Radar on Specialty Pharmacy

Bayer recently revealed that it will work with the FDA to voluntarily withdraw the New Drug Application (NDA) for its cancer drug Aliqopa (copanlisib). The therapy is the latest phosphoinositide 3-kinase — also called phosphatidylinositol 3-kinase — (PI3K) inhibitor/indication with accelerated approval to treat a hematologic malignancy to be pulled from the U.S. market, potentially spurring payers to take a closer look at these agents.

The agency gave Aliqopa accelerated approval on Sept. 14, 2017, for the treatment of adults with relapsed follicular lymphoma (FL) who have received at least two systemic therapies. Approval was based on the CHRONOS-1 Phase II clinical trial. In the confirmatory study, CHRONOS-4, adding Aliqopa to standard immunochemotherapy regimens did not meet the primary endpoint of progression-free survival (PFS) vs. the standard immunochemotherapy control arm. Bayer says it will publish the trial results “in a timely manner.”

Scrutiny of the FDA’s accelerated approval process has shown no signs of slowing, with an FDA committee holding a recent meeting on the program’s use for oncology indications, as well as on two cancer drugs lacking confirmatory trial data long after their accelerated approvals. And an FDA leader recently asserted his stance on granting the designation only when companies already have started postmarketing trials. Still, these developments should not dampen interest by either manufacturers or payers in drugs hitting the market via the accelerated pathway, says one industry expert.

The FDA established the accelerated approval pathway in 1992 to bring HIV/AIDS medicines onto the market sooner during the ongoing epidemic. Between that time and Dec. 31, 2020, the agency has granted more than 253 accelerated approvals, according to a 2021 report from the Institute for Clinical and Economic Review (ICER) examining the designation. Of those, 125 — 49.4% — later received full approval, with a median time of 3.2 years from accelerated approval to full. Sixteen — 6.3% — of the indications have been withdrawn, and the remaining 112 drugs have been on the market for a median of 1.9 years.

On March 23, 2010, then-President Barack Obama signed the Affordable Care Act (ACA) into law, establishing the 351(k) biosimilar pathway via the Biologics Price Competition and Innovation Act (BPCIA), which amended the Public Health Service (PHS) Act. Since then, the FDA has approved more than 40 biosimilars, with only a handful of those gaining interchangeable status. That designation, however, may not carry quite the distinction it historically has had if proposed guidance from the agency — which would replace previous guidance — on labeling for interchangeable biosimilars is finalized. At the same time, the change may help promote uptake of biosimilars, bringing cost savings to the U.S. health care system, say industry sources.

In contrast to the EU, whose European Medicines Agency (EMA) and the Heads of Medicines Agencies (HMA) clarified in September 2022 that all biosimilars approved in the EU are interchangeable, the FDA has created two levels of biosimilars: biosimilars and interchangeable biosimilars.

Celltrion USA, Inc. signed a contract with Ventegra to include its Yuflyma (adalimumab-aaty) as a preferred drug on its formulary in both public and private insurance markets, the manufacturer said Oct. 5. Ventegra is a “major U.S. Medical Benefits Manager (MBM) who administers pharmacy benefits through its Pharmacy Services Administration (PSA) model that has been effectively displacing traditional” PBMs. The move gives Celltrion access to approximately 3.6% of the U.S. The company says it will increase its marketing so that the drug has “coverage and/or access for 40% of the U.S. population by the end of the year.” Yuflyma, a high-concentration biosimilar of AbbVie Inc.’s Humira (adalimumab), launched in July with a wholesale acquisition cost of $6,576. Celltrion also said that Yuflyma has been added to CarePartners Pharmacy’s cost savings programs. With its partners, the specialty pharmacy has access to more than 10 million plan members. It is fully accredited and licensed to service patients in all 50 states and Washington, D.C. According to The Korea Economic Daily, “CarePartners will sidestep the original Humira from its roster and refrain from enlisting other competing adalimumab biosimilars, effectively making Yuflyma the sole adalimumab product in its offerings.”

Since the FDA approved the first biosimilar — Zarxio (filgrastim-sndz) from Sandoz, then a division of Novartis Pharmaceuticals Corp. — on March 6, 2015, the agency has approved more than 40 additional agents via the 351(k) pathway established under the Biologics Price Competition and Innovation Act (BPCIA), itself part of the Affordable Care Act (ACA). This past year has been especially busy in the space, with highlights including the launch of nine biosimilars of AbbVie Inc.’s Humira (adalimumab) and approvals of the first biosimilars of three different biologics: Biogen’s Tysabri (natalizumab), Actemra (tocilizumab) from Genentech USA, Inc., a member of the Roche Group and Stelara (ustekinumab) from the Janssen Pharmaceutical Companies of Johnson & Johnson. As the FDA approves more biosimilars, uptake of these agents will continue to increase, say industry experts.

