Fri.Dec 08, 2023

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Addressing Burnout: Why Psychological Safety Matters at Work

Pharmacy Times

Psychological safety substantially contributes to team effectiveness, learning, employee retention, and—most critically—better decisions and better performance.

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In historic decision, FDA approves a CRISPR-based medicine for treatment of sickle cell disease

STAT

The Food and Drug Administration on Friday approved the world’s first medicine based on CRISPR gene-editing technology, a groundbreaking treatment for sickle cell disease that delivers a potential cure for people born with the chronic and life-shortening blood disorder. The new medicine , called Casgevy, is made by Vertex Pharmaceuticals and CRISPR Therapeutics.

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FDA Approves Casgevy, Lyfgenia for the Treatment of Sickle Cell Disease

Pharmacy Times

This FDA approval represents the first cell-based gene therapies for the treatment of sickle cell disease in patients 12 years of age and older.

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Opinion: After living with sickle cell disease for 39 years, I’m both excited and skeptical about the newly approved gene therapies

STAT

For 39 years, I have lived with sickle cell disease. As of late, the sickle cell community has heard a lot of excitement about gene therapy’s potential to be the cure we’ve all been waiting for. That potential took a step toward fulfillment Friday when the Food and Drug Administration approved Casgevy from Vertex Pharmaceuticals’ and Lyfgenia from Bluebird Bio, both gene therapy treatments for people with sickle cell.

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From Diagnosis to Delivery: How AI is Revolutionizing the Patient Experience

Speaker: Simran Kaur, Founder & CEO at Tattva Health Inc.

The healthcare landscape is being revolutionized by AI and cutting-edge digital technologies, reshaping how patients receive care and interact with providers. In this webinar led by Simran Kaur, we will explore how AI-driven solutions are enhancing patient communication, improving care quality, and empowering preventive and predictive medicine. You'll also learn how AI is streamlining healthcare processes, helping providers offer more efficient, personalized care and enabling faster, data-driven

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FDA Approves Isavuconazonium Sulfate to Treat Invasive Aspergillosis and Invasive Mucormycotic

Pharmacy Times

The approval marks isavuconazonium sulfate as the only granted azole antifungal therapy for pediatric individuals AI and IM that are as young as 1 years old.

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FDA approves bluebird's sickle cell disease gene therapy. Can Lyfgenia overcome CRISPR’s halo?

Fierce Pharma

Alongside a historic approval for the first therapy utilizing the Nobel Prize-winning CRISPR/Cas9 gene-editing technology, the FDA has cleared a rival gene replacement therapy, also for sickle cell | Alongside a historic approval for the first therapy utilizing the Nobel Prize-winning CRISPR/Cas9 gene-editing technology, the FDA has cleared a rival gene replacement therapy, also for sickle cell disease (SCD).

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STAT+: A Q&A with the CEO of CRISPR Therapeutics on a historic approval and the road ahead

STAT

Editas Medicine, CRISPR Therapeutics, and Intellia Therapeutics were all started in the 2013-2014 time frame with the same goal of turning the nascent CRISPR gene-editing technology into medicines. With the approval of Casgevy , the world’s first gene-edited therapy and a potential cure for sickle cell disease, CRISPR Therapeutics won the race.

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The Future of Immunotherapy in Breast Cancer Involves Engineered T-Cell Therapies, Better Prediction of Adverse Effects

Pharmacy Times

As the use of sequencing, proteomics, and immunopeptidomics increases, more targets will also be discovered, as well as more biomarkers that predict responses to treatments.

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STAT+: Tough road ahead for Bluebird Bio despite FDA approval for sickle cell therapy

STAT

The Food and Drug Administration’s approval on Friday of two gene therapies for sickle cell disease sets up what could be an intense competition between Bluebird Bio, maker of Lyfgenia, and the partnership of Vertex Pharmaceuticals and CRISPR Therapeutics, whose drug is Casgevy. For the first time, physicians and patients will have a choice of potentially curative treatments for an inherited disease that affects nearly 100,000 Americans.

