Costs Unsustainable for New Angioedema Preventive Therapies in US, Real-world Data Show

Costs are unsustainable for preventive treatment of hereditary angioedema (HAE) attacks with approved therapies Takhzyro or Haegarda, according to an analysis of six-month real-world data performed by the pharmacy benefit manager Prime Therapeutics.

In addition, on-demand therapy plus Takhzyro cost $26,000 more than the same regimen plus Haegarda.

“Due to the remarkable expense of these treatments, value-based arrangements and preferred product strategies should be considered,” Kevin Bowen, MD, principal health outcomes researcher at Prime Therapeutics, said in a press release.

The analysis by Prime was presented at the Academy of Managed Care Pharmacy 2020 virtual learning event, April 20–24, in a poster titled “Hereditary Angioedema (HAE) Real-World Prophylactic and On-demand Treatment Cost in a 15 Million Commercially Insured Population: Comparison of C-1 Inhibitor (Haegarda®) versus Lanadelumab (Takhzyro®) Treated Members.”

The U.S. Food and Drug Administration (FDA) has approved three preventive treatments (prophylaxis) for HAE attacks: Cinryze and Takhzyro, both by Shire (now part of Takeda Pharmaceuticals), and Haegarda by the Australian biopharmaceutical company CSL Behring.

Haegarda and Takhzyro, both given as an under-the-skin injection, were approved in the past two years as long-term preventive treatment for HAE attacks. However, little is known about their real-world use or costs.

Among Prime’s 15 million insured members, 58 were new to treatment with Haegarda (29 patients, mean age 40.1 years) or Takhzyro (29 patients, mean age 43.5 years). These patients were treated with only one of these therapies. Moreover, they had been enrolled in Prime before, during, and after the preventive therapy.

The analysis compared the cost of the two therapies over a period of six months (180 days). Cost was defined as the plan plus member cost after network discounts.

Results showed that over the six months, the mean cost for Haegarda ($226,989) was about $51,000 lower than for Takhzyro ($278,267).

On-demand therapies, sometimes needed to deal with swelling of the face, throat, or intestines, were used by 62.1% (18 out of 29) of patients given Haegarda and 65.5% (19 out of 29) of those given Takhzyro. The mean cost of on-demand therapy for six months among patients given Haegarda was $108,025, and it was $82,591 for those taking Takhzyro, a difference of $25,434 and considered not statistically different.

On-demand therapies included Berinert by CSL Behring, and Kalbitor or Firazyr, both by Takeda.

The total costs (preventive plus on-demand therapy) over six months was $361,000 for those taking Takhzyro and $335,000 for those on Haegarda — a difference of $26,000 more for those using Takhzyro plus on-demand therapy.

Overall, and despite the small sample of patients analyzed, these real-world data showed that during the first six months, preventive treatment with Takhzyro was more than $50,000 higher than Haegarda treatment. However, no differences were seen in “the percentage of members with any use of on-demand HAE agents and no significant difference in the mean cost of on-demand therapy, although Takhzyro averaged $25,500 less,” the researcher wrote.

Taken together, Prime concluded that preventive HAE treatment with Takhzyro or Haegarda carries unsustainable costs, with Takhzyro being more expensive.

“These study results are meaningful despite the small sample size,” said Bowen. “Taking the real-world cost data of both medical and pharmacy claims to evaluate two FDA recently approved prophylaxis HAE drugs is extremely important to help make critical decisions about formulary placement, contracting with manufacturers and the most cost-effective treatment of members.”