Subject:
Hemophilia Products - Factor VIII (Hemophilia A) and Von Willebrand disease (VWD): Alphanate, Humate-P, and Wilate
Description:
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IMPORTANT NOTE:
The purpose of this policy is to provide general information applicable to the administration of health benefits that Horizon Blue Cross Blue Shield of New Jersey and Horizon Healthcare of New Jersey, Inc. (collectively “Horizon BCBSNJ”) insures or administers. If the member’s contract benefits differ from the medical policy, the contract prevails. Although a service, supply or procedure may be medically necessary, it may be subject to limitations and/or exclusions under a member’s benefit plan. If a service, supply or procedure is not covered and the member proceeds to obtain the service, supply or procedure, the member may be responsible for the cost. Decisions regarding treatment and treatment plans are the responsibility of the physician. This policy is not intended to direct the course of clinical care a physician provides to a member, and it does not replace a physician’s independent professional clinical judgment or duty to exercise special knowledge and skill in the treatment of Horizon BCBSNJ members. Horizon BCBSNJ is not responsible for, does not provide, and does not hold itself out as a provider of medical care. The physician remains responsible for the quality and type of health care services provided to a Horizon BCBSNJ member.
Horizon BCBSNJ medical policies do not constitute medical advice, authorization, certification, approval, explanation of benefits, offer of coverage, contract or guarantee of payment.
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Hemophilia is a rare disorder in which the blood does not clot normally. It is a medical condition in which the ability of the blood to clot is severely reduced, causing the sufferer to bleed severely from even a slight injury. The condition is typically caused by a hereditary lack of a coagulation factor, most often factor VIII. Hemophilia A and B are X-linked recessive diseases that present in male children of carrier females. However, hemophilia must sometimes be differentiated from other bleeding disorders when the family history is negative or unknown. Differentiation between hemophilia and other conditions, such as some types of von Willebrand disease or acquired factor inhibitors, and distinction between hemophilia A and B are crucial for appropriate management.
Hemophilia typically refers to an inherited bleeding disorder caused by deficiency of coagulation factor VIII (hemophilia A), factor IX (hemophilia B), or factor XI (hemophilia C). Hemophilia A – Inherited deficiency of factor VIII; is an X-linked recessive disorder. Hemophilia A is more common than hemophilia B, representing 80-85% of the total hemophilia population.
Von Willebrand disease (VWD) is the most common type of bleeding disorder however it is generally less severe than other bleeding disorders. People with VWD have a problem with a protein in their blood called von Willebrand factor (VWF) that helps control bleeding. For most people with VWD, the disorder causes little or no disruption to their lives except when there is a serious injury or need for surgery. It is estimated that up to 1% of the world’s population suffers from VWD, but because many people have only very mild symptoms, only a small number of them know they have it.
There are three main types of VWD. Within each type, the disorder can be mild, moderate, or severe. Bleeding symptoms can be quite variable within each type depending in part on the VWF activity.
1. Type 1 VWD is the most common form. People with Type 1 VWD have lower than normal levels of VWF. Symptoms are usually very mild. Still, it is possible for someone with Type 1 VWD to have serious bleeding.
2. Type 2 VWD involves a defect in the VWF structure. The VWF protein does not work properly, causing lower than normal VWF activity. Symptoms are usually moderate.
3. Type 3 VWD is usually the most serious form. People with Type 3 VWD have very little or no VWF. Symptoms are more severe. People with Type 3 VWD can have bleeding into muscles and joints, sometimes without injury.
Clinical manifestations of hemophilia A and VWD relate to bleeding from impaired hemostasis, sequelae from bleeding, or complications of coagulation factor infusion. Patients with moderate hemophilia often bleed in response to minor intercurrent injury and invasive procedures. Bleeding is less frequent than in severe hemophilia and typically occurs four to six times yearly. However, more frequent bleeding may occur if a target joint develops. Some patients with moderate hemophilia may express a more severe phenotype requiring the use of prophylactic treatment regimens. In contrast, individuals with mild hemophilia generally only have bleeding in response to injury/trauma or surgery, and bleeding may not become clinically apparent until later in life. Delayed bleeding can occur after minor surgical procedures such as tooth extraction, even in patients with mild disease.
