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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?
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.
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.
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.
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. Finally, psychiatry clinicaltrials must also address the variability in treatment responses.
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.
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.
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?
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.
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. They recently published a paper in Trials that explores the issue in further detail: Mann, S.
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.
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 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.
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 post March 4, 2025: PRIM-ER Team Develops Innovative Statistical Techniques for Stepped-Wedge Trials appeared first on Rethinking ClinicalTrials. The study was supported by the National Institute on Aging. Learn more about PRIM-ER.
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.
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.
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.
However, Amgen and other pharmaceutical companies have been criticised when they opted to choose the drug’s dosage based on the MTD model, which is commonly used in dose-finding trials. In clinicaltrials, dose-ranging is testing different drug doses against each other to establish which works best.
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.
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.
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.
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.
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.
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.
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. Contact us today to learn more.
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.
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.
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.
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.
Umoja Biopharma has signed a research agreement with IASO Biotherapeutics (IASO Bio) to develop off-the-shelf therapies for haematological malignancies. The post Umoja and IASO partner to develop therapies for haematological malignancies appeared first on Pharmaceutical Technology.
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.
Ast ellas Pharma has announced plans to make a strategic investment to back the development of Taysha Gene Therapies’ adeno-associated virus (AAV) development programmes for Rett syndrome and giant axonal neuropathy (GAN). This would allow the companies to boost the development of new therapy options for Rett syndrome and GAN patients.
Aviceda Therapeutics (Aviceda) has entered into a strategic partnership with the Patrick G Johnston Centre for Cancer Research at Queen’s University Belfast (QUB) for the development of next-generation glyco-immune therapeutics. Aviceda Glycotech has been created as an oncology-focused spin-off company from Aviceda Therapeutics and Queen’s.
The NIH Pragmatic Trials Collaboratory will hold a virtual workshop on February 5 and 6, 2025, on Digging Into Dilemmas of Pragmatic ClinicalTrials. Recordings and materials will be archived on the NIH Pragmatic Trials Collaboratory’s website at rethinkingclinicaltrials.org.
An unusual collaboration among gene therapy developers suggests certain mutations could be behind "peculiar" side effects experienced by several patients treated in clinicaltrials.
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?
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.
Gilead company Kite has entered an international strategic partnership with Arcellx for the joint development and commercialisation of the latter’s T-cell therapy, CART-ddBCMA, to treat relapsed or refractory multiple myeloma patients. Additionally, Kite will make other potential payments to Arcellx.
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.
C4X Discovery (C4XD) and AstraZeneca have entered an exclusive global licensing agreement worth up to $402m to develop oral therapy to treat inflammatory and respiratory ailments. The deal has been signed to develop the C4X NRF2 Activator programme for these ailments. This includes an upfront payment of $2m from AstraZeneca.
We need to make it possible for busy doctors and clinics to be in a learning environment,” she said. Developing a health system that fosters collaboration and constant learning is paramount to obtaining generalizable medical evidence. However, several barriers hinder the development of such a system.
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