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Expert Discusses Pharmacists Role on Rural Access to Care, Aiding Health Equity

Pharmacy Times

Tera Moore, PharmD, BCACP, discusses how the integration of pharmacists into team-based models could benefit patient access to care in rural populations.

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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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STAT+: New proposals for scientific misconduct investigations worry some research universities

STAT

Research universities are voicing concerns over some proposed changes to the process for reviewing scientific misconduct allegations, citing worries that new government requirements would hobble their own independent procedures and constitute a huge administrative burden. Nearly two decades since it last released research misconduct rules, the U.S. Office of Research Integrity (ORI) is ready to update its requirements for ensuring the scientific standards of projects funded by the U.S.

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Vertex, CRISPR score landmark FDA approval for sickle cell disease gene therapy Casgevy

Fierce Pharma

Groundbreaking? Game-changing? Transformational? Historic? | Vertex Pharmaceuticals and CRISPR Therapeutics have won an historic FDA approval for their sickle cell disease gene therapy Casgevy. The transformational treatment is a potential cure for the debilitating and life-threatening disease which affects more than 100,000 in the United States, most of them Black.

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STAT+: Vertex gets a win as Colorado board leaves cystic fibrosis drug off a list of unaffordable medicines

STAT

In a win for Vertex Pharmaceuticals, a Colorado state board voted not to place the company’s best-selling cystic fibrosis medication on a list of drugs for which payment limits will be set for most residents, including those whose insurance is covered by a government agency or a commercial health plan. The Colorado Prescription Drug Affordability Board voted unanimously that the expensive medicine, which carries a wholesale price of about $310,000 in the U.S. before any rebates or discoun

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Bristol Myers Squibb bids farewell to dealmaking czar Elizabeth Mily

Fierce Pharma

Three years on, Bristol Myers Squibb is bidding adieu to its top dealmaker. | Three years on, Bristol Myers Squibb is bidding adieu to its top dealmaker. Elizabeth Mily, who replaced BMS’ former M&A czar Paul Biondi back in 2020, is headed for the exit, the company confirmed Friday.

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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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Opinion: Clients bring politics into the therapy room. Here’s what that means for the therapist

STAT

“Can I tell you what I really think about what’s going on?” she asks, looking down and clutching her coffee, her right sneaker tapping. I had been fairly sure about two things ahead of our session: one, that she would talk about the Conflict, and two, that I would dislike what she was going to say. I gulp what I hope is a hidden gulp, every inch of me wanting to tell her no actually, can we please not go there and just stick with your relationship unhappiness?

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Expert: Study Results Could Improve Glycemic Outcomes with Pharmacist Management of Insulin

Pharmacy Times

Cole Daniels, PharmD, BCPS, discusses a quality improvement initiative for glycemic outcomes with pharmacist management of insulin.

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Chronic fatigue syndrome estimated to affect 3.3 million in U.S., more than previously thought: CDC

STAT

Health officials on Friday released the first nationally representative estimate of how many U.S. adults have chronic fatigue syndrome: 3.3 million. The Centers for Disease Control and Prevention’s number is larger than previous studies have suggested, and is likely boosted by some of the patients with  long COVID. The condition clearly “is not a rare illness,” said the CDC’s Dr.

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How Health-System Specialty Pharmacists’ Actions Impact External Pharmacy Patients

Drug Topics

The analysis revealed deeper issues created by payer and manufacturer lockouts placed on Integrated Health System Specialty Pharmacies.

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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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Pfizer’s initiative to return clinical trial data to patients

pharmaphorum

Pfizer’s initiative to return clinical trial data to patients Mike.