The diagnostic evaluation in cases of suspected hemophilia typically begins with a thorough review of the patient's personal bleeding history and family history. Screening tests are then performed and the diagnosis is confirmed with a specific clotting factor activity measurement(s) and/or genetic testing. Laboratory testing is similar for the majority of patients with hemophilia. Initial testing includes screening tests of hemostasis, including prothrombin time (PT), activated partial thromboplastin time (aPTT), and platelet count. Mixing studies for the aPTT assay are performed if the aPTT is prolonged. If mixing studies show correction, consistent with a factor deficiency rather than an inhibitor, factor activity levels are then measured. An exception is diagnosis in a male neonate with a positive family history, in which factor levels are often measured directly on cord blood.
Table 1: The 2nd edition World Federation of Hemophilia (WFH) guidelines for management of hemophilia screening tests for hemophilia
Interpretation of screening tests
| Possible diagnosis | PT (prothrombin time) | APTT* (activated partial thromboplastin time) | BT (bleeding time) | Platelet Count |
| Normal | Normal | Normal | Normal | Normal |
| Hemophilia A or B** | Normal | Prolonged* | Normal | Normal |
| VWD | Normal | Normal or prolonged* | Normal or prolonged | Normal or reduced |
*Results of APTT measurements are highly dependent on the laboratory method used for analysis
**The same pattern can occur in presence of FXI, FXII, prekallikrein, or high molecular weight kininogen deficiencies
Table 2: The 2nd edition World Federation of Hemophilia (WFH) guidelines for management of hemophilia severity classification criteria for hemophilia
Relationship of bleeding severity to clotting factor level
| Severity | Clotting Factor Level | Bleeding Episodes |
| Severe | <1 IU/dl (<0.01 IU/ml) or <1% of normal | Spontaneous bleeding into joints or muscles, predominantly in the absence of identifiable hemostatic challenge |
| Moderate | 1-5 IU/dl (0.01-0.05 IU/ml) or 1-5% of normal | Occasional spontaneous bleeding; prolongs bleeding with minor trauma or surgery |
| Mild | 5-40 IU/dl (0.05-0.40 IU/ml) or 5-<40% of normal | Severe bleeding with major trauma or surgery. Spontaneous bleeding is rare. |
Table 3: Laboratory Tests for Von Willebrand Disease Diagnosis and Classification, adapted from the National Heart, Lung, and Blood Institute
Initial Tests
| Diagnosis | VWF:RCO (IU per dL)* | VWF:AG (IU per dL)* † | Factor VIII |
| Type 1 | < 30 | < 30 | ↓ or normal |
| Type 2A | < 30 | < 30 to 200 | ↓ or normal |
| Type 2B | < 30 | < 30 to 200 | ↓ or normal |
| Type 2M | < 30 | < 30 to 200 | ↓ or normal |
| Type 2N | 30 to 200 | 30 to 200 | ↓ ↓ |
| Type 3 | < 3 | < 3 | ↓ ↓ ↓ (<10 IU per dL) |
| Low VWF | 30 to 50§ | 30 to 50§ | Normal |
| Normal | 50 to 200 | 50 to 200 | Normal |
Note: These values represent prototypical cases without additional VWF (or other disease) abnormalities. Exceptions occur, and repeat testing and clinical experience may be necessary for interpretation of laboratory test results. Arrows refer to an increase or decrease in the test result compared with the laboratory reference range.
[VWD = von Willebrand disease; VWF = von Willebrand factor; VWF:Ag = VWF antigen; VWF:RCo = VWF ristocetin cofactor activity]
*VWF levels of less than 30 IU per dL are recommended for the definite diagnosis of VWD (especially type 1 disease) because many persons in the United States have type O blood, which is associated with low VWF levels without VWD; bleeding symptoms are common in persons without VWD; and no abnormality in the VWF gene has been identified in many persons with mildly to moderately decreased VWF levels.
