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This paper elucidates and rationalizes the ethical governance system for healthcare AI research, as outlined in the 'Research Ethics Guidelines for AI Researchers in Healthcare' published by the South Korean government in August 2023. In developing the guidelines, a four-phase clinical trial process was expanded to six stages for healthcare AI research: preliminary ethics review (stage 1); creating datasets (stage 2); model development (stage 3); training, validation, and evaluation (stage 4); application (stage 5); and post-deployment monitoring (stage 6). Researchers identified similarities between clinical trials and healthcare AI research, particularly in research subjects, management and regulations, and application of research results. In the step-by-step articulation of ethical requirements, this similarity benefits from a reliable and flexible use of existing research ethics governance resources, research management, and regulatory functions. In contrast to clinical trials, this procedural approach to healthcare AI research governance effectively highlights the distinct characteristics of healthcare AI research in research and development process, evaluation of results, and modifiability of findings. The model exhibits limitations, primarily in its reliance on self-regulation and lack of clear delineation of responsibilities. While formulated through multidisciplinary deliberations, its application in the research field remains untested. To overcome the limitations, the researchers' ongoing efforts for educating AI researchers and public and the revision of the guidelines are expected to contribute to establish an ethical research governance framework for healthcare AI research in the South Korean context in the future. [ABSTRACT FROM AUTHOR]

Background: In studies of diagnostic performance that fail to detect a statistically significant difference between compared techniques, investigators often declare evidence of equivalence or similarity without having actually tested that assertion due to incorrect methodology or insufficient statistical power.Objective: The purpose of our investigation is to measure the prevalence of unfounded assertions of equivalence or similarity in comparison studies presented at the International Pediatric Radiology (IPR) meeting of 2016 and promote awareness of this fallacy of inference to the pediatric radiology community.Materials and Methods: Two pediatric radiologists independently reviewed the methodology and reporting quality of the 194 scientific paper abstracts from the 2016 IPR meeting. All comparison studies were identified and those failing to detect a statistically significance difference and making a claim of equivalence or similarity in the results or conclusion were assessed for a description of the study design type, statistical power and sample size estimator calculation.Results: Of 194 scientific paper abstracts, 112 (58%) were comparison studies. Of these, 36/112 (32%) made unfounded inferences of equivalence or similarity in diagnostic imaging performance. No study had an equivalence or non-inferiority design. No abstract specified the statistical power of the study, and only one abstract acknowledged a small sample size as a limitation in detecting a statistically significant difference.Conclusion: Inadequate reporting and unfounded inferences of equivalence or similarity were common in diagnostic performance comparison studies presented at IPR 2016. Failure to recognize these limitations could have adverse consequences by leading to the adoption of unvalidated imaging techniques. [ABSTRACT FROM AUTHOR]

The past few years have seen rapid changes in the methods of decision-analytic modelling of healthcare programmes for the purposes of economic evaluation. This paper focuses on four developments in modelling that have emerged over the past few years or have become more widely used.First, no one optimal method for extrapolating outcomes from clinical trials has yet been established. Modellers may draw from a set of varied assumptions about survival extrapolation that encompass a range of possibilities from highly optimistic to extremely cautious.Secondly, the practicality and appeal of microsimulation as a method for analysing healthcare decision problems has increased dramatically with the speed of computing technology. Individual instantiations of a system are generated by using a random process to draw from probability distributions a large number of times (also known as Monte Carlo or probabilistic simulation). Microsimulation is moving in new directions, such as discrete-event simulations that simulate sequences of events by drawing directly from probability distributions of event times; this approach is now being broadly applied to model situations where populations of patients interact with healthcare delivery systems. Microsimulation modelling of transmission systems at the population level is also rapidly developing.Thirdly, model calibration is emerging as a new tool that may offer health scientists a means of generating important fundamental knowledge about disease processes. Model calibration allows evidence synthesis in which observations on observable quantities are used to draw inferences about unobservable quantities. The methodology of model calibration has advanced considerably, drawing on theories of numerical analysis and mathematical programming such as gradient methods, intelligent grid search algorithms, and many more.As a fourth issue, an area of extraordinary activity is in the use of transmission models to analyse interventions for infectious diseases, including population-wide effects of vaccination. Transmission models use differential equations to simulate, deterministically for the most part, transitions among infection-related health states. Only recently have modelling methodologies been combined so that cost-effectiveness analyses can consider explicitly not only the patient-level benefits of interventions but also the secondary benefits through transmission dynamics.Advances in technology allow more realistic and complex healthcare models to be simulated more rapidly. However, decision makers will not readily accept results from models unless they can understand them intuitively and explain them to others in relatively simple terms. The challenge for the next generation of modellers is not only to harness the power available from these newly accessible methods, but also to extract from the new generation of models the insights that will have the power to influence decision makers. [ABSTRACT FROM AUTHOR]

Decision-analytic models for cost-effectiveness analysis are developed in a variety of software packages where the accuracy of the computer code is seldom verified. Although modeling guidelines recommend using state-of-the-art quality assurance and control methods for software engineering to verify models, the fields of pharmacoeconomics and health technology assessment (HTA) have yet to establish and adopt guidance on how to verify health and economic models. The objective of this paper is to introduce to our field the variety of methods the software engineering field uses to verify that software performs as expected. We identify how many of these methods can be incorporated in the development process of decision-analytic models in order to reduce errors and increase transparency. Given the breadth of methods used in software engineering, we recommend a more in-depth initiative to be undertaken (e.g., by an ISPOR-SMDM Task Force) to define the best practices for model verification in our field and to accelerate adoption. Establishing a general guidance for verifying models will benefit the pharmacoeconomics and HTA communities by increasing accuracy of computer programming, transparency, accessibility, sharing, understandability, and trust of models. [ABSTRACT FROM AUTHOR]

Since the introduction of the cost-effectiveness acceptability curve (CEAC) in 1994, its use as a method to describe uncertainty around incremental cost-effectiveness ratios (ICERs) has steadily increased. In this paper, first the construction and interpretation of the CEAC is explained, both in the context of modelling studies and in the context of cost-effectiveness (CE) studies alongside clinical trials. Additionally, this paper reviews the advantages and limitations of the CEAC. Many of the perceived limitations can be attributed to the practice of interpreting the CEAC as a decision rule while it was not developed as such. It is argued that the CEAC is still a useful tool in describing and quantifying uncertainty around the ICER, especially in combination with other tools such as plots on the CE plane and value-of-information analysis. [ABSTRACT FROM AUTHOR]

This paper investigates the benefits of using less intrusive wireless technologies for heart monitoring. By replacing well established heart monitoring devices (i.e. Holter) with wireless ECG based Body Area Networks (BAN), improved healthcare performance can be achieved, reflected in (1) high quality ECG recordings during physical activities and (2) increased patient satisfaction. A small scale clinical trial was conducted to compare both technologies and the results illustrate that the wireless ECG monitor was able to detect ECG signals intended for arrhythmia diagnostics. Furthermore, from a patient’s perspective, both technologies were evaluated using three dimensions, namely; hygienic aspects, physical activity, and skin reactions. Results demonstrate that the wireless ECG BAN showed better performance, especially regarding the hygienic aspects. It was also favourable for use during physical activities, and the signal quality of the wireless sensor system demonstrated good performance regarding signal noise and artefact disturbances. This paper concludes that wireless cardiac monitoring systems have significant benefits from a patient’s perspective, and further clinical trials should be conducted to further evaluate the new ECG based BAN system, to identify the possibility of widespread adoption and utilisation of wireless technology for arrhythmia diagnostics. [ABSTRACT FROM AUTHOR]

