The clinical-economic trial: Promise,problems, and challenges

The clinical-economic trial is a study design that is appearing with greater frequency in medical and public health literature. Some experienced investigators view these trials with skepticism; to policy makers they represent a promising step in the control of rising health care costs. The success of clinical-economic trials in meeting the important goal of more rational and efficient use of health care resources will depend on the strengths and limitations of the research method. As part of a report to the Office of Technology Assessment of the U.S. Congress on new health care assessment techniques, we describe the reasons why economic data collection and analysis are being considered in clinical trials, identify and discuss various designs and methods for gathering economic trial data, and evaluate the strengths and limitations of different methods for providing sound data for decision making on appropriate use of health care interventions. Because of the potential significance and increasing visibility of such research, experts in research methods should give more attention to methodological research for clinical-economic trials. Future efforts should be directed at comparing different techniques for collecting data, examining the incremental value of precision in economic measurements and ensuring appropriate interpretation of data from clinical-economic trials.     

Efficacy of clinical laboratory: Therapeutic TAT,safety, and medical errors

Health care services rely on continued technological advances and management of the operational systems for optimum reduction of medical errors. Significant gains in health care outcomes as indicated by recorded increases in life expectancies have been achieved due to the availability and application of technological advances for medical services. The inadequacies in the application of these systems for maximum benefit of the health care systems have however been the subject of recent publications dealing with patient safety and medical errors [1], [2], [3], [4]. Estimates by the Institute of Medicine (IOM) indicate that approximately 44,000–98,000 deaths occur each year as a consequence of inadequate safety and failure to prevent errors in the health care system. This puts medical errors in the top four leading causes of deaths per the IOM report. Other studies in the USA states of Colorado, Utah, and New York suggest that medical errors occur in 2–4% of hospitalizations. The paper by Raab et al. denoted a 6.7% discrepancy between original report and secondary case review, and 5% of the discrepancies have modest to significant effect on patient care [Raab SS, Grzybicki DM, Zarbo RJ, Meier FA, Geyer SJ, Jensen C. Anatomic pathology databases and patient safety. Arch Pathol Lab Med 2005;129:459–66]. This presentation focuses on the health care safety and medical errors relative to clinical laboratory. The impact of laboratory operations with resultant delays in test turn around times (TAT) and other laboratory errors on the health care services are presented. The role of governmental (US Department of Health and Human Services) and non-governmental regulatory agencies (CAP, AACC, IFCC, CLSI, etc) in mitigating these clinical laboratory errors is discussed. The use of payment system as a mechanism for improving the quality of laboratory services is also presented to illustrate the checks and balance systems aimed at reduction of medical errors. The presentation will conclude with the recommendation that majority of the clinical laboratory delays in turn around time and other errors can be prevented with appropriate analytical systems and operational processes under the overall guidance of the right regulatory agencies.     

Footprints of Clinical Pharmacology in Turkey: Past,Present, and Future

Clinical pharmacology is an interdisciplinary field that encompasses all components of the relationship between drugs and humans. All clinical pharmacology professionals aim to support an improved quality of drug-oriented health services by providing teaching, research, and routine health care services that ensure more tolerable and more effective, suitable, and cost-effective use of drugs. Subsections of clinical pharmacology include clinical trials, pharmacoepidemiology and drug use, pharmacovigilance, pharmacoeconomics, the rational use of medicines, pharmacotherapy consultation, drug monitoring, counseling to authorities and industry, pharmacogenetics, and other practices. By approaching these subsections as part of 3 main aspects of clinical pharmacology—education, research, and health care—this review aims to provide local and international practitioners with detailed information about clinical pharmacology practices in Turkey and to contribute to building the network of communication and collaboration. This review also aims to play an encouraging and pioneering role for Turkey's national community and other countries that have not yet made clinical pharmacology functional in improving the quality of health services, promoting the dissemination of rational use of medicines, helping the set-up of clinical pharmacology organizations, enhancing quantity and quality of the clinical pharmacology workforce, and increasing the infrastructural facilities.     

