Mon.Mar 20, 2023

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FDA staff signal support for Biogen ALS drug, but highlight data hurdles

Bio Pharma Dive

In documents released Monday, agency staff appeared inclined to approve Biogen’s tofersen, which failed in its main trial. Gathering confirmatory evidence of its benefit could be difficult, however.

Drugs 282
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RVAC and University of Pennsylvania to develop mRNA vaccines

Pharmaceutical Technology

RVAC Medicines has announced a research collaboration with the University of Pennsylvania (Penn) for the discovery and development of mRNA vaccines. The collaboration, under a sponsored research agreement, will focus on mRNA vaccines that can help in modulating the normal immune response of the body for the treatment of certain autoimmune diseases as well as allergic conditions.

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Karuna’s schizophrenia drug succeeds in a second late-stage trial

Bio Pharma Dive

The results add to a growing body of evidence that Karuna’s drug, known as KarXT, could be a valuable new option for patients living with the brain disorder.

Drugs 276
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Centralisation of drug licensing may not be the right path to improve quality of drugs: SPIC

AuroBlog - Aurous Healthcare Clinical Trials blog

Empowering of the Central Drugs Standard Control Organisation (CDSCO) through centralisation of drug licensing may not be the path to improve the quality of drugs manufactured in the country both for domestic and international markets, says SME Pharma Industries Confederation (SPIC), the apex organisation of small and medium pharma industries in the country.

Licensing 152
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European Clinical Supply Planning: Balancing Cost, Flexibility and Time

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Addressing manufacturing challenges in gene therapy development

Bio Pharma Dive

As new gene therapies are developed to treat a growing number of indications, patients may soon have access to novel treatment options and potential cures.

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New partner: Telemedicine Technologies

Deltaclinical

With 20+ years of experience in clinical trials, Telemedicine Technologies is not only a recognised eClinical software editor but also an experienced Data Management service provider. With its CleanWeb® eClinical Suite, Telemedicine Technologies offers high-quality focused, flexible and cost-effective solutions that are dedicated to the electronic management of clinical trials and registries.

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New partner: Telemedicine Technologies

Deltaclinical

With 20+ years of experience in clinical trials, Telemedicine Technologies is not only a recognised eClinical software editor but also an experienced Data Management service provider. With its CleanWeb® eClinical Suite, Telemedicine Technologies offers high-quality focused, flexible and cost-effective solutions that are dedicated to the electronic management of clinical trials and registries.

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Illumina, facing Icahn challenge, ‘moving quickly’ on resolution for Grail

Bio Pharma Dive

The DNA-sequencing company said the activist investor hasn’t offered any better solutions for satisfying regulators’ concerns about the acquisition.

DNA 141
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Race for a first-to-market therapy for NASH heats up

Pharmaceutical Technology

The race for a first-to-market therapy in non-alcoholic steatohepatitis (NASH) has been a marathon, with many companies facing numerous setbacks and failures. However, it appears that two companies have gained momentum and are in a sprint to the finish line. Leading the race to commercialisation is Intercept Pharmaceuticals, which, in December 2022, resubmitted the new drug application (NDA) for its obeticholic acid (OCA), an FXR agonist, as a Class 2 resubmission (which has a six-month review p

Marketing 130
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Early decisions to de-risk the transition to combination products

Bio Pharma Dive

Speeding up the drug development process through a less optimized containment and delivery system may have been an option in the past, but both expectations and the competitive environment are changing.

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The New Age of Decentralized Clinical Trials

White paper that delves into the complex topic of Decentralized Clinical Trials and how to master them within the confines of FDA Regulations

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Predictive Oncology partners with Cancer Research Horizons

Pharmaceutical Technology

Predictive Oncology and Cancer Research Horizons, a part of Cancer Research UK (CRUK), has collaborated to drive cancer drug development using the former’s PEDAL platform. The partnership will use the artificial intelligence (AI) and machine learning platform, PEDAL technology, to determine which cancer types and patient populations are most likely to respond to treatment with Cancer Research Horizon’s pre-clinical drug inhibitors of glutaminase.

