An America First Approach to Medical Innovation

FDA Voices

By: Kyle Diamantas, J.D., Acting Commissioner of Food and Drugs; Karim Mikhail, B.Pharm, MSc, Director, Center for Biologics Evaluation and Research; and, Michael Davis, M.D., Ph.D., Director, Center for Drug Evaluation and Research

America has long been the world’s premier destination for medical innovation. Our universities make groundbreaking discoveries. Our biotechnology companies turn them into medicines. Our capital markets finance risks few other countries can match. And the U.S. Food and Drug Administration ensures patient safety and efficacy.

This leadership can’t be taken for granted.

Today, a first-in-human clinical trial can take years to initiate in the United States versus just months in China and Australia. When clinical development happens faster overseas, America risks losing investment, intellectual property, scientific talent and, most important, early access to promising new therapies.

That is why the FDA is launching the Expedited Investigational New Drug (IND) Pilot on September 15. This is a key priority of the Trump Administration to retain American leadership in biomedical advancements.

The premise is simple: We can move faster without lowering our standards.

For decades, the IND process has helped protect patients as experimental therapies move from the laboratory into humans. Those protections are indispensable.

However, drug development increasingly involves complex biologics, cell and gene therapies and other novel technologies that scarcely existed when much of this regulatory architecture was designed. Sponsors today preparing for a first-in-human trial often must navigate a process that is can be unclear and lengthy.

Uncertainty has a cost. If a company isn’t sure what the FDA needs, the rational response is to do more. But data that may not be necessary to protect participants in a Phase 1 trial can consume months unnecessarily.

The FDA has already begun addressing this problem by clarifying phase-appropriate requirements. Our updated resources clearly explain what companies need for first-in-human development rather than leaving them to speculate, saving up to 12 months in the pre-IND phase.

The Expedited IND Pilot goes further by changing how sponsors, research institutions and the FDA work together.

Under the pilot, participating drug sponsors partner with qualified research institutions (QRI) with scientific and regulatory expertise, something America possesses in abundance. These can include leading academic medical centers, contract research organizations and other institutions capable of helping sponsors prepare high-quality IND applications.

A QRI will work alongside the sponsor as it prepares its IND, providing substantive scientific input and helping validate individual components before they reach the FDA. The FDA, in turn, will be able to review nonclinical evidence, manufacturing, and product-characterization on a rolling basis rather than waiting.

The traditional process forced much of that work to accumulate before regulatory review begins. Under the pilot, participating sponsors and their QRI partners will be able to submit components as they become ready.

FDA scientists can identify questions earlier. Sponsors can resolve issues earlier. Problems that might otherwise emerge at the end of the process can be addressed while development is ongoing.

The formal 30-day IND review period doesn’t disappear; it begins once the final component is submitted. Nor does the FDA surrender any of its oversight — we will continue to determine whether a clinical investigation may proceed and whether a clinical hold is necessary.

What changes is what is happening before the clock starts.

The goal is to replace unnecessary waiting with productive scientific engagement. Better-prepared submissions mean fewer surprises during formal review, greater predictability for sponsors and a shorter path from discovery to the first patient enrolled in a clinical trial.

However, the FDA review is only one part of the overall delay drug innovation in America is facing.

Even after an IND proceeds, a trial can be delayed by Institutional Review Board (IRB) review, contracting, site preparation and activation. These steps frequently occur after FDA submission, when they often could occur in parallel. Through Operation TrialBlazer, the Trump Administration is confronting all these bottlenecks and working with other agencies across HHS to address them holistically.

The Expedited IND model helps connect these pieces and will support activities such as IRB review, contracting and site preparation beginning alongside regulatory work instead of waiting in line behind it.

That is what genuine regulatory modernization should look like — implementing changes to improve efficiency without changing standards that protect patient safety. The FDA expects that the pilot will initially select eight to 10 sponsor-QRI pairs. We will study what works, what doesn’t, and whether the model can support a more durable system.

Michael Davis, M.D., Ph.D.

The stakes extend far beyond the FDA.

When an American scientist discovers a promising therapy, the fastest route to testing it in patients should run through the United States — not around it. American patients should not have to travel abroad because a promising treatment entered the clinic somewhere else first. American entrepreneurs should not conclude that another country offers a better path for testing an invention developed here. They should feel confident to invest at home.

President Trump and Secretary Kennedy have directed us to use every tool available to restore American leadership in medical innovation. The Expedited IND Pilot is part of that effort.

For generations, innovators around the world have looked to the United States as the best place to turn a scientific discovery into a safe and effective medicine.

Our job is to make sure they still do.