Ten months after biotech leaders reached out to Sen. Bill Cassidy, M.D., with concerns about the future of the FDA, the Senate Health, Education, Labor and Pensions (HELP) Committee chairman has had a chance to review the situation and share his vision for the future of the agency.
A new report (PDF), which Cassidy says is backed by input from a collection of stakeholders, offers proposed changes to the FDA review process and touches on the agency's rare disease approach, its use of AI, biosimilar regulations and other topics.
“Ultimately, it should be easier to make Americans healthy by empowering them with the tools and information they need to make healthy choices and live better lives,” Cassidy wrote in the 18-page report, noting that the HELP committee plans to spearhead a “collaborative, bipartisan process” for FDA reforms.
In his report, Cassidy highlights the FDA’s stance on clinical trials and alternative evidence models, pointing out that the agency “could do more” to expand the use of novel study designs and embrace technologies that can collect “better data” from studies, all while allowing more patients to join trials and cutting costs.
Currently, regulatory burdens for early-phase trials can drive drug sponsors out of the U.S., Cassidy said, citing China's recent R&D ascent.
The FDA should also take more responsibility in rare disease drug development, as the field “advances at unprecedented rates,” Cassidy noted, pointing to the 48 cell and gene therapies that have been approved through December 2025. In this area, the agency’s “increasing use of clinical holds” has “raised concern” as they can derail costly clinical trials, he said. The senator recommended that the FDA “judiciously pursue” transparent and constructive dialogue with drugmakers in order to avoid delays in getting new innovative drugs to patients.
He also suggested that the FDA’s accelerated approval pathway be utilized beyond its current focus in oncology, as more than 80% of accelerated approvals issued between 2010 and 2021 were for drugs that can treat cancers, Cassidy said.
Additionally, certain rare disease drugs could benefit from a modernized regulatory approach, as the current framework is “often ill-suited for personalized medicines,” according to the report. High costs under the existing regulatory requirements for these drugs are often paid by smaller companies, which are responsible for about 70% of rare disease therapies in late-stage trials, according to Cassidy.
Cassidy also uses the report to discuss biosimilars, a topic that health officials have already homed in on with a draft guidance document from the FDA. The guidance looks to improve access to biosims by reducing clinical testing burdens and labeling all approved biosimilars as “interchangeable,” which allows pharmacists to freely swap from branded reference meds to copycat biologics.
Although “questions remain regarding the position the current administration will ultimately adopt” on interchangeable biosimilars, Cassidy views FDA Commissioner Marty Makary's stance on the topic as a “notable signal that the FDA may be open to further reform in this area,” he wrote. However, the senator urges Congress to consider “whether additional legislative action is warranted to codify FDA’s current thinking regarding the types of studies needed to demonstrate biosimilarity and interchangeability.”
Congress could also consider reforms around generic drugs, as “certain discrete problems” could stand to delay additional generic drug competition, Cassidy noted.
According to Cassidy, potential FDA reforms coming from Congress should center on ensuring “accountability, transparency, and flexibility” in the drug review process. The senator also used his report to suggest greater consistency around regulating the use of AI in healthcare.
“While FDA has made progress, there remains enormous potential for the agency to support better and more affordable products for patients,” Cassidy concluded. “The HELP Committee looks forward to coordinating with FDA under its new leadership to promote patient access, accelerate U.S. competitiveness, and improve the health of American families.”
In his paper, Cassidy mentioned President Donald Trump’s mandates to “cut red tape and make America healthy again” as sticking points that challenge the FDA, Congress and other stakeholders to “work together to examine the extent to which FDA’s regulatory framework works efficiently,” he wrote.
Since the Trump administration settled in, the FDA has seen numerous shakeups, including mass layoffs through a wide-spanning restructuring that sent many agency leaders out the door. Cassidy and the HELP committee were called to task on this issue last year in a letter supported by over 200 biotech figures that voiced concern over “the current state of the agency and its future.”
Elsewhere, Cassidy has recently been outspoken about his qualms on the changing U.S. vaccine landscape. The senator, a Republican from Louisiana, voted to advance the nomination of “Make America Healthy Again” champion Robert F. Kennedy Jr. as the Secretary of the Department of Health and Human Services (HHS) last year after voicing his concern over Kennedy’s position on vaccines. Since then, the secretary has ushered in a volatile vaccine environment by dismantling the prior slate of the CDC’s Advisory Committee on Immunization Practices, among other controversial moves.
https://www.fiercepharma.com/pharma/help-committees-bill-cassidy-proposes-paths-widespread-fda-reform-new-report
