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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.
As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. A surrogate endpoint is a marker used in clinical trials as a substitute for a direct clinical outcome.
In an era where clinical trials are increasingly global, it’s more imperative than ever to leverage international expertise. Data and safety monitoring boards (DSMBs), also known as data monitoring committees (DMCs), play a critical role in overseeing a clinical trial’s safety and efficacy.
In the realm of vaccine development, mega trials — studies enrolling 5,000 subjects or more — have been instrumental in the fight against many pathogens, including influenza, rotavirus, malaria, RSV and most recently in the rapid development of vaccines against COVID-19.
1] [2] Adverse effects The US Food and Drug Administration prescription label for fitusiran contains a boxed warning for thrombotic events (blood clotting) and gallbladder disease (with some recipients requiring gallbladder removal). [2] Fitusiran 1711.0g/mol, 1] It is an antithrombin-directed small interfering ribonucleic acid. [1]
Rare Disease Innovation & Partnering Summit. Hybrid Event May 17-19, 2022 | Boston, MA www.informaconnect.com/rare. Don’t miss the Rare Disease Innovation & Partnering Summit, coming up May 17-19, 2022 in Boston, MA. Visit www.informaconnect.com/rare for further details and to register. d/b/a Drug Channels Institute.
“Patient-centric” is used frequently to describe today’s clinical trial and health care landscape. The industry applauds that point of view and strives to include a patient-centric approach in all aspects of clinical trial designs. Education on the disease state and regular reinforcement.
The figure depicts the neuroprotective role of crocin in Parkinson's disease. Abstract Parkinson's disease is among the most common forms of neurodegenerative illness, with present treatment being primarily symptomatic and frequently coming with substantial adverse effects.
Pulmonary arterial hypertension (PAH) is a progressive and fatal lung disease that is caused or influenced by multiple factors. Their early definition — as well as plans for recording and tracking — is a major factor in a trial’s success. Historically, the available drugs and U.S. None offers a cure for PAH.
Ladders to Cures (L2C) Accelerator By Maria Nemchuk November 25, 2024 Breadcrumb Home Ladders to Cures (L2C) Accelerator The Ladders to Cures (L2C) Accelerator aims to catalyze progress across the research ecosystem and accelerates advances leading to treatments and cures for patients with rare genetic diseases.
In this podcast interview, Dan Fisher, managing director and practice lead for IPM.ai, explains the use of artificial intelligence for stitching together key medical events that may happen over a r | IPM.ai helps brands assess a unique patient population, accelerate clinical trial recruitment, and optimize commercial outcomes
The Untitled Letter notes that the confirmatory trial for LYTGOBI is currently ongoing and has not been completed; therefore, clinical benefit of LYTGOBI has not yet been confirmed. As a single-arm trial (i.e., DCR is defined as the sum of complete response (CR), partial response (PR) and stable disease (SD).
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.
For neurodegenerative disease research, the care partner, or caregiver, is equally important as the person with dementia. Even with stronger awareness of studies, drug developers must contend with the inherent lack of trust between the dementia community and clinical trials. There’s little time to lose.
Positive high-level results from the FoCus Phase III trial in Wilson disease showed ALXN1840 met the primary endpoint with a statistically significant improvement in daily mean copper mobilisation from tissues, demonstrating superiority compared with standard-of-care (SoC) treatments.
95% of rare diseases don’t have a treatment approved and won’t anytime soon. Over the next few decades, that drug made its way through clinical trials, securing approval in 2007—just 36 months before Rob was diagnosed in 2010. Uplifting Athletes (UA) is on a mission to change that. But what was truly remarkable?
By adjusting the activity of this receptor, these drugs can influence brain functions associated with mood, cognition and pain, positioning them as potential treatments for conditions like depression, cognitive disorders and neurological diseases. “We call this phenomenon ‘event-driven pharmacology.”
Brain disorders are difficult to study and many drug candidates have failed in clinical trials, causing pharmaceutical companies to reduce their investments or even exit the field entirely. Without robust biomarkers, we cannot account for this mechanistic diversity and appropriately match patients with candidate treatments in clinical trials.
functions to reset the immune system and potentially provide remission for allergic diseases? In allergic diseases, APCs identify and present unknown antigens to the immune system. This can lead to inadequate long-term effectiveness with low rates of disease remission. They also require frequent administration.
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.
Assessing and reporting adverse events (AEs) in clinical trials is critical to ensuring the study is as safe as possible and the participants have the most up to date information so they can decide whether to continue their participation in the research study. Who Should Assess AEs in Clinical Trials? regulations.
As one of the more recent fields to emerge in the clinical space, psychedelics have gained significant traction as a potential treatment for various neuropsychiatric diseases. Psychedelic Research at Worldwide Since 2017, Worldwide Clinical Trials have supported a myriad of sponsors leading the way in psychedelic research programs.
By harnessing the vast amounts of data generated throughout the development pipeline, pharmaceutical companies can accelerate the discovery of novel therapies, optimize clinical trial design, enhance drug safety monitoring, and deliver personalized medicine, ultimately improving patient outcomes and transforming the future of healthcare.
