Radar on Specialty Pharmacy

✦ Nov. 9: The FDA expanded the label of Foundation Medicine, Inc.’s FoundationOne Liquid CDx to identify people with BRCA1, BRCA2 and/or ATM alterations in metastatic castration-resistant prostate cancer who may be appropriate for treatment with AstraZeneca and Merck & Co., Inc.’s Lynparza (olaparib). The agency initially approved the liquid biopsy test in August (RSP 9/20, p. 8); it’s now approved as a companion diagnostic for seven targeted therapies across four tumor types. The manufacturer says it is the only FDA-approved blood-based test that can analyze more than 300 genes. Visit https://bit.ly/3gINu3h.

✦ Nov. 13: The FDA gave accelerated approval to Merck’s Keytruda (pembrolizumab) in combination with chemotherapy for the treatment of people with locally recurrent unresectable or metastatic triple-negative breast cancer whose tumors express programmed death-ligand 1 (PD-L1) as determined by an FDA-approved test (see brief below). The agency initially approved the programmed death receptor-1 (PD-1) inhibitor in 2014 (RSP 9/14, p. 4); it now has almost 30 approvals across almost 20 types of cancer. This newest approval is its first in breast cancer. Dosing for this indication is 200 mg every three weeks or 400 mg every six weeks via a 30-minute intravenous infusion. The list price for dosing every three weeks is $9,869.94; for every six weeks, it’s $19,739.88, according to the Keytruda website. Visit www.keytruda.com.

The most-favored-nation (MFN) interim final rule issued in late November is not the first time the current administration has attempted to implement some kind of drug pricing model based on prices in other countries.

The first effort, the International Pricing Index (IPI) model (83 Fed. Reg. 54546, Oct. 30, 2018), was unveiled on Oct. 25, 2018, in an Advanced Notice of Proposed Rulemaking (ANPRM). It proposed that instead of CMS reimbursing Medicare Part B drugs at their average sales price (ASP) plus 6%, it would reimburse these medications per an IPI based on drug pricing data from not only the U.S. but also 16 other developed countries (RSP 11/18, p. 1).

With its third FDA approval in less than two-and-a-half years, Alnylam Pharmaceuticals, Inc. is bringing another orphan drug for a rare condition to the U.S. market. And similar to the first two, Oxlumo (lumasiran) comes with a costly price tag. To help provide payers with some predictability around costs, the company is extending a new value-based agreement, as well as offering another VBA that the company introduced with its previous approval.

On Nov. 23, the FDA approved Alnylam’s Oxlumo for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients. The agency says it is the first treatment approved for the rare genetic disorder, in which the body overproduces oxalate. This, in turn, results in the deposition of calcium oxalate in the kidneys and urinary tract, which can lead to kidney stones, nephrocalcinosis, kidney failure and organ damage throughout the body.

The FDA has approved only a few drugs to treat triple-negative breast cancer (TNBC), and last month saw Merck & Co., Inc’s Keytruda (pembrolizumab) gain accelerated approval for the condition. Commercial payers have indicated a willingness to cover it at parity with Roche Group member Genentech USA, Inc.’s Tecentriq (atezolizumab), as well as preferring Keytruda over Tecentriq, but providers are a bit more uncertain about how they will prescribe the newly approved therapy. The condition, though, remains largely unmanaged by payers, a source tells AIS Health.

Some breast cancers test positive for estrogen receptors, progesterone receptors or overexpression of human epidermal growth factor receptor 2 (HER2), but people with TNBC do not have these proteins, which limits their treatment options, according to the American Cancer Society. It also is a fairly aggressive cancer and recurs more often than other breast cancers.

As the Supreme Court decides on the fate of the Affordable Care Act (ACA), much of the focus understandably has been on the millions of people who would lose health insurance coverage and protections for pre-existing conditions if the law is overturned. Another not-so-often-discussed ramification of such a ruling is that the biosimilars market could be completely upended. While industry experts tell AIS Health that such an outcome probably will not occur, they still recommend that stakeholders prepare for this possibility.

The Biologics Price Competition and Innovation Act of 2009 (BPCIA) created the 351(k) biosimilar pathway. After more than one attempt to get a stand-alone bill to pass, lawmakers made it part of the ACA, and it became law on March 23, 2010 (RSP 4/10, p. 1).