When FDA reviewers discovered that a generative-AI pilot could slash a 3-day document review task to mere minutes—a time-savings of 99%!—it became clear that artificial intelligence is no longer an aspirational tool but a present-day catalyst for regulatory transformation. That single statistic captures why FDA is fast-tracking AI: the volume and complexity of regulatory submissions…
On January 6 and 7, 2025, the FDA released approximately 30 draft or final guidance documents1 covering a wide array of regulatory topics, including: These guidance documents reflect priorities the FDA deems urgent, especially in light of the new administration’s efforts to alter regulatory approaches. This article will focus on the draft guidance updates to…
The US Food and Drug Administration (FDA) has just taken a historic step by releasing its first-ever draft guidance on using artificial intelligence (AI) for regulatory decision-making related to the safety, effectiveness, or quality of drugs and biologics.1 Published in January 2025, this framework comes at a pivotal time, as AI’s potential to transform healthcare…
In February 2024, we reported on anticipated changes to the format of FDA advisory committee meetings (AdComs) based on public comments from FDA Commissioner Robert Califf and other FDA officials earlier this year. Some of those changes have, indeed, come to pass—most notably, a full‑throated return to in-person AdComs at the White Oak facility in…
This year, Rare Disease Day is on February 29, 2024. Rare Disease Day is observed on the last day of February, which is the “rarest of calendar dates1 to underscore the nature of these diseases” and serves to raise awareness of rare diseases and those individuals affected by them. FDA is holding a virtual meeting2…
Changes Are Coming to Advisory Committee Meetings Advisory Committee meetings (AdComs) are a long-standing feature of the FDA drug approval process. How and where those meetings are conducted has been evolving over the past few years, since the introduction of virtual meetings, and even more considerable reforms may be coming sooner rather than later. According…
Today, the US Food and Drug Administration (FDA) approved exagamglogene autotemcel (exa‑cel), the first CRISPR-based gene therapy for the treatment of sickle cell disease (SCD). Exa‑cel was co-developed by Vertex Pharmaceuticals and CRISPR Therapeutics. This is a landmark approval that paves the way not only for better outcomes for patients with SCD, but for many…
The field of neurology is experiencing a significant upswing in innovative therapeutic development, propelled by advances in genetics, neuroimaging techniques, and biomarker research. However, neurological diseases are inherently difficult to treat, and there remains an urgent need to rapidly translate these advances into more effective treatments. It is timely then, that several recent drug approvals…
Real-world data (RWD) can be used to create historical control groups for clinical trials in rare diseases where a randomized controlled trial (RCT) is not feasible. But what happens when the US Food and Drug Administration (FDA) doesn’t accept it? Since passage of the 21st Century Cures Act in 2016, FDA has promoted the use…
In Part 1 of our blog series on the saga of PI3K inhibitors, we reviewed the FDA’s recent Oncologic Drugs Advisory Committee (ODAC) meeting on April 21, 2022, to discuss the agency’s concerns about PI3K inhibitors (PI3Kis). The panel voted resoundingly (16 yes votes; 1 abstention) that future approvals of PI3Kis should be supported by…
