Jun 28, 2021 2:47:25 PM | 11 Min Read

The High Cost of Hemophilia A Treatments

The High Cost of Hemophilia A Treatments

Managing the costs of a rare and complex disease

What is Hemophilia A?
Hemophilia A is a rare, chronic, inherited disease that occurs in about 1 of every 5000 male births in the United States (hemophilia A is rarely symptomatic in females). During the period of 2012-2018, it was estimated that 20,000-33,000 males in the United States were living with hemophilia A. It is a bleeding disorder caused by lack of a crucial factor in the body’s coagulation process, known as factor VIII. People with hemophilia are susceptible to internal bleeding which can be fatal if it occurs in the brain. Bleeding into the joints, which can cause severe disfigurement, is common and can result in significant disability.

Aside from caution to avoid injury and bruising, treatment historically has been based on infusion of the missing coagulation factor, either in response to an active bleed or to maintain levels in the bloodstream sufficient to prevent bleeding. There are numerous factor VIII replacement products (FVIII) available that may be plasma derived or recombinant, and standard-acting and long-acting. FVIII infusions continue to be the primary treatment for hemophilia, but in November of 2017 a biological product for treating hemophilia A called Hemlibra (generic name, emicizumab) was approved by the FDA. Hemlibra acts by binding with other factors involved in the coagulation process, allowing bypass of factor VIII in that process.

FVIII is dosed differently based on the person’s own baseline factor VIII levels: “mild” hemophilia is defined as levels that are 6-40% of normal, “moderate” is levels 1-5% of normal, and “severe” is defined as levels less than 1% of normal. Treatment with FVIII products can be complicated by development of “inhibitors”, part of the body’s immune response that inactivate factor VIII. Once a person has developed inhibitors, the amount of FVIII needed to prevent or treat bleeding can greatly increase; in some cases, special treatments are used to bypass or mimic the role of factor VIII. The CDC estimates that there are about 1500 people in the United States living with inhibitors.

The Cost Of Hemophilia A Treatment
A person with severe hemophilia may incur annual drug costs exceeding $250,000. Development of inhibitors can increase that cost to $1,000,000 per year. And although availability of recombinant FVIII products and extensive screening for donated blood have drastically reduced the risk of contracting HIV or hepatitis from treatments, that risk remains and can mean years of suffering and even higher cost for those who are victims.

The current market just for hemophilia FVIII replacement products and drugs has been estimated as close to $11 billion, and it is expected that the market will continue to expand.

A major disrupter is potentially in the wings for hemophilia treatment. Known as Roctavian (generic name valoctocogene roxaparvovec), this gene therapy product is likely to have a very large (estimated seven-figure) up-front cost like other potentially curative gene therapies such as Zolgensma. Although it may be cost effective in the long run, given the possibility of eliminating millions of dollars’ worth of care for each person treated, coverage is problematic because 1) the payer that covers the treatment cost is not likely to retain the member long enough to recoup the cost through future savings; and 2) some payers may simply not have funding to pay such a large sum at one time. This dilemma is not unique to hemophilia gene therapy, and experts are still experimenting with coverage models that will solve it. In the meantime, FDA approval of Roctavian, which had been anticipated in October of 2020, has been delayed as the FDA is requiring further research on the long-term benefits of the product.

Variables In Product And Cost Management

FVIII Assays

Stocking and dispensing hemophilia products is a bit tricky. No two vials contain exactly the same amount, even though they may be labeled the same. The exact amount or “assay” of FVIII in the vials is indicated on the label, and pharmacies must take this figure into account when they are determining the number of vials to dispense for a given prescription.

Adding to the complexity is the fact that the supply of these products is sometimes limited. A pharmacy may or may not be able to obtain the vial size they desire, even though the manufacturer advertises that they produce it. The result of this is the need to allow some variance between the dose prescribed and the dose dispensed. The standard of practice is to allow 5-10% more or less than the prescribed dose to be dispensed.

Payers should ensure that dispensing pharmacies are staying within those margins; some even demand guarantees.

Patient Supplies

Persons with hemophilia typically keep a supply of FVIII on hand to quickly respond to unexpected bleeding. Given the cost of these products, however, it is important to ensure that patients are not “stockpiling”. In addition, since these back-up stores may never be used, there is potential for them to reach their expiration dates and require disposal.

Due to the cost of FVIII, both of these possibilities warrant monitoring by the payer or its agent (such as a specialty pharmacy).

Hemophilia Treatment Centers and 340b

Hemophilia treatment centers (HTCs) are rapidly becoming the preferred providers for persons with hemophilia. These federally funded centers were created to provide single facilities where comprehensive hemophilia care takes place. There are about 141 of them in the US. Because they focus solely on hemophilia, all their resources are devoted to support for people with hemophilia. In addition to providing treatments, they address the physical, emotional, psychological, educational, financial and vocational needs of patients and families, provide education to providers and the general public, and support and/or participate in research. The 340B Drug Pricing Program is a federal program which allows certain hospitals and other health care providers called “covered entities” to obtain discounted prices on “covered outpatient drugs” (prescription drugs and biologics other than vaccines) from drug manufacturers. HTCs are covered entities eligible to participate in the 340b program. This means that drugs dispensed by these centers for people who see physicians employed by or contracted with them are purchased at a highly discounted rate. These savings may be passed along to the payer, or the HTC may use the funds to provide more comprehensive services. At least 103 HTCs have established 340b programs.

