The term “translational research” generally refers to the bench-to-bedside enterprise of harnessing knowledge from basic sciences to produce new drugs, devices and treatment options for patients. However, as the cost of health care continues to rise and the demand for patient-specific treatment increases, the knowledge that is acquired through translational research is being applied beyond traditional product development to assess product value, quality and appropriateness. This landscape presents expanded research opportunities for academic medical centers, demands more evidence from manufacturers and ultimately expands patient access to treatment options.
For many, the term “translational research” refers to the bench-to-bedside enterprise of harnessing knowledge from basic sciences to produce new drugs, devices and treatment options for patients. But for health services researchers and public health investigators whose study focuses on the delivery of effective health care as the primary outcome, this broader perspective on translational research ensures that new treatments reach the patients or populations for whom they are intended. In other words, bringing a new drug, device or diagnostic to market becomes the starting point, not the end of the process of medical innovation.
Why does it matter?
In the U.S., patients only receive half of services recommended to them by their physicians. Translating research into practice seeks to close that gap and improve quality of care by improving access, reorganizing and coordinating systems of care, helping clinicians and patients to change behaviors and make more informed choices, providing reminders and point-of-care decision support tools, and strengthening the patient-clinician relationship. Foundations, industry, patient advocacy organizations and individual hospitals and health systems have implemented translational research programs to facilitate the interaction between basic research and clinical medicine. The federal government is making the advancement of translational research a priority:
- The National Institutes of Health (NIH) recognized that a broad re-engineering effort is needed to create greater opportunity to catalyze the development of a new discipline of clinical and translational science. As part of its Roadmap for Medical Research, NIH has formed centers of translational research through Clinical and Translational Science Awards (CTSA). Initiated in 2006 with 12 academic health centers, the consortium has expanded to 38 sites across the country. By 2012, there are projected to be 60 CTSA sites providing a total of $500 million annually.
- Faced with new projections that Medicare and Medicaid will consume 12 percent of the economy by the midpoint of this century, the Congressional Budget Office (CBO) is exploring ways to improve public health and achieve cost savings from preventive services and is working on two volumes of research to be completed by the end of 2009. The publications are expected to include evidence that “pinpointed” preventive care can garner savings to federal health programs.
- As the nation’s health bill rises, comparative effectiveness research is considered a valuable tool for enabling physicians and patients to select the best therapy for their individual condition, and may also influence coverage decisions. Currently, the federal government invests $30 million annually to conduct and support research with a focus on outcomes, comparative clinical effectiveness and appropriateness of pharmaceuticals, devices and health care services. Congressional leaders and President-elect Obama have indicated that they will expand the current effort and establish an institute to guide reviews and research on the comparative effectiveness of alternative diagnostic and therapeutic interventions. The institute would work with experts and stakeholders to prioritize treatments for research – including surgical procedures, pharmaceuticals, medical devices and other measures. The findings would be incorporated into practice guidelines, standards and other evidence-based decision tools for doctors and patients.
- For conditions that we cannot prevent, translational research is also a key component of the move toward personalized medicine. Given our better understanding of the human genome, we can predict our individual susceptibility to disease; provide more useful and person-to-person specific tools for preventing disease; detect the onset of disease at the earliest moments; pre-empt the progression of disease (as a result of early detection); and target medicines and dosages more precisely and safely to each patient. While the field of personalized medicine holds much promise, significant barriers for broader adoption remain. However, it is likely that the new administration will seek to address some of these issues, including lack of financial incentives, regulatory uncertainty, clinical education, and access and integration of supportive evidence.
What should you do to prepare?
- Clinical and Translational Science Awards: As the potential for funding through this mechanism narrows, universities and communities that are interested in this opportunity must position themselves for an ultra-competitive environment. NIH clearly is looking for innovative approaches among the CTSA applications. Among the successful CTSA applications to date, partnership with industry and clinical practices is a recurring theme. In order to establish an infrastructure for managing these various partners, the applicants need to establish a public/private partnership as part of the proposal.
- Public Health and Prevention: If policymakers on the Hill and in the administration can demonstrate that preventive care will yield long-term savings for the federal government, providers and manufacturers should be prepared for increased demand for preventive services and products as well as diagnostic tools that can be used to identify conditions. Companies should invest in developing additional prevention tools to meet the demand and those with disease-specific technologies should consider partnering with patient advocacy organizations to advance legislative initiatives that would increase the uptake of their products.
- Comparative Effectiveness: Medical technology manufacturers are concerned that research on clinical treatments will morph into a cost control mechanism and way for insurers to deny patients access to products. In preparation for the establishment of a comparative effectiveness institute, manufacturers should consider preparing and compiling the clinical evidence that will demonstrate the value of their products against those of their competitors. Manufacturers should also monitor how comparative effectiveness will be assessed to understand whether cost or other economic considerations will be included.
- Personalized Medicine: As the march toward personalized medicine continues, academic medical centers and manufacturers that are positioned to take advantage of the incentives that will be offered will be at the forefront of providing expanded treatment opportunities for patients. For example, some molecular diagnostic tests already use genetic information to steer treatment, such as testing for the HER2/neu receptor to determine whether to prescribe Herceptin® (Genentech USA, Inc.) for cancer patients. In addition, a number of pharmacogenomic tests are under development, including several for warfarin dosing, which could provide cost savings of up to $1 billion per year for the health care system. Personalized health care will require new business models and reimbursement approaches that will focus more on detecting disease earlier and prevent it more effectively with diagnostics and therapies commonly associated as a paired unit.