Regulatory affairs

Every month, thousands of drug-promo packages, ranging from TV spots to banner ads and updated labels, land in the FDA’s queue for 2253 review. The agency tracks “the number of required submissions of promotional communications to FDA on Form 2253 and the total number of submitted materials, as a submission may contain more than one…

The FDA is betting big on genAI to transform tedious workflows, setting a June 30 deadline for agency-wide deployment. While details of its plans are scarce at present, using genAI to reduce FDA’s paperwork burden seems inevitable. The broader goal doesn’t qualify as “a high-order AI problem today,” said Vik Bajaj, founder and CEO of…

Branding U.S. drug pricing an “egregious imbalance” in which a nation with “less than five percent of the world’s population … funds around three‑quarters of global pharmaceutical profits,” President Donald Trump on 12 May signed the Delivering Most-Favored-Nation Prescription Drug Pricing to American Patients order. The executive order describes the current dynamic as one in…

Determined to slash bureaucratic drag on drug approvals, FDA chief Dr. Martin Makary is ordering every center to plug generative-AI tools into its review pipeline by June 30, an agency-wide gamble that, he says, will turn days of paperwork into minutes of analysis. Multiple independent studies, including from McKinsey, Bain, Capgemini, the Federal Bar Association,…

On the surface, President Donald Trump’s latest drug‑pricing order looks like a populist coup. The White House says it will “put American patients first once again,” capping insulin at three cents and promising deeper Medicare savings. Yet nested in the fine print is a directive for HHS to work with Congress to delay Medicare’s power…

The pharma industry faces uncertainty as new HHS Secretary Robert F. Kennedy Jr. spearheads a major departmental overhaul aiming for a total reduction of about 20,000 positions (nearly a quarter of the workforce). The cuts build on roughly 10,000 voluntary departures earlier in 2025. A formal plan announced March 27, 2025, initiates Reduction-in-Force (RIF) actions…

In the classic Bill Murray comedy Groundhog Day, the main character is stuck reliving the same day—again and again. Biotech startups often face a similar fate when they sprint to first-in-human trials without planning for the realities of large-scale manufacturing. “Many times, researchers rush to get their product tested in humans with processes that are…

An analysis of FDA approvals across these five years shows small molecules remain central to modern therapeutics, even as complex biologics—particularly monoclonal antibodies (mAbs), antibody–drug conjugates (ADCs), and next-generation modalities—are on the rise. Steady R&D investment in these advanced approaches reflects growing clinical confidence in targeted therapies that address difficult or rare disease pathways. 1.…

Novartis has released positive topline results from the phase 3 STEER trial evaluating OAV101 IT (intrathecal onasemnogene abeparvovec) in treatment-naïve patients with spinal muscular atrophy (SMA) Type 2, aged two to under 18 years. The STEER trial is a pivotal, sham-controlled study designed to evaluate OAV101 IT against a procedure that mimics drug administration without…

A growing chorus of experts agree: 2025 signals a year when AI and its generative variants (genAI) move beyond early experimentation into a period of tangible, large-scale impact — including in the life sciences. While challenges remain as EY recently noted, the future will likely be one where the focus shifts from anticipating breakthrough genAI…

YTD 2024 FDA approvals: By the numbers 44 Novel Drug Approvals As of December 5, 2024, the FDA had approved a total of 44 novel drugs, spanning a diverse array of therapeutic areas and patient populations. These new treatments address both widespread public health concerns, such as cardiovascular disease and COPD, and more narrowly defined…

The understanding that there is a connection between breath and diseases can date back to over two thousand years ago, when Hippocrates described fetor oris and fetor hepaticus in his report on breath aroma (1). Since then, research into volatile organic compounds (VOCs) exhaled in breath and their involvement in disease physiology has led to…

