Neighborhood Health Plan RI Hemophilia Coverage Update

Coverage and Medical Necessity Criteria

Hemophilia A — Initial therapy

Covered when ALL of the following are met for Hemophilia A (congenital factor VIII deficiency):

Hemophilia A diagnosis: Diagnosis of congenital factor VIII deficiency confirmed by blood coagulation testing

Permitted uses: Used as treatment for control and prevention of bleeding episodes (episodic treatment of acute hemorrhage) OR perioperative management (authorization valid for 1 month) OR routine prophylaxis to prevent or reduce frequency of bleeding episodes

Perioperative management authorization valid for 1 month

Prophylaxis type: Primary prophylaxis for patients with severe factor VIII deficiency (factor VIII level <1%) OR secondary prophylaxis for patients with at least TWO documented episodes of spontaneous bleeding into joints

von Willebrand disease — Initial therapy

Covered when ALL of the following are met for von Willebrand disease (vWD):

vWD diagnosis: Diagnosis of von Willebrand disease confirmed by blood coagulation and von Willebrand factor testing

Permitted uses vWD: Used for spontaneous and trauma‑induced bleeding episodes OR surgical bleeding prophylaxis during major or minor procedures in patients in whom desmopressin is ineffective or contraindicated (authorization valid for 1 month) OR on‑demand treatment and control of bleeding episodes in patients with severe vWD or in mild/moderate vWD when desmopressin is ineffective or contraindicated OR routine prophylaxis to reduce frequency of bleeding episodes

Authorization valid for 1 month for perioperative use

vWD exclusions: Alphanate is not indicated for patients with severe (type 3) vWD undergoing major surgery or treatment of spontaneous/trauma‑induced bleeding episodes

See product‑specific labeling and exclusions

Renewal criteria

Renewal coverage conditions:

General renewal: Patient continues to meet Section III criteria AND absence of unacceptable toxicity (examples: anaphylaxis/hypersensitivity, thromboembolic events, development of neutralizing antibodies) AND no increase in breakthrough bleeding when adherent to therapy

Authorization will allow up to 5 doses on‑hand for treatment of acute bleeding episodes

Routine prophylaxis renewal: Renewals approved for 12 months AND patient has demonstrated beneficial response to therapy (reduced frequency of bleeding episodes from pre‑treatment baseline)

Routine prophylaxis renewals = 12 months

Perioperative renewals: Perioperative management/surgical bleeding prophylaxis coverage may NOT be renewed

Perioperative authorization periods are limited (see initial perioperative criteria)

Medical necessity: Indications and dosing

Covered when used for FDA‑approved indications and supported by clinical evidence per product labeling:

Indication: Product is prescribed for on‑demand treatment of bleeding, perioperative management, or routine prophylaxis in Hemophilia A or von Willebrand disease consistent with manufacturer labeling and supporting clinical studies

Clinical efficacy and safety summarized in product evidence sections

Product‑specific dosing guidance: Dosing must follow weight‑based calculations using VWF:RCo or FVIII targets; examples and ranges for minor, major, maintenance, and prophylaxis dosing are provided per product labeling (see Humate‑P, Wilate, and Alphanate dosing tables)

If IVR is not available, an IVR of 2.0 IU/dL per IU/kg may be assumed for Humate‑P

Individualization: Dosing and frequency should be titrated to clinical response, measured VWF:RCo and FVIII levels, and pharmacokinetic variability (IVR, half‑life)

Adjust per product labeling and measured laboratory response

Alphanate is not indicated for patients with severe (type 3) von Willebrand disease when used for major surgery or for treatment of spontaneous or trauma‑induced bleeding episodes. Do not authorize Alphanate for these scenarios unless documentation supports an alternative indication consistent with product labeling and policy exceptions.

For INTEGRITY (Medicare‑Medicaid Plan) members, these coverage criteria apply only in the absence of an applicable CMS National Coverage Determination (NCD) or Local Coverage Determination (LCD). When an NCD or LCD exists for the indication, the NCD/LCD guidance takes precedence for coverage decisions.

Dispensing beyond the permitted on‑hand limit or without required documentation may result in non‑authorization. Rendering providers must ensure patients do not have more than 5 extra doses on‑hand for treatment of acute bleeding, and must submit the required monthly authorization documentation (original prescription, requested amount, vial sizes, clinical history and patient product inventory) prior to delivering factor product. Failure to comply with these dispensing and documentation requirements may lead to denial or limitation of coverage.

