U.S. drugmakers have increasingly looked to China to source new experimental medicines, taking advantage of heavy government life sciences investment and a regulatory environment that can enable clinical trials to begin within 18 months of discovery of a new therapeutic molecule.
Now, the federal government is responding, publishing a blueprint on Monday that aims to ease the burden on biotechnology companies preparing to start clinical trials as well as expand patient access to trials. The HHS is also examining whether paying stipends to trial enrollees, in addition to reimbursing them for health insurance cost-sharing and other expenses, could be legal under federal anti-kickback laws.
The plan opens another front in the government’s efforts to protect U.S.-based pharma and biotech manufacturing and innovation from overseas competition. President Donald Trump has strongarmed big pharma into spending money to open and expand manufacturing plants in the U.S., while members of Congress have been advancing legislation to mandate Treasury and Defense Department review of U.S. drugmakers’ deals with Chinese counterparts.
Former Food and Drug Administration Commissioner Scott Gottlieb praised the proposal. The U.S. “has an inherent advantage in conducting proof-of-concept studies, because good medical care, to support patients receiving complex therapies, is key and U.S. does that best,” Gottleib wrote on X. “But the time and cost advantage can’t tilt so heavily against U.S.-based trials or we’ll lose our edge.”
The blueprint focuses heavily on experimental drugs being readied for their first-in-human tests. The FDA now will specify what data is necessary before filing an Investigational New Drug application, seeking to reduce unnecessary documentation on toxicology or chemistry, manufacturing and controls. The agency is also seeking to make trial protocols more flexible to keep drug developers from having to file complex amendments when adjustments to a trial plan must be made.
The FDA is proposing, too, a pilot project that will permit developers to consult with a network of research institutions as they prepare their IND applications. Additionally, the federal agency will create a “rolling submission” platform that will allow the FDA to review the institutions’ recommendations and provide “timely guidance” to drug developers.
HHS’ proposal is also looking at ways to expand enrollment in clinical trials with U.S.-based patients by helping physicians identify eligible people and better integrating trials with everyday care practices, as well as potentially expanding the types of compensation, including stipends, that trials can offer to encourage patients to join.
The National Institutes of Health will play a role in this effort, promoting technologies and practices like telehealth, remote monitoring, real-world data and artificial intelligence that can help people from rural and underserved areas participate in trials.
