Hemophilia Groups Protest BCBS of Tennessee Formulary Exclusions

As pharmacy benefit managers (PBMs) increasingly exclude drugs from their formularies, advocates for patients with hemophilia are concerned about the drugs for bleeding disorders that BlueCross BlueShield of Tennessee (BCBST) has dropped from its 2023 formulary.

The patient advocates say that in many cases the exclusions do not leave patients with acceptable alternatives. “The published BCBS formularies for 2023 exclude 17 to 19 different hemostatic agents. In doing so they endanger the health of potentially hundreds of Tennesseans who live with serious inherited blood disorders,” the Hemophilia Federation of America and the National Hemophilia Foundation wrote in a December 2022 letter to BCBST. The Tennessee Hemophilia and Bleeding Disorder Foundation has also objected to the formulary exclusions.

The hemophilia groups say that the Tennessee Blues plan has excluded all treatments for patients who develop resistance to standard clotting factor therapy. Treatments for rare bleeding disorders have also been excluded, such as factor VII and factor X deficiencies. The most common types of hemophilia, A and B, are characterized by deficiencies in factors VIII and IX, respectively.

The patient groups say that BCBST formulary has also cut off patients needing factor VIII therapy from an injectable option, requiring more frequent intravenous treatment.

“Denied access to life-sustaining treatment, affected individuals and their doctors would be forced to engage in challenging and time-consuming exception requests or appeals,” the advocacy groups said in a letter to BCBST, contending worse outcomes and higher overall healthcare costs will result.

A spokeswoman for BCBST told Managed Healthcare Executive that the 2023 formulary changes for hemophilia medication were reviewed by an independent panel of clinicians and that BCBST “reviewed and responded to outreach from hemophilia advocacy groups on several occasions” prior to implementing those changes.

“We continue to cover medications to treat hemophilia A, von Willebrand and hemophilia B. We also continue to cover popular drugs like Hemlibra (emicizumab-kxwh) in many of our plans,” said Dalya Qualls White, M.S., senior vice president and chief communications officer for BCBST. “We are willing to work with providers who wish to request specific drugs that are not on our formulary and have expanded the option to discuss those cases to ensure that any special circumstances have been considered.”

Hemophilia advocates estimated that BCBST has 75% of the Tennessee market for individual and group health insurance, which suggests many patients will be affected by the changes.

Qualls White said, though, that most of the bleeding disorder medications BCBST has removed from its formulary this year currently have zero utilization among its members and, in some cases, haven’t been used by its members in many years. Like most insurers, Qualls White added, BCBST has prior authorization requirements to promote use of the most appropriate and cost-effective treatments.

Costs of hemophilia care have soared over the past 15 years, when prophylaxis treatment started to become widespread. Drugs with longer half-lives mean fewer treatments for patients, but they have fuel the cost trend. Treatments to overcome immune system resistance (hemophilia with inhibitors) have also added to the tab.

The innovations have placed enormous cost pressure on payers. Estimates of average annual hemophilia treatment expense vary widely. An annual cost figure of $300,000 is widely used. Eloctate, a factor VIII prophylaxis product, can cost close to $1 million a year.

And there are even more expensive treatments. The new, one-time gene therapy for hemophilia B, Hemgenix (etranacogene dezaparvovec) that the FDA approved in November 2022 is priced at $3.5 million. However, the hope is that treatment will eliminate or drastically reduce the need for factor replacement and so possibly result in savings in the long run.

“As potentially curative gene therapies for hemophilia A and B are poised to enter the market, there is little sign of a pricing slowdown,” the American Society of Hematology wrote in 2020.

Magellan Rx stated in its 2022 pharmacy trend report that 69% of payers have resorted to care management including dose consolidation to reduce costs of hemophilia treatment.

The three largest PBMs — Optum Rx, CVS, and Express Scripts — have removed a number of hemophilia drugs from their formularies, although there is little overlap in the drugs selected for removal. For example, Optum Rx removed Sevenfact (coagulation factor VIIa (recombinant)-jncw), a bypassing agent for inhibitors, but CVS and Express Scripts kept it on their formularies. Optum Rx also removed Esperoct (antihemophilic factor (recombinant), glycopegylated-exei), an extended half-life factor VIII replacement therapy, but Express Scripts allows it and CVS makes no mention of it.

The battle that these national hemophilia advocacy groups are fighting against BCBST appears to be an isolated case.

Hemophilia drugs are among the priciest in the nation. In 2018, AllianceBernstein estimated the market value at $4.6 billion, yet the patient population is just around 20,000.

Competition has failed to bring down prices because payers are leery of the tarnished image that could result from denying essential care, and manufacturers reach out directly to patients and their families, making it difficult for doctors to insist on certain drugs, according to Kaiser Health News.

This story originally on Managed Healthcare Executive.