Under President Joe Biden, pharmaceutical regulation may see increased FDA guidance, new strategies to speed up innovation and regulatory review, and a renewed focus on diseases with unmet needs, among other expectations.
With President Joe Biden sworn in as the 46th US president, attention has turned to what actions he will take in his first 100 days in office, and beyond. While much of the president’s focus will initially be on the COVID-19 pandemic, increasing vaccination numbers, and economic relief, the pharmaceutical and biotechnology industries, which have been at the forefront of the executive branch’s attention since the start of COVID-19, will likely continue to garner significant policy focus. Specifically, over the next four years, we expect to see more Food and Drug Administration (FDA) independence, transparency, and funding; the application of new strategies to facilitate pharmaceutical product development; increasing international cooperation; efforts to facilitate more domestic manufacturing; an increase in enforcement; a refocusing of efforts on diseases with unmet medical needs; and increased FDA guidance.
RENEWED FOCUS ON SCIENCE AS PRIMARY DRIVER OF FDA ACTION
COVID-19 intensified presidential and public focus on FDA’s internal processes, with publicly voiced disagreements among FDA, the US Department of Health and Human Services (HHS), and President Donald Trump. While this spotlight is a natural consequence of the pandemic’s national impact, the Biden administration has confirmed that the “administration will always listen to science.” With the selection of Dr. Janet Woodcock as acting FDA commissioner, the administration is signaling its confidence in the agency’s historic focus on science and rigorous product review. Dr. Woodcock’s knowledge of FDA’s processes and the pressure points for drug research and development, developed over her several decades at FDA, also allows the de-risking of decisions by an experienced and well-respected regulator. It may also mean that we may see a renewed focus on regulatory transparency, especially with respect to FDA decisionmaking.
Finally, with an increased focus on the scientific expertise within FDA through the seventh iteration of the Prescription Drug User Fee Act (PDUFA), which will cover fiscal years 2023-2027, FDA may gain personnel funding, especially within the Center for Biologics Evaluation and Research, the center responsible for the review of pandemic vaccines, as well as new medical technologies, such as cell and gene therapies.
CONTINUATION AND DEVELOPMENT OF REGULATORY STRATEGIES TO SPEED INNOVATION AND REGULATORY REVIEW
As a direct result of FDA’s COVID-19 experience and success in substantially speeding up clinical trials and application review, FDA will likely continue current strategies and/or implement new strategies to facilitate and expedite the development, review, and approval of medical products. Through the COVID-19 pandemic, FDA developed the Coronavirus Treatment Acceleration Program, a process to ensure that COVID-19 product sponsors are able to quickly and efficiently discuss development plans with the agency, and pushed out industry guidance at record speed.
FDA became more comfortable with the use of remote clinical trial procedures and other innovative designs, while also looking for ways to further utilize real-world data. By example, during FDA’s September 10, 2020 Grand Rounds session, Dr. Amy Abernathy, the FDA principal deputy commissioner and acting chief information officer, described how the agency is collaborating with stakeholders and other government agencies on the COVID-19 Evidence Accelerator (Accelerator). Through the Accelerator, FDA developed research questions relevant to its COVID-19 regulatory review, created structured data elements for real-world data and evidence (RWD/RWE), and developed standardized RWD/RWE protocols and analytic methods to answer questions regarding the pandemic. As Dr. Abernathy discussed, this work demonstrated the potential of RWD/RWE in the selection of drugs for repurposing; clinical trial planning, prioritization, and design; safety evaluations; and, with further development, the assessment of treatment effects and use as comparator and synthetic control arms. We expect FDA to continue to work with stakeholders to help build efficiencies into the clinical trial and product development process.
INCREASED GLOBAL REGULATORY HARMONIZATION
With the new administration, we are also likely to see greater global regulatory harmonization, in large part as a result of COVID-19. As a result of travel restrictions, FDA has, in part, relied on international counterparts for manufacturing inspections, including through the Mutual Recognition Agreement program. This is a trend that will likely continue, as it frees up agency resources to focus on higher risk areas and other regulatory priorities.
Additionally, we are likely to see greater international cooperation and collaboration around product review. The United States already rejoined the World Health Organization (WHO), with Dr. Anthony Fauci leading a US delegation at the WHO’s annual meeting on January 21, 2021. The Biden administration also intends to “re-launch and strengthen” the US Agency for International Development’s PREDICT program, which was initiated in 2009 to detect and discover potential pandemic zoonotic viruses, and will join COVAX, one portion of the WHO’s Access to COVID-19 Tools Accelerator, which aims to facilitate development of and equitable access to COVID-19 vaccines.
The United States is further likely to work with international regulators to standardize regulatory priorities and undertake parallel reviews of products for rare and life-threatening diseases.
CONTINUED EMPHASIS ON DEVELOPING DOMESTIC PHARMACEUTICAL INFRASTRUCTURE
In parallel, we are likely to also see a continuation of US efforts to shore up the domestic pharmaceutical supply chain. Under the Coronavirus Aid, Relief, and Economic Security (CARES) Act, the National Academies of Sciences (NAS) will be assessing US medical supply chain security, including the United States’ reliance on foreign suppliers for critical drugs. NAS will also be making recommendations regarding promotion of domestic manufacturing.
Any FDA efforts, however, to enhance domestic manufacturing of drugs and biologics will likely require a coordinated review of the inspection process for active pharmaceutical ingredients, excipients, and finished formulations.
POTENTIAL INCREASE IN ENFORCEMENT ACTIONS
Once the United States is able to move beyond pandemic response, and more standard FDA inspection and monitoring activities ramp up, we expect to see an increase in FDA enforcement actions. By example, FDA is likely to focus on current Good Manufacturing Practice (cGMP) and data integrity issues that may have developed as a result of pandemic challenges. However, how much latitude and time FDA will provide manufacturers to realign processes post-COVID-19 remains unclear.
With respect to pharmaceutical promotion, FDA is likely to continue its more restrained approach as a result of historic First Amendment challenges. However, FDA is likely to continue to focus on social media promotion and increase enforcement where promotional practices adversely impact drug prices and healthcare costs. Whether there will be focus on the promotion of COVID-19 vaccines once there are multiple vaccine alternatives remains to be seen.
RENEWED FOCUS ON DISEASES AND CONDITIONS WITH UNMET NEEDS, ESPECIALLY IN ONCOLOGY
After its initial focus on pandemic response, FDA will likely refocus on diseases and conditions with unmet needs, including in oncology, as well as on personalized therapeutics. At the end of President Barack Obama’s second term, Congress passed the 21st Century Cures Act (Cures Act). This law incorporated many programs to address serious diseases or conditions with inadequate treatment options, including substantial research funding allocations, the Regenerative Medicine Advanced Therapy Designation, the Precision Medicine Initiative, and a program for targeted or variant protein drugs. President Biden also previously headed up the Cancer Moonshot program, a national effort to end cancer. Over the next four years, we are likely to see renewed attention to these areas, especially in the upcoming PDUFA legislation.
INCREASE IN GUIDANCE
Finally, over the last year, we have seen a significant uptick in FDA’s issuance of guidance documents. Specifically, over the course of 11 months, FDA issued more than 35 immediately effective drug and biologic guidances related to COVID-19. As FDA heads into the new administration, we would expect that the agency will continue its trend of swiftly issuing guidance to industry, related to both COVID-19 and other areas, such cell, gene, and other personalized therapies, an area of significant FDA focus.
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