Physicians through the ages have practiced their trade with more or less regard to the effects of their treatment on their patients' sense of well-being, if not as much as on the disease itself.

Until recently, however, little attempt has been made to measure the effect of disease upon quality of life and how this quality is or is not improved by the treatment. Several means have now evolved and in areas of medicine where the treatment may profoundly affect the patient's sense of well-being, effort is being made to gauge these consequences.

Objectives: In April 1998, the Medical Services Advisory Committee (MSAC) was established by the Australian federal government. Since that time, all new medical procedures must be evaluated for safety, effectiveness, and cost-effectiveness as a condition of the surgeon receiving public funding by means of the Medicare Benefits Schedule (MBS). Over these first 8 years, a significant number of applications for the public funding of new procedures have been given negative recommendations by the MSAC based on insufficient clinical evidence or lack of cost-effectiveness. In August 2006, after almost 2 years of processing, the MSAC made the decision to fund the new procedure, laparoscopic remotely assisted radical prostatectomy (LRARP). However, they stated that there was still uncertainty about the comparative cost-effectiveness.

Methods: An observational study using provisional cost-utility data for LRARP based on a combination of costs taken from consecutive patients at the Epworth Hospital, Melbourne, Australia, and utilities from the prospectively collected data on all patients undergoing surgery for prostate cancer over a 4-year period at the Vattikuti Urology Institute, Michigan, United States.

Results: The incremental cost for LRARP compared with the open surgery alternative is A$2,264 or A$24,457 per quality-adjusted life-year, well below the range accepted by the Australian pharmaceutical equivalent of the MSAC (the PBAC) of A$42,000 and A$76,000. This figure does not take into account additional benefits such as reduced time away from employment, reduced blood loss, reduced possibility of infection, and reduced scarring.

Conclusions: This case study of LRARP demonstrates that there is sufficient crude evidence to show that this new procedure is likely to be superior to the existing procedure in terms of safety, effectiveness, and cost-effectiveness. The decision to allow MBS funding was correct and will allow for the collection of additional evidence, on both economic and clinical outcomes.

It has been suggested that preoperative total parentetal nutrition may be used to reduce the risk of nutrition-associated postoperative complications in high-risk patients. These patients can be identified based on their nutritional status. The efficiency of this intervention is assessed using the technique of cost-utility analysis. Data from multiple sources is integrated to perform the economic assessment.

The cost-utility ratios for treating several malnourished patients with localized upper gastrointestinal cancer are below $40,000. These cost-utility ratios compare favorably with published results of other programs. The ratios increase considerably if patients who are better nourished (at lower risk of postoperative complication) receive the intervention. The analysis is very sensitive to the efficacy of the intervention.

A range of fecal occult blood tests are presently on the market and could potentially be used in population screening programs for the detection of colorectal neoplasms. This paper estimates the relative cost-effectiveness of alternative tests and concludes that the unhydrated Hemoccult II is the most cost-effective. However, the incremental costs per life-year of the HemeSelect test and the rehydrated Hemoccult II test are in line with incremental costs observed in breast cancer and cervical cancer programs.

Including information and values from patients in HTA has the potential to improve both the process and outcomes of health technology policy decisions. Accordingly, funding and structural incentives to include patients in HTA activities have increased over the past several years. Unfortunately, these incentives have not yet been accompanied by a corresponding increase in resources, time, or commitment to responsiveness. In this Perspectives piece, we reflect on our collective experiences participating in, conducting, and overseeing patient engagement activities within HTA to highlight the ethical challenges associated with this area of activity. While we remain committed to the idea that patient engagement activities strengthen the findings, relevance, and legitimacy of health technology policy, we are deeply concerned about the potential for these activities to do ethical harm. We use this analysis to call for action to introduce strong protections against ethical violations that may harm patients participating in HTA engagement activities.

Objective: The aim of this study was to present a clear process of synthesizing test accuracy data when conducting economic evaluations of diagnostic tests for health technology assessment (HTA) assessors and health economists.

Methods: We appraised the methods advocated for using diagnostic test accuracy data in economic evaluations. We used a case study of fetal anemia in which data from a screening test are used in combination with a confirmatory test.

Results: We developed a step-by-step guide and consider two scenarios: when data on test accuracy from several studies are based on (i) the same test threshold for positivity and (ii) different test thresholds.

