Just a few weeks ago, it looked like FDA would finally gain a permanent commissioner after a year of waiting, and that industry agreement on revised drug user fees would begin a steady march through Congress to timely approval this year. Now a range of contentious political issues have jeopardized these and other initiatives vital to the agency’s effectiveness and stature, both at home and on the important global stage. Democrats and Republicans are questioning the capabilities and credibility of Robert Califf as a leader for the agency, despite his valuable experience in biomedical R&D and as the head of FDA several years ago. Moreover, the legislators appear willing to delay action on new user fee agreements to air a broad range of grievances about agency decisions and policies.
The House Energy & Commerce Health subcommittee held hearing yesterday to officially kick off the debate and review process for renewing new user fee programs to support FDA regulation of drugs (PDUFA VII), generic drugs (GDUFA III), and biosimilars (BsUFA III).1 The session provided a forum for FDA officials and industry leaders to outline program updates and to emphasize the importance of renewing the fee programs by Sept. 30.2 The legislators unveiled both their continued support for the program, as well as concerns and complaints about FDA’s performance and priorities.
The agreed-on fees are significant, even by federal spending standards: $1.4 billion a year for prescription drugs, $600 million to speed approvals of generics, and about $50 million to advance biosimilars. The considerable fee amounts generate concerns about industry influence on the agency, along with praise for enabling cutting-edge advances and efficient operations by FDA’s staff. Notably absent was any discussion of fees for medical devices, due to continued disagreement over program parameters by manufacturers and FDA’s Center for Devices and Radiological Health.
The range of complaints and concerns with FDA decisions and actions raised by the legislators signals the shape of the debate to come. The agency’s accelerated approval (AA) process is at the top of the list, as seen in both vocal challenges to FDA’s approval of the Alzheimer’s treatment Aduhelm, along with complaints that Medicare patients will be denied access to the drug due to the proposed coverage-with-evidence-development policy under consideration by the Centers for Medicare and Medicaid Services (CMS). In response to some heated questions, Center for Drug Evaluation and Research (CDER) Director Patrizia Cavazzoni emphasized that FDA did not consult with CMS on its controversial coverage proposal, while acknowledging the need for improvements in the AA program, notably in requiring sponsors to begin confirmatory studies at time of approval and in facilitating FDA’s authority to pull products that fail to provide timely follow-on evidence.
The legislators also voiced concerns about limited development of new antimicrobials and treatments for rare conditions and on the lack of diversity in clinical trials. Peter Marks, director of the Center for Biologics Evaluation and Research (CBER), outlined efforts to address drug development policies for future pandemics and the need for reliable real-world evidence to support new products. Several legislators raised questions about how FDA will transition to “normal” procedures and practices for field inspections and remote meetings when the public health emergency is declared over.
Meanwhile, prospects have dimmed for quick Senate confirmation of Robert Califf as FDA commissioner. His strong record as an expert in clinical research and FDA policies, as seen in his previous stint as commissioner during the last year of the Obama administration, was supposed to speed his nomination through the Senate process. But lingering concerns about his ties to the pharmaceutical industry have generated opposition, as have charge that he failed to tackle FDA’s involvement in the approval of lethal opioids. Califf recently moved to offset “revolving-door” charges by pledging to recuse himself for four years after his time as FDA commissioner—instead of the required two years—from industry involvement. That won over some Democrats, but not all.
At the same time, Republican support has fallen off with an attack by right-to-life groups on FDA’s “pro-abortion agenda.” A main target is the agency’s recent decision to remove curbs on prescribing and dispensing medications that induce abortion at early stages of pregnancy. In response to heated questions, Califf said he would review that policy, but not necessarily revoke it, prompting most Republicans to withhold support for the candidate. Even the editors of The Wall Street Journal objected to such criticisms of Califf from the pro-life movement, noting that FDA is not an abortion regulator and that Califf doesn’t deserve such abuse from the left or the right.3
Califf’s most vocal supporter is HELP Committee ranking Republican Sen. Richard Burr of North Carolina. Burr championed the appointment of his fellow Carolinian as a knowledgeable scientist, experienced policymaker, and greatly needed leader for FDA, attributes also praised by former FDA commissioners and leaders of the biomedical research community.
Evidently, the White House expected Burr to bring in sufficient Republican support to move Califf’s confirmation through the Senate. But with much of the opposition coming from Democrats, the Biden administration has had to put more pressure on waffling legislators or risk leaving FDA in limbo once again. Acting Commissioner Janet Woodcock can remain in her post for much of the year if all else fails, but that temporary status is not fair to her—or to FDA.