Clinical and Translational Science Awards: a framework for a national research agenda

Clinical and Translational Science Awards: a framework for a national research agenda T he advances made in the past 5 years in fields such as genomics, proteomics, and bioimaging will enable tools for health care that are predictive, preemptive, and targeted. The improved molecular understanding of diseases, along with development of biomarkers for risk factors, will provide health-care professionals and their patients with early warnings for disease development as well as strategies to prevent progression. Patients with active diseases, both chronic and acute, will receive targeted therapies that have a reduced likelihood of adverse effects, while reducing morbidity and mortality. This new vision of health care requires sustainable and integrated efforts in translational and clinical research that can yield new products, approaches, and diagnostic tools in an efficient, seamless manner. As a result of extensive dialog with the community, the National Institutes of Health (NIH) developed the request for applications (RFAs) for the Clinical and Translational Science Awards (CTSA)s, whose purpose is to provide academic homes that can be centers, institutes, or departments that will provide and integrate intellectual and physical resources essential to clinical and translational science.1 The clinical and translational researchers of the future, who will be trained through degree-granting programs in these academic homes, will learn how to work as interdisciplinary teams and will understand the complexities of translating basic discoveries into clinical trials and studies, followed by implementation of the results into the practice of medicine. They will learn the best way in which to form partnerships with other organizations, such as industry, and will have the knowledge to interact with the U.S. Food and Drug Administration as they develop new drugs or new uses for approved drugs. The integration of efforts in biostatistics with clinical research can lead to the development of new clinical trial designs that will ensure patient safety and reduce duplication of effort. Active involvement of the community is essential to the success of appropriate trial design and recruitment of participants. These academic homes will be responsible for integrating informatics efforts and will be part of the larger Translational Research 2006;148:4 –5. 1931-5244/$ – see front matter © 2006 Mosby, Inc. All rights reserved. doi:10.1016/j.lab.2006.05.001 4 national activities conducted by the NIH and other federal and nonfederal organizations to develop interoperable clinical research informatics systems for programs ranging from clinical research networks to large databases that are ideal for the exploration of phenotypes and genotypes. In recognizing the importance of clinical research informatics, the NIH developed the Roadmap initiative “the NECTAR Network.” The initial efforts have produced an inventory of all clinical trial networks in the country; pilot studies on interactions in connectivity between networks are ongoing. The groundwork for the development of the CTSA program has been formed through the doubling of the NIH budget. An example of the explosion in genetic information and technology is the International HapMap Project, led by the NIH, which was completed in 2005. This project showed the power and the relatively low cost of using selected single nucleotide polymorphisms (SNPs) to study human haplotypes. These new findings can be applied to the study of large populations and will have immense benefit in clinical trials and studies that are storing DNA samples. Activities in genomics, as well as in development of new molecular entities for treatment of diseases, can be undertaken as partnerships with industry through welldefined agreements. The CTSAs can ensure that, through their degree-granting programs, researchers will have the training to work in multidisciplinary teams that can use the new tools in genomics and proteomics as they design their clinical studies and trials. The response to the RFA for the CTSAs and for the planning grants has been robust. Awards for 4 to 7 CTSAs, along with approximately 50 awards for planning grants, will be made in 2006. The CTSAs, which will number approximately 60 by 2012, will be catalysts and test beds for policies and practices that can benefit clinical and translational research organizations throughout the country. The investigators that are trained in the degree-granting programs will disseminate their knowledge and skills as they develop careers in public and private research organizations that may not have a CTSA. For the CTSAs to flourish, they must form a matrix that includes nonCTSA institutions, industry, and local as well as regional communities. They will reach out to populations that are underserved or that have reduced access to health care. Translational Research Volume 148, Number 1 The CTSA program will need to be evaluated in stages because its true worth will not be evident immediately. Benefits should be seen in the development of investigators that are trained and at ease in working between the borders of clinical and translational research; they will be knowledgeable about how to work with industry and with regulatory organizations. They will understand the processes required for the bidirectional flow of information between preclinical research and clinical trials and studies, although their own efforts may be focused in one particular area. An increased public awareness and trust in clinical research will be another outcome, along with increased efficiencies in developing new treatments and approaches for both rare diseases and common diseases. The ultimate test is for the CTSAs to become an integral and essen- 5 tial part of health-care delivery that can benefit all persons in this country. ELIAS A. ZERHOUNI Director National Institutes of Health Bethesda, Maryland BARBARA ALVING Acting Director National Center for Research Resources National Institutes of Health Bethesda, Maryland REFERENCE 1. Zerhouni E. Translational and clinical science-time for a new vision. N Engl J Med 2005;353:1621–3.