Pharma Faces Sweeping Legislative Changes

The bills aim to speed the discovery of new cures, improve patient representation in clinical trials, and enhance the FDA's ability to ensure safety, efficacy, and quality of the drug supply. 

The subcommittee on health of the Energy and Commerce (E&C) recently held a hearing on a string of legislation that brings about sweeping changes throughout the pharmaceutical industry.

The 22 proposed bills tackle different areas in healthcare, particularly in pharma, that need major legislative change. The mostly bipartisan bills aim to speed the discovery of new cures, improve patient representation in clinical trials and enhance the Food and Drug Administration's (FDA) ability to fulfill its mission of ensuring the safety, efficacy, and quality of the drug supply.

Industry trade associations BIO, PhRMA, and the Association for Accessible Medicines (AAM) each provided testimony during the March 17 hearing in support of certain legislative changes.

A new high-stakes research agency focused on biotech was also discussed during the hearing.

"This hearing is an enormous legislative undertaking," said E&C health subcommittee chairwoman Anna Eshoo (D-CA), whose bill was the first discussed. The Advanced Research Projects Agency for Health Act would establish a new independent agency, dubbed ARPA-H, to be housed under the National Institutes of Health (NIH) with a 2022 budget of $6.5 billion.

ARPA-H

President Biden first proposed ARPA-H in 2021 as a biomedical version of the U.S. military's Defense Advanced Research Projects Agency (DARPA), known for backing innovations like the internet. The new agency would have the authority to approve and terminate project funding, establish milestones, as well as coordinate with other health agencies, including the NIH.

It "will embody the nimble spirit of the highly regarded and successful defense advanced research project agency" to "pursue large-scale high-risk projects," Eshoo said. "It'll break the mold for federal research agencies by being uniquely focused on solving the valley of death to delivery transformational cures." The agency will be charged with closing the existing gap between NIH research and the development of the private sector's commercial products.

ARPA-H's mission will drive scientific breakthroughs to improve the country's health and help meet the president's goal of ending cancer, as well. The president signed into law the bipartisan Consolidated Appropriations Act of 2022 recently, providing $1 billion to establish ARPA-H.

"This is a momentous first step in creating an agency that will be a beacon of hope for the American people," Eshoo added. "But our work isn't done yet. Our committee needs to pass the ARPA-H legislation to provide the agency with the full authorities it needs to be successful from day one, including ensuring that it will be a nimble, dynamic, and independent agency."

The goals place emphasis on delivering "high-need cures." The term refers to a drug, biological product, or device that should be prioritized to detect, diagnose, mitigate, prevent, or treat any disease or medical condition for which incentives in the commercial market are unlikely to result in the adequate or timely development of such product. Goals include revolutionizing diagnosis, mitigation, prevention, and treatment of diseases via the development of health technologies and high-need cures; and promoting high-risk, high-reward innovation to develop high-need cures.

PDUFA and BsUFA

There was consensus among the trade groups around the need for the timely reauthorization of both the Prescription Drug User Fee Act (PDUFA) and the Biosimilar User Fee Act (BsUFA).

The latest reauthorization of PDUFA (PDUFA VI) was signed into law in August 2017 as part of the FDA Reauthorization Act of 2017 (FDARA) and took effect in October 2017. The enactment of FDARA also renewed BsUFA for a second time. These authorize FDA to collect user fees to support the review processes of prescription drugs and biosimilar biological products. But the current legislative authority for PDUFA VI and BsUFA II is set to expire September 30, 2022.

"Both the PDUFA VII and BsUFA III agreements were carefully considered by the biopharmaceutical industry and negotiated with FDA to ensure that the agency is equipped with the necessary resources to help us deliver new treatments and cures to meet patients' unmet medical needs," said Lucy Vereshchagina, vice president of science and regulatory advocacy at PhRMA. The agreements also ensure that the agency is "able to keep pace with ever-evolving science and have the necessary resources to ensure a timely review of innovative drugs and biologics, as well as biosimilar and interchangeable biosimilar products."

Cartier Esham, chief scientific officer at BIO, said the group "strongly supports timely enactment of the PDUFA VII and BsUFA III commitment letters." The resources these provide "will serve to maintain FDA's global leadership and enable the agency to keep pace with the medical and scientific advances of today and tomorrow," Esham added.

The industry user fee reauthorization process kicked off in Congress earlier this year. Top FDA officials Patrizia Cavazzoni, director of the Center for Drug Evaluation and Research (CDER), and Peter Marks, director of the Center for Biologics Evaluation and Research (CBER) testified to the success of the agency's current user fee programs and on the enhancements the FDA seeks.

Other legislation of interest to BIO included those related to building a new clinical development paradigm that is more inclusive, more patient-centric, and more informative about clinical outcomes; the FDA's accelerated approval process; continuing the momentum with pediatric drug development; and advanced manufacturing technologies to keep pace with product innovation.

AAM, meanwhile, focused its advocacy efforts on other areas. These include ensuring access to affordable medicines; addressing the current backlog of FDA manufacturing facility inspections; enhancing domestic manufacturing; promoting innovation in drug manufacturing; among others.