Benefit Coverage

Yet another confounding issue in managing hemophilia products relates to benefit coverage.

These products require intravenous infusion, an administration method that is usually provided by health care professionals in a doctor’s office, clinic, infusion center or through a home health agency. Most people associate these sites of care with coverage under the medical benefit. However, due to the urgency needed when treating a bleed, many persons with hemophilia (or their caregivers) are trained to prepare and infuse doses themselves. Self-administration of drugs is more commonly associated with the pharmacy benefit.

Coverage under the medical benefit is different in many ways from coverage under the pharmacy benefit. These differences include sourcing of drug products, retrospective vs. point of service claims adjudication and availability/handling of manufacturer concessions like rebates. Given all of these variables, it is not surprising that hemophilia products can be found in both pharmacy and medical claims.

In polling three different hemophilia resources, Confidio received three different answers when asked which benefit these products are typically covered under. One resource stated that most coverage is under medical. Another source told us that approximately 60% of FVIII prescriptions are covered under pharmacy, and a third source responded that it varies but there is a trend towards covering under the pharmacy benefit. It appears that the answer is, “it depends”.

It is important for a plan to examine all aspects of hemophilia product coverage before deciding where to direct these claims. These include product discounts, rebates and reimbursement rates available through each benefit. Will your PBM specialty pharmacy be able to access and pass through any savings associated with a 340b program or are the savings only available through a HTC pharmacy / home infusion provider? Note that any products sourced under a 340b program will not qualify for rebates. Another key factor to consider is whether the medical carrier has a hemophilia case management program and if carved out pharmacy benefit claims are used to trigger case management. If hemophilia products are covered under the pharmacy benefit, the medical carrier may not identify the member as being a candidate for their case management program.

Payers have a lot of things to consider when addressing the high cost of hemophilia treatments. At a high level, these fall into the following categories:
  • Maximizing member cost share assistance programs
  • Investigating hemophilia-specific foundation support programs
  • Assuring that evidence-based utilization and care management programs sponsored by your benefit provider are in place
  • Requiring the dispensing pharmacy to tightly adhere to appropriate assay and dispensing management practices
  • Investigating whether your benefit offers a Center of Excellence or similar offering that includes Hemophilia Treatment Centers
  • Exploring any relevant high cost therapy benefit protection insurance offerings (such as ones for gene therapy)

Clients trying to manage the cost of hemophilia products need to leverage every opportunity for cost savings. In addition to the hemophilia-specific considerations listed above, standard pharmacy benefit management measures should be optimized. These include patient cost share assistance programs, manufacturer rebates, utilization management (prior authorization, quantity limits, etc.), formulary/drug preferencing, dispensing channel management, case management and/or medication therapy management programs, etc.

Paying for treatment of hemophilia is complex and can be difficult. To assist our clients, Confidio has generated a detailed, comprehensive checklist with actionable steps that payers can take to ensure they have optimal clinical management and cost reduction strategies for hemophilia treatments.

Follow up with your Confidio representative to learn more about our proprietary checklist.

  1. CDC. (2020, October 29). Inhibitors and Hemophilia. Retrieved from cdc.gov: https://www.cdc.gov/ncbddd/hemophilia/inhibitors.html
  2. Cichocki, M. (2020, May 23). What Is the Risk of HIV in Hemophiliacs? Retrieved from www.verywellhealth.com: https://www.verywellhealth.com/hemophilia-andhiv48852#:~:text=In%20fact%2C%20since%20the%20earliest%20part%
  3. Gateway Hemophilia Association. (2020, October 30). Hemophilia Treatment Centers. Retrieved from Gatewayhemophilia.org: https://gatewayhemophilia.org/resources/treatment-centers/
  4. IBM Watson. (2020, January 9). IBM Micromedex. Armonk, New York, United States.
  5. IPD Analytics. (2020). Hemophilia–Update on Treatment Management. Bay Harbor Islands, FL: IPD Analytics.
  6. Malouin RA, M. L. (2018). Impact of the 340B Pharmacy Program on Services and Supports for Persons Served by Hemophilia Treatment Centers in the United States. Maternal and Child Health Journal, https://doi.org/10.1007/s10995-018-2545-7.
  7. National Hemophilia Foundation. (2015, July 16). NHF Policy Statement: Hemophilia Treatment Center Participation in the 340B Drug Discount Program. Retrieved from www.hemophilia.org: https://www.hemophilia.org/sites/default/files/document/files/NHF-Position-Statement-Regarding-Hemophilia-Treatment-Center-Participation-in-the340BDrugDiscount
  8. NHF. (2020, October 30). MASAC Recommendations Regarding Standards of Service for Pharmacy Providers of Clotting Factor Concentrates for Home Use to Patients with Bleeding Disorders. Retrieved from www.hemophilia.org: https://www.hemophilia.org/Researchers-Healthcare-Providers/Medical-and-Scientific-Advisory-Council-MASAC/MASAC-Recommendations/MASAC-Recommendations-Regarding-Standards-of-Service-for-Pharmacy-Providers-of-Clotting-Factor-Concentrates-for-Home-Use-to-Pat

Topics: Hemophilia, Treatments

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