NHS Rolls Out £1.7 Million Gene Therapy for Sickle Cell Disease
One of the world's most expensive medicines will be used to treat some patients with sickle cell disease in England using the state-funded National Health Service.
The one-time gene-editing therapy Casgevy, developed by Vertex Pharmaceuticals Inc. and Crispr Therapeutics AG, has a list price of £1.7 million ($2.1 million), but England's drug cost regulator said it has negotiated a lower, undisclosed price.
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4 hours ago
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England is launching a gonorrhea vaccine. Is Canada next?
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These data paint a complex picture of therapeutic inertia, growing anticipation for emerging treatments, and deep-seated frustration with limitations of current treatment options. Most transfusion-dependent beta thalassemia patients follow some regular transfusion schedule. That said, many receive them at relatively extended intervals: often once a month or even less frequently, though a small subset still require more frequent transfusions. This variation in treatment patterns, along with persistent symptoms and unmet needs highlighted in the chart audit, points to a strong opportunity for new therapies to improve care for a broad range of patients. Among surveyed hematologists, mitapivat (Agios) currently holds a slight edge in name recognition over etavopivat (Novo Nordisk). Interestingly, despite this difference in familiarity, physicians identified slightly more patients as potential candidates for etavopivat, hinting at a broader perceived clinical fit once awareness and understanding of the agent catch up. Chart audit data allows users to probe further into specific patient types which physicians perceive as better fits for either agent, as well as those who they do not perceive as candidates. This growing interest in emerging oral therapies reflects a broader desire among hematologists for more accessible, lower-burden treatment options. While most patients are theoretically eligible for curative approaches like gene therapy, the market still favors lower-barrier alternatives. These alternatives include oral therapies that do not require referral to specialized centers or extensive pre-conditioning, and brands with which they are more familiar given other indications such as Reblozyl (Bristol Myers Squibb). Disease-modifying therapies that are easier to prescribe and integrate into routine practice have the promise to significantly improve the lives of patients who would not qualify or want gene therapy. Access barriers compound the issue, with over half of physicians voicing frustration over payer restrictions that make it difficult to initiate optimal care. These challenges highlight a critical need – not just for clinical innovation, but also for strong manufacturer support in navigating reimbursement and expanding access. As the beta thalassemia landscape continues to evolve, hematologists are increasingly looking toward emerging therapies that are easier to prescribe, carry fewer logistical burdens than gene therapy, and offer clear reductions in transfusion dependency. 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The seasoned team of Spherix experts provides an unbiased and holistic view of the landscape within rapidly evolving specialty markets, including dermatology, gastroenterology, rheumatology, nephrology, neurology, ophthalmology, and hematology. Spherix clients stay ahead of the curve with the perspective of the extensive Spherix Physician Community. As a trusted advisor and industry thought leader, Spherix's unparalleled market insights and advisory services empower clients to make better decisions and unlock opportunities for growth. To learn more about Spherix Global Insights, visit or connect through LinkedIn. For more details on Spherix's primary market research reports and interactive dashboard offerings, visit or register here: NOTICE: All company, brand or product names in this press release are trademarks of their respective holders. The findings and opinions expressed within are based on Spherix Global Insight's analysis and do not imply a relationship with or endorsement of the companies or brands mentioned in this press release. CONTACT: Sarah Hendry, Hematology Franchise Head Spherix Global Insights 4848794284 in to access your portfolio
Newsweek
2 days ago
- Newsweek
Dad Makes 'Promise' to His Little Girl, 15 Years Later He Keeps His Word
Based on facts, either observed and verified firsthand by the reporter, or reported and verified from knowledgeable sources. A dad has revealed how a recent accident allowed him to keep a promise he had made to his daughter 15 years earlier. Peter Morrell, from Kent in the U.K., is the first to admit he is a "big kid at heart" and never misses an opportunity to get involved in whatever shenanigans his children get up to. "I like to join in on the trampoline, bike rides and water fights," Morrell told Newsweek. Unfortunately, that eagerness to get involved landed him in big trouble recently. "I bought a scooter to ride around with the kids on theirs, and I lost control and fell at an awkward angle," Morrell said. "Snapped my tibia clean in half and required surgery." While the care he received from the National Health Service in the U.K. was "amazing," Morrell was informed he would have to wear a cast on his leg for a minimum of six weeks while the broken bone healed. It was then that Morrell recalled a promise he made to his daughter Hollie, a decade and a half earlier. Children pay attention to the promises parents make and the excuses they come up with when they don't deliver. That was highlighted in a 2022 study from the journal Cognitive Development in which 64 children aged between 3 and 5 were shown videos where puppets promised to show them a toy then left only to return empty-handed. The puppets then provided the children with either a valid excuse for not producing the toy, a bad excuse or no excuse at all. The children were then quizzed on whether they felt the puppets' actions were wrong and why. Researchers discovered that, regardless of the excuse, there was consensus among the children that breaking a promise was wrong. However, they did show greater understanding if the puppet provided a valid excuse over a bad one or none at all. Thinking back to sometime around 2010, Morrell was reminded by Hollie of the promise he had made to her after she broke her leg. "She was coming up to 3 years old and was very unhappy about breaking her leg," he said. "She asked me why I couldn't have a pink cast like her, so I jokingly said if I ever break my leg, I'll get a pink sparkly cast just like her." All these years later, it turned out Hollie still remembered: "So I thought, 'Why not?' I did promise," Morrell said. But, while she had recalled the promise her dad made, Hollie was surprised to discover her dad had actually gone through with it. "When I showed her the cast on FaceTime, she burst out laughing and couldn't believe I actually did it," Morrell said. "She showed it to all her friends and teachers at school." Morrell has only a couple more weeks until the cast comes off. In the meantime, the story of how he came through for Hollie, all these years later, has captivated social media; a clip posted to TikTok under the handle @pete_m84 has gone viral with nearly 200,000 views. "I think it resonates with people as it's something that they can relate to. A promise is a promise," Morrell said. "I could have said no, but it's a bit of fun and a nice memory for us both to laugh at in a few years."
