Biennial Report of the Director

Overview of NIH Research Portfolio
Postclinical Translational Research

Postclinical translational research ensures that evidence-based interventions are broadly applied and accessible to those who need them most. NIH pursues this effort primarily through its support of health services research. Health services research is a multidisciplinary field, both basic and applied, that examines how social factors, financing systems, organizational structures and processes, health technologies, and personal beliefs and behaviors affect access to and utilization of healthcare, the quality and cost of healthcare, and ultimately our health and well-being. The goals of health services research are to identify the most effective ways to organize, manage, finance, and deliver high-quality care. 35

NIH undertakes a number of activities to ensure that the rich evidence base created through basic and clinical research is translated and utilized to enhance health and reduce the burdens of illness and disability. The focus of health services research supported by NIH is on optimizing the health care delivery system to supply care based on scientific evidence. As efficacious interventions are developed and tested, a more detailed understanding is needed to establish that they are effective in real world settings, including ensuring that they are adopted and implemented appropriately, and with sustained investment.

Health services research at NIH addresses topics such as institutional and organizational influences on health, including studies of the organization of and access to health care; its effectiveness in real world settings; its cost efficiency; and its social and cultural acceptability. It may also involve research related to macroeconomic phenomena (e.g., business cycles), community and neighborhood organization, and the structure and functioning of families, as well as how these variables influence the consumption and choice of health care and decision-making concerning health procedures. Finally, this category includes research on how successful approaches to the organization and delivery of health services can be translated into public policy.

35 Report of the Blue Ribbon Task Force on Health Services Research at the National Institute on Drug Abuse, 2004 https://ww2.drugabuse.gov/about/organization/nacda/HSRReport.pdf.

Partnering with Health Care Delivery Organizations

Healthcare delivery organizations are critical partners with NIH efforts to study the methods and models for adopting and sustaining evidence-based interventions. Through research within actual healthcare delivery settings, studies may provide crucial information that can help us deliver interventions faster and more effectively. An additional benefit is having access to the immense resources that healthcare delivery organizations offer, such as electronic medical records for thousands of patients. Already a number of NIH Institutes support collaborative activities between healthcare delivery organizations such as health maintenance organizations (HMOs), and biomedical researchers to implement large studies with real-world benefits.

Tackling real-world clinical issues and generating evidence that will be of immediate value to practitioners and patients is the central goal of the NIDCR-supported dental Practice-based Research Networks (PBRNs). Conducting research in dental practices draws on the experience and insight of practicing clinicians to help identify and frame research questions. Because PBRN studies address practice-based problems, their results tend to be more quickly translated into daily clinical care.

Leveraging the infrastructure of established dental practices for conducting PBRN studies also can be a powerful and cost-effective means to conduct clinical research. For example, the past decade brought reports that people who take bisphosphonates, a class of drug prescribed for osteoporosis or to treat the bone-wasting effects of cancer, can develop osteonecrosis (bone death) of the jaw. To address the problem, the three regional PBRNs, taking advantage of their presence in practices spanning multiple states, teamed up to carry out a collaborative study on osteonecrosis of the jaw. The study results, published in 2010, confirmed that bisphosphonate use is a risk factor for osteonecrosis of the jaw, and provided additional important evidence to guide clinicians in their treatment of this challenging condition.

The NCI HMO Cancer Research Network (CRN) consists of the research programs, enrolled populations, and data systems of 14 HMOs nationwide that, collectively, provide care to almost 11 million individuals. Co-funded by the Agency for Healthcare Research and Quality, CRN research focuses on the characteristics of patients, clinicians, communities, and health systems that lead to the best possible outcomes in cancer prevention and care. The CRN allows for large, multi-center, multidisciplinary intervention research that addresses the spectrum of cancer control, including studies of prevention, early detection, treatment, survivorship, surveillance, and end-of-life care. The CRN also develops and utilizes standardized approaches to data collection, data management, and analysis across health systems. CRN activities have generated more than 140 journal publications in a range of disciplines.

In FY 2010, NIMH launched a major initiative, the Mental Health Research Network (MHRN), which connects nine established public domain research centers based in integrated, not-for-profit health care systems. These systems provide care to a diverse population of 10 million people in 11 states, and they share rich and compatible data resources to support a wide range of effectiveness research. Researchers have begun to use this network to address several important issues, including the development of a geographically and ethnically diverse autism spectrum disorder research registry; a pilot study for a new type of therapy for postpartum depression; and, a longitudinal analysis of how suicide warning labels on antidepressants affect later suicide among youth.

A new initiative of the NIH Common Fund in FY 2010, the Health Care Systems Collaboratory builds on these kinds of investments to create a large infrastructure that leverages the resources of healthcare delivery organizations to implement pragmatic research studies in real world health care delivery settings. This program develops networks of Health Care Delivery Systems to provide a framework of implementation of methods and best practices that will enable the participation of many health care systems in clinical research.