Conclusions: We conclude that each approach has its strengths and limitations. We show that the optimal operating point of the test should be identified to determine the true cost-effectiveness of the test. We advocate that these issues require a multidisciplinary team of health economists, decision modelers and statisticians.

Objectives: To improve processes of ambulatory care for patients with type 2 diabetes in a nationwide program.

Methods: Interrupted time-series analysis with audits of practice. To implement selected recommendations of national guidelines, educational outreach visits (office visits or phone discussions) were offered to all French physicians who diagnosed one case of type 2 diabetes during a six-month intervention period. Outcome measures were the number of HBA1c measurements recorded monthly in the medical insurance computer database and the proportion of diabetic patients for whom one test had been reimbursed during the previous six months (HBA1c, fasting blood glucose) or previous twelve months (serum cholesterol, serum creatinine, urine microalbumin, electrocardiogram, ophthalmologic examination).

Results: A total of 15,522 office visits and 9,062 telephone discussions were performed among 22,940 physicians. The increase in the monthly proportion of the number of HBA1c tests to the total number of laboratory tests was higher during the intervention period than during the preintervention (p value<.0001) and postintervention periods (p value<.001). Between the first audit (n=651,574) and the third audit (n=911,871), HBA1c measurements increased from 41.2% to 60.5% and blood glucose measurements performed alone decreased from 38.8% to 18.7%. Urine microalbumin measurements increased from 10.6% before to 15.3% after intervention. Only a slight increase was observed for other tests.

Conclusions: Physician to physician outreach visits can be an effective way to improve the processes of care for diabetes and to routinize nationwide use of practice guidelines.

Objectives: The aim of this study was to demonstrate how value of information analysis can measure the upper limit on returns to future research and identify the research priorities for computer-assisted total knee replacement (CAS-TKR).

Methods: Using a previous economic analysis of CAS-TKR compared with conventional TKR, the population expected value of perfect information (EVPI) was calculated using Monte Carlo simulation to provide an estimate of the upper limit on returns to future research. The population expected value of partial perfect information (EVPPI) for both individual parameters and groups of parameters was estimated to inform specific future research priorities.

Results: The UK individual EVPI would be £21.4 if the willingness to pay for one QALY (quality-adjusted life-year) were £30,000. The population EVPPI would be £8.3 million, assuming a 10-year time horizon for CAS-TKR. In this instance, the expected value of information is positively related to willingness to pay for one QALY for the range of £0 to £50,000. Although each individual parameter had an EVPPI of £0, groups of utility parameters had positive EVPPI. Population EVPPI was £5.6 million for utility parameters, £20,000 for transition probabilities relating to CAS-TKR, and £5,000 for transition probabilities related to conventional TKR.

Conclusions: The study provides evidence on which parameters further information may be of most value. Focusing research on the utility values associated with health states relating to TKR would be of greatest value.

The consensus development program for health care is an American invention which dates back to 1977. It is derived out of concern for the appropriate use of medical technology and designed to formalize the process by which research pertinent to health care is disseminated and used.

Health systems are placing more and more emphasis on designing and delivering services that are focused on the patient, and there is a growing interest in patient aspects of health policy research and health technology assessment (HTA). Only a few HTA agencies use and invest in scientific methods to generate knowledge and evidence about the patient aspects of a given technology. This raises questions about how knowledge is produced in HTA reports and what kind of knowledge is considered relevant. This article uses a Danish HTA on patient education from 2009 as empirical material for a critical examination and discussion of knowledge and knowledge production about the patient aspects of HTA.

Objectives: The aim of this study was to support health technology assessment (HTA) capacity building in Member States of the European Union with limited experience or without institutionalized HTA. The main output is a Handbook on HTA Capacity Building.

Methods: The methods used were worldwide surveys of (i) HTA organizations, (ii) information management units, and (iii) HTA educational programs. The results of two surveys (i & ii) were combined with expert opinion to produce the Handbook on HTA Capacity Building.

Results: Survey of HTA organizations (n = 41, response rate 35 percent). Most of the organizations were established by the government (61 percent), and all were not-for-profit. Working on HTA (80.5 percent) and doing research (63.4 percent) were the main lines of activity. Survey on information management units (n = 23, response rate 23 percent). Most (74.2 percent) of the responding HTA agencies reported having personnel dedicated to HTA information services. Survey on HTA educational programs (n = 48, response rate 60 percent). In total, nine Master of Science (MSc) programs were identified (three MSc in HTA and six MSc in HTA-related areas). Handbook on HTA Capacity Building. A group of twenty experts from thirteen countries developed the handbook. It consists of nine chapters focusing on HTA institutional development (structural setup, work processes, and visibility).

