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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 drugmaker, one of the largest vaccine developers globally, won't move forward with either of two candidates it's been testing after disappointing data in early trials.
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.
Moderna’s Stéphane Bancel and Pfizer’s Albert Bourla hinted Monday at greater dealmaking to come for their companies, while Gilead gave investors an update on an important cancer drug trial.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
The vaccine didn’t spur a strong enough immune response against a particular flu strain in a Phase 3 trial, leading the companies to ponder adjustments.
Hookipa Pharma and Roche have signed a strategic partnership and licence agreement for developing HB-700 and another undisclosed arenaviral immunotherapy. Under the deal, Hookipa will carry out research and initial clinical development through Phase Ib for HB-700 to treat KRAS-mutated cancers.
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.
Now the trial is the source of much debate, and its outcome could influence how future treatments for the disease are developed. A study testing Alnylam's drug Onpattro in a deadly heart condition was once seen as a sure bet.
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.
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.
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.
Different approaches that are studied include antisense oligonucleotides (ASOs), and gene therapies, which are in early clinical trials. Other lines of research look at the genetic overlap between FTD and amyotrophic lateral sclerosis (ALS), which could be used in the development of treatments for both conditions.
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.
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.
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.
As with other targeted therapies, radiopharmaceutical development requires unique and specialized strategies to ensure successful execution. Are you aware of the challenges you must address for a successful radiopharmaceutical trial? These include ensuring informed consent, particularly around the risks and benefits of participation.
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.
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.
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.
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.
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.
Rosa Gonzalez-Guarda The NIH Pragmatic Trials Collaboratory’s Health Equity Core developed a written aid to offer guidance on inclusive language and terms to use when referring to specific people, groups, and communities. View the Equitable Language Cheat Sheet.
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