Wed.Dec 13, 2023

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Gene therapy offered this 7-year-old freedom. The price: a grueling year

STAT

PHILADELPHIA — The meds Shelby Campbell needed for her rare blood disorder stopped working just after her sixth birthday. She lost her appetite and was often doubled over in pain. She continued getting blood transfusions but her doctors struggled to manage side effects that threatened her organs. By the time she turned 7, the doctors told her parents they had to do something — soon.

Hospitals 363
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A former CDC head on key R&D areas, public trust and if we learned anything from COVID-19

PharmaVoice

Dr. Julie Gerberding, now president and CEO of the Foundation for the National Institutes of Health, shares lessons learned from a long career in academic, public health and industry.

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Trending Sources

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STAT+: Male birth control gets another shot with small new trial of non-hormonal pill

STAT

From several types of hormonal pills to implants, IUDs, and vaginal rings, women have a lot of birth control choices that are both non-surgical and reversible. Men have just one: Condoms, which are 87% effective in preventing pregnancy, and thus less reliable than many other methods. Attempts to change the status quo have been scarce and unsuccessful.

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What’s being done to prepare for the next pandemic?

PharmaVoice

Will the world be ready when a new outbreak hits? Here’s a look at the efforts to combat emerging viral threats.

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Position Your Pharmacy for Expansion

Speaker: Chris Antypas and Josh Halladay

Access to limited distribution drugs and payer contracts are key to pharmacy expansion. But how do you prepare your operations to take the next step? Meaningful data: Collect and share clinical data regarding outcomes, utilization, and more Reporting: Limited distribution models require efficient tracking and reporting systems Workflows: Align workflows with specific pharma and payer contractual requirements For in-depth, expert insights on pharmacy expansion, watch this webinar from Inovalon.

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Opinion: Exceptions to abortion restrictions often fail people like Kate Cox whose babies are sure to die

STAT

Imagine being in the midst of your pregnancy, and you learn that your baby is unlikely to live for more than a few fleeting moments after birth. There is no cure for their condition. Nothing can prevent their death. Most people think they will never be in such a situation. And most people are right. But Kate Cox’s case in Texas this week serves as a stark wake-up call to the nation: This could happen to you or someone you care about, and medical exceptions to anti-abortion laws won’

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NHSE plans ‘greater role’ for pharmacies in delivering seasonal jabs

The Pharmacist

A new national vaccine strategy for England promises to ‘take vaccines into the heart of communities’ and overcome vaccine hesitancy. The plan, published today by NHS England (NHSE), designates community pharmacy as a ‘core’ setting for adult seasonal vaccinations, with local systems expected to ‘enable community pharmacy to play a greater role in seasonal vaccination […] The post NHSE plans ‘greater role’ for pharmacies in delivering seasonal jabs appeared first on The Pharmacist.

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Pharmacist in practice: tackling winter infections and improving AMS

The Pharmacist

An innovative scheme at a south coast general practice aims to improve antimicrobial stewardship and speed up time between symptoms and treatment of winter infections. Every patient under the age of 12 at the Brighton Health and Wellbeing Centre GP practice has received a ‘winter pack’, containing information about common winter conditions and what a […] The post Pharmacist in practice: tackling winter infections and improving AMS appeared first on The Pharmacist.

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STAT+: New federal rules demand transparency into AI models used in health decisions

STAT

Federal health technology regulators on Wednesday finalized new rules to force software vendors to disclose how artificial intelligence tools are trained, developed, and tested — a move to protect patients against biased and harmful decisions about their care. The rules are aimed at placing guardrails around a new generation of AI models gaining rapid adoption in hospitals and clinics around the country.

Hospitals 312
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Not enough pharmacists in England’s most deprived PCNs

The Pharmacist

‘Skewed’ funding arrangements mean that insufficient pharmacists are being employed by primary care networks (PCNs) in the most deprived areas of England, according to new research from the Health Foundation. The report Doing More For Less said that areas with high deprivation were missing out because the greater health needs of their populations were not adequately taken […] The post Not enough pharmacists in England’s most deprived PCNs appeared first on The Pharmacist.

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STAT+: After decade-long dispute over CRISPR-Cas9, Editas strikes deal with Vertex to cash in

STAT

For a decade, leading academic institutes and their associated companies fought a bruising, headline-grabbing fight over who held patent rights to CRISPR-Cas9, the revolutionary genome editing tool. Editas Medicine, the winner of that battle in the U.S., will now cash in. Vertex Pharmaceuticals and Editas announced Wednesday that Vertex will pay Editas $50 million, along with a series of annual payments through 2034, in exchange for rights to use CRISPR-Cas9 in Casgevy , the sickle cell therapy

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5 Reasons to Upgrade Your Pharmacy Management Software

Are you still using workarounds to manage your daily operations? To achieve peak performance, it's time to explore other options for specialty and infusion pharmacy software. Streamline pharmacy operations and improve clinical performance with automated processing, real-time data exchange, and electronic decision support. Download this helpful infographic to: Drive efficiency and patient adherence from referral receipt to delivery and ongoing care – all with our Pharmacy Cloud.

