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Registry-based randomized controlled trials are defined as pragmatic trials that use registries as a platform for case records, data collection, randomization, and follow-up. Recently, the application of registry-based randomized controlled trials has attracted increasing attention in health research to address comparative effectiveness research questions in real-world settings, mainly due to their low cost, enhanced generalizability of findings, rapid consecutive enrollment, and the potential completeness of follow-up for the reference population, when compared with conventional randomized effectiveness trials. However several challenges of registry-based randomized controlled trials have to be taken into consideration, including registry data quality, ethical issues, and methodological challenges. In this article, we summarize the advantages, challenges, and areas for future research related to registry-based randomized controlled trials.

This article presents the current state of patient-reported outcome measures and explains new opportunities for leveraging the recent adoption of electronic health records to expand the application of patient-reported outcomes in both clinical care and comparative effectiveness research. Historic developments of patient-reported outcome, electronic health record, and comparative effectiveness research are analyzed in two dimensions: patient centeredness and digitization. We pose the question, “What needs to be standardized around the collection of patient-reported outcomes in electronic health records for comparative effectiveness research?” We identified three converging trends: the progression of patient-reported outcomes toward greater patient centeredness and electronic adaptation; the evolution of electronic health records into personalized and fully digitized solutions; and the shift toward patient-oriented comparative effectiveness research. Related to this convergence, we propose an architecture for patient-reported outcome standardization that could serve as a first step toward a more comprehensive integration of patient-reported outcomes with electronic health record for both practice and research. The science of patient-reported outcome measurement has matured sufficiently to be integrated routinely into electronic health records and other electronic health solutions to collect data on an ongoing basis for clinical care and comparative effectiveness research. Further efforts and ideally coordinated efforts from various stakeholders are needed to refine the details of the proposed framework for standardization.

The Core Outcome Measures in Effectiveness Trials (COMET) database is a publically available, searchable repository of published and ongoing core outcome set (COS) studies. An annual systematic review update is carried out to maintain the currency of database content. The methods used in the fourth update of the systematic review followed the same approach used in the original review and previous updates. Studies were eligible for inclusion if they reported the development of a COS, regardless of any restrictions by age, health condition or setting. Searches were carried out in March 2018 to identify studies that had been published or indexed between January 2017 and the end of December 2017. Forty-eight new studies, describing the development of 56 COS, were included. There has been an increase in the number of studies clearly specifying the scope of the COS in terms of the population (n = 43, 90%) and intervention (n = 48, 100%) characteristics. Public participation has continued to rise with over half (n = 27, 56%) of studies in the current review including input from members of the public. The rate of inclusion of all stakeholder groups has increased, in particular participation from non-clinical research experts has risen from 32% (mean average in previous reviews) to 62% (n = 29). Input from participants located in Australasia (n = 17; 41%), Asia (n = 18; 44%), South America (n = 13; 32%) and Africa (n = 7; 17%) have all increased since the previous reviews. This update included a pronounced increase in the number of new COS identified compared to the previous three updates. There was an improvement in the reporting of the scope, stakeholder participants and methods used. Furthermore, there has been an increase in participation from Australasia, Asia, South America and Africa. These advancements are reflective of the efforts made in recent years to raise awareness about the need for COS development and uptake, as well as developments in COS methodology.

Core outcome sets (COS) comprise a minimum set of outcomes that should be measured and reported in all trials for a specific health condition. The COMET (Core Outcome Measures in Effectiveness Trials) Initiative maintains an up to date, publicly accessible online database of published and ongoing COS. An annual systematic review update is an important part of this process. This review employed the same, multifaceted approach that was used in the original review and the previous two updates. This approach has identified studies that sought to determine which outcomes/domains to measure in clinical trials of a specific condition. This update includes an analysis of the inclusion of participants from low and middle income countries (LMICs) as identified by the OECD, in these COS. Eighteen publications, relating to 15 new studies describing the development of 15 COS, were eligible for inclusion in the review. Results show an increase in the use of mixed methods, including Delphi surveys. Clinical experts remain the most common stakeholder group involved. Overall, only 16% of the 259 COS studies published up to the end of 2016 have included participants from LMICs. This review highlights opportunities for greater public participation in COS development and the involvement of stakeholders from a wider range of geographical settings, in particular LMICs.