Considerable time and energy are expended in the scientific community to discuss the validity, importance, and applicability of the results of clinical trials. Depending on the goals, perspectives, and other motivating factors, protagonists and skeptics come to different conclusions, even when using the same methods and tools for critical appraisal. The aim of this study was to complement existing methods and tools with minor modifications to provide a prototype instrument that generates commonly accepted versions of critical appraisals. As a pilot experiment, one university-based and one industry-based referee independently completed the twin assessment of five trials published in well-recognized journals. They identified the study questions, defined the simplest, i.e., ideal, study designs to answer these questions, and checked eight validity criteria. Identical positive or negative answers of both referees increased or decreased the validity score. A maximum of two disagreements (0 score) was allowed. This procedure, which had been tested by two referees in a pilot experiment, was repeated with 19 third-year medical students and their supervisor at the Universidade Federal Fluminense, Niterói/RJ, Brasil. Four students each played the roles of the industry-based and university-based referees and finally recorded their consensus. The two referees of the pilot experiment agreed in all but one answer to the five investigated publications. The points of criticism differed in various papers. The consensus reached by the students considerably differed from the consensus reached by the referees. A consensus score generated by two referees or by two groups of students is feasible, but the achieved result is not necessarily reproducible. The critical appraisal of the study question in connection with the applied study design deserves special attention. It is time consuming but possible to identify and describe the possible flaws in the design conduct and report of clinical trials, but it is unlikely to reach a reproducible interpretation. These data indicate the problems with even evidence-based assessments and appraisals: the assessments may well be reproducible, but not the appraisals. Quality scores that include also the appraisal may therefore be interpreted with caution. Appraisals or quality scores may be used for interim decisions until data are available that confirm under real-world conditions what was predicted by the results generated under ideal but artificial conditions of a clinical trial. [ABSTRACT FROM AUTHOR]

In 2005, the National Institutes of Health (NIH) consensus conference published a series of papers recommending methods to improve the conduct of clinical trials in chronic GVHD. Although the NIH recommendations were primarily aimed at strengthening research, several papers addressed issues relevant for clinical practice, particularly diagnosis, severity scoring, and ancillary and supportive care practices. We conducted an international survey to assess the uptake of these recommendations, identify barriers to greater use and document the use and perceived effectiveness of available treatments. The response rate for the American survey of 1387 practitioners was 21.8%, and it was 24.6% for 407 centers surveyed in Europe, Asia, Australia and Africa. Most respondents were familiar with the NIH consensus recommendations (94-96%) and used them in practice. Multiple barriers to greater use were reported. Besides lack of time (55-62%), unfamiliarity with the recommendations, scarcity of evidence supporting the impact of recommendations on outcomes, insufficient training/experience in chronic GVHD management and inaccessibility of subspecialists were also endorsed. Systemic corticosteroids were reported to be the most effective treatment for chronic GVHD, but many others were perceived to have moderate or great success. Therapeutic management of steroid-refractory chronic GVHD was identified as the highest priority for research. [ABSTRACT FROM AUTHOR]

Ototoxicity diagnosis and management has historically been approached using a variety of methods. However, in recent years a consensus on useful and practical approaches has been developed through clinical guidelines of the American Speech Language Hearing Association, the American Academy of Audiology, and multiple clinical trials published in peer-reviewed literature. Some of the guidelines and approaches are used to detect and monitor ototoxicity, while others are used to grade adverse events. Some of the audiologic measures are primary, while others are adjunct measures and may be tailored to the specific needs of the patient or clinical trial. For some types of monitoring, such as drug-induced tinnitus or dizziness, validated paper survey instruments can be both sensitive and easy for fragile patients. This review addresses the characteristics of some of the most common clinical ototoxins and the most common methods for detecting and monitoring ototoxicity in clinical practice and clinical trials. [ABSTRACT FROM AUTHOR]

Purpose: Patient-reported outcome measures (PROMs), which are generic or condition-specific, are used for a number of reasons, including clinical care, clinical trials, and in national-level efforts to monitor the quality of health care delivery. Creating PROMs that meet different purposes without overburdening patients, healthcare systems, providers, and data systems is paramount. The objective of this study was to test a generalizable method to incorporate condition-specific issues into generic PROM measures as a first step to producing PROMs that efficiently provide a standardized score. This paper outlines the method and preliminary findings focused on a PROM for osteoarthritis of the knee (OA-K).Methods: We used a mixed-methods approach and PROMIS® measures to test development of a combined generic and OA-K-specific PROM. Qualitative methods included patient focus groups and provider interviews to identify impacts of OA-K important to patients. We then conducted a thematic analysis and an item gap analysis: identified areas covered by existing generic PROMIS measures, identified "gap" areas not covered, compared gap areas to legacy instruments to verify relevance, and developed new items to address gaps. We then performed cognitive testing on new items and drafted an OA-K-specific instrument based on findings.Results: We identified 52 existing PROMIS items and developed 24 new items across 14 domains.Conclusions: We developed a process for creating condition-specific instruments that bridge gaps in existing generic measures. If successful, the methodology will create instruments that efficiently gather the patient's perspective while allowing health systems, researchers, and other interested parties to monitor and compare outcomes over time, conditions, and populations. [ABSTRACT FROM AUTHOR]

This paper reports on the requirements for, design of, and preliminary evaluation of a novel pervasive healthcare system for supporting the care of premature infants as they transition from hospital to home. In support of this system, we report the results of gesture sensing in a clinical setting and of interviews and focus groups with caregivers and clinicians who are involved in the postnatal transition to the home. From these results, we developed prototype systems for monitoring and tracking observations of behavioral and health-related data in the home, including a mobile phone-based capture and access system for caregivers, a sensing platform, and an activity recognition algorithm for automatically documenting infant movement. We describe the results of preliminary trials of both systems with an emphasis on the synergistic importance of bridging this transition. The results of these trials indicate that clinically relevant monitoring can be accomplished in the home, but there is still more to do to integrate these approaches into a comprehensive monitoring system for this population. [ABSTRACT FROM AUTHOR]

Decision-analytical modelling is widely used in health-care economic evaluations, especially in situations where evaluators lack clinical trial data, and in circumstances where such evaluations factor into reimbursement pricing decisions. This paper aims to improve the understanding and use of modelling techniques in this context, with particular emphasis on Markov modelling. We provide an overview, in this paper, of the principles and methodological details of decision-analytical modelling. We propose a common route for practicing modelling that accommodates any type of decision-analytical modelling techniques. We use the treatment of chronic hepatitis B as an example to indicate the process of development, presentation and analysis of the Markov model, and discuss the strengths, weaknesses and pitfalls of different approaches. Good practice of modelling requires careful planning, conduct and analysis of the model, and needs input from modellers and users. [ABSTRACT FROM AUTHOR]

