Promacta (eltrombopag)

POLICY NUMBER

L.5.01.463

Promacta (eltrombopag)

Please perform a formulary drug search on your patient’s member ID to ensure the prescription drug is covered under their benefit plan. The medication(s) in this medical policy may not be covered under a specific member’s benefit plan.

DESCRIPTION

Promacta (eltrombopag) is an orally bioavailable, small molecule thrombopoietin (TPO) receptor agonist for oral administration. Eltombopag interacts with the transmembrane domain of the TPO-receptor (also known as cMpl) leading to increased platelet production.

Promacta (eltrombopag) is indicated for the treatment of thrombocytopenia in patients 1 year and older with persistent to chronic immune thrombocytopenia (ITP) who have had an insufficient response to corticosteroids, immunoglobulins or splenectomy, for the treatment of thrombocytopenia in patients with chronic hepatitis C to allow the initiation and maintenance therapy of interferon-based therapy, for the treatment of patients with severe aplastic anemia who have had an insufficient response to immunosuppressive therapy, and in combination with standard immunosuppressive therapy for the first-line treatment of patients 2 years of age and older with severe aplastic anemia.

Related medical policies:

• Immune Thrombocytopenia

POLICY

Prior authorization is required.

The use of samples by an individual will not be considered current or stable therapy to satisfy of Medical Policy requirements.

Initial Criteria

Promacta (eltrombopag) may be considered medically necessary when ALL of the following criteria are met:

1. ONE of the following:

   1. The individual has a diagnosis of persistent or chronic immune thrombocytopenia (ITP) for ≥3 months and ALL of the following:

      1. The individual is at least 1 year of age;

      2. ONE of the following:

         1. Platelet count <30 x10⁹/L); OR

         2. Platelet count <50 x 10⁹/L with significant bleeding symptoms (e.g., mucosal bleeding) or documented high risk for bleeding (e.g., peptic ulcer disease, liver disease, >60 years of age, concurrent anticoagulation/anti-platelet therapy);

      3. ONE of the following:

         1. The individual has had an inadequate response (defined as platelet count <30 x10⁹/L or platelet count >30 x10⁹/L but with bleeding symptoms) to ALL of the following therapies, with or without a splenectomy:

            1. Corticosteroids (after a 4-6 week duration of therapy);

            2. An adequate trial of IVIG; AND

            3. An adequate trial of rituximab; OR;

         2. The individual has a documented intolerance, FDA-labeled contraindication, or hypersensitivity to corticosteroid, IVIG, and rituximab therapies; AND

      4. The prescriber is or has consulted with a specialist in an area of the individual's diagnosis (i.e., a hematologist);

   2. The individual has a diagnosis of thrombocytopenia associated with chronic hepatitis C and ALL of the following:

      1. The individual is at least 18 years of age;

      2. ONE of the following:

         1. The individual’s baseline (prior to therapy with the requested agent) platelet count is <75 x10⁹/L AND the intent is to increase platelet counts sufficiently to initiate interferon therapy; OR

         2. The individual is on concurrent therapy with a pegylated interferon and ribavirin AND is at risk for discontinuing HCV therapy due to thrombocytopenia; AND

      3. The prescriber is or has consulted with a specialist in an area of the individual's diagnosis (i.e., a gastroenterologist, a hepatologist, or infectious disease specialist); OR

   3. The individual has a diagnosis of severe aplastic anemia and ALL of the following:

      1. The individual is at least 2 years of age;

      2. BOTH of the following:

         1. The individual has at least TWO of the following baseline (prior to therapy with the requested agent) blood criteria:

            1. Neutrophil counts <0.5 X 10⁹/L;

            2. Platelet counts < 20 X 10⁹/L; AND

            3. Reticulocyte count <60 X 10⁹/L; AND

         2. The individual has at least ONE of the following marrow criteria:

            1. Severe hypocellularity (i.e. <25%); OR

            2. Moderate hypocellularity (i.e., 25-50%) with hematopoietic cells representing less than 30% of residual cells;

      3. ONE of the following:

         1. The requested agent will be used as a first-line treatment in combination with standard immunosuppressive therapy (i.e., antithymocyte globulin [ATG]) AND cyclosporine); OR

         2. The individual has had an inadequate response, intolerance, or FDA-labeled contraindication to both ATG and cyclosporine; AND

      4. The prescriber is or has consulted with a specialist in an area of the individual's diagnosis (i.e., hematologist);

2. The requested agent will not be used in combination with another thrombopoietin receptor agonist (e.g., Nplate) or spleen tyrosine kinase inhibitor (e.g., Tavalisse);