†VWF:Ag is less than 50 IU per dL in most patients with type 2A, 2B, or 2M VWD.
§— Does not preclude the diagnosis of VWD in patients with a VWF:RCo of 30 to 50 IU per dL if there is supporting clinical evidence or family history of VWD, and does not preclude the use of agents to increase VWF levels in those who have a VWF:RCo of 30 to 50 IU per dL and may be at risk of bleeding.
Policy:
(NOTE: For Medicare Advantage, please refer to the Medicare Coverage Section below for coverage guidance.)
I. When anti-hemophilia products are considered medically necessary, initial therapy will be approved based on the following:
1. The provider must provide the actual prescribed dose with ALL of the following supporting documentation:
a. Patient’s age
b. Patient’s weight
c. Severity of the factor deficiency
i. (i.e., severe = <1% factor activity, moderate = ≥1 to ≤5% factor activity, mild = >5 to 40% factory activity)
d. Bleed history
e. Inhibitor status
f. Intended use/regimen: prophylaxis, on-demand, peri-operative
II. Alphanate, Humate-P, and Wilate are considered medically necessary for any of the following FDA-approved conditions:
1. Hemophilia A (congenital factor VIII deficiency)
· Diagnosis of congenital factor VIII deficiency has been confirmed by blood coagulation testing; AND
· Used as treatment in at least one of the following:
· Control and prevention of acute bleeding episodes (hemorrhage); OR
· Routine prophylaxis to prevent or reduce the frequency of bleeding episodes; OR
· Patient must have severe hemophilia A (factor VIII level of <1%); OR
· Patient must have at least two documented episodes of spontaneous bleeding into joints
· Perioperative management;
· If the request is for routine prophylaxis and the requested dose exceeds a total weekly dose of 160 IU/kg, a half-life study should be performed to determine the appropriate dose and dosing interval.
· For members with a BMI ≥ 30, a half-life study should be performed to determine the appropriate dose and dosing interval.
· For minimally treated patients (< 50 exposure days to factor products) previously receiving a different factor product, inhibitor testing is required at baseline in any patient that has received factor, then at every comprehensive care visit (yearly for the mild and moderate patients, semi-annually for the severe patients).
2. Von Willebrand disease (VWD)
· Diagnosis of von Willebrand disease has been confirmed by blood coagulation and von Willebrand factor testing; AND
· Treatment of spontaneous and trauma-induced bleeding episodes; OR
· Used as surgical bleeding prophylaxis during major or minor procedures in patients with vWD in whom desmopressin acetate (DDAVP) is either ineffective or contraindicated; AND
· Alphanate is not indicated for patients with severe (type 3) vWD undergoing major surgery
· Humate-P is not indicated for the prophylaxis of spontaneous bleeding episodes in vWD.
III. When medically necessary, Alphante, and Humate-P, and Wilate will be approved based on the following FDA approved dosing (see tables below).
1) Initial authorization periods vary by specific indication:
a. Control and prevention of acute bleeding episodes (hemorrhage)
i. Unless otherwise specified, the initial authorization for acute treatment will be 6 months and may be renewed.
§ The cumulative amount of medication(s) the patient has on-hand will be taken into account when authorizing. The authorization will allow up to 5 doses on-hand for the treatment of acute bleeding episodes as needed for the duration of the authorization.
b. Routine prophylaxis to prevent or reduce the frequency of bleeding episodes
i. Unless otherwise specified, the initial authorization for prophylaxis will be 12 months and may be renewed.
c. Perioperative management
i. Unless otherwise specified, the authorization is valid for 1 month
2) For patients whom PK test is recommended, initial approval will be for 3 months and renewal will be based upon completion of the PK test.
