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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.
However, recent breakthroughs in AI, such as predictive modelling, clinical trial optimisation, and personalised medicine, have demonstrated its potential. We spoke with Aaron Smith, a mathematician-turned-machine learning scientist and the founder of Unlearn , a company leading the charge in applying AI to optimise clinical trial efficiency.
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.
A surrogate endpoint is a marker used in clinical trials as a substitute for a direct clinical outcome. For example, transcriptomic processes are showing the potential to identify and track failures in gene expression and gene regulation of amyloid and tau-related biomarkers, understood as precursors to the onset of Alzheimers disease (AD).
As the CEO of iOnctura, an innovative oncology biopharmaceutical company she co-founded in 2017, Catherine has played a key role in advancing the development of highly targeted small molecules aimed at revolutionising cancer treatment. Additionally, we are preparing to launch several other trials over the coming months.
Neuropsychiatric treatment is on the verge of a major transformation. Neuropsychiatric disorders, affecting millions worldwide, disrupt the brain’s intricate processes of mood regulation, cognition and behaviour. “Zelquistinel is a positive modulator of NMDA receptors,” Donello explains.
An innovative stem cell-based treatment for Type 1 diabetes can meaningfully regulate blood glucose levels and reduce dependence on daily insulin injections, according to new clinical trial results from the University of British Columbia (UBC) and Vancouver Coastal Health (VCH).
By: Christine Moore, PhD, Vice President, Neuroscience, Scientific Solutions Patients with borderline personality disorder (BPD) face a heightened risk of substance abuse and suicide, as well as substantial delays in receiving treatment.
Though they do positively impact patients’ quality of life and hemodynamic parameters, these treatments — prostacyclin analogs and receptor agonists, phosphodiesterase 5 inhibitors, endothelin-receptor antagonists, and cGMP activators — have shown limited beneficial effects on survival and disease progression.
Developing treatments for individuals living with rare diseases is critical, but orphan drug development is laden with unique obstacles that necessitate innovative, multifaceted approaches. This limited pool poses significant barriers to conducting statistically significant studies and validating the efficacy and safety of new treatments.
Authors: Matt Cooper, PhD, Executive Director, Therapeutic Strategy Lead, Oncology; Megan Morrison, Vice President, Asia Pacific Strategy Lead Adaptive trial designs have become essential in oncology, offering a flexible and efficient approach for conducting clinical trials.
Despite their exciting potential, the smooth operation of cell therapy development trials 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.
These therapies have broadened treatment options for patients to expand beyond the more traditional small molecule drug alternatives. ADCs have the potential to redress the poor balance between safety and efficacy seen with traditional cancer treatment options. 3D rendering of Antibody Drug Conjugate Molecules.
Revolutionizing Treatment Through the Skin with Transdermal Drug Delivery Systems The human skin, often perceived as a passive barrier to the external environment, holds potential as a route for drug administration. This membrane regulates the diffusion of the drug from the reservoir into the skin.
By: Sue Batchelor, Executive Director, Oncology Project Management Radiopharmaceuticals represent a cutting-edge frontier in oncology treatment, offering the promise of highly targeted therapy with the potential to revolutionize cancer care. Are you aware of the challenges you must address for a successful radiopharmaceutical trial?
Obesity treatment is undergoing a major shift, much like the advances seen in cancer care. Phenomix Sciences , led by CEO Mark Bagnall, is using precision medicine to make treatments more targeted and effective. Phenomix Sciences, built on over a decade of clinical research at the Mayo Clinic, is disrupting this outdated approach.
The Buzhong Yiqi decoction regulated the PI3K-AKT signaling pathway in non-small cell lung cancer A549 cells. The Buzhong Yiqi decoction (BZYQD), traditional Chinese medicine (TCM) formula, has been reported to exhibit clinical efficacy in the treatment of NSCLC. Nevertheless, the underlying molecular mechanism remains elusive.
Patients are the backbone of clinical trials, playing an essential role in the drug development process. This engagement is often less understood and is underutilized by sponsors, meaning a significant element of the trial and drug experience is missed during sponsor engagement with the FDA.
Tobolowsky & Véronique Li, Senior Medical Device Regulation Expert & David B. Moreover, DMCs are being used in trials of modest size and in the context of increased globalization of medical product development. In another update, the recent draft guidance added “entities reviewing safety data” and adaptation committees.
For more than 35 years, Advarra has been committed to protecting the rights and welfare of clinical trial participants while helping to improve healthcare outcomes, advancing medical knowledge, and bringing innovative, life-extending treatments to market that benefit millions of patients worldwide.
In China, the high incidence of solid tumors is driving an urgent need for advanced therapies, spurring the push for new treatment approaches. There has also been an increase in government and regulatory support for CGT trials, in conjunction with an increase in investments for these products to get to market.
Cost and complexity go hand-in-hand The rising costs and growing complexity in clinical trials are deeply linked, with patient recruitment, extended timelines and meeting regulatory demands emerging as some of the key drivers. Nearly half (49%) of clinical trial sponsors surveyed identified rising costs as their foremost concern in 2024.
