Public Policy Corner
From the bedside to the community: Comparative effectiveness, health services, and implementation research†
Potential conflict of interest: Nothing to report.
Do, or do not. There is no try! (Jedi Master Yoda1)
For 60 years, the American Association for the Study of Liver Diseases (AASLD) and its membership have championed the following activities: (1) fundamental research elucidating hepatopathophysiology, (2) translational discovery identifying preventive and therapeutic targets, and (3) clinical studies demonstrating the efficacy of medical and surgical interventions in patients with liver disease. Moreover, the AASLD is at the forefront of establishing evidence-based guidelines for the diagnosis and management of a broad range of liver conditions.2 Despite the enormous scientific and medical progress in the management of liver disease, a substantial gap remains between the recommended standards of hepatology care and the care actually delivered to patients within our communities. Consequently, we call for greater investment in research focused on the development and implementation of innovative approaches to the systematic delivery of high-quality hepatology care to all Americans.
As reported in a previous AASLD Public Policy Corner,3 the final, least tested, and most important steps for effectively applying scientific and medical discoveries to improve health are the application of evidence-based guidelines to health practice [termed phase 3 translational (T3) research] and the evaluation of real-world outcomes of specific health care interventions [termed phase 4 translational (T4) research].3 Although hepatologists have contributed to a deep understanding of disease pathophysiology [phase 0 translational research and phase 1 translational (T1) research] and the optimal management of individual patients with liver disease [phase 2 translational (T2) research], the development and implementation of health care delivery strategies (T3 research) and the analysis of their effects on clinical outcomes (T4 research) have been limited. The National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) has instituted the Action Plan for Liver Disease Research. This plan includes the following specific goals, which will require T3 and T4 research to be successfully achieved4:
Improve the success rate of hepatitis C therapy.
Develop effective therapies that can be used in both alcoholic and nonalcoholic fatty liver disease.
Develop regimens of antiviral therapy that are effective in the long-term management of hepatitis B.
Develop sensitive and specific means of screening individuals at high risk for early hepatocellular carcinoma.
Improve the safety and define the optimal use of living donor liver transplantation.
Decrease the mortality rate from liver disease.
This NIDDK framework is committed to advancing prevention, effective therapy, screening, safety, optimization of limited resources (e.g., liver transplantation), standardization of care, and decreased mortality from liver disease within 10 years. Accomplishing these goals calls for not only the elucidation of hepatopathophysiology and discovery of improved therapies but also the development and implementation of effective health care delivery approaches.
This Public Policy Corner article looks at three emerging research fields that focus on different aspects of improving health care delivery to the community: (1) comparative effectiveness research, (2) health services research, and (3) implementation science research.
Comparative Effectiveness Research (CER)
The importance of translating medical advances to the community has been recognized by the US government through its recent and unprecedented action of turning to physicians and researchers to develop and test different health care delivery strategies within the broad community.5, 6 CER evaluates an intervention's effectiveness in real-life clinical situations, whereas more traditional clinical research examines efficacy, which is defined as “the probability of benefit from a medical technology applied for a given medical problem under ideal conditions of use” (i.e., a clinical trial).7, 8 CER goes by a variety of names, including patient-centered outcomes research. CER grants and contracts are challenging researchers to rigorously test health care interventions within multisite health care delivery systems. Approximately 1.1 billion dollars of the American Recovery and Reinvestment Act, Washington's recent economic stimulus package, has been allocated to CER projects.9, 10 As the US Congress debates the establishment of health insurance for 50 million uninsured Americans and the ways in which to pay for it, CER has emerged as a process for developing and testing strategies that contain health care costs while improving quality.11, 12 This is a unique opportunity for hepatology researchers who have the skill, experience, and passion to create and evaluate different clinical interventions in actual practice. The AASLD mission statement, “to advance the science and practice of hepatology, liver transplantation and hepatobiliary surgery, thereby promoting liver health and optimal care of patients with liver and biliary tract diseases,” underscores the importance of merging scientific knowledge with optimal evidence-based health care delivery practices. A recent commentary in HEPATOLOGY13 and an AASLD 2010 public policy statement14 support exploring CER research to improve liver health, enhance medical treatment, reduce health disparities, and prevent disease.
