clinical trials

The U.S. Food and Drug Administration (FDA) recently released a draft guidance titled “Frequently Asked Questions — Developing Potential Cellular and Gene Therapy Products.”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…

Today’s large language models can be as unreliable as they are eloquent. Their tendency to fabricate facts and lose the thread makes them risky tools for scientific research, especially in highly regulated industries like pharmaceuticals and chemistry. They also struggle to provide sources and will fabricate a bogus academic journal without batting an eye. Speaking…

In 2024, we saw the expanded use of synthetic data and natural language processing transform drug discovery and development. In a batch of predictions published in December of 2023, one expert predicted that synthetic data was set to “take off” in drug research. In 2025, the pendulum could begin swinging back the other way, according…

This case study is sponsored by Infor.  In the complex landscape of clinical trials and medical research, data fragmentation remains a major obstacle, hindering progress and patient outcomes. The case study “Advancing Clinical Trials and Research with Cloverleaf” showcases how Infor Cloverleaf significantly enhances clinical trials by integrating Real-World Evidence (RWE) to overcome data fragmentation.…

For millions living with rheumatoid arthritis (RA), the path to effective treatment is often a frustrating cycle of trial and error. Typically beginning with disease-modifying antirheumatic drugs (DMARDs) like methotrexate, the goal is low disease activity. Yet RA’s heterogeneity means many patients cycle through various therapies—biologic DMARDs like anti-TNF agents, JAK inhibitors, IL-6 receptor antagonists,…

Introduction Endometriosis is a chronic incurable gynecological condition with cyclical pelvic pain, heavy bleeding, infertility, and reduced quality of life as its main manifestations. Almost half of women with it experience infertility. [1] No symptoms are specifically pathognomonic or characteristic of endometriosis. Often referred to as a ‘silent disease’ because of how often the diagnosis…

Nine months into 2024, the search for effective neurodegenerative disease therapies continues, yielding a mix of promising advances and frustrating setbacks. After something of a lull in the 1990s and early 2000s, research into novel treatments for neurodegenerative diseases has surged back to life. One factor propelling the trend is the uptick in neurodegenerative disease…

Alzheimer’s clinical trials are among the most challenging and expensive studies to conduct with a 2021 study pegging the total costs since 1995 at $42.5 billion. Phase 3 trials are the biggest cost drivers, involving hundreds or thousands of participants. “We’re talking about studies with sometimes two thousand people per phase three clinical trial in…

Recursion Pharmaceuticals and Exscientia, two of the most prominent AI-driven drug discovery companies, are merging. The deal, which the firms expect to be official in early 2025, would create a portfolio of clinical and near-clinical programs with an expected 10 clinical readouts over the the next 18 months with “peak sales opportunities in excess of…

Liraglutide, a GLP-1 receptor agonist medication that first won FDA approval in 2010, may reduce cognitive impairment in Alzheimer’s patients. At this year’s Alzheimer’s Association International Conference, data from a Phase 2b clinical trial with 204 mild AD patients serving as participants was revealed, showing that the drug lowered decline in cognition by up to…

When it comes to medical AI, the biggest names aren’t necessarily delivering the best results. While tech giants race to build ever-larger language models, a new preprint reveals that when it comes to clinical accuracy and physician trust, a smaller player is outperforming the industry heavyweights. Putting large language model-based systems to the test Researchers…

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…

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…

The recent FDA approval of Lilly’s Kisunla (donanemab) represents a significant win in Alzheimer’s drug development, a field that has seen a  decades of false starts and fizzled hopes. This approval, along with that of lecanemab in 2023, could mark a turning point in AD treatment. Alzheimer’s disease is reaching epidemic proportions with an estimated…

As pharma giants like Darmstadt, Germany–based Merck KGaA announce new pacts with AI companies, the drug development landscape is witnessing a familiar pattern of emerging tech adoption. AI’s promise of faster, more efficient drug discovery has captured the industry’s imagination, leading Big Pharma to ramp up investment in AI startups and partnerships. Yet, the technology’s…

In the emerging field of digital pathology, Aleksandra Zuraw, DVM, Ph.D., DACVP, is one of the most prominent voices. Her journey to this position, however, was far from straightforward. When Zuraw was 12, she dreamed of becoming a veterinarian, picturing herself following in the footsteps of the British veterinary surgeon James Herriot, tending to “All Creatures…

Today, Lilly announced promising data for the SURMOUNT-OSA phase 3 clinical trials testing tirzepatide in adults with moderate-to-severe obstructive sleep apnea (OSA) and obesity. The company shared the data at the American Diabetes Association’s (ADA) 84th Scientific Sessions while publishing them in The New England Journal of Medicine (NEJM). The SURMOUNT-OSA phase 3 trial consists…

Featured on the cover of Nature Machine Intelligence, one of Iambic Therapeutics‘ AI platforms, known as NeuralPLexer, can accurately predict protein-ligand complex structures. The platform excels at predicting the complex 3D structures formed when proteins bind with drug-like molecules. In a benchmark study featured in the publication, NeuralPLexer outperformed other systems, including AlphaFold2, in its…

Imagine tripling or quadrupling the speed at which eligible patients are matched to potentially life-saving cancer trials. That’s the promise of ConcertAI’s AI-powered screening technology, which demonstrated a 3 to 4 times efficiency gain over traditional EMR-based methods in a recent study presented at the American Society of Clinical Oncology (ASCO) conference. Focusing on relapsed…

What’s the difference between a multimodal generative AI system to plan a vacation itinerary versus guiding cancer treatment? For the former, data quality is a definite plus, but for the latter, it’s indispensable. “If I ask ChatGPT to plan a vacation for me and it spits out something, I might sit there and second guess…

It’s something of a truism that about nine out of ten drug candidates in clinical trials fail to reach the market. While some amount of guess-work is unavoidable in drug development, Dr. Ali Pashazadeh, co-founder and CEO of Treehill Partners sees faulty decision-making and poor communication as chief factors contributing to high failure rate. While…

 Over the past year, North American biotech firms continued to lear the world in funding by a significant margin in an analysis that included a various equity fundraising types drawn from Crunchbase. Argenx, a publicly traded Dutch biotech, raised $1.1 billion from investors in July 2023 through a global offering. Next in line was…

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…

On March 6, 2024, the U.S. Securities and Exchange Commission approved a rule to require some companies to report some of their carbon emissions (Scope 1 and 2, but not Scope 3)1. While some feel this rule doesn’t go far enough, it certainly demonstrates the need to better understand our respective carbon footprints and develop…

Ozempic, Rybelsus and Wegovy have transformed the diabetes and weight loss treatment landscape, but when it comes to the impact of their active ingredient, semaglutide, on fetal development, “the answer is we do not know,” said Dr. Marijane Hynes, clinical professor of medicine at the George Washington University School of Medicine and Health Sciences. Hynes…