Objectives: The objective of this paper was to describe primary care prescribers' perspectives on electronic prescribing drug alerts at the point of prescribing.Design: We used a mixed-method study which included clinician surveys (web-based and paper) and focus groups with prescribers and staff.Participants: Prescribers (n = 157) working in one of 64 practices using 1 of 6 e-prescribing technologies in 6 US states completed the quantitative survey and 276 prescribers and staff participated in focus groups.Measurements: The study measures self-reported frequency of overriding of drug alerts; open-ended responses to: "What do you think of the drug alerts your software generates for you?"Results: More than 40% of prescribers indicated they override drug-drug interactions most of the time or always (range by e-prescribing system, 25% to 50%). Participants indicated that the software and the interaction alerts were beneficial to patient safety and valued seeing drug-drug interactions for medications prescribed by others. However, they noted that alerts are too sensitive and often unnecessary. Participant suggestions included: (1) run drug alerts on an active medication list and (2) allow prescribers to set the threshold for severity of alerts.Conclusions: Primary care prescribers recognize the patient safety value of drug prescribing alerts embedded within electronic prescribing software. Improvements to increase specificity and reduce alert overload are needed. [ABSTRACT FROM AUTHOR]

This article reviews models for the treatment of coronary heart disease (CHD). Whereas most of the models described were developed to assess the cost effectiveness of different treatment strategies, other models have also been used to extrapolate clinical trials, for capacity and resource planning, or to predict the future population with heart disease. In this paper we investigate the use of modelling techniques in relation to different types of health intervention, and we discuss the assumptions and limitations of these approaches. Many of the models reviewed in this paper use decision tree models for acute or short term interventions, and Markov or state transition models for chronic or long term interventions. Discrete event simulation has, however, been used for more complex whole system models, and for modelling resource-constrained interventions and operational planning. Nearly all of the studies in our review used cohort-based models rather than population based models, and therefore few models could estimate the likely total costs and benefits for a population group. Most studies used de novo purpose built models consisting of only a small number of health states. Models of the whole disease system were less common. The model descriptions were often incomplete. We recommend that the reporting of model structure, assumptions and input parameters is more explicit, to reduce the risk of biased reporting and ensure greater confidence in the model results. [ABSTRACT FROM AUTHOR]

Early health technology assessment is increasingly being used to support health economic evidence development during early stages of clinical research. Such early models can be used to inform research and development about the design and management of new medical technologies to mitigate the risks, perceived by industry and the public sector, associated with market access and reimbursement. Over the past 25 years it has been suggested that health economic evaluation in the early stages may benefit the development and diffusion of medical products. Early health technology assessment has been suggested in the context of iterative economic evaluation alongside phase I and II clinical research to inform clinical trial design, market access, and pricing. In addition, performing early health technology assessment was also proposed at an even earlier stage for managing technology portfolios. This scoping review suggests a generally accepted definition of early health technology assessment to be "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". The present review also aimed to identify recent published empirical studies employing an early-stage assessment of a medical product. With most included studies carried out to support a market launch, the dominant methodology was early health economic modeling. Further methodological development is required, in particular, by combining systems engineering and health economics to manage uncertainty in medical product portfolios. [ABSTRACT FROM AUTHOR]

The article reports that Dr Boldt had a prolific publication record of randomized clinical trials when readers questioned the authenticity of a study comparing albumin and hydroxyethyl starch (HES) for cardiopulmonary bypass priming. It mentions information on suspicion of unethical conduct of trials. It presents need for methods of computerized identification of falsification such as testing for non-random sampling.

Introduction: Patient-reported outcome measures (PROs) were originally developed for comparing groups of people in clinical trials and population studies, and the results were used to support treatment recommendations or inform health policy, but there was not direct benefit for the participants providing PROs data. However, as the experience in using those measures increased, it became obvious the clinical value in using individual patient PROs profiles in daily practice to identify/monitor symptoms, evaluate treatment outcomes and support shared decision-making. A key issue limiting successful implementation is clinicians' lack of knowledge on how to effectively utilize PROs data in their clinical encounters. Methods: Using a change management theoretical framework, this paper describes the development and implementation of three programs for training clinicians to effectively use PRO data in routine practice. The training programs are in three diverse clinical areas (adult oncology, lung transplant and paediatrics), in three countries with different healthcare systems, thus providing a rare opportunity to pull out common approaches whilst recognizing specific settings. For each program, we describe the clinical and organizational setting, the program planning and development, the content of the training session with supporting material, subsequent monitoring of PROs use and evidence of adoption. The common successful components and practical steps are identified, leading to discussion and future recommendations. Results: The results of the three training programs are described as the implementation. In the oncology program, PRO data have been developed and are currently evaluated; in the lung transplant program, PRO data are used in daily practice and the integration with electronic patient records is under development; and in the paediatric program, PRO data are fully implemented with around 7,600 consultations since the start of the implementation. Conclusion: Adult learning programs teaching clinicians how to use and act on PROs in clinical practice are a key steps in supporting patient engagement and participation in shared decision-making. Researchers and clinicians from different clinical areas should collaborate to share ideas, develop guidelines and promote good practice in patient-centred care. [ABSTRACT FROM AUTHOR]

The article presents the author's views on a paper on a study of antineoplastons in patients with brainstem glioma. The author states while efforts are needed for treatment of diffuse intrinsic pontine glioma (DIPG) but the paper is of no help. It is stated that the magnetic resonance imaging (MRI) reported are by far not typical of DIPG.

The article presents abstracts of papers presented at the 39th European Society of Clinical Pharmacy (ESCP) European Symposium on Clinical Pharmacy and the 3th Société Française de Pharmacie Clinique (SFPC) Congress: Clinical Pharmacy at the Front Line of Innovations in Lyon, France, including "Hallucinations Due to Therapeutic Doses of Ertapenem," "Carboplatin Dosage in Overweight Patient: Case Report" and "Severe Hypokalemia Associated With Flucloxacillin."

This paper examines inequality and polarization in self-assessed health, contributing towards the limited research existing on health economics. We use data from the European Health Interview Survey (EHIS) to investigate the relationship between health inequality and polarization across 27 European countries in two periods: 2006-2009 and 2013-2015. As our key variable is of an ordinal nature, we employ median based measures. Our empirical results suggest that Greece is the country with the highest level of health polarization in both periods, whereas Ireland has the lowest one when we consider countries where the median category is "very good", coinciding with the findings obtained in the inequality index. Estonia, Hungary and Lithuania have the highest degree of health polarization in both periods while Malta, The Netherlands and Spain are the countries with the lowest when we focus on those countries whose median category is "good" health. [ABSTRACT FROM AUTHOR]

