Hemophilia

Adynovate^®, Afstyla^®, Alprolix^®, Altuviiio™, Eloctate^®, Esperoct^®, Idelvion^®, Jivi^®, and Rebinyn^® Approval Criteria:

• An FDA approved indication; AND
• Requested medication must be prescribed by a hematologist specializing in hemophilia, or a mid-level practitioner with a supervising physician that is a hematologist specializing in hemophilia; AND
• A patient-specific, clinically significant reason why the member cannot use the following:
  • Hemophilia A: Advate® or current factor VIII replacement product; OR
  • Hemophilia B: Benefix® or current factor IX replacement product; AND
• A half-life study must be performed to determine the appropriate dose and dosing interval.
• Initial approval will be for the duration of the half-life study. If the half-life study shows significant benefit in prolonged half-life, subsequent approvals will be for the duration of one year. 

Alhemo^® (Concizumab-mtci) Approval Criteria:

• An FDA approved diagnosis of hemophilia A or B with inhibitors; and
• Member must be 12 years of age or older; and
• Member’s recent weight (taken within the past 3 months) must be provided and must be ≥25kg; and
• Member must not be undergoing immune tolerance induction (ITI); and
• Member must not have a history of or be at high risk for thromboembolic events; and
• Female members of reproductive potential must meet the following:
  • Must not be pregnant; or
    • If member is pregnant or becomes pregnant during treatment, the risk to the fetus must be weighed against the benefit to the mother; and
  • Must agree to use effective birth control during treatment and for at least 7 weeks after the last dose; and
• Prescriber must agree the member will not be continuing on other prophylactic therapies; and
• Must be prescribed by a hematologist practicing in a federally recognized Hemophilia Treatment Center (HTC) or mid-level practitioner under the supervision of a physician at an HTC; and
• Prescriber must verify that the member or caregiver has been trained on the subcutaneous administration and counseled on the storage of Alhemo^®; and
• Prescriber must verify that the member has been counseled on the potential risk of thrombosis and use of bypassing agents at the lowest possible dose for breakthrough bleeding episodes based on severity and location of bleed; and
• Requests must be for an FDA approved dosing regimen as outlined in the package labeling; and
• Initial approvals will be for 3 months for the loading dose of 1mg/kg on day 1 and 0.2mg/kg daily until individualization of the maintenance dose has been achieved. Subsequent approvals will be the duration of 1 year if there is documentation of clinical effectiveness. 

Alhemo® PA Form

Approval Criteria:

• An FDA approved indication; AND
• Coagadex® must be prescribed by a hematologist specializing in rare bleeding disorders, or a mid-level practitioner with a supervising physician that is a hematologist specializing in rare bleeding disorders; AND
• A half-life study must be performed to determine the appropriate dose and dosing interval.
• Initial approval will be for the duration of the half-life study and immediate needs. After a half-life study is performed and appropriate dose and interval is determined, subsequent approvals will be for the duration of one year.  

Approval Criteria:

• An FDA approved indication; AND
• Corifact® or Tretten® must be prescribed by a hematologist specializing in rare bleeding disorders, or a mid-level practitioner with a supervising physician that is a hematologist specializing in rare bleeding disorders; AND
• A half-life study must be performed to determine the appropriate dose and dosing interval.
• Initial approval will be for the duration of the half-life study and immediate needs. After a half-life study is performed and appropriate dose and interval is determined, subsequent approvals will be for the duration of one year. 

Feiba® (Anti-Inhibitor Coagulation Complex) Approval Criteria:

• Member must be diagnosed with hemophilia A or B with an inhibitor; AND
  • For a diagnosis of hemophilia A with an inhibitor, a patient-specific, clinically significant reason why the member cannot use Alhemo^® (concizumab-mtci), Hemlibra^®, or Qfitlia™ (fitusiran) for prophylaxis therapy must be provided; or
  • For a diagnosis of hemophilia B with an inhibitor, a patient-specific, clinically significant reason why the member cannot use Alhemo^® (concizumab-mtci) or Qfitlia™ (fitusiran) for prophylaxis therapy must be provided; and
• Feiba® must be prescribed by a hematologist specializing in rare bleeding disorders practicing in a federally recognized Hemophilia Treatment Center (HTC) or a mid-level practitioner under the supervision of a physician at an HTC. 

