RADAR on Specialty Pharmacy

Judge Strikes Down CMS’s So-Called ‘Accumulator Rule’

A U.S. district court judge has struck down a CMS rule that would have narrowed the exclusions from Medicaid best price for manufacturer-provided patient-assistance programs. The rule, which was set to go into effect on Jan. 1, would have required drugmakers to determine exactly where their patient assistance is going. If 100% of it was not reaching the patient — particularly via copayment accumulators and maximizers when payers are taking this assistance rather than allowing it to count toward patients’ deductibles and out-of-pocket maximums — that assistance would need to have been included in Medicaid best price and average manufacturer price (AMP) calculations for prescription drugs. This decision, as well as a recent pharma lawsuit against a maximizer company, may spur more pushback against these copay programs, one industry expert tells AIS Health, a division of MMIT.

FDA’s Rinvoq Approval Brings New Ulcerative Colitis Option

The FDA recently gave an additional indication to AbbVie Inc.’s Rinvoq (upadacitinib) in ulcerative colitis, broadening that therapeutic class even more. And while a study revealed some concerns around another agent with a similar mechanism of action, payers and gastroenterologists last year expressed interest in Rinvoq over other late-stage pipeline agents.

On March 16, the FDA expanded the label of Rinvoq to include the treatment of adults with moderately to severely active ulcerative colitis who have had an inadequate response or intolerance to at least one tumor necrosis factor (TNF) blocker. The agency initially approved the Janus kinase (JAK) inhibitor on Aug. 16, 2019. The recommended starting dose for the tablet is 45 mg once daily for eight weeks, followed by a maintenance dose of 15 mg once daily. The wholesale acquisition cost for a 30-day supply is $5,671.26.

People With Rare Diseases Face Challenges, Require Support

In the U.S., orphan diseases are conditions impacting fewer than 200,000 people. There are more than 7,000 of these rare conditions affecting an estimated 30 million Americans — and more than 300 million people globally — and new diseases continue to be discovered. Most of them are inherited conditions caused by gene mutations, but some can be caused by environmental factors. These diseases may be serious and even life-threatening, and about half of them affect children.

Before the Orphan Drug Act was passed in 1983, not much research was done into treatments for rare diseases. But that law created financial incentives for pharmaceutical manufacturers, and since then, hundreds of orphan drugs have been developed. As of early 2020, the FDA had approved therapies for more than 800 rare diseases.

New FDA Approvals: FDA Grants Additional Indication to Olumiant

May 10: The FDA granted full approval to Eli Lilly and Co.’s Olumiant (baricitinib) for the treatment of COVID-19 in hospitalized adults requiring supplemental oxygen, noninvasive or invasive mechanical ventilation, or extracorporeal membrane oxygenation. The agency first approved the Janus kinase (JAK) inhibitor on May 31, 2018. The drug has been available for COVID treatment since Nov. 19, 2020, under Emergency Use Authorization (EUA). The EUA remains in place for hospitalized people between the ages of 2 and 18 years old who require various degrees of oxygen support. The recommended dose of the tablet for the newest use is 4 mg once daily for 14 days or until hospital discharge, whichever occurs first. Alternative modes of administration via oral dispersion, gastrostomy tube, nasogastric tube or orogastric tube are available. The drug’s list price for a 30-day supply of 2 mg tablets is $2,497.20.

News Briefs: Biogen and Samsung Bioepis Launch Byooviz

Biogen Inc. and Samsung Bioepis Co., Ltd. said on June 2 that they had launched Byooviz (ranibizumab-nuna), and the medication will be commercially available “through major distributors across the U.S.” on July 1. The drug is the first FDA-approved ophthalmology biosimilar and references Roche Group unit Genentech USA, Inc.’s Lucentis (ranibizumab). On Sept. 20, 2021, the FDA approved Byooviz for the treatment of neovascular (wet) age-related macular degeneration, macular edema following retinal vein occlusion and myopic choroidal neovascularization. The list price of the intravitreal injection is $1,130 per single use vial, which is 40% less than Lucentis’ list price.

Do Pharma/PBM Contracts Play Role in Drugmakers’ Revenue Leakage?

Pharma manufacturers depend on contracts with PBMs — and, increasingly, their group purchasing organizations (GPOs) — to ensure favorable formulary positioning with PBMs’ health plan and employer clients. But as those contracts have grown more complex and less transparent, drugmakers may be at risk of losing significant amounts of money, according to some industry experts.

