Radar on Specialty Pharmacy

Researchers continue to make progress in developing cell and gene therapies that offer the promise of slowing a disease’s progression to even offering a potential cure to patients. And while these agents may offer hope to patients, some challenges exist, including access to the treatments. In order for these products to reach their full potential, stakeholders must work together to overcome these potential barriers.

With its Feb. 28 FDA approval, the Janssen Pharmaceutical Companies of Johnson & Johnson and Legend Biotech USA, Inc.’s Carvykti (ciltacabtagene autoleucel; cilta-cel) became the sixth chimeric antigen receptor T-cell (CAR-T) therapy approved in the U.S. In addition, the existing CAR-Ts continue to get additional FDA-approved indications added to their labels, including for use in earlier line settings. And in August and September alone, the FDA approved two bluebird bio, Inc. gene therapies: Zynteglo (betibeglogene autotemcel or beti-cel) and Skysona (elivaldogene autotemcel or eli-cel).

The FDA recently approved the first novel long-acting granulocyte colony-stimulating factor (G-CSF) in more than 20 years. Payers say they are likely to manage the new agent similar to existing ones, but some oncologists have indicated that they are willing to prescribe it in place of other neutropenia agents, according to Zitter Insights. Still, the leader in the space has a unique quality that has allowed it to continue to retain market share, which may prove challenging for the new drug — at least for the time being.

On Sept. 9, the FDA approved Spectrum Pharmaceuticals, Inc.’s Rolvedon (eflapegrastim-xnst) to decrease the incidence of infection, as manifested by febrile neutropenia, in adults with nonmyeloid malignancies receiving myelosuppressive anti-cancer drugs associated with clinically significant incidence of febrile neutropenia. The company developed the drug with South Korea’s Hanmi Pharmaceutical Co. The recommended dose is 13.2 mg administered subcutaneously once per chemotherapy cycle.

As FDA approvals of biosimilars continue and agents expand into new indications, more payers are using these drugs and seeing cost savings through that utilization, according to Zitter Insights.

When the FDA approved Fresenius Kabi’s Stimufend (pegfilgrastim-fpgk) on Sept. 1, it was the sixth biosimilar of Amgen Inc.’s Neulasta (pegfilgrastim) that the agency had approved. It also was the 38th biosimilar approved since the first one, Novartis Pharmaceutical Corp. division Sandoz Inc.’s Zarxio (filgrastim-sndz), was approved March 6, 2015, referencing Amgen’s Neupogen (filgrastim).

Oct. 21: The FDA granted another indication to AbbVie Inc.’s Rinvoq (upadacitinib) for the treatment of adults with active non-radiographic axial spondyloarthritis with objective signs of inflammation who have had an inadequate response or intolerance to tumor necrosis factor (TNF) inhibitors. The agency first approved the Janus kinase (JAK) inhibitor on Aug. 16, 2019. Dosing of the extended-release tablets for the newest indication is 15 mg once daily. The wholesale acquisition cost (WAC) of a 30-day supply is $5,671.26.

Oct. 21: The FDA approved AstraZeneca’s Imjudo (tremelimumab-actl) in combination with the company’s Imfinzi (durvalumab) for the treatment of adults with unresectable hepatocellular carcinoma. Dosing for people weighing at least 30 kg is 300 mg of Imjudo, a cytotoxic T-lymphocyte-associated protein 4 (CTLA-4) inhibitor, via intravenous infusion one plus 1,500 mg of Imfinzi, a programmed death-ligand 1 (PD-L1) inhibitor, also via intravenous infusion at cycle one/day and then Imfinzi as a single agent every four weeks. For those weighing less than 30 kg, dosing is 4 mg/kg of Imjudo plus 20 mg/kg of Imfinzi at cycle one/day one and then Imfinzi every four weeks.

Biosimilars have produced about $21 billion in savings for the U.S. health care system over the past six years, according to the 2022 Amgen Biosimilar Trends Report. That competition is resulting in decreasing average sales prices (ASPs) for both biosimilars and their reference products, found the ninth edition of the report, and uptake of biosimilars continues to increase. In the second quarter of 2022, drug spend savings were estimated to be $3.2 billion. Biosimilars for more classes, pharmacy benefit drugs and interchangeable biosimilars are among the trends expected over the next few years.

Rheumatologists are reporting a growing familiarity with biosimilars, with three-quarters of respondents to a Cardinal Health survey saying they are “very familiar” with the agents compared with 53% who said that in 2020. The Rheumatology Insights: October 2022 report found that, among other things, almost two-thirds of respondents said they are “very comfortable” with prescribing the drugs. New patients and existing patients whose payers have mandated the use of a biosimilar are the top categories of patients for whom respondents are likely to prescribe biosimilars, both cited by about 40% of respondents.