Research 130
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‘That scares me’: New childhood obesity guidelines still face a long road to consensus

STAT News

This is part of a series about new obesity drugs that are transforming patients’ lives, dividing medical experts, and spurring one of the biggest business battles in years. Read more about The Obesity Revolution. The rise of childhood obesity in the United States did not happen quickly. But, to medicine, “it sort of cropped up overnight,” says Bob Siegel, a pediatric obesity specialist at Cincinnati Children’s.

Medicine 137
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Colorectal cancer is rising among Gen X, Y & Z. Here are 5 ways to protect yourself

NPR Health - Shots

If you're in your 20s, 30s or 40s, you need to know the signs to watch for and when to seek screening or treatment for colorectal cancer.

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Opinion: Will there be any emergency doctors to see you in the future?

STAT News

In the emergency room today, everyone is suffering. Many emergency medicine physicians are struggling to provide quality care amid staffing shortages, increased pressure to meet productivity metrics, and frustrated patients battling prolonged wait times. Their situation is compounded by a sense that they have been abandoned by hospital leadership, leading to increasing levels of physician burnout and attrition.

Doctors 128
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Unlocking Excellence: How Catalent Is Transforming Japan’s Clinical Research

Planning on running clinical trials in Japan? How can you reliably supply these studies? Discover Catalent’s clinical supply packaging facility in Shiga, Japan. Strategically located between Tokyo and Osaka, and one of largest in Japan, this 6,000 square meter facility offers comprehensive services including primary and secondary clinical packaging and labelling, comparator sourcing, cold chain storage, local and global distribution, local language support and white glove service to support stud

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The potentially deadly Candida auris fungus is spreading quickly in the U.S.

NPR Health - Shots

Cases of the fungus that did not respond to antifungal medicine tripled in 2021. It can be deadly, but does not seem to have adverse effects on healthy people.

Medicine 121
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‘A safe place to fail’: ARPA-H chief on building a culture of risk-taking in science

STAT News

Renee Wegrzyn has been assembling teams her whole career — in academia, industry, and government. Now she’s in charge of a multibillion-dollar operation tasked with spotting talent, incubating breakthroughs, and doing for the life sciences what DARPA did for technology. Wegrzyn is director of the Advanced Research Projects Agency for Health , or ARPA-H, a newly formed organization tasked with investing in promising ideas across the life sciences.

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Lighting up tumors could help surgeons remove them more precisely

Medical Xpress

A new technique that combines highly detailed, real-time images of inside the body with a type of infrared light has, for the first time, been used during surgery to differentiate between cancerous tumors and healthy tissue.

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STAT+: ‘We’re getting much more aggressive’: Microsoft’s Nuance adds GPT-4 AI to its medical note-taking tool

STAT News

An AI medical scribe platform is the latest entrant into the AI arms race. On Monday, Microsoft-owned Nuance Communications announced it is integrating GPT-4 into its Dragon Ambient Intelligence platform, which is used by hospitals around the country to ease doctor workloads by using AI to listen to patient–provider conversations and write medical visit notes.

Doctors 105
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The New Age of Decentralized Clinical Trials

This new white paper defines and details the impact of Decentralized Clinical Trials on the Pharmaceutical industry and how the impact can be measured along with steps companies can take to ensure adoption.

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Fruit and veg supply issues associated with raised blood pressure, study shows

Medical Xpress

It is recognized that low fruit and vegetable consumption is a major, modifiable, risk factor associated with raised blood pressure. However, how the national supply of fruit and vegetables has impacted blood pressure trends is little-known.

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Higher cancer rates found in military pilots, ground crews

STAT News

WASHINGTON — A Pentagon study has found high rates of cancer among military pilots and for the first time has shown that ground crews who fuel, maintain, and launch those aircraft are also getting sick. The data had long been sought by retired military aviators who have raised alarms for years about the number of air and ground crew members they knew who had cancer.

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Understanding viral load and hepatitis B infections

Antidote

Hepatitis B , sometimes abbreviated to HBV, is a liver infection that is one of the most serious in the world. If left untreated, hepatitis B can cause fibrosis, cirrhosis, hepatocellular carcinoma, and end-stage liver disease.

