Advancing Regulatory Science

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Science  25 Feb 2011:
Vol. 331, Issue 6020, pp. 987
DOI: 10.1126/science.1204432

Ensuring the safety and quality of food and medical products has never been more complicated. Societies around the world face increasingly complex challenges that require harnessing the best available science and technology on behalf of patients and consumers. This effort requires a strong field of regulatory science to develop new tools, standards, and approaches that efficiently and consistently assess the safety, efficacy, quality, and performance of products. Yet, despite being a critical component of the scientific enterprise, regulatory science has long been underappreciated and underfunded.

Today, we are neither effectively translating scientific discoveries into therapies nor fully applying knowledge to ensure the safety of food and medical products. We must bring 21st-century approaches to 21st-century products and problems. Toxicology is a prime example. Most of the toxicology tools used for regulatory assessment rely on high-dose animal studies and default extrapolation procedures and have remained relatively unchanged for decades, despite the scientific revolutions of the past half-century. We need better predictive models to identify concerns earlier in the product development process to reduce time and costs. We also need to modernize the tools used to assess emerging concerns about potential risks from food and other product exposures.


The U.S. Food and Drug Administration (FDA) is prepared to lead the way in strengthening regulatory science and transforming toxicology. But this will require collaborations and partnerships with academia, industry, and other government agencies. Fortunately, this work has already begun. For example, the FDA and the European Medicines Agency have recently worked to characterize novel biomarkers that identify drug-induced kidney toxicity in preclinical animal models, and several of these biomarkers have now been qualified for regulatory use. And last year, the FDA and the U.S. National Institutes of Health (NIH) launched a new NIH-FDA Regulatory Science Initiative to encourage new research in the field; we recently awarded our first set of grants—$9.4 million over 3 years to support four research projects. The FDA will continue to make targeted investments in such collaborations, including, if resources are available, Centers of Excellence in Regulatory Science housed in academic settings and focused on collaborative, multidisciplinary, multisectoral regulatory science research.

With an advanced field of regulatory science, new tools, including functional genomics, proteomics, metabolomics, high-throughput screening, and systems biology, can replace current toxicology assays with tests that incorporate the mechanistic underpinnings of disease and of underlying toxic side effects. This should allow the development, validation, and qualification of preclinical and clinical models that accelerate the evaluation of toxicities during drug development. The goals include developing biomarkers to predict toxicity and screen at-risk human subjects during clinical trials, as well as after new products are on the market. The FDA is also working to eventually replace animal testing with a combination of in silico and in vitro approaches. The inherent complexity of the vertebrate reproductive system represents a major challenge to developing such technologies that replace whole-animal tests, and advanced regulatory science is needed to address this challenge. We must also develop new science to protect the safety of our food supply: for example, to identify the effect of food production, processing, preparation, and use on nutrient content, toxic contaminant generation, and inactivation of naturally occurring toxins.

Ultimately, investments in regulatory science can lead to a new era of progress and safety. Because such investments will promote not only public health but also the economy, job creation, and global economic competitiveness, they have major implications for the nation's future. Policy-makers, industry leaders, and the scientific community have the opportunity and the power to answer this call to action. It cannot wait any longer.

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