Introduction: The aim of this clinical update is to summarize articles and guidelines published in the last year with the potential to change current clinical practice as it relates to women's health.Methods: We used two independent search strategies to identify articles relevant to women's health published between March 1, 2007 and February 29, 2008. First, we reviewed the Cochrane Database of Systematic Reviews and journal indices from the ACP Journal Club, Annals of Internal Medicine, Archives of Internal Medicine, British Medical Journal, Circulation, Diabetes, JAMA, JGIM, Journal of Women's Health, Lancet, NEJM, Obstetrics and Gynecology, and Women's Health Journal Watch. Second, we performed a MEDLINE search using the medical subject heading term "sex factors." The authors, who all have clinical and/or research experience in the area of women's health, reviewed all article titles, abstracts, and, when indicated, full publications. We excluded articles related to obstetrical aspects of women's health focusing on those relevant to general internists. We had two acceptance criteria, scientific rigor and potential to impact women's health. We also identified new and/or updated women's health guidelines released during the same time period.Results: We identified over 250 publications with potential relevance to women's health. Forty-six articles were selected for presentation as part of the Clinical Update, and nine were selected for a more detailed discussion in this paper. Evidence-based women's health guidelines are listed in Table 1. Table 1 Important Women's Health Guidelines in 2007-2008: New or Updated Topic Issuing organization Updated recommendations and comments Mammography screening in women 40-4917 ACP Individualized risk assessment and informed decision making should be used to guide decisions about mammography screening in this age group. To aid in the risk assessment, a discussion of the risk factors, which if present in a woman in her 40s increases her risk to above that of an average 50-year-old woman, is provided in the guidelines. In addition, available risk prediction models, such as the NIH Web site calculator (http://www.cancer.gov/bcrisktool/) can also be used to estimate quantitative breast cancer risk. This model was updated in 2008 with race-specific data for calculating risk in African-American women.18 The harms and benefits of mammography should be discussed and incorporated along with a woman's preferences and breast cancer risk profile into the decision on when to begin screening. If a woman decides to forgo mammography, the decision should be readdressed every 1 to 2 years. STD screening guidelines19 USPSTF and CDC Routine screening for this infection is now recommended for ALL sexually active women age 24 and under, based on the recent high prevalence estimates for chlamydia It is not recommended for women (pregnant or nonpregnant) age 25 and older, unless they are at increased risk for infection. STD treatment guidelines20 CDC Flouroquinolones are NO longer recommended for treatment of N. gonorrhea, due to increasing resistance (as high as 15% of isolates in 2006). For uncomplicated infections, treatment of gonorrhea should be initiated with ceftriaxone 125 mg IM or cefixime 400 mg PO and co-treatment for chlamydia infection (unless ruled out with testing). Recent estimates demonstrate that almost 50% of persons with gonorrhea have concomitant chlamydia infection21. STD = sexually transmitted disease, NIH = National Institutes of Health, ACP = American College of Physicians, USPSTF = United States Prevention Services Task Force, CDC = Centers for Disease Control. [ABSTRACT FROM AUTHOR]

Systematic reviews systematically evaluate and summarize current knowledge and have many advantages over narrative reviews. Meta-analyses provide a more reliable and enhanced precision of effect estimate than do individual studies. Systematic reviews are invaluable for defining the methods used in subsequent studies, but, as retrospective research projects, they are subject to bias. Rigorous research methods are essential, and the quality depends on the extent to which scientific review methods are used. Systematic reviews can be misleading, unhelpful, or even harmful when data are inappropriately handled; meta-analyses can be misused when the difference between a patient seen in the clinic and those included in the meta-analysis is not considered. Furthermore, systematic reviews cannot answer all clinically relevant questions, and their conclusions may be difficult to incorporate into practice. They should be reviewed on an ongoing basis. As clinicians, we need proper methodological training to perform good systematic reviews and must ask the appropriate questions before we can properly interpret such a review and apply its conclusions to our patients. This paper aims to assist in the reading of a systematic review.Am J Gastroenterol 2009; 104:1086–1092; doi:10.1038/ajg.2009.118 [ABSTRACT FROM AUTHOR]

OBJECTIVES:Surgical treatment is a therapeutic option for patients with gastroesophageal reflux disease (GERD). It is unclear which patient characteristics influence postoperative success. The purpose of this paper was to review the literature on prognostic factors for patients with GERD treated with fundoplication.METHODS:We searched Medline and the Cochrane Library Central for studies from 1966 through July 2007. We identified additional studies by reviewing bibliographies of retrieved articles and by consulting experts. We included English language studies that evaluated factors potentially affecting the outcomes after surgical treatments in patients with GERD. We recorded baseline patient characteristics associated with treatment efficacy, details on the study design, comparators, and definitions of outcomes.RESULTS:We assessed 6,318 abstracts; 53 cohorts and 10 case–control studies met our inclusion criteria. Age, body mass index, sex, esophagitis grade, and dysmotility were generally not associated with treatment outcomes. There were no consistent associations between preoperative response to acid suppression medications, baseline symptoms, baseline acid exposure, degree of lower esophageal sphincter competence, or position of reflux and surgical outcomes. Certain psychological factors might be associated with worse treatment outcomes.CONCLUSIONS:Although several preoperative predictors of surgical outcomes have been described, the quality and consistency of the data were mixed and the strength of the associations remains unclear. Additional studies with improved methodological designs are needed to better define which patient characteristics are associated with surgical outcomes following fundoplication.Am J Gastroenterol 2009; 104: 752–758; doi:10.1038/ajg.2008.123; published online 3 February 2009 [ABSTRACT FROM AUTHOR]

Nanotechnology is an important platform technology which will add new features like improved biocompatibility, smaller size, and more sophisticated electronics to neuro-implants improving their therapeutic potential. Especially in view of possible advantages for patients, research and development of nanotechnologically improved neuro implants is a moral obligation. However, the development of brain implants by itself touches many ethical, social and legal issues, which also apply in a specific way to devices enabled or improved by nanotechnology. For researchers developing nanotechnology such issues are rather distant from their daily work in the lab, but as soon as they use their materials or devices in medical applications such as therapy of brain diseases they have to be aware of and deal with them. This paper is intended to raise sensitivity for the ethical, legal and social aspects (ELSA) involved in applying nanotechnology in brain implants or other devices by highlighting the short term problems of testing and clinical trials within the existing regulatory frameworks (A), the short and medium-term questions of risks in the application of the devices (B) and the long-term perspectives related to problems of enhancement (C). To identify and address such issues properly nanotechnologists should involve ethical, legal and social experts and regulatory bodies in their research as early as possible. This will help to remove pressure from regulatory bodies, to settle public concern and to prevent non-acceptable developments for the benefit of the patients. [ABSTRACT FROM AUTHOR]

The application of Bayesian statistical analyses has been facilitated in recent years by methodological advances and an increasing complexity necessitated within research. Substantial debate has historically accompanied this analytic approach relative to the frequentist method, which is the predominant statistical ideology employed in clinical studies. While the essence of the debate between the two branches of statistics centres on differences in the use of prior information and the definition of probability, the ramifications involve the breadth of research design, analysis and interpretation. The purpose of this paper is to discuss the application of frequentist and Bayesian statistics in the pharmacoeconomic assessment of healthcare technology. A description of both paradigms is offered in the context of potential advantages and disadvantages, and applications within pharmacoeconomics are briefly addressed. Additional considerations are presented to stimulate further development and to direct appropriate applications of each method such that the integrity and robustness of scientific inference be strengthened. [ABSTRACT FROM AUTHOR]