[INFORMATIONAL NOTE: Requirements for half-life study are a part of the hemophilia management program. This information is not meant to replace clinical decision making when initiating or modifying medication therapy and should only be used as a guide.]
3) Dispensing Requirements for Renderings Providers
a. Prescriptions cannot be filled without an expressed need from the patient, caregiver or prescribing practitioner. Auto-filling is not allowed.
b. Monthly, rendering provider must submit for authorization of dispensing quantity before delivering factor product. Information submitted must include:
· Original prescription information, requested amount to be dispensed and patient clinical history (including patient product inventory and bleed history)
· Factor dose should not exceed +1% of the prescribed dose and a maximum of three vials may be dispensed per dose. If unable to provide factor dosing within required range of prescribed dose, then rendering provider must provide proof of all available vial sizes from the manufacturer prior to dispensing factor product and the lowest possible dose able to be achieved above +1% should be dispensed. Prescribed dose should not be increased to meet assay management requirements.
c. The prescriber should discuss with the patient that a treatment log should be maintained and a copy should be submitted (via prescriber or pharmacy) upon request for renewal purposes.
Alphanate
| Indication | Dose |
| Control and prevention of bleeding Congenital Hemophilia A | The expected in vivo peak increase in FVIII level expressed as IU/dL (or % normal) can be estimated using the following formulas:
Dosage (units) = body weight (kg) x desired FVIII rise (IU/dL or % normal) x 0.5 (IU/kg per IU/dL)
Minor
FVIII:C levels should be brought to 30% of normal (15 IU FVIII/kg twice daily) until hemorrhage stops and healing has been achieved (1-2 days).
Moderate
FVIII:C levels should be brought to 50% (25 IU FVIII/Kg twice daily). Treatment should continue until healing has been achieved (2-7 days, on average).
Major
FVIII:C levels should be brought to 80-100% for at least 3-5 days (40-50 IU FVIII/kg twice daily). Following this treatment period, FVIII levels should be maintained at 50% (25 IU FVIII/kg twice daily) until healing has been achieved. Major hemorrhages may require treatment for up to 10 days. Intracranial hemorrhages may require prophylaxis therapy for up to 6 months. |
| Perioperative management Congenital Hemophilia A | Prior to surgery, the levels of FVIII:C should be brought to 80-100% of normal (40-50 IU FVIII/kg). For the next 7-10 days, or until healing has been achieved, the patient should be maintained at 60-100% FVIII levels (30-50 IU FVIII/kg twice daily). |
| Control and prevention of bleeding and perioperative management von Willebrand Disease (VWD) | The ratio of VWF:RCo to FVIII in Alphanate varies by lot, so with each new lot, check the IU VWF:RCo/Vial to ensure accurate dosing.
Minor
Pre-operative/pre-procedure dose (Target FVIII:C Activity – 40-50 IU/dL):
Adults: 60 IU VWF:RCo/kg body weight.
Pediatrics: 75 IU VWF:RCo/kg body weight.
Maintenance dose (Target FVIII:C Activity – 40-50 IU/dL):
Adults: 40 to 60 IU VWF:RCo/kg body weight at 8 to 12 hour intervals as clinically needed for 1-3 days.
Pediatrics: 50 to 75 IU VWF:RCo/kg body weight at 8 to 12 hour intervals as clinically needed for 1-3 days.
Major
Pre-operative/pre-procedure dose (Target FVIII:C Activity – 100 IU/dL):
Adults: 60 IU VWF:RCo/kg body weight.
Pediatrics: 75 IU VWF:RCo/kg body weight.
Maintenance dose (Target FVIII:C Activity – 100 IU/dL):
Adults: 40 to 60 IU VWF:RCo/kg body weight at 8 to 12 hour intervals as clinically needed for at least 3-7 days.