Hemgenix^® (Etranacogene Dezaparvovec-drlb) Approval Criteria:

• Diagnosis of severe or moderately severe congenital, X-linked, hemophilia B; and
• Member must not have a history of an inhibitor or a recent positive screening, defined as ≥0.6 Bethesda units, prior to administration of etranacogene dezaparvovec-drlb; and
• Member must not have an AAV5 neutralizing antibody titer >700; and
• Member must be a male 18 years of age or older; and
• Member must be on prophylactic therapy with continued frequent breakthrough bleeding episodes or has experienced a life-threatening bleeding episode; and
• Member must have had >150 previous exposure days of treatment with factor IX; and
• Member must not have active hepatitis B or C; and
• Members with human immunodeficiency virus (HIV) must be controlled with antiviral therapy; and
• Member must not have received prior treatment with any gene therapy for hemophilia B; and
• Prescriber must perform baseline liver health assessment including:
  • Enzyme testing (ALT, AST, ALP); and
  • Hepatic ultrasound; and
• Member’s recent weight must be provided (taken within the last month) to ensure appropriate dosing; and
• Must be prescribed by a hematologist practicing in a federally recognized Hemophilia Treatment Center (HTC) or mid-level practitioner under the supervision of a physician at an HTC; and
• Must be administered in a clinical setting and monitoring performed for at least 3 hours post-infusion; and
• Prescriber must monitor liver enzymes weekly for 3 months following administration of etranacogene dezaparvovec-drlb and continue monitoring until liver enzymes return to baseline; and
  • Prescriber must agree to begin corticosteroids if indicated; and
• Approvals will be for 1 dose per member per lifetime.

Hemlibra® (Emicizumab-kxwh) Approval Criteria:

• Member must have a diagnosis of hemophilia A; and
• Hemlibra^® must be prescribed by a hematologist specializing in rare bleeding disorders practicing in a federally recognized Hemophilia Treatment Center (HTC) or a mid-level practitioner under the supervision of a physician at an HTC.; and
• Prescriber must be able to monitor appropriate blood clotting tests and levels utilizing testing which accounts for the interaction of Hemlibra^® and blood factors by following the Medical and Scientific Advisory Council (MASAC) guidance; and
• For members with hemophilia A with an inhibitor to factor VIII:
  • A treatment plan must be developed to address breakthrough bleeds and procedures. Prescriber must counsel member and/or caregiver on the risks of utilizing Feiba^® for breakthrough bleeding while on Hemlibra^®, and member should be monitored closely if any bypassing agent is given; or
• For members without an inhibitor and having severe hemophilia A or those with moderate hemophilia A presenting as severe:
  • Treatment plan must be made to address breakthrough bleeds and procedures; and
  • Routine lab screenings must occur for factor VIII inhibitor while using Hemlibra^® since this would change the treatment plan for bleeds and procedures; and
• Prescriber must agree the member will not be continuing other prophylactic therapies; and
• First dose must be given in a health care facility; and
• In order to calculate appropriate dosing, the member’s recent weight must be provided and been taken within the last 3 months; and
• Initial approvals will be for 3 months of therapy. Subsequent approvals will be for the duration of 1 year, if there has been a decrease in the member’s spontaneous bleeding episodes since initiating Hemlibra^® treatment.

