Evaluation of bottom-up interventions targeting community-dwelling frail older people in Belgium: methodological challenges and lessons for future comparative effectiveness studies.

BACKGROUND: Optimizing the organization of care for community-dwelling frail older people is an important issue in many Western countries. In Belgium, a series of complex, innovative, bottom-up interventions was recently designed and implemented to help frail older people live at home longer. As the effectiveness of these interventions may vary between different population groups according to their long-term care needs, they must be evaluated by comparison with a control group that has similar needs. METHODS: The goal was to identify target groups for these interventions and to establish control groups with similar needs and to explore, per group, the extent to which the utilization of long-term care is matched to needs. We merged two databases: a clinical prospective database and the routine administrative database for healthcare reimbursements. Through Principal Component Analysis followed by Clustering, the intervention group was first stratified into disability profiles. Per profile, comparable control groups for clinical variables were established, based on propensity scores. Using chi-squared tests and logistic regression analysis, long-term care utilization at baseline was then compared per profile and group studied. RESULTS: Stratification highlighted five disability profiles: people with low-level limitations; people with limitations in instrumental activities of daily life and low-level of cognitive impairment; people with functional limitations; people with functional and cognitive impairments; and people with functional, cognitive, and behavioral problems. These profiles made it possible to identify long-term care needs. For instance, at baseline, those who needed more assistance with hygiene tasks also received more personal nursing care (P < 0.05). However, there were some important discrepancies between the need for long-term care and its utilization: while 21% of patients who were totally dependent for hygiene tasks received no personal nursing care, personal nursing care was received by 33% of patients who could perform hygiene tasks. CONCLUSIONS: The disability profiles provide information on long-term care needs but not on the extent to which those needs are met. To assess the effectiveness of interventions, controls at baseline should have similar disability profiles and comparable long-term care utilization. To allow for large comparative effectiveness studies, these dimensions should ideally be available in routine databases.

Optimizing the leveraging of real-world data to improve the development and use of medicines.

Health research, including health outcomes and comparative effectiveness research, is on the cusp of a golden era of access to digitized real-world data, catalyzed by the adoption of electronic health records and the integration of clinical and biological information with other data. This era promises more robust insights into what works in health care. Several barriers, however, will need to be addressed if the full potential of these new data are fully realized; these will involve both policy solutions and stakeholder cooperation. Although a number of these issues have been widely discussed, we focus on the one we believe is the most important-the facilitation of greater openness among public and private stakeholders to collaboration, connecting information and data sharing, with the goal of making robust and complete data accessible to all researchers. In this way, we can better understand the consequences of health care delivery, improve the effectiveness and efficiency of health care systems, and develop advancements in health technologies. Early real-world data initiatives illustrate both potential and the need for future progress, as well as the essential role of collaboration and data sharing. Health policies critical to progress will include those that promote open source data standards, expand access to the data, increase data capture and connectivity, and facilitate communication of findings.

Traumatic Brain Injury Rehabilitation Comparative Effectiveness Research: Introduction to the Traumatic Brain Injury-Practice Based Evidence Archives Supplement.

This supplement of the Archives of Physical Medicine and Rehabilitation is devoted to the Traumatic Brain Injury-Practice Based Evidence study, the first practice-based evidence study, to our knowledge, of traumatic brain injury rehabilitation. The purpose of this preface is to place this study in the broader context of comparative effectiveness research and introduce the articles in the supplement.

Were There "Additional Foreseeable Risks" in the SUPPORT Study? Lessons Not Learned from the ARDSnet Clinical Trials.

Even though the interventions were adapted from standard clinical practice, the way they were provided meant that the care given infants in the study was distinctly different from standard care, with different risk profiles. Parents should have been informed about those differences.

SUPPORT and the Ethics of Study Implementation: Lessons for Comparative Effectiveness Research from the Trial of Oxygen Therapy for Premature Babies.

