FDA

For health plans, managing prescription drug coverage has become a highly complex task for myriad reasons, including never-ending regulatory changes and ever-rising prices. And that task is made even more difficult by the fact that an increasing number of drugs are coming to market with limited evidence that they are effective, panelists said during a March 13 session of the AHIP Medicare, Medicaid, Duals & Commercial Markets Forum in Baltimore.

“One of the challenges for insurers going forward will be the increasing need to manage uncertainty,” said Daniel Ollendorf, Ph.D., chief scientific officer at the Institute for Clinical and Economic Review (ICER). Many drug launch prices, he said, “are being driven by approvals that happen on an accelerated basis with very limited evidence.”

Late last year, the FDA approved a new regimen for urothelial cancer that could transform the treatment of the condition, according to industry experts. However, the cost of the combination — which is estimated to be more than half a million dollars annually — could pose a barrier to its uptake.

On Dec. 15, 2023, the FDA approved Pfizer Inc. and Astellas Pharma Inc.’s Padcev (enfortumab vedotin-ejfv) in combination with Merck & Co., Inc.’s Keytruda (pembrolizumab) for the treatment of people with locally advanced or metastatic urothelial cancer (la/mUC). The agency gave accelerated approval to this combination for people with la/mUC who are not eligible for cisplatin-containing chemotherapy on April 3, 2023. The newest approval converts that accelerated approval to full and expands the labeled indication to include the treatment of people eligible for cisplatin chemotherapy. The application had priority review and breakthrough therapy designation.

Feb. 9: The FDA approved Aurobindo Pharma Limited’s deflazacort for the treatment of Duchenne muscular dystrophy (DMD) in people at least 5 years old. The corticosteroid is the first generic of PTC Therapeutics, Inc.’s Emflaza that the agency has approved. It has approval as a tablet for all of Emflaza’s dosage strengths: 6 mg, 18 mg, 30 mg and 36 mg. Emflaza also is available as an oral suspension. That agent is approved for the treatment of DMD in people at least 2 years old; it has exclusivity for the treatment of people ages 2 to 5 years old until June 2026. The recommended daily dosing is 0.9 mg/kg. Drugs.com lists the price of 30 18 mg tablets of Emflaza as more than $9,706.

Feb. 9: The FDA approved Takeda Pharmaceuticals U.S.A., Inc.’s Eohilia (budesonide) for the treatment of people at least 11 years old with eosinophilic esophagitis. The corticosteroid is the first and only FDA-approved oral treatment for the indication. Recommended dosing is 2 mg twice daily for 12 weeks; the agent is available in 2 mg/10 mL single-dose stick packs. Its monthly wholesale acquisition cost is $1,875.

The Federal Trade Commission (FTC) and HHS issued a request for information (RFI) on Feb. 14 regarding the role of health care group purchasing organizations (GPOs) and drug wholesalers in widespread generic drug shortages. The agencies are seeking public input about market concentration and contracting practices in those two industries and their involvement in the generic drug market. The agencies noted in a press release that they want information about “how both entities may influence the pricing and availability of pharmaceutical drugs.” Last year, the FTC announced an investigation into rebate-aggregating GPOs tied to major PBMs.

A federal judge on Feb. 12 dismissed a lawsuit brought by the trade group Pharmaceutical Research and Manufacturers of America (PhRMA) challenging the Medicare drug price negotiation program. PhRMA sought to overturn the section of the Inflation Reduction Act creating the program, which allows the federal government to negotiate the price of select high-cost medications. Individual drugmakers such as Merck & Co., Bristol Myers Squibb Co., Johnson & Johnson and AstraZeneca PLC all have similar suits underway. CMS on Feb. 1 sent initial price offers to the manufacturers of the first 10 medications that were selected for the Medicare drug price negotiation program, which goes into effect in 2026.

Jan. 12: The FDA broadened the label of Merck & Co., Inc.’s Keytruda (pembrolizumab) to include, in combination with chemoradiotherapy, the treatment of people with FIGO (International Federation of Gynecology and Obstetrics) 2014 Stage III-IVA cervical cancer. The agency initially approved the programmed death receptor-1 (PD-1) inhibitor on Sept. 4, 2014. The indication is the drug’s third in cervical cancer and 39th overall. The application was granted priority review. That review was conducted under Project Orbis in collaboration with the Australian Therapeutic Goods Administration and the Brazilian Health Regulatory Agency, and it used the Assessment Aid. Dosing is 200 mg every three weeks or 400 mg every six weeks via a 30-minute intravenous infusion. The agent’s price for every-three-weeks dosing is $11,115; for every-six-weeks dosing, it’s $22,230.

