Specialty Drugs

The FDA recently gave an additional approval to BeiGene, Ltd.’s Brukinsa (zanubrutinib), which is already approved for three other rare types of non-Hodgkin lymphoma. Respondents to a Zitter Insights survey said that while its availability will result in a lower level of unmet need in the treatment of chronic lymphocytic leukemia (CLL), there is still moderate or high unmet need for the condition.

On Jan. 19, the FDA expanded the label of Brukinsa to include the treatment of adults with CLL or small lymphocytic lymphoma (SLL). CLL and SLL are the same disease, a type of non-Hodgkin lymphoma, except CLL cancer cells are mostly in the blood and bone marrow, while in SLL, the cells are mainly in the lymph nodes.

A new study published in the Journal of Clinical Oncology found that cancer afflicting adolescents and young adults cost the health care system $23.5 billion in 2021, and $259,324 for the average patient over a lifetime. The findings come as the American Cancer Society reported that incidence of colorectal cancer is becoming more common among that cohort: 20% of new colorectal cancer diagnoses in 2019 were for patients younger than 55, compared with 11% in 1995, per the Wall Street Journal. Experts say that to help, insurers can educate patients and improve access to screenings and care coordination.

Fortunately, the overwhelming majority of adolescents and young adults who are diagnosed with cancer have a positive prognosis: in 2019, the 5-year survival rate for people in that cohort was 85% “with prompt diagnosis and timely delivery of appropriate therapy,” per the Journal of Clinical Oncology study. Approximately 90,000 people ages 15-39 in the U.S. are diagnosed with cancer annually — that’s about 5% of all new cancer cases. The increasing incidence of colorectal cancers contrasts with falling adolescent and young adult death rates “each year between 2010 and 2019,” the study said. Experts say systemic issues make detecting and treating cancers in the adolescent and young adult cohort difficult.

Breast cancer is the most common cancer diagnosed in American women and the most common cancer globally. Estimates show that more than 300,000 people in the U.S. are expected to be diagnosed this year alone, almost 3,000 of them men.

When the FDA first approved Genentech, Inc.’s Herceptin (trastuzumab) on Sept. 25, 1998, for the treatment of people with breast cancer whose tumors overexpress the human epidermal growth factor receptor-2 (HER2) protein, the drug offered a new targeted approach to treating the disease. In the years since, researchers have identified other subtypes of the cancer and developed newer agents indicated for specific types and stages of the disease.

The FDA accepted its first application for a biosimilar denosumab, Sandoz disclosed on Feb. 6. The Amgen Inc. reference drug is available as both Prolia and Xgeva. According to AmerisourceBergen’s Feb. 6 U.S. Biosimilar Report, nine companies have denosumab biosimilars in clinicals trials, with five of those in Phase III trials. Prolia and Xgeva are approved for multiple conditions, including for the treatment of postmenopausal women with osteoporosis at high risk for fracture and for the prevention of skeletal-related events in people with multiple myeloma and in people with bone metastases from solid tumors. Sandoz’s application is for all of the drugs’ indications. Prolia’s list price is $1,564.31 per injection every six months, and the per-dose price for Xgeva — which is dosed every four weeks — is $2,877.36.

Elevance Health said on Feb. 15 that it had closed its acquisition of BioPlus, a specialty pharmacy subsidiary of CarepathRx, a portfolio company of Nautic Partners. BioPlus will operate as part of CarelonRx, Elevance’s PBM within Carelon, its health care services brand. Elevance said Nov. 9 that it had entered into an agreement to purchase the company. It did not disclose financial terms of the deal. The firm also said that it “plans to expand BioPlus’ service models across more complex disease treatment areas so the combined company can continue to provide timely access to medications, deliver leading support services for both providers and patients, and ensure individuals receive distinctive clinical expertise and service.

The U.S. biosimilars market is a dynamic and ever-evolving space, and a recent report shows growing acceptance of the medications among providers. Last year saw a handful of milestones related to these agents, and this year, particularly with the launches of biosimilars of AbbVie Inc.’s Humira (adalimumab), is likely to continue the trend, according to a recent report from Cardinal Health.

Titled 2023 Biosimilars Report: tracking market expansion and sustainability amidst a shifting industry, the report is based on responses to web-based surveys conducted in 2022 from both community- and hospital-based practices. Providers were from “therapeutic areas with the most potential for disruption in the year ahead.” Cardinal Health polled more than 100 rheumatologists in July and August, 125 dermatologists in September, 70 gastroenterologists in September and October, and 60 retina specialists in September and October.

