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.

As part of a broader resurgence in pharmaceutical pricing reform and manufacturing policies in 2025, President Donald Trump and bipartisan congressional leaders have introduced contemporaneous proposals to lower prescription drug prices under “most-favored-nation” (MFN) drug pricing models.

Distributors, dispensers, and retailers of medical device products face a patchwork of federal and state requirements governing distribution chain compliance. Given the evolving and expanding requirements for device distribution activities, companies must take proactive measures to assess and mitigate risk. Below are key considerations that companies operating in this space should consider to safeguard their operations and maintain compliance.

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.”

The US Food and Drug Administration (FDA) recently announced a strategic roadmap to phase out animal testing requirements for certain drug products, starting with monoclonal antibodies. This shift marks a significant change in regulatory policy, reflecting advancements in science and technology that offer alternative testing methods.

The pharmaceutical industry is a critical component of the global economy, impacting public health, national security, and economic stability. Recent developments—including investigations into the national security implications of pharmaceutical imports, executive actions aimed at reducing prescription drug prices, and the evolving role of pharmacy benefit managers (PBMs)—highlight the complex interplay of policy, economics, and healthcare.

The US Food and Drug Administration (FDA) recently published two new draft guidances to implement provisions of the Food and Drug Omnibus Reform Act (FDORA) aimed at refining the accelerated approval pathway. These guidances seek to address regulatory challenges and enhance oversight of drug products granted accelerated approval. Additionally, a recent report from the Office of the Inspector General (OIG) underscores FDA’s efforts in refining the accelerated approval pathway. These efforts raise important implications for the rare disease community.

Private foundations providing support, resources, and advocacy for individuals and families affected by rare diseases have played an important role for more than 30 years in the advancement of treatments for rare diseases. Much of this support has been in the form of grants to nonprofit institutions to help fund research on promising treatments.

The value of merger and acquisition (M&A) deals of rare disease companies has increased significantly over the past few years (from $18.9 billion in 2019 to $50.6 billion in 2022 according to a 2024 article in Nature Reviews Drug Discovery). This combined with recent billion-dollar acquisitions in the rare disease space have piqued the interest of large pharmaceutical companies, as well as investors, and may be indicative of further growth of this life sciences sector in the years ahead. This article analyses some of the key challenges faced by parties to an M&A transaction in the rare disease space and outlines strategies that can be deployed to ensure a successful closing.