The design of SUPPORT has been widely misunderstood. This confusion has driven much of the debate about the trial - and threatens the whole enterpise of comparative effectiveness research.

Changing patterns of use of osteoporosis medications in the years after launch: implications for comparative effectiveness research.

PURPOSE: Newly marketed medications may be used selectively in patients with more severe disease. Changes in patterns of use following a drug's introduction to the market can greatly influence results from non-experimental comparative effectiveness research. We sought to explore this issue by characterizing trends in oral and injectable prescription drug claims for the prevention and treatment of osteoporosis. METHODS: We examined a post-menopausal population of women age 55 years and older in the Truven Health Analytics MarketScan® Databases. We used propensity score (PS) methods to describe how predictors of new users of oral and injectable osteoporosis medications change over time. RESULTS: We found that injectable osteoporosis medications tended to be used more selectively in the higher risk patients shortly after launch. Over time, they appeared to be used increasingly in lower risk patients. CONCLUSION: If disease severity is incompletely captured in the data, comparative effectiveness of novel osteoporosis medications may be difficult to accurately estimate, particularly when medications are new to market.

Collaborative chronic care models for mental health conditions: cumulative meta-analysis and metaregression to guide future research and implementation.

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.

Comparative effectiveness research for prostate cancer radiation therapy: current status and future directions.

Comparative effectiveness research aims to help clinicians, patients and policymakers make informed treatment decisions under real-world conditions. Prostate cancer patients have multiple treatment options, including active surveillance, androgen deprivation therapy, surgery and multiple modalities of radiation therapy. Technological innovations in radiation therapy for prostate cancer have been rapidly adopted into clinical practice despite relatively limited evidence for effectiveness showing the benefit for one modality over another. Comparative effectiveness research has become an essential component of prostate cancer research to help define the benefits, risks and effectiveness of the different radiation therapy modalities currently in use for prostate cancer treatment.

Reengineering acute episodic and chronic care delivery: the Geisinger Health System experience.

Comparative effectiveness research (CER) represents an evolution in clinical decision-making research that allows for the study of heterogeneous groups of patients with complex diseases processes. It has foundations in decision science, reliability science, and health care policy research. Health care finance will increasingly rely on CER for guidance in the coming years. There is increasing awareness of the importance of decreasing unwarranted variation in health care delivery. In the past 7 years, Geisinger Health System has performed broad reengineering of its acute episodic and chronic care delivery models utilizing macrosystem-level application of CER principles. These provider-driven process initiatives have resulted in significant improvement across all segments of care delivery, improved patient outcomes, and notable cost containment. These programs have led to the creation of novel pricing models, and when "hardwired" throughout a care delivery system, they can lead to correct medical decision making by 100% of providers in all patient encounters. Neurosurgery as a specialty faces unique challenges and opportunities with respect to broad adoption and application of CER techniques.

Wisconsin Spine Outcome Study-Pilot: preliminary data.