As the PBM and specialty pharmacy worlds have become more integrated, PBMs now are providing more specialty management services than they previously did. Only 15% of respondents to a recent survey said they carve out specialty drugs from the PBM that is providing their pharmacy benefit management, while 26% said they were considering taking this step, according to the 2023 Pharmacy Benefit Manager Customer Satisfaction Report from Pharmaceutical Strategies Group (PSG), an EPIC company.

The 85% of respondents whose PBMs provide both specialty and traditional pharmacy benefit management ranked formulary management of specialty drugs and financial reporting in the pharmacy benefit as the services they were most satisfied with, while they were least satisfied with management of specialty agents in the medical benefit and offering competitive discounts on specialty medications.

The FDA recently approved the first biosimilar of Actemra (tocilizumab) from Genentech USA, Inc., a member of the Roche Group, for multiple indications. Both payers and rheumatologists responding to a Zitter Insights survey said they expected the new drug to have some impact on their management of and prescribing for rheumatoid arthritis (RA). However, the drug’s lack of an additional formulation may hurt its uptake, say industry sources.

On Sept. 29, the FDA approved Bio-Thera and Biogen Inc.’s Tofidence (tocilizumab-bavi) intravenous formulation for the treatment of adults with moderately to severely active rheumatoid arthritis who have had an inadequate response to at least one disease-modifying antirheumatic drug (DMARD), people at least 2 years old with active polyarticular juvenile idiopathic arthritis and people at least 2 years old with active systemic juvenile idiopathic arthritis. Dosing of the intravenous infusion is based on the indication.

Oct. 3: The FDA granted interchangeability to Biogen Inc. and Samsung Bioepis Co., Ltd.’s Byooviz (ranibizumab-nuna) for the treatment of neovascular (wet) age-related macular degeneration, macular edema following retinal vein occlusion and myopic choroidal neovascularization (mCNV). The agency first approved the biosimilar of Lucentis (ranibizumab) from Roche Group member Genentech USA, Inc. on Sept. 17, 2021. The vascular endothelial growth factor (VEGF) is administered by intravitreal injection once a month. Dosing for mCNV is up to three months. Drugs.com lists the price of a single-dose 10 mg/mL for 0.05mL vial as more than $1,199.

Oct. 11: The FDA expanded the approval of Fresenius Kabi’s Idacio (adalimumab-aacf) for the treatment of adults with moderate-to-severe hidradenitis suppurativa. The agency first approved the biosimilar of AbbVie Inc.’s tumor necrosis factor (TNF) blocker Humira (adalimumab) on Dec. 13, 2022. Dosing starts with 160 mg via subcutaneous injection on day one or split over two consecutive days, then 80 mg on day 15, and then on day 29 and subsequent doses, 40 mg every week or 80 mg every other week. The price of two single-dose prefilled pens or two single-dose prefilled glass syringes is $6,576.

The first biosimilar for the treatment of multiple sclerosis recently received FDA approval, and when it launches, Tyruko (natalizumab-sztn) from Polypharma Biologics and Sandoz Inc. will enter a competitive therapeutic class that is a high priority for management. Payers have said they expect the new treatment to have a moderate impact on their management of the other agents available to treat the condition.

On Aug. 24, the FDA approved Tyruko for the treatment of two indications: (1) adults with relapsing forms of multiple sclerosis, including clinically isolated syndrome, relapsing-remitting disease and active secondary progressive disease, and (2) adults with moderately to severely active Crohn’s disease with evidence of inflammation who have had an inadequate response to, or are unable to tolerate, conventional Crohn’s therapies and tumor necrosis factor (TNF) inhibitors.

A U.S. district court judge recently struck down a federal rule allowing health plans to not count copayment assistance against members’ out-of-pocket costs. The move clears the way for plans to have to discontinue use of copay offset programs such as copayment accumulators, which have been popular tools to keep their costs down and often impact specialty therapies. But as a result of the ruling, industry experts say that patient adherence likely will improve.

U.S. District Judge John D. Bates of the U.S. District Court for the District of Columbia issued the ruling on Sept. 29.

To help patients pay for costly specialty drugs, pharma manufacturers offer assistance that can help cover their out-of-pocket costs. Companies claim that the assistance helps improve patient adherence to medications that often treat rare and deadly conditions. But critics of them say such programs incentivize drugmakers to raise prices of these agents.