For months, everyone in the pharma industry has been trying to divine which direction commissioner Marty Makary will take the FDA in 2025. A change in FDA leadership always brings a new set of priorities—and, inevitably, a flurry of debate about whether the Agency’s updated vision will deliver real progress. Well, the wait is over….
Digital health therapeutics (DTx), applications increasingly powered by AI and/or machine learning, are on the rise and promise to increase access to healthcare, provide real-time monitoring of health data, improve patient engagement and adherence to treatment, and reduce healthcare costs. What Are Digital Health Therapeutics? DTx are evidence-based therapeutic interventions driven by software and technology…
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 This article will focus on the draft guidance updates to…
The US Food and Drug Administration (FDA) has just taken a historic step by releasing its first-ever draft guidance on using artificial intelligence (AI) for regulatory decision-making related to the safety, effectiveness, or quality of drugs and biologics.1 Published in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…
The FDA’s new Digital Health Advisory Committee (DHAC) convened for the first time on November 20-21, 2024. The topic of discussion was medical devices that rely on generative AI (GenAI) models, which hold transformative potential for healthcare but pose unique regulatory challenges. For example, how to define the device’s intended use, manage errors like hallucinations…
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…
When chimeric antigen receptor (CAR)‑T cells first rolled off the assembly line 7 years ago, with the approval of Kymriah, they were the newest and most technically advanced immunotherapy against cancer, and they demonstrated the potential to elicit profound and durable responses in patients with relapsed/refractory hematologic malignancies. The patient’s own (autologous) T cells are genetically modified in…
The FDA has approved Madrigal Pharmaceuticals’ Rezdiffra™ (resmetirom) for nonalcoholic steatohepatitis (NASH). The approval is a watershed event, marking a historic turning point in the long-standing quest to find an effective treatment for the millions of Americans afflicted by this devastating condition. A targeted treatment for NASH has eluded pharmaceutical scientists for nearly a half century……
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…
“Silent killer” is an apt description for a potentially fatal disease that progresses slowly, without any obvious symptoms in the early stages. When you couple this insidious type of pathology with a rapidly increasing prevalence, the disease is often described in terms akin to an epidemic. Nonalcoholic steatohepatitis (NASH)—a disease characterized by fat accumulation in the…
Preparing for an FDA advisory committee meeting (AdCom) is a daunting task that can take anywhere from 3 to 9 months of intense preparation. At ProEd Regulatory, we partner with our clients to ensure that they’re well prepared to present their data and answer questions from the committee with confidence and poise. Our clients have…
Introduction Throughout history, humans have had a complex relationship with psychedelics. For millennia, ancient indigenous cultures used them for spiritual and healing purposes. For example, in 2007, archaeologists in Spain discovered mushrooms found at a burial site dated more than 7,000 years old. The mushrooms found at the site were identified as psilocybin, a type…
Artificial Intelligence (AI) is rapidly transforming how we work, and the pharmaceutical industry is no exception. In particular, the emergence of Generative Pre-trained Transformer (GPT) models has the potential to revolutionize several aspects of the biotech business, including drug discovery and clinical trials. According to a recent survey, 39% of healthcare professionals see AI (including…
Artificial Intelligence (AI) is significantly transforming pharmaceutical regulatory affairs. Rather than replacing regulatory professionals, AI enhances their capabilities and allows them more time for strategic activities. This blog post explores how AI is revolutionizing the regulatory submission process, the challenges posed by the rapidly evolving AI regulatory landscape, and what regulatory affairs professionals need to…
