Subject: Promacta 1928-A SGM P2022

Drug

PROMACTA  (eltrombopag)

Policy:

       I.  INDICATIONS

            The indications below including FDA-approved indications and
            compendial uses are considered a covered benefit provided
            that all the approval criteria are met and the member has no
            exclusions to the prescribed therapy.

            A.  FDA-Approved Indications
                 1.  Treatment of thrombocytopenia in adult and pediatric
                      patients 1 year and older with persistent or chronic
                      immune thrombocytopenia (ITP) who have had an
                      insufficient response to corticosteroids,
                      immunoglobulins, or splenectomy
                 2.  Treatment of thrombocytopenia in patients with chronic
                      hepatitis C to allow the initiation and maintenance of
                      interferon-based therapy
                 3.  First-line treatment of severe aplastic anemia in adult
                      and pediatric patients 2 years and older in combination
                      with standard immunosuppressive therapy
                 4.  Treatment of patients with severe aplastic anemia who
                      have had an insufficient response to immunosuppressive
                      therapy
           
            B.  Compendial Uses
                 1.  MYH9-related disease with thrombocytopenia
                 2.  Myelodysplastic syndromes, for lower risk disease in
                      patients with severe or refractory thrombocytopenia
                      following disease progression or no response to
                      hypomethylating agents, immunosuppressive therapy,
                      or clinical trial.
                 3.  Myelodysplastic syndromes, in combination with equine
                      anti-thymocyte globulin with or without cyclosporine, for
                      treatment of lower risk disease in select patients
                      (generally ≤60 years old and with ≤5% marrow blasts,
                      or those with hypocellular marrows, PNH clone
                      positivity, or STAT-3 mutant cytotoxic T-cell clones) with
                      clinically relevant thrombocytopenia or neutropenia.

            All other indications are considered
            experimental/investigational and not medically necessary.

 
     II.  DOCUMENTATION

           Submission of the following information is necessary to initiate
           the prior authorization review:
           A.  Chronic or persistent immune thrombocytopenia:
                pretreatment and current platelet counts
           B.  Aplastic anemia continuation of therapy: current platelet
                counts

 
   III.   EXCLUSIONS

            Coverage will not be provided for members with the following
            exclusion: concomitant use of Promacta with other
            thrombopoietin receptor agonists (e.g., Nplate, Doptelet,
            Mulpleta) or with spleen tyrosine kinase inhibitors (e.g.,
            Tavalisse)

     IV.  PRESCRIBER SPECIALTIES

 
           A.  For diagnosis of persistent or chronic thrombocytopenia,
                severe aplastic anemia, MYH9-related disease with
                thrombocytopenia and myelodysplastic syndromes, this
                medication must be prescribed by or in consultation with a
                hematologist or oncologist.
           B.  For diagnosis of hepatitis C, this medication must be
                prescribed by or in consultation with a hepatologist,
                gastroenterologist, or infectious disease specialist.

 
      V.  CRITERIA FOR INITIAL APPROVAL

            A.  Chronic or persistent immune thrombocytopenia
                 (ITP)
                 Authorization of 6 months may be granted for treatment of
                 chronic or persistent ITP when both of the following criteria
                 are met:
                 1.  Inadequate response or intolerance to prior therapy with
                      corticosteroids, immunoglobulins, or splenectomy
                 2.  Untransfused platelet count at any point prior to the
                      initiation of the requested medication is less than
                      30x10⁹/L OR 30x10⁹/L to 50x10⁹/L with symptomatic
                      bleeding (e.g., significant mucous membrane bleeding,
                      gastrointestinal bleeding or trauma) or risk factors for
                      bleeding (see Section VII).

            B.  Thrombocytopenia associated with chronic hepatitis C
                 Authorization of 6 months may be granted to members who
                 are prescribed Promacta for the initiation and maintenance
                 of interferon-based therapy for the treatment of
                 thrombocytopenia associated with chronic hepatitis C.

            C.  Aplastic anemia
                 1.  Authorization of 6 months may be granted for first-line
                      treatment of severe aplastic anemia when Promacta will
                      be used in combination with standard
                      immunosuppressive therapy (e.g., horse antithymocyte
                      globulin (h-ATG) and cyclosporine).
                 2.  Authorization of 6 months may be granted for treatment
                      of aplastic anemia which had an insufficient response to
                      immunosuppressive therapy.

            D.  MYH9-related disease with thrombocytopenia
                 Authorization of 12 months may be granted to members
                 with thrombocytopenia associated with MYH9-related
                 disease.

            E.  Myelodysplastic Syndromes
                 1.  Authorization of 12 months may be granted for
                      treatment of myelodysplastic syndromes with severe or
                      refractory thrombocytopenia when both of the following
                      criteria are met:
                      i.      Member has lower risk disease defined as Revised
                              International Prognostic Scoring System (IPSS-R)
                              (Very Low, Low, Intermediate), International
                              Prognostic Scoring System (IPSS)
                              (Low/Intermediate-1), WHO classification-based
                              Prognostic Scoring System (WPSS) (Very Low,
                              Low, Intermediate).
                      ii.     Member has severe or refractory
                              thrombocytopenia following disease progression or
                              no response to hypomethylating agents (such as
                              azacitidine and decitabine), immunosuppressive
                              therapy, or clinical trial.
                 2.  Authorization of 12 months may be granted for
                      treatment of myelodysplastic syndromes when all of the
                      following criteria are met:
                      i.      Member has lower risk disease defined as Revised
                              International Prognostic Scoring System (IPSS-R)
                              (Very Low, Low, Intermediate), International
                              Prognostic Scoring System (IPSS)
                              (Low/Intermediate-1), WHO classification-based
                              Prognostic Scoring System (WPSS) (Very Low,
                              Low, Intermediate).
                      ii.     Member has clinically relevant thrombocytopenia
                              or neutropenia.
                      iii.    Promacta will be used in combination with equine
                              anti-thymocyte globulin.