Hepatobiliary disease has been designated by the Institute of Medicine as an area for focused CER.15 Within the field of digestive diseases, liver disease and viral hepatitis together compose the second leading diagnosis on hospital discharge records and the second leading cause of death.16 In addition, the prevalence of nonalcoholic fatty liver disease is estimated to be 23% for Europe and North America and will likely increase with the rising rate of obesity.17 The rising US burden of cirrhosis and hepatocellular carcinoma related to the long-term consequences of hepatitis C virus (HCV) and fatty liver disease will further increase the economic impact of health care.18, 19 As a result, chronic liver disease is a significant health and economic burden in the United States and globally.16, 20 Moreover, inequities in health care access and quality are well documented in populations suffering from chronic liver disease.3, 21-28 Clearly, interventions aimed toward improving the quality of and access to hepatology care throughout the population will have a significant impact, particularly with the expansion of treatments for viral hepatitis and the rising prevalence of nonalcoholic fatty liver disease.17 Hence, disorders of the liver represent a ripe target for CER.
Health services and implementation science research are investigative fields closely related to CER that develop and assess innovative health care delivery models. Physicians trained in these fields will be in a strong position to take advantage of new grant funding, such as that coordinated by the Patient-Centered Outcomes Research Institute recently authorized as part of the Patient Protection and Affordable Care Act.29, 30 More importantly, these individuals will be poised to address problems of cost and inequity within our country's health care system. Hence, health services and implementation science investigators will also be valuable catalysts for improvements in hepatology care for clinicians and their patients.
Health Services Research
Health services research examines the structure, process, and resulting outcomes of health care in order to improve care delivery. A commonly accepted definition of health services research is that it is “the multidisciplinary field of scientific investigation that studies how social factors, financing systems, organizational structures and processes, health technologies and personal behaviors affect access to healthcare, the quality and cost of healthcare, and ultimately our health and well being.”31 In contrast to basic research, in which reagents, methods, and products are typically well defined, the variables and outcomes of health service research can be complex and difficult to define. The complex and fluid environment of health services research encourages fresh ideas, novel methods, and original approaches, which make it an exciting and meaningful scientific field for clinicians and investigators enthusiastic about advancing the quality of hepatology care in real-life practice.
Implementation Science Research
Implementation science research, which overlaps health services research, is the rigorous study of methods for promoting the systematic uptake of clinical research findings and other evidence-based practices into routine practice and thereby improving the quality and effectiveness of health care.32 Implementation science research encompasses the following: (1) methods for implementing evidence-based practices or delivery systems to improve clinical outcomes, particularly in care settings in which quality/performance is inadequate; (2) formative evaluations that include rigorous assessment processes designed to identify potential and actual influences on the progress and effectiveness of implementation efforts; (3) interventions that include studying a method or technique to facilitate change and thereby the adoption of best practice recommendations; and (4) evaluations of implementation strategies or programs rather than individual interventions.32 The essential importance of implementation science research has been formally recognized within the NIDDK Action Plan for Liver Disease Research.4
New Approaches: Disruptive Innovations and Public Health
To meet the 10-year aims of the NIDDK action plan and especially the expectation that health care discoveries will reach the wider community, we believe that hepatologists and hepatology researchers will need to broaden their approaches to research and health care delivery. We suggest that the translation of scientific and medical research into medical practice will be facilitated by the application of disruptive innovations and public health partnerships, strategies that have succeeded in other fields.
In other industries, great effort is expended to discover disruptive innovations that competitively transform the market. Disruptive innovations fundamentally expand access to services by substantially changing the cost-performance ratio. Examples of disruptive innovations include personal computers and internet purchasing of goods and services, which have dramatically transformed the performance of diverse industries. This type of business model thinking has been proposed as an important next step in ushering affordable, accessible, and high-quality health care.33 Examples of potentially disruptive innovations in health care include electronic referral management, retail clinics, telemedicine, and medical tourism.34-37 These innovations and others provide new models and options that might be harnessed by hepatologists and hepatology investigators to increase system-wide access to hepatology care and its quality. We suggest the value of infusing the concept of disruptive innovation into academic and biomedical research models to facilitate the development of T3 and T4 research activities.
Since 1900, the average lifespan of the US population has been lengthened by more than 30 years; most of this gain can be attributed to the application of medical, technological, and sociological research findings to public health measures within the community.38, 39Ten Great Public Health Achievements—United States, 1900-1999,38, 39 a 1999 publication by the Centers for Disease Control and Prevention, includes vaccination, motor vehicle safety, safer workplaces, control of infectious diseases, declines in deaths from coronary heart disease and stroke, safer and healthier foods, healthier mothers and babies, family planning, fluoridation of drinking water, and the recognition of tobacco as a health hazard. Fields in which health care interventions are integrated with public health strategies appear to have the greatest potential for completing the National Institutes of Health (NIH) bench to bedside to community progression. We suggest that public health approaches and partnerships may facilitate the accomplishment of the objectives of the NIDDK 10-year plan targeting the prevention and care of viral and fatty liver conditions and their complications for all Americans.
