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Envisioning Successful Regulatory Science at FDA1

Emphasizing the neglected state of regulatory science at FDA, Goodman noted that science is critical to the agency’s mission to promote and protect the public health and safety, as well as to the integrity of its decision making. Improvements in the practice of regulatory science must occur at all levels, including population, epidemiology, clinical, manufacturing, and behavioral science. Understanding a molecule or a nanoparticle is not useful unless the information can benefit people.

Pharmaceutical companies perform excellent scientific assessments of their products, said Goodman, but they are limited by the costs and risks associated with innovation. Therefore, a major function of FDA is to engage in a science-based manner both internally and with industry, academia, patients, and other agencies throughout the product development and evaluation processes.

KEY AREAS OF SCIENTIFIC EMPHASIS

As FDA’s Chief Scientist whose office has primary responsibility for overseeing the scientific capacity of FDA, Goodman shared his vision of a successful regulatory science infrastructure within the agency. He described ideal circumstances in which product development, treatments for rare diseases, regenerative medicine, predictive medicine, and infor-

1

This section is based on the presentation of Jesse Goodman, Chief Scientist and Deputy Commissioner for Science and Public Health, FDA.



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7 Envisioning Successful Regulatory Science at FDA1 Emphasizing the neglected state of regulatory science at FDA, Goodman noted that science is critical to the agency’s mission to pro - mote and protect the public health and safety, as well as to the integrity of its decision making. Improvements in the practice of regulatory science must occur at all levels, including population, epidemiology, clinical, manufacturing, and behavioral science. Understanding a molecule or a nanoparticle is not useful unless the information can benefit people. Pharmaceutical companies perform excellent scientific assessments of their products, said Goodman, but they are limited by the costs and risks associated with innovation. Therefore, a major function of FDA is to engage in a science-based manner both internally and with industry, academia, patients, and other agencies throughout the product develop - ment and evaluation processes. KEy AREAS OF SCIENTIFIC EMPHASIS As FDA’s Chief Scientist whose office has primary responsibility for overseeing the scientific capacity of FDA, Goodman shared his vision of a successful regulatory science infrastructure within the agency. He described ideal circumstances in which product development, treatments for rare diseases, regenerative medicine, predictive medicine, and infor- 1 This section is based on the presentation of Jesse Goodman, Chief Scientist and Deputy Commissioner for Science and Public Health, FDA. 5

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6 ESTABLISHMENT OF REGULATORY SCIENCE FOR DRUG DEVELOPMENT mation technology would be made scientifically robust through agency- wide adoption of a regulatory science discipline. Goodman outlined the following areas of scientific emphasis and the vision of success for each: • ransform product development—Given calls for a paradigm shift T in drug development, Goodman predicted that the process will be radically different upon successful implementation of regulatory science at FDA. He described the ideal process as agile and adap- tive to new information, with the ability to consolidate clinical and biological information and identify population subgroups that can uniquely benefit from new drugs, with the ultimate goal of delivering products to patients efficiently and safely. Personalized medicine, diagnostics, biomarkers, innovative clinical trial designs, and combination interventions can all benefit from this change. • dentify unmet public health needs—The ability to respond I rapidly to a pandemic with prepared countermeasures is crucial to the safety and security of the nation and to the national and global public health. Sound science, technology, and methods are essential for the development of products to respond to global diseases, emerging infectious disease threats, and bioterrorism; vaccines; and diagnostics. An ideal regulatory system will be able to identify innovative products with the potential to address unmet medical needs and provide countermeasures for public health and stability. • ocus on regenerative medicine—Stem cells, engineered tissues, F and combination products are areas of rapidly emerging technol- ogy; however, FDA’s intervention will be necessary to bridge the gap between innovation and the market. Despite limited resources, stated Goodman, FDA recognizes the importance of regenerative medicine and has made an effort to interact with the development community and NIH to consider standards and models in this area. The hope is that such medicines will be successfully developed to treat serious diseases, replace damaged organs and tissues, and create new treatments for diabetes and cardiac and neurogenera- tive diseases. • odernize predictive science—As with regenerative medicine, M FDA has the potential to improve predictive science and translate advances in life science and engineering into practice. With a sound science base and the right resources, the agency can modernize in vitro toxicology and product characterization, rapidly detect patho- gens and contaminants in food and medical products, and assess environmental and chemical hazards.

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 ENVISIONING SUCCESSFUL REGULAORY SCIENCE AT FDA • se informatics to enhance outcomes—Goodman envisioned u informatics being used to enhance safety and health outcomes and transform health care. The application of informatics in a regula- tory science infrastructure would include monitoring safety using vast amounts of clinical, health care, and biological data. In part - nership with the health care sector and community settings, the knowledge derived through informatics could result in optimized outcomes, clinical trials, and patient safety and ultimately speed up product development. Finally, Goodman suggested that methods for resolving scientific disputes internally and externally are needed to match the rapid pace of research. There is never a single scientific truth. FDA will need to foster the creation of a culture that allows for diverse opinions to take advantage of all available scientific evidence in regulatory decision making.

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