Specialty Drugs

The FDA has approved multiple new agents for the treatment of acute lymphoblastic leukemia (ALL) — which is also known as acute lymphocytic leukemia — and recently converted an accelerated approval it had given one of them to full. However, even with all these options, respondents to a Zitter Insights survey said that unmet need exists in treating the disease.

On June 21, 2023, the FDA granted full approval to Amgen Inc.’s Blincyto (blinatumomab) for the treatment of adults and pediatric patients with CD19-positive B-cell precursor ALL first or second complete remission with minimal residual disease (MRD) greater than or equal to 0.1%. The agency first approved the CD19-directed CD3 T-cell engager on Dec. 3, 2014; the accelerated approval for MRD-positive B-cell ALL was granted on March 29, 2018.

Specialty drug trend continues to be in consistent double digits, with increases due to both spend and trend. That’s one of the findings of the 2023 Artemetrx Specialty Spend & Trend Report from the Pharmaceutical Strategies Group (PSG), an EPIC company. And with the specialty drug pipeline continuing to pump out more and more expensive agents, opportunities for savings exist through the use of biosimilars and generic specialty drugs, among other strategies.

Published July 25, the report is sponsored by Walmart Specialty Pharmacy and includes data from 2022. Findings are based on an analysis of 347 million medical claims and 133 million pharmacy claims from PSG’s Artemetrx book of business. Artemetrx is a proprietary SaaS platform developed by PSG. This is the seventh annual version of the report, which started under the Pharmacy Benefit Management Institute (PBMI) moniker. In April 202, MJH Life Sciences acquired PBMI’s trademarks, conference, website, education and membership assets, while the current and future research and analytics projects remained with PSG.

A new study from the Employee Benefit Research Institute highlights the high — and growing — markups that hospital outpatient departments assign to biologic drugs, while also examining the variation in how HOPDs and physician offices (POs) treat innovator biologics compared to their biosimilars.

The study analyzed medical and pharmacy claims data from Merative MarketScan Commercial Database — which covers nearly 25 million people with private health insurance — from 2013 to 2020, and it focused on seven innovator biologics and their biosimilars that had been launched as of 2020.

The large markups that hospital outpatient departments (HOPDs) place on biologic medicines are both causing plan sponsors’ costs to soar and muting the potential savings that could come from biosimilars, according to a new report from the Employee Benefit Research Institute (EBRI). One of the report’s coauthors says that health care provider consolidation is largely the culprit — and it’s an issue that plan sponsors may struggle to combat.

While physician practices also mark up the prices of provider-administered drugs, HOPDs generally raise them even more, explains Paul Fronstin, Ph.D., director of health benefits research at EBRI. “And that’s because they’ve got purchasing power, and the purchasing power is increasing as they acquire more and more physician practices,” he adds.

Over the next few years, numerous biosimilars are expected to hit the U.S. market, leading to more competition and potentially lower costs for some expensive medications. However, that will not be enough to offset the entrance of new high-cost drugs and expanded indications for specialty medicines, according to the latest Pharmaceutical Strategies Group (PSG) “Spend and Trend” report.

The analysis, published on July 25, found that the per member per year (PMPY) cost for specialty drugs was $1,169 in 2022, a 14.1% increase from the previous year. After factoring in rebates, the PMPY increased by 12.5%, from $822 in 2021 to $925 in 2022.

PSG projects that the pre-rebate and post-rebate PMPY in 2025 will be $1,712 and $1,370, representing a 46.5% and 48.5% increase from last year, respectively.

The FDA recently converted accelerated approval to full for Amgen Inc.’s Blincyto (blinatumomab) for certain patients with acute lymphoblastic leukemia (ALL). While the drug is one of many others approved for the condition, respondents to a Zitter Insights survey said there is still unmet need in treating the disease.

On June 21, 2023, the FDA granted full approval to Blincyto for the treatment of adults and pediatric patients with CD19-positive B-cell precursor ALL — which is also known as acute lymphocytic leukemia — in first or second complete remission with minimal residual disease (MRD) greater than or equal to 0.1%. The agency first approved the CD19-directed CD3 T-cell engager on Dec. 3, 2014; the accelerated approval for MRD-positive B-cell ALL was granted on March 29, 2018.

As expected, early July saw the launch of multiple biosimilars of AbbVie Inc.’s Humira (adalimumab). Most of them launched with wholesale acquisition costs (WACs) just slightly less than that of the reference drug, but a few undercut that price fairly significantly.

Of the seven agents — including a branded and an unbranded version of Hyrimoz (adalimumab-adaz) from Sandoz, a division of Novartis Pharmaceuticals Corp., that will be spun off into a new publicly traded standalone company in the second half of this year — five launched with a WAC 5% off Humira’s $6,922 price tag:

The FDA last month approved two new gene therapies for Duchenne Muscular Dystrophy (DMD) and hemophilia A, and major PBMs tell AIS Health, a division of MMIT, that they have not yet decided how to cover the new treatments. If current trends are any indication, health plans will impose strict utilization management requirements and attempt to negotiate outcomes-based reimbursement pacts with the treatments’ manufacturers.

The FDA on June 22 granted accelerated approval to Sarepta Therapeutics, Inc.’s Elevidys (delandistrogene moxeparvovec-rokl), the DMD treatment. It will have a list price of $3.2 million. BioMarin Pharmaceutical Inc.’s Roctavian (valoctocogene roxaparvovec), for hemophilia A, gained full FDA approval on June 29, and will have a list price of $2.9 million. Both therapies are administered as a one-time dose. Roctavian also typically requires an indefinite period of simultaneous treatment with corticosteroids to reduce patients’ immune-system response to the gene therapy.

Plan sponsors’ use of copayment accumulators, copay maximizers and alternative funding companies to keep spending on specialty drugs down has been a fairly recent trend but one that continues to pick up speed, noted longtime industry expert Adam J. Fein, Ph.D., CEO of Drug Channels Institute, during a recent webinar. But as that use continues to increase, multiple questions exist around the practices and what the future holds for them.

The focus of the June 23 webinar, titled PBMs and the Battle Over Patient Support Funds: Accumulators, Maximizers, and Alternative Funding, mainly affects specialty drugs, which now represent more than half of payer spending on drugs due to two main reasons, he said. The first is the specialty drug pipeline and the costly prices of those agents, while the second is the dramatic growth in generic dispensing over the past 20 years.

Eisai Co., Ltd. and its partner Biogen, Inc. crossed over the biggest barrier in their quest to commercialize Leqembi (lecanemab) now that Medicare will cover the cost of the Alzheimer’s disease drug. The next hurdles for doctors and patients will be obtaining blood tests and PET scans for confirming amyloid pathology in the brain, genetic testing to assess APOE4 status, MRIs to monitor for amyloid-related imaging abnormalities (ARIA) and infusion centers to administer the medicine — facilities that exist, but not in the numbers and locations needed to serve all of the US patients eligible for treatment.

CMS said that it will cover the cost of Leqembi on July 6, the day that the FDA converted the amyloid protofibril-targeting antibody’s accelerated approval for the treatment of mild cognitive impairment or mild dementia associated with Alzheimer’s disease (AD) into full approval. Eisai and others believe that while uptake of Leqembi will be slow as the health care system catches up with the diagnostic, monitoring and infusion requirements associated with the therapy, Medicare coverage gives providers the confidence they need to offer those services.