Pediatrics: 50 to 75 IU VWF:RCo/kg body weight at 8 to 12 hour intervals as clinically needed for at least 3-7 days. |
Humate-P
| Indication | Dose |
| Control and prevention of bleeding Congenital Hemophilia A | One International Unit (IU) of Factor VIII (FVIII) activity per kg body weight will increase the circulating FVIII level by approximately 2.0 International Units (IU)/dL.
Minor
Loading dose 15 IU FVIII:C/kg to achieve a FVIII:C plasma level of approximately 30% of normal; one infusion may be sufficient. If needed, half of the loading dose may be given once or twice daily for 1-2 days.
Moderate
Loading dose 25 IU FVIII:C/kg to achieve a FVIII:C plasma level of approximately 50% of normal, followed by 15 IU FVIII:C/kg every 8-12 hours for the first 1-2 days to maintain the FVIII:C plasma level at 30% of normal. Continue the same dose once or twice daily for up to 7 days or until adequate wound healing is achieved.
Major
Initially 40-50 IU FVIII:C/kg, followed by 20-25 IU FVIII:C/kg every 8 hours to maintain the FVIII:C plasma level at 80-100% of normal for 7 days. Continue the same dose once or twice daily for another 7 days to maintain the FVIII:C level at 30-50% of normal. |
| Control and prevention of bleeding von Willebrand Disease (VWD) | Administer 40 to 80 International Units (IU) VWF:RCo (corresponding to 17 to 33 International Units (IU) FVIII in Humate-P) per kg body weight every 8 to 12 hours. Adjust the dosage based on the extent and location of bleeding. Administer repeat doses as long as needed based on monitoring of appropriate clinical and laboratory measures |
| Perioperative management von Willebrand Disease (VWD) | Loading Doses
Major
VWF:Rco Target Peak Plasma Level – 100 IU/dL
Target FVIII:C Activity – 80-100 IU/dL
((Target peak plasma VWF:RCo level – baseline plasma VWF:RCo level) –Body wt
(kg)) /IVR (in vivo recovery)
If the IVR is not available, assume an IVR of 2.0 IU/dL per IU/kg and calculate the
loading dose as follows: (100 – baseline plasma VWF:RCo) x BW (kg)/2.0
Minor
VWF:Rco Target Peak Plasma Level – 50-60 IU/dL
Target FVIII:C Activity – 40-50 IU/dL
((Target peak plasma VWF:RCo level – baseline plasma VWF:RCo level) –Body wt (kg)) /IVR (in vivo recovery)
Emergency
VWF:Rco Target Peak Plasma Level – 100 IU/dL
Target FVIII:C Activity – 80-100 IU/dL
Administer a dose of 50-60 IU VWF:RCo/kg body weight.
Maintenance Doses
The initial maintenance dose of Humate-P for the prevention of excessive bleeding during and after surgery should be half of the loading dose, irrespective of additional dosing required to meet FVIII:C targets. Subsequent maintenance doses should be based on the patient’s VWF:RCo and FVIII levels. |
Wilate
| Indication | Dose |
| Hemophilia A (Routine Prophylaxis) | 20 to 40 IU/kg every 2 to 3 days. Adjust dose based on clinical response. |
| Hemophilia A (Control of bleeding episodes) | Minor hemorrhage: 30 to 40 IU/kg every 12 to 24 hours. At least 1 day, until the hemorrhage has resolved.
Moderate hemorrhage: 30 to 40 IU/kg every 12 to 24 hours. Continue treatment at least 3 to 4 days and until hemorrhage stops.
Major hemorrhage: 35 to 50 IU/kg every 12 to 24 hours. Continue treatment at least 3 to 4 days and until hemorrhage stops.
Life-threatening hemorrhage: 35 to 50 IU/kg every 8 to 24 hours. Continue treatment until threat has resolved. |
| Control of bleeding episodes VWD | The ratio between VWF:RCo and FVIII activities in Wilate is approximately 1:1. The dosage should be adjusted according to the extent and location of the bleeding.
