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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 clinical trials, even for trials deemed to have minimal risk.
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.
The NIH Pragmatic Trials Collaboratory will hold a virtual workshop on February 5 and 6, 2025, on Digging Into Dilemmas of Pragmatic Clinical Trials. To date, the NIH Collaboratory has launched 35 pragmatic trials that are conducted within healthcare systems at over 1,400 clinical sites.
Clinical trials 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?
Clinical development organizations face a wide array of challenges when it comes to data, many of which can impact the operational effectiveness of their clinical trials. In this whitepaper, experts from Revvity Signals explore how solutions like TIBCO® Spotfire® enable better, more streamlined studies.
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 NIH and the Patient-Centered Outcomes Research Institute (PCORI) are major funders of pragmatic clinical trials embedded in healthcare systems. An existing chapter of the Living Textbook of Pragmatic Clinical Trials provided guidance on how to Develop a Compelling Grant Application for the NIH.
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.
When selecting a clinical supply provider, consideration often focuses upon the manufacturing, packaging, storage and distribution capabilities available that will, at face-value, be sufficient to meet the needs of the sponsor and their trial.
The biotech, best known for its success developing migraine medicines, will seek approval of a treatment that failed a Phase 3 trial but showed signs of a positive effect.
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 clinical trial of BNT163 – an HSV vaccine candidate.
Despite advancements in neuroscience and psychiatry, clinical trials 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.
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 regulator cited concerns around single-country trials in turning back Hutchmed's pancreatic cancer treatment, while manufacturing issues held up Junshi and Coherus' throat cancer medicine.
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 clinical trial. The clinical advancement follows a thorough review of Phase I trial data under the FDAs Regenerative Medicine Advanced Therapy (RMAT) designation.
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 clinical trials and marketing of gene therapy products developed leveraging the new AAV capsids of Cyagen.
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 clinical trials.
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.
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 clinical trial methods. However, spillover due to resource constraints is absent from the major frameworks that inform clinical trial methods.
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.
Global rare disease clinical trials face unique challenges due to small patient populations, complex logistics, diverse regulatory environments and cultural differences. Clinical Trial Manager/Sr. These measures enhance recruitment, retention and overall trial success.
Syros Pharmaceuticals announced that its cancer drug tamibarotene failed to meet its primary endpoint in the SELECT-MDS-1 Phase III trial. The placebo-controlled SELECT-MDS-1 trial involved 190 patients with complete response rate (CRR) being the study’s primary endpoint. percent and 18.8
The surge in MASH cases stresses the need for early diagnosis, timely intervention and precise, reliable methodologies in clinical trials to evaluate new therapies effectively. Nicola Owen, PhD, MBBS, Medical Directorthey shared insights into the operational and medical strategies that support the design and conduct of MASH clinical trials.
Over the past two decades, industry-sponsored clinical trials 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.
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. CLDN 18.2).
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.
Early Phase Unit, Worldwide Clinical Trials 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.
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 clinical trials, 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.
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.
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 Clinical Trials.
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. We prioritize cost-effectiveness, providing essential services for your trial’s success at competitive rates. Discover the difference we can make for your trial.
Patients are the backbone of clinical trials, 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.
In the trial, platelet response, defined as achieving at least 50,000 platelets per microliter or doubling baseline counts, was seen in 65 percent of patients on rilzabrutinib, compared to 33 percent on placebo. Participants received either rilzabrutinib or placebo for 24 weeks, followed by an open-label extension.
On February 7, at a town hall organised to discuss clinical trial designs for gene therapies, FDA experts pushed pharma players to look for ways to establish clinical effectiveness despite the challenges in recruiting patients with rare diseases.
mg), underperformed in its latest clinical trial, leading to a dip in the companys share price. In response to the trial outcomes, Novos shares declined by approximately 8% this week. Novo shared the latest data from the Phase III REDEFINE 2 trial evaluating the drug in overweight or obese patients with type 2 diabetes.
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.
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.
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