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STAT+: Top FDA official: Agency needs to start using accelerated approval for gene therapies

STAT News

DALLAS — A top Food and Drug Administration official said Monday that the agency needs to start using accelerated approval , a much-debated path commonly used for advancing cancer drugs, to advance gene therapies for rare disease. “We always are going to keep safety, front and foremost,” Peter Marks, head of the Center for Biologics Evaluation and Research, told attendees at the annual meeting of the Muscular Dystrophy Association.

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Roles and Responsibilities of Specialized Clinical Supply Experts

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. However, there are human-based and knowledge-driven factors that are often overlooked that go beyond these basic physical capabilities and are integral to the development and delivery of high performing clinical supply chains.

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Almost all countries around the world criminalize abortion in some circumstances, finds global review

Medical Xpress

Almost all countries around the globe criminalize abortion in some circumstances, despite the public health risks and impact on human rights, finds a review of the scope of penalties for the procedure in 182 nations, published in the open access journal BMJ Global Health.

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STAT+: Karuna’s treatment for schizophrenia achieves study goals, bolsters FDA filing plans

STAT News

Karuna Therapeutics said Monday that its treatment for schizophrenia reduced psychosis reported by patients — achieving the main goal of a large clinical trial and supporting similarly positive results from previously conducted studies. The company intends to file a marketing application for its drug, called KarXT, with the Food and Drug Administration by the middle of the year.

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Enhanced recombination among omicron variants shown contributes to viral immune escape, finds study

Medical Xpress

In January 2022, around the time that the omicron variant of SARS-CoV-2 started spreading rapidly, a team of researchers at the Indian Institute of Science (IISc) led by Shashank Tripathi, Assistant Professor at the Department of Microbiology & Cell Biology, and Centre for Infectious Diseases Research, noticed that there was an unusually high increase in the number of recombinant strains of the omicron variant.

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STAT+: Hospitals are not crumbling, Medicare experts tell Congress

STAT News

Hospitals’ financial situations are not nearly as dire as industry groups are making them out to be, Medicare policy experts are telling Congress. Profit margins hit all-time highs in 2021, and almost $200 billion of taxpayer subsidies provided hospitals with ample cushion to get through the worst of the pandemic, the Medicare Payment Advisory Commission said in its newest report.

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How Machine Learning Drives Clinical Trial Efficiency

Clinical trial data management is increasingly challenging as studies grow in complexity. Quickly accessing and analyzing study data is vital for assessing trial progress and patient safety. In this paper, we explore real-time data access and analysis for proactive study management. We investigate using adverse event (AE) data to monitor safety and discuss a clinical analytics platform that supports collaboration and data review workflows.

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Can lymph nodes boost the success of cancer immunotherapy?

Medical Xpress

Cancer treatment routinely involves taking out lymph nodes near the tumor in case they contain metastatic cancer cells. But new findings from a clinical trial by researchers at UC San Francisco and Gladstone Institutes shows that immunotherapy can activate tumor-fighting T cells in nearby lymph nodes.

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Watch: P-value, an important measurement in clinical trials, explained

STAT News

When testing new treatments, drugmakers must show that their formulation bears out better results than a placebo in clinical trials. In fact, the new drug must outperform the placebo by a statistically significant margin using a measurement called “p-value.” In this short video, STAT national biotech reporter Damian Garde explains what p-value is, how it is used, and why it is still considered the gold standard for statistical significance , despite its shortcomings.

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Unrealistic vaping views? Nearly 50 percent of parents confident they'd know if their child vapes

Medical Xpress

Nearly half of parents say they would definitely know if their child was vaping, despite characteristics of vaping devices that make it easy to hide or disguise their use, a new national poll suggests.

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STAT+: Pharmalittle: Manufacturers say bird flu shots for humans can be made quickly; obesity guidelines for children disappoint

STAT News

Good morning, everyone, and welcome to another working week. We hope the weekend respite was relaxing and invigorating, because that oh-so familiar routine of phone calls, online meetings, and deadlines has returned. To cope, we are firing up the coffee kettle and brewing a few cups of stimulation. Our choice today is chocolate marshmallow, for those tracking this sort of thing.

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Accelerating Clinical Supply Through Integrated Drug Development

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. There are two approaches that can be used to speed a drug from development to clinic faster: timeline compression and parallel processing, but only one that considers the benefits of integrating clinical supply into the overall drug development process.