Early health technology assessment (HTA), which includes all methods used to inform industry and other stakeholders about the potential value of new medical products in development, including methods to quantify and manage uncertainty, has seen many applications in recent years. However, it is still unclear how such early value assessments can be integrated into the technology innovation process. This commentary contributes to the discussion on the purposes early HTA can serve. Similarities and differences in the perspectives of five stakeholders (i.e., the hospital, the patient, the assessor, the medical device industry, and the policy maker) on the purpose, value, and potential challenges of early HTA are described. All five stakeholders agreed that integrating early HTA in the innovation process has the possibility to shape and refine an innovation, and inform research and development decisions. The early assessment, using a variety of methodologies, can provide insights that are relevant for all stakeholders but several challenges, for example, feasibility and responsibility, need to be addressed before early HTA can become standard practice. For early evaluations to be successful, all relevant stakeholders including patients need to be involved. Also, nimble, flexible assessment methods are needed that fit the dynamics of medical technology. Best practices should be shared to optimize both the innovation process and the methods to perform an early value assessment.

Objectives: The reduction in cognitive decline depends on timely diagnosis. The aim of this systematic review was to analyze the current available information and communication technologies-based instruments for cognitive decline early screening and detection in terms of usability, validity, and reliability.

Methods: Electronic searches identified 1,785 articles of which thirty-four met the inclusion criteria and were grouped according to their main purpose into test batteries, measures of isolated tasks, behavioral measures, and diagnostic tools.

Results: Thirty one instruments were analyzed. Fifty-two percent were personal computer based, 26 percent tablet, 13 percent laptop, and 1 was mobile phone based. The most common input method was touchscreen (48 percent). The instruments were validated with a total of 4,307 participants: 2,146 were healthy older adults (M = 73.59; SD = 5.12), 1,104 had dementia (M = 74.65; SD = 3.98) and 1,057 mild cognitive impairment (M = 74.84; SD = 4.46). Only 6 percent were administered at home, 19 percent reported outcomes about usability, and 22 percent about understandability. The methodological quality of the studies was good, the weakest methodological area being usability. Most of the instruments obtained acceptable values of specificity and sensitivity.

Conclusions: It is necessary to create home delivered instruments and to include usability studies in their design. Involvement of people with cognitive decline in all phases of the development process is of great importance to obtain valuable and user-friendly products. It would be advisable for researchers to make an effort to provide cutoff points for their instruments.

Evince-I is a desktop expert system for the differential diagnosis of dementia, implemented on a personal computer. It is intended to assess the effectiveness of this new technology in modeling a psychiatrist who uses international guidelines for diagnosing dementia. EVINCE-I was tested in diagnosing 19 patients with varying stages of dementia and 10 patients showing other disorders except dementia. EVINCE-I and the human expert were in perfect agreement on the diagnosis of dementia and correlated highly on the diagnosis of dementia of the Alzheimer type and multiple infarct dementia. EVINCE-I thus offers important possibilities as a tool in investigating the data and procedures used by the human expert.

Objectives: The purpose of this study is to provide a comparison of the benefits of reduction mammaplasty (RM) for women with heavy breasts often termed macromastia or breast hypertrophy (BH) surgery. The rationale is to provide information to allow decision-makers to make judgments about the cost-effectiveness of this intervention and make comparisons with other interventions which are commonly undertaken within publicly financed health-care systems.

Methods: Data from a previous outcomes study in Sweden is re-analyzed to derive quality of life measures, from which a mean level of benefit outcome is derived and a cost per quality-adjusted life year is calculated (cost per QALY).

Results: The low Cost per QALY suggests that reduction mammaplasty is cost-effective when compared with other treatments which are commonly undertaken.

Conclusions: The authors suggest that the evidence in favor of funding reduction mammaplasty is strong and that decision-makers review their policy in light of this new evidence.

Objectives: To compare the cost-effectiveness of a multidisciplinary rehabilitation program with traditional treatment within primary care in terms of the health-related quality of life (HRQOL) in patients with prolonged musculoskeletal disorders (MSD) on the one hand and the costs to society on the other. Predictors of total costs, such as motivation, socio-economic level, age, pain, and working environment, were investigated.

Methods: A prospective, matched, controlled 2-year follow-up study was designed. The main outcome measures were HRQOL using the Nottingham Health Profile (NHP) and patient-specific total costs due to society. The cost-effectiveness was expressed as a quotient of the total costs to society/NHP global score difference value.

Results: Patients with prolonged MSD generate substantial total costs to society, chiefly in the area of indirect costs. Multidisciplinary rehabilitation improved HRQOL more cost-effectively. Motivation was revealed as a predictor of total costs. The relationship in savings in terms of indirect costs between the highly-motivated and the less-motivated patients was calculated at 4:1.

Conclusions: The large group of patients with prolonged MSD generate substantial total costs, and even small reductions in direct and indirect costs could be of importance to society. The multidisciplinary rehabilitation program applied here was more cost-effective as compared with conventional treatment within primary care when it came to improving the patients' perceived HRQOL. Motivation could be a predictor of total costs, which has to be addressed in the process of identifying the patient as a partner in the rehabilitation process.