The development of Proteolysis Targeting Chimeras, or “PROTACs*,” has garnered significant attention in the pharmaceutical industry due to their potential to target and degrade disease-causing proteins previously considered undruggable. As a novel therapeutic modality, PROTACs offer a promising alternative to traditional small-molecule inhibitors and biologics. However, despite the excitement around these therapies, preclinical evaluations of…

The U.S. Food and Drug Administration (FDA) recently released a draft guidance titled “Frequently Asked Questions — Developing Potential Cellular and Gene Therapy Products.” This 40-page document, while not legally binding, offers insights into best practices for common questions received by the Office of Therapeutic Products (OTP). While each question is answered in brief, the…

In August 2024, MDMA, which is better known as ecstasy, faced a setback as a potential therapy for PTSD—a complete response letter from the FDA. While its developer, Lykos Therapeutics, plans on launching a new Phase 3 trial with tighter scrutiny, another psychedelic company, MindMed, is readying a pivotal study of LSD for generalized anxiety…

Manufacturers in the biotech sector face unique challenges ranging from stringent regulatory requirements to complex production processes. Implementing an ERP (enterprise resource planning) system tailored to their needs is crucial for ensuring compliance, efficiency, and scalability. Whether you are a life sciences CMO (contract manufacturing organization), a pharmaceutical manufacturer with an R&D department, or a…

Real-world evidence and real-world data The use of real-world evidence (RWE) to support regulatory and reimbursement decision-making received increasing attention over the past decades. The US FDA provides a definition of RWE as “the clinical evidence about the usage and potential benefits and risks of a medical product”1. RWE can be used across the entire…

Large molecule therapies hold immense promise for treating an array of diseases, but their development presents unique challenges in preclinical testing. Toxicology studies form a cornerstone of this process, providing essential data on the drug’s biological effects. While small molecule drugs have been the focus of drug development for decades, large molecule therapies present unique challenges…

Medicare is taking its scalpel to drug prices, excising billions in costs for popular medications like Eliquis and Jardiance. The Biden-Harris Administration announced significant agreements with drug makers that would cut list prices by up to 79% for the first 10 drugs selected under the new Medicare drug price negotiation program. CMS announced last year…

While clinical trials and regulatory filings offer a semi-structured view of drug safety, a large amount of insights lie in sources ranging from patient support programs (PSPs) to social media posts. As Natural Language Processing (NLP) evolves, a growing number of tools are becoming available to unlock this potential. Deepanshu Saini, Director of Program Management…

The pandemic, the rise of decentralized trials and a wave of technological advances have each played a role in reshaping clinical trials and safety reporting. While these events have been changing the industry, the internet’s growing convenience and accessibility has also empowered and transformed the patient experience. Patients are increasingly turning to online platforms, particularly…

Phesi, a Connecticut-based clinical development analytics company, has published research in Bone Marrow Transplantation showing the potential of digital twins to replace standard-of-care control arms in clinical trials. The study focused on chronic graft versus host disease (cGvHD), a serious complication affecting 30–50% of the 50,000 cancer patients who receive hematopoietic cell transplantation (HCT) each…

The pharmaceutical industry has long faced something of a perfect storm of regulatory upheaval and technological disruption. But now is different. The Inflation Reduction Act represents a unique challenge for pharma companies as the U.S. government can force them to negotiate drug prices for some Medicare drugs. And despite its name, some pundits believe the…

A group of prominent researchers and clinicians have published a consensus statement backing the use of MDMA-assisted therapy for post-traumatic stress disorder (PTSD). This statement comes as FDA is reviewing a New Drug Application for midomafetamine (MDMA) for the condition. “On the basis of the safety and efficacy data we have seen, assuming the integrity…

In terms of treatment outcomes, the LGBTQ+ community continues to face significant disparities. For instance, a recent study in JAMA on breast cancer outcomes reported that those from sexual and gender minority groups were more likely to face delays in diagnosis. LGBTQ+ patients were also more likely to decline oncologist-recommended therapies while experiencing a three-fold…