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 ≥1 year of age;

      2. ONE of the following:

         1. The individual has a baseline platelet count <30,000/µL (30 x10⁹/L) and severe bleeding (e.g., intracranial, gastrointestinal);

         2. The individual has a baseline platelet count of <20,000/µL (20 x10⁹/L);

      3. ONE of the following:

         1. The individual has had an inadequate response (i.e., failure to achieve and maintain a platelet count ≥30,000/µL (30 x10⁹/L) for 3-6 months following completion of treatment) to all of the following therapies, with or without a splenectomy:

            1. corticosteroids;

            2. immunoglobulins (IVIg);

            3. a rituximab product;

         2. The individual has a documented intolerance, FDA-labeled contraindication, or hypersensitivity to ALL of the above treatments;

      4. The individual will not be prescribed combination therapy with thrombopoietin receptor agonists (i.e., Nplate, Doptelet, Mulpleta, etc.);

      5. The prescriber is a specialist 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 hepatitis C associated thrombocytopenia and ALL of the following:

      1. The individual is ≥18 years of age;

      2. ONE of the following:

         1. The individual’s platelet count is <75 x10⁹/L AND the intent is to increase platelet counts sufficiently to initiate interferon therapy;

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

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

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

      1. The individual is ≥2 years of age;

      2. All of the following:

         1. At least TWO of the following blood criteria:

            1. Neutrophils less than 0.5 X 10⁹/L;

            2. Platelets less than 20 X 10⁹/L;

            3. Reticulocytes less than 1% corrected (percentage of actual hematocrit [Hct] to normal Hct) or reticulocyte count <20 X 10⁹/L;

         2. At least ONE of the following marrow criteria:

            1. Severe hypocellularity: <25%;

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

      3. One of the following:

         1. The individual has had an insufficient response to immunosuppressive therapy (defined as failure to Atgam (antithymocyte globulin [ATG]) and cyclosporine);

         2. The individual has an FDA labeled contraindication, intolerance, or hypersensitivity to both ATG and cyclosporine;

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

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

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

  Length of Approval:  

• Immune thrombocytopenia: 8 weeks

• Hepatitis C associated thrombocytopenia: Up to 12 weeks (length of approval will not exceed antiviral treatment)

• Aplastic anemia: 4 months

Renewal Criteria

Promacta 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. The individual has a diagnosis of Immune thrombocytopenia or Aplastic anemia and ALL of the following:

   1. The individual has shown clinical improvement (i.e. increase in platelet count from baseline and at least doubling of the baseline platelet count);

   2. Current (within the last 90 days) platelet count is <400 x 10⁹/L;

   3. The individual is not being treated using combination therapy with thrombopoietin receptor agonists (e.g., Nplate, Doptelet, Mulpleta);

3. The prescriber is a hematologist or has consulted with a hematologist;

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

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

  Length of Approval: 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:

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

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

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

4. 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 failure to achieve and maintain a platelet count ≥30,000/L for 3-6 months following completion of treatment. 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.

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