3. The individual does not have any FDA-labeled contraindication(s) to therapy with the requested agent; AND

4. The prescribed dosage is within the program quantity limits based on FDA approved labeled dosage.

  Length of Approval:

• Immune thrombocytopenia: 4 months

• Thrombocytopenia associated with chronic hepatitis C: Up to 6 months (length of approval will not exceed duration of antiviral therapy)

• Aplastic anemia: 4 months

Renewal Criteria

Promacta (eltrombopag) may be approved for RENEWAL when ALL of the following criteria are met:

1. The individual has been previously approved for therapy with the requested agent through BCBSMS PA process;

2. ONE of the following:

   1. Persistent or Chronic ITP: The individual has documented clinical response (i.e., achievement and maintenance of a platelet count >30 x 10⁹/L, at least doubling of the baseline platelet count, and/or platelet count increased sufficiently to avoid clinically significant bleeding);

   2. Severe aplastic anemia:The individual has shown clinical improvement from baseline (i.e. increase in platelet count, reduction in red blood cell transfusions, hemoglobin increase, and/or absolute neutrophil increase); OR

   3. Thrombocytopenia associated with chronic hepatitis C: The individual has increased platelet count and is receiving concomitant interferon-based therapy;

3. The requested agent will not be used in combination with another thrombopoietin receptor agonists (e.g., Nplate) or spleen tyrosine kinase inhibitor (e.g., Tavalisse);

4. The prescriber is or has consulted with a specialist in an area of the individual's diagnosis (i.e., hematologist);

5. The individual does not have any FDA-labeled contraindication(s) to therapy with the requested agent; AND

6. The prescribed dosage is within the program quantity limits based on FDA approved labeled dosage;

  Length of Approval:

• Immune thrombocytopenia: 12 months

• Thrombocytopenia associated with chronic hepatitis C: Up to 6 months (length of approval will not exceed duration of antiviral therapy)

• Aplastic anemia: 12 months

POLICY EXCEPTIONS

State Health Plan (State and School Employees): The prescription drug(s) in this medical policy may be covered under a prescription drug benefit plan administered by the State Health Plan’s Pharmacy Benefit Manager. Please perform a formulary drug search at https://www.dfa.ms.gov/cvs-caremark and submit any required Prior Authorization Requests for coverage determination to the Plan’s Pharmacy Benefit Manager. Services related to delivery and/or administration of a medication determined to be not medically necessary will also be considered not medically necessary. Services related to delivery and/or administration of a self-administered drug are not covered.

POLICY GUIDELINES

The coverage guidelines outlined in the Medical Policy Manual should not be used in lieu of the Member's specific benefit plan language.

Medically Necessary is defined as those services, treatments, procedures, equipment, drugs, devices, items or supplies furnished by a covered Provider that are required to identify or treat a Member's illness, injury or Mental Health Disorders, and which Company determines are covered under this Benefit Plan based on the criteria as follows in A through D:

A. consistent with the symptoms or diagnosis and treatment of the Member's condition, illness, or injury; and

B. appropriate with regard to standards of good medical practice; and

C. not solely for the convenience of the Member, his or her Provider; and

D. the most appropriate supply or level of care which can safely be provided to Member. When applied to the care of an Inpatient, it further means that services for the Member's medical symptoms or conditions require that the services cannot be safely provided to the Member as an Outpatient.

For the definition of Medically Necessary, “standards of good medical practice” means standards that are based on credible scientific evidence published in peer-reviewed medical literature generally recognized by the relevant medical community, and physician specialty society recommendations, and the views of medical practitioners practicing in relevant clinical areas and any other relevant factors. BCBSMS makes no payment for services, treatments, procedures, equipment, drugs, devices, items or supplies which are not documented to be Medically Necessary. The fact that a Physician or other Provider has prescribed, ordered, recommended, or approved a service or supply does not in itself, make it Medically Necessary.

BCBSMS may request medical records for determination of medical necessity. When medical records are requested, letters of support and/or explanation are often useful, but are not sufficient documentation unless all specific information needed to make a medical necessity determination is included.

Medication Failure

Medication failure is defined as disease progression at generally accepted doses at generally accepted durations of therapy as appropriate for the disease state being treated. Dosages below the recommended dose for the specific condition being treated and/or experience of common side effects of medication will not be considered medication failure or lack of response for the purpose of this review.

