Modeling death rates has been suggested as a potential method to screen hospitals and identify superior and substandard providers. This article begins with a review of one hospital death rate study and focuses upon its findings and limitations. It also explores the inherent limitations in the use of large data sets to assess quality of care.

Advances in the digitization of health systems and expedited regulatory approvals of innovative treatments have led to increased potential for the use of real-world data (RWD) to generate real-world evidence (RWE) to complement evidence from clinical trials. However, health technology assessment (HTA) bodies and payers have concerns about the ability to generate RWE of sufficient quality to be pivotal evidence of relative treatment effectiveness. Consequently, there is a growing need for HTA bodies and payers to develop guidance for the industry and other stakeholders about the use of RWD/RWE to support access, reimbursement, and pricing. We therefore sought to (i) understand barriers to the use of RWD/RWE by HTA bodies and payers; (ii) review potential solutions in the form of published guidance; and (iii) review findings with selected HTA/payer bodies. Four themes considered key to shaping the generation of robust RWE for HTA bodies and payers were identified as: (i) data (availability, governance, and quality); (ii) methodology (design and analytics); (iii) trust (transparency and reproducibility); and (iv) policy and partnerships. A range of guidance documents were found from trusted sources that could address these themes. These were discussed with HTA experts. This commentary summarizes the potential guidance solutions available to help resolve issues faced by HTA decision-makers in the adoption of RWD/RWE. It shows that there is alignment among stakeholders about the areas that need improvement in the development of RWE and that the key priority to move forward is better collaboration to make data usable for multiple purposes.

Objectives: The aims of the present study were to investigate the awareness among dentists and dental hygienists of evidence-based reports and guidelines on tobacco cessation activities and the impact these publications had on clinical practice.

Methods: A questionnaire was mailed to dental hygienists and dentists in Stockholm County, Sweden, and the results were compared with a previous investigation.

Results: Among the respondents, awareness of a popular science version of a systematic review on smoking and its effect on oral health was reported by 90 percent of the hygienists and 66 percent of the dentists. The information was used in clinical work by 34 percent of the dentists and 54 percent of the hygienists. Reported changes in patterns of practice were more frequent recommendations to use nicotine replacement therapy and a more widespread use of setting quit dates. Approximately one quarter of the dental professionals reported that they had increased tobacco cessation consultation because of the results from the reports.

Conclusions: Changes in patterns of practice were observed after dissemination of evidence-based information on tobacco cessation. Methods that were proven to be effective in the evidence-based report such as discussing quit dates and recommending nicotine replacement therapy were more commonly used after the publication of the report. Short, popular versions of extensive systematic reviews seem to be useful for implementing evidence-based knowledge and changing clinical practice.

Objectives: The assessment of electronic fetal monitoring (EFM) carried out by the authors in the late 1970s provides an early case of a systematic review of evidence in health technology assessment. This paper identifies lessons pertinent for the present day in this field.

Methods: We reviewed our own files for the description of our assessment and reactions to it. We also reviewed recent literature to evaluate our observations in relation to recent evidence.

Results: Our findings of insufficient evidence of efficacy and concerns about safety have been confirmed by subsequent research. Still, despite findings and recommendations of prominent professional and governmental bodies, EFM continues in widespread use in the United States and Europe and is spreading into developing countries around the world. Aggressive attacks on our assessment as well as our skills and integrity have been mirrored in recent years by criticism of other researchers in health technology assessment.

Conclusions: The case of EFM points to the limitations of assessment without other actions to assure the implementation of results. Health technologies that are accepted by the majority of clinicians in a particular field may require extraordinary efforts to assure appropriate use of technology assessments.

Large data sets are an attractive source of information for outcome assessment, but their use involves certain problems and risks. Data base evaluations are retrospective and unblinded; they often represent the result of multiple analyses of multiple endpoints, and it is difficult to identify the procedures used and analytic choices made because critical details are often omitted. While data bases can suggest problems and offer answers, they cannot prove them; data base analyses must be followed by trials.

Objectives: The aim of this report was to assess the clinical effectiveness of two Gene expression profiling (GEP) and two expanded immunohistochemistry (IHC) tests compared with current prognostic tools in guiding the use of adjuvant chemotherapy in patients with early breast cancer.

Methods: A systematic review of the evidence on clinical effectiveness of OncotypeDX, IHC4, MammaPrint, and Mammostrat, compared with current clinical practice using clinicopathological parameters, in women with early breast cancer was conducted. Ten databases were searched to include citations to May 2016.

Results: Searches identified 7,064 citations, of which forty-one citations satisfied the criteria for the review. A narrative synthesis was performed. Evidence for OncotypeDX demonstrated the impact of the test on decision making and there was some support for OncotypeDX predicting chemotherapy benefit. There were relatively lower levels of evidence for the other three tests included in the analysis. MammaPrint, Mammostrat, and IHC4 tests were limited to a small number of studies. Limitations in relation to study design were identified for all tests.

Conclusions: The evidence base for OncotypeDX is considered to be the most robust. Methodological weaknesses relating to heterogeneity of patient cohorts and issues arising from the retrospective nature of the evidence were identified. Further evidence is required for all of the tests using prospective randomized controlled trial data.

