Subject: Promacta 1928-A SGM P2021

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 

A.   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

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.  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 VI).

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 when both of
     the following criteria are met:
     1.   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).
     2.   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.

V.    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).

Special Notes:

VI.   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; February 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, 2021.
4. The NCCN Clinical Practice Guidelines in Oncology® Myelodysplastic Syndrome (Version 2.2020). © 2020 National Comprehensive Cancer Network, Inc. https://www.nccn.org. Accessed June 15, 2021.
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 Med2017;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 23, 2021