Welcome back to the FDA, Robert Califf. Let’s get to work on your big idea

Now, more than ever, the U.S. needs scientific leaders who value Americans’ health and safety, who want to improve research and testing, and who are passionate about innovative ideas.

With Robert Califf once again confirmed as the commissioner of the Food and Drug Administration, it’s time to actualize a powerful idea he presented during his previous tenure: make the data submitted to the FDA during the drug development process publicly available.

With the world in the grips of a multiyear pandemic, implementing this idea should be among Califf’s top priorities. Potential pharmaceutical solutions need to be quickly assessed for safety and effectiveness, and sharing data will aid scientists in evaluating new methods that help them do so. What’s more, access to results of failed efforts can help others make more informed decisions about how to proceed with related work.

Pharmaceutical development is currently misled by outdated methods of assessment, methods that cannot reliably model human outcomes because they are conducted on mice, rats, rabbits, dogs, pigs, and monkeys.

Before a therapeutic can be sold in the U.S., it must first survive the FDA’s drug approval process. This extensive testing is intended to ensure that products will be safe for patients and will work for their intended purposes. This has traditionally included testing in both animals and humans, with multiple species of animals being used during nonclinical testing, which occurs before and concurrent with human testing in clinical trials.

Much of the initial research and testing done on animals, which is used to justify clinical trials, often cannot be reproduced, and much of the data submitted to FDA during drug development is never published — meaning it is never shared with the scientific community.

In 2016, Califf explained at an event hosted by the National Library of Medicine that a publicly available database would “free up all the data from the drug development process that is never seen by anyone. Because there is no compelling reason to publish them … if they’re unsuccessful, they get dropped …”

Providing public access to both nonclinical and clinical data submitted to the FDA — after being modified to protect privacy and trade secrets, of course — would allow scientists to assess the relevance of nonclinical animal testing, to assess the relevance of new human-specific testing methods that could significantly improve the nonclinical testing process, protect animals, and prevent wasteful, duplicative testing.

Modern, human-specific, nonanimal methods for testing drug safety and efficacy include the use of human cells, tissues, and data to improve the predictive ability of nonclinical studies. These advanced in vitro and computational approaches also offer the opportunity to study and account for patient-specific biological differences affecting drug outcomes, improving efficiency in drug development and paving the way for personalized medicine.

Many such human-specific methods have been commercialized, including micro-tissue models, organ chips, and biomarker-based metabolomics platforms, but many more are stalled in the evaluation process as scientists work to determine how they compare to demonstrated human outcomes, a standard that traditional animal tests have not been held to. This evaluation process could be accelerated by easier access to drug development data.

More than 88% of drugs that pass the preclinical phase end up failing in clinical trials. Without a doubt, this statistic can partially be attributed to the use of animal studies. Scientifically evaluated human-specific methods of assessment can only help improve this statistic.

With the launch of its Predictive Toxicology Roadmap in 2017, the FDA acknowledged the need to integrate new approaches for modeling human outcomes to improve drug testing. Some advanced human-specific methods, like reconstructed human epidermis, are appearing in industry submissions to the FDA, but many more are needed to bring safer and more effective medicines to the people who need them.

Making nonclinical and clinical data submitted to the FDA as part of the drug development process publicly available in a way that protects identity and trade secrets is the big idea that will accelerate the development of treatments for Covid-19 and many other diseases that continue to ail the global population.

We urge Califf to get to work ASAP on his big idea.

Elizabeth Baker is the regulatory policy director at the Physicians Committee for Responsible Medicine, an organization that advocates for changes in medical research and product testing that improve human outcomes while reducing and replacing the use of animals. Eryn Slankster-Schmierer is a science consultant to the organization.