Conclusions: Setting up organizational structures and establishing effective HTA programs that guide key policy decisions is a challenging task. There are no standard models or pathways. “One size fits all” is not a useful principle because of the wide systemic and cultural differences between countries. The Handbook on HTA Capacity Building includes approaches for overall institutional development, especially in formulating objectives, setting up structures, and defining work processes.

Health technology assessment (HTA) is commonly used to guide evidence-informed decisions to optimize resource use, prioritize policies, and support countries to achieve universal health coverage. Producing HTAs requires time, scientific expertise, and political commitment, but these are not available in all settings – especially in low- and middle-income countries (LMIC) where HTA processes may be less institutionalized. Transferring and adapting existing HTAs to local settings may offer a solution while reducing duplication efforts. This scoping review aims to provide an overview of tools, methods, approaches, and considerations which can aid HTA transfers. We systematically searched (from 2005 to 2020) six databases and, using predefined inclusion criteria, included twenty-two studies. Data extraction followed a structured process, while synthesis was more iterative. We identified a common approach for HTA transfers. It follows the de novo process of undertaking original HTAs, but with additional steps to assess relevance (applicability), quality, and transferability, as well as steps to adapt parameters where necessary. The EUnetHTA Adaptation Toolkit was the only tool that provided guidance for adapting multiple HTA domains. Other tools were specific to systematic reviews (n = 1) or economic evaluations (n = 12), where one provided guidance for systematic reviews of economic evaluations. Eight papers reported transferring an HTA, with only one transferring to an LMIC. Finally, we reported issues that may facilitate or hinder transferability. In conclusion, we identified fourteen transfer approaches in the form of guidance or checklists, but harmonized and pragmatic guidance for HTA transfers to suit settings with limited HTA capacity seems warranted.

That new health technologies often diffuse into the health care systems of developed countries without adequate evaluation has long been a cause of concern (I). In addition, where clinical or economic assessments have been carried out, they often contain methodological weaknesses which reduce their usefulness to health policy makers or clinical practitioners (5,6).

This study provides a sociological commentary on the current debates within health technology assessment (HTA), specifically in response to the approaches taken in France, The Netherlands, Sweden, and the United Kingdom. It argues that HTA is part of a wider reflexive innovation system that seeks to order current and prospective technologies. The study discusses the socio-political process of HTA priority setting, the rhetorical role of HTA, the localised and contingent use of HTA, and the policy gap between guidelines and practice. It argues for the development of new types of methodologies for assessment and for a stronger social embedding of HTA practice.

Health technology assessment (HTA) agencies vary in their use of quantitative patient preference data (PP) and the extent to which they have formalized this use in their guidelines. Based on the authors' knowledge of the literature, we identified six different PP “use cases” that integrate PP into HTA in five different ways: through endpoint selection, clinical benefit rating, predicting uptake, input into economic evaluation, and a means to weight all HTA criteria. Five types of insight are distinguished across the use cases: understanding what matters to patients, predicting patient choices, estimating the utility generated by treatment benefits, estimating the willingness to pay for treatment benefits, and informing distributional considerations. Summarizing the literature on these use cases, we recommend circumstances in which PP can add value to HTA and the further research and guidance that is required to support the integration of PP in HTA. Where HTA places more emphasis on clinical outcomes, novel endpoints are available; or where there are already many treatment options, PP can add value by helping decision makers to understand what matters to patients. Where uptake is uncertain, PP can be used to estimate uptake probability. Where indication-specific utility functions are required or where existing utility measures fail to capture the value of treatments, PP can be used to generate or supplement existing utility estimates. Where patients are paying out of pocket, PP can be used to estimate willingness to pay.

Objectives: The University of Minnesota has maintained a home monitoring program for over 10 years for lung and heart–lung transplant patients. A cost analysis was completed to assess the impact of home monitoring on the cost of post-transplant medical care.

Methods: Clinical information gathered with the monitoring system includes spirometry, vital signs, and symptom data. To estimate the impact of this system on medical costs, we completed a retrospective analysis of the effects of home monitoring on the cost of post-lung transplant medical care. The cost analysis used multivariate linear regression with inpatient, outpatient, and total medical care costs as the dependent variables. The independent variables for the regression include home monitoring adherence, underlying disease, ambulatory diagnostic group mapping variables, transplant type, and patient demographics.

