Drug Approvals

A congressional panel found that major PBMs and health insurers are charging members cost sharing and imposing other coverage requirements on birth control products in violation of the Affordable Care Act. The “investigation identified more than 30 birth control products for which most health insurers and PBMs reviewed impose cost-sharing requirements or coverage exclusions,” according to a press release. Among the companies examined were CVS Health Corp., UnitedHealth Group, Elevance Health Inc., Cigna Corp., Humana Inc. and Prime Therapeutics, a PBM owned by a consortium of Blues affiliates. “From 2015 to 2021, companies denied exceptions requests on average four or more times out of ten,” the press release added.

In its latest report highlighting notable drugs nearing FDA approval, UnitedHealth Group’s Optum Rx PBM singles out two therapies designated as orphan drugs and one that has been granted priority review. Experts tell AIS Health that payers deciding how to cover these drugs will undoubtedly have to weigh factors such as a high unmet need for treatment, the durability of patients’ response to the therapy, and potentially high price tags.

For example, regarding the only non-orphan drug included in the report, Apellis Pharmaceuticals, Inc.’s pegcetacoplan, there are currently no other approved treatments for the condition it’s targeting, “so that will have an impact on how payers approach coverage,” says Bill Dreitlein, senior director of Pipeline & Drug Surveillance at Optum Rx.

The FDA recently approved the first novel long-acting granulocyte colony-stimulating factor (G-CSF) in more than 20 years. Payers say they are likely to manage the new agent similar to existing ones, but oncologists have indicated that they are willing to prescribe it in place of other neutropenia agents, according to Zitter Insights.

On Sept. 9, the FDA approved Spectrum Pharmaceuticals, Inc.’s Rolvedon (eflapegrastim-xnst) to decrease the incidence of infection, as manifested by febrile neutropenia, in adults with nonmyeloid malignancies receiving myelosuppressive anti-cancer drugs associated with clinically significant incidence of febrile neutropenia. The company developed the drug with South Korea’s Hanmi Pharmaceutical Co. The recommended dose is 13.2 mg administered subcutaneously once per chemotherapy cycle. The company says it expects the product to be available in fourth-quarter 2022. It has not revealed the agent’s price yet.

A new formulation of a narcolepsy drug may get uptake among providers, according to Zitter Insights research. But payers indicate that they are likely to require patients to fail at least one generic drug before getting access to the new agent and other branded medications. Still, the agent’s less onerous dosing may allow it to pull market share from similar medications within the class.

On July 19, Avadel Pharmaceuticals plc said that the FDA had granted tentative approval to its Lumryz (sodium oxybate) for the treatment of excessive daytime sleepiness or cataplexy — an abrupt loss of muscle tone that can be triggered by strong emotion — in adults with narcolepsy. The drug — which is also known as FT218 — is a once-at-bedtime extended-release version of Jazz Pharmaceuticals plc’s Xyrem, a drug that requires one dose at bedtime and then another dose between two-and-a-half to four hours later.

For decades, a successful vaccine for respiratory syncytial virus (RSV) — which sends thousands of infants and older adults to the hospital each year — has remained elusive. Yet several heavyweight pharmaceutical companies are now poised to fill the void, as they’re nearing the finish line in the development of RSV vaccines that could ultimately comprise a multibillion-dollar market. Coverage of the new vaccines, according to an industry expert, will hinge on the all-important Advisory Committee on Immunization Practices (ACIP), which requires payers to fully reimburse any inoculation that it recommends.

Sept. 9: The FDA approved Bristol Myers Squibb’s Sotyktu (deucravacitinib) for the treatment of adults with moderate-to-severe plaque psoriasis who are candidates for systemic therapy or phototherapy. The agent is a first-in-class, oral, selective, allosteric tyrosine kinase 2 (TYK2) inhibitor. The recommended dosage of the tablet is 6 mg once daily. The list price for a 30-day supply is $6,164.

