Gene Therapy Has Arrived. But So Will the $$$$$ Bills. | AMCP Nexus 2022

Gene therapy, once the stuff of scientific aspiration, is fast becoming a clinical reality in U.S. healthcare but with staggering price tags. In her keynote talk today on specialty drugs at AMCP Nexus 2022, Aimee Tharaldson, Pharm.D., listed 12 approved gene therapies and another seven that could be approved next year.

Tharaldson, senior clinical pharmacist, emerging therapeutics, at Evernorth, told the large audience for her morning talk that 500 gene therapies are in clinical trials and that as many 1 million Americans could be treated with a gene therapy by 2034.

But Tharaldson also noted the huge cost associated gene therapy, especially the one-time gene replacement therapies that are priced in the millions of dollars. Citing a paper from the National Bureau of Economic Research, Tharaldson said the cumulative annual cost of gene therapies could reach $25 billion by 2034.

“It is going to be interesting to see what happens because they are very expensive,” Tharaldson said in a brief interview after her talk, which drew a large audience on the third day of the AMCP Nexus meeting in National Harbor, Maryland. “Some of them are coming to market with pretty good data and some will likely come to market with not the best data. They are just going to need to be evaluated carefully to make sure they are effective and safe and have a durable effect.”

Some of the gene therapies have been promoted as curative but Tharaldson sounded a note of caution :“I don’t think we have enough information at this point to say they're curative.”

Tharaldson said one of the gene therapies that the FDA may approve next year is delandistrogene moxeparvovec, a treatment for Duchenne muscular dystrophy. The drug's manufacturer, Sarepta, may price the treatment at $3 million.

Three of the gene therapies that might be approved next year are for dystrophic epidermolysis bullosa, a rare skin disease, she said.

Tharaldson also listed eight hemophilia gene therapies that are in late-stage development and could be approved over the next several years. One, etranacogene dezaparvovec, may be approved next month.

Value-based contracts have been discussed as one way to possibly cope with the cost impacts of the gene therapies. Tharaldson mentioned that Bluebird Bio has proposed a value-based contract for Zynteglo (betibeglogene autotemcel), its gene therapy for beta thalassemia, a rare blood disorder that results in low hemoglobin levels. Bluebird priced the one-time gene replacement therapy, which was approved by the FDA in August 2022, at $2.8 million, but the company has also said that it would reimburse payers for perhaps as much as 80% of that cost if patients do not stay "transfusion independent" for two years.

The FDA approved another Bluebird gene replacement therapy, Skysona (elivaldogene autotemcel), a treatment for cerebral adrenoleukodystrophy, a rare neurological condition, in September 2022. The price of $3 million is similar to price Zynteglo but Tharaldson said Bluebird hasn't offered any value-based terms for Skysona.

Here are some of the other highlights from Tharaldson’s information-dense talk

• Biosimilars. The FDA has approved 39 biosimilars and 23 of them have come on the marketplace, according to Tharaldson. She said 10 Humira biosimilars are expected to come on the market next year. But Enbrel (etanercept) is not expected to see biosimilar competition till 2029.
• Inflammatory conditions. Inflammatory conditions, which include chronic autoimmune conditions such as rheumatoid arthritis and Crohn’s disease, continue to be the most expensive therapy class on a per member, per year basis. Tharaldson listed eight drugs in the pipeline for inflammatory conditions, including three for ulcerative colitis (mirikzumab, etrasimod and risankizumab-rzaa). She also discussed four drugs under development for atopic dermatitis, which is also inflammatory conditions
• Multiple sclerosis. Tharaldson listed six drugs in the multiple sclerosis pipeline. Approval of one of them — ublituximab — is expected this year but the other five on her list were will not be approved till 2024 and 2025.
• Alzheimer’s disease. Despite the checkered history with Aduhelm (aducanumab), many drugs based on the hypothesis that Alzheimer’s is caused by beta amyloid deposits in the brain remain on the market. Tharaldson had three of them on pipeline list: lecanemab (which may get approved in January 2023) donanemab and gantenerumab. A fourth drug on her list, semorinemab, is based on an alternative theory that Alzheimer’s is more directly caused by clumps of tau protein.