Key concepts of clinical trials: a narrative review

The recent focus of federal funding on comparative effectiveness research underscores the importance of clinical trials in the practice of evidence-based medicine and health care reform. The impact of clinical trials not only extends to the individual patient by establishing a broader selection of effective therapies, but also to society as a whole by enhancing the value of health care provided. However, clinical trials also have the potential to pose unknown risks to their participants, and biased knowledge extracted from flawed clinical trials may lead to the inadvertent harm of patients. Although conducting a well-designed clinical trial may appear straightforward, it is founded on rigorous methodology and oversight governed by key ethical principles. In this review, we provide an overview of the ethical foundations of trial design, trial oversight, and the process of obtaining approval of a therapeutic, from its pre-clinical phase to post-marketing surveillance. This narrative review is based on a course in clinical trials developed by one of the authors (DJM), and is supplemented by a PubMed search predating January 2011 using the keywords "randomized controlled trial," "patient/clinical research," "ethics," "phase IV," "data and safety monitoring board," and "surrogate endpoint." With an understanding of the key principles in designing and implementing clinical trials, health care providers can partner with the pharmaceutical industry and regulatory bodies to effectively compare medical therapies and thereby meet one of the essential goals of health care reform.     

Exploring the continuum: medical information to effective clinical practice. Paper II. Towards aetiology-centred clinical practice

Contemporary clinical practice increasingly functions within a disease management paradigm aimed at finding and implementing therapeutic interventions that demonstrate efficacy in clinical trials. Disease prevention, elucidation of illness aetiology and proactive health promotion have taken a back seat. Current clinical care often includes a 'fast-food' type of medical encounter, which frequently neglects disease causality. The medical community is presently challenged by unique administrative and professional adversities as well as undue commercial influence; these factors contribute to a lethargic response to escalating rates of chronic illness and to mainstream medicine's relative inattention to emerging research about disease aetiology. Individual medical practitioners and the medical establishment must strategically seek to advance patient health and maintain professional relevancy by a renewed emphasis on the following principles: patient-based clinical care, aetiology-centred medicine, and proactive health promotion.     

Epidemiologic trends and costs of fragmentation

The data that were reviewed in this article documented that in health systems, which manage behavioral health disorders independently from general medical disorders, the estimated 10% to 30% of patients with behavioral health service needs can expect (1) poor access or barriers to medical or mental health care; (2) when services are available, most provided will not meet minimum standards for expected outcome change; and (3) as a consequence of (1) and (2), medical and behavioral disorders will be more persistent with increased complications, will be associated with greater disability, and will lead to higher total health care and disability costs than will treatment of patients who do not have behavioral health disorders. This article proposes that these health system deficiencies will persist unless behavioral health services become an integral part of medical care (ie, integrated). By doing so, it creates a win-win situation for virtually all parties involved. Complex patients will receive coordinated general medical and behavioral health care that leads to improved outcomes. Clinicians and the hospitals that support integrated programs will be less encumbered by cross-disciplinary roadblocks as they deliver services that augment patient outcomes. Health plans (insurers) will be able to decrease administrative and claims costs because the complex patients who generate more than 80% of service use will have less complicated claims adjudication and better clinical outcomes. As a result, purchaser premiums, whether government programs, employers, or individuals, will decrease and the impact on national budgets will improve. Ongoing research will be important to assure that application of the best clinical and administrative practices are used to achieve these outcomes.     

Multisite randomized controlled trials in health services research: scientific challenges and operational issues

Although well-designed randomized controlled trials (RCT) provide the strongest evidence regarding causation, only relatively recently have they been used by health services researchers to study the organization, delivery, quality, and outcomes of care. More recent yet is the extension of multisite RCTs to health services research. Such studies offer numerous methodological advantages over single-site trials: (1) enhanced external validity; (2) greater statistical power when studying conditions with a low incidence or prevalence, small event rate in the outcome (eg, mortality), and/or large variance in the outcome (eg, health care costs); and (3) rapid recruitment to provide health care organizations and policy makers with timely results. This paper begins by outlining the advantages of multisite RCTs over single-site trials. It then discusses both scientific challenges (ie, standardizing eligibility criteria, defining and standardizing the intervention, defining usual care, standardizing the data collection protocol, blinded outcome assessment, data management and analysis, measuring health care costs) and operational issues (ie, site selection, randomization procedures, patient accrual, maintaining enthusiasm, oversight) posed by multisite RCTs in health services research. Recommendations are offered to health services researchers interested in conducting such studies.     

