As Prescribed

On June 2, 2025, FDA announced the launch of Elsa, a generative AI tool designed to “help employees—from scientific reviewers to investigators—work more efficiently.” Per FDA, the tool “modernizes agency functions and leverages AI capabilities to better serve the American people.” While Elsa may add efficiencies to FDA’s review processes, it also raises a number of questions for regulated industry.

While the US Food and Drug Administration has been experiencing recent reorganization and cuts in personnel, the United Kingdom has its own upcoming policy changes making it easier and faster to initiate and maintain UK clinical trials, which will create attractive new options and strategy decisions for pharma research and development companies.

Drug approval is unequivocally the linchpin to any drug development effort and, given the role of the US market in the global marketplace, the drug approval standard employed by FDA remains a perennial focus of stakeholders across the industry. In light of recent remarks from the FDA commissioner on the development of a new conditional approval pathway, we revisit FDA’s existing approval standard and the broader regulatory and policy context that has, to date, underpinned FDA’s drug approval practices and anchored the agency as the worldwide “gold standard.”

As we discussed in our blog post last week, 2024 saw the Department of Health and Human Services Office of Inspector General (HHS OIG) provide more concrete regulatory guidance for programs sponsored by pharmaceutical manufacturers that provide financial assistance and free genetic testing and counseling services to patients suffering from rare diseases. While the favorable treatment from HHS OIG is a positive development for stakeholders and patients, the Advisory Opinions should not be interpreted as permission slips for stakeholders to push the limits of facilitating testing, assistance, or treatment of federal healthcare program beneficiaries. Recent False Claims Act (FCA) enforcement developments demonstrate that stakeholders should carefully consider the structure and risk associated with providing assistance to patients, regardless of disease state.

In response to calls from industry stakeholders for increased innovation, coordination, and tailored regulatory approaches to the development of treatments in rare disease, in recent years the US Food and Drug Administration (FDA) has established a number of rare disease-focused programs within the agency. Building on other recent FDA initiatives such as the Center for Drug Evaluation and Research (CDER) Accelerating Rare disease Cures (ARC) Program and the Center for Biologic Evaluation and Research (CBER) Rare Disease Program, FDA expanded its rare disease toolkit to include its Rare Disease Innovation Hub in 2024 to serve as a point of collaboration between CDER and CBER with the overarching goal of enhancing collaboration across centers to improve patient outcomes and addressing common challenges in drug development for rare diseases.

Several members of our firm’s life sciences team were on the ground at the 43rd Annual J.P. Morgan Healthcare Conference in San Francisco from January 13 to 16, 2025. It was an exciting and sunny four days, during which key players from across the life sciences industry gathered to engage in deal discussions and consider upcoming trends for the pharmaceutical, biotechnology, and healthcare industries. Although there was a notable increase in the level of security at the conference given recent events, the overall sentiment of the conference was one of optimism.

The life sciences industry has long been at the forefront of innovation, and 2025 promises to continue this trajectory with exciting developments in intellectual property (IP), licensing, and mergers and acquisitions (M&A). As the sector navigates a dynamic landscape of scientific advancements, economic pressures, and regulatory changes, stakeholders are increasingly leveraging strategic transactions to gain a competitive edge.

In response to rising prescription costs and overall state-level healthcare spending, numerous states, including Maryland and Oregon, have established prescription drug affordability review boards (PDABs) to review certain high-cost prescription drugs and determine if states should take action to reduce those prices.

Aiming to expedite drug development, enhance the body of clinical evidence supporting new and existing therapies, and improve participation and diversity in clinical trial populations, the US Food and Drug Administration (FDA) recently issued three guidance documents that impacted the clinical trial landscape and established a new hub for clinical trial innovation.

While audits are part of doing business in the ordinary course, businesses are generally less than eager to open the books. And the same is true when it comes to the 340B Drug Pricing Program. Audits are now making the list of ongoing disputes between manufacturers and covered entities (CEs) participating in the program.