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FDA is suffering from a deficit of high quality, reliable evidence needed to support regulatory determinations of safety, efficacy and appropriate indications for marketed medical products, says Commissioner Robert Califf. He seeks a solution in expanded access to electronic health records and clinical registries, which can provide rich and diverse sources of digital data. And by employing sophisticated analytical tools, “we are at a tipping point” for being able to tap these resources to generate the scientific evidence needed to make good decisions, Califf observed in a keynote speech to the annual meeting of the Food and Drug Law Institute on May 5. The traditional way to gain answers to research questions is to collect needed evidence through randomized clinical trials that feature restrictive inclusion and exclusion criteria and are conducted at special study sites, Califf explained. Now FDA is working diligently to tap the nation’s investment in electronic health records to generate quality evidence more quickly. The agency seeks to use clinical evidence in “creative ways,” utilizing modern informatics and clinical tools for data analysis in order to tailor diagnostic and therapeutic strategies to match individual characteristics. Califf was careful to emphasize that using “real world data” without a prospective research plan and careful attention to data sources and research methods is “not of great use to the FDA at this time.” But prospectively designed registries and cohort studies “are highly valuable,” as are randomized “pragmatic” clinical trials conducted in the context of clinical practice. Real world data and randomization are “not polar opposites,” Califf explained. In fact, a randomized trial using real world data will be a “highly valuable and efficient approach to generating evidence.” This will be beneficial to the development of precision medicine, he noted, which aims to treat patients based on their unique genes and biological systems. This supports FDA’s “flexible, adaptive regulatory approach,” he said, which “fosters innovation” and ensures patient access to accurate and meaningful test results.
Need for transparency
Califf proceeded to highlight the importance of disclosure and transparency in clinical trial results to support “robust investment” in new medical products. Innovation is stimulated, he said, by “shining a light on what is known” in order to make improvements in technologies and therapies. Data transparency, though, has to be balanced with protecting patient privacy and commercial trade secrets, Califf acknowledged. He expressed optimism that such issues can be resolved and that more access to data will support broad “confidence in the conclusions” of research. Rigorous and complete data are “essential to making valid decisions” about accurate product labeling and appropriate patient treatment, he said. But that raises questions about the standards that apply to communication of that information, particularly by manufacturers promoting medical products. FDA is “reviewing our policies” in order to provide more “clarity” to stakeholders in this evolving environment. Califf concluded his comments by raising concerns about the scope and possible risks of certain provisions of the 21st Century Cures legislation moving through Congress. The bill needs to be “carefully crafted” to avoid posing “significant risks” for FDA and American patients, he said. While the larger goal of the legislation is to promote innovation and speed delivery of medical products to patients, it is equally important for policy makers to preserve the current safety and effectiveness standard. “Innovative therapies are not helpful to patients if they don’t work, or worse, cause harm,” the commissioner warned, urging efforts to “strike the right balance between fast access and good science.” And just as important is for any new legislation that establishes new programs and requirements for FDA to also provide the resources needed for the agency to meet such new obligations. Otherwise, FDA might be forced to “divert its already limited resources from critical public health activities” such as the review of new medical products, Califf warned. And that’s “not good for patients or for industry.” Califf’s full speech is available at