Neighborhood Health Plan RI Factor VIII/VWF Coverage

Coverage Criteria for Factor VIII/VWF Products

Initial coverage criteria

Covered when ALL of the following are met for Hemophilia A or vWD respectively:

Hemophilia A - diagnostic and indication criteria: Diagnosis of congenital factor VIII deficiency has been confirmed by blood coagulation testing; AND used as treatment for one of the following: treatment and control of acute bleeding episodes (episodic treatment of acute hemorrhage); OR perioperative management (authorization valid for 1 month); OR routine prophylaxis to prevent or reduce the frequency of bleeding episodes; AND used as primary prophylaxis in patients with severe factor VIII deficiency (factor VIII level of <1%); OR used as secondary prophylaxis in patients with at least TWO documented episodes of spontaneous bleeding into joints.

supported by [[6]] [[10]]

von Willebrand disease (vWD) - diagnostic and indication criteria: Diagnosis of von Willebrand disease has been confirmed by blood coagulation and von Willebrand factor testing; AND used as treatment for one of the following: spontaneous and trauma-induced bleeding episodes; OR surgical bleeding prophylaxis during major or minor procedures in patients with vWD in whom desmopressin (DDAVP) is ineffective or contraindicated (authorization valid for 1 month); OR perioperative management of bleeding; OR on-demand treatment and control of bleeding episodes in patients with severe vWD or in patients with mild or moderate vWD in whom desmopressin is known or suspected to be ineffective or contraindicated; OR routine prophylaxis to reduce the frequency of bleeding episodes; AND note that Alphanate is not indicated for patients with severe (type 3) vWD undergoing major surgery or treatment of spontaneous/trauma-induced bleeding episodes.

supported by [[8]] [[11]]

Renewal criteria

Renewal coverage conditions differ by indication:

Renewal for acute/on-demand indications: Patient continues to meet initial criteria; absence of unacceptable toxicity (examples include anaphylaxis/hypersensitivity, thromboembolic events, development of neutralizing antibodies/inhibitors, etc.); any increases in dose must be supported by an acceptable clinical rationale (e.g., weight gain, half-life study results, increased breakthrough bleeding despite adherence); the cumulative amount of medication the patient has on-hand will be taken into account when authorizing (authorization will allow up to 5 doses on-hand for treatment of acute bleeding episodes); Renewals for treatment and control of acute/on-demand bleeding episodes will be approved for a 6-month authorization period.Up to 5 on-hand doses permitted

supported by [[15]]

Renewal for routine prophylaxis: Patient continues to meet initial criteria; absence of unacceptable toxicity; patient has demonstrated a beneficial response to therapy (reduction in frequency of bleeding episodes from pre-treatment baseline); renewals will be approved for a 12-month authorization period.

supported by [[16]]

Perioperative management:

Coverage criteria for factor/VWF products

Covered when ALL of the following are met

Indication and prior therapy: Patient has hemophilia A or von Willebrand disease and requires control/prevention of bleeding or perioperative management; for VWD, product use is indicated when desmopressin (DDAVP) is ineffective or contraindicated; use must align with product-specific indications and supporting clinical evidence.

Supported by product indications and summary of evidence

Dosing targets by severity: Dosing and target plasma levels must correspond to bleed severity or surgical need. Examples from product labeling: minor targets — FVIII:C approximately 30–50% (30–50 IU/dL) or VWF:RCo target ~40–60 IU/dL; major/perioperative targets — FVIII:C 80–100% and VWF:RCo peak 100 IU/dL. Use weight-based calculations provided in product literature to compute IU dose.Examples: FVIII:C 30–50% (minor), 80–100% (major); VWF:RCo peak 100 IU/dL

Use product dosing tables (Humate-P, Wilate) to calculate dose per kg and target levels

Documentation for authorization: Prescriber must document patient weight, baseline FVIII and/or VWF:RCo levels, desired target level, calculation of IU dose (weight-based), and clinical indication (type of bleed or surgical procedure). For perioperative use include intended peak target levels and planned duration of therapy per product labeling.

General coverage criteria

Covered when use aligns with FDA-approved labeling or relevant clinical literature and plan criteria, with consideration of cost-effectiveness.

Plan review requirements: Use must be consistent with FDA-approved labeling or relevant clinical literature; prescriber must submit clinical information for individual consideration; coverage decisions will consider cost-effective options when applicable and appropriate; criteria apply in absence of Medicare NCD/LCD requirements for Medicare-Medicaid Plan members.

Supported by P&T committee rationale and plan review statements

The policy notes an explicit product-specific limitation for Alphanate: it is not indicated for patients with severe (type 3) von Willebrand disease undergoing major surgery or for treatment of spontaneous/trauma‑induced bleeding episodes. This statement is drawn from the Alphanate product information and the coverage rationale summarized in the evidence section, and should be used to guide authorization decisions when Alphanate is the requested product.

