This site uses cookies to improve your experience. To help us insure we adhere to various privacy regulations, please select your country/region of residence. If you do not select a country, we will assume you are from the United States. Select your Cookie Settings or view our Privacy Policy and Terms of Use.
Cookie Settings
Cookies and similar technologies are used on this website for proper function of the website, for tracking performance analytics and for marketing purposes. We and some of our third-party providers may use cookie data for various purposes. Please review the cookie settings below and choose your preference.
Used for the proper function of the website
Used for monitoring website traffic and interactions
Cookie Settings
Cookies and similar technologies are used on this website for proper function of the website, for tracking performance analytics and for marketing purposes. We and some of our third-party providers may use cookie data for various purposes. Please review the cookie settings below and choose your preference.
Strictly Necessary: Used for the proper function of the website
Performance/Analytics: Used for monitoring website traffic and interactions
Advanced strategies and tools are being implemented to establish the safety and efficacy of new therapeutic modalities, with the development of new biomarkers becoming one of the most relevant approaches for enhancing the precision and utility of early-stage studies.
Developing treatments for individuals living with rare diseases is critical, but orphan drugdevelopment is laden with unique obstacles that necessitate innovative, multifaceted approaches. Moreover, in some rare disease trials, it is unethical to design a control group of patients with a placebo.
Our annual look at the state of the drugdevelopment industry highlights a dual set of challenges complicating progress. Pressure and requirements to engage diverse patient populations in trials have become more challenging and expensive, requiring tailored strategies that can stretch both resources and budgets.
However, as we note in that post, the design, timing of initiation, and timely conduct of confirmatory trials are also important considerations in FDAs determination of whether accelerated approval is appropriate. This blog post focuses on interpreting these new authorities with respect to timely conduct of confirmatory trials.
Food and Drug Administration (FDA) plays a pivotal role in fostering the development of treatments for rare diseases through its Orphan Products Grants Program. Each year, FDA selects a limited number of clinical trials to fund to help sponsors pursue development of medical products for rare diseases and advance their field.
To understand the significance of this goal, it is important to recognise the current drugdevelopment process. This uncertainty contributes to the high failure rate and enormous costs of drugdevelopment. Currently, understanding these aspects requires extensive animal testing followed by human trials.
Drugdevelopment is plagued by complex challenges, but multimodal AI is unlocking new opportunities. By integrating diverse data sources – from genomics to clinical insights – this approach is accelerating drug discovery, improving patient stratification and boosting success rates.
“Protocol development can be a complex and meticulous process,” he explains, “as it’s crucial to ensure that trial candidates meet the necessary inclusion criteria.” Prior treatments and patient histories often introduce variables that can affect outcomes, making thoughtful trial design essential for generating reliable data.
A personal journey to Alltrna Michelle Werner’s career has spanned over 20 years in the pharmaceutical industry, where she developed her expertise in oncology drugdevelopment at leading companies such as Bristol Myers Squibb, AstraZeneca, and Novartis.
As Vice President of Oncology Early Development (OED) at AbbVie , she leads the charge on translating bold scientific ideas into real-world impact – and she’s doing it with a focus on some of the most tenacious cancers out there. Her team sits right at the edge of innovation, where discovery science meets clinical execution. “In
The trial met both its primary and key secondary endpoints, signaling a potential breakthrough in a condition with few, if any, effective treatment options. The full dataset is expected to be presented at an upcoming international scientific conference, which will provide greater clarity on the magnitude of fenfluramine’s clinical benefits.
This approach has led to the discovery of numerous potential drug candidates. From Lab to Market: The Long Road of DrugDevelopment Once a promising compound is identified, it enters the long and costly process of drugdevelopment. Q: How long does a drug patent last?
Altasciences At CPHI Americas 2025 pmjackson Thu, 07/03/2025 - 13:41 In the ever-changing drugdevelopment world, staying agile is essential to enabling more informed decisions, faster. It’s a challenge that affects a large percentage of new drug candidates—one we’re passionate about helping to solve.
With more than 40 clinical and commercial-stage assets in active development, BeOne Medicines is demonstrating a scale of ambition matched by few in the field. BeOne Medicines’ rise has been fueled by a strategic commitment to innovation at every level of the drugdevelopment continuum.
