Radar on Specialty Pharmacy

March 13: The FDA approved BeiGene, Ltd.’s Tevimbra (tislelizumab-jsgr) for the treatment of adults with unresectable or metastatic esophageal squamous cell carcinoma after prior systemic chemotherapy that did not include a programmed death receptor-1 (PD-1)/programmed death-ligand 1 (PD-L1) inhibitor. Recommended dosing for the PD-1 inhibitor is 200 mg via intravenous infusion once every three weeks. The initial dose is administered over 60 minutes; if it is tolerated, subsequent doses can be administered over 30 minutes. The drug will be available in the second half of 2024.

March 13: The FDA gave an additional indication to Mirum Pharmaceuticals, Inc.’s Livmarli (maralixibat) for the treatment of cholestatic pruritus in people at least 5 years old with progressive familial intrahepatic cholestasis (PFIC). The agency first approved the ileal bile acid transporter inhibitor on Sept. 29, 2021. The new indication has orphan drug designation, as well as breakthrough therapy designation for PFIC type 2. The starting dose for the newest use of the oral solution is 285 mcg/kg once daily in the morning, increasing to 285 mcg/kg twice daily, then 428 mcg/kg twice daily and then 570 mcg/kg twice daily. Drugs.com lists the price of 9.5 mg/mL for 30 milliliters is more than $56,239.

The FDA recently approved a first-in-class agent to treat a particularly deadly cancer. The therapy, Iovance Biotherapeutics, Inc.’s Amtagvi (lifileucel), signifies a promising new development in the treatment of solid tumors, which represent about 90% of all cancers in the U.S. While the drug comes with a price tag of more than $500,000, as well as additional costs, payers’ experience with chimeric antigen receptor T cells (CAR-T) therapies should be helpful in their coverage of the new agent, say industry sources.

On Feb. 16, the FDA gave accelerated approval to Amtagvi for the treatment of adults with unresectable or metastatic melanoma previously treated with a programmed death receptor-1 (PD-1) inhibitor and, if BRAF V600 positive, a BRAF inhibitor with or without a MEK inhibitor. The agency gave the therapy orphan drug, regenerative medicine advanced therapy, fast track and priority review designations.

As CMS engages in the initial round of Inflation Reduction Act (IRA)-mandated drug price negotiations with manufacturers, one of the agents on the list of Medicare Part D drugs to be negotiated has certain aspects that make it a not-so-straightforward candidate. Stelara (ustekinumab) from the Janssen Pharmaceutical Companies of Johnson & Johnson has particular qualities that could result in unintended consequences, asserts one industry expert.

Stelara is unique among the first drugs to be negotiated in that it is available in both subcutaneous and intravenous formulations. The human interleukin-12 and -23 antagonist is approved for subcutaneous use for the treatment of people at least 6 years old with moderate to severe plaque psoriasis who are candidates for phototherapy or systemic therapy and people at least 6 with active psoriatic arthritis. It also is approved for the treatment of adults with moderately to severely active Crohn’s disease and adults with moderately to severely active ulcerative colitis, for whom treatment is initiated with a single intravenous dose, followed by subcutaneous maintenance dosing.

Specialty pharmacies and oncology practices dispensing costly specialty medications have long complained that Medicare Part D direct and indirect remuneration (DIR) fees are not appropriate for these drugs. Efforts to do away with these retroactive fees were finally successful, but revamped reimbursement has brought a new problem — underwater reimbursement — claims the Community Oncology Alliance (COA).

DIR includes rebates and price concessions that occur after the point of sale. According to CMS, total DIR “has been growing significantly in recent years.…In 2020, pharmacy price concessions accounted for about 4.8 percent of total Part D gross drug costs ($9.5 billion), up from 0.01 percent ($8.9 million) in 2010.”