Executive Summary

Advances in biomedical research are yielding significant opportunities to improve cancer prevention, detection, and treatment. However, the ability to translate biomedical discoveries into meaningful advances in cancer care depends on an effective clinical trials system. Publicly funded clinical trials play a vital role by addressing questions that are important to patients but are less likely to be top priorities of industry, which has an important primary focus on new drug development and Food and Drug Administration (FDA) registration. For example, companies may have less incentive to

  • conduct clinical trials to compare the effectiveness of different treatment options that are already approved for clinical use,

  • combine novel therapies developed by different sponsors,

  • develop therapies for rare diseases,

  • determine optimal duration and dose of treatment with drugs in clinical use,

  • test multimodality therapies, such as radiation therapy, surgery, or devices in combination with drugs,

  • study screening and prevention strategies, or

  • focus on rehabilitation and quality of life following therapy.

The National Cancer Institute (NCI) supports the largest U.S. network for clinical trials of any type. The largest component of that network is the Clinical Trials Cooperative Group Program, which comprises 10 Groups that involve more than 3,100 institutions and 14,000 investigators who



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Executive Summary Advances in biomedical research are yielding significant opportuni- ties to improve cancer prevention, detection, and treatment. However, the ability to translate biomedical discoveries into meaningful advances in cancer care depends on an effective clinical trials system. Publicly funded clinical trials play a vital role by addressing questions that are important to patients but are less likely to be top priorities of industry, which has an important primary focus on new drug development and Food and Drug Administration (FDA) registration. For example, companies may have less incentive to • conduct clinical trials to compare the effectiveness of different treatment options that are already approved for clinical use, • combine novel therapies developed by different sponsors, • develop therapies for rare diseases, • determine optimal duration and dose of treatment with drugs in clinical use, • test multimodality therapies, such as radiation therapy, surgery, or devices in combination with drugs, • study screening and prevention strategies, or • focus on rehabilitation and quality of life following therapy. The National Cancer Institute (NCI) supports the largest U.S. network for clinical trials of any type. The largest component of that network is the Clinical Trials Cooperative Group Program, which comprises 10 Groups that involve more than 3,100 institutions and 14,000 investigators who 1

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2 A NATIONAL CANCER CLINICAL TRIALS SySTEM enroll more than 25,000 patients in clinical trials each year. The results of Cooperative Group trials have steadily improved the care of patients with cancer in the United States and worldwide for more than 50 years. One of the Program’s strengths is the extensive involvement of physi- cians and patients from the community setting. Participation by the diverse patient populations treated in the community setting helps to ensure that the results of clinical trials are meaningful to a broad segment of the U.S. population and provides these patients with access to promising, innovative therapies as they are developed and tested. The clinical trials conducted by the Cooperative Groups also provide a valuable mechanism for the training of clinical investigators. However, despite these important contributions and a long record of accomplishments, the Cooperative Group Program is at a critical juncture. Numerous challenges threaten its ability to conduct the timely, large-scale, innovative clinical trials needed to improve patient care. With many itera- tive layers of oversight, the complex trials system has become inefficient and cumbersome. The average time required to design, approve, and activate a trial is 2 years and many of the trials undertaken are not completed. Furthermore, since 2002 funding for the Cooperative Group Program has decreased by 20 percent, whereas new knowledge of the molecular changes underpinning cancer and the use of predictive biomarkers in cancer therapy not only increase the potential impact of trials but also add to their com- plexity and cost. The director of NCI asked the Institute of Medicine (IOM) to conduct a consensus study of cancer clinical trials and the Cooperative Group Pro- gram and to develop recommendations on how to improve the system. To address the charge, the IOM appointed a 17-member committee with a broad range of expertise and experience. The committee concluded that a robust, standing cancer clinical trials network is essential to effectively translate discoveries into clinical benefits for patients. There are hundreds of cancer therapies in development and a continuous need for design and implementation of new clinical trials, so it would be highly inefficient to fund and develop infrastructures and research teams separately for each new trial. Thus, it is imperative to preserve and strengthen the unique capabilities of the Cooperative Group Program as a vital component in NCI’s translational continuum. However, the current structure and processes of the entire clinical trials system need to be redesigned to improve value by reducing redundancy and improving the effectiveness and efficiency of trials. Numerous changes are needed, including an evaluation and justification of the unique contribu- tion of each Cooperative Group and a shift in the primary focus of NCI from oversight to the facilitation of Cooperative Group trials. The Program

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 EXECUTIVE SUMMARy needs to move beyond cooperation to integration, which can be achieved by reorganizing clinical trial structures and operations in a truly national trials network. The revised system must also be sufficiently funded to enable the rapid completion of well-designed, high-priority trials. In addition, govern- ment agencies need to streamline and coordinate the oversight process, with parallel, concurrent, or ideally, joint reviews to the extent possible. In sum, the academic, government, and commercial sectors must join with the pub- lic to develop a 21st-century multidisciplinary clinical trials system to more effectively leverage scientific advancements and translate them into public health benefits by improving the science; technology; efficiency; and timely creation, launch, and completion of the highest-priority cancer clinical tri- als. With adequate funds and support, a more effective and efficient clinical trials system will speed the pace of advances in cancer patient care. On the basis of a review of the available published literature, along with input from experts in the field and interested individuals, the commit- tee’s recommendations (Box ES-1) focused on four broad goals to enhance the value of national Cooperative Group clinical trials in cancer: Consolidation and Efficiency. Improve the efficiency and reduce the average time for the design and launch of innovative clinical trials by con- solidating functions, committees, and Cooperative Groups; streamlining oversight processes; facilitating collaboration; and streamlining and stan- dardizing data collection and analysis. Science. Incorporate innovation in science and trial design, for example, in studies identifying biomarkers that can predict therapeutic response. Funding and Support. Adequately support those clinical trials that have the greatest possibility of improving survival and the quality of life for cancer patients, and increase the rate of clinical trial completion and publication. Participation. Incentivize the participation of patients and physicians in clinical trials by providing adequate funds to cover the costs of research and by reimbursing the costs of standard patient care during the trial.

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 A NATIONAL CANCER CLINICAL TRIALS SySTEM BOX ES-1 Summary of the Committee’s Goals and Recommendations Goal I. Improve the speed and efficiency of the design, launch, and conduct of clinical trials   1.   eview  and  consolidate  some  front  office  operationsa  of  the  Cooperative  R Groups on the basis of peer review   2.   onsolidate  back  office  operations  of  the  Cooperative  Groups  and  improve  C processesb   3.   treamline and harmonize government oversight S   4.  mprove collaboration among stakeholders I Goal II. Incorporate innovative science and trial design into cancer clinical trials   5. Support and use biorepositories   6. Develop and evaluate novel trial designs   7. Develop standards for new technologies Goal III. Improve the means of prioritization, selection, support, and comple- tion of cancer clinical trials   8. Reevaluate the role of NCI in the clinical trials system   9. Increase the accrual volume, diversity, and speed of clinical trials 10. Increase funding for the Cooperative Group Program Goal IV. Incentivize the participation of patients and physicians in clinical trials 11. Support clinical investigators 12. Cover the cost of patient care in clinical trials aFront office operations refer primarily to the Cooperative Group scientific committees and  statistical offices, which are responsible for activities such as trial design, prioritization, and  data analysis. bBack  office  operations  refer  to  administrative  structures  and  activities  that  include  such  things  as  data  collection  and  management,  data  queries  and  reviews,  patient  registration,  audit functions, case report form processing, image storage and retrieval, drug distribution,  credentialing of sites, and funding and reimbursement for patient accrual.