The aim of this paper is to assess the benefit to treat plantar fasciitis with low-dose energy extracorporeal shock wave therapy (ESWT) and the efficacy of such treatment to abate the painful symptoms allowing a rapid return to the running activity. Our study included 54 running athletes treated for plantar fasciitis associated with a heel spur who received four sessions (once weekly) of low-dose ESWT, and followed prospectively on average 45 days, 6 and 24 months after their last session. The clinical results were excellent in 59% of cases, good in 12%, satisfactory in 21% and distinctly unsatisfactory in 8%. No patient was observed a significant modification of the heel spur at the follow-up X-ray. The ultrasound examination at 24 months showed a disappearance of the inflammation signs in 61% of cases. A strong correlation between ultrasound improvement and clinical results were found. Low-energy ESWT seems to be a good mean to treat plantar fasciitis in runners with a 71% of good or excellent results and a persistent improvement lasting 24 months. A randomized multicentric study seems to be necessary to define the type of energy that should be used in the future to treat plantar fasciitis, in particular in the athletic patients, to allow a faster return to sports activities. [ABSTRACT FROM AUTHOR]

The past few years have seen rapid changes in the methods of decision-analytic modelling of healthcare programmes for the purposes of economic evaluation. This paper focuses on four developments in modelling that have emerged over the past few years or have become more widely used.First, no one optimal method for extrapolating outcomes from clinical trials has yet been established. Modellers may draw from a set of varied assumptions about survival extrapolation that encompass a range of possibilities from highly optimistic to extremely cautious.Secondly, the practicality and appeal of microsimulation as a method for analysing healthcare decision problems has increased dramatically with the speed of computing technology. Individual instantiations of a system are generated by using a random process to draw from probability distributions a large number of times (also known as Monte Carlo or probabilistic simulation). Microsimulation is moving in new directions, such as discrete-event simulations that simulate sequences of events by drawing directly from probability distributions of event times; this approach is now being broadly applied to model situations where populations of patients interact with healthcare delivery systems. Microsimulation modelling of transmission systems at the population level is also rapidly developing.Thirdly, model calibration is emerging as a new tool that may offer health scientists a means of generating important fundamental knowledge about disease processes. Model calibration allows evidence synthesis in which observations on observable quantities are used to draw inferences about unobservable quantities. The methodology of model calibration has advanced considerably, drawing on theories of numerical analysis and mathematical programming such as gradient methods, intelligent grid search algorithms, and many more.As a fourth issue, an area of extraordinary activity is in the use of transmission models to analyse interventions for infectious diseases, including population-wide effects of vaccination. Transmission models use differential equations to simulate, deterministically for the most part, transitions among infection-related health states. Only recently have modelling methodologies been combined so that cost-effectiveness analyses can consider explicitly not only the patient-level benefits of interventions but also the secondary benefits through transmission dynamics.Advances in technology allow more realistic and complex healthcare models to be simulated more rapidly. However, decision makers will not readily accept results from models unless they can understand them intuitively and explain them to others in relatively simple terms. The challenge for the next generation of modellers is not only to harness the power available from these newly accessible methods, but also to extract from the new generation of models the insights that will have the power to influence decision makers. [ABSTRACT FROM AUTHOR]

Clinical studies for the development of new drugs in the pharmaceutical industry consist of a number of individual tasks which have to be carried out in a pre-defined chronological order. Each task requires certain types of medical personnel. This paper investigates the scheduling of clinical studies to be performed during a short-term planning horizon, the allocation of workforce between the studies, and the assignment of individual employees to tasks. Instead of developing a complex monolithic decision model, a hierarchical modelling approach is suggested. In the first stage, a compact integer optimization model is solved in order to determine the start-off times of the studies and the required staffing while taking the limited availability of personnel into account. The objective is to minimize total staffing costs. The assignment of individual employees to tasks is then made in the second stage of the procedure using a binary optimization model. [ABSTRACT FROM AUTHOR]

Objective: A combination of enteral (EN) and parenteral nutrition (PN) is often used as a strategy to optimize nutritional intake in critically ill patients; however, the effects of this intervention on clinically important outcomes have not been widely studied. This paper systematically reviewed studies that compare EN + PN to enteral nutrition (EN) alone in critically ill patients.Methods: We searched bibliographic databases, personal files, and relevant reference lists to identify randomized controlled trials that compared combination EN + PN to EN alone.Results: Only five studies met the inclusion criteria. In all these studies PN was started at the same time as EN in the experimental group. When the results of these trials were aggregated, EN + PN had no significant effect on mortality. There was no difference between the two groups in rates of infectious complications, length of hospital stay, or ventilator days.Conclusions: In critically ill patients who are not malnourished and have an intact gastrointestinal tract, starting PN at the same time as EN provides no benefit in clinical outcomes over EN alone. More research is needed to determine the effects of combination EN + PN on clinical outcomes in critically ill patients who are poorly intolerant to EN. [ABSTRACT FROM AUTHOR]

This paper provides a synthetic review of research on school-based mental health services. Schools play an increasingly important role in providing mental health services to children, yet most school-based programs being provided have no evidence to support their impact. A computerized search of references published between 1985 and 1999 was used to identify studies of school-based mental health services for children. Study inclusion was determined by (i) use of randomized, quasi-experimental, or multiple baseline research design; (ii) inclusion of a control group; (iii) use of standardized outcome measures; and (iv) baseline and postintervention outcome assessment. The application of these criteria yielded a final sample of 47 studies on which this review is based. Results suggest that there are a strong group of school-based mental health programs that have evidence of impact across a range of emotional and behavioral problems. However, there were no programs that specifically targeted particular clinical syndromes. Important features of the implementation process that increase the probability of service sustainability and maintenance were identified. These include (i) consistent program implementation; (ii) inclusion of parents, teachers, or peers; (iii) use of multiple modalities; (iv) integration of program content into general classroom curriculum; and (v) developmentally appropriate program components. Implications of these findings and directions for future research are discussed. [ABSTRACT FROM AUTHOR]

Background: A prospective cost-effectiveness analysis undertaken as part of the CAESAR (Canada, Australia, Europe, South Africa) placebo-controlled clinical trial showed that adding lamivudine to zidovudine-containing regimens for 1 year reduced progression to AIDS or death and, in addition, significantly reduced the number of hospitalisations, unscheduled outpatient visits and the requirement for medications for HIV-related illness. Data from all 1840 patients included in the intent-to-treat population of the CAESAR trial were used in the analysis reported in this paper. Because a third-party payer perspective was adopted, possible savings associated with increased productivity (indirect costs) were not taken into account. All costs were adjusted to 1997 prices. Results: The savings associated with reduced healthcare resource use in the CAESAR study were estimated to be 3045 Deutschmarks (DM) [German analysis] or 432 pounds sterling (£) [UK analysis] per patient for the 1-year time period. These savings partly offset the cost of lamivudine in the 2 countries. The German analysis showed that the addition of lamivudine to zidovudine-containing regimens resulted in an incremental cost-effectiveness ratio of DM22 405 [95% confidence interval (CI): -DM2199 to DM59 154] for progression to AIDS/death avoided and of DM8869 (95% CI: -DM1047 to DM23 365) for HIV-related illness avoided. The corresponding ratios for the UK analysis were £12 030 (95% CI: £6752 to £21 888) for progressions avoided and £4762 (95% CI: £2796 to £9484) for new and recurrent HIV-related illness avoided. Conclusions: Our findings indicate that treatments that slow the progression of HIV infection to AIDS or death have the potential to facilitate healthcare savings during the period that the treatment is effective. The results also demonstrate that it is possible to undertake economic evaluations in parallel with a major clinical end-point study. [ABSTRACT FROM AUTHOR]