BCBSMS determines patient medication trial and adherence by a review of pharmacy claims data over the preceding twelve months. Additional information may be requested on a case-by-case basis to allow for proper review. If individual is new to BCBSMS and pharmacy records are needed to confirm medication trials and adherence, it is the responsibility of the individual and/or requesting provider to obtain said records and to submit them to BCBSMS upon request. Medical records from the provider that list previously prescribed medications will not be sufficient to show medication trials or adherence.

POLICY HISTORY

01/01/2014: New policy added.

11/12/2014: Policy statement criteria regarding hepatitis C associated thrombocytopenia updated to change the platelet count from "<100 x109/L" to "<75 x10⁹/L." Added criteria for Promacta for severe aplastic anemia to the policy statement.

08/14/2015: Code Reference section updated for ICD-10.

05/31/2016: Policy number L.5.01.463 added. Investigative definition updated in Policy Guidelines.

06/13/2016: Approved by Pharmacy & Therapeutics (P&T) Committee.

05/16/2017: Policy reviewed and approved by Pharmacy & Therapeutics (P&T) Committee.

03/27/2018: Policy reviewed and approved by Pharmacy & Therapeutics (P&T) Committee.

08/05/2020: Policy reviewed and approved by Pharmacy & Therapeutics (P&T) Committee. Policy title changed from "Promacta" to "Promacta (eltrombopag)." Policy description updated to add indications for Promacta (eltrombopag). Policy section updated to add the following: 1) Prior authorization is required; 2) The use of samples by a Member will not be considered current or stable therapy for purposes of Medical Policy review. Updated approval criteria for Promacta (eltrombopag). Quantity limits table updated to include the generic name and to remove GPI information. Added length of approval for listed conditions. Policy Guidelines updated to define medication failure. Sources updated.

02/01/2023: Policy Exceptions updated to add the following: State Health Plan (State and School Employees): The prescription drug(s) in this medical policy may be covered under a prescription drug benefit plan administered by the State Health Plan’s Pharmacy Benefit Manager. Please perform a formulary drug search at https://www.dfa.ms.gov/cvs-caremark and submit any required Prior Authorization Requests for coverage determination to the Plan’s Pharmacy Benefit Manager. Services related to delivery and/or administration of a medication determined to be not medically necessary will also be considered not medically necessary. Services related to delivery and/or administration of a self-administered drug are not covered.

10/01/2024: Policy reviewed and approved by the Pharmacy & Therapeutics (P&T) Committee. Policy description updated. Revised medically necessary criteria for persistent or chronic immune thrombocytopenia, hepatitis C associated thrombocytopenia, and severe aplastic anemia. Removed quantity limits table. Length of approval updated for hepatitis C associated thrombocytopenia and aplastic anemia. Added renewal criteria. Policy Exceptions updated to remove statement that Promacta prior authorization is not required for Federal Employee Program (FEP) members. Policy Guidelines updated regarding BCBSMS request for medical records and to define medication failure. Changed "Nervous/Mental Conditions" to "Mental Health Disorders" and removed investigative definition from Policy Guidelines. Sources updated. Effective 12/01/2024.

09/09/2025: Policy reviewed by Pharmacy & Therapeutics (P&T) Committee; no changes to policy statement. Sources updated.

04/17/2026: Policy updated to revise coverage criteria for individuals with a diagnosis of persistent or chronic immune thrombocytopenia, thrombocytopenia associated with chronic hepatitis C, and severe aplastic anemia. Revised statement regarding combination therapy. Updated length of approval for initial and renewal criteria. Policy Guidelines updated regarding medication failure.

SOURCE(S)

1. Arnold, D., Cuker, A. Immune thrombocytopenia (ITP) in adults: Clinical manifestations and diagnosis. In: UpToDate, Connor RF (Ed), Wolters Kluwer. Accessed on June 24, 2025. Available from

   https://www.uptodate.com/contents/immune-thrombocytopenia-itp-in-adults-clinical-manifestations-and-diagnosis

2. DeZern AE, Churpek JE. Approach to the diagnosis of aplastic anemia. Blood Adv. 2021 Jun 22;5(12):2660-2671. doi: 10.1182/bloodadvances.2021004345.

3. Neunert C, Terrell DR, Arnold DM, et al. American Society of Hematology 2019 guidelines for immune thrombocytopenia. Blood Adv 2019; 3:3829-3866.

4. Promacta prescribing information. Novartis Pharmaceuticals Corporation.  June 2025. Last accessed July 2025.

CODE REFERENCE

None