Disseminating and Implementing Clinical Research Discoveries

Dissemination and implementation research is intended to bridge the gap between clinical research and everyday practice by building a knowledge base that addresses how health information, interventions, and new clinical practices are transmitted and translated for public health and health care service use in specific settings. For example, NIDA has created two implementation infrastructures, NIDA’s National Drug Abuse Treatment Clinical Trials Network (CTN) and the Criminal Justice-Drug Abuse Treatment Studies (CJ-DATS) to enhance the implementation of evidence-based substance abuse and HIV screening and treatment interventions in community treatment programs and the criminal justice system, respectively. CTN is exemplary of efforts to translate research into practice, testing feasibility and measuring variables tied to implementation success. This research infrastructure promotes feedback from multiple stakeholders, which is then integrated to improve drug abuse and addiction treatments, making them more feasible and readily available to those who need them. Similarly, CJ-DATS tests evidence-based approaches and innovative implementation strategies within the criminal justice system as well as upon re-entry into the community.

Tackling real-world clinical issues and generating evidence that will be of immediate value to practitioners and patients is the central goal of the NIDCR-supported dental Practice-based Research Networks (PBRNs). Conducting research in dental practices draws on the experience and insight of practicing clinicians to help identify and frame research questions. Because PBRN studies address practice-based problems, their results tend to be more quickly translated into daily clinical care.

Leveraging the infrastructure of established dental practices for conducting PBRN studies also can be a powerful and cost-effective means to conduct clinical research. For example, the past decade brought reports that people who take bisphosphonates, a class of drug prescribed for osteoporosis or to treat the bone-wasting effects of cancer, can develop osteonecrosis (bone death) of the jaw. To address the problem, the three regional PBRNs, taking advantage of their presence in practices spanning multiple states, teamed up to carry out a collaborative study on osteonecrosis of the jaw. The study results, published in 2010, confirmed that bisphosphonate use is a risk factor for osteonecrosis of the jaw, and provided additional important evidence to guide clinicians in their treatment of this challenging condition.

To date, nearly 1,000 practitioner-investigators have participated in network projects, and over 30,000 patients from their practices have been enrolled in more than 30 different PBRN studies. These studies include comparisons of the benefits of a variety of dental procedures, dental materials, and diagnostic strategies for patients with diverse clinical conditions. Individual studies have addressed, for example, controlling pain associated with root canal therapy, improving dental restorations, and testing the feasibility of measuring blood glucose levels in dental practice.

In another example, the NIDDK supports translational research efforts to develop affordable, scalable adaptations of the landmark Diabetes Prevention Program (DPP) clinical trial, which found that a lifestyle intervention could prevent or delay type 2 diabetes by 58 percent in people at risk. While the original DPP lifestyle intervention was found to be cost-effective, providing it to a significant fraction of the 79 million Americans estimated to have pre-diabetes requires still greater efficiency. The positive effects of the DPP continue as new research, building on the study’s results, seek the most effective ways to prevent, delay, or even reverse diabetes. Subsequent NIDDK-funded translational research efforts utilized local Ys (formerly YMCAs) for delivering a group-based adaptation of the DPP lifestyle intervention. A pilot study showed that this group-based approach reduces costs to deliver the intervention, while achieving similar levels of weight loss in participants; a larger trial is ongoing. Based on evidence from this clinical and translational research, the CDC, in partnership with the Y and UnitedHealth Group, launched the National Diabetes Prevention Program36. Efforts to scale and sustain the National Diabetes Prevention Program continue as a growing number of programs offer the lifestyle change program nationwide. Organizations offering a lifestyle change program consistent with the Diabetes Prevention Recognition Program Standards and Operating Procedures can participate in CDC’s Diabetes Prevention Recognition Program. Organizations are recognized by CDC for achieving results that are consistent with what research showed to be effective in preventing type 2 diabetes.

In addition, the NIH Office of Medical Applications of Research37 works closely with ICs to assess, translate, and disseminate the results of biomedical research that can be used in the delivery of health services. OMAR coordinates periodic consensus conferences with the goal of reviewing areas of NIH-supported research where there may be a gap between research accomplishments and clinical care. To date, NIH has conducted more than 120 consensus development conferences and 30 state-of-the-science conferences. Consensus and state-of-the-science statements are disseminated widely after the conference either to modify clinical practice when evidence strongly supports using or not using a particular intervention or to direct future research when important gaps in knowledge have been identified. The consensus statements that result from these conferences are shared widely with healthcare providers, policymakers, patients, and the media.

36 For more information, see https://www.cdc.gov/diabetes/prevention/.
37 In 2012, the Office of Medical Applications of Research combined resources, staff, and key activities within the Office of Disease Prevention.

Health Economics Research

Economic factors are major drivers in healthcare delivery and thus affect the translation of biomedical research findings into health improvements. NIH supports research designed to understand these factors, such as, for example, financing and reimbursement issues related to the adoption and sustainability of newly developed interventions. Under the Common Fund, the Health Economics Program aims to support basic and applied research to understand how innovations in treatments, diagnosis, and preventative strategies can be most effectively implemented in a health care setting, so that past and future investments by NIH may have greater impact.

Results from health services research can inform the entire healthcare sector (clinicians, hospitals, insurers, public health and community centers, and policymakers) about the most efficient and effective means of preventions, screening, and treatment, and opportunities for improvement.