Comparative effectiveness research (CER) aims to provide decision-makers the evidence needed to evaluate the benefits and harms of alternative clinical management strategies. CER has become a national priority, with considerable new research funding allocated. Cardiovascular disease is a priority area for CER. This workshop report provides an overview of CER methods, with an emphasis on practical clinical trials and observational treatment comparisons. The report also details recommendations to the National Heart Lung and Blood Institute for a new framework for evidence development to foster cardiovascular CER, and specific studies to address eight clinical issues identified by the Institute of Medicine as high priorities for cardiovascular CER.
comparative effectiveness; research methods; clinical trials
This paper describes our process to engage regional stakeholders for prioritizing comparative effectiveness research (CER) in cancer diagnostics. We also describe a novel methodology for incorporating stakeholder data and input to inform the objectives of selected CER studies.
Materials & methods
As an integrated component to establishing the infrastructure for community-based CER on diagnostic technologies, we have assembled a regional stakeholder group composed of local payers, clinicians and state healthcare representatives to not only identify and prioritize CER topics most important to the western Washington State region, but also to inform the study design of selected research areas. A landscape analysis process combining literature searches, expert consultations and stakeholder discussions was used to identify possible CER topics in cancer diagnostics. Stakeholders prioritized the top topics using a modified Delphi/group-nominal method and a standardized evaluation criteria framework to determine a final selected CER study area. Implementation of the selected study was immediate due to a unique American Recovery and Reinvestment Act funding structure involving the same researchers and stakeholders in both the prioritization and execution phases of the project. Stakeholder engagement was enhanced after study selection via a rapid analysis of a subset of payers’ internal claims, coordinated by the research team, to obtain summary data of imaging patterns of use. Results of this preliminary analysis, which we termed an ‘internal analysis,’ were used to determine with the stakeholders the most important and feasible study objectives.
Stakeholders identified PET and MRI in cancers including breast, lung, lymphoma and colorectal as top priorities. In an internal analysis of breast cancer imaging, summary data from three payers demonstrated utilization rates of advanced imaging increased between 2002 and 2009 in the study population, with a great deal of variability in use between different health plans. Assessing whether breast MRI affects treatment decisions was the top breast cancer study objective selected by the stakeholders. There were other high-priority research areas including whether MRI use improved survival that were not deemed feasible with the length of follow-up time following MRI adoption.
Continuous stakeholder engagement greatly enhanced their enthusiasm for the project. We believe CER implementation will be more successful when undertaken by regional stakeholders.
breast cancer; cancer imaging; comparative effectiveness research; research prioritization; stakeholder involvement
Stakeholder engagement is fundamental to comparative effectiveness research (CER), but lacks consistent terminology. This paper aims to define stakeholder engagement and present a conceptual model for involving stakeholders in CER.
Materials & methods
The definitions and model were developed from a literature search, expert input and experience with the Center for Comparative Effectiveness Research in Cancer Genomics, a proof-of-concept platform for stakeholder involvement in priority setting and CER study design.
Definitions for stakeholder and stakeholder engagement reflect the target constituencies and their role in CER. The ‘analytic-deliberative’ conceptual model for stakeholder engagement illustrates the inputs, methods and outputs relevant to CER. The model differentiates methods at each stage of the project; depicts the relationship between components; and identifies outcome measures for evaluation of the process.
While the definitions and model require testing before being broadly adopted, they are an important foundational step and will be useful for investigators, funders and stakeholder groups interested in contributing to CER.
cancer genomics; comparative effectiveness research; consumer participation; deliberative methods; public participation; stakeholder engagement; stakeholders
The increasing burden of chronic diseases presents not only challenges to the knowledge and expertise of the professional medical community, but also highlights the need to improve the quality and relevance of clinical research in this domain. Many patients now turn to complementary and integrative medicine (CIM) to treat their chronic illnesses; however, there is very little evidence to guide their decision-making in usual care. The following research recommendations were derived from a CIM Stakeholder Symposium on Comparative Effectiveness Research (CER): (1) CER studies should be made a priority in this field; (2) stakeholders should be engaged at every stage of the research; (3) CER study designs should highlight effectiveness over efficacy; (4) research questions should be well defined to enable the selection of an appropriate CER study design; (5) the CIM community should cultivate widely shared understandings, discourse, tools, and technologies to support the use and validity of CER methods; (6) Effectiveness Guidance Documents on methodological standards should be developed to shape future CER studies. CER is an emerging field and its development and impact must be reflected in future research strategies within CIM. This stakeholder symposium was a first step in providing systematic guidance for future CER in this field.
