FDA’s new predictive toxicology roadmap will improve human safety

FDA’s new predictive toxicology roadmap will improve human safety
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What happens when you replace an outdated paper map with Google Maps? Predictive technology helps you find the best route, avoiding bottlenecks that may be blocking your way, so that you can safely and quickly reach your destination.

The U.S. Food and Drug Administration’s new Predictive Toxicology Roadmap will do the same for drug testing by ensuring that high-tech methods such organs-on-chips reduce and replace the use of animal tests to better predict the safety of FDA-regulated medical products.

The FDA says the six-part framework — including an organizing committee, training, continued communication, collaborations, research, and oversight — will help develop a more efficient process. This process will help with identifying and qualifying technologies that can more quickly move products to market and prevent dangerous ones from ever reaching market.


“Moreover, in many cases, these technologies are reducing the need for animal testing — advancing FDA’s long sought goal of refining, reducing, and replacing testing on animals,” wrote Denise M. Hinton, FDA’s acting chief scientist, and Suzanne Fitzpatrick, Ph.D., senior advisor for toxicology in FDA’s Center for Food Safety and Applied Nutrition, in FDA Voice.

FDA commissioner Scott Gottlieb, M.D., has consistently addressed the need for more predictive approaches to protecting human health and supporting scientific innovation.  During congressional hearings prior to being appointed to his position, he said that both speed and safety are achievable through advances in regulatory science, that “we can have our cake and eat it too.”

He also said that the FDA should consider approach that can make the drug development process more predictive, such as better tools to evaluate safety and effectiveness and to bring down cost.

FDA’s Predictive Toxicology Roadmap will help reach that goal — and not a moment too soon. A new drug can take 10 to 15 years and more than $2 billion to develop, and 95 percent fail in human studies, according to the National Institutes of Health.

Organs-on-chips — devices the size of a memory stick that use human cells to model the structure and function of human organs — are already being used to help overcome these obstacles. Researchers at Wake Forest Institute for Regenerative Medicine recently combined micro hearts, lungs, and livers to create a body-on-a-chip to test how the whole body would respond to a drug.


“If you screen a drug in livers only, you’re never going to see a potential side effect to other organs,” said one of the researchers. “By using a multi-tissue organ-on-a-chip system, you can hopefully identify toxic side effects early in the drug development process, which could save lives as well as millions of dollars.”

The FDA’s new plan could also help encourage the development of technologies to help avert a drug disaster in the United States like the one that occurred in France last year. A previously healthy man died and four others experienced brain damage during a clinical trial of the experimental drug BIA 10-2474, which had previously been tested in animals.

Following the tragedy, a group of researchers screened the compound using human cells and brain tissues. The researchers found that BIA 10-2474 may disrupt how neurons in the brain metabolize lipids, effects not seen in preclinical experiments on mice, rats, monkeys, and dogs.

Another method the FDA is recommending is “read across,” which allows scientists to apply toxicity data from a chemical that has already been tested to a similar chemical that has not been tested.

Earlier this year, we recommended this method, among others, to the FDA at the Interagency Coordinating Committee on the Validation of Alternative Methods’ annual public forum. Congress established ICCVAM in 2000 to promote acceptance of tests that either refine procedures to lessen animal pain or distress, reduce the number of animals used in a particular test, or replace animals with test methods such as cells, tissues, or predictive computer models.

Continuing these collaborations and training scientists on these new methods — both of which the Roadmap calls for — will be essential for the FDA to reach its final destination of improved safety for humans while reducing and replacing animal testing.

Elizabeth Baker, Esq., is a senior science policy specialist with the nonprofit Physicians Committee for Responsible Medicine. Baker works to modernize drug development to save human and animal lives by shifting preclinical research from the use of animal models to modern technologies that are relevant to human biology.