Hereditary angioedema (HAE) is a high-cost category with an increasing number of therapies that treat the condition. To help stakeholders absorb datapoints and perceptions from all directions and synthesize those insights into a tangible strategy for HAE, Zitter Insights surveys a leading panel of pharmacy and therapeutics (P&T) decision makers at payers and integrated delivery networks. The Managed Care Biologics and Injectables Index: Hereditary Angioedema combines deep payer insights with the industry standard in market access information to produce quarterly reports and insights summaries on leading specialty brands.
Payers with more than one-third of commercial lives said that managing branded treatments for HAE is a high or very high priority, while those covering less than half of Medicare lives rated it an average priority. Payers representing more than one-third of commercial lives and more than half of Medicare lives said they are somewhat aggressive in their management of the therapies. More than half of physicians expressed high satisfaction with the available agents to treat the disease.
Around half of both payers and physicians stated that high unmet need exists in the treatment of HAE. More than four-fifths of physicians said that people with the condition have a high level of disease burden. Almost half of payers said that available treatments have an average budget impact. Almost two-thirds of providers said that patients have high out-of-pocket spending on treatments for HAE.
In the year prior to the survey, providers prescribed BioCryst Pharmaceuticals, Inc.’s Orladeyo (berotralstat) to the largest amount of unique patients, followed closely by Takeda Pharmaceuticals U.S.A., Inc.’s Cinryze (C1 esterase inhibitor [human]) and CSL Behring’s Berinert (C1 esterase inhibitor [human]). Cinryze had the greatest number of high-prescribing physicians, which are those who prescribe a product to at least 80% of patients, while Takeda’s Kalbitor (ecallantide) was the agent with the most low-prescribing physicians, which prescribe a product to 5% or less of their patients. The drug with the highest percentage of prior authorization requests denied upon first request was Pharming Healthcare, Inc.’s Ruconest (C1 esterase inhibitor [recombinant]), while Berinert had the lowest percentage of first-time PA request denials.
KalVista Pharmaceuticals, Inc.’s sebetralstat is an oral plasma kallikrein inhibitor that is in a Phase III clinical trial as an on-demand therapy for HAE attacks. Payers representing almost half of commercial lives and more than one-third of Medicare lives said they are moderately aware of the agent. Payers with around half of both commercial and Medicare covered lives said that if the drug is approved, they expect it to have a moderate impact on the other available treatments for HAE, meaning that some products’ tiering and/or utilization management criteria are likely to change. Almost half of physicians said that sebetralstat would have a moderate impact on their treatment approach for a considerable portion of patients.
Payers, oncologists and practice managers rated manufacturers of HAE treatments across a range of performance metrics: overall commitment to the disease, account representative support, patient and family support programs and resources (excluding copay assistance), patient copay assistance, physician education and support, and hub services. Payers gave Pharming top marks in five of the six metrics and said CSL Behring was best at overall commitment to the disease. Physicians said that CSL Behring was the top HAE therapy manufacturer in five of the categories and cited BioCryst as No. 1 in overall commitment to the disease. Practice managers’ responses were more varied: They said Pharming was tops in overall commitment to the disease and hub services, named BioCryst the best at patient copay assistance and cited CSL Behring as No. 1 in account representative support, patient/family support programs/resources and physician education/support.
Message: “The three main types of HAE are Type 1, affecting 85% of patient population; Type 2, affecting 15% of patient population; and, Type 3, which is extremely rare. Usually, early childhood attacks equates to a worse condition, and untreated patients tend to have attacks up to four days per month.”
Payer Thoughts: “Ensuring there is adequate access to patients for both acute and chronic conditions is vital to prevent higher ancillary costs and even mortality.”
FDA Expands Patient Population of Takeda’s Takhzyro
On Feb. 3, 2023, the FDA expanded the patient population for Takeda’s Takhzyro (lanadelumab-flyo) to include the prevention of hereditary angioedema attacks in people at least 2 years old. The agency initially approved the plasma kallikrein inhibitor on Aug. 3, 2018. Dosing of the subcutaneous injectable in people at least 12 years old is 300 mg every two weeks. In people at least 6 and less than 12, dosing is 150 mg every two weeks, and for people 2 years old and younger than 6, dosing is 150 mg every four weeks. Drugs.com lists the price of one single-dose 300 mg/2 mL subcutaneous solution as more than $26,165.
FDA Approves First Oral Therapy for Prevention of HAE Attacks
When the FDA approved BioCryst Pharmaceuticals, Inc.’s Orladeyo (berotralstat) last month, the drug became the first oral treatment for prophylaxis to prevent hereditary angioedema (HAE) attacks. According to Zitter Insights, payers with nearly three-quarters of covered lives plan to manage it at parity to other prophylactic treatments.