The Clinical Researcher—An "Emerging" Species

The discrepancy between current medical practice and the capabilities for improvement is greater now than at any time since the early part of the last century. In the early 1900s, the emerging sciences of chemistry, biochemistry, anatomy, physiology, pathology, physics, and microbiology provided the potential to transform medical practice. Nonetheless, the field was anecdotal, unscientific, and unregulated. In 1910, the Flexner Report influenced the development of the modern academic medical center, where students are educated and trained by research-oriented faculty who practice in teaching hospitals.¹ This integration of the core missions of education, research, and clinical care led to understanding the pathophysiologic basis of diseases and therapeutic modes capable of modifying them.

Fueled by enlightened federal investments in biomedical research, there has been an explosion in research capabilities, medical technologies, and allied industries. In particular, since the mid-1970s, the biotechnology revolution has fueled new sciences, including genomics, proteomics, nanotechnologies, molecular imaging, and information technologies. Ironically, despite this array of new capabilities, medical practice remains grounded in models that are essentially based in a prior century. That is, health care and the training of physicians are focused on disease, not health, and the delivery of health care is uncoordinated, expensive, and inefficient. New technologies are introduced piecemeal, largely to treat late-stage chronic disease, and they generally drive up expenses.

A new approach, "prospective health care," is necessary, using personalized health planning to quantify each individual's risk of disease, incorporating refined capabilities for early detection and intervention, and centered on standards of care based on evidence that defines the interventions that are best suited for that patient.^{2 - 4} This type of health care would place far more responsibility and support tools in the hands of patients and enable the scientific advances of the last 2 decades to improve health, minimize disease, and enhance the value of each dollar spent. It would also facilitate continuous improvement of care through analysis of outcomes.

Factors preventing a move to this approach to the health care system include inadequate practice models and perverse reimbursement methods. Less well appreciated but equally important is the lack of a robust clinical research enterprise mobilized to translate basic discoveries into clinical relevance. This undernourished part of the US health care system is the critical link that provides the only mechanism to develop the evidence needed to define effective health strategies and monitor their effectiveness. To accomplish this, a full range of clinical research is needed, from translation to outcomes analysis. Without clinical research, the rational application of research discoveries to the development of prospective care and personalized health planning cannot occur.²

The many obstacles preventing expansion of clinical research have been well documented.^{5 - 8} In this issue of THE JOURNAL, the report by Kotchen et al⁹ examines the role of the National Institutes of Health (NIH) peer review process as a barrier to clinical investigation. The authors cite the oft-repeated concern among clinical investigators that the NIH peer review process discriminates against clinical research.

The problem of a decreasing pool of clinical investigators has been addressed, in part, by the NIH through the development of new models for support of training, changes in the structure of study sections, and increases in the priority of clinical research.^{10 - 11} Kotchen et al⁹ provide an update and analysis of the current status of NIH funding for clinical research. The authors present data indicating that physicians fare well in the peer review process in general but that grants for clinical research do "modestly" less well. However, the differences are indeed substantial, with approximately 25% of nonclinical research grants funded compared with only about 20% of grants involving human subjects. A striking finding is that almost 40% of K08 awardees never applied for an R01 award. This finding highlights a root cause for the lack of a robust clinical research enterprise. Many of the small numbers of physicians in training who enter a career in clinical research never even apply for an independent research grant. One can assume that they are discouraged by the daunting issues they face in a clinical research career.

The "parity gap" in peer review funding is only a symptom of a larger, more complex problem. Clinical research has not yet developed into a well-recognized, well-respected, and economically attractive career option. As a result, the pool of well-trained clinical researchers is inadequate. Much has been done to address training support,^{10 - 11} and this may yet have a positive effect. However, neither the medical profession, its institutions, the public, nor payers fully understand the practical importance of clinical research for improving the effectiveness of health care and the efficiency of expenditures. As a consequence, clinical research has not yet attained the market value it needs and deserves.

Academic institutions, with their increasingly strained finances, by and large do not allocate sufficient means to support clinical research and the faculty needed to do it well. Clinical researchers are often limited by lack of formal standardized training, appropriate certification, and adequate time for research. They also do not have sufficient respect from their professional colleagues. As the clinical practices in academic centers are increasingly placed under financial pressure, time for clinical investigation or to experiment with the development of novel health care delivery approaches has diminished. Prestigious organizations and study groups have proposed solutions,^{12 - 15} ranging from training to career advancement and promotion to funding. The importance of clinical research to the mission of the NIH is well presented in its "roadmap."¹⁶ However, the clinical researcher must also be recognized as an esteemed member of institutional and professional organizations and must be provided with opportunities to perform clinical research as a valid academic function. The public, payers, and the government must be educated to recognize that the true value and need for clinical research is to develop better models of health care delivery with more effective and efficient use of technology for the individual patient.

A number of academic medical centers have begun to recognize the critical role that clinical research plays in their academic and social missions.^{12 - 13} This is a positive movement, and leaders of academic medicine can foster its momentum by positioning their institutions to address more fully the expanding needs for the clinical researcher. For example, development of master's-level clinical research training programs provides the means for students, trainees, and faculty to participate and be certified. Tenured tracks can be established for clinical researchers within clinical departments. Establishment of clinical research institutes at medical centers provides a mechanism for clinical researchers to collaborate with statisticians and other research professionals required to address clinical and outcomes research questions in today's complex research environment.¹⁷ Less research-intensive institutions also can participate in significant clinical research through interdisciplinary and interinstitutional collaborations (including with NIH), thereby enhancing the opportunity for clinical research faculty.

To expand clinical research, the suggestions provided by the Association of American Medical Colleges, the Clinical Research Roundtable of the Institute of Medicine,^{12 - 14} and others are constructive steps that should be taken. However, until the health care market fully understands the importance of clinical research in creating more cost-effective health care, the resources needed to support this field will remain scarce. Health care needs and market forces will eventually reward clinical research as they have other areas of critical shortage, such as nursing. Nonetheless, physicians and health care administrators must be strong advocates to bring this about more quickly. The increased fragility of the current US health care system coupled with the increasingly clear understanding of the benefits of prospective care will magnify the support for clinical research if a clear case is made for its importance. To jump-start this process, academic medical leaders must foster quality clinical research centers in their institutions. Moreover, the positive impact of practical application of new discoveries, technologies, and knowledge must be advocated.

Clinical research, when properly applied, will save money and improve care. The reward of doubling the NIH budget will be realized only through clinical applications of its discoveries within a better delivery system. However, this will not occur unless a substantial proportion of that budget is spent on clinical research.¹⁸ To demonstrate the value of clinical research on the cost-effectiveness of health care, idealized pilot models incorporating clinical research to enable personalized health planning and prospective care must be developed. Pilots should be funded by appropriate governmental agencies including the NIH, Agency for Healthcare Research and Quality, and Centers for Medicare and Medicaid Services, as well as by enlightened private payers. The currents of change in the capabilities of clinical research and medical care along with the disparate needs of the health care system require a transformation equal in magnitude to what occurred a century ago. Through coordinated efforts, the medical community can help transform the clinical researcher from an endangered to an emerging species.