“In order to take advantage of today’s advancements in science, drug development, and patient treatment, the U.S. Food and Drug Administration’s (FDA’s) structure needs reorganization to focus its resources and ensure the best outcomes for patients. Friends of Cancer Research (Friends) proposes enhancing coordination at the FDA based on specific disease areas, rather than approving treatments on a cumbersome product-by-product basis, to reflect 21st century science and modern medical care.”
This Friends statement was the basis of an hour-long panel discussion moderated by Michael McCaughan, Founding Member, Prevision Policy, Washington, DC.1
The five panelists agreed that the FDA does a good job, despite a severe shortage of funds and personnel, but its efficiency could be greatly improved. They also agreed that exactly how to go about doing this is a conundrum.
Congress has not updated the FDA’s organizational structure for medical products since the 1970s, and the existing regulatory framework has been defined by some as a “divide-and-conquer” approach to oversight: separate centers to regulate three major categories of products: drugs, biologics, and devices.
Moonshot and FDA
An idea that has been around for some time is to establish four Centers of Excellence at the FDA (oncology, neurodegenerative, cardiovascular, and infectious disease)—that is, switching to a more disease-oriented approach to product development and regulation.
The Centers of Excellence idea received a boost from President Obama when he announced the White House Cancer Moonshot Task Force in his State of the Union address this year. Vice President Biden called it an effort to “end cancer as we know it.” He set an ambitious goal of “making a decade’s worth of advances in 5 years—a quantum leap on the path to a cure.” The President also slated an additional $75 million for creation of a virtual Oncology Center of Excellence at the FDA.
Although the Moonshot Task Force will be advisory only—and it’s not yet clear what the “virtual” part entails—its main function will be, according to the White House, “to leverage the combined skills of regulatory scientists and reviewers with expertise in drugs, biologics, and devices. This center would expedite the development of novel combination products and support an integrated approach.” Specific areas will include evaluating products; supporting development of companion diagnostic tests; using combination drugs, biologics, and devices to treat cancer; and developing and promoting methods created through precision medicine.
FDA does a good job, but it could do a better one. So it is up to us to think about what the next steps for the agency could be—and we have to do it right now.
— Ellen V. Sigal, PhD
It also will encourage coordination of federal efforts and make recommendations about how to expand clinical trials, improve data collection, remove unnecessary regulations, and develop public-private partnerships, all bolstered by the $75 million—a good beginning but not nearly enough, said Ellen V. Sigal, PhD, Friends Chair and Founder.
FDA Must Keep Up
Steve Galson, MD, MPH, Senior Vice President, Global Regulatory Affairs and Safety, Amgen, Inc—and former Director of the FDA Center for Drug Evaluation and Research—said, “New types of genetic and diagnostic tests, along with new drugs and the devices required to deliver them, no longer fit into FDA’s organizational structure with its fixed scientific and regulatory boundaries. Increasingly complex legislation, as well as the authority that the agency requires, make it mandatory that FDA reassess the way it does business. Things are dramatically different than they were even a decade ago.”
He spoke of the compelling need for change. “All Amgen products are a combination of drugs and biologics, and all require companion diagnostics and devices. Their development and manufacture mean that our many scientists work together to produce these highly complex products. FDA needs to mirror this ability.”
He continued, “The agency currently regulates cellular and immunologic therapies, devices, and biomarker tests in separate areas. Now is the time to bring them under one roof to reflect the way these products are used in medical practice.”
[The FDA] must create a more collaborative system to expedite discovery and approval, especially in biosimilars, immunotherapies, and combination products.
— Mark McClellan, MD, PhD
Mark McClellan, MD, PhD, Director, Duke-Robert J. Margolis, MD, Center for Health Policy at Duke University, and former Commissioner of both the FDA and Centers for Medicare & Medicaid Services, reminded the audience and other panelists that the FDA suffers from extremely limited resources, but despite that, it is time to increase coordination across all its divisions and to streamline the agency. “At the same time, it must create a more collaborative system to expedite discovery and approval, especially in biosimilars, immunotherapies, and combination products.”
Otis Brawley, MD, Chief Medical Officer and Vice President, American Cancer Society, added that cancer science is now so advanced that it is no longer acceptable to study it in terms of location or anatomic structure. “Rather, we have to ask whether the tumor expresses a certain molecular marker and whether the drug intended for the marker actually hits it.”
The definition of cancer has changed over the past 20 years with the advent of genomics; therefore, the FDA has to keep up with the science and with drug development, and it has to make its approval and regulatory processes more comprehensive and collaborative, he added.
George D. Demetri, MD, Director, Ludwig Center at Harvard Medical School, agreed. “Cancer is thousands of different diseases and thus requires comprehensive and coordinated research.” He said that although Harvard Medical School does not have its own hospital, it is affiliated with a half-dozen or more in the Boston area. “Each treats cancer patients, and we have developed a consortium of cancer centers that operates cooperatively. Harvard, of course, is known for its fiefdoms and competitive atmosphere, but in this area we have managed to work together.”
Dr. Sigal reminded those in attendance that all cancer research and development involves benefit and risk—and it is always about the patient. “FDA does a good job,” she said. “But it could do a better one, so it is up to us to think about what the next steps for the agency could be—and we have to do it right now.”
She added, “Yes, of course, it needs better funding. We have always wanted a cancer center at FDA because today’s treatment options have moved far beyond the ‘silo’ approach that FDA still uses. It has, however, become increasingly obvious that this will not be achieved without impetus from outside the agency.”