Hympavzi™ (Marstacimab-hncq) Approval Criteria:

• A diagnosis of moderately severe to severe hemophilia A (FVIII <2%) without inhibitors or moderately severe to severe hemophilia B (FIX activity <2%) without inhibitors; and
• Member must be 12 years of age or older and weigh at least 35kg; and
• Member must not have a current inhibitor or documented history of an inhibitor; and
• For females of reproductive potential:
  • Member must not be pregnant and must have a negative pregnancy test prior to therapy initiation; and
  • Member must be willing to use effective contraception during and after treatment for at least 2 months after the last dose; and
• Member must not have uncontrolled human immunodeficiency virus (HIV) as shown by CD4+ counts ≤200cells/mm³; and
• Prescriber must agree the member will not be continuing other prophylactic therapies; and
• Must be prescribed by a hematologist practicing in a federally recognized Hemophilia Treatment Center (HTC) or mid-level practitioner under the supervision of a physician at an HTC; and
• Prescriber must verify that the member or caregiver has been trained on the subcutaneous administration and counseled on the storage of Hympavzi™; and
• Prescriber must verify that the member has been counseled on the use of factor replacement therapy at the lowest possible dose for breakthrough bleeding episodes; and
• Initial approvals will be for 3 months of therapy. Subsequent approvals will be the duration of 1 year if there is documentation of clinical effectiveness; and
• Approvals will be for 300mg loading dose followed by 150mg weekly doses. Approvals may be granted for dose escalation to 300mg weekly when the following are met:
  • Member weighs ≥50kg; and
  • There have been ≥2 spontaneous bleeding episodes which were treated with factor replacement therapy in the last 6 months despite compliance; and
  • Absence of inhibitor development.

NovoSeven® RT [Coagulation Factor VIIa (Recombinant)] Approval Criteria:

• An FDA approved diagnosis of one of the following:
  
  • Hemophilia A or B with inhibitors; or
    • For a diagnosis of hemophilia A with an inhibitor, a patient-specific, clinically significant reason why the member cannot use Alhemo^® (concizumab-mtci), Hemlibra^® (emicizumab-kxwh), or Qfitlia™ (fitusiran) for prophylaxis therapy must be provided; or
    • For a diagnosis of hemophilia B with an inhibitor, a patient-specific, clinically significant reason why the member cannot use Alhemo^® (concizumab-mtci) or Qfitlia™ (fitusiran) for prophylaxis therapy must be provided; or
  • Congenital factor VII deficiency; or
  • Glanzmann’s thrombasthenia with refractoriness to platelet transfusions, with or without antibodies to platelets; or
  • Acquired hemophilia; and
   
• NovoSeven^® RT must be prescribed by a hematologist specializing in rare bleeding disorders practicing in a federally recognized Hemophilia Treatment Center (HTC) or a mid-level practitioner under the supervision of a physician at an HTC.

Approval Criteria:

• An FDA approved indication; AND
• Obizur® must be prescribed by a hematologist specializing in hemophilia, or a mid-level practitioner with a supervising physician that is a hematologist specializing in hemophilia; AND
• A patient-specific, clinically significant reason why the member cannot use Feiba® (anti-inhibitor coagulant complex) or NovoSeven® RT [coagulation factor VIIa (recombinant)]; AND
• A half-life study must be performed to determine the appropriate dose and dosing interval. 
• Initial approval will be for the duration of the half-life study. After a half-life study is performed and appropriate dose and interval is determined, subsequent approvals will be for the duration of one year.

Qfitlia™ (Fitusiran) Approval Criteria:

• A diagnosis of severe hemophilia A or B, with or without factor inhibitors; and
• Member must be 12 years of age or older; and
• Member must not have a history of or be at high risk for thromboembolic events; and
• Member must not have clinically significant liver disease; and
• Member must not have active hepatitis C; and
• Member must not have an acute or chronic hepatitis B infection; and
• Members with human immunodeficiency virus (HIV) must not be uncontrolled with antiviral therapy as shown by CD4+ counts ≤200cells/mm³ or viral load ≥20 copies/mL; and
• In a member with a history of symptomatic gallbladder disease, a reason why the member cannot use other available treatments must be provided; and
• Must be prescribed by a hematologist practicing in a federally recognized Hemophilia Treatment Center (HTC) or mid-level practitioner under the supervision of a physician at an HTC; and
• Prescriber must agree the member will not be continuing other prophylactic therapies for longer than 7 days after initiation of fitusiran; and
• Prescriber must agree to perform an FDA-cleared test for antithrombin activity at weeks 4, 12, 20, and 24 and adjust the dosing as outlined in the package labeling; and
• Prescriber must agree to perform baseline liver tests prior to initiation of fitusiran and monthly for at least 6 months and after any dose increase; and
• Prescriber must verify that the member or caregiver has been trained on the subcutaneous administration and counseled on the storage of fitusiran; and
• Prescriber must verify that the member has been counseled on the use of factor replacement therapy or bypassing agent as outlined in the prescribing information for breakthrough bleeding episodes; and
• Initial approvals will be for 3 months of therapy. Subsequent approvals will be the duration of 1 year if there is documentation of clinical effectiveness.