 
     IV.  CONTINUATION OF THERAPY

 
            A.  Chronic or persistent ITP
                 1.  Authorization of 3 months may be granted to members
                      with current platelet count less than 50x10⁹/L for whom
                      the platelet count is not sufficient to prevent clinically
                      important bleeding and who have not received a
                      maximal Promacta dose for at least 4 weeks.
                 2.  Authorization of 12 months may be granted to members
                      with current platelet count less than 50x10⁹/L for whom
                      the current platelet count is sufficient to prevent
                      clinically important bleeding.
                 3.  Authorization of 12 months may be granted to members
                      with current platelet count of 50x10⁹/L to 200x10⁹/L.
                 4.  Authorization of 12 months may be granted to members
                      with current platelet count greater than 200x10⁹/L to
                      less than or equal to 400x10⁹/L for whom Promacta
                      dosing will be adjusted to achieve a platelet count
                      sufficient to avoid clinically important bleeding.

             B.  Thrombocytopenia associated with chronic
                   hepatitis C
                  Authorization of 6 months may be granted to members
                  who are continuing to receive interferon-based therapy.

             C.  Aplastic anemia
                  1.  Authorization of up to 16 weeks total may be granted
                       to members with current platelet count less than
                       50x10⁹/L who have not received appropriately titrated
                       therapy with Promacta for at least 16 weeks.
                  2.  Authorization of up to 16 weeks total may be granted
                       to members with current platelet count less than
                       50x10⁹/L who are transfusion-independent.
                  3.  Authorization of 12 months may be granted to
                       members with current platelet count of 50x10⁹/L to
                       200x10⁹/L.
                  4.  Authorization of 12 months may be granted to
                       members with current platelet count greater than 200
                       x10⁹/L to less than or equal to 400x10⁹/L for whom
                       Promacta dosing will be adjusted to achieve and
                       maintain an appropriate target platelet count.

            D.  MYH9-related disease with thrombocytopenia
                 All members (including new members) requesting
                 authorization for continuation of therapy must meet all
                 initial authorization criteria.

            E.  Myelodysplastic Syndromes
                 Authorization of 12 months may be granted for continued
                 treatment of myelodysplastic syndromes in members who
                 experience benefit from therapy (e.g., increased platelet
                 counts, decreased bleeding events, reduced need for
                 platelet transfusions).

 
    VII.  APPENDIX

            Examples of risk factors for bleeding (not all inclusive)
            •    Undergoing a medical or dental procedure where blood loss
                  is anticipated
            •    Comorbidity (e.g., peptic ulcer disease, hypertension)
            •    Mandated anticoagulation therapy
            •    Profession (e.g., construction worker) or lifestyle (e.g.,
                  plays contact sports) that predisposes member to trauma

 

 

Place of Service:

Outpatient

The above policy is based on the following references:

1. Promacta [package insert]. Research Triangle Park, NC: GlaxoSmithKline; October 2021.
2. Pecci A, Gresele P, Klersy C, et al. Eltrombopag for the treatment of the inherited thrombocytopenia deriving from MYH9 mutations. 2010;116(26):5832-7.
3. The NCCN Drugs & Biologics Compendium^® © 2020 National Comprehensive Cancer Network, Inc. https://www.nccn.org. Accessed June 15, 2022.
4. The NCCN Clinical Practice Guidelines in Oncology® Myelodysplastic Syndrome (Version 3.2022). © 2020 National Comprehensive Cancer Network, Inc. https://www.nccn.org. Accessed June 15, 2022.
5. Nuenert C, Terrel DR, Arnold DM, et al. American Society of Hematology 2019 guidelines for immune thrombocytopenia. Blood Adv2019;3(23):3829–3866. 
6. Provan D, Arnold DM, Bussel JB, et al. Updated international consensus report on the investigation and management of primary immune thrombocytopenia. Blood Adv2019;3(22): 3780–3817. 
7. Provan D, Stasi R, Newland AC, et al. International consensus report on the investigation and management of primary immune thrombocytopenia. 2010;115(2):168-186.
8. Rodeghiero F, Stasi R, Gernsheimer T, et al. Standardization of terminology, definitions and outcome criteria in immune thrombocytopenic purpura of adults and children: report from an international working group. Blood. 2009;113(11):2386-2393.
9. Olnes MJ, Scheinberg P, Calvo KR, et al. Eltrombopag and improved hematopoiesis in refractory aplastic anemia. N Engl J Med. 2012;367(1):11-19.
10. Townsley DM, Scheinberg P, Winkler T, et al. Eltrombopag added to standard immunosuppression for aplastic anemia. N Engl J Med 2017;376:1540-1550.

Copyright Aetna Inc. All rights reserved. Pharmacy Clinical Policy Bulletins are developed by Aetna to assist in administering plan benefits and constitute neither offers of coverage nor medical advice. This Clinical Policy Bulletin contains only a partial, general description of plan or program benefits and does not constitute a contract. Aetna does not provide health care services and, therefore, cannot guarantee any results or outcomes. Participating providers are independent contractors in private practice and are neither employees nor agents of Aetna or its affiliates. Treating providers are solely responsible for medical advice and treatment of members. This Clinical Policy Bulletin may be updated and therefore is subject to change.

February 12, 2023