A new methodological framework for assessing agreement between cost-effectiveness endpoints generated using alternative sources of data on treatment costs and effects for trial-based economic evaluations is proposed. The framework can be used to validate cost-effectiveness endpoints generated from routine data sources when comparable data is available directly from trial case report forms or from another source. We illustrate application of the framework using data from a recent trial-based economic evaluation of the probiotic Bifidobacterium breve strain BBG administered to babies less than 31 weeks of gestation. Cost-effectiveness endpoints are compared using two sources of information; trial case report forms and data extracted from the National Neonatal Research Database (NNRD), a clinical database created through collaborative efforts of UK neonatal services. Focusing on mean incremental net benefits at £30,000 per episode of sepsis averted, the study revealed no evidence of discrepancy between the data sources (two-sided p values >0.4), low probability estimates of miscoverage (ranging from 0.039 to 0.060) and concordance correlation coefficients greater than 0.86. We conclude that the NNRD could potentially serve as a reliable source of data for future trial-based economic evaluations of neonatal interventions. We also discuss the potential implications of increasing opportunity to utilize routinely available data for the conduct of trial-based economic evaluations. [ABSTRACT FROM AUTHOR]

A condition-specific preference-based measure (CSPBM) is a measure of health-related quality of life (HRQOL) that is specific to a certain condition or disease and that can be used to obtain the quality adjustment weight of the quality-adjusted life-year (QALY) for use in economic models. This article provides an overview of the role and the development of CSPBMs, and presents a description of existing CSPBMs in the literature. The article also provides an overview of the psychometric properties of CSPBMs in comparison with generic preference-based measures (generic PBMs), and considers the advantages and disadvantages of CSPBMs in comparison with generic PBMs. CSPBMs typically include dimensions that are important for that condition but may not be important across all patient groups. There are a large number of CSPBMs across a wide range of conditions, and these vary from covering a wide range of dimensions to more symptomatic or uni-dimensional measures. Psychometric evidence is limited but suggests that CSPBMs offer an advantage in more accurate measurement of milder health states. The mean change and standard deviation can differ for CSPBMs and generic PBMs, and this may impact on incremental cost-effectiveness ratios. CSPBMs have a useful role in HTA where a generic PBM is not appropriate, sensitive or responsive. However, due to issues of comparability across different patient groups and interventions, their usage in health technology assessment is often limited to conditions where it is inappropriate to use a generic PBM or sensitivity analyses. [ABSTRACT FROM AUTHOR]

Traditionally, US teaching hospitals have subsidised research by charging higher costs for treatment; however, this approach is being challenged. The growth of managed-care organisations, concerned about maximising profits, has led many to argue that clinical research will be damaged, whether by the loss of internal funds for research or by reductions in the numbers of patients available for studies. This review examines the evidence on which this argument is based. There is some evidence that managed-care organisations are refusing to cover patients who are involved in clinical trials, although, in general, they are receptive towards research providing that it is explicit and seen as relevant. The indirect effects of competition are, arguably, more important. Although many academic centres have established strategies to protect research funds, those working in the most competitive healthcare environments are obtaining fewer externally funded research grants. They are also publishing fewer papers and are working in climates that are seen as less supportive, with less ability to undertake research that is not externally funded. There is little evidence that managed care is reducing access to patients for clinical research. The growth of managed care is, together with certain other trends, also influencing the nature of clinical research. The overall consequences of these different factors are difficult to predict, although there are grounds for concern about recruitment and retention of junior researchers. The relationship between the various actors involved in healthcare and research is dynamic and, as pressure is excerpted in a particular direction, others adapt. It may be some time before the consequences of policies being enacted now are apparent. [ABSTRACT FROM AUTHOR]

This article explores the moral issues and the preventive and treatment deficiencies for AIDS, as well as the systematic deficiencies beginning with a discussion of the implications of chronic illnesses like AIDS, which is now recognized as a chronic illness. The paper offers a framework for understanding AIDS in relation to several important health issues. The normal sequence of steps through which treatment drugs reach the public--screening by the U.S. Federal Drug Administration and then carefully controlled clinical trials has been altered by AIDS activists, who have created a national lobby bent on changing features of the health care system. AIDS has heightened tensions around certain moral dilemmas associated with current health care policy by raising issues of personal responsibility for illness and public responsibility for preventing and treating certain illnesses.

Astonishingly, 98% of all marketed medicines, including all covid-19 vaccines, have either insufficient or no safety data to guide dosing during pregnancy and lactation [[7]] I . i Meanwhile, a major adverse pregnancy outcome, preterm birth, is rising world-wide. A "Clinical Trial Cost Study" conducted in 2016 indicated the median cost of a study from protocol approval to final report at US$3.4 million for phase I trials, $8.6 million for phase II trials and $21.4 million for phase III trials [[21]]. Am J Med Genet C Semin Med Genet. Perinatal clinical, biomedical, and life sciences research are important means of improving the health and well-being of mothers and infants, and as health in infancy can establish life-long trajectories, adult health as well. [Extracted from the article]

Purpose: The purpose of this study was to develop a new measure to evaluate the ability to receive medical services when needed among persons with type 2 diabetes mellitus.Methods: The Healthcare Access measure was developed using data from 225 persons with type 2 diabetes mellitus who completed an item pool comprised of 54 questions pertaining to their experience accessing healthcare services.Results: Exploratory and confirmatory factor analyses supported the retention of 45 items. In addition, a constrained graded response model (GRM), as well as analyses that examined item misfit and differential item functioning (investigated for age, sex, education, race, and socioeconomic status), supported the retention of 44 items in the final item bank. Expert review and GRM item calibration products were used to inform the selection of a 6-item static short form and to program the Healthcare Access computer adaptive test (CAT). Preliminary data supported the reliability (i.e., internal consistency and test-retest reliability) and validity (i.e., convergent, discriminant, and known-groups) of the new measure.Conclusions: The new Healthcare Access item bank can be used to examine the experiences that persons with type 2 diabetes mellitus have with healthcare access, to better target treatment improvements and mitigate disparities; it will be available as a part of the Neuro-Qol measurement system through healthmeasures.net and the PROMIS Application Programmable Interface (API) in early 2023. [ABSTRACT FROM AUTHOR]

Purpose: The purpose of this study is to evaluate potential gender-based differences in interpreting the Kansas City Cardiomyopathy Questionnaire (KCCQ-23) and to explore if there are aspects of health-related quality of life (HRQOL) not captured by the KCCQ-23 that are important to assess in men and/or women with heart failure (HF). Methods: Patients ≥ 22 years of age with clinician-diagnosed HF and left ventricular ejection fraction ≤ 40% were recruited from two academic medical centers to participate in semi-structured concept elicitation and cognitive debriefing interviews. Enrollment was stratified by patient-identified gender (half women/half men). All interviews were conducted over the phone/web and audio recorded. Interviews were transcribed and descriptive qualitative content analysis was used to summarize findings overall and by gender. Results: Twenty-five adults (56% women) diagnosed with HF participated. The average age was 67 years (range: 25–88). Women attributed a wider variety of symptoms to HF than men. Some participants had difficulty differentiating whether their experiences were due to HF, side effects of their medications, or age. We found very little evidence that participants interpreted KCCQ-23 items differently based on gender. Conclusions: Overall, our findings indicate that interpretation of the KCCQ-23 items were similar in men and women. However, some modifications to items may improve clarity of interpretation for a wide range of patients. [ABSTRACT FROM AUTHOR]