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STAT+: Medicare’s advisory committee isn’t sold on big pay raises for doctors

STAT

On Thursday, the Medicare Payment Advisory Commission reviewed Medicare performance data and concluded that despite doctors’ protests that their pay hasn’t kept up with inflation over the last 20 years, doctors shouldn’t get more than a minor raise for 2025. On the other hand, the committee admitted hospitals could use a larger increase in payment rates.

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PharmStars digital health accelerator names fall 2023 grads

pharmaphorum

PharmStars digital health accelerator names fall 2023 grads Phil.

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No Significant Differences at 5 Year Follow-Up Between Palbociclib-Based Treatment Regimens

Pharmacy Times

Study results indicate insignificant differences between PFS and OS in patients with HR[+]/HER2[-] breast cancer who received either palbociclib-letrozole or palbociclib-fulvestrant treatment.

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Here’s what you need to know about the Lower Costs, More Transparency Act

Fierce Healthcare

A House vote on transparency regulations in healthcare could occur next week, potentially having substantial ramifications on how PBMs and hospitals operate. | Bipartisan anticipation is in the air for a potential vote in the House this week that would bring transparency to PBMs and ban spread pricing in Medicaid, but hospital groups are firmly opposed to provisions enacting site-neutral payment.

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Morning Rounds: 3D-printed organs, antimicrobial resistance in Ukraine, and other major health stories of the day

STAT

Understand how science, health policy, and medicine shape the world everyday. Sign up for our Morning Rounds newsletter here. Institutions push back on scientific misconduct rules Investigating scientific research misconduct is necessarily a heavy lift for universities and other institutions. Many are now saying that proposed  changes  from the U.S.

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Africa-led HIV vaccine trial ends in failure

pharmaphorum

Africa-led HIV vaccine trial ends in failure Phil.

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STAT+: New proposals for scientific misconduct investigations worry some research universities

STAT

Research universities are voicing concerns over some proposed changes to the process for reviewing scientific misconduct allegations, citing worries that new government requirements would hobble their own independent procedures and constitute a huge administrative burden. Nearly two decades since it last released research misconduct rules, the U.S. Office of Research Integrity (ORI) is ready to update its requirements for ensuring the scientific standards of projects funded by the U.S.

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Watch DSN: Walgreens’ Shah outlines the future of pharmacy

Drug Store News

Rina Shah shared her thoughts on the future of pharmacy and how their current VillageMD model serves as the template for the future.

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STAT+: Up and down the ladder: The latest comings and goings

STAT

Hired someone new and exciting? Promoted a rising star? Finally solved that hard-to-fill spot? Share the news with us, and we’ll share it with others.   That’s right. Send us your changes, and we’ll find a home for them. Don’t be shy. Everyone wants to know who is coming and going. And here is our regular feature in which we highlight a different person each week.

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FDA Approves Two Landmark Cell-Based Gene Therapies for Sickle Cell Disease

PharmExec

FDA approval of bluebird bio’s Lyfgenia and Vertex Pharmaceuticals' and CRISPR Therapeutics’ Casgevy marks significant milestone in the treatment of sickle cell disease.

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Why pharma needs to leave digital transformation behind and embrace digital business

pharmaphorum

Why pharma needs to leave digital transformation behind and embrace digital business Mike.

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Examining Tenofovir Alafenamide Versus Tenofovir Disoproxil Fumarate in the Treatment of HBV

Drug Topics

Two posters presented at ASHP Midyear 2023 looked at how the two medications differed and how clinical pharmacy services improved transitions.

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Life sciences industry predictions 2024: Curavit

pharmaphorum

Life sciences industry predictions 2024: Curavit Mike.

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Merck and Eisai's Keytruda-Lenvima combo comes up short yet again, this time in endometrial cancer

Fierce Pharma

Merck and Eisai’s combo of Keytruda and Lenvima has missed the mark yet again, rounding out a year of multiple trial flops for the pairing. | The combo missed both endpoints in a phase 3 study in certain patients with advanced or recurrent endometrial carcinoma, adding yet another entry to a long list of recent trial failures.

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