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In an article published online ahead of print , leaders from the NIH Pragmatic Trials Collaboratory share lessons learned about the importance of independent oversight by a safety office or data and safety monitoring board in pragmatic clinicaltrials, even for trials deemed to have minimal risk.
Clinicaltrials are the foundation of medical innovation, paving the way for groundbreaking therapies. Yet for many, the idea of joining a trial can feel intimidating or unclear. Have you ever wondered how life-saving treatments for cancer, Alzheimers, or chronic diseases come to life?
Despite advancements in neuroscience and psychiatry, clinicaltrials for mental health conditions such as major depressive disorder (MDD), schizophrenia, bipolar disorder and generalized anxiety disorder face unique challenges. One of the main challenges lies in defining and selecting appropriate trial populations.
Researchers with PRIM-ER, an NIH Collaboratory Trial, published 2 innovative statistical techniques for evaluating intervention effects in stepped-wedge, cluster randomized trials. In stepped-wedge trials, the clusters are randomized into several groups, and all groups start the trial in the control condition.
Clinicaldevelopment organizations face a wide array of challenges when it comes to data, many of which can impact the operational effectiveness of their clinicaltrials. In this whitepaper, experts from Revvity Signals explore how solutions like TIBCO® Spotfire® enable better, more streamlined studies.
Global rare disease clinicaltrials face unique challenges due to small patient populations, complex logistics, diverse regulatory environments and cultural differences. ClinicalTrial Manager/Sr. These measures enhance recruitment, retention and overall trial success.
The surge in MASH cases stresses the need for early diagnosis, timely intervention and precise, reliable methodologies in clinicaltrials to evaluate new therapies effectively. Read on to gain insights from Medpace experts about advancements in imaging, histology and innovative techniques for MASH clinicaltrials.
Over the past two decades, industry-sponsored clinicaltrials have targeted treatments for BPD, yielding some promising outcomes; however, broader psychiatric research often excludes BPD patients, a trend that extends to the emerging field of psychedelic studies.
This is known as negative spillover, or crowding out, in the field of development economics. However, spillover due to resource constraints is absent from the major frameworks that inform clinicaltrial methods. These conditions are fairly common in pragmatic trials. Trials 25, 833 (2024).
Clinical research has entered a new era, one that requires real-time analytics and visualization to allow trial leaders to work collaboratively and to develop, at the click of a mouse, deep insights that enable proactive study management.
The NIH Pragmatic Trials Collaboratory will hold a virtual workshop on February 5 and 6, 2025, on Digging Into Dilemmas of Pragmatic ClinicalTrials. To date, the NIH Collaboratory has launched 35 pragmatic trials that are conducted within healthcare systems at over 1,400 clinical sites.
Designing the Future of Biosimilars: Strategies for Success As the biosimilar market continues to grow, pharmaceutical companies are facing increasing pressure to develop high-quality, cost-effective treatments that meet the evolving needs of patients and healthcare systems. What are your thoughts on the future of biosimilars?
Early Phase Unit, Worldwide ClinicalTrials Neurodegenerative diseases represent a diverse group of progressive disorders characterized by the loss of structure and function of neurons. Although PET tracers for -syn are in development, they are not yet available for clinical use.
The NIH and the Patient-Centered Outcomes Research Institute (PCORI) are major funders of pragmatic clinicaltrials embedded in healthcare systems. An existing chapter of the Living Textbook of Pragmatic ClinicalTrials provided guidance on how to Develop a Compelling Grant Application for the NIH.
As the development pipeline for new drugs continues to grow, biopharmaceutical companies are re-evaluating how to best manage and balance resources across an increasing number of development projects and complex clinicaltrials.
The therapy's developer, Brainstorm Therapeutics, blamed the setback on better-than-expected results from placebo-treated patients. The biotech still sees a path forward, however.
The agency proposes that pharma move away from the common maximum tolerated dose (MTD) to trial designs that prioritise drug dosage optimisation at different stages. In clinicaltrials, dose-ranging is testing different drug doses against each other to establish which works best.
The Indian Council of Medical Research (ICMR) and Panacea Biotec have announced the initiation of the first-ever phase 3 clinicaltrial for a dengue vaccine in India. The trial will evaluate the efficacy of India’s indigenous tetravalent dengue vaccine, DengiAll, developed by Panacea Biotec.
Keith Goldfeld, principal investigators for PRIM-ER An evidence-based training program to improve the capacity of emergency department care teams to communicate with seriously ill older patients about palliative care did not lead to lower rates of hospital admission, according to the results of the PRIM-ER trial. Learn more about PRIM-ER.
Health Data Research Network (HDRN) Canada is now accepting applications for its Pragmatic Trials Training Program. There will be an emphasis on experiential learning for future trial leaders and postdoctoral fellows in the program through the development of a pragmatic trial protocol.
By Luisa Sterkel & Joana Loureiro , Tenthpin Consultants The promise and potential of cell and gene therapies (CGT) has emerged in the recent past and currently over 1.500 CGT are registered for clinicaltrials holding great hope for the treatment of challenging and uncurable diseases.
