March, 2024

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March 7, 2024: New Report Sets Out Posttrial Responsibilities in Pragmatic Clinical Trials

Rethinking Clinical Trials

Dr. Stephanie Morain In a new report from the NIH Pragmatic Trials Collaboratory, a team of bioethicists and implementation scientists argue for a “presumptive default” that the results of pragmatic clinical trials should be incorporated into healthcare delivery processes. This responsibility arises from a key rationale for conducting pragmatic trials: that they can facilitate uptake of their results by relevant decision-makers.

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The unique clinical trial challenges faced by digital therapeutics

pharmaphorum

On today’s podcast, host Jonah Comstock is joined by Meri Beckwith, co-founder of Lindus Health, a next-gen CRO that includes digital therapeutics among its specialties. They talk a little bit about the often unseen difficulties of validating a digital therapeutic through a clinical trial.

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What’s next for AAV gene therapies in 2024?

Pharmaceutical Technology

Upcoming regulatory events for pipeline AAV gene therapies, with a focus on neuromuscular and blood disorders, are setting high expectations for the space in 2024.

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Epigenetic Editing Explodes on the Heels of Gene Editing Success

BioSpace

Ubiquitous potential, possible safety advantages and the recent growth of cell and gene therapy are driving investment in a different type of genetic editing.

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European Clinical Supply Planning: Balancing Cost, Flexibility and Time

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Building the patient experience into observational studies produces stronger real-world evidence

pharmaphorum

Incorporating the patient experience into observational studies can enhance the quality of real-world evidence gathered. Learn how patient-centred approaches can lead to stronger research outcomes.

Research 119
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Amylyx ALS drug fails crucial study, putting company’s future in doubt

Bio Pharma Dive

The results have led Amylyx to pause promotion of Relyvrio and potentially pull it from the market in the coming weeks, a major blow to the company and ALS patients.

Drugs 342

More Trending

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Pain is reduced by almost half with cannabis-based medicines – report finds

Outsourcing Pharma

On March 7, Celadon Pharmaceuticals plc, a leading UK-based pharmaceutical company specializing in cannabis-based medicines, unveiled promising findings from an early economic analysis of its chronic pain clinical trial, dubbed CANPAIN.

Medicine 121
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Akums introduces Hydroxyurea oral suspension for sickle cell disease

Pharmaceutical Technology

Akums Drugs and Pharmaceuticals has introduced Hydroxyurea oral suspension, a room temperature-stable drug for treating sickle cell disease.

Drugs 306
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BioNTech Hit with NIH Notice of Default over COVID-19 Vaccine Royalties

BioSpace

The National Institutes of Health claims BioNTech is in default regarding alleged royalty payments the agency contends it is owed in connection with the company’s COVID-19 vaccine Comirnaty.

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Cannabinoids show promise in acute migraine clinical trial

pharmaphorum

Inhaled cannabinoids have been shown to perform better than placebo in providing pain relief for people suffering from acute migraine in a clinical trial

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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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Madrigal wins FDA approval of first drug for MASH

Bio Pharma Dive

Rezdiffra’s clearance is a turning point in the fight against the liver disease. But its sales potential is unclear, and competition could emerge from popular weight loss drugs.

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BioNTech hit with 'notice of default' from NIH in COVID-19 vaccine royalty dispute

Fierce Pharma

Even as Germany's BioNTech deals with ongoing declines of its revenue and share price, it's facing another serious concern: U.S. | Even as Germany's BioNTech deals with ongoing declines of its revenue and share price, it's facing another serious concern: U.S. officials are pressing the company to pay royalties linked to the commercialization of its lucrative Pfizer-partnered COVID-19 vaccine.

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What do volunteers for research studies do after finding a trial?

Antidote

For many people, participating in a research study presents an interesting opportunity with many benefits to volunteering. While there are many types of clinical research, all with distinct goals and timelines, trial volunteers will follow a similar path when taking part. Read on to learn more about the process.

Trials 119
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AD/PD 2024: TauRx’s HMTM promising despite lacking primary endpoint data

Pharmaceutical Technology

Participants cannot remain blind to their treatment arm due to the properties of TauRx's drug, which causes urinary discoloration.

Drugs 299
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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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Pfizer Terminates Phase III Sickle Cell Study Due to Slow Patient Recruitment

BioSpace

While Pfizer has ended one of its two Phase III studies for inclacumab in sickle cell disease, the company is still eyeing an approval for the antibody in the inherited blood disorder by 2026.

Antibody 145
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Nanoscope preps filing for retinitis pigmentosa gene therapy

pharmaphorum

Nanoscope Therapeutics is on the brink of filing for FDA approval of what could be the first gene therapy for incurable eye disease retinitis pigmentosa (RP) that can be used regardless of underlying genetic mutations.

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Biotech stock fundings headed for best quarter in 3 years, Jefferies says

Bio Pharma Dive

Publicly traded companies raised nearly $10 billion in follow-on stock offerings in January and February, a financing surge that’s driving a “sector recovery,” the investment bank said.

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AstraZeneca pledges £650M in UK investments to boost vaccine capabilities, expand near HQ

Fierce Pharma

AstraZeneca CEO Pascal Soriot, who has been critical of the U.K.’s business environment for the life sciences industry, is directing a big chunk of money into the Big Pharma’s home country. | AstraZeneca CEO Pascal Soriot, who has been critical of the U.K.’s business environment for the life sciences industry, is directing £650 million into the Big Pharma’s home country.

