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The standard regulatory threshold of “substantial evidence of effectiveness” has historically relied on large-scale randomized controlled trials (RCTs) to mitigate statistical noise. For ultra-rare genetic conditions, however, the laws of small numbers make traditional RCTs practically and ethically unfeasible. The FDA’s latest guidance introduces a “Plausible Mechanism Framework” to bridge this gap, allowing deep molecular … Read more

Keeping pace with the rapid evolution of medical technology requires a regulatory framework that is both rigorous and agile. The FDA’s latest announcement regarding the Food and Drug Administration Modernization Act of 1997 represents a vital step in aligning regulatory expectations with modern engineering. By updating its list of recognized consensus standards, the agency is … Read more

The FDA’s transition to a single-trial default for drug and biologic approvals marks a pivotal evolution in regulatory philosophy, moving away from the 1962 Kefauver-Harris dual-study precedent. For industry leadership, this shift offers a significant opportunity to accelerate speed-to-market, provided that clinical programs are built on a foundation of absolute statistical and biological rigor. However, … Read more

Artificial intelligence (AI) and advanced modeling are reshaping how drugs and biologics are developed, reviewed, and regulated worldwide. In late 2024 and early 2025, the FDA released draft guidance on AI use in regulatory submissions, while the International Council for Harmonization (ICH) finalized its M15 guideline on model-informed drug development (MIDD). Together, these documents signal … Read more

What if regulatory decisions reflected not just clinical endpoints, but what patients truly value? What if benefit–risk assessments explicitly accounted for patients’ willingness to accept uncertainty, risk, or trade-offs? The FDA’s adoption of ICH E22 signals its perspective on using patient preferences for regulatory decisions by formalizing how structured patient preference data can be designed, … Read more

For decades, pharmaceutical manufacturing steadily migrated overseas, leaving the U.S. dependent on fragile global supply chains. With the launch of the FDA PreCheck pilot program, the agency is offering manufacturers something long requested: regulatory predictability before product submission. FDA PreCheck is designed to strengthen the domestic pharmaceutical supply chain by facilitating the construction of U.S.-based … Read more

What’s the real impact of the FDA’s post-market safety authorities on drug safety actions? A new analysis published in JAMA Internal Medicine explores how post-approval safety measures, including withdrawals, boxed warnings, and safety communications, have evolved since the FDA Amendments Act (FDAAA) of 2007. The findings challenge assumptions about how quickly safety issues are identified … Read more

The 2025 CDRH Annual Report provides a comprehensive overview of the Center’s continued progress in balancing the review of new medical technologies with the rigorous oversight of patient safety. This latest update shows the FDA meeting its commitment to predictable regulatory pathways, ensuring that both manufacturers and patients can rely on consistent standards of excellence. … Read more

In the fast-paced world of oncology, every second counts for patients awaiting life-saving treatments. The FDA’s Assessment Aid is a game-changing tool designed to strip away administrative red tape and put the focus back on what matters most: critical scientific evaluation. By streamlining the communications between drug developers and regulators, this initiative is redefining the … Read more

The era of human-exclusive regulatory review has officially come to an end. With the formal update to SOPP 8217, the FDA is no longer just “experimenting” with machine learning; they are operationalizing it within the core of the IND review process. By introducing a dedicated AI reviewer from the Office of Biostatistics and Pharmacovigilance (OBPV), … Read more