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Finding that Spark

PharmExec

Lisa Conte, founder, president, and CEO of Jaguar Health, discusses how a climb up Mount Kilimanjaro would vault her to her true career calling—and spark a decades-long quest to accelerate breakthroughs in plant-based pharmaceuticals and supportive care.

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Opinion: Sobering centers offer a safe place to recover from intoxication. Every community should have one

STAT

Every day, thousands of individuals across the United States call 911 due to concern about someone who is intoxicated in public. Their concern is warranted: The potential harms from public intoxication are significant. They are at risk of being the target of assault, violence, or theft or may suffer injury or death from an underlying health condition.

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Study confirms safety of new flow-diverting stent to treat brain aneurysms

Pharma Times

In England, ruptured brain aneurysms occur in around one in 15,000 people every year - News - PharmaTimes

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STAT+: Vertex non-opioid drug reduces chronic nerve pain in mid-stage study

STAT

Vertex Pharmaceuticals said Wednesday that an experimental drug reduced pain in people with diabetes who have chronic nerve pain — mid-stage study results that support the biotech company’s efforts to develop an effective painkiller without the addictive potential of opioids. The drug, a pill called VX-548, is designed to precisely block pain-sensing neurons from signaling the brain.

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ACSR to use digitised pathology for HIV-associated malignancies

Pharma Times

The software has reduced the time to provide pathology data from days to hours - News - PharmaTimes

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STAT+: Pfizer says 2024 profits will fall far below expectations

STAT

Pfizer said Wednesday that its 2024 profits would fall far below investor expectations, leading shares to tumble 7% in early trading. The reduced earnings numbers cap a difficult year for the drug giant. Its shares have fallen 45% and it has struggled to maintain the momentum it achieved during the pandemic with the development of one of two widely used Covid vaccines and a Covid treatment, Paxlovid.

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Pfizer to deepen cost cuts as latest sales forecasts miss expectations

BioPharma Dive

The company is now expecting to save $4 billion by the end of 2024, with a majority of the spending cuts coming from drug research and development.

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Supreme Court will decide abortion pill access

STAT

WASHINGTON — The Supreme Court agreed Wednesday to take on whether Americans can access a commonly used abortion pill. It will be the court’s first major abortion case since it overturned Roe v. Wade last year, giving jurisdiction over abortion laws back to the states. Since then, 13 states have banned the procedure and seven others have enacted severe limits, though several of those restrictions are in court and voters have spurned others.

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Vertex builds case for non-opioid pain drug ahead of key study readouts

BioPharma Dive

The closely watched medicine showed promise treating a form of chronic nerve pain, with the results suggesting comparable effects to an available therapy.

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Opinion: Listen: When do tests hurt more than help?

STAT

Manil Suri and Daniel Morgan are an unusual team: Manil is a mathematics professor and author (of both fiction and nonfiction ), while Daniel is a physician and professor of epidemiology, public health, and infectious diseases. But — in what they say is a typical “Smalltimore” moment — both a neighbor and a student had told them they should work together because of a shared interest in false positives on diagnostics tests.

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Granules India gets ANDA approval for Pantoprazole Sodium DR tablets

Express Pharma

Granules India announced today that the US Food & Drug Administration (US FDA) has approved its Abbreviated New Drug Application (ANDA) for Pantoprazole Sodium Delayed-Release Tablets USP, 20 mg and 40 mg. It is bioequivalent and therapeutically equivalent to the reference listed drug (RLD), Protonix Delayed-Release Tablets, 20 mg and 40 mg, of Wyeth Pharmaceuticals.

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STAT+: Bankrupt Pear’s addiction treatments acquired by virtual care company

STAT

Half a year after selling its assets at bankruptcy auction , Pear Therapeutics’ most significant digital treatments will find a new life as part of PursueCare, which provides online addiction care and mental health counseling. PursueCare today announced it had raised a $20 million Series B round and that it had acquired Pear’s portfolio of apps that treat people with addiction problems.

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Europe publishes first list of critical medicines

European Pharmaceutical Review

The European Commission, the Heads of Medicines Agencies (HMA) and the European Medicines Agency (EMA) have published the first Union list of critical medicines. The list contains more than 200 active substances that are considered “critical for healthcare systems” across the EU/EEA. Continuity of supply is seen as a priority for these substances and shortages should be avoided.

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STAT+: Could sickle cell someday be treated not with CRISPR, but with a pill?

