Citing how its mission continues to be “at risk,” the FDA—in a new report from its Science Board—said the agency’s expansion and extraordinary range of increasingly complex responsibilities and insufficient resources needs to be addressed soon.

The 36-page report, titled “Mission Possible: How FDA Can Move at the Speed of Science,” highlights regulatory science efforts important for developing more novel drugs and biologics, including qualification of biomarkers and encouragement of more efficient and lower-cost clinical trials through the use of master protocols and trial networks.

Intended as a follow-up to the Science Board’s 2007 report, which stated that the FDA could not fulfill its mission due to a weak scientific base and inadequate scientific workforce, it also cited a disparity between the FDA’s responsibilities and resources that threatened the agency’s ability to perform its mission. In addition, there were doubts regarding its capacity to ensure the rapid entry into the market of life-saving new medical therapies—especially those resulting from new and emerging technologies. Another major concern from the 2007 report, titled “FDA Science and Mission at Risk,” was that the FDA did not have the capacity to ensure the safety of the nation’s food supply.

Fast-forward to 2015. The new report outlines significant progress since 2007, starting with the creation of the Office of the Chief Scientist to ensure better coordination and promotion of the FDA’s science, along with new statutory authority from Congress that not only improves the FDA’s ability to tackle public health problems but also expands the agency’s regulatory responsibilities. Among them: tobacco products, food safety, compounding pharmacies and biosimilars—the last prompting Janet Woodcock, director of the Center for Drug Evaluation and Research (CDER), to stress the importance of ensuring that the evaluation of new therapies is based on sound science.

“We have to get the science right,” Woodcock told a Senate Health, Education, Labor & Pensions Committee subcommittee during a discussion on biosimilar regulations, explaining the FDA’s lengthy and deliberate process for issuing important policy advisories on biosimilars.

In recent years, the FDA has launched initiatives across FDA programs to deal with new and emerging technologies such as stem cells, 3-D printing, predictive toxicology, genome sequencing and computer-related simulation. Also included was the Precision Medicine Initiative, in a partnership with the National Institutes of Health to revolutionize personalized medicine.

The new report, however, cautions that medical product and food manufacturing quality control procedures “have not kept pace with advances being made in research and product development and that more research is needed in such areas as product design, manufacturing and quality assurance.”

Despite substantial improvements since 2007, the report identified a range of concerns needed to support and stimulate biomedical innovation:

• Facilitate the qualification of biomarkers, including surrogate endpoints, where appropriate for the evaluation, especially for conditions such as Alzheimer’s disease that have no effective treatments.
• Increase the efficiency and lower the cost of clinical trials by making it easier for the development and encouragement in using clinical trial networks and “master protocols,” especially in the areas of oncology and antimicrobial therapy.
• Expand access to external experts in emerging technologies to help accelerate approvals.
• Use new data-mining and analytical tools to evaluate the efficacy and safety of new medical products, including full development of the FDA’s Sentinel Initiative to evaluate the effects of major safety warnings or label changes on the use of drugs.

The report also urged major improvements in recruiting a “21st century scientific workforce” that called for changing “an archaic and ponderous human resource system that has further impeded FDA’s ability to recruit outstanding talent.” That proposal includes implementing expanded retention to pay for hard-to-recruit specialists such as specialty physicians, biomedical engineers, biostatisticians and toxicologists. It also called for the FDA to seek authority to “offer special pay levels, up to that of the President, for outstanding and senior scientists, whom the Agency cannot now recruit due to restrictions on Federal pay.”

While initial reaction for continuing to keep pace with scientific changes drew praise from some nonprofit organizations, others questioned whether some of the priorities should become part of the FDA’s mission.
“[The report] provides a clear-eyed assessment of what needs to be done for the FDA to keep pace with rapidly advancing science and remain the cutting-edge public health agency that our nation needs,” said Jeff Allen, Ph.D., executive director of Friends of Cancer Research. “But some of these priorities should not be shouldered solely by the FDA. The report, however, puts it all on the FDA when the increasing growth costs should not be solely the responsibility of the FDA.”

Allen said new FDA approaches to clinical trials should not require master protocols, as that is where industry has a major role in deciding how to use them.

“For medical products, the National Institutes of Health and private industry will continue to be the engines that drive breakthroughs, but the FDA will continue to demonstrate leadership in advancing science in emerging areas like precision medicine,” said Cynthia Bens, vice president for public policy at the Alliance for Aging Research. “The issue, in my view, is not with how broad FDA’s mission is, but rather the inability of funding to keep pace with their resource needs as the agency’s responsibilities increase. No other federal, state or private agency provides a fallback if FDA can’t fulfill its ever-growing mission.”

Ronald Rosenberg is a former business and science reporter for The Boston Globe. He has written features for New Scientist and Inc. magazine. His lengthy journalism career includes editing an award-winning weekly newspaper in Cornwall, N.Y. Ron also was a media relations specialist for the science faculty at Boston University, and a Knight Science Journalism Fellow at the Massachusetts Institute of Technology. 

This article was reprinted from Volume 19, Issue 39, of CWWeekly, a leading clinical research industry newsletter providing expanded analysis on breaking news, study leads, trial results and more. Subscribe »