OBJECT: Given the pragmatic difficulties in developing randomized controlled trials in patients with disorders of the spine, the Wisconsin Spine Outcome Group has adopted the use of a prospective registry design to perform comparative effectiveness research on treatments for degenerative lumbar disorders. The goal of the Wisconsin Spine Outcome Study-Pilot (WISPOS-P) was to establish a Web-based, Health Insurance Portability and Accountability Act-compliant registry and to implement a patient registration paradigm that demonstrates at least 80% compliance in collecting pre- and posttreatment data in patients with lumbar disorders, regardless of the treatment they receive. The primary outcome measures were the percentage of patients with lumbar spine disorders who completed a Web-based survey preappointment, and at 1 and 3 months postappointment; the percentage of patients receiving a physician-assigned diagnosis in the registry; and the success of electronic data transition from the Web-based interface to a locally controlled registry. METHODS: The WISPOS-P uses a prospective, diagnosis-based registry design. A universally accessible and secure Internet-based data management platform was created that accrues self-entered patient data on validated disability indices, including the visual analog pain scale, Oswestry Disability Index (ODI), and the 36-Item Short Form Health Survey questionnaire. Data were obtained on patients, preappointment and at 1 and 3 months postappointment, regardless of the treatment rendered. A physician-entered diagnosis was assigned to each patient for data stratification. RESULTS: One hundred patients were invited into the WISPOS-P; 90 patients participated, and 10 withdrew for various reasons. Eighty-eight of 90 patients were assigned a diagnosis by the evaluating physician. Preliminary and qualitative assessment of the data shows that the major difference between patients who withdrew from the study and those who participated was the number of days between study invitation and clinic appointment (median 11 vs 20.5 days, respectively). In evaluating patients by mode of survey completion, the 2 largest groups were those who completed their intake forms electronically before their clinic appointment and those who used the paper format. The median age of patients electronically completing this survey was 14.34 years younger than those using the paper format. A significantly higher proportion of patients who completed their forms electronically had listed an email address. The 3 major diagnoses were disc disease (32 patients), stenosis (24 patients), and nonsurgical pain of spinal origin (14 patients). Patients with stenosis were older than those in the other 2 groups. Patients with nonsurgical pain of spinal origin had lower ODI scores compared with the other 2 groups. CONCLUSIONS: A diagnosis-based registry design is effective in collecting pretreatment data for patients with lumbar disorders. When stratified by diagnosis, comparative effectiveness analyses can be performed to identify optimum treatments for lumbar disorders given individual patient characteristics. The WISPOS-P has established a mechanism and proof of principle for the participation of patients in an outcomes registry.

From comparative effectiveness research to patient-centered outcomes research: policy history and future directions.

Containing growth in health care expenditures is considered to be essential to improving both the long-term fiscal outlook of the federal government and the future affordability of health care in the US. As health care expenditures have increased, so too have concerns about the quality of health care. Better information on the clinical effectiveness of alternative treatments and other interventions is needed to improve the quality of care and restrain growth in expenditures. This article explains the key role played by the federal government in defining the context and process of comparative effectiveness research as well as its funding. Subsequently, the article explores the mission, priorities, and research agenda of the Patient-Centered Outcomes Research Institute, which is an independent, nonprofit corporation established in 2010 by the Patient Protection and Affordable Care Act.

Publication patterns of comparative effectiveness research in spine neurosurgery.

OBJECT: The purpose of this study was to investigate publication patterns for comparative effectiveness research (CER) on spine neurosurgery. METHODS: The authors searched the PubMed database for the period 1980-2012 using the key words "cost analysis," "utility analysis," "cost-utility," "outcomes research," "practical clinical research," "comparator trial," and "comparative effectiveness research," linked with "effectiveness" and "spine neurosurgery." RESULTS: From 1980 through April 9, 2012, neurosurgery CER publications accounted for 1.38% of worldwide CER publications (8657 of 626,330 articles). Spine neurosurgery CER accounted for only 0.02%, with 132 articles. The journal with the greatest number of publications on spine neurosurgery CER was Spine, followed by the Journal of Neurosurgery: Spine. The average annual publication rate for spine neurosurgery CER during this period was 4 articles (132 articles in 33 years), with 68 (51.52%) of the 132 articles being published within the past 5 years and a rising trend beginning in 2008. The top 3 contributing countries were the US, Turkey, and Japan, with 68, 8, and 7 articles, respectively. Only 8 regular articles (6.06%) focused on cost analysis. CONCLUSIONS: There is a paucity of publications using CER methodology in spine neurosurgery. Few articles address the issue of cost analysis. The promotion of continuing medical education in CER methodology is warranted. Further investigations to address cost analysis in comparative effectiveness studies of spine neurosurgery are crucial to expand the application of CER in public health.

Comparative effectiveness of treatments for cerebral arteriovenous malformations: trends in nationwide outcomes from 2000 to 2009.

