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Tis Full and Very, Very Expensive: the 2023 Specialty Drug Pipeline | AMCP 2023

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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:

  • Zuranolone, a GABA-A modulator, for major depressive disorder and postpartum disorder, is similar to Zulresso (brexanolone) but is taken orally. They said it would be priced the same as Zulresso at about $23,000 a year.
  • Delandistrogene moxeparvovec would be the first gene therapy for Duchenne muscular dystrophy, according to Fish and Cassberg, and is expected to be price at between $2 million and $3 million. A competing gene therapy developed by Pfizer could be approved next year.
  • Donanemab would be the third anti-beta amyloid treatment for Alzheimer’s disease after Leqembi (lecanemab-irmb) and controversial Aduhelm (aducanumab). The drug was denied accelerated approval in January but Eli Lilly is expected to fill an application for full approval this year, which would likely mean an FDA decision in 2024.
  • The FDA approved Skyclarys (omaveloxolone) for Friedreich’s ataxia in February. Fish and Casberg estimate that between 4,000 and 5,000 people might be prescribed the treatment, but Fish said that its mechanism of action may invite off-label use for other conditions. Skyclarys is priced to cost $370,000 a year (the wholesale acquisition price)
  • Vyjuvek (beremagene geperpavec) is a gene-therapy gel for dystrophic epidermolysis bullosa, a rare skin condition that can cause blisters and “erosions” (open wounds). Fish noted that Vyjuvek involves ongoing treatment and is exception to the usual “one-and-done” paradigm of gene therapy. It is priced to cost about $300,000 a year, although the costs will vary depending on how often the erosions appear and respond to the treatment.
  • Seres Therapeutics, based in Cambridge, Massachusetts, has developed an oral treatment for C. difficile that could compete with Rebyota (fecal microbiota, live-jslm), which is administered rectally. The treatment, known provisionally as SER-109, consists oflive purified Firmicutes bacterial spores. An FDA approval decision is scheduled for April 26, 2023. Casberg and Fish said the oral treatment would be priced between $10,000 and $25,000 per treatment course, and a treatment course consists of four capsules a day for three days.
  • The FDA is expected to approve two RSV vaccines in May 2023: Pfizer’s Arexvy and GSK’s Abrysvo, according to Casberg and Fish. GSK has hired Johnson to be a spokesperson and social media influencer for its Sideline.RSV campaign.
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