Acute Myeloid Leukemia Practice Pearls: Considerations for Formulary Decisions

Acute myeloid leukemia (AML) is the most frequently occurring cause of acute leukemia in adults. This aggressive form of cancer is diagnosed in approximately 21,000 individuals annually in the United States and slightly more than half will die each year. In a Pharmacy Times® Practice Pearls video series, experts discussed best practices in the management of AML, including patient identification, formulary decisions, and providing therapy from an outpatient setting for optimal patient care.

The panelists—Katie Culos, PharmD, BCOP, a hematology/oncology clinical pharmacist at Vanderbilt University Medical Center in Nashville, Tennessee; Amanda Brahim, PharmD, BCOP, BCPS, BCACP, a hematology/oncology clinical pharmacist at Memorial Cancer Institute in Pembroke Pines, Florida; and Yehuda Deutsch, MD, a hematologist/oncologist in the Department of Malignant Hematology and Cellular Therapy at Memorial Cancer Institute—noted that myriad factors are involved in determining the appropriate care pathway for patients with AML.

Practice Pearl 2: Formulary

Culos noted a boom over the past 3 years with a much stronger amount of treatment options for AML. Brahim said her institution’s inpatient and outpatient formularies are similar, but have a few differences.

“On the outpatient side, we do have all of the oral targeted agents, as well. On the inpatient side, in terms of new medications for IVs [intravenous drugs], I believe we have everything,” she said. “We have added gemtuzumab ozogamicin when it came back to market. We did add CPX-351 when it came to market. We also have midostaurin, which we are starting on the inpatient side, for those patients that are getting the traditional 7+3 regimen but have a FLT3 mutation. We’ll start that on day 8, where the patient is still on the inpatient side, and they’re unable to obtain it and bring it into the hospital. So, we have added, from an inpatient perspective, many if not all of the newer agents to the formulary.”

She added her institution stocks venetoclax as well because some patients have an urgent need to start this medication.

“Through a specialty pharmacy, this may take a week, sometimes 2 weeks, to get to the patient’s house. We have recognized that, and we do stock it, although it’s not officially on our inpatient formulary,” she said.

In terms of P&T committee decisions, Brahim said a subcommittee exists specifically for hematology-oncology.

“Generally, when we go through the formulary process, a request is made, and a physician and pharmacist dyad…review the literature regarding the agent that’s proposed to be added to formulary. We then review it at the system-wide hematology-oncology P&T committee, and from there it moves on to the system overall formulary committee for final approval,” she said.

The panel also noted that important outcomes and efficacy endpoints that are critical to help support decision making.

“We always like to see a randomized, controlled clinical trial that has 100s, if not 1000s, of patients. Unfortunately, in AML, that’s not always feasible,” Brahim said. “So, we have approved agents based on phase 2 trials, based on open-label studies, based on smaller trials, where we did feel like there was enough evidence to say that the benefit outweighed the risk. I think more than anything, just because the need is so great in AML, some of these agents have been able to come to market and have been added to formulary.”

Deutsch added that the FDA has taken these factors into account as well.

“They have approved medications for AML, even before there is necessarily overall survival data or phase 3 data,” he said. “Some of the newer medications that have been approved recently were approved based on phase 2 data by the FDA, including venetoclax combinations.”

Click here for the full Pharmacy Times® Practice Pearls video series on AML.