The Division of Lung Diseases of the National Heart, Lung and Blood Institute (NHLBI) recently held a workshop to identify gaps in our understanding and treatment of childhood lung diseases and to define strategies to enhance translational research in this field. Leading experts with diverse experience in both laboratory and patient-oriented research reviewed selected areas of pediatric lung diseases, including perinatal programming and epigenetic influences;mechanisms of lung injury, repair, and regeneration; pulmonary vascular disease (PVD); sleep and control of breathing; and the application of novel translational methods to enhance personalized medicine. This report summarizes the proceedings of this workshop and provides recommendations for emphasis on targeted areas for future investigation. The priority areas identified for research in pediatric pulmonary diseases included: (1) epigenetic and environmental influences on lung development that program pediatric lung diseases, (2) injury, regeneration, and repair in the developing lung, (3) PVD in children, (4) development and adaptation of ventilatory responses to postnatal life, (5) nonatopic wheezing: aberrant large airway development or injury? (6) strategies to improve assessment, diagnosis, and treatment of pediatric respiratory diseases, and (7) predictive and personalizedmedicine for children.
epigenetics; pediatric; respiratory; lung disease
There is a need for more Comparative Effectiveness Research (CER) to strengthen the evidence base for clinical and policy decision-making. Effectiveness Guidance Documents (EGD) are targeted to clinical researchers. The aim of this EGD is to provide specific recommendations for the design of prospective acupuncture studies to support optimal use of resources for generating evidence that will inform stakeholder decision-making.
Document development based on multiple systematic consensus procedures (written Delphi rounds, interactive consensus workshop, international expert review). To balance aspects of internal and external validity, multiple stakeholders including patients, clinicians and payers were involved.
Recommendations focused mainly on randomized studies and were developed for the following areas: overall research strategy, treatment protocol, expertise and setting, outcomes, study design and statistical analyses, economic evaluation, and publication.
The present EGD, based on an international consensus developed with multiple stakeholder involvement, provides the first systematic methodological guidance for future CER on acupuncture.
Comparative effectiveness research; Effectiveness guidance document; Acupuncture
Comparative effectiveness research (CER) has received considerable research attention in recent months, and efforts to promote CER are part of the newly enacted Patient Protection and Affordable Care Act. In this paper, we define CER, how it complements traditional efficacy research in asthma, and discuss how CER can help provide the basis for rational decision-making about the care of individual patients with asthma and how best to deliver this care in real-world settings. We present information about the challenges and opportunities to conduct CER, including enhanced patient registries for observational CER and effectiveness trials (also called pragmatic trials). We discuss the urgent need to define the appropriate methodologies for CER and to develop and prioritize a research agenda for CER studies in asthma with the help of a diverse group of stakeholders.
Comparative effectiveness research; comparative clinical effectiveness research; observational studies; effectiveness trials; efficacy trials; pragmatic trials; explanatory trials; asthma
For decades, investigators have conducted innovative research on shared decision-making (SDM), helping patients and clinicians to discuss health decisions and balance evidence with patients' preferences for possible outcomes of options. In addition, investigators have developed and used rigorous methods for conducting comparative effectiveness research (CER), comparing the benefits and risks of different interventions in real-world settings with outcomes that matter to patients and other stakeholders. However, incorporating CER findings into clinical practice presents numerous challenges. In March 2012, we organized a conference at Washington University in St Louis (MO, USA) aimed at developing a network of researchers to collaborate in developing, conducting and disseminating research about the implementation of CER through SDM. Meeting attendees discussed conceptual similarities and differences between CER and SDM, challenges in implementing CER and SDM in practice, specific challenges when engaging SDM with unique populations and examples of ways to overcome these challenges. CER and SDM are related processes that emphasize examining the best clinical evidence and how it applies to real patients in real practice settings. SDM can provide one opportunity for clinicians to discuss CER findings with patients and engage in a dialog about how to manage uncertainty about evidence in order to make decisions on an individual patient level. This meeting highlighted key challenges and suggested avenues to pursue such that CER and SDM can be implemented into routine clinical practice.
