Regulatory affairs

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…

The pharmaceutical industry is grappling with the consequences of a significant data breach at Cencora, the prominent drug distributor formerly known as AmerisourceBergen. Recent cybersecurity rules from the U.S. Securities and Exchange Commissions (SEC), which came into effect in December 2023, have also played a role in a wave of data breach reports, highlighting the…

In 2023, the American Society of Anesthesiologists (ASA) issued guidelines advising patients to discontinue GLP-1 receptor agonists (GLP1-RAs) a week before surgery, citing concerns over potential operative complications, leading to delayed or rescheduled surgeries across the U.S. Dr. David Klonoff, a diabetes researcher and Endocrinologist at Mill Peninsula Medical Center, thought it was curious that…

Strategically selecting the right outsourcing models can accelerate the cost and time curve of drug development by maximizing quality, operational success, and financial efficiencies. To help meet increasingly demanding timelines, organizations are looking more to functional service partnership models to outsource specific functions of clinical trials (e.g., clinical operations, pharmacovigilance, medical writing, etc.). The increasing…

The success of any organization is dependent on consistent, clear, and reliable information sharing among all internal stakeholders. In the pharmaceutical industry, where there are multiple complex layers within manufacturing processes, communication among teams is especially critical. If information-sharing gaps occur among these layers, vulnerabilities could result in costly errors impacting all levels of the…

The global counterfeit drug trade has exploded into a deadly public health crisis, with the rise of online pharmacies and increasingly sophisticated criminal networks fueling the trend. A 2020 analysis from the Organisation for Economic Co-operation and Development (OECD) and the European Union Intellectual Property Office (EUIPO), found that the problem is global in scope…

The FDA’s Center for Drug Evaluation and Research (CDER) recently unveiled the launch of the CDER Center for Clinical Trial Innovation (C3TI), which aims to drive clinical trial innovation. When asked to assess the potential impact of the center, Charles Fisher, founder and CEO of Unlearn, said: “The truth is, I don’t know. I’m sort…