtain opinions on this topic from HTA agencies.

Methods: The risks and approaches to their management were compiled, drawing on experiences from HTA programs and the risk assessment literature. Opinion on this classification was obtained from members of the International Network of Agencies for Health Technology Assessment (INAHTA).

Results: Twenty-one risks for HTA programs were identified under the categories Formulation of HTA Questions, Preparation of the HTA Product, Dissemination, and Contracting. For each risk area, potential consequences and suggested management approaches were outlined. Responses from ten HTA programs indicated substantial agreement regarding the risks that had been identified and on the importance of risk management for their own operations.

Conclusions: Prudent management of HTA programs should take into account the risks related to external factors.

Health technology assessment (HTA) recommendations informed by patient concerns are seen to ensure democracy and legitimacy. We explored how written and oral patient involvement in two HTAs was reported on in publicly available final recommendations and discussion summaries of appraisal committees from three HTA bodies. We aimed to gain insights into how patient input was utilized by appraisal committees to better understand the goals of patient involvement and how these are being achieved. In each of the three HTA bodies, templated submission questionnaires provide a formal process for seeking written patient group input. Additionally, the National Institute for Health and Care Excellence (NICE) selects patient experts to provide a templated submission and attend appraisal committee meetings. For Scottish Medicines Consortium (SMC), a patient advocate and clinician combined meeting (PACE) discussed the cancer drug, referred to in the final recommendation. The discussion summaries of all appraisal committees contained references to patient involvement. Where two mechanisms for patient involvement were provided, oral input from the expert patients and PACE were more clearly documented than information from written patient group submissions. NICE reports focused on the perspective of the patient expert. The SMC report highlighted feedback from the PACE throughout. We suggest that the lack of clear reporting on the use of patient group input in deliberations and therefore accountability to patient groups limits progress in patient involvement in HTA. Patient groups may therefore not have a clear understanding of what information they can best provide to inform deliberations, and in reporting back to members.

Prevention of smoking in pregnancy is a mission of utmost importance since the fetal outcome is improved when the mother quits smoking. Methods to prevent smoking have to consider maternal attitudes and characteristics and be based on pedagogical and behavioral principles. A well-motivated staff, a self-help manual accepted by most women, and special support to those who want it are minimal requirements.

Objective: To study the costs and effects of incorporating ultrasonography in the triple assessment of palpable breast masses.

Methods: A decision analytic model was designed to compare a conventional strategy of performing fine-needle aspiration cytology after clinical examination and mammography, with three different experimental strategies of preceding ultrasonography. Empirical data were used from a prospective study in 522 breasts in 492 patients with a palpable mass, including 93 malignancies. In strategy 1, cases with probably benign, suspect malignant, and malignant ultrasonography results were referred for fine-needle aspiration cytology; in strategy 2, benign cases were also referred for fine-needle aspiration cytology; and in strategy 3, ultrasonography was only performed in patients with benign results on clinical examination and mammography, whereas immediate fine-needle aspiration cytology was performed in patients with suspicious lesions. Outcome variables included the total costs and the expected number of life years. Sensitivity analysis was performed on all parameters in the model.

Results: All strategies reported a similar life expectancy of 31.0 years. Cost-minimization demonstrated that experimental strategy 3 was the least expensive strategy (€ 3,013). Experimental strategy 2 was the most costly one (€ 3,512). Compared with the conventional strategy of immediate fine-needle aspiration cytology (€ 3,087), both ultrasonography strategies 1 and −3 were preferred.

Conclusions: Incorporating ultrasonography in the triple assessment of palpable breast masses can result in a reduction of the total costs for the diagnosis and treatment of breast cancer.