For years, payers have been concerned about the rising prices of prescription medications and how to cover newly approved drugs. Pharmaceutical experts tell AIS Health, a division of MMIT, that PBMs and plans will continue to be challenged in 2024 with similar issues, particularly when it comes to gene therapies, obesity medications and other expensive products.

Andy Szczotka, Pharm.D., chief pharmacy officer at AscellaHealth, notes that more than half of pharmacy benefit spending is on specialty medications even though only a small percentage of members use those drugs.

He says dealing with high-cost specialty products “is a focus for most payers” and adds there is a “large target on specialty drugs.”

Dec. 8: The FDA expanded the patient population of ADMA Biologics, Inc.’s Bivigam (immune globulin intravenous, human) to include the treatment of people at least 2 years old with primary humoral immunodeficiency. The agency initially approved the therapy on Dec. 21, 2012. The initial intravenous infusion rate is 0.5 mg/kg/minute for the first 10 minutes for a dose of 300-800 mg/kg every three to four weeks. The maintenance infusion rate may be increased every 20 minutes by 0.8 mg/kg/minute up to 6 mg/kg/minute. Drugs.com lists the price of a 50 mL intravenous solution as more than $756.

Dec. 13: The FDA approved US WorldMeds’ Iwilfin (eflornithine) to reduce the risk of relapse in adult and pediatric patients with high-risk neuroblastoma (HRNB) who have demonstrated at least a partial response to prior multiagent, multimodality therapy, including anti-disialoganglioside (anti-GD2) immunotherapy. Between 700 and 800 cases of neuroblastoma are diagnosed annually in the U.S., with 90% of those before age 5. About half of those cases in children are HRNB. The agency used the Real-Time Oncology Review pilot program and the Assessment Aid. It also granted the application priority review and breakthrough drug and orphan drug designations. The recommended dosing of the tablet is based on body surface area.

In December, the FDA approved the two newest cell and gene therapies, which were the first such agents approved for the treatment of sickle cell disease. As more of these products launch onto the U.S. market — the agency previously predicted that it would be approving 10 to 20 of the treatments by 2025 — payers are taking a variety of approaches to managing the therapies. Cost remains the main obstacle to their use, but their long-term durability also remains a question to some extent.

Dec. 8 saw the newest approvals, both for the treatment of sickle cell disease in people at least 12 years old: bluebird bio, Inc’s Lyfgenia (lovotibeglogene autotemcel; lovo-cel) and Vertex Pharmaceuticals Inc. and CRISPR Therapeutics’ Casgevy (exagamglogene autotemcel; exa-cel). The latter agent is the first CRISPR/Cas9 genome-edited cell therapy that the FDA has approved. The FDA gave both applications priority review, orphan drug, fast track and regenerative medicine advanced therapy designations. It also gave Lyfgenia rare pediatric disease designation.

The FDA recently approved the two newest cell and gene therapies, with one of them earning the distinction of being the first of its kind approved by the FDA. As more of these products launch onto the U.S. market — the agency previously predicted that it would be approving 10 to 20 of the treatments by 2025 — payers are taking a variety of approaches to managing the therapies.

Dec. 8 saw the newest approvals, both for the treatment of sickle cell disease in people at least 12 years old: bluebird bio, Inc’s Lyfgenia (lovotibeglogene autotemcel; lovo-cel) and Vertex Pharmaceuticals Inc. and CRISPR Therapeutics’ Casgevy (exagamglogene autotemcel; exa-cel). The latter agent is the first CRISPR/Cas9 genome-edited cell therapy that the FDA has approved. The one-time treatments come with hefty price tags: Lyfgenia is priced at $3.1 million and Casgevy at $2.2 million.

The ulcerative colitis (UC) space has seen multiple new approvals recently that could focus payer management even more on the condition. Payers already take an aggressive approach toward managing branded agents within the class, according to a Zitter Insights survey. That stance, say industry experts, will only grow with the new drugs and administration routes.

While no cure exists for the inflammatory bowel disease, the FDA has approved numerous agents to treat signs and symptoms of the condition. In addition to the nine biosimilars of AbbVie Inc.’s Humira (adalimumab) that have launched in the U.S. in 2023, a handful of other approvals happened from late September through late October. All of the agents offer maintenance dosing either through an oral or subcutaneous route of administration.