STAT

Two gene therapies were approved for sickle cell disease last week, but for many researchers the Holy Grail remains something far more modest: A pill. Only the pharmacists’ oldest tool, they say, can actually have an impact on the scale of sickle cell. The gene therapies, with the risks and hospital stays and bespoke manufacturing they entail, won’t be an option for the vast majority of U.S. patients or for virtually any of the 20 million patients across the Global South.

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A New Era in Clinical Trials

PharmExec

Current industry challenges present an opportunity for innovation and the adoption of new solutions, particularly in addressing barriers related to patient recruitment, engagement, and retention.

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STAT+: Pharmalittle: Employers hire virtual providers as obesity drug gatekeepers; Vertex non-opioid nerve pain drug succeeds in mid-stage trial

STAT

Good morning, everyone, and how are you today? We are doing just fine, thank you. Given that this is already the middle of the week and we have survived this far, no reason not to continue, yes? Just consider the alternatives. In fact, this modest accomplishment calls for celebration. So please join us in quaffing a ritual cup of needed stimulation.

Hospitals 235
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Acamprosate Versus Naltrexone in Alcohol Use Disorder

Med Ed 101

Alcohol use disorder can be managed with medications. American Psychiatric Association Guidelines recommend acamprosate or naltrexone as a first-line agent for moderate to severe management of alcohol use disorder. In this article, I will compare acamprosate versus naltrexone and highlight some of the most important differences. This comparison of acamprosate versus naltrexone would definitely be […] The post Acamprosate Versus Naltrexone in Alcohol Use Disorder appeared first on Med Ed 10

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A plot to solve the ‘whack-a-mole’ of drug discovery

STAT

Want to stay on top of the science and politics driving biotech today?  Sign up  to get our biotech newsletter in your inbox. Good morning, everyone. Damian here with some hotly anticipated news from Vertex Pharmaceuticals, a closer look at the latest FTC controversy, and a detailed look at what it’s like to actually receive a gene therapy.

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How simulations help device makers meet booming autoinjector demand

Pharmaceutical Technology

Advanced simulation methods and tools are not only paving the way for more efficient manufacturing operations. At SHL Medical, they’re also speeding up the development of new production facilities around the globe.

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Morning Rounds: For kids with high BMI, task force not ready to recommend weight loss drugs

STAT

Understand how science, health policy, and medicine shape the world everyday. Sign up for our Morning Rounds newsletter here. Good morning. Don’t miss Brittany Trang’s compelling story about seven-year-old Shelby Campbell’s life — and her family’s — while being treated for beta thalassemia with gene therapy.

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New CAR-T therapies in South Korea to face stringent regulatory oversight: GlobalData

Express Pharma

South Korea has issued guidelines to monitor the side effects of the two approved CAR-T therapies – Kymriah (tisagenlecleucel) from Novartis and Carvykti (ciltacabtagene autoleucel) from Janssen. This will ensure a comprehensive database of adverse events including malignancies, if any in the local population, thereby ensuring patient safety in the country.

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Trade & Channel Strategies 2023: A DSCSA Update

Pharmaceutical Commerce

The seminar examines where members of the pharma supply chain stand in terms of preparation, being that the act has officially been enacted.

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Indian Immunologicals celebrates 25 years of Human Biologicals, launches measles and rubella vaccine

Express Pharma

Human Biologicals Institute (HBI), a division of Indian Immunologicals celebrates its silver jubilee, 25 years since its establishment in 1998. HBI was created in an era where the need for indigenous vaccines is paramount and thus has contributed to a self-reliant India – “ Atmanirbhar Bharat ”. While celebrating the occasion of 25 th year of HBI, IIL launched Mebella (Measles and Rubella) vaccine for children.

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Eisai sets Leqembi price in Japan at a discount to US

pharmaphorum

Eisai and Biogen’s Alzheimer’s disease therapy Leqembi is due to be launched in Japan, its second market, on 20th December, and will be priced quite a lot lower there than in the US. The anti-amyloid antibody has been included in the Japan National Health Insurance (NHI) Drug Price List at just under JPY 3 million (around $20,500) per patient per year, whereas in the US its list price before discounts or rebates was set at $26,500.

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FOGSI and BSV organises run for gender equality

Express Pharma

The FOGSI Run for Gender Equality is specifically organised to spread awareness on Anemia and Rh negative pregnancies among women across the country Bharat Serums and Vaccines Limited (BSV) along with Federation of Obstetric and Gynecological Societies of India (FOGSI) organised the FOGSI Run for Gender Equality (the Run) at Airoli, Navi Mumbai. Part of a 28 city run, the Run for Gender Equality aimed at connecting the country by spreading awareness for specific medical conditions such as anemia