Objective: Prior meta-analysis indicates that collaborative chronic care models (CCMs) improve mental and physical health outcomes for individuals with mental disorders. This study aimed to investigate the stability of evidence over time and identify patient and intervention factors associated with CCM effects to facilitate implementation and sustainability of CCMs in clinical practice. Methods: We reviewed 53 CCM trials that analyzed depression, mental quality of life (QOL), or physical QOL outcomes. Cumulative meta-analysis and metaregression were supplemented by descriptive investigations across and within trials. Results: Most trials targeted depression in the primary care setting, and cumulative meta-analysis indicated that effect sizes favoring CCM quickly achieved significance for depression outcomes, and more recently achieved significance for mental and physical QOL. Four of 6 CCM elements (patient self-management support, clinical information systems, system redesign, and provider decision support) were common among reviewed trials, whereas 2 elements (health care organization support and linkages to community resources) were rare. No single CCM element was statistically associated with the success of the model. Similarly, metaregression did not identify specific factors associated with CCM effectiveness. Nonetheless, results within individual trials suggest that increased illness severity predicts CCM outcomes. Conclusions: Significant CCM trials have been derived primarily from 4 original CCM elements. Nonetheless, implementing and sustaining this established model will require health care organization support. Although CCMs have typically been tested as population-based interventions, evidence supports stepped care application to more severely ill individuals. Future priorities include developing implementation strategies to support adoption and sustainability of the model in clinical settings while maximizing fit of this multicomponent framework to local contextual factors.

While the United States leads the world in many measures of health care innovation, it has been suggested that it lags behind many developed nations in a variety of health outcomes. It has also been stated that the United States continues to outspend all other Organisation for Economic Co-operation and Development (OECD) countries by a wide margin. Spending on health goods and services per person in the United States, in 2007, increased to$7,290 – almost 2½ times the average of all OECD countries. Rising health care costs in the United States have been estimated to increase to 19.1% of gross domestic product (GDP) or $ 4.4 trillion by 2018. The increases are illustrated in both public and private sectors. Higher health care costs in the United States are implied from the variations in the medical care from area to area around the country, with almost 50% of medical care being not evidence-based, and finally as much as 30% of spending reflecting medical care of uncertain or questionable value. Thus, comparative effectiveness research (CER) has been touted by supporters with high expectations to resolve most ill effects of health care in the United States and provide high quality, less expensive, universal health care. CER is defined as the generation and synthesis of evidence that compares the benefits and harms of alternate methods to prevent, diagnose, treat, and monitor a clinical condition or to improve the delivery of care. The efforts of CER in the United States date back to the late 1970’s even though it was officially born with the Medicare Modernization Act (MMA) and has been rejuvenated with the American Recovery and Reinvestment Act (ARRA) of 2009 with an allocation of $1.1 billion. CER has been the basis for health care decision-making in many other countries. According to the International Network of Agencies for Health Technology Assessments (INAHTA), many industrialized countries have bodies that are charged with health technology assessments (HTAs) or comparative effectiveness studies. Of all the available agencies, the National Institute for Health and Clinical Excellence (NICE) of the United Kingdom is the most advanced, stable, and has provided significant evidence, though based on rigid and proscriptive economic and clinical formulas. While CER is making a rapid surge in the United States, supporters and opponents are expressing their views. Part I of this comprehensive review will describe facts, fallacies, and politics of CER with discussions to understand basic concepts of CER. Key words: Comparative effectiveness research, evidence-based medicine, Institute of Medicine, National Institute for Health and Clinical Excellence, interventional pain management, interventional techniques, geographic variations, inappropriate care.

The United States leads the world in many measures of health care innovation. However, it has been criticized to lag behind many developed nations in important health outcomes including mortality rates and higher health care costs. The surveys have shown the United States to outspend all other Organisation for Economic Co-operation and Development (OECD) countries with spending on health goods and services per person of$7,290 – almost 2½ times the average of all OECD countries in 2007. Rising health care costs in the United States have been estimated to increase to 19.1% of gross domestic product (GDP) or $ 4.4 trillion by 2018. CER is defined as the generation and synthesis of evidence that compares the benefits and harms of alternate methods to prevent, diagnose, treat, and monitor a clinical condition or to improve the delivery of care. The, comparative effectiveness research (CER) has been touted by supporters with high expectations to resolve most ill effects of health care in the United States providing high quality, less expensive, universal health care. The efforts of CER in the United States date back to the late 1970s and it was officially inaugurated with the enactment of the Medicare Modernization Act (MMA). It has been rejuvenated with the American Recovery and Reinvestment Act (ARRA) of 2009 with an allocation of $1.1 billion. CER has been the basis of decision for health care in many other countries. Of all the available agencies, the National Institute for Health and Clinical Excellence (NICE) of the United Kingdom is the most advanced, stable, and has provided significant evidence, though based on rigid and proscriptive economic and clinical formulas. While CER is taking a rapid surge in the United States, supporters and opponents are emerging expressing their views. Since interventional pain management is a new and evolving specialty, with ownership claimed by numerous organizations, at times it is felt as if it has many fathers and other times it becomes an orphan. Part 2 of this comprehensive review will provide facts, fallacies, and politics of CER along with discussion of potential outcomes, impact of CER on health care delivery, and implications for interventional pain management in the United States. Key words: Comparative effectiveness research, evidence-based medicine, Institute of Medicine, National Institute for Health and Clinical Excellence, interventional pain management, interventional techniques, geographic variations, inappropriate care.

