In the past year, medicines and therapeutics have made big strides—from first-in-class treatments to therapeutic milestones and a once-unthinkable “functional cure.”
That latter distinction belongs to Casgevy, the first CRISPR-based therapeutic approved by the Food and Drug Administration—which came to fruition less than a decade after the gene-editing technology was first developed. Developed by Vertex Pharmaceuticals, in partnership with CRISPR Therapeutics, Casgevy is a treatment for sickle cell disease that could benefit some 16,000 patients. But even as the Biden administration negotiates discounts for state Medicaid plans, the drug’s uptake could be limited by its $2.2 million price tag.
High list prices have become a hallmark of the most advanced therapies. Take the latest drug meant to help slow the progression Alzheimer’s disease. The therapy, Leqembi, offers unprecedented hope for the 6.7 million older Americans living with its effects. Developed by Eisai with partner Biogen and granted FDA full approval in July 2023, the infused therapy is the first treatment to clearly slow cognitive decline and reduce amyloid plaques in the brain, whose buildup is a key driver of the disease. About 1 million Alzheimer’s patients in the U.S. could qualify for the treatment, and the drug’s annual price tag of $26,500 along with frequent scans and monitoring could cost taxpayers $82,500 per patient per year, according to estimates from the nonprofit Institute for Clinical and Economic Review. Six months after approval, between 2,000 and 3,000 people in the U.S. were receiving the therapy.
But without a doubt, the top drug-cost story of 2023 was about category of blockbuster diabetes anti-obesity drugs known scientifically as GLP-1 receptor agonists. They’re better known by their brand names Ozempic, Wegovy, Mounjaro, and Zepbound. Developed and manufactured by longtime rivals Novo Nordisk and Eli Lilly, these drugs have rewritten the rules of weight loss and caused a profound shift in the pharma landscape.
“Medicaid and [many] insurance companies don’t even cover weight-loss drugs, yet this category is on track to be $50 billion to $100 billion, or more,” says Tim Opler, managing director in Stifel’s Global Healthcare Group. Despite retail pricing of around $1,000 a month, the widespread consumer demand “means that all of a sudden we have two pharma companies that can tell the US government and United Health to jump in a lake,” Opler says. There are also signs that the costs listed are not always what insurers or patients end up paying.
At the same time, Novo Nordisk has been working to show that semaglutide—the miracle molecule in Ozempic, Wegovy, and Rybelsus—is more than a weight-loss bombshell. Ongoing clinical trials are exploring the drug’s potential use in conditions including liver and kidney disease, substance abuse disorder, and Alzheimer’s. And in March, the FDA greenlit Wegovy for the treatment of cardiovascular disease in obese and overweight patients. That approval was based on a years-long study showing marked reductions in heart attack, stroke, and death in people taking the drug. The new indication could increase pressure on payers to cover these drugs, and on drug makers to negotiate their price.
In a bright spot for access and pricing to medications, in March, retailers and online pharmacies started selling Perrigo’s daily contraceptive Opill, the first over-the-counter birth-control pill ever available in the U.S. The price of $20 for a one-month supply, or $90 for six months, may also be covered by some insurance plans.
Explore the full 2024 list of Fast Company’s Most Innovative Companies, 606 organizations that are reshaping industries and culture. We’ve selected the firms making the biggest impact across 58 categories, including advertising, artificial intelligence, design, sustainability, and more.