Use of Factor VIII/VWF complex products for von Willebrand disease is described as appropriate when desmopressin (DDAVP) is ineffective or contraindicated. The document does not describe coverage for indications that are not supported by the product labeling; requests for off‑label indications should be evaluated against FDA‑approved indications and the clinical evidence summarized in the policy.

Within the excerpt provided there are no additional explicit exclusions beyond the product‑specific statements (e.g., the Alphanate limitation noted above). The P&T committee framework indicates criteria are intended to ensure use for medically accepted indications rather than to enumerate broad additional exclusions.

Perioperative management and surgical bleeding prophylaxis are authorized for the perioperative period only and, per the renewal criteria, coverage for perioperative/surgical bleeding prophylaxis may NOT be renewed beyond the initial authorization period. Perioperative authorizations are intended to cover the immediate perioperative timeframe rather than ongoing therapy.

The policy states that Neighborhood will apply these coverage criteria in the absence of applicable Medicare National or Local Coverage Determinations. In other words, Medicare NCDs/LCDs/LCAs take precedence where they apply; local NCD/LCD provisions may define additional coverage or not medically necessary conditions that should be followed for Medicare members.

The document emphasizes that the criteria are designed to ensure use for medically accepted indications and that individual requests will be reviewed based on submitted prescriber information. However, within the provided excerpt there are no discrete, itemized 'not medically necessary' conditions beyond references to applying NCD/LCD rules where relevant and the product‑specific limitations summarized elsewhere.

Billing, Codes, and Dispensing Limits

Product maximum units per supplymixed

55,200

Max billable units per supply (listed per product)

Billing HCPCS / J-codesHCPCS

J-code(s)

HCPCS drug J-code(s) for factor products (specific J-codes listed in provider billing guidance/HCPCS section)

NDCNDC

NDC(s)

National Drug Codes for specific product vials (refer to product vial NDC list)

Covered ICD-10 Diagnosis CodesICD-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

inv-15: On-hand doses — ≤ 5 extra doses for treatment of acute bleeding episodes

On-hand dose limitPatients should not have more than 5 extra doses on-hand for the treatment of acute bleeding episodes

Dispensing considerationCumulative on-hand inventory must be considered when authorizing dispensing quantity

Monthly submission requirementRendering provider must request authorization of dispensing quantity monthly before delivery and include patient product inventory

inv-16: Minimally treated patient threshold — < 50 exposure days

Minimally treated patient definitionPatients with fewer than 50 exposure days to factor products

Prior Authorization, Documentation, and Billing Instructions

Prior Authorization

Authorization period and perioperative exception — Initial auth generally 3 months; perioperative auth valid 1 month

Unless otherwise specified, initial authorizations are generally provided for 3 months and may be renewed. Perioperative authorizations are valid for 1 month. The cumulative amount of medication the patient has on-hand will be taken into account for authorizations; up to 5 on‑hand doses for treatment of acute bleeding episodes will be permitted at the time of the authorization request. Initial and renewal authorization periods may vary by specific covered indication.

Step Therapy

Step therapy exemption for recent users — MMP members with prior use in past 365 days exempt from step therapy

MMP members who have received Alphanate or Humate‑P within the past 365 days are exempt from step therapy requirements.

Note

DDAVP consideration — For VWD, DDAVP preferred/first-line when effective; products indicated where DDAVP is ineffective or contraindicated

Clinical Background

Hemophilia A is a congenital deficiency of factor VIII and von Willebrand disease (vWD) is a disorder of von Willebrand factor; both conditions can present with spontaneous or procedure‑related bleeding. Treatment options include intravenous Factor VIII/VWF concentrates (e.g., Alphanate, Humate‑P, Wilate) for control of acute bleeding, perioperative management, and routine prophylaxis. Diagnostic confirmation by appropriate coagulation and VWF testing is required prior to use, and product selection should align with FDA‑approved indications and clinical evidence as adopted by the plan’s P&T committee.

Definitions and Key Terms

inv-31: FDA approved indications / Orphan drug — FDA indications for Hemophilia A and vWD and orphan status

FDA-approved indicationsProducts indicated for control and prevention of bleeding in Hemophilia A and for treatment/perioperative management in von Willebrand disease

Orphan drug statusDocument marks these Factor VIII/VWF complex products with orphan drug designation

Indication sourcesFDA-approved labeling and supporting product literature referenced in policy (package inserts summarized)

inv-32: Minimally treated patient — < 50 exposure days; special inhibitor testing applies

Definition — minimally treated patientPatient with < 50 exposure days to factor products

Special testing requirementMinimally treated patients previously on a different factor product require baseline inhibitor testing

OpenPayer

Hemophilia Products - Factor VIII/VWF Complex: Alphanate, Humate-P, Wilate (Intravenous)

Coverage Criteria for Factor VIII/VWF Products

Billing, Codes, and Dispensing Limits

Prior Authorization, Documentation, and Billing Instructions

Clinical Background

Definitions and Key Terms