DARPin therapeutics were a prominent theme at AACR 2025 , with Molecular Partners presenting a trio of preclinical programmes spanning radiopharmaceuticals and immune cell engagers. “My passion for science drew me to DARPin technology, and I was eager to contribute to the development of new DARPin therapies.”
Antibody-drugconjugates (ADCs) represent a significant advancement in drug discovery, combining the precision of monoclonal antibodies with the cancer-killing power of cytotoxic drugs. This type of analysis is crucial for drugdevelopers, providing a framework to identify and mitigate toxicity risks early in the design process.
Dr Pooja Hingorani, Senior Medical Director of Oncology Early Development at AbbVie , shares her journey in STEM, from her early days in New Delhi to her impactful career in oncology research and drugdevelopment. In addition to this, I also chaired national trials evaluating an anti-GD2 antibody in pediatric osteosarcoma.
Two large and randomized clinical trials, IMpower-133 and CASPIAN, showing statistically significant improvements in outcomes when chemotherapy was combined with atezolizumab or durvalumab, respectively, in the first-line treatment of ES-SCLC. This Phase 3 trial compared tarlatamab with chemotherapy as second-line treatment.
4 Essential Topics to Cover in Your First CRO Meeting pmjackson Fri, 11/15/2024 - 19:22 Starting a partnership with a contract research organization (CRO) is a sometimes intimidating—yet exciting—first step in your drugdevelopment journey. Let the CRO know any key outcomes from each.
By: Juliane Mills, Senior Director, Therapeutic Strategy Lead, Rare Disease The rise of patient-led clinical research, particularly in rare disease, represents a significant shift in the clinical trial landscape. This proactive approach allows sponsors to address patient needs more effectively and streamline the development process.
AI has applications from the simple to the complex; from automating mundane tasks and accelerating drugdevelopment, to increasing accuracy in documentation and uncovering complex correlations within vast datasets. This presents three core conflicts: Opaque provenance: The data used to train the model is not transparent.
GPCRs are critical targets for drugdevelopment due to their involvement in numerous disease pathologies, with many medications working by either activating or inhibiting these receptors. 2022) Why 90% of clinical drugdevelopment fails and how to improve it?, References Sun D, et al. doi:10.1016/j.apsb.2022.02.002.
The drug, formerly known as TAK-861 but now called oveporexton, was evaluated in two Phase 3 studies in a main type of narcolepsy. No serious treatment-related side effects were reported, and more than 95% of study participants who completed the trials enrolled in a long-term extension study. You can unsubscribe at anytime.
These resources provide accurate and current strategies that strongly represent FDAs standpoint and have routinely been leveraged by Premiers team of strategists when guiding sponsors through their development plans. The summary presented in the table below features some of these initiatives. Program What is it?
In Issue 7 of The Altascientist , we delve into these factors, the importance of drug interaction studies, and how to limit adverse effects and maximize treatment response. The potential for an investigational drug to cause DDIs should be investigated in a stepwise manner during drugdevelopment.
Drugdevelopment is a complex and highly regulated process. Food and Drug Administration (FDA), the European Medicines Agency (EMA), and other global counterparts, set rigorous standards to ensure that drugs are safe, effective, and high-quality. Regulatory agencies, such as the U.S.
Despite their exciting potential, the smooth operation of cell therapy developmenttrials requires extraordinary orchestration, perfectly aligning the product and patient journeys. While clinical supply is essential to any successful trial, autologous cell therapy trials occupy the far end of the spectrum regarding risk tolerance.
As an undergraduate biology student, I spent some time in a TB lab working on antibiotic resistance — a growing concern for drugdevelopers. A 1994 review of 14 prospective trials and 12 case-control studies revealed that the BCG vaccine reduced the risk of TB by 50 percent. Subscribe to Asimov Press. tuberculosis.
SciePro via Getty Images Dive Brief: The Food and Drug Administration has approved a new oral medication for an uncommon kind of tumor, clearing Nuvation Bio’s Ibtrozi on Wednesday for certain people whose metastatic non-small cell lung cancer has a type of alteration in the gene ROS1. You can unsubscribe at anytime. Sanofi’s $9.1
Furthermore, training GenAI on healthcare data itself can present major regulatory challenges. By Jacob Bell • July 1, 2025 Kendall Davis/BioPharma Dive Tracker 10 clinical trials to watch the rest of 2025 Expected readouts in obesity, lung cancer and AATD headline a series of study results that could give the biotechnology sector a boost.