Real-world data paired with machine learning is a game changer in drug development RWD is becoming increasingly important in influencing the drug development landscape, particularly when used to develop the natural history and patient journey through disease. AI is also proving useful in drug repurposing or indication selection.
The landscape of clinical research is undergoing major changes with virtual clinical trials driving these transformations. These innovative trials promise to revolutionise the way medical research is conducted, making it more accessible, efficient, and patient-centric than ever before.
In practice, some patients may be left managing a syndrome associated with the therapy along with the disease. 2 However, when dosed at the MTD, ADCs display improved efficacy over small molecules in oncology trials. 2 However, when dosed at the MTD, ADCs display improved efficacy over small molecules in oncology trials.
recent announcements from Merck and Sanofi for obesity and broader metabolic disease). Still may be some merit if they are clinical-stage and can achieve once-daily dosing without too steep a cut in efficacy or worsened adverse events vs. peptides, but be wary of the intensely competitive landscape. Additional trials (e.g.,
AI also has the potential to incorporate real-world data (RWD) obtained from electronic health records (EHRs), medical claims or other data sources to inform the design and optimization strategy of clinical trials. A high-risk participant can be even excluded from the study based on the severity of the adverse event.
Phase III BRIDGE open-label, switch-over clinical trial met key objectives for safety and efficacy.
galactosidase-A product candidate under development for the treatment of Fabry disease. PRX-102 was well-tolerated in the study, with all adverse events being transient in nature without sequelae.
18, 2021 /PRNewswire/ — Aruvant Sciences, a private company focused on developing gene therapies for rare diseases, and Lonza announced today their agreement in support of ARU-1801, Aruvant’s one-time investigational gene therapy for sickle cell disease (SCD).
NEW YORK , Jan.
For others, this rare day is aptly the day we honor rare disease communities. Observed annually on the last day of February since 2008, Rare Disease Day has grown into a global movement for raising awareness, promoting research, and advocating for improved access to rare disease treatments and support services.
Target validation : Do we believe the target(s) plays a central role in disease biology and that modulation will modify disease? There is nothing more exciting than digging into a new target and trying to develop a thesis on whether modulation may be impactful in disease. Is the functionality of mutations known?
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.
Clinical trials for ultra-rare diseases can be particularly challenging to mount due to small, geographically-dispersed patient populations. For such trials, the US Food and Drug Administration (FDA) may allow the use of credible real-world data (RWD) and real-world evidence (RWE) in lieu of data collected in a Phase 3 trial.
But what does “working” really mean when efficacy means something subjective, like quality of life, reduced psychiatric events, or less symptoms? In most cases, the results you are really looking for and trying to prove in a trial can be complex. All key questions to consider and address before the therapy even leaves the lab.
A number of areas represent significant opportunities for Antibody Drug Conjugates (ADCs) beyond oncology, leveraging their ability to deliver therapeutic agents specifically to diseased cells or tissues while minimising off-target effects. Oncology also offers high returns and the opportunity to address severe, life-threatening conditions.
Rare Disease Day on February 28th is a chance for rare disease communities to come together as one and join in conversation with stakeholders searching for treatments, raising money to support research, and changing the way healthcare and governments interact with patients. In my case, my daughter Iris has GM1 gangliosidosis.
By: Lisle Kingery, PhD, Therapeutic Strategy Lead, Neuroscience With depression rates at an all-time high and limited new treatments available, depression clinical trials are more important than ever. Treatment Treatment can take various forms, including therapy, medication, a combination of both, or a clinical trial.
Among others, NGS has led to the identification of disease-causing variants and novel drug targets and an improved understanding of complex biological events, e.g., the heterogeneity of tumors. 2022) recommend matching interventions to specific patient characteristics should be implemented early in the disease course.
Sanofi and Translate Bio initiate Phase 1 clinical trial of mRNA influenza vaccine. The trial will evaluate the safety and immunogenicity of a monovalent flu vaccine candidate coding for the hemagglutinin protein of the A/H3N2 strain of the influenza virus. JUNE 22 , 2021. We look forward to sharing initial results by year-end. ”. “
Novartis today announced positive results from two Phase III clinical trials assessing Beovu ® (brolucizumab) 6 mg versus aflibercept 2 mg in patients with diabetic macular edema (DME). Both trials demonstrated an overall well-tolerated safety profile. 5%) overall adverse events were cataract and dry eye. for Beovu vs. 1.7%
Novo Nordisk today announced headline results from the ONWARDS 2 trial, a phase 3a, 26-week efficacy and safety treat-to-target trial investigating once-weekly insulin icodec vs insulin degludec in 526 people with type 2 diabetes switching from daily insulin. events per patient year exposed to once-weekly insulin icodec and 0.27
Of 202 treated participants, no safety signals have been identified and of the 4 serious adverse events reported, none were considered to be study drug related. Headquartered in Miami, Florida, Ridgeback Biotherapeutics LP is a biotechnology company focused on emerging infectious diseases. About Ridgeback Biotherapeutics.
Described in Nature , these ‘brain chimeroids’ provide an approach for studying how genetic background can affect the outcome of neurotoxic exposures, disease-associated genetic variants, and potential therapeutic compounds. This could lead to more accurate preclinical trials and better predictions of clinical outcomes.
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