As the biotech world flocks to San Diego today for the BIO International Convention, the Department of Health and Human Services has provided plenty of conversation fodder with the announcement of a series of efforts to regain ground on China’s surging early-stage clinical development capabilities.
In a call with journalists, Acting FDA Commissioner Kyle Diamantas and Jay Bhattacharya, M.D., Ph.D., director of the National Institutes of Health and acting director of the Centers for Disease Control and Prevention, sketched an outline of reforms meant to hasten phase 1 trials and encourage companies to conduct the studies in the U.S. rather than speedier locales like China and Australia.
The efforts are part of an HHS-wide initiative called Operation Trial Blazer, Diamantas said.
“We've been witness to a growing share of phase 1 clinical trials moving overseas, delaying opportunities for American patients and weakening the nation's position as a global leader in biomedical research,” the acting FDA chief said. “FDA is taking action to reverse that trend.”
The FDA’s efforts are meant to reduce phase 1 timelines by six to 12 months, Diamantas said. They include what he called an “expedited” investigational new drug pilot program, updated guidance on the use of advanced computer models for dose selection and updated frameworks for clinical trial master protocols.
A senior HHS official on the call provided further detail to Diamantas’ remarks, describing how the agency will be “clarifying what you need to do for a first-in-human phase 1 with respect to the chemistry manufacturing controls,” as well as issuing further guidance on shifting away from animal models for preclinical testing and providing more direction for sponsors on dosing.
In addition, the official said, the FDA will launch a website and call center specifically built for sponsors to find answers for all their phase 1 trial questions. This will make it easier for small companies especially to “navigate the agency to get those questions answered to keep those trials moving forward.”
HHS requested that all comments on the call outside of Diamantas’ and Bhattacharya’s opening remarks be attributed to a senior HHS official.
The FDA shared three draft guidances today related to the officials’ announcements. One reiterates the regulator’s previously stated preference for approvals to be based on only one clinical trial, while the other two outline the planned master protocol and dose selection reforms.
Recently departed FDA Commissioner Marty Makary, M.D., spoke frequently about his desire to revamp the phase 1 trial process. The efforts being undertaken at the agency long predate Makary’s departure, another senior official told Fierce on the call, with many being led by career staff.
“This work has begun long, long before Commissioner Makary left the agency, and it continues to this day,” the official said. “This still remains a top focus for us at the agency, across both drug product centers.”
In May, an HHS spokesperson told Fierce that reforms launched by Makary were continuing to move forward after his exit.
On NIH’s part, Bhattacharya said the agency plans to use its clinical trial network to test out potential new study designs that can then be rolled out more broadly.
“We'll augment our use of real-world data and causal inference methods to improve protocol feasibility, recruitment planning and evidence generation in ways that make trials look faster and more informative,” the NIH director said. There will also be a push to ensure that underserved and rural population can access trials, he added.
NIH will seek comment soon on its reform plans, Bhattacharya said, including plans to reshape requirements for institutional review boards to approve trial designs.
“The goal is to streamline trials while protecting human rights,” he said.
A press release shared with journalists highlighted numerous vague efforts being undertaken by other health centers, including the NIH’s National Center for Advancing Translational Sciences and National Cancer Institute as well as HHS’ Office of the National Coordinator for Health Information Technology.
Much has been made in recent years of China and Australia’s prowess in early-stage research, with America’s comparatively slower process often named as a key weakness in competition with China’s biotech industry. In the press release, HHS Secretary Robert F. Kennedy, Jr., decried this trend.
“America should be the best place in the world to develop new medicines, yet we have built a system that drives too much clinical research overseas,” Kennedy said. “HHS is launching a coordinated department-wide effort to restore America's leadership in clinical research, remove unnecessary barriers and bring more clinical research and investment back to the United States.”
“America led the world in medical innovation before,” Kennedy added. “We will lead again.”