Information about underlying genetic traits and markers can be applied to help determine whether drugs will be effective as well as to predict their toxicological effects. It is important to feed this type of information as well as knowledge about why drugs have failed in the past back into earlier parts of the drug development process so that if a drug will fail it is identified as early as possible, not after hundreds of millions of dollars have been spent. While the FDA is not allowed to share the confidential commercial information it receives, the agency is working with companies to make this information more available to inform drug development and applications of existing drugs, Hamburg said.

The FDA has many opportunities to apply better computational approaches, improved data mining techniques, better pattern recognition strategies, and other cutting-edge techniques to identify promising applications of existing drugs and better-defined pathways for drug development. Such applications of cutting-edge science will often require bringing together people with different perspectives and different expertise.

Hamburg concluded her formal remarks by pointing to the complex ecosystem involved in biomedical product development and innovation. Many policies besides regulatory policies affect this ecosystem, including intellectual property protections, access to capital, reimbursement policies, and direct government investments. All of these policies matter, said Hamburg. “If we’re going to have success in realizing the opportunities in science, we need to be thinking about investing in and supporting the ecosystem that will be the engine of success.”


Another increasingly important partner in collaborative efforts will be NCATS, which was represented at the workshop by its acting deputy director, Kathy Hudson. The mission of NCATS is to catalyze the generation of innovative methods and technologies in order to enhance the development, testing, and implementation of diagnostics and therapeutics. The goal is to develop the tools that will make drug discovery and development better, faster, and cheaper, Hudson said. “We are not in the drug development business.”

This is a nontraditional approach for NIH. It requires working collaboratively with all of the 27 NIH institutes and centers that are each conducting translational research; with its sister agencies, including the FDA; with industry; with patients; and with academic medical centers. “[Collaboration] is really essential and vital to our success,” Hudson said.

Hudson mentioned several examples of innovative work that NCATS is undertaking in a precompetitive space. Like the FDA, NCATS has an interest in new kinds of clinical trials that are faster, cheaper, and involve smaller

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