OBJECT: The development of multimodality approaches for the treatment of cerebral arteriovenous malformations (AVMs), including microsurgery, endovascular therapy, and radiosurgery, has shifted modern treatment paradigms in the last 10 years. This study examines these changes in detail from a nationwide perspective. METHODS: The authors examined data from 2001 to 2009 in the Nationwide Inpatient Sample (NIS) database, and they assessed the safety, quality, and cost-effectiveness, including the total number of discharges, discharge proportion, length of stay, and hospital charges. The authors also examined patient demographics (including age, sex, income level, and insurance), hemorrhage status at presentation, and trends in open surgical and endovascular treatment. RESULTS: A total of 33,997 inpatient admissions for patients with a primary diagnosis of intracranial AVM were identified, with a mean of 4191 patients admitted annually. The mean hospital charges increased 2-fold over the study period without significant differences in outcomes. There were substantial differences between surgical, endovascular, radiosurgical, and multimodality treatments. The proportion of AVMs treated microsurgically remained stable over this period, while the proportion treated endovascularly dramatically increased in size, and the data demonstrate important patient-level distinctions among groups. Outcomes and complication profiles were significantly different between treatment modalities and were impacted by age and hemorrhage status. CONCLUSIONS: Charges associated with treatment of cerebral AVMs to the payer and society have increased dramatically over the first decade of the 21st century without clear improvements in quality parameters. However, analysis of the 3 primary treatment modalities has demonstrated differences and warrants further investigation to understand which patient population would benefit maximally from each. Unfortunately, with only imprecise measurements of quality in health care delivery, it remains imperative to develop national databases in which parameters, such as survival, functional outcomes, quality of life, and complication rates, can be assessed to examine the value of care delivered in a more meaningful way. Demonstrating an ever-increasing value of delivered health care will be imperative in our evolving health care system.

Comparative effectiveness research: policy and politics.

Comparative effectiveness research (CER) is the basis for some of the fiercest rhetoric of the current political era. While it is a relatively old and previously academic pursuit, CER may well become the foundation upon which the future of health care in the US is based. The actual impact of CER on-and uptake among-doctors, patients, hospitals, and health insurers, however, remains to be seen. Political considerations and compromises have led to the removal of key aspects of CER implementation from policy legislation to prevent alienating stakeholders critical to the success of health care reform. Health care providers, including specialists such as neurosurgeons, will need to understand both the policies and political implications of CER as its practices becomes an indelible part of the future health care landscape.

Breaking up is hard to do: why disinvestment in medical technology is harder than investment.

Healthcare technology is a two-edged sword - it offers new and better treatment to a wider range of people and, at the same time, is a major driver of increasing costs in health systems. Many countries have developed sophisticated systems of health technology assessment (HTA) to inform decisions about new investments in new healthcare interventions. In this paper, we question whether HTA is also the appropriate framework for guiding or informing disinvestment decisions. In exploring the issues related to disinvestment, we first discuss the various HTA frameworks which have been suggested as a means of encouraging or facilitating disinvestment. We then describe available means of identifying candidates for disinvestment (comparative effectiveness research, clinical practice variations, clinical practice guidelines) and for implementing the disinvestment process (program budgeting and marginal analysis (PBMA) and related techniques). In considering the possible reasons for the lack of progress in active disinvestment, we suggest that HTA is not the right framework as disinvestment involves a different decision making context. The key to disinvestment is not just what to stop doing but how to make it happen - that is, decision makers need to be aware of funding disincentives.

Type and trends in outcomes research in breast cancer between 2000 and 2007.

