Senate HELP chair proposes reforms to FDA’s regulatory framework

The top lawmaker on the Senate Health, Education, Labor, and Pensions (HELP) Committee published a report this week on reforming the US Food and Drug Administration (FDA). The report argues the changes would cut red tape, make the US more attractive to clinical research, and accelerate the approval of medical products.

On Tuesday, HELP Committee Chair Bill Cassidy (R-LA) published a report outlining four broad proposed reforms to FDA.

“Biomedical innovation holds enormous promise for the American people... But ‘innovation’ is meaningless unless it creates products that actually help patients,” said Cassidy in a statement. “While many parts of FDA work well, unnecessary bottlenecks slow patients and consumers getting the products they need.

“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,” he added. “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.”

On top of the list, the report called for changes to FDA's premarket application process and evidentiary requirements to increase consistency and predictability during reviews.

The report noted that significant differences in product reviews leave sponsors feeling they are subject to a "reviewer lottery." It also highlighted that Congress has mandated that the agency take a "least burdensome" approach to regulating medical devices, which may set the tone for other products.

"To improve predictability, Congress and FDA should consider how to apply this principle to other product categories while maintaining patient safety and evidentiary standards," the report stated. "Additionally, as Congress considers reforms, proposals should ensure there is accountability, transparency, and flexibility in the review process to meet the agency’s resources."

In reforming the premarket application process and evidentiary requirements, the report also highlighted the need for more novel clinical trial approaches and alternative evidentiary models. It warned that companies are increasingly reporting they are starting their clinical trials outside the US, including in "adversarial nations like China," and seeks to reverse that trend. It emphasized the use of real-world evidence (RWE) and digital health technologies in clinical trials that can help decentralize trials.

"FDA must appropriately tailor its review of digital health technologies to the relevant risks," said the report. "In some cases, FDA expects products like monitors and sensors to meet medical device-like standards, despite the fact that most do not serve the function of a medical device.

"Even when FDA has approved or cleared certain remote patient monitors as medical devices, FDA resists their use for data collection in their trials," the report added. "FDA requires re-validation of these tools from study to study, which creates unnecessary duplication. Agencies should align on risk-based validation requirements for use across studies."

The report also asked FDA to assess its approach to artificial intelligence (AI). More specifically, it stated that the agency should take a risk-based approach to regulating AI products without stifling innovation and be clear about its expectations to sponsors.

The second broad area of reform detailed in the report is for FDA to take steps to help the development of cutting-edge and rare disease therapies. The report noted that there have been significant advances in rare disease research, such as cell and gene therapies, but also that the agency has been increasingly using clinical holds that are slowing such innovations from reaching the market.

"Clinical holds are highly disruptive for innovators, clinicians, and patients, often derailing complex and costly trials," the report stated. "Sponsors report opaque and procedurally rigid interactions with FDA, including late-stage outreach urging [investigational new drug (IND)] withdrawal on day 29 of the 30-day review period with little explanation, holds imposed using boilerplate regulatory citations without explanations that are specific to the trial in question, and feedback on trial design that arrives long after trials are completed.

"When issuing clinical holds, FDA should judiciously pursue a more transparent and constructive dialogue with sponsors in order to more expeditiously address safety concerns, without major delays to patient access to new treatments," the report added.

The report also argued that FDA's current approval framework is ill-suited for personalized medicine and rare diseases and needs reform. It said that it is encouraged by the potential for the agency's Rare Disease Innovation Hub to improve coordination across the agency to help get rare disease treatments to market.

The third broad area of reform listed by the report is to update FDA's regulatory framework to increase competition and innovation. It stated that the current regulatory process for biologics is limiting and needs to align better with the science. More specifically, it recommended creating a new pathway that is between biologics license applications (BLA) and supplemental biologics license applications (sBLA), like the 505(b)(2) pathway used by drug sponsors.

"This pathway permits sponsors, where appropriate, to rely on FDA’s previous findings of safety and efficacy for other drugs and published literature, allowing new products, or products with new features, to launch without having to start from scratch," said the report. "Extending a similar intermediate pathway to biologics would allow appropriate biosimilars and modified biologics to partially rely on FDA’s existing findings, streamlining review while preserving standards for safety and efficacy."

The report also stated that the premarket pathway for biosimilars is too burdensome, requiring sponsors to conduct comparative clinical efficacy studies that have no equivalent for generics on the drug side. It recommended that Congress consider additional legislation to update the pathway. Additionally, it said Congress should consider new legislation to protect and strengthen generic drug competition.

Finally, the report also broadly recommended updating FDA's framework for software, medical device review, and innovation. It stated that medical device sponsors still struggle with delays, inconsistent review practices, and limited opportunities for real-time engagement with agency reviewers.

"As FDA continues implementing new device authorities granted by Congress, the agency could strengthen coordination with key agencies, like the Office of the National Coordinator for Health Information Technology (ONC), and collaborate with manufacturers to ensure compliance with evolving requirements," said the report. "Product developers are also eager to see FDA fully embrace Congress’ recent directive to leverage predetermined change control plans (PCCPs), which allow developers to make certain postmarket improvements to their devices with minimal disruption."

The report recommended that Congress consider ways to improve the current medical device review pathways for software medical products, and FDA consider its current risk framework for AI applications. It also asked the agency to update its thinking on clinical decision support (CDS) software.

The report noted that Congress explicitly carved out many CDS tools from being categorized as medical devices in the 21st Century Cures Act, and yet the agency published a guidance in 2022 that broadly applied its regulatory authority over such applications that conflicts with the statutory definition.

"Stakeholders who develop and use CDS software have raised concerns with this guidance as they have historically had no regulatory interactions with FDA, including product review and required payment of user fees," said the report. "Requiring FDA review of these tools will likely stifle and slow advancements in digital health.

"While FDA recently published revised guidance this year, it is important that FDA continue to exercise its authority within the bounds of what Congress directed in the 21st Century Cures Act," the report added.

Report

Senate HELP chair proposes reforms to FDA’s regulatory framework

Related topics

Recommended content

Welcome to the new RAPS Digital Experience