Minor and Moderate
Loading dose: 20-40 IU/kg ; Maintenance dose: 20-30 IU/kg every 12-24 hours until VWF:Rco and FVIII activity trough levels > 30%, for up to 3 days
Major
Loading dose: 40-60 IU/kg ; Maintenance dose: 20-40 IU/kg every 12-24 hours until VWF:Rco and FVIII activity trough levels > 50%, for up to 5-7 days |
| Perioperative management of bleeding vWD | Minor
Loading dose: 30-60 IU/kg ; Maintenance dose: 15-30 IU/kg every 12-24 hours until wound healing achieved, up to 3 days. VWF:Rco trough levels > 30% and peak levels 50%.
Major
Loading dose: 40-60 IU/kg ; Maintenance dose: 20-40 IU/kg every 12-24 hours until wound healing achieved, up to 6 days or more. VWF:Rco trough levels > 50% and peak levels 100%. |
IV. For continuing therapy, Alphanate, and Humate-P, and Wilate are considered medically necessary and approved when ALL of the following criteria are met:
· Patient continues to meet criteria identified in section I and II and dosing identified in section III; AND
· Absence of unacceptable toxicity from the drug. Examples of unacceptable toxicity include the following:
· Symptoms of allergic-anaphylactic reactions (anaphylaxis, dyspnea, rash);
· Thromboembolic events (thromboembolism, pulmonary embolism);
o Monitor plasma levels of VWF:RCo and FVIII activities to avoid sustained excessive VWF and FVIII activity levels greater than 150 IU/dL, which may increase the risk of thrombotic events; particularly in patients with known risk factors. Monitor the vWF:RCo and FVIII levels of vWD patients using standard coagulation tests, especially in cases of surgery. It is advisable to monitor trough vWF:RCo and FVIII:C levels at least once a day in order to adjust the dosage as needed to avoid excessive accumulation of coagulation factors.
· Development of neutralizing antibodies (inhibitors); AND
o Monitor for development of FVIII and VWF inhibitors. Perform appropriate assays to determine if FVIII and/or VWF inhibitor(s) are present if bleeding is not controlled with expected dose.
· Any increases in dose must be supported by an acceptable clinical rationale (i.e. weight gain, half-life study results, increase in breakthrough bleeding when patient is fully adherent to therapy, etc.).
· Monitor plasma FVIII levels periodically to elevate individual patient response to the dosage regimen. If dosing studies have determined that a particular patient exhibits a lower/higher than expected response and shorter/longer half-life, adjust the dose and the frequency of dosing accordingly.
· Alphanate and Humate-P contain blood group isoagglutinins. When doses are very large or need to be repeated frequently (for example when inhibitors are present or when pre- and post-surgical care is involved), monitor patients of blood groups A, B, and AB for singns of intravascular hemolysis and decreasing hematocrit values and treat appropriately.
· The cumulative amount of medication (s) the patient has on-hand will be taken into account when authorizing. The authorization will allow up to 5 doses on-hand for the treatment of acute bleeding episodes as needed for duration of the authorization.
· For the following disease specific criteria, renewal will be approved for a 6 month authorization period:
· Treatment of acute bleeding episodes
· Treatment of spontaneous and trauma-induced bleeding episodes
· On-demand treatment of bleeding episodes
· For the following disease specific criteria, renewal will be approved for a 12 month authorization period:
· Prevention of acute bleeding episodes
· Routine prophylaxis to prevent or reduce the frequency of bleeding episodes
· Dispensing Requirements for Renderings Providers
a. Prescriptions cannot be filled without an expressed need from the patient, caregiver or prescribing practitioner. Auto-filling is not allowed.
b. Monthly, rendering provider must submit for authorization of dispensing quantity before delivering factor product. Information submitted must include:
· Original prescription information, requested amount to be dispensed and patient clinical history (including patient product inventory and bleed history)
o Factor dose should not exceed +1% of the prescribed dose and a maximum of three vials may be dispensed per dose. If unable to provide factor dosing within required range of prescribed dose, then rendering provider must provide proof of all available vial sizes from the manufacturer prior to dispensing factor product and the lowest possible dose able to be achieved above +1% should be dispensed. Prescribed dose should not be increased to meet assay management requirements.
c. The prescriber should discuss with the patient that a treatment log should be maintained and a copy should be submitted (via prescriber or pharmacy) upon request for renewal purposes.