comparative effectiveness research; shared decision-making
Comparative Effectiveness Research (CER) has the potential to transform the current healthcare delivery system by identifying the most effective medical and surgical treatments, diagnostic tests, disease prevention methods and ways to deliver care for specific clinical conditions. To be successful, such research requires the identification, capture, aggregation, integration, and analysis of disparate data sources held by different institutions with diverse representations of the relevant clinical events. In an effort to address these diverse demands, there have been multiple new designs and implementations of informatics platforms that provide access to electronic clinical data and the governance infrastructure required for inter-institutional CER. The goal of this manuscript is to help investigators understand why these informatics platforms are required and to compare and contrast six, large-scale, recently funded, CER-focused informatics platform development efforts. We utilized an 8-dimension, socio-technical model of health information technology use to help guide our work. We identified six generic steps that are necessary in any distributed, multi-institutional CER project: data identification, extraction, modeling, aggregation, analysis, and dissemination. We expect that over the next several years these projects will provide answers to many important, and heretofore unanswerable, clinical research questions.
Methods; Comparative Effectiveness Research; Organization and Administration; Medical Informatics; Methods
Recognizing the importance of improving lung health through lung disease research, the National Heart, Lung, and Blood Institute (NHLBI) convened a workshop of multidisciplinary experts for the following purpose: (1) to review the current scientific knowledge underlying the basis for treatment of adults and children with pulmonary vascular diseases (PVDs); (2) to identify gaps, barriers, and emerging scientific opportunities in translational PVD research and the means to capitalize on these opportunities; (3) to prioritize new research directions that would be expected to affect the clinical course of PVDs; and (4) to make recommendations to the NHLBI on how to fill identified gaps in adult and pediatric PVD clinical research. Workshop participants reviewed experiences from previous PVD clinical trials and ongoing clinical research networks with other lung disorders, including acute respiratory distress syndrome, chronic obstructive lung disease, and idiopathic pulmonary fibrosis, as well. Bioinformatics experts discussed strategies for applying cutting-edge health information technology to clinical studies. Participants in the workshop considered approaches in the following broad concept areas: (1) improved phenotyping to identify potential subjects for appropriate PVD clinical studies; (2) identification of potential new end points for assessing key outcomes and developing better-designed PVD clinical trials; and (3) the establishment of priorities for specific clinical research needed to advance care of patients with various subsets of PVDs from childhood through adulthood. This report provides a summary of the objectives and recommendations to the NHLBI concentrating on clinical research efforts that are needed to better diagnose and treat PVDs.
clinical trials; pediatrics; pulmonary hypertension; pulmonary vascular changes
We analyzed the extent to which comparative effectiveness research (CER) organizations share terms for designs, analyzed coverage of CER designs in Medical Subject Headings (MeSH) and Emtree, and explored whether scientists use CER design terms.
We developed local terminologies (LTs) and a CER design terminology by extracting terms in documents from five organizations. We defined coverage as the distribution over match type in MeSH and Emtree. We created a crosswalk by recording terms to which design terms mapped in both controlled vocabularies. We analyzed the hits for queries restricted to titles and abstracts to explore scientists' language.
Pairwise LT overlap ranged from 22.64% (12/53) to 75.61% (31/41). The CER design terminology (n = 78 terms) consisted of terms for primary study designs and a few terms useful for evaluating evidence, such as opinion paper and systematic review. Patterns of coverage were similar in MeSH and Emtree (gamma = 0.581, P = 0.002).
Stakeholder terminologies vary, and terms are inconsistently covered in MeSH and Emtree. The CER design terminology and crosswalk may be useful for expert searchers. For partially mapped terms, queries could consist of free text for modifiers such as nonrandomized or interrupted added to broad or related controlled terms.
