Sunday, October 17, 2010

Advancing Regulatory Science

“We’re more poised than ever to deliver science to serve patients but to do so requires advanced and more effective application of regulatory science—the discipline at the very heart of our mission at FDA,” said FDA’s Commissioner Dr. Margaret Hamburg.

Speaking at a National Press Club luncheon on October 6th, , she said “We’ve witnessed the discoveries that hold major promise for therapeutic advances in fields as diverse as genomics, synthetic biology, stem cells, and nanotechnology.”

She continued to say, “However, we lack the ability to effectively translate many of these developments into vital products to help those who need them. The number of new therapies is on the decline while the costs to bring the therapies to market have soared. Because of the troubling gap between advances in science and available patient care, we need to build a bridge across this gap and that bridge is referred to as regulatory science.”

Dr. Hamburg announced that the FDA white paper “Advancing Regulatory Science for Public Health”, a framework for FDA’s regulatory science initiative is now available. Section IV in the white paper, points out that the FDA at the present time does not have the right infrastructure, tools, and resources available to organize and analyze all of the large data sets across the multiple studies and data streams.

The report also explains how the vast data stored at FDA needs to be transformed into a harmonized format and organized in a common database so that it can be queried by topic and analyzed to address key questions. These goals require investments in informatics hardware and software but at the same time, standardized data models need to be developed for relational databases and scientific computing.

Also, FDA needs to concentrate on real-time monitoring of safety data using healthcare data. The goal for the agency is to expand and harmonize FDA’s electronic systems to receive, process, store, and analyze adverse event reports and other safety information for FDA-related products while at the same time ensuring patient privacy.

In addition according to the whitepaper, investments in new software tools are needed along with collaborative projects that bring together the latest technologies and approaches for mining complex data from clinical trials, healthcare settings, and biological studies.

Dr. Hamburg gave an example of how FDA and NIH, foundations, and industry have been working hard to develop an artificial pancreas for juvenile diabetes. The artificial pancreas is designed to continuously monitor blood sugar levels and inject the right amount of insulin automatically. This is a complex task, so scientifically solid testing path has to be developed to insure that the device controls blood sugar levels without risking hypoglycemia. Next month, FDA will be holding a meeting on establishing standards needed for approval of the artificial pancreas.

Dr. Hamburg described FDA efforts for 2011. As she sees it, the agency will continue to make strategic targeted investments in areas that support regulatory science. There are plans to establish pilots and feasibility studies and to expand these activities in the next few years to establish centers of excellence in regulatory science funded by FDA. The plan is to focus on collaborative, multidisciplinary, and multi-sector research activities that most likely will be housed in academic settings.

In addition, FDA is working with NIH on a new regulatory science initiative to collaboratively encourage research in the field. Last week, FDA/NIH awarded more than $9 million to go to four projects to research nanoparticles, develop a heart/lung model to test the safety of new drugs, develop innovative clinical trial design strategies, and to develop a novel strategy to predict eye irritation.

Dr. Hamburg concluded by saying, “We are living in the century of biology where scientific advances are ripe. With a collective effort, these advances can be transformed into therapies to alleviate or prevent much suffering, and enhance our quality of life.”

Go to to download the whitepaper.