Description:
Human antihemophilic factor (AHF, Factor VIII) maintenance therapy is used as an alternative to "on-demand" therapy to reduce the incidence of bleeding episodes and resultant joint damage in patients who have severe hemophilia A (AHF activity < 1% of normal). In on-demand therapy, human AHF concentrate is infused when trauma or joint pain is observed consistent with internal bleeding.

During maintenance therapy, also known as prophylactic therapy, human AHF concentrate is infused on a weekly schedule with the goal of maintaining AHF activity at a level sufficient to prevent bleeding (generally AHF activity greater than 1%).

This policy specifically applies to the following: 

ADVATE, HELIXATE FS, KOGENATE FS, RECOMBINATE, XYNTHA, NOVOEIGHT, ADYNOVATE, ELOCTATE, ALPHANATE, HUMATE-P, KOATE

(antihemophilic factor VIII [recombinant])  

HEMOFIL M, MONOCLATE-P

(antihemophilic factor VIII [human] monoclonal antibody purified)  

JIVI

(antihemophilic factor [recombinant] PEGylated-aucl)

HEMLIBRA^®

(emicizumab-kxwh)

Policy:
Coverage of these medications is provided when the FDA approved indications below are met and there has been a trial and failure of preferred therapy.

The use of human antihemophilic factor (AHF) maintenance therapy for severe hemophilia A (i.e., AHF activity < 1% of normal) is MEDICALLY NECESSARY for the follwoing:

• Maintain the trough AHF activity at greater than 1%
• For the treatment of acute and significant bleeding
• Member has had an insufficient response to desmopressin OR one of the following clinical reasons for not trying desmopressin first:
  • Age < 2 years
  • Pregnancy
  • Fluid/electrolyte imbalance
  • High risk of cardiovascular or cerebrovascular disease (especially elderly)
  • Predisposition to thrombus formation
  • Trauma requiring surgery
  • Life-threatening bleed
  • Contraindication or intolerance to desmopressin

Effective 01/01/2021 Hemlibra^® (emicizumab-kxwh) Will be considered MEDICALLY NECESSARY WHEN THE FOLLOWING CRITERIA IS MET:

1. ONE of the following:
   1. There is documentation that the patient is currently being treated with the requested agent (starting on samples is not approvable) OR
   2. The prescriber states the patient is using the requested agent (starting on samples is not approvable) and is at risk if therapy is changed OR
   3. ALL of the following:
      1. The patient has a diagnosis of hemophilia A with documented Factor VIII inhibitors AND
      2. The requested agent is prescribed for prophylactic use AND
      3. The prescriber is a specialist in the area of the patient’s diagnosis [e.g., prescriber working in a hemophilia treatment center (HTC), hematologist with hemophilia experience] or has consulted with a specialist in the area of the patient’s diagnosis AND
      4. ONE of the following:
         1. The patient’s inhibitor level is ≥ 5 BU OR
         2. The patient has tried and had an inadequate response to Immune Tolerance Therapy (ITT) (Immune Tolerance Induction (ITI)) OR
         3. The patient is using a bypassing agent (Feiba, NovoSeven) for ondemand treatment and is not adequately controlled OR
         4. The patient is using a bypassing agent for prophylaxis and had an inadequate response OR
         5. The patient is using more than 5 doses per week of a bypassing agent OR
         6. The patient has a documented intolerance, FDA labeled contraindication or hypersensitivity to a bypassing agent AND
2. If  the patient is using a Factor VIII product (e.g., Advate, Adynovate, Eloctate, Nuwiq, Recombinate, Xyntha) or a bypassing agent (e.g., Feiba, NovoSeven) for prophylaxis treatment, the prophylaxis with the Factor VIII product or bypassing agent will be discontinued  (on-demand treatment  is acceptable  to continue) AND
3. If the patient is receiving Feiba (activated prothrombin complex concentrate [aPCC])  for breakthrough bleeds, BOTH of  the following:
   1. The patient will be monitored for thrombotic microangiopathy and thromboembolism AND
   2. The prescriber  has counseled the  patient  on the  maximum dosages of Feiba to  be used (i.e., no more than 100 u/kg/24 hours) AND
4. ONE of the following:
   1. The prescriber has discussed with the patient that a treatment log, documenting at least 6 months of bleeds prior to starting the requested agent, and which includes ALL of the following must be maintained and a copy will be submitted (via prescriber or pharmacy) for renewal purposes
      1. Date of the bleed AND
      2. The treatment used (include the brand name and number of units administered) AND
      3. The number of doses required to treat the bleed OR
   2. If there is no historical treatment log, the prescriber has discussed with the patient that a treatment log for bleeds which includes ALL of the following must be maintained and a copy will be submitted (via prescriber or pharmacy) for renewal purposes
      1. Date of the bleed AND
      2. The treatment used (include the brand name and number of units administered) AND
      3. The number of doses required to treat the bleed AND
5. The patient does not have any FDA labeled contraindication(s) to therapy with the requested agent AND
6. The prescriber has provided the patient's weight  AND
7. The requested  dose is within the FDA labeled dosing based on the patient's weight AND
8. ONE of the following:
   1. The patient is requesting induction therapy and maintenance therapy and the requested quantity for maintenance therapy is NOT greater than the allowed quantity  (see Hemlibra Weight-Based Approvable Quantities chart) OR
   2. The patient is requesting maintenance therapy only and the requested quantity is NOT greater than the allowed quantity (see the Hemlibra Weight-Based Approvable Quantities chart) 

