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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 clinicaltrials to fund to help sponsors pursue development of medical products for rare diseases and advance their field.
In just two years, CTMC has advanced eight therapies into clinicaltrials, harnessing genetic engineering to enhance T-cell effectiveness in the fight against cancer. Ive been involved in therapeutics development for over 25 years, working with small, medium, and large biotech companies.
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.
Like many aspects of life in the 21st century, this era of scientific advancement and increased partnership is largely driven by next-generation technologies that are streamlining workflows, breaking down silos and empowering scientists to extract the most prescient insights from their data throughout the drug development process.
Neuropsychiatric treatment is on the verge of a major transformation. Historically, treatment options have been limited, with patients relying on daily medications that have minimal efficacy and troublesome side effects. “Zelquistinel is a positive modulator of NMDA receptors,” Donello explains.
More than 7,000 rare diseases – conditions that affect fewer than 1 in 2,000 people – have been identified, and1 in 17 people will develop a rare disease in their lifetime, with children disproportionately affected. Most of these conditions are genetic in origin and the majority have no effective treatment.
2 Unmet needs in lung cancer treatment Recent decades have seen significant advancements in lung cancer treatment, especially with the introduction of targeted therapies and immunotherapies, which have notably improved survival rates. 18 Indeed, early-phase clinical studies exploring TsAbs in solid tumours have recently commenced.
Developingtreatments for individuals living with rare diseases is critical, but orphan drug development 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.
Authors: Rich Worldwide ClinicalTrials Exec. This trend is on the rise despite recent disappointments with clinicaltrial outcomes, which have the potential to destabilize the industry in the short term regarding drug development strategy and optimal study designs. Director, Therapeutic Area Medical Lead.
Patients are the backbone of clinicaltrials, 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.
Despite significant progress, there are more than 6,500 rare diseases without a single approved treatment. As a provider of end-to-end clinicaldevelopment solutions, we plan to leverage our expertise to refine drug-disease matches identified by Every Cure’s use of AI and data analysis.
Written By: Derek Ansel, MS, CCRA, Executive Director, Therapeutic Strategy Lead, Rare Disease Given that 80% of rare diseases have a genetic etiology, genetic implications should be addressed at the onset of a clinical program to support trial enrollment. One diagnostic example that I discussed in my presentation is autism.
Regulatory bodies such as the FDA oversee clinicaltrials to ensure that studies’ design, conduction, analysis, and reporting are per established guidelines and laws. Any delays or missteps in bioanalysis during a Phase I trial can derail the trajectory of a promising drug.
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 clinicaltrials.
To keep a pulse on this rapidly changing industry, the PPD clinical research business of Thermo Fisher Scientific surveyed 150 leaders in the biopharma and biotech industries for the second consecutive year. Differences in size, resources and agility have led the two groups to take different approaches to clinicaldevelopment.
Instead of the standard approach where studies are designed to address a primary focus, a parallel study method — a format that incorporates multiple endpoints and objectives, modifications, treatment arms and dose selections into a single protocol — is being rapidly adopted.
Clissold — The trio of CDER, CBER, and CDRH released a new draft guidance titled “ Use of Data Monitoring Committees in ClinicalTrials ” that revises the 2006 guidance “Establishment and Operation of ClinicalTrial Data Monitoring Committees” and, when final, will replace the 2006 guidance.
Bicara has focused ficerafusp alfa clinicaldevelopment on head and neck squamous cell carcinoma (HNSCC), adding pembrolizumab to separately block the PD-1 pathway. No chemotherapy regimen is used in the trial. Another clinical program that has making headlines in HNSCC is petosemtamab, an anti-EGFR x LGR5 bispecific antibody.
FXR-targeted drugs have shown promise in late-stage clinicaltrials for non-alcoholic steatohepatitis. Cilofexor is one of few remaining FXR agonists in clinicaldevelopment. The farnesoid X receptor (FXR) is a nuclear receptor that controls bile acid, lipid, and cholesterol metabolism.
Clinicaltrial success is a key factor for pharma companies when designing and recruiting patients into trials. This data can also provide guidance in terms of diversifying clinicaltrials to include different types of population demographics such as age, gender, race, ethnicity, or geographic location.
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.
A review by the external Data Monitoring Committee (eDMC) determined that this effect was related to treatment with the combination of ISL08507; the greatest decreases were seen in the arms of the study receiving the highest doses of MK-8507 (200 mg and 400 mg). The company has notified investigators and paused development of MK-8507.
Author: Lona Sheeran, SVP, Clinical Operations, Early Phase At this year’s ClinicalTrials Nexus, I had the privilege of representing Worldwide ClinicalTrials as the sole CRO on a panel discussion: “Reversing the Conversation: What the ClinicalTrial Industry Really Wants from its Service Providers.”