Health technology assessment (HTA) conducted to inform developers of health technologies (development-focused HTA, DF-HTA) has a number of distinct features when compared to HTA conducted to inform usage decisions (use-focused HTA). To conduct effective DF-HTA, it is important that analysts are aware of its distinct features as analyses are often not published. We set out a framework of ten features, drawn from the literature and our own experience: a target audience of developers and investors; an underlying user objective to maximize return on investment; a broad range of decisions to inform; wide decision space; reduced evidence available; earlier timing of analysis; fluid business model; constrained resources for analysis; a positive stance of analysis; and a “consumer”-specific burden of proof. This paper presents a framework of ten features of DF-HTA intended to initiate debate as well as provide an introduction for analysts unfamiliar with the field.

Objectives: The aim of this study was to describe the process and challenges of developing a decision aid for the national public breast cancer screening program in Finland.

Methods: An expert team with stakeholder representation used European guidelines and other literature as basis for selecting relevant content and format for the decision aid for breast cancer screening. Feedback from women was sought for the draft documents.

Results: A decision aid attached to the invitation letter for screening was considered the best way to ensure access to information. In addition, tailored letter templates for all phases of the screening program, a poster, and a public website were developed. Initial feedback from users (women, professionals, and service providers), was mainly positive. Six months after publishing, the implementation of the decision aid was still incomplete.

Conclusions: Providing balanced information for women invited to breast cancer screening is demanding and requires careful planning. Professionals and service providers need to be engaged in the HTA process to ensure proper dissemination and implementation of the information. End user participation is essential in the formulation of information. There is a need to follow up the implementation of the decision aid.

Background: To our knowledge, there has been no overall systematic review focusing on the methodological quality of full economic evaluation studies of self-management interventions. Our objective was to systematically review the literature of full economic evaluation studies of self-management interventions in adult chronic patients and to investigate their methodological quality and cost-effectiveness.

Methods: A data extraction form was developed to assess general and randomized controlled trial (RCT) -related characteristics, quality, of the RCTs, economic information and quality of the economic evaluation studies by means of a quality assessment (CHEC-list for trial-based studies, adjusted CHEC-list for model-based studies).

Results: Twenty-three reports were found. Sixteen studies (73 percent) lack information on the control intervention(s). Only one study fulfilled all three criteria for quality of RCTs and five studies (23 percent) did not meet any of these criteria. This review included one model-based study; the other studies were trial-based economic evaluation studies based on a RCT. Eight studies (35 percent) used a societal perspective and 12 (60 percent) synthesized costs and effects. Seven studies were categorized into the highest category (<15 score), nine studies into the “moderate” group (9–14 score), six studies received a “low” score (<8) on the CHEC-list. Eighteen studies found the self-management intervention(s) to be cost-effective compared with other interventions

Conclusions: Self-management interventions for adult chronic patients were heterogeneous and there was no clear, well-considered definition of self-management. Overall, the methodological quality of the full economic evaluation studies was moderate and, therefore, cost-effectiveness results must be interpreted with caution. Future research will benefit from further improvements in methodological quality of both economic study design and analysis, as well as a taxonomy for describing self-management interventions and their contents.

Moderate psychosocial problems identified during early pregnancy in 78 women were found to predispose for various “complications” during gestation, but not for prematurity or obstetrical complications during delivery. However, from a normal postnatal status the children of these mothers demonstrated a significantly unfavorable somatic, as well as psychomotoric development, until the age of 4. Pregnant women with certain psychosocial problems must be early identified and property attended to in order to preclude an adverse child development.

Objectives: In cost-minimization studies, it is important to establish noninferiority in the clinical effect of the treatments under investigation. The relationship between the proportion of patients reaching the end point in a study, equivalence limit (δ), and power is investigated in the context of cost-minimization studies with dichotomous clinical end points. Two formulations of the null-hypothesis, absolute and relative formulations of δ, will be explored.

Methods: Sensitivity analysis was performed, in which the effect of the predicted proportions and δ on the power in a noninferiority setting was investigated. The patterns found are discussed in terms of the practical relevance within the cost-minimization framework.

Results: Sensitivity analyses show different patterns of results for both null-hypotheses. The differences in these results originate from the way δ is expressed. By expressing δ as absolute difference, power grows quite fast when sample proportions are smaller than expected. In the case of a proportional δ at small sample proportions, the power to establish noninferiority remains low.

Conclusions: To obtain valid results from a cost-minimization study, care has to be taken to adapt the correct methodology for noninferiority testing in clinical outcomes. Defining δ in terms of absolute differences between treatments can lead to obscured results. Although conservative, the expression of δ as a proportion of the effectiveness of the treatment as usual is found to be closer to clinical practice. The inflated δ, resulting from smaller clinical effects than expected when absolute formulation is applied, thus can be avoided.

When there is a scarcity of resources (therapists, modalities), the physical therapist must make decisions about whom to treat and why. This paper discusses ethical dilemmas confronting the therapist and addresses solutions based on approaches of procedural justice and distributive justice.

