FDA plans to release AI drug development guidance this year

The US Food and Drug Administration (FDA) plans to release a draft guidance this year on the use of artificial intelligence/machine learning (AI/ML) to support drug development. The guidance will be informed by the agency’s experience in reviewing submissions containing AI/ML elements, as well as feedback it received on two discussion papers on the topic released last year, said Tala Fakhouri, associate director for policy analysis in the Office of Medical Policy (OMP) at FDA’s Center for Drug Evaluation and Research (CDER).

Fakhouri made these comments at the agency’s Regulatory Education for Industry (REdI) conference on Wednesday. FDA’s progress in developing a framework for AI was one of the topics of discussion, as well as the growing use of AI in regulatory submissions.

She said that Issuance of such guidance will fulfill a mandate in President Biden’s Executive Order 14110 published last October, which requires government agencies, including the Department of Health and Human Services (HHS) to issue certain deliverables related to AI/ML; one of these deliverables was to define objectives and goals for regulating AI throughout the different phases of drug development.

Fakhouri told the meeting CDER that has received over 300 submissions with AI components; these elements traverse the landscape of drug development from drug discovery to post market safety monitoring and cover a range of therapeutic areas. Most of the submissions were in the oncology area, followed by psychiatry, gastroenterology, and neurology.

In 2016, there was one investigational new drug applications (IND) with AI elements, by 2021 this number had grown to 128 submissions with these components.

She noted that AI tools can be used to identify drug targets, in nonclinical studies, dose finding studies, clinical research, site selection, participant recruiting and retention, and to analyze real-world data (RWD). It can also be used in advanced pharmaceutical manufacturing and post-marketing safety surveillance.

Fakhouri said FDA received 800 comments from 60 organizations on the agency’s two discussion papers on incorporating AI into the pharmaceutical manufacturing and drug development regulatory framework. (RELATED: FDA publishes discussion paper on AI/ML in drug development, Regulatory Focus 12 May 2023; AI in drug manufacturing: Stakeholders call for harmonization, further guidance, Regulatory Focus 1 March 2023)

The responses sought clarity on the following points: the scope of FDA’s oversight of AI, how to operationalize a risk-based approach, ensuring transparency, and the level of detail and documentation to support AI/ML in applications submitted to the agency.

Respondents also urged FDA to harmonize its AI regulatory requirements with other regulators globally, to align these regulations with the Centr for Devices and Radiological Health (CDRH) and to establish partnerships to advance the creation and sharing of machine-readable data sets for drug development.

These comments, along with FDA’s experience in reviewing AI/ML submissions and current regulatory science research will be used to inform a guidance FDA plans to issue this year on AI/ML in drug development. The guidance will also be developed with input from FDA’s medical product centers, the Oncology Center for Excellence (OCE), and the Office of Combination Products.

Fakhouri added that “we really want your feedback so when the guidance goes final, this is informed by your experience and the work that you’re doing.”

Besides the guidance, FDA also plans to develop a set of best practices in using AI and a guidance on consistent terminologies related to AI.

REdI meeting

FDA plans to release AI drug development guidance this year

Related topics

Recommended content

Welcome to the new RAPS Digital Experience