Feb. 3: The FDA expanded the patient population for Takeda’s Takhzyro (lanadelumab-flyo) to include the prevention of hereditary angioedema attacks in people at least 2 years old. The agency initially approved the plasma kallikrein inhibitor on Aug. 3, 2018. Dosing of the subcutaneous injectable in people at least 12 years old is 300 mg every two weeks. In people at least 6 and less than 12, dosing is 150 mg every two weeks, and for people 2 years old and younger than 6, dosing is 150 mg every four weeks. Drugs.com lists the price of one single-dose 300 mg/2 mL subcutaneous solution as more than $26,165.

Feb. 8: The FDA granted another indication to Regeneron Pharmaceuticals, Inc.’s Eylea (aflibercept) for the treatment of retinopathy of prematurity in preterm infants. The agency first approved the drug on Nov. 18, 2011. Dosing of the vascular endothelial growth factor (VEGF) inhibitor for the newest indication is 0.4 mg via intravitreal injection; treatment may be given bilaterally on the same day. Injections may be repeated with an interval of at least 10 days. Drugs.com lists the price of a 40 mg/ml intravitreal solution as more than $1,957.

The FDA recently approved a new HIV drug for a small patient population desperately in need of treatments. And the twice-yearly medication’s annual price came below the level that respondents to a Zitter Insights poll said they would consider a good value. The medication comes with both potential advantages and disadvantages for patients, providers and payers, say industry experts.

On Dec. 22, the FDA approved Gilead Sciences, Inc.’s Sunlenca (lenacapavir) for the treatment, in combination with other antiretroviral(s) (ARVs), of HIV-1 infection in heavily treatment-experienced adults with multidrug resistant HIV-1 infection who are failing their current antiretroviral regimen due to resistance, intolerance or safety considerations. The agency gave the first-in-class capsid inhibitor priority review, fast track and breakthrough therapy designations.

Following a severe season of respiratory syncytial virus (RSV) in the U.S., several companies have revealed that they have promising vaccine candidates for various patient populations in the late-stage clinical trial pipeline. The FDA could approve some of them as early as this year, for what is estimated to be a market worth more than $10 billion. Currently, only one agent has FDA approval for RSV prevention, and one specialty pharmacy recently shared with AIS Health, a division of MMIT, how it manages that treatment and members taking it.

The CDC estimates that children younger than 5 years old account for more than 57,000 hospitalizations and about 100 to 500 deaths per year. Older adults experience approximately 177,000 hospitalizations and 14,000 deaths annually, but no FDA-approved treatments indicated for this population are available.

Legislative leaders in North Carolina have struck a deal to expand Medicaid in the state, although the measure won’t be voted on until later this month at the earliest. During a news conference on March 2, state House Speaker Tim Moore and Senate leader Phil Berger, both Republicans, touted the agreement as a major accomplishment for North Carolina, which is one of 11 states that has not yet expanded Medicaid eligibility under the Affordable Care Act. “What a huge policy direction this is that will provide help for so many in this state, but it’s going to do it in a way that’s fiscally responsible,” Moore said, according to the Associated Press. Under the agreement, which was still being drawn up at press time, the state’s 10% share of covering the Medicaid expansion population would be paid through assessments on hospitals. Previously, the Urban Institute estimated that expanding Medicaid could reduce the uninsured population by 30%, or 346,000 people, in North Carolina.

As the world enters the fourth year of the COVID-19 pandemic, a recent report from the IQVIA Institute for Human Data Science found — not surprisingly — that a significant shift has occurred in the outlook for global medicine spending. The report, titled The Global Use of Medicines 2023: Outlook to 2027, revealed that global spending on pharmaceuticals, including COVID vaccines and therapeutics, from 2020 to 2027 is anticipated to surpass the Institute’s pre-pandemic estimations by $497 billion.

“Here we are at the beginning of 2023, with perhaps more uncertainties still ahead of us than we might have hoped for a year ago,” observed Murray Aitken, executive director of the IQVIA Institute for Human Data Science, during a recent webinar presented by his company. “Despite the progress on the pandemic front, it’s still out there; it’s still shaping our lives to a greater or lesser extent, depending on where in the world we are. It’s also still shaping our health care systems.