The clinical utility is uncertain for many cancer genomic applications. Comparative effectiveness research (CER) can provide evidence to clarify this uncertainty.
To identify approaches to help stakeholders make evidence-based decisions, and to describe potential challenges and opportunities using CER to produce evidence-based guidance.
We identified general CER approaches for genomic applications through literature review, the authors’ experiences, and lessons learned from a recent, seven-site CER initiative in cancer genomic medicine. Case studies illustrate the use of CER approaches.
Evidence generation and synthesis approaches include comparative observational and randomized trials, patient reported outcomes, decision modeling, and economic analysis. We identified significant challenges to conducting CER in cancer genomics: the rapid pace of innovation, the lack of regulation, the limited evidence for clinical utility, and the beliefs that genomic tests could have personal utility without having clinical utility. Opportunities to capitalize on CER methods in cancer genomics include improvements in the conduct of evidence synthesis, stakeholder engagement, increasing the number of comparative studies, and developing approaches to inform clinical guidelines and research prioritization.
CER offers a variety of methodological approaches to address stakeholders’ needs. Innovative approaches are needed to ensure an effective translation of genomic discoveries.
evidence synthesis; evidence generation; stakeholder; clinical utility
The upper airway serves three important functions: respiration, swallowing, and speech. During development it undergoes significant structural and functional changes that affect its size, shape, and mechanical properties. Abnormalities of the upper airway require prompt attention, because these often alter ventilatory patterns and gas exchange, particularly during sleep when upper airway motor tone and ventilatory drive are diminished. Recognizing the relationship of early life events to lung health and disease, the National Heart, Lung, and Blood Institute (NHLBI), with cofunding from the Office of Rare Diseases (ORD), convened a workshop of extramural experts, from many disciplines. The objective of the workshop was: (1) to review the state of science in pediatric upper airway disorders; (2) to make recommendations to the Institute to fill knowledge gaps; (3) to prioritize new research directions; and (4) to capitalize on scientific opportunities. This report provides recommendations that could facilitate translation of basic research findings into practice to better diagnose, treat, and prevent airway compromise in children.
With the ongoing debate over health care reform in the United States, public health and policy makers have paid growing attention to the need for comparative effectiveness research (CER). Recent allocation of federal funds for CER represents a significant move toward increased evidence-based practice and better informed allocation of constrained health care resources; however, there is also heated debate on how, or whether, CER may contribute to controlling national health care expenditures. Economic evaluation, in the form of cost-effectiveness or cost-benefit analysis, is often an aspect of CER studies, yet there are no recommendations or guidelines for providing clinical investigators with the necessary skills to collect, analyze, and interpret economic data from clinical trials or observational studies. With an emphasis on multidisciplinary research, the Clinical and Translational Science Award (CTSA) consortium and institutional CTSA sites serve as an important resource for training researchers to engage in CER. In this article, the authors discuss the potential role of CTSA sites in integrating economic evaluation methods into their comparative effectiveness education goals, using the Columbia University Medical Center CTSA as an example. By allowing current and future generations of clinical investigators to become fully engaged not only in CER, but in the economic evaluations that result from such analyses, CTSA sites can help develop the necessary foundation for advancing research to guide clinical decision making and efficient use of limited resources.
Comparative effectiveness research (CER) can efficiently and rapidly generate new scientific evidence and address knowledge gaps, reduce clinical uncertainty, and guide health care choices. Much of the potential in CER is driven by the application of novel methods to analyze existing data. Despite its potential, several challenges must be identified and overcome so that CER may be improved, accelerated, and expeditiously implemented into the broad spectrum of cancer care and clinical practice.
To identify and characterize the challenges to cancer CER, we reviewed the literature and conducted semi-structured interviews with 41 cancer CER researchers at the Agency for Healthcare Research and Quality (AHRQ)'s Developing Evidence to Inform Decisions about Effectiveness (DEcIDE) Cancer CER Consortium.