Factors affecting the use of medical, mental health, alcohol, and drug treatment services by homeless adults

This study describes use of medical, mental health, alcohol, and drug services by 832 adult residents of the New York City homeless shelter system and examines associations between service use during the past three months and an array of predisposing, enabling, and need factors. Utilization rates were 23% for medical services, 13% for mental health services, and 10 and 7.5% for alcohol and drug treatment services, respectively. Service contacts were more often hospitals than ambulatory care clinics. Logistic regression analyses revealed that need factors were stronger predictors of all four types of service use. Predisposing factors other than education and black ethnic status were not significant, and the enabling factor of enrollment in Medicaid and/or Medicare was significant only for use of medical and drug services. Among the need factors, measures of mental health status were analyzed as indices of distress to test a stress-utilization model of prediction for all four types of service use. While these measures did not predict use of nonmental health services, physical health problems were associated with use of all four types of services. Implications for future health services research and for service delivery to the homeless are discussed, including the need for more information on availability of services and on psychosocial and cultural characteristics of homeless persons that may affect their help-seeking behavior.     

Surgical procedures and devices should be evaluated in the same way as medical therapy

This paper is a personal essay that starts and ends with the message that surgical procedures and devices should be evaluated in the same way as medical therapies, namely, by randomized clinical trials (RCTs). I discuss, with particular attention to surgical procedures and devices, the objections raised against RCTs in medical decision-making, a schema for utilizing the traditional phases of RCTs in the evaluation of surgical procedures and devices, the importance of RCTs to FDA approval, financial compensation, and health care costs, the impact of RCTs on clinical practice, the role of RCTs in academia, teaching, and research, and the surgeon's obligation to participate in a leadership role in RCTs. The belief is expressed that national funding of health care should mandate allocations for RCTs and that such expenditures, as well as the spending of health care dollars on the basis of the outcomes of such trials, will not only improve patient management in the shortest time but will eventually reduce health care costs. The RCT, by selecting effective and safe surgical procedures and devices, as well as diets and drugs, is the best means science has to assess the validity of patient management.     

Use of pathology services in re-engineered clinical pathways

A significant proportion of pathology tests ordered in hospital are unnecessary. Specific measures targeting the increasing appropriateness of pathology service use have been shown to decrease overall ordering of laboratory tests. However, it is not clear whether general programmes to improve quality of care will have any impact on the use of pathology services. Use of pathology services was compared within two separate prospective controlled clinical trials of re-engineered clinical pathways for both elective (surgical) patients and acute unplanned (medical) admissions. Trial One was a controlled trial of a re-engineered surgical service. Booked patients in the treatment group were admitted on the day of surgery, care was guided by a clinical pathway, and patients were discharged early with domiciliary post-acute care. Controls were admitted on the day before surgery, treated according to usual practice and discharged according to surgeons' preferences. In Trial Two, acute medical patients admitted to hospital through the Emergency Department (ED) were randomised into a treatment (Hospital in the Home) or a control (inpatient) care pathway. In both studies, patients on the re-engineered clinical pathways were well matched demographically and clinically. Health outcomes and satisfaction ratings were comparable. Seventy per cent fewer laboratory tests were ordered in the elective surgery intervention group (P < 0.0001), while the treatment group of the acute medical patients had 25% fewer tests ordered (P = 0.0133). Pooled results also showed a significantly lower rate of test ordering (P < 0.001) for the treatment group (Mann-Whitney U-Wilcoxon ranked sum test). The findings of these audits of controlled, prospective trials suggested overuse of laboratory tests in New South Wales public hospitals, and that savings can be generated by using clinical pathways and applying clinical criteria to the ordering of tests without adversely affecting health outcomes.     