V. All other uses of Alphanate, Wilate, and Humate-P are considered investigational.
Medicare Coverage:
Hemophilia Products for Factor VIII (Hemophilia A) and Von Willebrand disease (VWD) are covered when NCD 110.3 and LCD L35111 criteria is met. For additional information and eligibility, refer to NCD 110.3 and L35111.
National Coverage Determination (NCD) for Anti-Inhibitor Coagulant Complex (AICC) (110.3). Available to be accessed at CMS National Coverage Determinations (NCDs) Alphabetical Index search page: https://www.cms.gov/medicare-coverage-database/indexes/ncd-alphabetical-index.aspx.
Local Coverage Determination (LCD): Hemophilia Factor Products (L35111). Available to be accessed at Novitas Solutions, Inc., Medical Policy Search page: https://www.novitas-solutions.com/webcenter/portal/MedicareJL/LcdSearch?_afrLoop=90769712476969#!%40%40%3F_afrLoop%3D90769712476969%26centerWidth%3D100%2525%26leftWidth%3D0%2525%26rightWidth%3D0%2525%26showFooter%3Dfalse%26showHeader%3Dfalse%26_adf.ctrl-state%3D63y7eftob_46.
Medicaid Coverage:
For Horizon NJ Health members, please follow this link for the corresponding HNJH drug policy https://services3.horizon-bcbsnj.com/ddn/NJhealthWeb.nsf
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Horizon BCBSNJ Medical Policy Development Process:
This Horizon BCBSNJ Medical Policy (the “Medical Policy”) has been developed by Horizon BCBSNJ’s Medical Policy Committee (the “Committee”) consistent with generally accepted standards of medical practice, and reflects Horizon BCBSNJ’s view of the subject health care services, supplies or procedures, and in what circumstances they are deemed to be medically necessary or experimental/ investigational in nature. This Medical Policy also considers whether and to what degree the subject health care services, supplies or procedures are clinically appropriate, in terms of type, frequency, extent, site and duration and if they are considered effective for the illnesses, injuries or diseases discussed. Where relevant, this Medical Policy considers whether the subject health care services, supplies or procedures are being requested primarily for the convenience of the covered person or the health care provider. It may also consider whether the services, supplies or procedures are more costly than an alternative service or sequence of services, supplies or procedures that are at least as likely to produce equivalent therapeutic or diagnostic results as to the diagnosis or treatment of the relevant illness, injury or disease. In reaching its conclusion regarding what it considers to be the generally accepted standards of medical practice, the Committee reviews and considers the following: all credible scientific evidence published in peer-reviewed medical literature generally recognized by the relevant medical community, physician and health care provider specialty society recommendations, the views of physicians and health care providers practicing in relevant clinical areas (including, but not limited to, the prevailing opinion within the appropriate specialty) and any other relevant factor as determined by applicable State and Federal laws and regulations.
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Index:
Hemophilia Products - Factor VIII (Hemophilia A) and Von Willebrand Disease (VWD): Alphanate and Humate-P
Alphanate
Humate-P
Wilate
Factor VIII and Von Willebrand Disease
Hemophilia A and Von Willebrand Disease
References:
1. Alphanate [package insert]. Los Angeles, CA; Grifols Biologicals Inc.; June 2018.
2. Humate-P [package insert]. Kankakee, IL; CSL Behring LLC; September 2017.
3. MASAC RECOMMENDATIONS CONCERNING PRODUCTS LICENSED FOR THE TREATMENT OF HEMOPHILIA AND OTHER BLEEDING DISORDERS. 2013 National Hemophilia Foundation. MASAC Document #240; February 2016. Available at: http://www.hemophilia.org. Accessed June 2016.
