Specialty Drugs

As manufacturers continue to bring more advanced therapies onto the U.S. market, payers are grappling with how to afford these agents. And while the products may be life-changing for some patients, they may not have the desired outcome in others, leaving payers on the hook for an unsuccessful treatment. This has resulted in various contracting opportunities, including warranties.

While such agreements can make payers more confident in their coverage of treatments, particularly high-cost ones and products whose effectiveness or durability are uncertain, they also are beneficial for pharma companies, which are seeing utilization of their therapies.

Although the FDA has approved five biosimilars of Regeneron Pharmaceuticals, Inc.’s best-selling Eylea (aflibercept), patent infringement lawsuits by the drugmaker have kept those competitors off the U.S. market — until now. Following a successful defense of its Pavblu (aflibercept-ayyh), Amgen Inc. recently launched the drug at risk. The agent is entering an increasingly crowded therapeutic class, but it’s one that’s also costly for payers, which may be seeking some savings, say industry experts. But is its price good enough to pull market share?

Pavblu has approval for all of Eylea’s indications — neovascular (wet) age-related macular degeneration (AMD), macular edema following retinal vein occlusion (RVO), diabetic macular edema (DME) and diabetic retinopathy (DR) — except for retinopathy of prematurity. Among the vascular endothelial growth factor (VEGF) inhibitors approved for ocular use, Eylea is the only one with that indication on its label.

A recently approved treatment for psoriatic arthritis (PsA) brings a new mechanism of action to the therapeutic class. And while payers and rheumatologists varied in their expectations of what the drug’s impact on coverage of and prescribing for the class will be, almost half said they expect it to have at least some effect, according to a survey by Zitter Insights.

On Sept. 20, the FDA approved three more indications for UCB, Inc.’s Bimzelx (bimekizumab-bkzx) for the treatment of adults with active PsA, adults with active non-radiographic axial spondyloarthritis with objective signs of inflammation and adults with active ankylosing spondylitis. Two months later, on Nov. 20, the FDA gave the drug another approval, for the treatment of moderate-to severe hidradenitis suppurativa.

A Medicare Part D policy forbidding plan sponsors’ formularies from excluding nearly all drugs in six “protected” classes hinders payers’ ability to negotiate prices and may lead to significantly higher costs to the health care system, according to a recent analysis published in Health Affairs. The study’s lead author notes this is the first peer-reviewed trial suggesting Part D plans “can’t negotiate as high of rebates as they might otherwise be able to” for drugs in the protected classes.

Last month’s FDA approval of a drug to treat atopic dermatitis brings a new agent to a growing class of medications that payers say they consider a high priority to manage.

On Sept. 13, the FDA approved Lilly’s Ebglyss (lebrikizumab-lbkz) for the treatment of people at least 12 years old who weigh at least 40 kg with moderate-to-severe atopic dermatitis that is not well controlled despite treatment with topical prescription medications or when those medications are not advisable. Dosing for the interleukin-13 (IL-13) antagonist is 500 mg via two 250 mg subcutaneous injections at weeks zero and two and then 250 mg every two weeks until week 16 or later, when an adequate clinical response is achieved. At that point, maintenance dosing is 250 mg every four weeks. The drug’s WAC is $3,500 for a 250 mg/2 mL single-dose prefilled pen or prefilled syringe.

In the seven years since the approval of the first chimeric antigen receptor T-cell (CAR-T) therapy, the agents have proved to be effective in the treatment of non-Hodgkin lymphoma. But the one-time-use agents come with risks, including the potentially fatal cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS), so the FDA requires patients remain near their treatment center for four weeks after administration, which can be onerous for patients. But a recent study finds that side effects were rare after the first two weeks post-infusion, perhaps helping lead to less of a burden for patients.

An article in Blood Advances, a journal of the American Society of Hematology, revealed the findings of a retrospective study of 475 patients who received three CD19-directed CAR-Ts at nine different centers: Yescarta (axicabtagene ciloleucel) from Gilead Sciences Inc. subsidiary Kite Pharma, Inc., Novartis Pharmaceuticals Corp.’s Kymriah (tisagenlecleucel) and Breyanzi (lisocabtagene maraleucel) from Juno Therapeutics, Inc., a Bristol Myers Squibb company.

When the FDA approved the first injectable treatment for HIV pre-exposure prophylaxis (PrEP), the agency hailed the therapy as “an important tool in the effort to end the HIV epidemic” due to its every-two-months regimen, lessening the burden of oral treatments that were taken every day. But a new injectable agent may soon be approved that reduces that treatment burden to twice a year.

As President Joe Biden’s administration nears its end, two promised rules on copayment accumulators and maximizers have yet to be released. They stand to have a huge impact on whether pharma manufacturer-provided patient assistance — much of which is provided for specialty drugs — must be counted toward patients’ out-of-pocket responsibility.

The first concerns a lawsuit over the 2021 Notice of Benefit and Payment Parameters (NBPP) and its stance toward copay accumulators.

Aug. 7: The FDA approved Citius Pharmaceuticals, Inc.’s Lymphir (denileukin diftitox-cxdl) for the treatment of relapsed or refractory cutaneous T-cell lymphoma (CTCL) after at least one systemic therapy. The immunotherapy is the only CTCL treatment that targets the interleukin-2 (IL-2) receptor found in T-cell lymphomas and regulatory T cells (Tregs). The rare disease is a chronic non-Hodkin lymphoma that primarily affects the skin. The drug is a reformulation of Ontak (denileukin diftitox), which was on the U.S. market from 2008 to 2014, when it was voluntarily withdrawn “to enable manufacturing improvements.” The FDA gave the therapy orphan drug designation. The recommended dose is 9 mcg/kg per day based on actual body weight via a 60-minute intravenous infusion on days one through five of a 21-day cycle. The company says it expects to launch the agent within the next five months.

Ahead of the much-anticipated launch of the next wave of biosimilars for a high-cost specialty drug, The Cigna Group’s Evernorth Health Services has said that it will roll out a program to manage the drugs that is similar to one it first began offering this summer. But several aspects of the upcoming launch remain unclear, including whether the discount provided for the new drug will be available to all payers.

Evernorth revealed on Sept. 5 that it will offer an interchangeable biosimilar of Stelara (ustekinumab) from Johnson & Johnson Innovative Medicine for $0 for “eligible patients” of Accredo starting “early next year.” The agent will be produced by Quallent Pharmaceuticals, Cigna’s private-label subsidiary.

The new agent’s price will be more than 80% less than Stelara’s list price. “For many employers, unions, municipalities and other health plan sponsors that choose to work with Accredo as part of their specialty pharmacy network offering, this represents an opportunity for significant savings,” said Evernorth in a press release.