Gottlieb Takes to Twitter for AI, Next Generation Sequencing Announcements

FDA Commissioner Scott Gottlieb tours the agency's Center for Devices and Radiological Health (CDRH) labs. Image courtesy FDA’s Flickr page.

As is now common practice for government officials, the head of the FDA just made a pair of important announcements via Twitter. They concern new regulatory approaches to artificial intelligence (AI) and next-generation sequencing (NGS).

“Artificial Intelligence and Machine Learning hold enormous promise for the future of medicine,” Commissioner Scott Gottlieb, MD, tweeted. “FDA is taking steps to promote innovation and support the use of artificial intelligence based medical devices.”

He wrote that the agency is moving towards a pre-certification pathway for software that “needs to continue to learn and improve as it’s used.” He added that the FDA’s looking at an “efficient” way to evaluate algorithms, with an emphasis on real-world evidence and input from researchers, developers, providers, and consumers.

>>READ: Key Takeaways from Scott Gottlieb's Healthy Returns Interview

Gottlieb has expressed similar inclinations in the past, and the FDA has begun to act on them. Yesterday, the agency approved a first-of-its-kind AI system for diagnosis of diabetic retinopathy. “Today’s decision permits the marketing of a novel artificial intelligence technology that can be used in a primary care doctor’s office,” the agency’s Malvina Eydelman, MD, said in a statement. The program at hand, however, was approved for use in Europe nearly 5 years ago.

This morning, he teased another big announcement: “When it comes to complex, life-threatening diseases like cancer, promoting and protecting public health requires FDA to adopt policies to ensure regulation serves as bridge to innovation, not a barrier to it,” he tweeted.

The message included a link to the full prepared text of the keynote speech he delivered today at the 2018 Community Oncology Conference in National Harbor, Maryland. He detailed the promise that NGS technologies hold for finding new pathways to treat cancer and said that the FDA needs to keep pace with the “rapidly falling cost” and “fast-moving” developments in the field.

The speech is packed with interesting points. He mentioned costs—both for patients procuring prescriptions and for pharma companies developing new treatments—as 2 of the most important problems facing healthcare today. “The perverse reality of the market today is that cancer treatment comes with its own financial toxicity,” he said, and too much money is being spent on drugs that ultimately fail trials.

Encouraging smarter drug development with new regulatory pathways for NGS, he said, could help bring down overall costs. The agency has released 3 new draft guidance documents for NGS technologies to help steer developers:

• "Considerations for Design, Development, and Analytical Validation of NGS-Based In Vitro Diagnostics Intended to Aid in the Diagnosis of Suspected Germline Diseases," which describes the FDA’s analytical validation recommendations and could “spur the creation of consensus standards for NGS-based tests.”
• "Use of Public Human Genetic Variant Databases to Support Clinical Validity for Genetic and Genomic-Based In Vitro Diagnostics," which describes how developers can access large public genetic databases, like that National Institutes of Health ClinGen database, to gain verified scientific basis for premarket review.
• "Investigational In Vitro Diagnostics in Oncology Trials: Streamlined Submission Process for Study Risk Determination," which outlines a “streamlined submission process” for determining the amount of risk involved in an in vitro trial. Gottlieb called this guidance “step toward our goal of having a common filing for a drug and diagnostic system where the drug is co-developed with a diagnostic test.”

While development costs are astronomical, there is some debate about whether they are truly the driver of the high drug and treatment prices that patients have to pay—even for novel, groundbreaking new cancer treatments. Gottlieb himself comes from the pharmaceutical industry, and in a poll of other pharma executives conducted before his nomination to head the FDA he got 72% of the vote—at the time, some expressed concerns about conflicts of interest.

Few will deny, however, that NGS and AI technologies are evolving quickly and require special regulatory consideration. Today’s announcements—and the means by which he made them—fit Gottlieb’s overall goal of creating a streamlined FDA that bases its reviews on a risk-based approach.

“America’s innovation ecosystem depends, above all, on public confidence in the institutions and rules that support it,” he said in today’s speech.

Related Coverage:

First-of-its-Kind AI Tool for Diabetic Retinopathy Detection Approved by FDA

FDA Streamlines Review Pathway for Certain Glucose Monitoring Devices

Gottlieb Announces More Regulatory Updates to Encourage Device Innovation