We have seen how advanced software platforms empower companies across many sectors to develop personalised applications that deliver seamless customer experiences. Now, forward-thinking clinical technology providers are seeing an opportunity to apply these back-end approaches to advance software in the healthcare industry.
It’s even being used to review investigator invoices and automate financial reconciliations—a monthly headache every contract research organization, sponsor, and clinicaltrials operator will be more than happy to resign to machines. MGI puts the value of this contribution to clinicaldevelopment at $15 billion to $25 b illion.
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.
In clinical research, diversity is more than a prerequisite for a successful drug — it’s a critical component that enhances the validity and applicability of clinicaltrial outcomes. Diversity Action Plans have now moved from being a “nice-to-have” to a “must-have” in clinicaltrial designs.
Critical infrastructure in biopharma and medical devices plays a vital role in enabling innovation, bolstering research and development, and ensuring the production of novel drugs and products. It ensures scalability, quality control, and regulatory compliance.
The recent regulatory developments to relax clinicaltrial norms in India are poised to have a significant impact on the pharmaceutical industry and drug pricing. By easing regulations, Indian pharma could see a reduction in the costs associated with human studies.
Following on from its Covid-19 vaccine programmes, BioNTech has set its sights on a range of infectious diseases for vaccine development. The company saw major successes with its Covid-19 vaccine, developed in collaboration with Pfizer. In December 2022, BioNTech initiated a Phase I clinicaltrial of BNT163 – an HSV vaccine candidate.
BlueRock Therapeutics, a subsidiary of Bayer AG, is making waves in the treatment of Parkinsons disease by advancing its investigational cell therapy, bemdaneprocel, to a Phase III clinicaltrial. These stem cells are engineered to develop into dopamine-producing neurons, which are progressively lost in Parkinsons disease.
In a recent webinar, Challenges of Drug Development 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.
Velocity Clinical Research, the leading multi-specialty clinical research organization, has partnered with Verified ClinicalTrials (VCT) to set a new benchmark for patient safety and data quality in clinicaltrials through a new technological implementation at Velocitys 80+ global sites.
Early-phase clinicaltrials, including phase 1 and some phase 2a studies, serve as a pivotal step for biotech companies, laying the foundation for a drug candidate’s journey to market.
Fortunately, advances in clinical research are providing hope for better treatments and outcomes. With the support of global networks like ours at Worldwide ClinicalTrials (Worldwide), autoimmune and bone health research is accelerating, providing new solutions to patients around the world.
Tom Zhang, PhD, Chief Scientific Officer, Large Molecule Bioanalysis Revolutionizing Therapeutic Development with Cutting-Edge Bioanalysis Automation Cutting-edge advancements in bioanalysis (BioA) and drug R&D are reshaping the future of preventative and interventional care, but how do we harness these to their fullest potential?
Speaking at the NIH Pragmatic Trials Collaboratory’s 2024 Annual Steering Committee Meeting in May, Commissioner of Food and Drugs Robert Califf and NIH Director Monica Bertagnolli discussed the intersection of pragmatic research and national health priorities. However, several barriers hinder the development of such a system.
Fortunately, advances in clinical research are providing hope for better treatments and outcomes. With the support of global networks like ours at Worldwide ClinicalTrials (Worldwide), autoimmune and bone health research is accelerating, providing new solutions to patients around the world.
An unusual collaboration among gene therapy developers suggests certain mutations could be behind "peculiar" side effects experienced by several patients treated in clinicaltrials.
Regulatory bodies such as the FDA oversee clinicaltrials to ensure that studies’ design, conduction, analysis, and reporting are per established guidelines and laws. Bioanalysis to support drug development requires meticulous attention to detail across many clinic and laboratory disciplines.
Updated study snapshots and ethics and regulatory documentation are now available for the AIM-CP, ARBOR-Telehealth, and RAMP trials. The post February 24, 2025: Study Snapshots and Updated Ethics Documentation Available for 3 NIH HEAL InitiativeSupported Trials in Rural Populations appeared first on Rethinking ClinicalTrials.
Clinical-stage biopharmaceutical company TORL BioTherapeutics has raised $158m in a Series B financing round for advancing the development of new biologics for cancer treatment. TORL BioTherapeutics is a newly formed, US-based company focused on the development of novel antibody-based therapeutics for cancer patients.
Cyagen and Neurophth Therapeutics have entered a strategic partnership to jointly develop next-generation AAV gene therapy vectors for specific kinds of genetic ophthalmic ailments. Additionally, Neurophth will oversee the clinicaltrials and marketing of gene therapy products developed leveraging the new AAV capsids of Cyagen.
German pharmaceutical firm Merck has extended its partnership with Moderna to jointly develop and sell mRNA-4157/V940, an investigational personalised cancer vaccine (PCV). In 2016, the companies entered a strategic partnership to develop novel messenger RNA (mRNA) based PCVs.
The year ahead in clinicaltrials looks set to be one that is challenged and influenced by regulatory developments, new guidance, and broader political shifts.
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