CHAPTER 1 USP Drug Classification and Coverage of Bleeding Disorder Treatments 

 

EXPERTS:  Aaron Blocker1, Nathan Schaefer1, Miriam Goldstein2, Kollet Koulianos1 Amy Dunn 3,4 1 National Bleeding Disorders Foundation, 2. Artemis Policy Group, 3. The Ohio State University College of Medicine, 4. Nationwide Children’s Hospital

 

 

TOPIC 1: USP Drug Classification for Bleeding Disorder Products

 

BACKGROUND

  • Affordable Care Act (ACA) standards for assessing whether health plan formularies satisfy ACA essential health benefit (EHB) requirements reference the USP Medicare Model Guidelines (USP MMG), an independent drug classification system designed by US Pharmacopeia.  ACA regulations set a floor of at least one drug per USP MMG category and class. 
    • The USP MMG, however, comprises only Medicare Part D drugs. Bleeding disorders drugs – covered under Medicare Part B – are not included in the USP MMG. This exclusion creates a serious gap in EHB protections for bleeding disorders patients.
    • In 2022, CMS published a request for information  (CMS-9898-NC, 87 Fed. Reg. 74097) asking, among other things, whether CMS should switch from the USP MMG to a broader compendium that includes Part B drugs: the USP DC or some other drug classification system. 
    • National bleeding disorders groups submitted comments supporting a switch to the USP DC – but cautioned that merely substituting the USP DC for the USP MMG would not, by itself, ensure adequate formulary coverage of bleeding disorders products, due in part to the existing structure of the USP DC classification system. 

 

EXISTING USP DC

  • Currently, the USP DC (“a hierarchical therapeutic drug classification system designed to assist with formulary support outside of Medicare Part D”) places all clotting factor treatments and all nonfactor treatments for Hemophilia A, Hemophilia B, Von Willebrand Disease (VWD) and rare deficiencies into a single class (Blood Component / Deficiency) within a single category (Blood Products and Modifiers). 
  • Aggregating all bleeding disorders products into a single category and class ignores clinically important differences among the therapies.
  • Many/most bleeding disorders therapies are disease-specific. For example, a hemophilia A clotting factor cannot appropriately be prescribed for the treatment of hemophilia B and vice versa.
  • Some therapies can be used only prophylactically, and are neither indicated nor effective for treatment of acute bleeding. 
  • Available treatments also differ in their mechanisms of action, routes of administration, thrombotic risk, etc. 

All these differences have clinical significance. USP disregards these important distinctions when it places all bleeding disorders therapies in a single category and a single class.

  • (The 2025 USP DC does recognize separate pharmacotherapeutic groups for Hemophilia A, Hemophilia B, VWD, and rare deficiencies; however, USP states that the pharmacotherapeutic groups are “not intended to be used for review of formulary adequacy” – leaving plans free to exclude disease-specific therapies while remaining technically ACA-compliant). 

 

 

RECOMMENDATION 1.1

  • MASAC recommends that the USP Drug Classification (USP DC) system separate all bleeding disorder drugs into distinct classes by disease area (Hemophilia A with and without inhibitors, Hemophilia B with or without inhibitors, von Willebrand disease, and rare factor deficiencies), rather than grouping them into a single class. 
  • REMARK 0001: The national bleeding disorders groups have recommended that CMS reference the USP DC (a classification system that includes Part B as well as Part D drugs) rather than the narrower USP MMG in its regulations establishing quantitative formulary standards.

REMARK 0002: The current USP DC structure aggregates all bleeding disorders therapies within a single category and class (products for multiple disparate blood disorder conditions, and products with distinct mechanisms of action and routes of administration). This structure allows some health plans to claim compliance with ACA EHB standards – even as their formularies exclude most or all available treatments for certain bleeding disorders.

  • REMARK 0003: Examples include a formulary that lists only one infused clotting factor (FVIII) along with a handful of low-cost oral and injection medicines – but excludes all products for, e.g., hemophilia B; hemophilia A with inhibitors; severe VWD.  Other formularies have excluded all subcutaneously administered therapies (potentially requiring parents of infants to transition to infused products) or have included prophylactic agents but no products to halt active bleeding.
  • REMARK 0004: Disaggregating the USP category and class of bleeding disorders products would better reflect medical practice and serve patient needs, while still meeting USP principles (no class containing fewer than three drug examples, while aligning with clinical reality and ensuring nondiscriminatory access to therapies). 

 

 

FUTURE RESEARCH NEEDS:

  • Evaluate the impact of USP DC classification changes on formulary design, patient access and outcomes in bleeding disorders.
  • Assess insurance practices exploiting USP DC gaps (e.g. formulary exclusions, copay maximizers, non-EHB strategies, alternative funding models).

 

REFERENCES

  1. U.S. Pharmacopeia. USP Drug Classification. https://www.usp.org

  2. Patient Protection and Affordable Care Act, Pub. L. No. 111–148, 124 Stat. 119 (2010).
  3. National Bleeding Disorders Foundation, Hemophilia Federation of America, Coalition for Hemophilia B, Hemophilia Alliance. Joint Letter to USP on Draft USP DC 2025. October 31, 2024.
  4. National Hemophilia Foundation, Hemophilia Federation of America. Joint Letter to US Department of Health and Human Services on Request for Information, Essential Health Benefits. January 31, 2023.

  5. National Bleeding Disorders Foundation, Hemophilia Federation of America, Coalition for Hemophilia B, Hemophilia Alliance. Joint Letter to USP on Draft USP DC 2025. November 11, 2025

 

KEYWORDS: USP Drug Classification, Affordable Care Act, Hemophilia A, Hemophilia B, von Willebrand Disease, rare factor deficiencies, formularies, drug access

Treatment Recommendations
MASAC Document 296 - Recommendations for Laboratory Screening of Iron Deficiency with and without Anemia
MASAC Documents
MASAC Document 298 - National Bleeding Disorder Foundation Recommendations on USP Drug Classification for Bleeding Disorders