The US Food and Drug Administration (FDA) has made “significant strides” in strengthening its scientific capabilities, though there is still some concern about the agency’s ability to support and stimulate biomedical innovation, according to a new report from a subcommittee of FDA’s Science Board.
Six years after a 2007 report from the Science Board -- which, among other findings, called attention to FDA’s “weak scientific base and inadequate scientific workforce” and “highly doubtful” ability to ensure the rapid entry of life-saving new medical therapies – the Science Board, an advisory committee of national experts across various scientific disciplines, created a “Science Looking Forward Subcommittee.”
The subcommittee was tasked with assessing the progress of FDA since 2007, in terms of how it can “meet emerging and future trends in science and technology,” improve collaborations with outside groups and support a culture of science.
The subcommittee notes at the outset of its report, called “Mission Possible: How FDA Can Move at the Speed of Science,” that FDA has made many substantive changes “that significantly address issues identified in 2007.” Highlights include:
- the creation of the Office of the Chief Scientist and other offices to support regulatory science, health informatics, the professional development of scientists, scientific integrity, minority health and a group devoted specifically to countermeasures for acts of terrorism and emerging threats
- new authority from Congress and increased funding, from both appropriations and user fees, enabling FDA to improve its ability to tackle public health problems
- initiatives that deal with new and emerging technologies, including stem cells, 3D printing, predictive toxicology, genome sequencing and computer simulation
- the Precision Medicine Initiative, in collaboration with the National Institutes of Health (NIH)
- advances in regulatory science that promote the lifecycle approach to regulation for drugs, devices and biologics
- increased focus on active post-market surveillance, primarily related to the new Sentinel active surveillance system
However, the subcommittee “notes with concern” that medical product quality control procedures have not kept pace with advances in research and product development, and that patients and industry are concerned with FDA’s ability to support biomedical innovation.
More specifically, the subcommittee calls on the agency to facilitate the qualification of biomarkers, including surrogate endpoints, for evaluation of new therapies and providing guidance on how new biomarkers can be qualified as surrogate endpoints; help develop and encourage the use of clinical trial networks and master protocols, expand access to external experts in emerging technologies to expedite approvals and use data-mining and analytical tools to further evaluate the safety and efficacy of new drugs and devices.
Looking ahead, the subcommittee also says FDA needs to focus specifically on product manufacturing and quality, modernizing toxicology, being more assertive in adding new research university and foundation partnerships and offering more pay and other perks to help recruit and retain top scientists.
Echoing a similar call made by acting Commissioner Stephen Ostroff last week at the FDA Science Writers Symposium, the subcommittee says that “several resource deficits exist that the Subcommittee believes threaten FDA’s ability to advance in the future.”
The subcommittee points to the House-approved 21st Century Cures bill, which the Congressional Budget Office says could add $1 billion in new costs to FDA’s operations over the first five years following the bill’s enactment.
“Although the House bill has a provision for partial funding of those costs, even that partial funding is uncertain,” the report says. “Therefore, the Subcommittee expresses its strong opinion that an unfunded mandate of that magnitude could set back the progress of medical product development by forcing FDA to divert its scientific staff away from its currently successful review programs, thus harming patients and the progress of biomedical innovation.”
Science Looking Forward Subcommittee Report