Purpose: Efficient analytical methods are necessary to make reproducible inferences on single-item longitudinal ordinal patient-reported outcome (PRO) data. A thorough simulation study was performed to compare the performance of the semiparametric probabilistic index models (PIM) with a longitudinal analysis using parametric cumulative logit mixed models (CLMM). Methods: In the setting of a control and intervention arm, we compared the power of the PIM and CLMM to detect differences in PRO adverse event (AE) between these groups using several existing and novel summary scores of PROs. For each scenario, PRO data were simulated using copula multinomial models. Comparisons were also exemplified using clinical trial data. Results: On average, CLMM provided substantially greater power than the PIM to detect differences in PRO-AEs between the groups when the baseline-adjusted method was used, and a small advantage in power when using the baseline symptom as a covariate. Conclusion: Although the CLMM showed the best performance among analytical methods, it relies on assumptions difficult to verify and that might not be fulfilled in the real world, therefore our recommendation is the use of PIM models with baseline symptom as a covariate. [ABSTRACT FROM AUTHOR]

Purpose: A joint modeling approach is recommended for analysis of longitudinal health-related quality of life (HRQoL) data in the presence of potentially informative dropouts. However, the linear mixed model modeling the longitudinal HRQoL outcome in a joint model often assumes a linear trajectory over time, an oversimplification that can lead to incorrect results. Our aim was to demonstrate that a more flexible model gives more reliable and complete results without complicating their interpretation. Methods: Five dimensions of HRQoL in patients with esophageal cancer from the randomized clinical trial PRODIGE 5/ACCORD 17 were analyzed. Joint models assuming linear or spline-based HRQoL trajectories were applied and compared in terms of interpretation of results, graphical representation, and goodness of fit. Results: Spline-based models allowed arm-by-time interaction effects to be highlighted and led to a more precise and consistent representation of the HRQoL over time; this was supported by the martingale residuals and the Akaike information criterion. Conclusion: Linear relationships between continuous outcomes (such as HRQoL scores) and time are usually the default choice. However, the functional form turns out to be important by affecting both the validity of the model and the statistical significance. Trial registration: This study is registered with ClinicalTrials.gov, number NCT00861094. [ABSTRACT FROM AUTHOR]

The article reports on the two-year research fellowship of EUROSPINE-The Spine Society of Europe, which focuses on orthopedic surgery in Europe. It is said that the research covers the academic and scientific evaluation of the registry data on statistics and epidemiology. In addition, researchers will also determine the design and implementation of multicenter studies.

Bangladesh has the third largest population of poor older adults in the world and 73% of them live in rural areas. Disparity in the country's health services is evident that creates a substantial pressure, especially on rural elderly women who live in a compromised socio-cultural atmosphere. This is true that we know about rural elderly women's self-reported health and service use barriers, but no studies captured the views of health staff. This study presents a qualitative exploration of the views held by rural health staff whose role is to provide care to local elderly women. We conducted 11 interviews with clinicians, pharmacists and public health assistants in Sylhet district, Bangladesh. A critical thematic discourse analysis, using the critical social constructs of Habermas and Honneth, of the data informed the women's inadequate healthcare access and associated barriers that were complex and overlapping but had explicit institutional, subjective and material consequences. Five major themes emerged including: unequal distribution of health services; marginalization in patient-staff relationships; living with poverty; social relegation; and mistrust of clinical treatment. Rural areas were viewed with inequitably distributed health services and traditionally a large proportion of elderly women living in poverty who lacked social support and demonstrated a mistrust towards healthcare system. No recognition of the women and power differences were underpinned by economic factors and cultural societal values. The findings suggest a need for health policy solutions and education of healthcare staff and elderly women regarding accessing healthcare. [ABSTRACT FROM AUTHOR]

Background: National regulatory agencies often have to use cost-effectiveness (CE) data from multinational randomized controlled trials (RCTs) for national decision making on reimbursement of new drugs. We need to make the best use of these patient-level data to obtain estimates of country-specific CE. Several methods, ranging from simple to statistically complex, have existed for years. We investigated which of these methods are used to estimate CE ratios in economic evaluations performed alongside recent, multinational RCTs that enrolled at least 500 patients. Methods: In this systematic literature review, studies were classified based on whether resource use, unit costs, health outcomes and utility value sets were obtained from all countries, a subset of countries or one country. We recorded if the study presented trial-wide and country-specific CE results and reported the statistical analyses that were used to estimate them. Results: We included 21 studies, of which the majority used measurements of health care utilization and health outcomes from all countries to estimate CE. Thirteen studies used a one-country valuation of health care utilization; six used a multi-country valuation. Despite the availability of country-specific utility value sets, none of the studies that presented quality-adjusted life-years (QALYs) used multi-country valuation. Valuation of health care utilization and health outcomes was not always consistent within a study: three studies combined a multi-country valuation of health care utilization, with a one-country valuation of health outcomes. Most studies calculated trial-wide CE estimates, while 11 studies calculated country- or region-specific estimates. Thirteen studies used relatively simple methods, which do not take the possible interaction between the country and treatment effect on health care utilization and health outcomes into account. Eight studies used more advanced statistical methods. Three of them used a fixed-effects modeling approach. Five studies explicitly took the hierarchical structure of the data into account, which leads to more appropriate estimates of population average results and associated standard errors. In this way, they help improve transferability of the published results. Conclusion: Based on this systematic review, we concluded that the uptake of more advanced statistical methods has been relatively slow, while simpler naïve methods are still routinely employed. [ABSTRACT FROM AUTHOR]

Background: The generic health-related quality-of-life (HR-QOL) utility measures the EQ-5D and SF-6D are both commonly used to inform healthcare policy developments. However, their application to pharmacy practice is limited and the optimal method to inform policy developments is unknown. Objectives: Our objective was to test the sensitivity of the EQ-5D and SF-6D within pharmacy when measuring whether changes in health status or other co-variates at baseline affect the effectiveness of the intervention at follow-up. A further objective was to consider the implications of the findings for pharmacy research and policy. Methods: The EQ-5D and SF-6D utility measures were employed within a randomized controlled trial (RCT) of community pharmacy-led medicines management for patients with coronary heart disease. The intervention covered a baseline visit with the potential for follow-up. Simultaneous quantile regression assessed the impact of the intervention on both EQ-5D and SF-6D measures at follow-up, controlling for baseline health, appropriateness of treatment, personal characteristics and self-reported satisfaction. Results: No statistically significant difference in HR-QOL across the intervention and control groups at follow-up was reported for either measure. Increased health gain was however associated with the baseline utility score (with the EQ-5D more sensitive for those in worse health) and the appropriateness of treatment, but not patient characteristics or self-reported satisfaction. Conclusion: Neither generic measure detected a gain in HR-QOL as a result of the introduction of an innovative pharmacy-based service. This finding supports other work in the area of pharmacy, where health gains have not changed following interventions. Disease-specific utility measures should be investigated as an alternative to generic approaches such as the EQ-5D and SF-6D. Given that the RCT found an increase in self-reported satisfaction, broader measures of benefit that value patient experiences, such as contingent valuation and discrete-choice experiments, should also be considered in pharmacy. [ABSTRACT FROM AUTHOR]

