When Formularies Fail: The Policy Breakdown Driving Hemophilia Drug Denials and How Patients and HTCs Can Fight Back
by Jennifer Borrillo and Miriam Goldstein

“How does a hemophilia patient obtain their lifesaving medication when their health plan no longer includes their drug on the plan’s specialty drug formulary?”

With the start of a new health plan year, some commercially insured HTC patients – and the HTCs who serve them – are once again discovering that patients’ medications have been dropped from their health plan formularies. The Hemophilia Alliance is aware of formularies that, for example, list only a single clotting factor, or (alternatively) include only a single non-factor product for prevention of bleeding. These narrow formularies create challenging issues for HTCs and patients alike. A medication used to treat hemophilia A does not work for a patient with factor IX deficiency; an agent indicated solely for prophylaxis will do nothing to halt breakthrough bleeding; and an IV-administered product may be an unacceptable option for an infant who is stable on an injected therapy – to note just a few examples.

Narrow formulary designs, unfortunately, are technically allowable under existing law. However, the Hemophilia Alliance in tandem with national partners is advocating for more robust standards, to provide a foundation for balanced formularies that meet the needs of the full patient community. As this advocacy continues, HTCs have tools and a key role to play in ensuring that patients can access the medications they need in order to live healthy lives.  

Strengthening formulary adequacy standards: Current federal regulations governing formulary adequacy say that formularies must cover at least one drug per USP (US Pharmacopeia) category and class. There are two problems with this benchmark. First, the rule cites a USP classification system that comprises only drugs covered under Medicare Part D – but of course most bleeding disorders products are covered under Medicare Part B, so they don’t appear on that USP listing. Second, a separate USP list that does include Part B drugs also has major flaws, insofar as it groups all bleeding disorders medications in one single category and class, without regard to disease treated, prophylactic vs. acute indication, route of administration, etc. Due to these limitations, even the narrowest formularies can claim to be compliant with existing USP-based standards for formulary adequacy.​

The Hemophilia Alliance and national bleeding disorders patient groups have undertaken a sustained, multi-pronged campaign to address the issue of formulary inadequacy. We are engaging with USP via letters and meetings, urging that body to revise its drug classification system to appropriately differentiate among bleeding disorders products based on condition and population treated, mechanism of action, and route of administration.​ We also continue to advocate with federal health and insurance regulators for better enforcement of existing standards; this includes urging regulators to give full weight to qualitative standards in existing law and protect patients against discriminatory plan benefit designs, including inadequate formularies.

Accessing medication TODAY: Meanwhile, patients still need access to their medication. Here are steps that patients and HTCs can take now when they encounter formulary exclusions and denials.

First and foremost, do not take “no” as a final answer! The patient has legal rights to appeal and/or to file a request for an exception. You (the HTC) play a critical role in that process. 

1. File an Urgent Formulary Exception Request: The HTC should immediately submit a “Formulary Exception Request” or “Prior Authorization” request, making the case that the patient’s medication is medically necessary. Provide documentation including (1) the patient’s treatment records and clinical history, and (2) journal articles or treatment guidelines supporting the use of prescribed medication.
2. Highlight Clinical Distinctions: There are important clinical differences between and among bleeding disorder medications based on: condition treated; prophylactic vs. acute indication; mechanism of action (factor replacement vs. non-factor); half-life; mode of administration; etc. Describe how this specific prescription is carefully tailored to the patient’s health needs.
3. Use the “Step Therapy” Rule Against Them: If the plan demands that the patient try a different, specified product first (“step therapy” or “fail first”), explain why that course of action could be clinically dangerous. Include information on any other treatment regimens previously tried by the patient that proved to be ineffective or harmful.
4. Request a Peer-to-Peer Review: Ask for the opportunity for your hematologist to speak directly with the health plan’s medical director to explain the clinical necessity of the patient’s prescribed medicine.
5. File an Internal and External Appeal: If the exceptions request is denied, file for an internal appeal and, if necessary, an external review (by an independent third party). Make sure to file these appeals within the timeframes specified by the health plan.
6. Contact Patient Assistance Programs: If your patient is in immediate need, reach out to the manufacturer’s patient assistance programs for bridging medication while appealing. 

Please contact your MCR primary contact if you have questions or need assistance navigating these steps.