A number of datasets for cancer CER were identified and differentiated into an ontology of eight categories, and characterized in terms of strengths, weaknesses, and utility. Several themes emerged during development of this ontology and discussions with CER researchers. Dominant among them was accelerating cancer CER and promoting the acceptance of findings, which will necessitate transcending disciplinary silos to incorporate diverse perspectives and expertise. Multidisciplinary collaboration is required including those with expertise in non-experimental data, outcomes research, clinical trials, epidemiology, generalist and specialty medicine, survivorship, informatics, data, and methods, among others.
Recommendations highlight the systematic, collaborative identification of critical measures; application of more rigorous study design and sampling methods; policy-level resolution of issues in data ownership, governance, access, and cost; and development and application of consistent standards for data security, privacy, and confidentiality.
With the increasing availability of therapeutic strategies (drugs, devices, disease management systems) and the growing complexity of health care delivery, there is an attendant need for objective evidence of the tangible benefits of different approaches to care. This is particularly true for patients with heart failure, a common, morbid, and resource-intensive disease. There are few well-proven therapies for patients with acute decompensation or for patients with normal LVEF. Comparative effectiveness research (CER) offers an important avenue for making progress in the field. However, CER, like any well-designed research program, requires the explicit articulation of clinically important outcomes to be compared. For patients with heart failure, there is a need to develop endpoint measures that capture the totality of potential benefits and risks for alternative therapeutic approaches. Ultimately, for one therapeutic approach to be considered superior to another, it must improve one of three relevant endpoints: make patients live longer, make them feel better, or save money without adversely affecting the other two goals. Importantly, these outcomes must be measured directly and surrogates should be avoided, even if such surrogates appear to be associated with clinically meaningful, patient-centered outcomes. In this review, we discuss the available CER endpoint domains from both a clinical and a statistical perspective, summarize the wide variety of endpoints used in CER studies, and suggest steps for greater standardization of endpoints across CER studies of patients with heart failure.
Endpoints/end points; congestive heart failure; comparative effectiveness research; study design
To improve formulary design processes and support payers in providing more effective health care, policy makers should consider involving commercial payers in the development of comparative effectiveness research and creation of research and treatment guidelines.
The perspective of commercial payers on comparative effectiveness research (CER) has not been well researched. This study aims to describe how US commercial payers use and value CER for formulary decision making in different disease states.
We recruited 20 medical and pharmaceutical directors from national and regional plans who are involved in pharmaceutical and therapeutics committees to participate in the study. We conducted in-depth qualitative interviews with the payers and asked them to rate the usefulness of CER study types across various disease states and market conditions. The results were analyzed for thematic content.
Our findings indicate that payers are interested in a broad range of CER study types, are unsatisfied with the current state of CER, and would like to partner with research groups to develop research and treatment guidelines to better leverage CER. Payers value CER less so in oncology than in other disease states because of limitations in their ability to manage oncology therapies.
To improve formulary design processes and support payers in providing more effective health care, policy makers should consider involving commercial payers in the development of CER as well as in the creation of research and treatment guidelines.
Comparative effectiveness research and pragmatic clinical trials are valued methods to address the limitations of traditional randomized trials, answer questions of cost-effectiveness or noninferiority, and inform data-driven dialogue and decision making by stakeholders.
Although much effort has focused on identifying national comparative effectiveness research (CER) priorities, little is known about the CER priorities of community-based practitioners treating patients with advanced cancer. CER priorities of managed-care–based clinicians may be valuable as reflections of both payer and provider research interests.
We conducted mixed methods interviews with 10 clinicians (five oncologists and five pharmacists) at five health plans within the Health Maintenance Organization Cancer Research Network. We asked, “What evidence do you most wish you had when treating patients with advanced cancer?” and questioned participants on their impressions and knowledge of CER and pragmatic clinical trials (PCTs). We conducted qualitative analyses to identify themes across interviews.
