Radar on Specialty Pharmacy

July 14: The FDA expanded the patient population of Biocodex, Inc.’s Diacomit (stiripentol) for the treatment of seizures associated with Dravet syndrome in people between the ages of 6 months to 2 years and weighing at least 7 kg who are taking clobazam. The agency first approved the treatment on Aug. 20, 2018. The drug is available as a capsule and an oral suspension. Dosing is 50 mg/kg/day for both routes of administration. Drugs.com lists the price of 60 250 mg capsules and 60 250 mg powder for reconstitution as more than $1,589.

July 14: The FDA expanded the label of Pfizer Inc.’s Xalkori (crizotinib) to include the treatment of people at least 1 year old with unresectable, recurrent or refractory inflammatory anaplastic lymphoma kinase (ALK)-positive myofibroblastic tumors. The agency initially approved the kinase inhibitor on Aug. 26, 2011. The FDA gave the agent orphan drug designation and granted the application priority review; that review used the Assessment Aid, a voluntary submission from the applicant to assist the FDA in its analysis. Dosing for the newest indication of the capsule in adults is 250 mg twice daily. The recommended pediatric dosage is 280 mg.m² twice daily based on body surface area. Drugs.com lists the price of 60 250 mg capsules as more than $20,657.

As scrutiny over what Americans pay for prescription drugs continues, the Federal Trade Commission (FTC) is joining the action. In early June, the agency voted unanimously to launch an investigation of the business practices of the six largest PBMs. Then a little over a week later, it issued a statement on its enforcement policy on certain rebates and fees that manufacturers give PBMs that result in the exclusion of lower-cost drugs on their formularies. Industry experts say that PBMs should heed this “aggressive approach,” which appears to be challenging them to show that these rebates are justifiable and being passed on to patients and payers.

The focus comes after President Biden’s July 9, 2021, Executive Order on Promoting Competition in the American Economy in which he called for “a fair, open, and competitive marketplace” across numerous industries. “Americans are paying too much for prescription drugs and healthcare services — far more than the prices paid in other countries. Hospital consolidation has left many areas, particularly rural communities, with inadequate or more expensive healthcare options. And too often, patent and other laws have been misused to inhibit or delay — for years and even decades — competition from generic drugs and biosimilars, denying Americans access to lower-cost drugs.”

Only days before the end of CMS’s Oncology Care Model (OCM), the agency unveiled a successor that will start next year. While oncologists have been overall positive about the new program, they still have had some complaints.

Offered through the Center for Medicare and Medicaid Innovation (CMMI), the Enhancing Oncology Model (EOM) is a five-year, value-based, patient-centered care model that will start on July 1, 2023. Participants may include oncology physician group practices, private payers, Medicare Advantage plans and state Medicaid agencies. The application submission period started when the voluntary model was introduced on June 27 and will close Sept. 30.

As Congress again proposes drug pricing efforts, many of its members, as well as a couple of government agencies, have pharma manufacturers in their crosshairs for a somewhat related reason: their patent processes. The FDA and the U.S. Patent and Trademark Office (PTO) recently said they would be working together to scrutinize certain practices that could potentially lead to delays in competition from biosimilars and generics.

The move follows President Biden’s July 9, 2021, Executive Order on Promoting Competition in the American Economy in which he called for “a fair, open, and competitive marketplace” across numerous industries. “Too often, patent and other laws have been misused to inhibit or delay — for years and even decades — competition from generic drugs and biosimilars, denying Americans access to lower-cost drugs.”