Objectives: Technology assessment systems for interventional procedures (including surgical operations, minimally invasive procedures, and others) have lagged behind those for pharmaceutical treatments. Such systems have been introduced in some countries during the past decade amid debate about how they should be organized, but there is no collated information about where they exist or how they work. This study was designed to provide hitherto unavailable information about the existence, organization, methods, and outputs of systems aimed at influencing the use of interventional procedures in different countries.

Methods: Data were gathered from a questionnaire survey of key informers associated with healthcare technology assessment (HTA) organizations in different countries.

Results: Responses were received from key informers working for twenty-eight HTA organizations in twenty-five countries (response rate 83 percent). Information about a national system for assessing interventional procedures was obtained for fifteen countries. There was substantial variability in the type and funding of these organizations, the systems used for the selection of procedures, the types and sources of evidence used, the personnel involved in the appraisal of the evidence, the arrangements for consultation on the draft assessment, the format of assessment recommendations, the status of the guidance, and the use of guidance from other countries.

Conclusion: Guidance on interventional procedures is produced variably in different countries—and not at all in some. Greater international collaboration in the assessment of new interventional procedures could help to optimize the efficiency of existing systems as well as the quality of the assessments, by capitalizing on the outputs from scarce (international) resources and expertise.

More and more medical technologies are becoming available for the early detection of congenital abnormalities, such as amniocentesis, chorionic villus sampling, and ultrasound scanning. Recent research has shown that maternal serum can provide a wealth of information on the unborn fetus and that the testing method lends itself to mass screening. Although screening will prevent a great deal of grief, such developments also lead to extreme medicalization of pregnancy. However, owing to the fact that people show a strong desire to avert every possible approaching calamity, the “tentative pregnancy” seems to be unavoidable.

Objectives: The aim of this study was to analyze and describe process and outcomes of two pilot assessments based on the HTA Core Model, discuss the applicability of the model, and explore areas of development.

Methods: Data were gathered from HTA Core Model and pilot Core HTA documents, their validation feedback, questionnaires to investigators, meeting minutes, emails, and discussions in the coordinating team meetings in the Finnish Office for Health Technology Assessment (FINOHTA).

Results: The elementary structure of the HTA Core Model proved useful in preparing HTAs. Clear scoping and good coordination in timing and distribution of work would probably help improve applicability and avoid duplication of work.

Conclusions: The HTA Core Model can be developed into a platform that enables and encourages true HTA collaboration in terms of distribution of work and maximum utilization of a common pool of structured HTA information for national HTA reports.

Objectives: The aim of this study was to examine the evidence payers cited in their coverage policies for multi-gene panels and sequencing tests (panels), and to compare these findings with the evidence payers cited in their coverage policies for other types of medical interventions.

Methods: We used the University of California at San Francisco TRANSPERS Payer Coverage Registry to identify coverage policies for panels issued by five of the largest US private payers. We reviewed each policy and categorized the evidence cited within as: clinical studies, systematic reviews, technology assessments, cost-effectiveness analyses (CEAs), budget impact studies, and clinical guidelines. We compared the evidence cited in these coverage policies for panels with the evidence cited in policies for other intervention types (pharmaceuticals, medical devices, diagnostic tests and imaging, and surgical interventions) as reported in a previous study.

Results: Fifty-five coverage policies for panels were included. On average, payers cited clinical guidelines in 84 percent of their coverage policies (range, 73–100 percent), clinical studies in 69 percent (50–87 percent), technology assessments 47 percent (33–86 percent), systematic reviews or meta-analyses 31 percent (7–71 percent), and CEAs 5 percent (0–7 percent). No payers cited budget impact studies in their policies. Payers less often cited clinical studies, systematic reviews, technology assessments, and CEAs in their coverage policies for panels than in their policies for other intervention types. Payers cited clinical guidelines in a comparable proportion of policies for panels and other technology types.

Conclusions: Payers in our sample less often cited clinical studies and other evidence types in their coverage policies for panels than they did in their coverage policies for other types of medical interventions.

In our article “Romiplostim and eltrombopag for immune thrombocytopenia: methods for indirect comparison” published in the Journal (1), we presented an indirect comparison of the effectiveness of eltrombopag and romiplostim in raising platelet counts in patients with immune thrombocytopenia (ITP). Indirect comparison analyses are recommended by the National Institute for Health and Care Excellence (NICE) in cases where randomized head-to-head studies do not exist, and were used by NICE in their guidance for the eltrombopag Single Technology Appraisal submission (2).

In 1989, Poland started to slowly release itself not only from the burden of a half-century of communist indoctrination and soviet exploitation, but also from the consequences of the Semashko model of healthcare organization: low doctors' salaries, primary care based on multispecialty groups, overdeveloped hospital infrastructure, and limited access to sophisticated interventions overcome by patients' unofficial payments.

A few years after the 1998 workshop on health technology assessment (HTA) in Budapest, the first HTA reports were elaborated in the National Center for Quality Assessment in Health Care, which could mark the beginning of HTA in Poland. Several individuals and organizations have been involved in developing HTA, both from noncommercial and commercial standpoints.