With a particular focus on biologics, cell, and gene therapies, Galbraiths work is centred on equipping drugdevelopers with the tools, insights, and support needed to advance their therapies from concept to clinic. These innovative modalities present both exciting possibilities and unique challenges in drug discovery and development.
Vor is paying RemeGene $45 million upfront along with $80 million in warrants to purchase common stock in exchange for the drug, called telitacicept. Data from that trial is expected in 2027. “I You can unsubscribe at anytime. Telitacicept is in Phase 3 testing for generalized myasthenia gravis in the U.S., TechTarget, Inc.s
By: Nathan Chadwick, Therapeutic Strategy Lead, Rare Disease Over the last few years in clinical trials, particularly within the rare disease community, a notable shift is underway, where patients and caregivers are taking the lead in reaching out to clinical trial sites rather than the other way around.
Deep Dive Library Events Press Releases Topics Sign up Search Sign up Search Pharma Biotech FDA Clinical Trials Deals Drug Pricing Gene Therapy An article from Merck to buy Verona and its lung drug in $10B deal The New Jersey-based pharma has inked one of the biggest deals of the year in pursuit of a promising new way to treat COPD.
Incyte expects multiple pivotal trial readouts this year, along with proof-of-concept data for several pipeline candidates. New data visualizations published by BioPharma Dive capture the year-to-date funding trends for 2025, which so far includes a significant pullback in financings for cancer drugdevelopers.
Daily Dive M-F Commercialization Weekly Every Wednesday Gene Therapy Weekly Every Thursday Emerging Biotech Weekly Every Tuesday By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. You can unsubscribe at anytime. Informa PLCs registered office is 5 Howick Place, London SW1P 1WG. Registered in England and Wales.
In a recent webinar, Challenges of DrugDevelopment in Early Parkinsons Disease , Tom Babic, MD, PhD , our Vice President of Scientific Solutions in Neuroscience, uses his 40+ years of experience to share invaluable insights into the current state and future directions of early Parkinsons disease (PD) research.
Retail Pharmacies : Retail pharmacies present significant challenges in the PA-to-coverage conversion process. Daily Dive M-F Commercialization Weekly Every Wednesday Gene Therapy Weekly Every Thursday Emerging Biotech Weekly Every Tuesday By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. inhibitors.
Solid tumors present a significant challenge to clinical research due to their complex and heterogeneous nature. Have you optimized your trial designs to employ precision medicine approaches for your novel solid tumor intervention?
When researchers gave people willow bark extract corresponding to 240 mg of salicin, then looked at how much salicylic acid was present in their blood over time, it was the equivalent of taking 87 mg of aspirin ( 300 mg to 600 mg is recommended per dose, with up to 3600 mg allowed per day).
Thats why Worldwide Clinical Trials was committed to ensuring the rare disease community still had a space to share knowledge, resources, and support on Rare Disease Day 2025. We welcome opportunities to engage with the rare disease community and help contribute to the conversation between advocates, researchers, drugdevelopers, and more.
Following the American College of Cardiology (ACC) conference 2025, Worldwide Clinical Trials hosted a post-meeting webinar, On the Cutting Edge of Cardiovascular Discovery Learnings from ACC 2025 and Future Perspectives. The trial additionally expressed a 15.5%
The event also featured a presentation from Dr. JP Clancy, CFF VP of Clinical Research, about the latest medical and scientific advancements to treat cystic fibrosis. Understanding Cystic Fibrosis According to the CFF , cystic fibrosis is a progressive, genetic disease that affects the lungs, pancreas, and other organs.
Applying CAR T-cell therapy in new clinical settings presents unique challenges. Worldwide Clinical Trials is at the forefront, partnering with drugdevelopment teams to map this new path forward.
We organize all of the trending information in your field so you don't have to. Join 15,000+ users and stay up to date on the latest articles your peers are reading.
You know about us, now we want to get to know you!
Let's personalize your content
Let's get even more personalized
We recognize your account from another site in our network, please click 'Send Email' below to continue with verifying your account and setting a password.
Let's personalize your content