Executive Summary
Clinical research has always been an integral part of medical care: in many countries the two cannot be dissociated. Clinical Trials are the mechanism for the testing of new approaches to cancer prevention, diagnosis, and treatment. As such they are a critical component of the National Cancer Program and the National Cancer Institute's (NCI) research program. The NCI clinical trials program represents a unique opportunity to improve the survival and quality of life of patients with cancer, and to translate basic research into clinical application.
The clinical trials system is complex, involves many participants, and requires collaboration at all levels--between investigators and physicians, industry and academia, academia and NCI, and NCI and industry. In its entirety, the clinical trials system is an intricate and large research laboratory without walls. This complexity has bred inefficiencies and eroded the ability of the system to generate new ideas to reduce the cancer burden. Other challenges to clinical research, such as rapidly diminished opportunities for training, managed care, cost containment, low levels of participation in research, and diminishing levels of financial support for patient care and research have strained the system. The United States is now the world leader in both medical advances and in clinical treatment, in large part because of the excellence of clinical trials. Erosion of this structure will have a negative impact on our national health and economy over the long term.
At the same time several scientific and medical events have converged to force a reevaluation of the clinical trials system. Progress in cancer biology, genetics, immunology, imaging, and molecular biology has accelerated, creating new opportunities to pursue in clinical application. Opportunities offered through informatics and electronic communications offer an entirely new approach to communication and data transfer and analysis in the clinical research setting.
In April 1996 the Director of NCI and the Chair of the NCI Extramural Board of Scientific Advisors convened a group to review a series of issues affecting the NCI clinical trials effort. This report is a response to that review. It addresses the question of how the cancer clinical trials system can respond to the exponential increase in new therapeutics and new technology in a changing fiscal and health care environment.
The clinical research community must find productive ways to respond to these challenges reducing the costs of research through efficiencies and by redefining study parameters, endpoints, and outcomes. In sum, the forces of expanding opportunities and contracting resources call for a new approach to setting priorities for, developing, and conducting clinical trials. In considering these challenges, the Review Group considered the following broad issues which form the structure of this report: recruiting and retention of clinical scientists; recruiting of participants to clinical trials; improving clinical trials methodology; increasing collaboration and cooperation in clinical trials; and the organizational framework and structure for implementation of clinical trials at NCI.
The Review Group made the following major recommendations regarding review, funding, design, oversight, and administration of the NCI clinical trials system. The recommendations are intended to create a more efficient and effective clinical research effort and to help NCI enhance and maintain a critical endeavor which is continually facing new internal and external challenges. Additional recommendations appear in the body of the report.
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A patient-oriented clinical cancer research and training study section in the NIH Division of Research Grants is critical.
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The NCI should increase funding for cooperative groups to fully recommended levels.
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In designing clinical trials, data collection should be reduced so that only data pertinent to the study endpoints and patient safety are accrued. In addition, NCI-funded efforts should include some large, uncomplicated trials in common cancers with minimal data requirements and accrual goals large enough to establish treatment differences definitively.
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Uniformity of data collection for patients on clinical trials in cooperative groups and cancer centers is essential.
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NCI should enlist the clinical trials and patient advocate communities as well as the pharmaceutical industry to work with the Food and Drug Administration to develop uniform standards and reporting requirements for everyone involved in oncology clinical trials (e.g., pharmaceutical industry, academia, cooperative groups, cancer centers).
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To be able to create and prioritize the best new ideas in cancer treatment and prevention, the NCI-funded cooperative groups and cancer centers should be provided with the means to access all relevant electronic databases, and should be primary participants in the development and testing of the new NCI informatics system.
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For phase III and phase II studies not involving new agents, the Cancer Therapy Evaluation Program of the Division of Cancer Treatment, Diagnosis and Centers should approve study concepts and collaboratively establish research priorities, and its authority should be otherwise limited to regulatory and safety issues and prevention of unnecessary duplication.
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Representatives of patient and high-risk communities must be integrated into the clinical trials decision making process.
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Therapeutic trials conducted through the Community Clinical Oncology Program should be transferred to the Division of Cancer Treatment, Diagnosis and Centers. Cancer prevention studies conducted across the NCI clinical trials system should be the responsibility of a newly configured Division of Cancer Prevention and Control.
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To insure the continued success of cancer clinical trials, NCI should increase training opportunities for new and mid career investigators.
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NCI should develop strategies, including necessary databases, to convince payers that clinical trials are the preferred way to manage cancer patients, that they represent a better standard of care, and ultimately result in decreased costs.
The Review Group believes that if NCI considers and implements the recommendations outlined in this report, the overall structure and performance of the nation's clinical trials system will more efficiently and effectively explore new approaches to reducing the morbidity and mortality of cancer.