Specialty Pharmacy

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.

During their recent fourth-quarter and full-year 2022 earnings calls, the companies that own the country’s largest PBMs all said they expect that part of their business to gain momentum this year thanks to successful selling seasons in which their increasingly diversified offerings resonated with clients.

In 2022, “Optum Rx revenues grew 9%, approaching $100 billion for the year, driven by continued strong sales and the expansion of our pharmacy services businesses,” UnitedHealth Group Executive Vice President and Chief Financial Officer John Rex said during the company’s Jan. 13 conference call to discuss financial results. “Both customer retention and new customer wins were among the highest Optum Rx has ever delivered, laying a strong foundation for continued market-leading growth,” he added.

AstraZeneca will permanently withdraw Lumoxiti (moxetumomab pasudotox-tdfk) from the U.S. market, the company revealed recently. In a letter to health care providers, the company said it will direct its distributors to halt distribution of the agent in August 2023 and will request that those distributors return any Lumoxiti packs. The FDA approved the medication on Sept. 13, 2018, for the treatment of relapsed or refractory hairy cell leukemia in people who had received at least two prior systemic therapies including a purine nucleoside analog. The manufacturer said that the drug’s withdrawal is not related to its safety or efficacy and that the medicine has had “very low clinical uptake” since its approval, “due to the availability of other treatment options and possibly due to the specialized complexity of administration, toxicity prophylaxis and safety monitoring needs for patients.” The company advised providers not to start treatment of the drug. For patients already taking it, providers have “adequate time to complete six cycles of treatment.”

The FDA recently approved the fifth and sixth gene therapies, with four of those decisions coming in the second half of 2022. While payer respondents to a Zitter Insights survey have expressed interest in hemophilia B treatment Hemgenix (etranacogene dezaparvovec-drlb), its price may prove to be an obstacle to coverage.

On Nov. 22, the FDA approved uniQure N.V.’s Hemgenix for the treatment of people at least 18 years old with hemophilia B who currently use factor IX prophylaxis therapy or have current or historical life-threatening hemorrhage or have repeated serious spontaneous bleeding episodes. CSL Behring LLC, a CSL business, will market the gene therapy. The agency gave the first-in-class adeno-associated virus A5-based gene therapy — which previously was known as EtranaDez — priority review and orphan and breakthrough therapy designations.

Paying for specialty drugs continues to be a top concern for U.S. payers, and employers are utilizing various strategies to try to get a handle on their costs. Some relief should come, at least for Medicare, with the Inflation Reduction Act, which will start to have an impact this year as CMS names its top medications for negotiations on Sept. 1. AIS Health, a division of MMIT, spoke with multiple industry experts about what they’re expecting to see in 2023.

AIS Health: What are some specialty pharmacy issues to keep an eye on in 2023, and why?

Elan Rubinstein, Pharm.D., principal at EB Rubinstein Associates: Increasing acceptance of biosimilars by prescribers and patients may give payers confidence to implement coverage mandates and benefit designs that advantage their use.

The pharmaceutical industry likely will continue to battle challenges posed by a variety of factors in 2023, including global economic issues, competition and the Inflation Reduction Act (IRA). The biosimilars space in particular is expected to undergo a change as the world’s top selling drug, AbbVie Inc.’s Humira (adalimumab), finally faces competition from a number of companies. But industry experts tell AIS Health, a division of MMIT, that they expect a number of positive developments in the space in 2023.

The pharmaceutical industry and the broader health care services market currently are experiencing a series of trends that are likely to persist into 2023, said Adam J. Fein, Ph.D., CEO of Drug Channels Institute, during a Dec. 16 webinar titled Drug Channels Outlook 2023. These include pressure on insurers’ traditional coverage of generics from patient-paid prescriptions, ongoing 340B litigation and providers’ increased presence within the specialty pharmacy market. But the impact of the biggest disruption, the Inflation Reduction Act (IRA), is yet to come. In this second of a two-part series, AIS Health highlights these trends projected by the longtime industry expert.

HHS set important deadlines for Medicare drug price negotiation on Jan. 11: Biden administration officials said that the first 10 drugs selected for negotiation will be announced on Sept. 1 of this year, and the maximum fair price for those drugs will be revealed on the same day in 2024. Those prices will go into effect at the start of 2026. HHS also released more information about the process it designed to select drugs and negotiate prices, which will include public comment periods and official consultations with stakeholders including manufacturers, insurers and providers.

HHS has a “plan to voluntarily solicit public comments on key elements of the program,” said CMS Administrator Chiquita Brooks-LaSure during a press conference call. “Input from our partners is essential to effectively and expeditiously implementing the Medicare drug pricing negotiation authority. We are committed to engaging with our partners through our timeline to maximize transparency, predictability and collaboration.”

Both the specialty pharmacy market and the home infusion space continued to grow in 2022. Congress finally passed legislation that will allow Medicare to negotiate drug prices with manufacturers for the first time, and many of the impacted agents are expected to be specialty medications. As payers grappled with their drug costs, some implemented new utilization management strategies. And the COVID-19 pandemic, now approaching its fourth year, remained a disruptor in both the specialty pharmacy and home infusion markets. AIS Health, a division of MMIT, spoke with various industry experts on multiple 2022 issues impacting those industries.