A goal to establish a national HTA agency appeared among the priorities of the Polish Ministry of Health in 2004 and was realized a year later. The Agency for HTA in Poland published guidelines on HTA and established a sound and transparent two-step (assessment-appraisal) process for preparing recommendations on public financing of both drugs and nondrug technologies. The recommendations of the Agency's Consultative Council were warmly welcomed by the public payer. However, the recent major restructuring of the Agency and new drug reimbursement decisions aroused doubts as to keeping transparency of the decision-making processes.

The elemental resource model (ERM) attempts to provide a quantitative and straightforward framework for characterizing the human system, tasks, and their interface. It evolved in large part from the general systems performance theory (GSPT), which was developed first and independently. Resource constructs are used exclusively for modeling the abstract idea of system performance and for subsequent measurement of performance resource capacities. Resource economic principles provide a cause-and-effect description of the human-task interface. While argued to have immediate utility, it also provides the motivation to consider coordinated, collaborative, long-term developments that could facilitate effective decision making and technology utilization in rehabilitation.

Objective: To examine the relative importance of factors influencing health insurers' coverage of new genetic technologies.

Methods: A national survey in which the decision makers for private health insurers were asked whether they would cover cystic fibrosis (CF) carrier screening, testing for genetic susceptibility to breast cancer (BRCA test), and medical costs of a clinical trial of gene therapy for CF under a variety of conditions.

Results: Respondents' coverage of the two tests and of medical costs of clinical trials was low at the time of the study (4%–15.5% of insurers.) Their coverage of CF carrier screening and BRCA testing would be increased significantly if the group tested was restricted to those at high risk, if detection rates were higher and costs lower, and if testing was endorsed by a national professional group or consensus conference. Coverage of the medical costs of a trial of CF gene therapy would be significantly more likely if the trial was restricted to children or adults with severe CF, safety and effectiveness was proven, and therapy could be administered in a regional hospital or an outpatient setting rather than in a research hospital.

Conclusions: Health insurers play a critical role in the diffusion of new genetic technologies. The validity of genetic tests and the safety and effectiveness of new therapies are primary factors influencing health insurers' coverage. Lower costs and approval of professional groups are other factors associated with increased coverage.

The Swedish routine program for antenatal care is based on a number of scheduled visits for the detection of symptomless complications, as well as psychosocial support and health education. The scientific evidence to support the present timing and contents of routine visits is unsatisfactory, and there is a great need for evaluation both of single diagnostic procedures and interventions and of programs of antenatal care.

Objectives: The aim of this study is to review how health technology assessments (HTA) of medical tests incorporate intermediate outcomes in conclusions about the effectiveness of tests on improving health outcomes.

Methods: Systematic review of English-language test assessments in the HTA database from January 2005 to February 2010, supplemented by a search of the Web sites of International Network of Agencies for Health Technology Assessment (INAHTA) members.

Results: A total of 149 HTAs from eight countries were assessed. Half evaluated tests for screening or diagnosis, a third for disease classification (including staging, prognosis, monitoring), and a fifth for multiple purposes. In seventy-one HTAs (48 percent) only diagnostic accuracy was reported, while in seventeen (11 percent) evidence of health outcomes was reported in addition to accuracy. Intermediate outcomes, mainly the impact of test results on patient management, were considered in sixty-one HTAs (41 percent). Of these, forty-seven identified randomized trials or observational studies reporting intermediate outcomes. The validity of these intermediate outcomes as a surrogate for health outcomes was not consistently discussed; nor was the quality appraisal of this evidence. Clear conclusions about whether the test was effective were included in approximately 60 percent of HTAs.

Conclusions: Intermediate outcomes are frequently assessed in medical test HTAs, but interpretation of this evidence is inconsistently reported. We recommend that reviewers explain the rationale for using intermediate outcomes, identify the assumptions required to link intermediate outcomes and patient benefits and harms, and assess the quality of included studies.

Objective: Economic evaluations such as cost-effectiveness and cost-utility analyses generally fail to incorporate elements of intangible costs and benefits, such as anxiety and discomfort associated with the screening test and diagnostic test, as well as the magnitude of utility associated with a reduction in the risk of dying from cancer. This paper seeks to include all costs and effects incurred by introducing mammography screening through the application of discrete ranking modeling.

Methods: In the present analysis, 207 women were interviewed and asked to rank, according to priority, a number of alternative breast cancer screening setups. The alternative programs varied with respect to number of tests performed, risk reduction obtained, probability of a false-positive outcome, and extent of copayment. Using discrete ranking modeling, the stated preferences were analyzed and the relative weighting of the program attributes identified. For a range of hypothetical breast cancer programs, relative utilities and corresponding willingness-to-pay estimates were derived.

Results: A comparison of cost and willingness to pay for each of the programs suggested that net benefits are maximized when screening person aged 50–74 years biennially. More intensive screening produces lower or similar levels of utility at a higher cost.

Conclusion: Discrete ranking modeling can aid decision making by identifying inferior healthcare programs, i.e., programs that are more costly but less beneficial.