As Prescribed

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 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.

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.

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 US Food and Drug Administration (FDA) recently issued a notice of noncompliance to the FADOI Foundation, citing the organization’s failure to submit required clinical trial results to ClinicalTrials.gov. The notice reflects FDA’s increasing attention toward noncompliance with the agency’s requirements for submission of clinical trials information and may signal an increase in enforcement that we may see from FDA going forward.

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.

The US Food and Drug Administration (FDA) has announced a final rule expanding the approval pathway for nonprescription drug products by allowing the approval of a nonprescription drug product with an “Additional Condition for Nonprescription Use” (ACNU). This rule aims to expand consumer access to certain nonprescription drugs—that would otherwise be available only via prescription—by permitting their retail sale with an additional condition beyond labeling, such as a digital questionnaire to help the consumer decide if the medication is right for them.