Oncology

On Dec. 8, the U.S. Department of Commerce’s National Institute of Standards and Technology and HHS unveiled draft guidance (88 Fed. Reg. 85593) on framework for exercising march-in rights on taxpayer-funded drugs and other products. The framework “specifies that price can be a factor in considering whether a drug is accessible to the public,” the administration said. That was one of other “new actions to promote competition in health care and support lowering prescription drug costs for American families.” Comments must be received by 5 p.m. Eastern on Feb. 6.

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.

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.”

The specialty drug landscape continues to be a dynamic space, as new agents enter the market and existing ones gain FDA approval for additional indications. Global health care and specialty pharmacy solutions organization AscellaHealth recently released its quarterly breakdown of insights into treatments within the segment.

The Q3 2023 Specialty & Rare Pipeline Digest examines new approvals and launches of specialty drugs, including biosimilars, generics, and cell and gene therapies, as well as ones in the pipeline.

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.

Boehringer Ingelheim’s adalimumab-adbm interchangeable biosimilar is now available at an 81% discount to the price of its reference drug, AbbVie Inc.’s Humira (adalimumab), the manufacturer said Oct. 2. In early July, the company launched a branded version of the agent, Cyltezo, at a 5% discount. Both are citrate-free formulations available in various strengths for multiple inflammatory conditions.

Takeda Pharmaceuticals U.S.A., Inc. will voluntarily withdraw Exkivity (mobocertinib) after the Phase III EXCLAIM-2 confirmatory trial did not meet its primary endpoint, the company disclosed Oct. 2. The FDA gave the kinase inhibitor accelerated approval on Sept. 15, 2021, for the treatment of adults with locally advanced or metastatic non-small cell lung cancer with epidermal growth factor receptor (EGFR) exon 20 insertion mutations, as detected by an FDA-approved test, whose disease has progressed on or after platinum-based chemotherapy. The company says it made the decision in consultation with the FDA and is working with the agency on withdrawal timing. It also is working with other regulatory agencies to withdraw the drug globally. In the meantime, Takeda says it is working to make sure that people receiving Exkivity can maintain access to the medication.

The specialty drug landscape continues to be a dynamic space, as new agents enter the market and existing ones gain FDA approval for additional indications. Global health care and specialty pharmacy solutions organization AscellaHealth recently released its quarterly breakdown of insights into treatments within the segment.

The Q3 2023 Specialty & Rare Pipeline Digest examines new approvals and launches of specialty drugs, including biosimilars, generics, and cell and gene therapies, as well as ones in the pipeline.

Among the highlighted agents in the recently released report were Alzheimer’s disease drug Leqembi (lecanemab-irmb) from Eisai Inc. and Biogen, myasthenia gravis therapy Rystiggo (rozanolixizumab-noli) from UCB, Inc. and Pfizer Inc.’s alopecia areata medication Litfulo (ritlecitinib).

Sept. 8: The FDA approved BioLineRx Ltd.’s Aphexda (motixafortide) in combination with filgrastim to mobilize hematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in people with multiple myeloma. The agency gave the agent orphan drug designation. Dosing for Aphexda is 1.25 mg/kg based on actual body weight via subcutaneous injection after filgrastim has been administered daily for four days and 10 to 14 hours prior to the start of apheresis. The drug’s price is $5,900 per vial, and the company has said that it expects most patients will need two vials.

Sept. 8: The FDA expanded the label of Sandoz Inc.’s Hyrimoz (adalimumab-adaz) to include the treatment of adults with non-infectious intermediate and posterior uveitis and panuveitis. The agency first approved the biosimilar of AbbVie Inc.’s TNF inhibitor Humira (adalimumab) on Oct. 30, 2018. Dosing starts with 80 mg via subcutaneous injection, followed by 40 mg every other week starting one week after the initial dose. The agent is available in a low-concentration, citrate-free version of its reference drug. The product launched in July with two wholesale acquisition costs: a branded version with a WAC of $6,576 and an unbranded one for $1,315 for a carton of two syringes or autoinjectors.

Prominent insurance brokers are expecting large percentage increases in employer-plan costs next year, brought on by factors like rising labor costs and higher health care utilization. But there isn’t likely to be any relief in coming years, and experts say plan sponsors will have to be creative and disruptive if they hope to cushion the blow of increasing expenses.

Brokers Mercer Inc., Aon PLC and WTW PLC project that average employer plan costs will increase between 5.4% and 8.5% in plan year 2024.

And while employers don’t have to pass that onto employees in the form of higher monthly plan rates, some industry experts expect rising premiums are on the horizon, too.