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What the FDA's New Dosage Guidance Means for the Future of Clinical Research

Speaker: Dr. Ben Locwin - Biopharmaceutical Executive & Healthcare Futurist

What will the future hold for clinical research? A recent draft from the FDA provides valuable insight. In "Optimizing the Dosage of Human Prescription Drugs and Biological Products for the Treatment of Oncologic Diseases," the FDA notes that "targeted therapies demonstrate different dose-response relationships compared to cytotoxic chemotherapy, such that doses below the Maximum Tolerated Dose (MTD) may have similar efficacy to the MTD but with fewer toxicities.

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Antidote anecdotes: A conversation with an MS care partner

Antidote

MS is a chronic disease that is estimated to impact over a million Americans. The condition causes damage to the body’s central nervous system, leading to numbness, memory problems, sight issues, and difficulties with coordination.

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The growing uses of medicinal cannabis

Pharmaceutical Technology

Demand for medicinal cannabis is increasing globally to treat a range of illnesses and conditions.

Medicine 340
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FDA Approves Madrigal’s Rezdiffra as First MASH Therapy

BioSpace

Madrigal Pharmaceuticals’ Rezdiffra (resmetirom) is the first-ever approved therapy for metabolic dysfunction-associated steatohepatitis—a decision experts say could signal a sea change in treatment of the disease.

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AI in Drug Discovery: Day One

pharmaphorum

There is no denying that artificial intelligence (AI) has captured the imagination of innovators working across the pharmaceutical sector. The drug discovery space is no different, with myriad applications promising to revolutionise the way that researchers and sponsors approach identifying and progressing new drug candidates.

Drugs 126
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2022 Research: The Rapid Rise of Ocean Freight Visibility

A research study conducted by The Journal of Commerce and FourKites surveyed hundreds of international shippers, exploring how their usage of global supply chain visibility technology has evolved since the onset of global disruptions caused by COVID-19. For international shippers, ocean freight visibility has evolved from optional to essential and satisfaction with visibility varies greatly depending on how it is obtained and delivered.

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Madrigal, FDA approval in hand, outlines plan to sell MASH drug

Bio Pharma Dive

The company expects initial uptake of Rezdiffra, which costs $47,400 per year, to be slow as doctors and insurers establish the protocols needed to find patients eligible for treatment.

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FDA approves first MASH drug: Madrigal's Rezdiffra breaks ground in notorious biopharma graveyard

Fierce Pharma

The decades-long wait for an effective treatment for metabolic dysfunction-associated steatohepatitis (MASH) has ended, as the FDA has approved the first drug for the fatty liver disease. | The decades-long wait for an effective treatment for metabolic dysfunction-associated steatohepatitis (MASH) has ended. The FDA has approved Madrigal's resmetirom under the brand name Rezdiffra as the first drug for the fatty liver disease.

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World Bipolar Day 2024: Innovations in Bipolar Disorder Treatment

XTalks

World Bipolar Day, observed on March 30th each year, is an important global awareness day focusing on bipolar disorder and eliminating the social stigma often associated with mental health issues. March 30 is also the birthday of the well-known Dutch artist Vincent van Gogh who was posthumously diagnosed to have had bipolar disorder. World Bipolar Day started as a partnership initiative of the International Bipolar Foundation (IBPF), the Asian Network of Bipolar Disorder (ANBD) and the Internati

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Measles cases continue to rise in US amid lagging vaccination rates

Pharmaceutical Technology

According to the CDC, over 64 cases of measles have been documented in the US in 2024, already exceeding 2023's total of 58.

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An Innovative & Creative Problem Solver Approach to Selling in the Medical Device Space

Speaker: Steve Goldstein, Sales Leader

Are you currently in sales, or involved in a business that depends on strong sales results? What about the extremely competitive world of medical device sales? What are some of the top challenges your customers face and how do you approach understanding what’s most important to them? Join Steve Goldstein, Sales Success Coach, Motivational Speaker and Medical Device Sales Leader from Gold Selling LLC., to discover critical strategies and approaches you can take to engage your customers, achieve g

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Merck, Others Look to Immunotherapy Combos as Next Frontier in Oncology

BioSpace

Drugmakers are testing a variety of biologics and small molecules in conjunction with therapies aimed at modulating the immune system to target and destroy cancer cells.

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Why gut bacteria is the next frontier in medicine

pharmaphorum

Discover why gut bacteria are considered the next frontier in medicine in this informative article. Learn about the importance of the microbiome, its impact on health, and how it relates to conditions like Clostridiales difficile.

Bacteria 126
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After surprise trial failure, ALS doctors brace for one less treatment option

Bio Pharma Dive

Physicians are preparing for tough conversations with patients on Amylyx Pharmaceuticals’ drug, Relyvrio, while holding out hope it still may help some with the disease.

Doctors 306
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In banner year, Lilly rewards CEO David Ricks with 24% boost in total pay to $26.6M

Fierce Pharma

In 2023, Eli Lilly CEO David Ricks received a 24% boost in pay from $21.4 million to $26.6 million. The increase coincides with the company's booming sales.

Sales 143
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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