The goal of comparative effectiveness research is to assess medical therapies and allow patients, health care providers, payers, and policy makers to make evidence-based decisions about the most appropriate therapies in routine clinical practice. To conduct this type of research and to inform health care delivery, data about the impact of interventions on patient outcomes are needed. Methods of generating evidence for comparative effectiveness research provide opportunities to engage patients and understand their experiences with illness and its treatment. In this article we assess the need for, uses of, and strengths and weaknesses of patient-generated data. We also review in brief federal and medical society efforts to create new streams of patient-generated data for clinical and research use. We observe that the key to high-quality patient-generated data is to have immediate and actionable data so that patients experience the importance of the data for their own care as well as research purposes. We conclude that leveraging the emerging wealth of \"big data\" being generated by patient-facing technologies such as systems to collect patient-reported outcomes data and patient-worn sensors is critical to developing the evidence base that informs decisions made by patients, providers, and policy makers in pursuit of high-value medical care.

The Affordable Care Act offers strong support for comparative effectiveness research, which entails comparisons among active treatments, to provide the foundation for evidence-based practice. Traditionally, a key form of research into the effectiveness of therapeutic treatments has been placebo-controlled trials, in which a specified treatment is compared to placebo. These trials feature high-contrast comparisons between treatments. Historical trends in placebo-controlled trials have been evaluated to help guide the comparative effectiveness research agenda. We investigated placebo-controlled trials reported in four leading medical journals between 1966 and 2010. We found that there was a significant decline in average effect size or average difference in efficacy (the ability to produce a desired effect) between the active treatment and placebo. On average, recently studied treatments offered only small benefits in efficacy over placebo. A decline in effect sizes in conventional placebo-controlled trials supports an increased emphasis on other avenues of research, including comparative studies on the safety, tolerability, and cost of treatments with established efficacy. [PUBLICATION ABSTRACT]

The Patient-Centered Outcomes Research Institute (PCORI) was established by the Affordable Care Act of 2010 to promote comparative effectiveness research (CER) to assist patients, clinicians, purchasers, and policy-makers in making informed health decisions by advancing the quality and relevance of evidence concerning the manner in which diseases, disorders, and other health conditions can effectively and appropriately be prevented, diagnosed, treated, monitored, and managed through research and evidence synthesis. The development of PCORI is vested in the Medicare Modernization Act (MMA) and the American Recovery and Reinvestment Act (ARRA). The framework of CER and PCORI describes multiple elements which are vested in all 3 regulations including stakeholder involvement, public participation, and open transparent decision-making process. Overall, PCORI is much more elaborate with significant involvement of stakeholders, transparency, public participation, and open decision-making. However, there are multiple issues concerning the operation of such agencies in the United States including the predecessor of Agency for Healthcare Research and Quality (AHRQ), the Agency for Healthcare Policy and Research (AHCPR), AHRQ Effectiveness Health Care programs, and others. The CER in the United States may be described at cross-roads or at the beginnings of a scientific era of CER and evidence-based medicine (EBM). However the United States suffers as other countries, including the United Kingdom with its National Health Services (NHS) and National Institute for Health and Clinical Excellence (NICE), with major misunderstandings of methodology, an inordinate focus on methodological assessment, lack of understanding of the study design (placebo versus active control), lack of involvement of clinicians, and misinterpretation of the evidence which continues to be disseminated. Consequently, PCORI and CER have been described as government-driven solutions without following the principles of EBM with an extensive focus on costs rather than quality. It also has been stated that the central planning which has been described for PCORI and CER, a term devised to be acceptable, will be used by third party payors to override the physician’s best medical judgement and patient’s best interest. Further, stakeholders in PCORI are not scientists, are not balanced, and will set an agenda with an ultimate problem of comparative effectiveness and PCORI that it is not based on medical science, but rather on political science and not even under congressional authority, leading to unprecedented negative changes to health care. Thus, PCORI is operating in an ad hoc manner that is incompatible with the principles of evidence-based practice. This manuscript describes the framework of PCORI, and the role of CER and its impact on interventional pain management. Key words: Patient-Centered Outcomes Research Institute (PCORI), comparative effectiveness research (CER), National Institute for Health and Clinical Excellence (NICE), Patient Protection and Affordable Care Act (ACA), Medicare Modernization Act (MMA), American Recovery and Reinvestment Act (ARRA), interventional pain management, interventional techniques, evidencebased medicine, systematic reviews.