Revenue leakage — unintended revenue loss because of process inefficiencies — can be a huge financial drain on pharma manufacturers. It also may potentially result in compliance risks with the Anti-Kickback Statute and its discount safe harbor protections, “so it always has to be clearly defined as to what the rebate or any monies between pharma and the PBM being exchanged; there has to be a reason,” explains Stephanie Seadler, vice president of Trade Relations at EmsanaRx.

Novartis’ Pluvicto Brings New Mechanism of Action to mCRPC Options

A new prostate cancer drug is sparking interest among payers and oncologists alike, according to a survey by Zitter Insights. While the product offers a new mechanism of action for the indication, the manufacturer recently halted production of the therapy temporarily in two of its three global sites “out of an abundance of caution” due to “potential quality issues” that could pose a glitch in initial uptake of the therapy.

On March 23, the FDA approved Novartis Pharmaceuticals Corp.’s Pluvicto (lutetium Lu 177 vipivotide tetraxetan) (formerly referred to as 177Lu-PSMA-617) for the treatment of prostate-specific membrane antigen (PSMA)-positive metastatic castration-resistant prostate cancer (mCRPC) in people who have been treated with androgen receptor pathway inhibition and taxane-based chemotherapy. The product from Novartis unit Advanced Accelerator Applications USA, Inc. is the first FDA-approved targeted radioligand therapy for eligible people with mCRPC that combines a targeting compound with a therapeutic radioisotope.

Study Finds PBC Drug’s Real-World, Trial Side Effects Are Similar

A recent study of a new drug to treat primary biliary cholangitis (PBC) found that common symptoms experienced by real-world patients were similar to those experienced by people in clinical trials for the agent.

PBC is a chronic disease that causes the liver’s small bile ducts to be destroyed, resulting in permanent liver damage and putting people at risk for liver failure and death. There is no cure for PBC, and the goal of treatment is to slow progression of the condition and manage its symptoms, which most commonly are itching, also known as pruritis, and fatigue.

New FDA Approvals: The FDA Approved Amneal’s Alymsys

April 13: The FDA approved Amneal Pharmaceuticals, Inc.’s Alymsys (bevacizumab-maly) for the treatment of multiple conditions: (1) first- or second-line treatment of metastatic colorectal cancer in combination with intravenous fluorouracil-based chemotherapy; (2) second-line treatment of metastatic colorectal cancer in combination with fluoropyrimidine-irinotecan or fluoropyrimidine-oxaliplatin chemotherapy in people who have progressed on a first-line bevacizumab product; (3) first-line treatment of unresectable, locally advanced, recurrent or metastatic non-squamous non-small cell lung cancer in combination with carboplatin and paclitaxel; (4) recurrent glioblastoma in adults; (5) metastatic renal cell carcinoma in combination with interferon alfa; (6) persistent, recurrent or metastatic cervical cancer in combination with paclitaxel and cisplatin or paclitaxel and topotecan; and (7) epithelial ovarian, fallopian tube or primary peritoneal cancer in combination with paclitaxel, pegylated liposomal doxorubicin or topotecan for platinum-resistant recurrent disease in people receiving no more than two prior chemotherapy regimens. The vascular endothelial growth factor inhibitor is the third biosimilar of Roche Group member Genentech USA, Inc.’s Avastin (bevacizumab) that the agency has approved. Dosing of the intravenous infusion is based on indication.

News Briefs: TG Therapeutics Withdraws Pending Applications for Ublituximab/Ukoniq Combo

TG Therapeutics, Inc. said April 15 that it voluntarily withdrew its pending Biologics License Application (BLA)/supplemental New Drug Application (sNDA) for the combination of ublituximab and Ukoniq (umbralisib) for the treatment of adults with chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma. The company said it made the decision based on updated overall survival data from the UNITY-CLL Phase III trial. The company also said that it voluntarily withdrew Ukoniq from sale for two indications: (1) for adults with marginal zone lymphoma who have received at least one anti-CD20-based regimen, and (2) for adults with follicular lymphoma who have received at least three prior systemic therapies. The FDA gave the drug accelerated approval for those indications on Feb. 5, 2021. On Feb. 3, 2022, the company disclosed that the FDA was investigating a possible increased risk of death with Ukoniq based on initial findings from the UNITY clinical trial. The FDA had scheduled an April 22 meeting to discuss the sNDA for the combination therapy, as well as Ukoniq’s accelerated approvals. Following TG Therapeutics’ withdrawal of the BLA/sNDA and Ukoniq’s existing indications, the agency cancelled the meeting. The FDA is expected to make a decision on the BLA for ublituximab in relapsing forms of multiple sclerosis by Sept. 28, 2022.