A Rocky Road Ahead for FDA and Industry

The coming year promises to embroil federal health agencies, providers, and biomedical research organizations in a range of contentious issues, as promising research on breakthrough therapies confronts policymakers looking to limit government spending on public health, R&D, and regulatory operations. Politics also will shape global health initiatives to combat antimicrobial resistance and to prepare for future pandemics, while drug coverage and costs remain a hot topic. FDA’s accelerated approval program will be in the spotlight, with manufacturers facing more pressure to withdraw from the market products that fail to confirm efficacy in timely postapproval studies. Drug shortages will also continue
to draw attention and proposed remedies, as rising infection rates send patients to hospitals and drive a surge in demand for treatments.

An anticipated end in 2023 to the public health emergency adopted two years ago to facilitate the approval of new treatments and preventives to COVID-19 will limit FDA emergency use authorizations (EUAs) that have helped speed promising treatments to patients. The change also may limit telehealth flexibilities and broader healthcare coverage for lower income patients.

At the same time, FDA faces tight deadlines for implementing a host of new programs and policies established by recently reauthorized user fee legislation for drugs and medical products. These include initiatives to modernize manufacturing processes and for testing innovative therapies and monitoring their effects. Looking ahead, 2023 promises to be a big year for cell and gene therapies, as seen in FDA’s formation of an expanded Office of Therapeutic Products to handle the surge in regenerative, genetic, and messenger RNA therapies.

Additionally, FDA and industry face a November 2023 deadline for full implementation of the Drug Supply Chain Security Act. This ten-year-old program requires all prescription drugs to be traceable from manufacturers to distributors and pharmacies, but it has been difficult to establish and has been subject to multiple delays.

These and other FDA initiatives require the agency to attract hundreds of new scientists and analysts able to evaluate innovative therapies, handle complex regulatory requirements, and monitor manufacturing operations needed to ensure quality standards. The likely retirement of dozens of senior staffers, moreover, will further challenge agency capabilities.

Politics in play

Notable changes in Congress promise to alter the tone and substance of the legislative debate and put pressure on federal funding of healthcare and biomedical research. New GOP committee chairs in the House are readying probes of Biden administration mismanagement of the pandemic and failure to fully assess the Chinese origins of COVID-19. Republican promises to reduce federal spending also threaten anticipated increases in budgets for FDA, the National Institutes of Health, and Medicare and Medicaid. Cost-cutting for Medicare includes an increase in the eligibility age for seniors, reduced payments to providers and hospitals, and further revisions in Medicare reimbursement and coverage of prescription drugs.

Although Democrats continue to control the Senate, there will be notable changes at the Senate Health, Education, Labor, and Pensions (HELP) Committee, as Sen. Bernie Sanders of Vermont is in line to become chair. Sanders has long championed universal health care and attacked high drug costs, and it remains to be seen where he can gain support from Republicans on the panel.

The battle over abortion drugs will continue to escalate, with pro-life organizations challenging FDA’s approval of mifepristone based on alleged inadequate safety testing. While the suit appears weak to legal experts, this attack strategy will consume considerable agency attention and resources. The fight is slated to go global, moreover, as some advocates work to increase access to abortion pills in other countries, while others seek curbs on abortion access.

Drug pricing contentious

Continued Democratic control of the Senate makes it unlikely that Congress will revise the provisions in the Inflation Reduction Act (IRA) that authorized Medicare price negotiations, curbs on certain drug price increases, and a price cap on insulin for Medicare beneficiaries. Democrats still want to extend the insulin cap and price hike limits to all patients, but that may be too costly to advance. Sky-high launch prices on new treatments, though, draw criticism, as seen in the $3.5 million list price for CSL’s new hemophilia B gene therapy (1). FDA leaders are exploring if less costly clinical testing strategies could reduce development costs for such bespoke therapies.

In the name of reducing outlays for medicines, some states will continue to push for FDA authorization of drug import plans, as permitted by policies adopted during the Trump administration. Several states have devised such Canadian drug import programs, but so far, FDA has not approved any due to continued concerns that the administrators cannot prevent distribution of unsafe or unregulated products.

Pharmacy benefit managers (PBMs) also will be in the spotlight, as Congressional leaders move to examine their role in drug access and pricing, issues already under investigation by the Federal Trade Commission (2). Both parties are reviewing the role PBMs play in determining patient outlays for drugs and in influencing Medicare drug coverage and costs.

While FDA avoids direct involvement in drug pricing issues, the agency will continue to support the development and marketing of generic drugs to promote market competition, particularly for products with complex formulations and dosage forms. Some members of Congress look to ensure that generic makers can access innovator products needed for testing and support efforts to advance the development of biosimilars by simplifying testing requirements for interchangeable therapies. The growth in follow-on treatments, though, promises to generate even more patent disputes, as will efforts to limit international patent protections strongly opposed by industry.

References

1. CBS News. FDA Approves Most Expensive Drug Ever, a $3.5 Million-per-dose Gene Therapy for Hemophilia B. CBSNews.com. Nov. 23, 2022.
2. FTC. FTC Launches Inquiry into Prescription Drug Middlemen Industry. Press Release. June 7, 2022.

About the author

Jill Wechsler is Washington reporter for Pharmaceutical Technology and BioPharm International.

Article details

BioPharm International
Vol. 36, No. 1
January 2023
Pages: 8–9

Citation

When referring to this article, please cite it as J. Wechsler. A Rocky Road Ahead for FDA and Industry. BioPharm International 2023 36 (1).