Subject: Promacta 1928-A SGM P2023

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 (MDS)

          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.  Persistent or chronic immune thrombocytopenia:
               1.  For initial requests: pretreatment platelet count
               2.  For continuation requests: current platelet count
          B.  Aplastic anemia continuation of therapy: current platelet
               count


  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 thrombocytopenia with 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.  Persistent or chronic immune thrombocytopenia (ITP)
               Authorization of 6 months may be granted for treatment of
               persistent or chronic 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
               Authorization of 12 months may be granted for treatment of
               myelodysplastic syndromes (MDS).


   VI.  CONTINUATION OF THERAPY

 
          A.  Persistent or chronic 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).


VIII.  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; March 2023.
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^® © 2023 National Comprehensive Cancer Network, Inc. https://www.nccn.org. Accessed June 2, 2023.
4. The NCCN Clinical Practice Guidelines in Oncology® Myelodysplastic Syndrome (Version 1.2023). © 2023 National Comprehensive Cancer Network, Inc. https://www.nccn.org. Accessed June 2, 2023.
5. Nuenert C, Terrel DR, Arnold DM, et al. American Society of Hematology 2019 guidelines for immune thrombocytopenia. Blood Adv.2019;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 Adv.2019;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.

December 09, 2023