Makary, Prasad set out their vision for the FDA
Top priorities at the FDA in the coming years will include shorter review timelines for drug development, embracing the use of artificial intelligence and big data, and speeding up approvals for biosimilar and generic medicines, according to top agency officials.
In an editorial in medical journal JAMA, FDA Commissioner Marty Makary and the new director of the Center for Biologics Evaluation and Research (CBER), Vinay Prasad, also said they aim to work closely with the pharma industry, but "guard against a cozy relationship that has characterised the agency in the past and led to allegations of industry capture."
The article emerged as Health and Human Services (HHS) Secretary Robert F Kennedy Jr fired all the members of the CDC's Advisory Committee on Immunisation Practices (ACIP), accusing them of "rubber stamping" decisions on behalf of vaccine developers.
Alluding to that move, the FDA officials also said that at a recent meeting of FDA's Vaccines and Related Biological Products Advisory Committee (VRBPAC), they granted no so-called '502' waivers, which waive voluntary disclosure of potential conflicts of interest.
Makary and Prasad also repeat various themes repeatedly visited by Kennedy on his Make America Healthy Again (MAHA) soapbox, focusing on chronic diseases – particularly in children – and pledging to look at possible links with "ultraprocessed foods, food additives, and environmental toxins."
Specific plans include a pilot programme for accelerated reviews that could deliver decisions for sponsors "within weeks," aided by pathways allowing information on manufacturing, labelling, and packaging to be submitted even before trials are completed.
"The time from when pivotal trial results are known to when decisions are made must be shortened. This has implications for public welfare and will improve the risk-reward calculation of drug development," they wrote.
AI and big data
Earlier this month, Makary announced the early launch of a large language model (LLM) AI called Elsa to speed up reviews, sparking concerns that AI is being adopted by the regulator with over-inflated claims about its capabilities and a lack of "guardrails" to ensure it is secure and fit for purpose.
In the editorial, Makary and Prasad reiterate that AI is a "top priority" for improving efficiencies at the agency, which had around 3,500 staffers axed in the first quarter of this year, raising fears about its ability to meet deadlines on regulatory reviews. They also promise to speed up reviews of AI-based technologies and increase the use of AI-powered modelling to reduce the need for animal testing.
On big data, the plan is to harness it to explore the root causes of chronic diseases and monitor FDA-approved products for safety.
"Advances in causal inference in [non-randomised data], including the use of target trials, which attempt to balance confounding and time zero, have potential to yield actionable causal conclusions, in many cases at lower cost," they wrote.
Finally, while acknowledging that the FDA has little power to control drug prices, the FDA will work to streamline the approval process for biosimilar and generic medicines and will support President Donald Trump's recently announced most favoured nation (MFN) policy, which would link prices to those offered in other comparable economies.
"In summary, the FDA will be focused on delivering faster cures and meaningful treatments for patients, especially those with neglected and rare diseases, healthier food for children, and common-sense approaches to rebuild the public trust," said Makary and Prasad.