BACKGROUND: Interest in outcomes research (OR) derives from the need to know the value and the effectiveness of health interventions, especially for oncology. We focused our research on OR in breast cancer, providing an overview of the trend of publications. PATIENTS AND METHODS: We carried out a Medline search to retrieve all articles in English published from 2000 through 2007. The abstracts were reviewed and classified according to the research topics and the primary design of the trial. RESULTS: We selected 405 articles: their number remained constant until 2003, rose during 2004-2005 and decreased during the last 2 years. The most common topic was surgery (n = 234), alone or in association with other interventions. The category more investigated was the process. Clinical outcomes, and among them disease-free survival, were more frequent than other outcomes. The median value of 2007 Impact Factor of the journals publishing the selected references was 2.466 (range 0.272-25.547) and the median value of Citation Index was 8 (range 0-143). CONCLUSIONS: Our research showed a decreasing interest in OR during the more recent years. We are hopeful that it will regenerate interest, particularly by the light of the funds allocated to the comparative effectiveness research in the United States.

Comparative effectiveness research in urology.

BACKGROUND: Comparative effectiveness research (CER) has come to remain at the forefront of political and scientific debates of health care. The aim of this article is to describe the attributes of CER and implications to the field of urology. METHODS: The authors reviewed the published urology literature and recent CER publications including the Institute of Medicine reports. RESULTS: CER is defined as the evaluation of the relative efficacy of diagnostic tests, treatments, and health care services against the current standards of care, with the goal of reducing health care costs while simultaneously improving quality. Significant government funds were recently devoted to CER efforts. The Institute of Medicine identified 25 of the most urgently needed areas for research of which three pertain to Urology: screening for prostate cancer, comparing effectiveness of treatments for localized prostate cancer, and use of imaging in diagnosing, staging, and monitoring cancer patients. Some of the important required strategies to formulate successful CER include involving health care stakeholders to aid in selecting appropriate topics, utilizing study populations that represent real life practice, and the development of large-scale national databases and registries. CONCLUSION: Several topics of urologic care have already been identified in the first quartile of CER priorities, and many others need to be added. Following the proposed research, designs for CER should be done while abiding by robust methodology, maintaining transparency, and effectively translating research into clinical practice.

[Achievements and problems of modern trials of antihypertensive drugs].

Most important value of lowering of substantially elevated arterial pressure (AP) for improvement of outcomes in patients with arterial hypertension (AH) was convincingly confirmed by large truly placebo controlled randomized clinical trials (RCT) with the use of mainly diuretics, and/or beta-adrenoblockers in the 60-80ths. Later comparative RCT confirmed equal antihypertensive efficacy of 5 main drug classes relative to AP level in brachial artery. In this review we discuss merit of auxiliary class-specific properties of antihypertensive agents potentially affecting prognosis besides AP lowering. We also discuss problems related to decline of significance of quantitative criteria of AH and consideration of AP level in general context of cardiovascular risk; problems of external validity of RCT; extrapolation of RCT results obtained in patients with complicated AH and very high cardiovascular risk on young patients with uncomplicated AH; significance of hard and surrogate end points.

Hospital-based comparative effectiveness centers: translating research into practice to improve the quality, safety and value of patient care.

Hospital-based comparative effectiveness (CE) centers provide a model that clinical leaders can use to improve evidence-based practice locally. The model is used by integrated health systems outside the US, but is less recognized in the US. Such centers can identify and adapt national evidence-based policies for the local setting, create local evidence-based policies in the absence of national policies, and implement evidence into practice through health information technology (HIT) and quality initiatives. Given the increasing availability of CE evidence and incentives to meaningfully use HIT, the relevance of this model to US practitioners is increasing. This is especially true in the context of healthcare reform, which will likely reduce reimbursements for care deemed unnecessary by published evidence or guidelines. There are challenges to operating hospital-based CE centers, but many of these challenges can be overcome using solutions developed by those currently leading such centers. In conclusion, these centers have the potential to improve the quality, safety and value of care locally, ultimately translating into higher quality and more cost-effective care nationally. To better understand this potential, the current activity and impact of hospital-based CE centers in the US should be rigorously examined.