Length of Approval: 6 months

Renewal Evaluation

1. The patient has been previously approved for the requested agent AND
2. The prescriber is a specialist in the area of the patient's diagnosis (e.g., prescriber working in a hemophilia treatment center [HTC], hematologist with hemophilia experience) or has consulted with a specialist in the area of the patient's diagnosis AND
3. The prescriber has provided a copy of the patient's treatment logs for bleeds that includes ALL of the  following:
   1. Date of bleed AND
   2. The treatment used (include the brand name and number of units administered) AND
   3. The number of doses required to treat the bleed AND
4. ONE of the following:
   1. The patient has shown clinical benefit since starting the requested agent (i.e., less breakthrough bleeds as documented in the treatment log) OR
   2. The prescriber has submitted documentation supporting the continued use of the requested agent AND
5. If  the  patient is receiving  Feiba (activated  prothrombin complex concentrate [aPCC])  for breakthrough bleeds, the patient will be monitored for thrombotic microangiopathy and thromboembolism AND
6. The patient does not have any FDA labeled contraindication(s) to therapy with the requested agent AND
7. The prescriber has provided the patient's weight AND
8. The requested  dose is within the FDA labeled dosing  based on the  patient's weight AND
9. The requested quantity for maintenance therapy is NOT greater than the allowed quantity  (see the Hemlibra Weight-Based Approvable Quantities chart)

Length of approval: 12 months  

These medications will be covered for the following conditions with clinical documentation of diagnosis and medical necessity:

• Factor IX deficiency (hemophilia B, Christmas disease, plasma thromboplastin component)
• Von Willebrand’s disease
• Acquired hemophilia (acquired Factor VIII autoantibodies most frequently) and other coagulation factor deficiencies, intrinsic circulating anticoagulants, antibodies or inhibitors
• Congenital deficiencies of other clotting factors (such as congenital afibrinogenemia and others)

Benefit Application
BlueCard/National Account Issues
Human antihemophilic factor is a blood derivative applicable for processing under the Blood benefit if defined to include blood derivatives; otherwise, it may be processed under Prescription Drugs when utilized and defined as drugs.

References:

1. Hemlibra prescribing information. Gentech Inc. November 2017.
2. Centers for Disease Control and Prevention. Hemophilia data & Statistics. https://www.cdc.qov /ncbddd/hemophilia/data.html. Accessed on January 2018.
3. National Hemophilia Foundation Inhibitors & other Complications. https://www.hemophilia.org/ Bleedinq-Disorders/Inhibitors-other-Complications. Accessed on January 2018
4. National Hemophilia Foundation. Steps for Living. The Basics of Bleeding Disorders/Treatment Basics. 2016.
5. Haemophilia (2013), 19, e1-e47.
6. World Federation of Hemophilia Guidelines for  the  Management of Hemophilia. 2nd edition. 2012.
7. National Hemophilia Foundation Bleeding Disorders Inhibitors & Other Complications Inhibitors for Providers. https://www.hemophilia.org/Bleedinq-Disorders/Inhibitors-other-Complications/Inhibitors-for-Providers/The-Challenges-of-Inhibitors. Accessed January 2018.
8. Carcao MD, Avila L, Leissinger C et al. An International Prophylaxis Study Group (IPSP) survey of prophylaxis in inhibitor positive children/adults with severe haemophilia. Haemophilia. 2017. Sep; 23(5) : e444-e447.
9. Leissinger CA, Konkle BA, Antunes SV. Prevention of bleeding in hemophilia patients with high-titer inhibitors. Expert Rev Hematol. 2015 Jun;8(3) :375-82.
10. Kasper CK, Hemophilia: a cautionary status report . Blood 2016 127:2949-2050.
11. National Hemophilia Foundation-McMaster University Guideline on Care Models for Hemophilia Management. Menaka P, Nigel SK, Skinner M et al. Haemophilia volime 22 Supplement 3. July 2016.
12. Institute for Clinical and Economic Review. Emicizumab for Hemophilia A:Effectivess and Value. Draft Evidence Report. January 26, 2018.
13. Medical and Scientific Advisory Council (MASAC). MASAC Safety Information Update on Emicizumab (Hemlibra). April 24, 2018.
14. American Red Cross. Practice Guidelines for Blood Transfusion:  A Compilation from Recent Peer Reviewed Literature. May 2002. 

Coding Section

Procedure and diagnosis codes on Medical Policy documents are included only as a general reference tool for each policy. They may not be all-inclusive.

This medical policy was developed through consideration of peer-reviewed medical literature generally recognized by the relevant medical community, U.S. FDA approval status, nationally accepted standards of medical practice and accepted standards of medical practice in this community, Blue Cross Blue Shield Association technology assessment program (TEC) and other non-affiliated technology evaluation centers, reference to federal regulations, other plan medical policies, and accredited national guidelines.

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