Sept. 9: The FDA approved Spectrum Pharmaceuticals, Inc.’s Rolvedon (eflapegrastim-xnst) to decrease the incidence of infection, as manifested by febrile neutropenia, in adults with nonmyeloid malignancies receiving myelosuppressive anti-cancer drugs associated with clinically significant incidence of febrile neutropenia. The manufacturer says it is the first novel long-acting granulocyte colony-stimulating factor (G-CSF) approved in more than 20 years. The recommended dose is 13.2 mg administered subcutaneously once per chemotherapy cycle. The company says it expects the product to be available in fourth-quarter 2022.

The FDA on Sept. 29 approved Relyvrio (sodium phenylbutyrate/taurusodiol), making it only the third medication to treat patients with amyotrophic lateral sclerosis (ALS). While ALS advocates and some clinicians applauded the agency’s decision, other providers have questioned the drug’s effectiveness, and some experts believe the drug is not cost-effective with its approximately $158,000 annual wholesale acquisition cost (WAC).

For instance, the nonprofit Institute for Clinical and Economic Review (ICER) found that Relyvrio, which was formerly known as AMX0035, would be cost-effective if it was priced at between $9,100 and $30,600 per year. And Bruce Booth, a former scientist who’s now a partner at the Atlas Venture biotechnology venture capital firm, wrote on Twitter that the $158,000 price for Relyvrio was “even more egregious than I thought!”

The FDA’s controversial accelerated approval of Biogen Inc.’s Aduhelm (aducanumab) has yielded new fallout, as members of the U.S. House of Representatives have introduced a bill seeking to unwind CMS’s national coverage determination that limited access to the Alzheimer’s treatment — and prevent CMS from issuing restrictive NCD decisions on future drugs.

D.C. insiders tell AIS Health, a division of MMIT, that the bipartisan bill’s fate will likely depend on the outcome of the upcoming midterm elections. They add that the NCD bill represents a renewed effort by the pharmaceutical industry to flex its lobbying muscle after it faced an unprecedented defeat with the passage of Medicare drug price negotiation as part of the Inflation Reduction Act.

The manufacturers of many drugs granted accelerated approval by the FDA do not complete timely confirmatory trials of the drugs’ efficacy, according to a recent study published in the journal Health Affairs — meaning that the pricing for many accelerated approval drugs has nothing to do with their clinical efficacy. The study’s author tells AIS Health, a division of MMIT, that “the market doesn’t work very well” for drugs that have received accelerated approval, and “what it leads us to is overpaying at the beginning and underpaying, potentially, later.”

After a drug is granted accelerated approval, the FDA mandates that the drug be evaluated using confirmatory clinical trials. The accelerated approval designation is given to new, unproven drugs that could potentially meet a dire need for a new or more effective therapy to treat a terminal disease. The intention behind the confirmatory trial system is to make sure that the drug actually does what its developer says it will.

Walgreens Boots Alliance, Inc. (WBA) will assume full control of specialty pharmacy Shields Health Solutions for approximately $1.37 billion cash, the firm said Tuesday. Walgreens already controlled the pharmacy, holding 71% of the company’s stock thanks to a $970 million deal that closed last year. WBA CEO Roz Brewer said in a press release that the deal will allow “further progress on our strategy through Shields’ integrated model, increasing our value to health systems, expanding access to payor partners and supporting improved outcomes and lower costs.”

Centene Corp. agreed to pay $165.6 million to the state of Texas over the state's contention that the insurer overcharged the state's Medicaid program for PBM services, according to legal documents obtained by Kaiser Health News (KHN). The settlement is the largest of a series of settlements from at least a dozen states over similar charges. The Texas settlement is the largest such arrangement, with Centene paying out at least $475 million so far to the states of Arkansas, Illinois, Kansas, Mississippi, New Hampshire, New Mexico, Ohio, Texas, and Washington; three other states have not released the value of their settlements with the Medicaid-focused insurer, per KHN. Centene has divested most of its PBM assets over the past year, and told investors in 2021 that it set aside $1.25 billion to wind down its liability to states in this area.