A review of market access for acute lymphoblastic leukemia treatments shows that under the pharmacy benefit, about 39% of the lives under commercial formularies are covered with utilization management restrictions. Around 33% of the lives under Medicare formularies are not covered for at least one of the drugs.
Under the medical benefit, about 59% of the lives under commercial policies are covered with utilization management restrictions. Almost 67% of the lives under Medicare policies have access to at least one of the drugs without utilization management restrictions.
For about 95% of the covered lives, payer pharmacy benefit formularies do not require step therapy (ST). Of the lives that require ST, about 31% of the lives require multiple steps. Around 69% of payer-controlled pharmacy benefit covered lives require prior authorization, with 5% of those lives covered by policies that are restrictive as compared with a product’s FDA-approved label.
In June 2023, the FDA converted accelerated approval to full approval for Amgen Inc.’s Blincyto (blinatumomab) for the treatment of people with CD19-positive B-cell precursor acute lymphoblastic leukemia in first or second complete remission with minimal residual disease greater than or equal to 0.1%. The agency first approved the CD19-directed CD3 T-cell engager on Dec. 3, 2014. Dosing consists of 28 days of continuous intravenous infusion via an infusion pump and then a 14-day treatment-free interval. For a treatment course of four cycles, people weighing at least 45 kg are administered 28 mcg/day for the first 28 days of each cycle. People weighing less than 45 kg are given 15 mcg/m2/day on the same schedule. Drugs.com lists the price of one 35 mcg lyophilized powder in a single-dose vial as more than $5,169.
In November 2022, the FDA approved a new dosing regimen for Jazz Pharmaceuticals plc’s Rylaze (asparaginase erwinia chrysanthemi [recombinant]-rywn) as a component of a multiagent chemotherapeutic regimen for the treatment of acute lymphoblastic leukemia and lymphoblastic lymphoma in people at least 1 month old who have developed hypersensitivity to E. coli-derived asparaginase. The new regimen allows people to receive 25 mg/m2 via intramuscular injection on Monday and Wednesday mornings and 50 mg/m2 on Friday afternoons. The original dosing schedule of 25 mg/m2 intramuscularly every 48 hours — approved June 30, 2021 — remains an option. The review for the newest approval was done through Project Orbis in collaboration with the Australian Therapeutic Goods Administration, Health Canada and Switzerland’s Swissmedic; it also used the Real-Time Oncology Review pilot program. Rylaze has orphan drug designation. Drugs.com lists the price of one 10 mg/0.5 mL vial as more than $14,382.
In November 2021, the FDA approved Zydus Cadila’s nelarabine 250 mg/50 mL (5 mg/mL) for the treatment of people with T-cell acute lymphoblastic leukemia and people at least 1 year old with T-cell lymphoblastic lymphoma whose disease has not responded to or has relapsed following treatment with at least two chemotherapy regimens. It is the first approved applicant for the generic of Novartis Pharmaceuticals Corp.’s Arranon and has 180 days of market exclusivity. Dosing for adults is 1,500 mg/m2 via a two-hour intravenous infusion on days one, three and five repeated every 21 days. Pediatric dosing is 650 mg/m2 through a one-hour intravenous infusion for five consecutive days repeated every 21 days. Website Drugs.com lists the price of a 5 mg/mL vial as more than $777.
Market Events Drive Changes
In June 2023, the FDA converted accelerated approval to full approval for Amgen Inc.’s Blincyto (blinatumomab) for the treatment of people with CD19-positive B-cell precursor acute lymphoblastic leukemia (ALL) in first or second complete remission with minimal residual disease greater than or equal to 0.1%. In November 2022, the agency approved a new dosing regimen for Jazz Pharmaceuticals plc’s Rylaze (asparaginase erwinia chrysanthemi [recombinant]-rywn) as a component of a multiagent chemotherapeutic regimen for the treatment of ALL and lymphoblastic lymphoma in people at least 1 month old who have developed hypersensitivity to E. coli-derived asparaginase. In May 2022, the FDA withdrew its approval of the new drug application for Acrotech Biopharma’s Marqibo (vinCRIStine sulfate LIPOSOME injection), which was approved for the treatment of adults with Philadelphia chromosome-negative ALL in second or greater relapse or whose disease has progressed following at least two anti-leukemia therapies.
Competitive Market Landscape
Novartis Pharmaceuticals Corp.’s Kymriah (tisagenlecleucel) was the first FDA-approved chimeric antigen receptor T-cell therapy indicated for patients up to 25 years of age with relapsed or refractory B-cell ALL, while Tecartus (brexucabtagene autoleucel) from Kite Pharma, Inc., a Gilead Sciences, Inc. company, was the first CAR-T approved for adults with ALL. There is no current cure for ALL except an allogeneic hematopoietic stem cell transplant (HSCT), which has a high cure rate.
Pharmacy, Medical Benefit Implications
The first-line treatment for many of the variants of ALL is to enroll eligible patients into clinical trials. If this is not done, most medications require prior authorization. Most drugs are covered under either the pharmacy or medical benefit, and coverage is dependent upon the route of administration.