Ninety percent of participants had heard of CER, 20% had heard of PCTs, and all rated CER/PCTs as highly relevant to patient and health plan decision making. Each participant offered between three and 10 research priorities. Half (49%) involved head-to-head treatment comparisons; another 20% involved comparing different schedules or dosing regimens of the same treatment. The majority included alternative outcomes to survival (eg, toxicity, quality of life, noninferiority). Participants cited several limitations to existing evidence including lack of generalizability, funding biases, and rapid development of new treatments.
Head-to-head treatment comparisons remain a major evidence need among community-based oncology clinicians, and CER/PCTs are highly valued methods to address the limitations of traditional randomized trials, answer questions of cost-effectiveness or noninferiority, and inform data-driven dialogue and decision making by all stakeholders.
The Clinical and Translational Science Award (CTSA) Workgroup for Comparative Effectiveness Research (CER) Education, Training, and Workforce Development identified a need to delineate the competencies that practitioners and users of CER for patient centered outcomes research, should acquire. With input from CTSA representatives and collaborators, we began by describing the workforce. We recognize the workforce that conduct CER and the end users who use CER to improve the health of individuals and communities. We generated a preliminary set of competencies and solicited feedback from the CER representatives at each member site of the CTSA consortium. We distinguished applied competencies (i.e., skills needed by individuals who conduct CER) from foundational competencies that are needed by the entire CER workforce, including end users of CER. Key competency categories of relevance to both practitioners and users of CER were: 1) Asking relevant research questions; 2) Recognizing or designing ideal CER studies; 3) Executing or using CER studies; 4) Using appropriate statistical analyses for CER; and 5) Communicating and disseminating CER study results to improve health. While CER is particularly broad concept, we anticipate that these preliminary, relatively generic competencies will be used in tailoring curricula to individual learners from a variety of programmatic perspectives.
The Clinical and Translational Science Awards (CTSAs) were initiated to improve the conduct and impact of NIH's research portfolio, transforming training programs and research infrastructure at academic institutions and creating a nationwide consortium. They provide a model for translating research across disciplines and offer an efficient and powerful platform for comparative effectiveness research (CER), an effort that has long struggled but enjoys renewed hope under health care reform. CTSAs include study design and methods expertise, informatics, and regulatory support; programs in education, training, and career development in domains central to CER; and robust programs in community engagement, both of the general public and of clinical practice communities.
Albert Einstein College of Medicine of Yeshiva University and Montefiore Medical Center have entered a formal partnership that places their CTSA at a critical intersection for clinical and translational research. Their CTSA leaders were asked to develop a strategy for enhancing CER activities, and in 2010 they developed a model that encompasses four broadly defined “compartments” of research strength that must be coordinated for this enterprise to succeed: evaluation and health services research, biobehavioral research and prevention, efficacy studies and clinical trials, and social science and implementation research.
This article provides historical context for CER, elucidates Einstein-Montefiore’s CER model and strategic planning efforts, and illustrates how a CTSA can provide a vision, leadership, coordination, and services to support an academic health center’s collaborative efforts to develop a robust CER portfolio and thus contribute to the national effort to improve health and health care.
Randomized clinical trials, particularly for comparative effectiveness research (CER), are frequently criticized for being overly restrictive or untimely for health-care decision making.
Our prospectively designed REsearch in ADAptive methods for Pragmatic Trials (RE-ADAPT) study is a ‘proof of concept’ to stimulate investment in Bayesian adaptive designs for future CER trials.
We will assess whether Bayesian adaptive designs offer potential efficiencies in CER by simulating a re-execution of the Antihypertensive and Lipid Lowering Treatment to Prevent Heart Attack Trial (ALLHAT) study using actual data from ALLHAT.
We prospectively define seven alternate designs consisting of various combinations of arm dropping, adaptive randomization, and early stopping and describe how these designs will be compared to the original ALLHAT design. We identify the one particular design that would have been executed, which incorporates early stopping and information-based adaptive randomization.
While the simulation realistically emulates patient enrollment, interim analyses, and adaptive changes to design, it cannot incorporate key features like the involvement of data monitoring committee in making decisions about adaptive changes.
This article describes our analytic approach for RE-ADAPT. The next stage of the project is to conduct the re-execution analyses using the seven prespecified designs and the original ALLHAT data.