Mental Health Aspects of Emergency Medical Services for Children Summary of a Consensus Conference

OBJECTIVE: To address the mental health needs of children involved in emergency medical services (EMS). METHODS: A multidisciplinary consensus conference convened to identify mental health needs of children and their families related to pediatric medical emergencies, to examine the impact of psychological aspects of emergencies on recovery and satisfaction with care, and to delineate research questions related to mental health aspects of medical emergencies involving children. RESULTS: The consensus group found that psychological and behavioral factors affect physical as well as emotional recovery after medical emergencies. Children's reactions are critically affected by age and developmental level, characteristics of the emergency medical event, and parent reactions. As frontline health care providers, EMS staff members are in a pivotal position to recognize and effectively manage the mental health needs of patients and their families. CONCLUSIONS: Ecological changes in emergency departments, such as linkages to mental health follow-up services, training of EMS providers and mental health professionals, and focused research that provides an empirical basis for practice, are necessary components for improving current standards of health care.     

Quality assurance and technology assessment: pieces of a larger puzzle

Increasing integration of health care and health services research has resulted in an overlap between disciplines involved in the evaluation of clinical practice. We have examined the relationships of quality assurance (QA), medical technology assessment (TA), clinical epidemiology (CE) and evidence-based medicine (EBM) from an historical perspective. Clinicians, patients and administrators need local information on effectiveness of routine care. Information from trials alone, efficacy data, will not suffice nor can it be culled from administrative databases designed for other purposes. The current activities of QA should be therefore be expanded to include the study of the effectiveness of interventions in terms of appropriateness of use, patient outcomes and study of the determinants of outcomes, as seen from the perspective of doctors, patients, administrators and policy makers, using data collected during the course of routine patient care. With the assistance of information technology, with methodological support and multidisciplinary cooperation, clinicians can do this as part of a more broadly defined clinical research. Quality assurance and TA both evolved with the objective of studying clinical care but have quite different historical roots, complementary perspectives and objectives, use different methods and involve a different set of practitioners. Quality assurance is a type of 'formative' evaluation conducted in the clinical setting using indicators as flags of process or outcome events of interest, simple surveys and audit studies. Its primary aim is to achieve incremental improvement rather than to simply pass judgement. An important underlying assumption is that health care behaves as a complex dynamic system. Technology assessment, a form of summative evaluation with an orientation towards policy, synthesises information from formal scientific studies of efficacy in the form of clinical trials and studies of cost-effectiveness. For the evaluation of the impact of any technology more complex than a drug, the complementary contributions of both of these disciplines is needed, and QA and TA should work cooperatively in tandem with the support of CE and EBM.     

Embedding nursing and therapy consultantship: the case of stroke consultants

Aims and objectives. As the basis for the design of career development opportunities for current and aspiring nursing and therapy consultants, we aimed to explore the factors that shape how these roles have embedded in UK stroke services. Background. The non‐medical consultant role has been introduced into UK health care services to provide opportunities for experienced practitioners to progress their careers in clinical practice. Whilst there have been evaluations of the impact of the role on service delivery, little attention has been paid to the pathways towards consultantship. Design. An exploratory design, incorporating focus group discussions, was used to address the research questions. Participating consultants, both nurses and allied health professionals, worked in stroke services, although it is anticipated that the results will have wider application. Methods. Two focus groups were held with non‐medical consultants in stroke from across the UK. Participants had the opportunity to comment on an interim paper prior to publication of the results. Thirteen consultants took part in the study. Results. A lack of consensus about the nature of clinical expertise and a diverse range of pathways towards consultantship were identified. Health care policy had presented the opportunity for consultants to be entrepreneurial in the development of stroke services, although this had limited the scope for the development of professional knowledge. Inflexible programmes to support aspiring consultants may limit the opportunities to develop these entrepreneurial skills. Conclusions. This study challenges health care organizations and the education and research departments that support them to think creatively in the way that the non‐medical consultant role is embedded, and that this should draw on the commitment of existing consultants to support succession planning. Relevance to clinical practice. The identification of those aspects of career pathways that current consultants have found to be helpful will be useful in designing opportunities for aspiring consultants.     