Results: The multivariate regression of the overall cost results predicts a 52.4 percent reduction in total costs with 100 percent patient adherence; this rate includes a 72.24 percent reduction in inpatient costs and a 46.6 percent increase in outpatient costs. The actual first year average patient adherence was 74 percent.

Conclusions: Adherence to home monitoring increases outpatient costs and reduces inpatient costs and provides an overall cost savings. The break-even point for patient adherence was 25.28 percent, where the net savings covered the cost of home monitoring. This is well within the actual first year adherence rates (74 percent) for subjects in the lung transplant home monitoring program, providing a net savings with adherence to home monitoring.

Objectives: The aim of the study was to investigate the feasibility and effectiveness of searching selected databases to identify information required to populate a decision-analytic model.

Methods: Methods of searching for information to populate a decision-analytic model were piloted using a case study of prophylactic antibiotics to prevent recurrent urinary tract infections in children. This study explored how the information requirements for a decision-analytic model could be developed into searchable questions and how search strategies could be derived to answer these questions. The study also assessed the usefulness of three published search filters and explored which resources might produce relevant information for the various model parameters.

Results: Based on the data requirements for this case study, 42 questions were developed for searching. These questions related to baseline event rates, health-related quality of life and outcomes, relative treatment effects, resource use and unit costs, and antibiotic resistance. A total of 1,237 records were assessed by the modeler, and of these, 48 were found to be relevant to the model. Search precision ranged from 0 percent to 38 percent, and no single database proved the most useful for all the questions.

Conclusions: The process of conducting specific searches to address each of the model questions provided information that was useful in populating the case study model. The most appropriate resources to search were dependent on the question, and multiple database searching using focused search strategies may prove more effective in finding relevant data than thorough searches of a single database.

South Africa has embarked on major health policy reform to deliver universal health coverage through the establishment of National Health Insurance (NHI). The aim is to improve access, remove financial barriers to care, and enhance care quality. Health technology assessment (HTA) is explicitly identified in the proposed NHI legislation and will have a prominent role in informing decisions about adoption and access to health interventions and technologies. The specific arrangements and approach to HTA in support of this legislation are yet to be determined. Although there is currently no formal national HTA institution in South Africa, there are several processes in both the public and private healthcare sectors that use elements of HTA to varying extents to inform access and resource allocation decisions. Institutions performing HTAs or related activities in South Africa include the National and Provincial Departments of Health, National Treasury, National Health Laboratory Service, Council for Medical Schemes, medical scheme administrators, managed care organizations, academic or research institutions, clinical societies and associations, pharmaceutical and devices companies, private consultancies, and private sector hospital groups. Existing fragmented HTA processes should coordinate and conform to a standardized, fit-for-purpose process and structure that can usefully inform priority setting under NHI and for other decision makers. This transformation will require comprehensive and inclusive planning with dedicated funding and regulation, and provision of strong oversight mechanisms and leadership.

Objectives: The aim of this study was to establish a list of priority topics for methods research to support decision making at the National Institute for Health and Clinical Excellence (NICE).

Methods: Potential priorities for methods research topics were identified through a focused literature review, interviews, an email survey, a workshop and a Web-based feedback exercise. Participants were members of the NICE secretariat and its advisory bodies, representatives from academia, industry, and other organizations working closely with NICE. The Web exercise was open to anyone to complete but publicized among the above groups.

Results: A list of potential topics was collated. Priorities for further research differed according to the type of respondent and the extent to which they work directly with NICE. Priorities emerging from the group closest to NICE included: methodology for indirect and mixed treatment comparisons; synthesis of qualitative evidence; research relating to the use of quality-adjusted life-years (QALYs) in decision making; methods and empirical research for establishing the cost-effectiveness threshold; and determining how data on the uncertainty of effectiveness and cost-effectiveness data should be taken into account in the decision-making process. Priorities emerging from the broadest group of respondents (through the Web exercise) included: methods for extrapolating beyond evidence observed in trials, methods for capturing benefits not included in the QALY and methods to assess when technologies should be recommended in the context of further evidence gathering.

Conclusions: Consideration needs to be given to the needs of those who use the outputs of research for decision making when determining priorities for future methods research.