Specialty drugs are expected to comprise 80% of the FDA approvals this year. Among them are gene therapies for hemophilia A and Duchenne muscular dystrophy priced at between $2 million and $3 million.
“We're in a whole new world. The specialty world has taken over,” said Jeffrey Casberg, M.S.
Casberg, and his colleague, Leslie Fish, Pharm.D.. both senior vice presidents of clinical pharmacy at IPD Analytics, a pharmaceutical analytics and consulting firm, gave an hourlong tour of that world on the final day of the annual meeting of the Academy of Managed Care Pharmacy, which was held in San Antonio.
More specifically, they thumbnailed the 31 specialty drugs in late-stage development that have been or are likely to be approved by the FDA this year. It is a group that includes the first gene therapies for hemophilia A and Duchenne muscular dystrophy, the first treatment for a rare neurological disorder called Friedreich’s ataxia that may have off-label uses, a third anti-beta amyloid drug for Alzheimer’s disease, an oral treatment for Clostridioides difficile that might be preferable to treatment delivered rectally that is currently on the market, and two different vaccines for respiratory syncytial virus, one of which may become associated with Magic Johnson.
Nearly 80% of the drugs that the FDA is expected to approve this year are specialty drugs, a category that is getting increasingly difficult to define but includes drugs for relatively rare diseases or need special handling. Most of the specialty drugs are costly. “The average price is probably 2, 3 $400,000 (per year). Ten years ago, it was a fraction of that,” Casberg said in a short interview he and Fish gave their presentation. Casberg noted that the costs are borne by insurers and maybe especially commercial insurers because Medicare is pushing down on drug prices. “The commercial (insurers) are really going to take a hit. I would watch out for commercial premiums,” he said.
Fish noted that many of specialty drugs are approved through the FDA’s accelerated approval pathway, which has lower standards that the agency’s full approval process.
“We're approving these drugs, and we're paying a very high cost, not really knowing what true outcomes are,” she said in a post-presentation interview. Fish said the accelerated approval pathway is needed but has sometimes been abused.
She pointed to Roctavian (valoctocogene roxaparvovec), the gene therapy, as a counterexample. The FDA delayed the treatment’s approval because it wanted more proof that the effects of the one-time gene therapy would last. The FDA is scheduled to decide on whether to approve the treatment in June after the manufacturer, BioMarin Pharmaceutical, submitted more data, Fish said.
Here are few of the treatments that Casberg and Fish discussed in their pipeline review: