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☆ Insight: What happens when a $2 million gene therapy is not enough
▼ + stars: | 2023-08-12 | by ( Deena Beasley | ) www.reuters.com time to read: +13 min

Their experience raises broader questions around other high-cost gene therapies coming to market, sometimes after accelerated regulatory approvals, drug pricing experts said. Gene therapies work by replacing genes – the body's blueprint for its development. The gene Zolgensma delivers instructs the body to make a protein vital for muscle control. If gene therapies do fall short, it becomes harder to justify prices that researchers have argued are already poor value. More recently, the first hemophilia gene therapy approved by the U.S. Food and Drug Administration was priced by CSL Behring at $3.5 million; 26 more gene therapies are in late-stage development, according to IQVIA.

Persons: Elizabeth Kutschke, Ben, Zolgensma, Ben Kutschke, neurologists, Sitra Tauscher, Wisniewski, Ben's, Roger Hajjar, Brigham Gene, Kutschke, Vasant Narasimhan, Stacie Dusetzina, Roche's, Biogen, Roche, Maha Radhakrishnan, Steven Pearson, It's, Sree Chaguturu, Amanda Cook, Weston, Jackson, Cook, Elizabeth, Jerry Mendell, Russell Butterfield, –, Biogen's, Mendell, UMR, Spinraza, Eric Cox, Caroline Humer, Sara Ledwith Organizations: Reuters, U.S, Novartis, IQVIA Institute, Human Data, Novartis Gene Therapies, Mass, Cell Therapy, U.S . Food, Drug Administration, CSL Behring, CSL, Nashville's Vanderbilt University School of Medicine, Clinical, Economic, CVS Health, Aetna, SMA, Nationwide Children's Hospital, University of Utah Health, Children's, UnitedHealth, Thomson Locations: Oak Park, Berwyn , Illinois, Swiss, U.S, Lebanon , Virginia, United States, Columbus , Ohio, Russia, Kazakhstan, Chicago

[1/3] A pharmacist holds a bottle of the drug Eliquis, made by Bristol Myers Squibb and Pfizer, at a pharmacy in Provo, Utah, U.S. January 9, 2020. The government will launch the negotiation process in September by naming the first drugs it plans to target. "We couldn't have the other parts of the IRA without this Medicare negotiation," said Sean Dickson, director of the West Health Policy Center, a non-partisan healthcare think tank. Eliquis, which Bristol Myers (BMY.N) shares with Pfizer (PFE.N), Ibrance, and Imbruvica, sold by AbbVie and Johnson & Johnson (JNJ.N), appear on every list. Pfizer, Novo Nordisk and J&J declined to comment on the likelihood their drugs would be included in the first round of negotiations.

☆ Drug companies favor biotech meds over pills, citing new U.S. law
▼ + stars: | 2023-01-13 | by ( Deena Beasley | ) www.reuters.com time to read: +5 min

The pharmaceutical industry, whose members gathered in the thousands this week in San Francisco for the annual JP Morgan Healthcare conference, opposed the legislation and has begun implementing strategies to mitigate its impact. "In 10 years, we'll have far fewer small molecules being developed than we do today." He questioned the benefit of "rules that really just disincentivize investment in what ends up being convenient drugs, drugs for tough conditions like cancer and drugs that get really cheap when they go generic." Most medicines on the market today are small molecules, which can be taken by mouth, absorbed into the bloodstream and easily penetrate cell membranes. He noted it is not unusual for pharmaceutical companies to choose not to pursue a drug they once thought promising.

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