Roctavian™ (Valoctocogene Roxaparvovec-rvox) Approval Criteria:

• An FDA approved diagnosis of severe congenital (or X-linked) hemophilia A; and
• Member must be a male 18 years of age or older; and
• Member must not have a history of or a recent positive screening of an inhibitor defined as ≥0.6 Bethesda units; and
• Member must be on prophylactic therapy with continued frequent breakthrough bleeding episodes or has experienced a life-threatening bleeding episode; and
• Member must not have acute infections; and
• Member must not have chronic active infections such as hepatitis B or C; and
• Member must not have uncontrolled human immunodeficiency virus (HIV) as shown by CD4+ counts ≤200u/L; and
• Member must not be taking efavirenz; and
• Member must not have antibodies to AAV5; and
• Member must not have any of the following:
  • Significant liver fibrosis:
    • Defined as ≥3 as rated on a scale of 0-4 on the METAVIR scoring system or equivalent grade on an alternative scale; and
    • Measured by ultrasound and elastography or laboratory assessments; or
  • Liver cirrhosis; or
  • Significant liver dysfunction with any of the following abnormal lab results:
    • Alanine aminotransferase (ALT) >1.25x upper limit of normal (ULN); or
    • Aspartate aminotransferase (AST) >1.25x ULN; or
    • Gamma-glutamyl transferase (GGT) >1.25x ULN; or
    • Total bilirubin >1.25x ULN; or
    • Alkaline phosphatase >1.25x ULN; or
    • International normalized ratio (INR) ≥1.4; and
• Must be prescribed by a hematologist practicing in a federally recognized Hemophilia Treatment Center (HTC) or mid-level practitioner under the supervision of a physician at an HTC; and
• Prescriber must counsel member to not donate semen, and if member is of reproductive potential then their female partners must agree to prevent or postpone pregnancy for 6 months after treatment with valoctocogene roxaparvovec-rvox; and
• Valoctocogene roxaparvovec-rvox must be administered in an appropriate clinical setting and member must be monitored for at least 3 hours post infusion; and
• Prescriber must follow liver enzymes weekly for 26 weeks, every 1 to 2 weeks for weeks 26 through 52, every 3 months in the second year, and every 6 months thereafter; and
• Prescriber agrees to start corticosteroids (or other immunosuppressives if corticosteroids are contraindicated) as outlined in the package labeling; and
• Prescriber agrees to monitor factor VIII levels weekly for 26 weeks, every 1 to 2 weeks for weeks 26 through 52, every 3 months in the second year, and every 6 months thereafter; and
• Approvals will be for 1 treatment per member per lifetime.

Sevenfact^® [Coagulation Factor VIIa (Recombinant)-jncw] Approval Criteria:

• An FDA approved diagnosis; and
  • For a diagnosis of hemophilia A with an inhibitor, a patient-specific, clinically significant reason why the member cannot use Alhemo^® (concizumab-mtci), Hemlibra^® (emicizumab-kxwh), or Qfitlia™ (fitusiran) for prophylaxis therapy must be provided; or
  • For a diagnosis of hemophilia B with an inhibitor, a patient-specific, clinically significant reason why the member cannot use Alhemo^® (concizumab-mtci) or Qfitlia™ (Fitusiran) for prophylaxis therapy must be provided; and
• Sevenfact^® must be prescribed by a hematologist specializing in rare bleeding disorders practicing in a federally recognized Hemophilia Treatment Center (HTC) or a mid-level practitioner under the supervision of a physician at an HTC.