The impact of social stressors and social networks on pediatric medical care use

Substantial differences in the use of pediatric medical resources reinforce the need for identifying and understanding factors that influence the use of medical services for children. This research assesses the simultaneous impact of sociodemographic characteristics, health attitudes and beliefs, psychologic distress, social stressors, and social networks on the use of pediatric acute care services during a 12-month period. Using a prospective longitudinal study design, data were obtained on 513 children and their families enrolled in a prepaid group practice. Linear modeling results showed that health attitudes and social networks were important predictors of acute care utilization in addition to child's age, birth order, baseline health status, and ethnic group. The authors were able to show significant effects for network size, dispersion, and tendency to use one's network members. Individuals with large nondispersed networks are more likely to use pediatric health services, apparently due to the transmission of the networks' pro-medical care health beliefs. Also the tendency to call on network members modifies an individual's propensity to seek care for minor pediatric medical problems and can make a difference by as much as 1.6 visits per year per child for acute care episodes.     

Using electronic health record data for substance use Screening,Brief Intervention,and Referral to Treatment among adults with type 2 diabetes: Design of a National Drug Abuse Treatment Clinical Trials Network study

BackgroundThe Affordable Care Act encourages healthcare systems to integrate behavioral and medical healthcare, as well as to employ electronic health records (EHRs) for health information exchange and quality improvement. Pragmatic research paradigms that employ EHRs in research are needed to produce clinical evidence in real-world medical settings for informing learning healthcare systems. Adults with comorbid diabetes and substance use disorders (SUDs) tend to use costly inpatient treatments; however, there is a lack of empirical data on implementing behavioral healthcare to reduce health risk in adults with high-risk diabetes. Given the complexity of high-risk patients' medical problems and the cost of conducting randomized trials, a feasibility project is warranted to guide practical study designs.MethodsWe describe the study design, which explores the feasibility of implementing substance use Screening, Brief Intervention, and Referral to Treatment (SBIRT) among adults with high-risk type 2 diabetes mellitus (T2DM) within a home-based primary care setting. Our study includes the development of an integrated EHR datamart to identify eligible patients and collect diabetes healthcare data, and the use of a geographic health information system to understand the social context in patients' communities. Analysis will examine recruitment, proportion of patients receiving brief intervention and/or referrals, substance use, SUD treatment use, diabetes outcomes, and retention.DiscussionBy capitalizing on an existing T2DM project that uses home-based primary care, our study results will provide timely clinical information to inform the designs and implementation of future SBIRT studies among adults with multiple medical conditions.     

Integrating health services research into nursing doctoral programs: the evolution of nursing research

Traditionally, nursing research has focused on the effect of an intervention on selected patients without considering the influence of the system of care and its myriad characteristics. Health services research (HSR) focuses on organization and financing of health services; access to health care; quality of care; clinical evaluation and outcomes research; informatics and clinical decision making; practitioner, patient, and consumer behavior; health professions workforce; health policy formulation and analyses; and health care model and service use. Doctoral students can benefit from HSR's broad perspective if it is included in existing nursing curricula. Ultimately, HSR could help the nursing profession achieve the capacity to develop health policy and new systems of health care for the 21st century.     

Patterns of medical services utilization by infants discharged from a neonatal intensive care unit

A decreasing neonatal mortality rate has increased the number of infants who survive their stay in a neonatal intensive care unit only to need continued medical care. Medical services utilization by families with high-risk infants has been widely studied, but research is sparse on high-risk infants who do not receive follow-up care through medical specialists' services or speciality clinics. After dividing medical services utilization into four patterns, ranging from use of only primary care to use of three kinds of medical services, the authors analyzed a set of predictors of utilization. The factors most likely to affect pattern of utilization significantly are related to health need and illness level. The infants with more serious medical conditions diagnosed at birth were found to use the most follow-up services. No effects of location of residence or social class were found, which suggests that families had no significant problems of access to medical care services for their infants in the defined region of the study.     

Patient care cancer clinical trials at the National Cancer Institute: a resource for payers and providers

Clinical trials form the evidence base for medical decision making and may provide patients with life-threatening conditions their best chance to find an effective treatment. A growing number of states and the federal government are mandating coverage of the routine costs of cancer clinical trials, although the extent of coverage varies. Individual health plans are following suit on behalf of their beneficiaries. Trials conducted at the National Cancer Institute (NCI) are an attractive resource for payers, because NCI provides medical services at no charge, enables patient access to promising care, emphasizes continuity with patients' regular physicians, and makes the referral and enrollment process easy and efficient through its Clinical Studies Support Center's toll-free information line 1-888-NCI-1937.