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SUMMARY: THE ROLES OF THE NCI AND FDA IN DEVELOPING ANTICANCER AGENTS

NCI has played, and should continue to play, an important role in the development of new cancer therapeutics. That role has changed and will continue to evolve as the basic science underlying cancer and the development of agents to fight cancer evolve. That is NCI’s challenge: to continue to fill gaps and catalyze new fields, both on its own and in collaboration with other agencies, academia, and industry. This may involve initiating new programs and phasing out others, a process that has gone on for decades at NCI, both on its own initiative and pursuant to congressional directives. In defining its role, then, in the development of new cancer therapeutics, NCI may variably emphasize basic science and discovery of new agents or development and introduction of agents of others in coordination with FDA.

For NCI, adapting to new science and a changing mix of actors has meant: modifying drug screens and developing new in vitro and in vivo models; exploring more effective toxicologic evaluations; reviewing the organization of clinical trials; setting up compound repositories; providing support to academic and other drug developers; a recent emphasis on fields of molecular targeting, biomarkers, genomics, proteomics, assays and databases of genetic changes in cancer; working more closely with industry; and an array of research supports; among others; and, in general, balancing its cancer research roles with responsibilities to encourage quality cancer care and advances in therapeutics. The role of the NCI in developing new therapeutics for cancer is not as precisely defined in statute and regulation as is that of the FDA. It is unarguable, however, that, as a research agency, NCI, with congressional support, needs to continue to advance the basic and clinical cancer science that is the foundation of new and better cancer therapeutics.

FDA has been responsible for review and approval of cancer therapeutics in the United States as defined in the statutes reviewed in this background paper, and on its own and at congressional directive has made changes in its procedures and internal structure to improve the efficiency of reviews and approvals and communications to drug and biologics sponsors.

For FDA, this has meant: meeting the legal requirements for assessing safety and efficacy of all drugs, including cancer drugs, and for all biologics, including cancer biologics when this responsibility was transferred from NIH; developing evaluative science and the evaluation criteria for cancer drugs and performance goals for reviews and approvals; improving communications and guidance documents for sponsors; developing policies for accelerated approvals and post-marketing confirmatory trials of cancer drugs and appropriate endpoints for cancer trials and approvals; exploring and openly discussing ways of approaching trials, reviews, and approvals of combinations of cancer drugs; thinking through problems and solutions relevant to more precisely molecularly targeted drugs and approaches to cancer and populations with identified



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Federal Agency Roles in Cancer Drug Development from Preclinical Research to New Drug Approval: The National Cancer Institute and the Food and Drug Administration 4 SUMMARY: THE ROLES OF THE NCI AND FDA IN DEVELOPING ANTICANCER AGENTS NCI has played, and should continue to play, an important role in the development of new cancer therapeutics. That role has changed and will continue to evolve as the basic science underlying cancer and the development of agents to fight cancer evolve. That is NCI’s challenge: to continue to fill gaps and catalyze new fields, both on its own and in collaboration with other agencies, academia, and industry. This may involve initiating new programs and phasing out others, a process that has gone on for decades at NCI, both on its own initiative and pursuant to congressional directives. In defining its role, then, in the development of new cancer therapeutics, NCI may variably emphasize basic science and discovery of new agents or development and introduction of agents of others in coordination with FDA. For NCI, adapting to new science and a changing mix of actors has meant: modifying drug screens and developing new in vitro and in vivo models; exploring more effective toxicologic evaluations; reviewing the organization of clinical trials; setting up compound repositories; providing support to academic and other drug developers; a recent emphasis on fields of molecular targeting, biomarkers, genomics, proteomics, assays and databases of genetic changes in cancer; working more closely with industry; and an array of research supports; among others; and, in general, balancing its cancer research roles with responsibilities to encourage quality cancer care and advances in therapeutics. The role of the NCI in developing new therapeutics for cancer is not as precisely defined in statute and regulation as is that of the FDA. It is unarguable, however, that, as a research agency, NCI, with congressional support, needs to continue to advance the basic and clinical cancer science that is the foundation of new and better cancer therapeutics. FDA has been responsible for review and approval of cancer therapeutics in the United States as defined in the statutes reviewed in this background paper, and on its own and at congressional directive has made changes in its procedures and internal structure to improve the efficiency of reviews and approvals and communications to drug and biologics sponsors. For FDA, this has meant: meeting the legal requirements for assessing safety and efficacy of all drugs, including cancer drugs, and for all biologics, including cancer biologics when this responsibility was transferred from NIH; developing evaluative science and the evaluation criteria for cancer drugs and performance goals for reviews and approvals; improving communications and guidance documents for sponsors; developing policies for accelerated approvals and post-marketing confirmatory trials of cancer drugs and appropriate endpoints for cancer trials and approvals; exploring and openly discussing ways of approaching trials, reviews, and approvals of combinations of cancer drugs; thinking through problems and solutions relevant to more precisely molecularly targeted drugs and approaches to cancer and populations with identified

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Federal Agency Roles in Cancer Drug Development from Preclinical Research to New Drug Approval: The National Cancer Institute and the Food and Drug Administration genetic changes; implementing organizational changes to emphasize and focus on cancer therapeutics; among others; and, in general, balancing its role as a consumer protection agency with its obligation to make useful medications available to the marketplace and patient care on a timely basis. Both agencies are responding to the promise and challenges posed by advances in basic and clinical science in cancer and related fields and the need to adjust strategies and programs to this progress in scientific understanding. Both agencies have issued statements and taken steps to coordinate, collaborate, and cooperate with the objective of ensuring that the potential of recent and projected scientific progress in cancer is translated to new anticancer therapeutics that are less toxic, more specific to precise molecular targets, and more effective in tumor control. NCI and FDA collaborations and continuing work to update procedures and policies for drug identification and development appear to be encouraging directions for the future. The information reviewed in this background paper, however, suggests that scientific, procedural, and policy problems remain that will require continuing effort from these agencies, singly and in concert, if their goals in the fight against cancer are to be realized in the most efficient and effective directions.