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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.
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.
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.
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?
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.
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.
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 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.
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.
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.
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.
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 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.
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.
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.
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?
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.
Ei s ai and Bliss Biopharmaceutical have entered into a joint development agreement for antibody-drug conjugate (ADC), BB-1701, to treat cancers. The collaboration will help in further development of BB-1701 across the world and advance the ADC towards late stage of development.
The NIH Pragmatic Trials Collaboratory’s Health Equity Core released a new Health Equity in Pragmatic ClinicalTrials guide designed to help research teams define health equity and provide examples of how to promote it in clinical research. ” View the complete guide. . ” View the complete guide.
However, the current MS pipeline offers new hope for PPMS patients, with three late-stage agents currently in development. Nonetheless, the increased focus on the development of PPMS treatment is a welcome game-changer for patients.
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.
Researchers have developed a blood test to determine a person’s risk of developing anxiety, while also providing insight into its current severity and best course of treatment.
Selecting the right CRO to partner with on your development program is a pivotal decision that can shape the trajectory of your product’s development. Our consultative approach ensures success by integrating research methodology, regulatory affairs strategy, and clinical operations. You have the oversight you need.
Amidst a shifting clinical landscape characterized by increasingly complex trial designs and growing patient subpopulations, many contract research organizations (CROs) have adopted a “one-stop-shop” strategic approach. Moreover, these delays impact getting potentially lifesaving therapies to patients in need.
The year ahead in clinicaltrials looks set to be one that is challenged and influenced by regulatory developments, new guidance, and broader political shifts.
Termination of the branaplam program, according to Novartis, was due to an “overall assessment of the risk-benefit profile” observed in a mid-stage clinicaltrial that raised safety concerns last year.
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