How We Need to Move From Bench to Bedside to the Community: Two Examples
Improve the Success Rate of Hepatitis C Therapy.
Despite the swift progression in our knowledge of hepatitis C from the identification of the virus in 198940 to the development of evidence-based guidelines for its management and treatment in 1997,41 the rates of screening, access to treatment, and successful outcomes of treatment are unacceptably low.42, 43 Indeed, the three primary recommendations of the recent Institute of Medicine report on the prevention and control of HCV are (1) to improve disease surveillance, (2) to improve patient and community education, and (3) to integrate and enhance viral hepatitis services.44 Furthermore, the AASLD and NIH recognize that it is especially difficult to initiate and manage antiviral treatment in several populations that are disproportionately affected by hepatitis C, including current or recent illicit drug users and patients without stable housing.45, 46 We have yet to establish health care models in the United States that effectively identify, treat, and manage the diverse individuals infected with HCV. With the advent of promising new HCV therapies, it is critical to improve the current health care delivery systems for hepatitis C. We believe that improved viral hepatitis surveillance, management, and treatment outcomes will require the use of public health strategies and the adoption of disruptive innovations, such as integrated care models or HCV treatment delivery within methadone or homeless clinics.47-49 It is incumbent upon hepatology investigators with health service research and implementation science expertise to develop effective strategies and models of viral hepatitis surveillance, management, and treatment.
Develop Effective Therapies That Can Be Used in Patients With Fatty Liver Disease.
In contrast to HCV, fatty liver disorders are biologically more heterogeneous with a more complex pathophysiology. This may explain the longer interval between the characterization of the syndrome in 198050 and the only recent demonstration of efficacious therapies.51 Indeed, the development of specific treatments for these disorders is challenged by the fact that fatty liver conditions are typically only one manifestation of an underlying metabolic or toxic pathology. Despite concerted efforts to understand the pathophysiology of nonalcoholic fatty liver disorders, identify targets for therapy, and perform rigorous efficacy trials,4, 52, 53 the number of individuals with fatty liver disorders and their complications continues to swell. This growth in patients with fatty liver disease is largely due to societal factors that have increased the number of obese or diabetic individuals.54 Although further laboratory and clinical trial investigation (T1 and T2 research) focused on fatty liver disorders is important, it seems certain that comprehensive and effective management of nonalcoholic fatty liver disease will require public health measures to control obesity and diabetes. Such T3 interventions might include initiatives to resolve the paucity of fresh fruits and vegetables in lower socioeconomic areas, to reintroduce physical education within many of our public school systems, and to develop collaborative partnerships between health care systems and local community groups.55–57 In addition to public health interventions, the enormous number of individuals affected by fatty liver disorders suggests that disruptive innovations that qualitatively expand access to proven health care services will be essential to addressing fatty liver disease for all affected people. The sheer magnitude and complexity of hepatological conditions such as hepatitis C and fatty liver disease suggest that disruptive innovations and public health strategies applied by hepatologists and hepatology investigators within the context of comparative effectiveness, health services, and implementation science research will be critical to their prevention and control.
To improve the health of all Americans with liver disease, we need to bridge the gap between the care that each patient should receive and the actual practice of hepatology within the community. Hence, greater investment in comparative effectiveness, health services, and implementation science research is needed. Toward this objective, the public policy committee will do the following:
- 1Advocate for the development of curricula and funding for the training of junior and mid-level investigators in comparative effectiveness, health services, and implementation science research directed toward patients with liver disease.
- 2Promote increased federal support (e.g., NIH, Agency for Healthcare Research and Quality, Centers for Disease Control and Prevention, Veterans Administration, and Health Resources and Services Administration) and private support of comparative effectiveness, health services, and implementation science research.
- 3Encourage the presentation and publication of rigorously performed, clinically significant comparative effectiveness, health services, and implementation science research at the Liver Meeting and in Hepatology.
- 4Organize a series of programs at Digestive Disease Week and the Liver Meeting for the AASLD membership focused on comparative effectiveness, health services, and implementation science research.
Knowing is not enough; we must apply. Willing is not enough; we must do. (Goethe58)