Standards of Care for pharmacies providing factor replacement products:  

• The Provider/Pharmacy shall be licensed as a pharmacy by the Oklahoma State Board of Pharmacy. The Pharmacist-in-Charge must be licensed as a pharmacist in Oklahoma. 
• The Provider/Pharmacy agrees that it will provide the following services:
  • The Provider/Pharmacy shall be capable of providing a full range of factor products including all available vial sizes. 
  • The Provider/Pharmacy shall provide support services to patients on a “24/7” basis in order to assure availability of appropriate support in the event of an after-hours emergency. 
  • The Provider/Pharmacy staff shall deliver factor within 24 hours (with a delivery goal of four hours) of notification of a need due to a current bleeding episode.  If the patient is not having an emergency/current bleeding episode, the Provider/Pharmacy shall deliver factor within three days of notification of need. 
  • The Provider/Pharmacy shall provide all necessary supplies for the appropriate preparation and administration of the factor product as well as appropriate sharps and bio-hazardous disposal unit (to include retrieval and destruction of the disposal unit). If the items are SoonerCare compensable, such items must be billed as durable medical equipment (DME) via a DME contract. 
  • The Provider/Pharmacy must provide access to multilingual interpreters for those patients and families for whom English is not their primary language. Interpreters must be available on a “24/7” basis, in order to assure availability in the event of an after-hours emergency. 
  • Case Management:
    • Case Management can be performed by a pharmacist, nurse, social worker, or case manager. 
    • An in-home patient assessment must be performed upon initiation of services and at least yearly thereafter.
      • An assessment must include, at a minimum:
        • Verification of appropriate and adequate storage; AND 
        • A current inventory of factor product and supplies; AND 
        • Verification of access to a bio-hazardous waste disposal unit; AND 
        • A review of current infusion/treatment records/logs; AND
           A assessment of educational opportunities to be performed by appropriately trained staff (please refer to 3 b ii below); AND 
        • Identification of any adverse events. 
      • In the event a patient or caregiver refuses entry to the home, the pharmacy must re-attempt the in-home assessment within three months. If the patient or caregiver continues to deny access, the pharmacy must discuss this issue with the prescribing provider and develop an action plan to verify items set forth in subparagraph 2(f)(ii)(1) above.  Documentation must be kept of any refusal, re-attempt, and action plan.
      • The in-home assessment must be completed annually and must be documented and signed by patient or caregiver and pharmacy personnel acknowledging the availability of patient and/or caregiver training and the patient/caregiver’s understanding of the items set forth in subparagraph 2(f)(ii)(1) above, together with any additional information discussed. 
    • Regular follow up with the patient via telephone, video call, or in-person.  This contact should be at least quarterly and must address, at a minimum:
      • All recent bleeding episodes reported should be forwarded to the prescribing practitioner immediately.
      • Current inventory:
        • Number of factor doses on hand; AND 
      • Expiration dates of vials on hand.
        Confirmation of factor storage.
      • Adverse events:
        • If adverse events are reported to a non-clinical case manager, a clinician should become involved immediately. 
    • Coordination of care including nursing, DME, treating practitioner, and all medications, regardless of source. 
  • Educational requirements:
    • Staff Education: 
      • Staff having contact with the patient via telephone, video calling, or in-person, must be appropriately trained and knowledgeable about hemophilia and other bleeding disorders. 
      • Two hours of Continuing Education (CE) on hemophilia or other related bleeding disorders must be completed annually. Licensed staff must use accredited CE based on their license type. Non-licensed staff may use non-accredited CE provided by a licensed professional.  
        • Staff members, whether employed or contracted by the pharmacy, who are required to complete CE include but are not limited to the following:
          • Pharmacist in Charge; AND 
          • Nurse manager; AND 
          • Nurse performing direct patient care; AND 
          • Social worker; AND 
          • Case Manager (including customer service representatives) 
        • Documentation of educational activity completed must be maintained by the pharmacy and must include the CE certificate or date of activity, staff in attendance, and name and license of professional providing activity. 
      • Member and Caregiver Education:
        • Pharmacy staff shall encourage engagement with a comprehensive hemophilia treatment center. Studies have shown better clinical outcomes for those patients engaged with a comprehensive hemophilia treatment center.
        • Pharmacy staff must discuss educational needs of the patient with the treating practitioner. Once educational opportunities are identified, the pharmacy staff must provide training for the patients and family members in accordance with the treating physician’s or mid-level practitioner’s recommendations. All patient efforts must be documented. Areas of education may include but are not limited to the following:
          • Proper storage for factor products and ancillary supplies; and
          • Proper disposal of bio-hazardous waste; AND 
          • Preparation of factor and supplies; AND 
          • Training on self-infusion; AND 
            • Prescriber to provide order 
              • Professional licensed nurse (LPN or RN) to train patients or caregivers for peripheral venous access.  
              • Licensed RN to train patients or caregivers on central line care (e.g.  PICC line, InfusaPort, etc.) which includes but is not limited to access, flushing, infusions, and dressing changes.   
            • Training must be in accordance with the MASAC guidelines. 
          • Infusion/treatment record keeping; AND 
          • Factor and supply management. 
    • Factor Product Dispensing and Delivery:
      • Prescriptions cannot be filled without an expressed need from the patient, caregiver or prescribing practitioner. Auto-filling is not allowed. If a prescription is written for prophylaxis with additional doses for breakthrough bleeding, then the monthly prophylaxis dispensing should not include further additional doses absent documented use of doses for breakthrough bleeding.
      • Factor products must be packaged in such a way that a patient or caregiver can easily determine what is to be used for each dose:
        • If the factor dose to be infused only consists of one vial/box, the vial/box should be labeled as such; AND 
        • If the factor dose to be infused consists of two or more vials/boxes then each dose should be packaged as a group of appropriate vials/boxes and labeled as an individual dose. 
      • Factor dose dispensed must be within 5% of the prescribed dose.
        • If unable to provide factor dosing within 5% of prescribed dose, then pharmacy must provide proof of all available vial sizes from the manufacturer at the time dispensing occurred.
        • Any dose requiring more than 3 vials/boxes to be used must be approved by the prescribing practitioner and documented.
        •  Pharmacy staff must, by the 10th of every month, fax or email to the Oklahoma Health Care Authority a record of dispensing for the previous month, to include but not limited to the member’s name, SoonerCare ID, date dispensed, prescriber name, product, prescribed dose, units per vial dispensed, quantity of each vial size, how the doses were packaged if more than one vial was to be used per dose, type of treatment (prophylaxis, episodic, or breakthrough), and delivery confirmation with member or caregivers’ signature. 
      • Any factor product which is short-dated (expiring within 6 months) may only be dispensed after approval from the prescribing practitioner and must be documented.
      • The pharmacy staff must assure appropriate storage of the factor products and supplies including cold chain supply shipping and delivery. The pharmacy must be able to trace the supply chain from manufacturer to patient delivery.
      • The pharmacy must keep records of all lots of factor products dispensed to each patient and notify patient and treating practitioner of any recalls of dispensed factor products. The pharmacy must participate in the National Patient Notification System for clotting factor recalls.
      • The pharmacy provider must have a plan in place for delivery of factor products to the patient in the event of a natural disaster. 
    • The Provider/Pharmacy must originally attest to the Oklahoma Health Care Authority these standards of care will be followed and must re-attest yearly.
    • Oklahoma Health Care Authority (OHCA) Auditing:
      • The OHCA has the right to audit records of the blood clotting factor providers to assure all requirements are being met. The OHCA will audit these records which include but is not limited to the following:
        • In-home assessment records; AND 
        • Educational information and training provided; AND 
        • Adverse Event records including reports to other state and federal agencies; AND 
        • Sharps and bio-hazardous waste disposal units, delivery proof, and education on proper disposal in patient record; AND 
        • Patient records, including:
          • Original Prescriptions; AND 
          • Dispensing records (including lot numbers and expiration dates). 
• The pharmacy will be excluded from providing blood factor products if OHCA finds that the pharmacy is out of compliance with the requirements as outlined.