In the rapidly evolving landscape of oncology drug development, artificial intelligence (AI) has emerged as a revolutionary force, altering not just the pace but the very methodology of discovery and development. What sets RADR ® apart is its robust data-driven approach.
to enhance outcomes, new treatments which will target kidney-specific disease mechanisms are highly desired,” said Hiddo L. Food and Drug Administration (FDA) supported the positive results of the FIDELIO-DKD phase III clinicaltrial study for patients with CKD and T2D.
A new treatment for G4-targeted solid tumors including pancreatic cancer has been administered to patients for the first time in a Phase 1a clinicaltrial. This latest development follows the US Food and Drug Administration (FDA) giving clearance for QN-302 in July 2023 to proceed to this initial clinicaltrial stage.
External Control Arms (ECAs) provide comparative evidence when recruiting patients is difficult or unethical in randomized controlled trials. ECAs have significant potential to save resources and accelerate access to innovative treatment.
However, accelerated approval comes with a condition: sponsors must conduct confirmatory trials to verify the drug’s anticipated clinical benefits using robust outcome measures. Concerns have arisen over delays—sometimes spanning over 7–8 years—that may expose patients to risks before confirmatory trials are completed.
As the strategists for Worldwide ClinicalTrials’ rare disease sponsors, we design customized solutions that make a difference in achieving successful outcomes. This philosophy is encapsulated in our belief: “If you’ve seen one trial for a rare indication, you’ve seen one trial.”
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.
Additionally, in 2021, a literature review estimated the average capitalized research and development (R&D) costs per new cancer medicine at between $944 million and $4.54 Typical clinicaldevelopment timelines for anticancer drugs average an estimated 6.7
Manguso, who’d recently graduated from college and was conducting research at the University of Copenhagen as a Fulbright scholar, moved back to the Boston area to be with his mother as she underwent treatment. By then, a few cancer immunotherapies had entered clinicaltrials and only a handful had been approved by the FDA.
This has aligned with the broader curation of a number of new dose-escalation and efficacy designs for clinicaldevelopment in oncology. A nuanced understanding of these considerations enables sponsors to ask more complex questions like:
In addition, as part of the label update, this indication has been revised to be for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who are not eligible for any platinum-containing chemotherapy. Source link: [link].
We are very pleased to share our initial Phase 2 infectivity data at this important conference, which remains at the forefront for critical clinical scientific information in infectious diseases,” shared Dr. Wendy Painter, Chief Medical Officer of Ridgeback Biotherapeutics. “At About Molnupiravir.
Read the Article Both of these studies reflect a global effort to understand the genetic underpinnings of disorders so people can find the right treatments sooner. In addition to benefiting awarded sponsors, this pilot program will help create best practices of communication with the FDA during rare disease therapeutic development.
Five Promising Treatment Areas in Early-Phase Drug Development in 2024 aasimakopoulos Wed, 04/17/2024 - 15:52 Early-phase drug development is an ever-changing landscape, as emerging science leads to new promising areas of research for the treatment of human health issues.
mg dose of Ozempic ® (once-weekly semaglutide subcutaneous injection), a glucagon-like peptide-1 (GLP-1) analogue for the treatment of adults with type 2 diabetes. mg doses for the treatment of type 2 diabetes in adults. The FDA approval is based on the results from the SUSTAIN FORTE trial. mg and 2.0
The primary efficacy outcome was met showing a statistically significant reduction in the 24-hour cough count (average hourly cough frequency based on 24-hour sound recordings) of up to 27% for eliapixant 75 mg twice daily over placebo after 12 weeks of treatment.
The high rate of unvaccinated people will continue to put a strain on hospitals and healthcare systems around the world, furthering the need for effective treatments for patients hospitalised with COVID-19,” said Levi Garraway, M.D., Chief Medical Officer and Head of Global Product Development, Roche. Actemra/RoActemra is not U.S.
Approval based on Phase 3 CheckMate -9ER trial results showing Opdivo in combination with Cabometyx significantly improved overall survival and doubled median progression-free survival and objective response rates compared to sunitinib. vice president, development program lead, genitourinary cancers, Bristol Myers Squibb.
Bayer’s established collaboration with the Broad Institute has already resulted in three clinical oncology candidates over the past decade. We look forward to continuing our work with renowned Broad scientists to advance additional innovative cancer targets into clinicaldevelopment.”
Sandoz, a Novartis division, today announced progress in the late-stage clinicaldevelopment program for its proposed biosimilar aflibercept. Sandoz will begin enrolling the first patient in MYLIGHT, a clinical Phase III confirmatory efficacy and safety study, shortly 1. Aflibercept is a key treatment in ophthalmology.
What challenges has the industry faced in developing and commercialising treatments for neurological diseases, including rare diseases? But commitment to neuroscience drug development is critically important given the significant unmet medical needs and the ways in which patients, caregivers, and families suffer.
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