At the recent World Orphan Drug Congresses (WODC) in Europe and the United States, Worldwide Clinical Trials Derek Ansel , Vice President, Therapeutic Strategy Lead, Rare Disease, facilitated roundtable discussions to explore the operational challenges and ethical barriers surrounding genetic testing.
However, limited treatment options are available for those with recurrent disease, and there is a need to identify alternative treatment options for the advanced setting. Advanced-stage endometrial cancer patients typically receive a combination of platinum and paclitaxel chemotherapy.
This process can be daunting, but understanding how to manage feedback effectively is crucial for developing and ultimately gaining approval for new therapies, especially in oncology clinical trials. Additionally, consider the locations of your trial; if it is multiregional, you can start planning for your overall product approval strategy.
Contract research organizations (CROs) and pharmaceutical companies can leverage these cutting-edge technologies to streamline clinical trials and introduce automation in drug discovery. As clinical trials grow in complexity, the volume of data being gathered and utilized for these studies is expanding.
Rapid and substantialimprovement in depressive symptoms achieved by 44% of patients at 2 weeks, 67% at 6 weeks (MADRS response), and sustained with long-term treatment.
points at Week 6 (primary timepoint), with AXS-05 treatment. points at Week 6 (primary timepoint), with AXS-05 treatment.
NEW YORK, Dec.
BRAF and downstream mitogen-activated extracellular signal-regulated kinase (MEK) inhibitors are combined to treat BRAF-mutated melanoma, for example. Classically, rational drug combinations are designed to target two nodes in the same oncogenic signalling pathway.
Logistics For Cell & Gene Therapy Trials: Specific Needs Demand Special Skills Download now to learn more about intricate trial logistics, the importance of expertise, navigating regulations, secure data management, and real case studies like viral vector therapy for Spinal Muscular Atrophy. A specialized provider is essential.
Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.
Clinical trial data management is critical to pharmaceutical research, yet it remains a significant challenge for many organizations. Outdated systems: Many organizations rely on legacy data management tools that fail to meet the demands of modern clinical trials.
Involved in various physiological processes, such as vision, taste, smell, immune response and neurotransmission, GPCRs are activated by various molecules including hormones, neurotransmitters and environmental stimuli, which trigger a cascade of cellular events that help regulate bodily functions.
By: Sarah Bly, Regulatory Science and Innovation and Matt Cooper, Executive Director, Therapeutic Strategy Lead, Oncology The European Union (EU) presents a unique set of regulatory challenges and opportunities for clinical trials in oncology. Increased Transparency : Ensures that information on clinical trials is more accessible.
The increased adoption of decentralized clinical trial (DCT) designs has revolutionized how the industry approaches clinical studies. In short, for clinical trial design to truly benefit from the digital revolution, data flow, management and analysis are the next frontiers.
Food and Drug Administration (FDA) issued draft guidance, Clinical Trial Considerations to Support Accelerated Approval of Oncology Therapeutics , regarding clinical trial design specific to oncology with considerations to support accelerated approval applications.
SH ) submitted its pivotal phase III clinical trial application (IND) of national class I innovative drug Chiauranib to NMPA, for the treatment of Small Cell Lung Cancer (SCLC) as a single agent for the patients after 2nd-line systemic chemotherapy and recurrence afterwards. SHENZHEN, China , Jan.
PROs in clinical trials are important as they capture the patient’s perspective and ensure that the impact of an intervention is comprehensively evaluated. What are PROs in clinical trials? From a regulatory standpoint, agencies like the U.S. These aspects influence a patient’s perceived health-related quality of life (QoL).
The World Health Organization (WHO) defines clinical trials as a type of research that studies new tests and treatments and evaluates their effects on human health outcomes (1). Clinical trials […]. The post Clinical Trial Approval Process in Australia appeared first on ProRelix Research.
Clinical trials are necessary in advancing medical research and health care options by allowing new treatments to be introduced in the market through safety and efficacy testing in humans. Successful […] The post Clinical Trials and Human Subject Protection as per US FDA appeared first on ProRelix Research.
Managing clinical trial budgets efficiently is necessary for the success and sustainability of clinical research sites. Effective budget management not only ensures trials are financially viable but also maximizes return on investment (ROI). the impact and value of the data produced).
Clinical trials are research studies in which participants volunteer to test new treatments, therapies, or tests in the hopes of preventing, detecting, treating, or managing a wide range of diseases […]. The post Advantages of Conducting Clinical Trials in Australia appeared first on ProRelix Research.
Food and Drug Administration (FDA) has designated as a Fast Track development program the investigation of Brilacidin as a potential treatment for COVID-19. A molecular screening study of 11,552 compounds also supports Brilacidin as a promising novel coronavirus treatment. WAKEFIELD, Mass.,
SRP-001 targets the central nervous system (CNS) by producing N-arachidonoylaminophenol (AM404) in the midbrain’s periaqueductal grey (PAG) region, crucial for pain sensation and regulation. In Phase I clinical trials, SRP-001 showed no serious adverse events among 56 healthy volunteers, highlighting its safety.
FDA Approves Danyelza (naxitamab-gqgk) for the Treatment of Neuroblastoma. the “Company” or “Y-mAbs”) (Nasdaq: YMAB) a commercial-stage biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer, today announced that the U.S. NEW YORK, Nov.
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