Congress has taken the necessary step of including critical reauthorizations for the Prescription Drug User Fee Act in its continuing resolution that will avert a partial government shutdown. Successful reauthorization of the act, which sets the funding structure for the Food and Drug Administration, will ensure that it has the funds to fulfill its vital mission.
But by passing a “clean” reauthorization of PDUFA, Congress is missing a rare opportunity to improve public health policies that could greatly benefit people with cancer and others by expanding access to cutting-edge therapies through clinical trials and improving the FDA’s existing Accelerated Approval Program to ensure that drugs meet certain safety and efficacy standards.
During the previous several months, members of the Senate’s Committee on Health, Education, Labor and Pensions and the House Committee on Energy and Commerce, in consultation with leadership in both chambers, had been negotiating a number of policy “riders” that would attach new policies to the “must-pass” PDUFA that every five years puts in place a structure for the fees drug and device makers must pay to the FDA to review applications for marketing approval.
The fees make up a significant share of the revenue that enables the FDA to function on behalf of the public; the legislation must get congressional approval so that the FDA does not run out of funding to pay its staff.
During at least the last three rounds for this PDUFA reauthorization, important bipartisan policies have been included that pertain to expedited review of products designed for rare diseases such as recalcitrant and pediatric cancers, and to improve patient-focused drug development. While some provisions in earlier PDUFA versions remain controversial, several proposals enjoyed broad support. Ultimately agreeing to drop even these common-sense policies from the user fee reauthorization is a major swing and miss for Congress that ultimately hinders regulatory policy advancements and passes on a prime opportunity to better serve people with serious illnesses such as brain tumors.
To see a key example of how this failure to act has real-world consequences, look no further than the recent high-profile controversy over Aduhelm, an experimental Alzheimer’s drug. Aduhelm was approved through the FDA’s Accelerated Approval Program, which uses surrogate measures of effectiveness to predict the ultimate clinical benefit of a drug to patients. In this case, a decline in the level of amyloid, a protein associated with Alzheimer’s, was used as a stand-in for a decline in cognitive impairment. Calling the drug’s efficacy into question, as well as the use of surrogate endpoints, critics pounced on the opportunity to point out flaws in the current accelerated approval process and regulations.
In response to concerns about rigor of this approval process, the House Energy and Commerce Committee bill, sponsored by Rep. Anna Eshoo (D-Calif.), called for policy that would make simple yet effective changes to strengthen regulations relating to confirmatory clinical trials. Such a provision would ensure that patients still get early access to the most cutting-edge therapies being developed, while ultimately holding biopharmaceutical companies accountable for completing studies that confirm whether a drug delivers ultimate clinical benefit to the intended patient population. It also importantly codifies the FDA’s authority to withdraw approval if a drug sponsor fails to complete the confirmatory study, or the drug is found to be unsafe or ineffective. The House bill also tasks the FDA with developing more guidance on the types of endpoints (measures of a drug’s benefit to patients) that can be used in clinical trials.
By failing to address this critical issue, Congress has left the door open for ongoing controversies related to accelerated approvals, pitting patients, biopharmaceutical companies, research scientists, clinicians, payers, and public health advocates against each other in a circular firing squad.
During the Covid-19 pandemic, the FDA shined in creating guidance that gave more flexibility to clinical trial sponsors to adjust for pandemic-related safety protocols. In some cases, this included allowing certain trial procedures to take place at partnering local medical centers or via telehealth and other virtual interactions. Doing so allowed trials to keep operating during the height of the pandemic. It also showed how such hybrid or decentralized clinical trials could reduce barriers to patient participation such as distance, travel time, and cost of transportation.
Congress had the chance to increase access and boost enrollment in these studies by preserving a provision in the final bill that called for the FDA to develop more guidance on the use of decentralized clinical trial designs. Congress must now act on separate measures to address this policy not only to advance research but to give people easier access to cutting-edge treatments in their own communities.
It is hard to pass legislation in Congress these days, especially legislation with support from both parties. Voting for a “clean” user fee reauthorization package averts disaster but wastes an easy chance to enact valuable patient-centered policies and regulations that would greatly improve research and reduce barriers for access to medical innovations.
Great policy forged through intense deliberations with multiple stakeholders across the health care ecosystem was ready to be put in place. And the imperative to pass user fee reauthorization was a once-in-five-years chance to easily do so. The question now is, “If not now, then when?” Congress still has the opportunity to address these and other no-brainer pieces of legislation that put patients first, and must find the will to do so.
David F. Arons is the president and chief executive officer of the National Brain Tumor Society. He was part of the blue-ribbon panel of advisors on the National Cancer Moonshot, led by then-Vice President Biden.