4. First Coast Service Options, Inc. Local Coverage Determination (LCD): Hemophilia Clotting Factors (L33684). Centers for Medicare & Medicaid Services, Inc. Updated on 01/21/2016 with effective date 01/01/2016. Accessed February 2016.
5. Novitas Solutions, Inc. Local Coverage Determination (LCD): Hemophilia Clotting Factors (L35111). Centers for Medicare & Medicaid Services, Inc. Updated on 01/22/2016 with effective date 01/01/2016. Accessed February 2016.
6. Annual Review of Factor Replacement Products. Oklahoma Health Care Authority Review Board. Updated April 2016. Access June 2016.
7. Graham A1, Jaworski K. Pharmacokinetic analysis of anti-hemophilic factor in the obese patient. Haemophilia. 2014 Mar;20(2):226-9.
8. Croteau SE1, Neufeld EJ. Transition considerations for extended half-life factor products. Haemophilia. 2015 May;21(3):285-8.
9. Mingot-Castellano, et al. Application of Pharmacokinetics Programs in Optimization of Haemostatic Treatment in Severe Hemophilia a Patients: Changes in Consumption, Clinical Outcomes and Quality of Life. Blood. 2014 December; 124 (21).
10. Blanchette VS, Key NS, Ljung LR, et al. Definitions in hemophilia: communication from the SSC of the ISTH. J Thromb Haemost 2014; 12:1935.
11. Hemlibra (emicizumab-kxwh) [package insert]. Genentech, Inc. South San Francisco, CA. November 2017. Accessed March 2018.
12. Guidelines for the management of hemophilia, 2nd edition. 2012 World Federation of Hemophilia. July 2012. Available at: https://www1.wfh.org/publication/files/pdf-1472.pdf. Accessed March 2018.
13. Collins PW, Chalmers E, Hart DP, et al. UK Haemophilia Centre Doctors. Diagnosis and treatment of factor VIII and IX inhibitors in congenital haemophilia: (4th edition). UK Haemophilia Centre Doctors Organization. Br J Haematol. 2013 Jan; 160(2): 153-70. Available on PubMed.
14. National Heart, Lung, and Blood Institute. The diagnosis, evaluation, and management of von Willebrand disease. Bethesda, Md.: National Institutes of Health; December 2007:36. NIH publication no. 08-5832. http://www.nhlbi.nih.gov/guidelines/vwd. Accessed March 7, 2018.
15. World Federation of Hemophilia. What is von Willebrand Disease (VWD)? Montreal, Quebec. Updated May 2014. https://www.wfh.org/en/page.aspx?pid=673. Accessed March 7, 2018.
16. Wilate [package insert]. Hoboken, NJ; Octapharma USA; September 2019.
Codes:
(The list of codes is not intended to be all-inclusive and is included below for informational purposes only. Inclusion or exclusion of a procedure, diagnosis, drug or device code(s) does not constitute or imply authorization, certification, approval, offer of coverage or guarantee of payment.)
CPT*
HCPCS
J7186
J7187
J7183
* CPT only copyright 2020 American Medical Association. All rights reserved. CPT is a registered trademark of the American Medical Association.
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Medical policies can be highly technical and are designed for use by the Horizon BCBSNJ professional staff in making coverage determinations. Members referring to this policy should discuss it with their treating physician, and should refer to their specific benefit plan for the terms, conditions, limitations and exclusions of their coverage.
The Horizon BCBSNJ Medical Policy Manual is proprietary. It is to be used only as authorized by Horizon BCBSNJ and its affiliates. The contents of this Medical Policy are not to be copied, reproduced or circulated to other parties without the express written consent of Horizon BCBSNJ. The contents of this Medical Policy may be updated or changed without notice, unless otherwise required by law and/or regulation. However, benefit determinations are made in the context of medical policies existing at the time of the decision and are not subject to later revision as the result of a change in medical policy
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