Objectives: The aim of this study was to estimate thresholds for production volume, durability, and cost of care for the cost-effective adoption of liver organ replacement technologies (ORTs).

Methods: We constructed a discrete-event simulation model of the liver allocation system in the United States. The model was calibrated against UNOS data (1994–2000). Into this model, we introduced ORTs with varying durability (time to failure), cost of care, and production volume. Primary outputs of interest were time to 5 percent reduction in the waiting list and time to 5 percent increase in expected transplant volume.

Results: Model output for both calibration and validation phases closely matched published data: waiting list length (±2 percent), number of transplants (±2 percent), deaths while waiting (±5 percent), and time to transplant (±11 percent). Reducing the waiting list was dependent on both ORT durability and production volume. The longer the durability, the less production volume needed to reduce the waiting list and vice versa. However, below 250 ORT/year, durability needed to be >2 years for any significant change to be seen in the waiting list. For base-case costs, all ORT production volume and durability scenarios result in more transplants per year at less total cost of care/patient than the current system. ORTs remain cost saving until manufacturing costs are >5 times base-case costs, production is less 500 ORT/year, and durability <6 months.

Conclusions: Although there remain many technical challenges to overcome, as long as ORTs can meet these threshold criteria, they have the potential of transforming the world of end-stage liver disease.

Coronary artery bypass graft surgery (CABGS) has become an important procedure for the treatment of coronary heart disease. Over 100,000 of these surgical procedures are performed each year (1) at an aggregate cost of about $2 billion (2). Despite its current widespread acceptance, this major surgical innovation has generated considerable controversy concerning its effectiveness (3), and there still remains substantial confusion in assessing its overall impact (4).

With a growing number of economic evaluations being conducted alongside trials, there is a need to address, and if necessary, investigate the generalizability of results derived from trials. This paper explores the generalizability of costs from a trial investigating the frequency of breast cancer screening in the United Kingdom.

Objectives: The objective of this study is to analyze statistically the possible determinants and implications of including or not including recommendations in health technology assessments (HTAs).

Methods: A sample of 433 HTAs published by eleven leading institutions or agencies in nine countries was reviewed and analyzed statistically by multiple logistic regression.

Results: The extent of policy and research recommendations in HTAs varies greatly from country to country. The content and scope of HTAs have some impact on recommendations. Extensive assessment of economic and organizational aspects increases the likelihood of including policy recommendations. Extensive assessment of technological and patient aspects increases the likelihood of including research recommendations, whereas extensive focus on economic aspects is negatively related to research recommendations. The most striking result is that the use of external partners for assessment increases the likelihood of including research recommendations in HTAs but not policy recommendations.

Conclusions: HTA commissioners, agencies, institutions, and funding authorities need to be aware of the consequences of the choices they make in advance of assessing health technologies. Outsourcing HTA to external partners suggests a greater likelihood of being told that “more research is needed.” The scope and content of HTAs has an impact on the type of recommendations, and country-specific preferences are strong predictors of recommendations in HTAs.

The focus of this article is on contemporary roots of medical care quality measurement, while it also examines some of the most important historical precedents. Specifically, it singles out Dr. Ernest Avery Codman and his early 20th-century work emphasizing the assessment of outcome as the paramount indicator of medical care quality. The standard that he set for himself and others is the one that late 20th-century researchers must uphold, and to which we must answer.

Objectives: Although disease modifying antirheumatic drugs (DMARDs) are the first choice drugs in the treatment of rheumatoid arthritis, many patients still take nonsteroidal anti-inflammatory drugs (NSAIDs) as well. These drugs may cause serious gastric adverse events with continuous usage. Cyclooxygenase-2 (COX2) inhibitors were supposed to have a gastrointestinal (GI) friendly side effect profile. The aim of the study is to compare three therapeutic strategies: conventional NSAIDs, NSAID in combination with proton pump inhibitors (PPIs), and the selective COX2 inhibitor therapy (celecoxib).

Methods: A decision tree model was developed, for 1 year, to simulate cohorts within the three arms (NSAIDs, NSAID + PPI, celecoxib). The efficacy of the different active agents of NSAIDs in therapeutically relevant doses was assumed to be the same, consequently differences can be seen in the side effect profile of the drugs. Medical costs, the costs of the side effects (GI, cardiovascular [CV] events), and quality-adjusted life-years (QALYs) were calculated to gain an incremental cost-effectiveness ratio (ICER). Evaluations were made from a third party payer's perspective. We performed one-way deterministic sensitivity analyses; the results were displayed in tornado diagrams.

Results: Our model indicates that NSAID + PPI offers extra health gain for extra costs compared with conventional NSAIDs (ICER:14,287 euro/QALY), while it dominates celecoxib because of celecoxib's higher costs and lower effectiveness. According to the sensitivity analyses, QALYs had the highest influence on ICER.

Conclusions: Although COX2 inhibitors have elevated GI efficacy compared with NSAIDs, celecoxib seems to be an adequate choice only for a limited group of patients with specific conditions because of the significantly higher price and CV risk profile.