low and burdensome” is how President Donald Trump has described the Food and Drug Administration’s drug approval process. His promise to speed it up by slashing regulations raised major safety concerns among scientists, regulators, and patient groups. But speed and safety do not have to be at odds. Modern tests that are physiologically relevant to humans because they are based on human biology — not on the biology of other animals — offer scientists more predictive information more quickly. FDA should focus on evaluating these technologies and work with industry to ensure they are implemented.
Dr. Scott Gottlieb, the new FDA commissioner, addressed the false dichotomy of speed versus safety in his congressional confirmation hearing. Through advances in regulatory science, he said that “we can have our cake and eat it too.” Gottlieb also said that the FDA should consider anything that can make the drug development process more predictive, such as better tools to evaluate safety and effectiveness and to bring down cost.
Implementing new test methods and protocols to assess the safety and efficacy of new drugs is essential. More than 95 percent of new drugs fail in humans after passing preclinical in vitro and animal tests. There were 25 clinical trial failures in the first quarter of 2017 alone.
This challenging situation provides an opportunity for FDA scientists and regulators to modernize preclinical drug testing. And it seems the FDA may be up for the challenge.
In fact, the FDA has already begun working to modernize drug development. The Critical Path Initiative seeks to improve the way drugs are developed, evaluated, and manufactured. And the America’s Got Regulatory Science talent competition seeks to help students work on innovative solutions to regulatory science challenges.
Gottlieb also said in his hearing that improving the quality of tools the FDA uses to test drugs — while still meeting the high bar for safety and efficacy — would be a priority. Greater support for research and development of new tools like tissue chips (also known as organs on chips), computer simulations, and 3-D bioprinting would a step in the right direction.
Tissue chips are transparent microchips about the size of a house key that are lined with living cells from the lung, liver, heart, skin, and other tissues or organs and that contain features designed to replicate the biological functions of those tissues and organs.
An added advantage of these approaches would be to reduce the use of animals in medical research, something that my organization, the Physicians Committee for Responsible Medicine, has worked to achieve since 1985.
The FDA already collaborates with the National Center for Advancing Translational Sciences on its tissue chips for drug screening program. Since 2012, this program has worked to develop tissue chips that can better predict whether a drug will be safe or toxic in humans.
The FDA also works with the Defense Advanced Research Projects Agency on an initiative to develop a system of organ chips to better predict the safety of a substance.
While tissue chips were initially developed to evaluate whether a drug or chemical is likely to be safe or toxic, the FDA may expand their reach. Earlier this month, the FDA announced it will begin a multiyear project to evaluate the technology for foods, cosmetics, and dietary supplements. The research will begin with a liver chip. The ultimate goal of this work is to predict how specific organs will respond to potential chemical hazards with greater precision than current methods, such as animal tests.
In addition to supporting the development of innovative new tools through advances in regulatory science, the FDA should also update its policies that prioritize traditional tests in animals. Current FDA regulations and internal policies discourage the use of modern tests like tissue chips by requiring data from animal testing. As more of the modern technologies that are based on human biology are developed and implemented as early in the testing process as possible, the review process will become more efficient without sacrificing necessary patient protections.
“It is a challenge and an opportunity to make sure that the FDA has the best training and tools, and is forward-leaning in adopting the best science into the principles used to govern the review process,” Gottlieb told Congress.
Advancing modern technologies that are based on human biology is the best way for the FDA to achieve both speed and safety in drug testing, reduce animal testing, and protect human health.
Elizabeth Baker, Esq., is senior science policy specialist at the Physicians Committee for Responsible Medicine. One goal of the organization is to help find alternatives to the use of animals for testing drugs, cosmetics, and other substances to be used by humans.