Billing, Diagnosis and Coding

Billing codes and identifiersHCPCS | NDC

J-code	Manufacturer-specific J-code(s) per drug (see billing section)
NDC	Product NDC per vial (see billing section)

Covered Diagnosis Codes (ICD-10)ICD-10Covered

D66	Hereditary factor VIII deficiency
D68.01	Von Willebrand disease, type 1
D68.020	Von Willebrand disease, type 2A
D68.021	Von Willebrand disease, type 2B
D68.022	Von Willebrand disease, type 2M
D68.023	Von Willebrand disease, type 2N
D68.03	Von Willebrand disease, type 3
D68.04	Acquired von Willebrand disease
D68.09	Other von Willebrand disease

Expected VWF:RCo or FVIII rise per IU/kg — IVR and conversion

Expected rise (Wilate guidance)Approximately 1 IU VWF:RCo per kg body weight raises plasma VWF activity by ~2% (2 IU/dL). Use formula: Expected VWF:RCo rise (% of normal) = 2 x administered IU/kg.

Expected rise (Wilate — Factor VIII)For Factor VIII, 1 IU per kg raises plasma FVIII activity by ~2% (2 IU/dL) when measured by one‑stage clotting assay.

Humate‑P IVR guidanceWhen calculating Humate‑P dosing use measured IVR (in vivo recovery). If IVR unavailable, assume IVR = 2.0 IU/dL per IU/kg for loading dose calculations.

Dose calculation example (Humate‑P)Loading dose formula: (Target plasma VWF:RCo - baseline plasma VWF:RCo) x body weight (kg) / IVR; if IVR unknown use 2.0 IU/dL per IU/kg.

Authorization, Prior Authorization, and Documentation Requirements

Prior Authorization

Authorization periods and renewal

Initial authorization generally provided for 3 months; renewals considered per renewal criteria and may be approved for a 6-month period. The cumulative amount of medication the patient has on-hand will be taken into account when authorizing; up to 5 on-hand doses for treatment of acute bleeding episodes may be permitted at the time of the authorization request. Renewal decisions require continued meeting of clinical criteria and absence of unacceptable toxicity.

Initial authorization: typically 3 months
Renewal authorization: may be approved for 6 months when renewal criteria met
Plan will consider on-hand inventory; up to 5 on-hand doses allowed

Denial Risk

Medical necessity review requirement

Requests are reviewed for medical necessity to ensure alignment with FDA labeling and relevant clinical literature. The plan's Pharmacy & Therapeutics (P&T) committee favors use according to FDA-approved indications and may require justification when use deviates from labeling or when less costly, clinically appropriate alternatives exist. Neighborhood will give individual consideration to each request based on submitted prescriber information and available plan data.

Clinical Background

Hemophilia A is a congenital deficiency of factor VIII that presents with bleeding episodes requiring replacement of factor VIII; management includes on‑demand treatment of acute bleeds, perioperative replacement for surgical procedures, and routine prophylaxis in appropriately selected patients. von Willebrand disease (vWD) is a disorder of von Willebrand factor leading to mucocutaneous and surgical bleeding; management can include von Willebrand factor ± factor VIII replacement when desmopressin is ineffective or contraindicated. Factor VIII/VWF complex products (Humate‑P, Wilate, Alphanate) provide VWF:RCo and factor VIII activity and are used for control and prevention of bleeding, perioperative management, and routine prophylaxis as described in product labeling; dosing is weight‑based and individualized to clinical response and measured VWF:RCo or FVIII levels.

Key Definitions and Terms

Primary prophylaxis definition

DefinitionPrimary prophylaxis = routine prophylaxis for patients with severe factor VIII deficiency (factor VIII level <1%).

PurposeIntended to prevent or reduce the frequency of bleeding episodes in patients with severe congenital hemophilia A.

Authorization noteRoutine prophylaxis renewals are approved for 12 months when benefit demonstrated (reduced bleeding frequency from pre‑treatment baseline).

Secondary prophylaxis definition

DefinitionSecondary prophylaxis = routine prophylaxis for patients with at least two documented episodes of spontaneous bleeding into joints.

Use caseApplied to reduce recurrent joint bleeds in patients who have experienced ≥2 spontaneous joint bleeds.

OpenPayer

Hemophilia Products Factor VIII/ VWF Complex: Alphanate, Humate‑P, Wilate

Coverage and Medical Necessity Criteria

Billing, Diagnosis and Coding

Authorization, Prior Authorization, and Documentation Requirements

Clinical Background

Key Definitions and Terms