The identification of subgroups of patients from randomized clinical trials that are of specific interest for guiding clinical decisions can be an attractive idea; however, since such trials are designed for the comparison of groups of patients, performing subgroup analyses can result in misinterpretation of the data. Such analyses must, therefore, be performed and evaluated with caution: these should be pre-planned and included in the design of a suitably powered trial. Data obtained should be analyzed using formal statistical tests of interaction on proper subgroups rather than improper subgroups of patients, the results obtained should be delineated carefully, and details of how these analyses were performed, and how the data should be interpreted, should be reported in the trial paper. The caveats associated with this approach, such as the occurrence of false positive or false negative effects, chance differences in observed effects, lack of power to perform the analysis, floor or ceiling effects, issues relating to multiple statistical testing, and over-reporting and under-reporting are discussed in this review. Subgroup analyses can, however, provide valuable, albeit predominantly exploratory, information on which to base clinical decisions if they are performed in accordance with recommendations and guidelines, and do, therefore, have a legitimate place in rheumatology clinical trials. [ABSTRACT FROM AUTHOR]

Background: Results of 2 trials of postmenopausal hormone therapy (HT) challenged established practice patterns; 1 was not associated with changes in HT use, whereas the other was associated with substantial decline. Differential coverage by lay newspapers may have contributed to the differential impact.Objective: To examine newspaper coverage of HT before and after the publication of the Heart and Estrogen Replacement Study (HERS) in August 1998, and the main findings of the estrogen plus progestin therapy arm of the Women's Health Initiative (EPT-WHI) in July 2002.Design: Longitudinal review of newspaper articles, 1998 to 2003 (n=663).Setting: Twenty local and 6 regional/national newspapers.Measurements: Number and content of articles about HT.Results: The average number of articles about HT published during the month of the publication of the EPT-WHI was at least 8-fold greater than the number of articles published on the topic during any prior period. While the majority of articles in all periods presented information about the potential benefits of HT, information about harms became more common than information about benefits during the 2 months before the publication of the EPT-WHI, when the trial participants were notified of the early termination of the study. The presentation of specific health harms was more common after the publication of the EPT-WHI than after the publication of HERS. Few articles in any period used visual aids.Conclusions: The publication of the EPT-WHI was associated with a change in both the volume and content of newspaper coverage about HT. [ABSTRACT FROM AUTHOR]

OBJECTIVES: To determine effectiveness and costs of different guideline dissemination and implementation strategies. DATA SOURCES: MEDLINE (1966 to 1998), HEALTHSTAR (1975 to 1998), Cochrane Controlled Trial Register (4th edn 1998), EMBASE (1980 to 1998), SIGLE (1980 to 1988), and the specialized register of the Cochrane Effective Practice and Organisation of Care group. REVIEW METHODS: INCLUSION CRITERIA: Randomized-controlled trials, controlled clinical trials, controlled before and after studies, and interrupted time series evaluating guideline dissemination and implementation strategies targeting medically qualified health care professionals that reported objective measures of provider behavior and/or patient outcome. Two reviewers independently abstracted data on the methodologic quality of the studies, characteristics of study setting, participants, targeted behaviors, and interventions. We derived single estimates of dichotomous process variables (e.g., proportion of patients receiving appropriate treatment) for each study comparison and reported the median and range of effect sizes observed by study group and other quality criteria. RESULTS: We included 309 comparisons derived from 235 studies. The overall quality of the studies was poor. Seventy-three percent of comparisons evaluated multifaceted interventions. Overall, the majority of comparisons (86.6%) observed improvements in care; for example, the median absolute improvement in performance across interventions ranged from 14.1% in 14 cluster-randomized comparisons of reminders, 8.1% in 4 cluster-randomized comparisons of dissemination of educational materials, 7.0% in 5 cluster-randomized comparisons of audit and feedback, and 6.0% in 13 cluster-randomized comparisons of multifaceted interventions involving educational outreach. We found no relationship between the number of components and the effects of multifaceted interventions. Only 29.4% of comparisons reported any economic data. CONCLUSIONS: Current guideline dissemination and implementation strategies can lead to improvements in care within the context of rigorous evaluative studies. However, there is an imperfect evidence base to support decisions about which guideline dissemination and implementation strategies are likely to be efficient under different circumstances. Decision makers need to use considerable judgment about how best to use the limited resources they have for quality improvement activities. [ABSTRACT FROM AUTHOR]

This commentary describes the transition to remote delivery of cognitive-behavioral therapy (CBT) for anxiety in children with autism spectrum disorder (ASD) who participates in a clinical trial during the COVID-19 pandemic. The effects of COVID-19 on children's anxiety and on the family functioning are discussed. Modifications to CBT necessitated by telehealth delivery were aimed at maximizing engagement of children and their parents while maintaining treatment fidelity and adhering to the research protocol. Treatment targets were updated to address new sources of anxiety and CBT exposure exercises were modified to accommodate the new reality of quarantine restrictions. If the COVID-19 pandemic continues to affect treatment delivery it may require a widespread utilization of telehealth for treating anxiety in children with ASD. [ABSTRACT FROM AUTHOR]

In 1990, the Fast Track Project was initiated to evaluate the feasibility and effectiveness of a comprehensive, multicomponent prevention program targeting children at risk for conduct disorders in four demographically diverse American communities (Conduct Problems Prevention Research Group [CPPRG], 1992). Representing a prevention science approach toward community-based preventive intervention, the Fast Track intervention design was based upon the available data base elucidating the epidemiology of risk for conduct disorder and suggesting key causal developmental influences (R. P. Weissberg & M. T. Greenberg, 1998). Critical questions about this approach to prevention center around the extent to which such a science-based program can be effective at (1) engaging community members and stakeholders, (2) maintaining intervention fidelity while responding appropriately to the local norms and needs of communities that vary widely in their demographic and cultural/ethnic composition, and (3) maintaining community engagement in the long-term to support effective and sustainable intervention dissemination. This paper discusses these issues, providing examples from the Fast Track project to illustrate the process of program implementation and the evidence available regarding the success of this science-based program at engaging communities in sustainable and effective ways as partners in prevention programming. [ABSTRACT FROM AUTHOR]

The COVID-19 pandemic has been a catalyst for the implementation of decentralized clinical trials (DCTs) enabled by digital health technologies (DHTs) in the field while curtailing in-person interactions and putting significant demands on health care resources. DHTs offer improvements in real-time data acquisition remotely while maintaining privacy and security. Here, we describe the implications of technologies, including edge computing, zero-trust environments, and federated computing in DCTs enabled by DHTs. Taken together, these technologies—in the setting of policy and regulation that enable their use while protecting the users—extend the scope and accelerate the pace of clinical research. [ABSTRACT FROM AUTHOR]