This paper discusses an important element that is missing from the existing algorithm of preconception care, namely, comparative effectiveness research (CER). To our knowledge, there has been limited assessment of the comparative effectiveness of diverse interventions that promote preconception health, conditions under which these are most effective, for which particular populations, and their comparative costs. CER can improve the decision making process for the funding, development, implementation, and evaluation of comprehensive preconception care programs, specifically by identifying the most effective interventions with acceptable costs to society. This paper will examine the framework behind preconception care and how the inclusion of comparative effectiveness research and evaluation into the existing algorithm of preconception care could foster improvement in maternal and child health. We discuss challenges and opportunities regarding the utilization of CER in the decision making process in preconception health, and finally, we provide recommendations for future directions.
Preconception care; Comparative effectiveness; Cost-effectiveness; Decision making
Federal legislation placed Comparative Effectiveness Research (CER) and Patient Centered Outcomes Research (PCOR) at the center of current and future national investments in health care research. The Role of CER and PCOR in emergency care has not been well described. This report proposes an agenda for researchers and health care providers to consider CER and PCOR methods and results in order to improve the care for patients who seek, use, and require emergency care. This objective will be accomplished by: 1) exploring the definitions, frameworks, and nomenclature for CER and PCOR; 2) describing a conceptual model for CER in emergency care, 3) identifying specific opportunities and examples of emergency care related CER; and 4) categorizing current and planned funding for CER and PCOR that can include emergency care delivery.
Increasing numbers of Americans are living with multiple chronic conditions (MCCs) and disabilities. Addressing health care needs of persons with MCCs or disabilities presents challenges on many levels. For health services researchers, priorities include (1) considering MCCs and disabilities in comparative effectiveness research (CER) and assessing quality of care; and (2) identifying and evaluating the data needed to conduct CER, performance measure development, and other research to inform health policy and public health decisions concerning persons with MCCs or disabilities. Little information is available to guide CER or treatment choices for persons with MCCs or disabilities, however, because they are typically excluded from clinical trials that produce the scientific evidence base. Furthermore, most research funding flows through public and private agencies oriented around single organ systems or diseases. Likely changes in the data landscape—notably wider dissemination of electronic health records (EHRs) and moving toward updated coding nomenclatures—may increase the information available to monitor health care service delivery and quality for persons with MCCs and disabilities. Generating this information will require new methods to extract and code information about MCCs and functional status from EHRs, especially narrative texts, and incorporating coding nomenclatures that capture critical dimensions of functional status and disability.
Chronic conditions; disability; functional status; comparative effectiveness research; quality measurement; health information technology; coding nomenclatures
Comparative effectiveness research (CER) documents important differences in antidepressants in terms of efficacy, safety, cost, and burden to the patient. Decision aids can adapt this evidence to help patients participate in making informed choices. In turn, antidepressant therapy will more likely reflect patients’ values and context, leading to improved adherence and mood outcomes.
The objective of this study is to develop the Depression Medication Choice decision aid for use during primary care encounters, and to test its efficacy by conducting a clustered practical randomized trial comparing the decision aid to usual depression care in primary care practices.
We will use a novel practice-based, patient-centered approach based on participatory action research that involves a multidisciplinary team of designers, investigators, clinicians, patient representatives, and other stakeholders for the development of the decision aid. We will then conduct a clustered practical randomized trial enrolling clinicians and their patients (n = 300) with moderate to severe depression from rural, suburban and inner city primary care practices (n = 10). The intervention will consist of the use of the depression medication choice decision aid during the clinical encounter. This trial will generate preliminary evidence of the relative impact of the decision aid on patient involvement in decision making, decision making quality, patient knowledge, and 6-month measures of medication adherence and mental health compared to usual depression care.
Upon completion of the proposed research, we will have developed and evaluated the efficacy of the decision aid depression medication choice as a novel translational tool for CER in depression treatment, engaged patients with depression in their care, and refined the process by which we conduct practice-based trials with limited research footprint.
Clinical Trials.gov: NCT01502891
Depression; Comparative effectiveness research; Shared decision making; Decision aid; Implementation; Randomized controlled trial