This policy version was replaced July 4, 2022. To find the newest version, go to https://www.bluecrossmn.com/providers/medical-policy-and-utilization-management, read and accept the Blue Cross Medical Policy Statement, then select “Blue Cross and Blue Shield of Minnesota Medical Policies.” This will bring up the Medical Policy search screen. Enter the policy number without the version number (last 3 digits).

Romiplostim is a thrombopoietin (TPO) receptor agonist which increases platelet production through binding and activation of the TPO receptor, a mechanism analogous to endogenous TPO. Romiplostim is administered by subcutaneous injection by a healthcare professional.

The U.S. Food and Drug Administration (FDA) has approved romiplostim (Nplate®) for the treatment of thrombocytopenia in;

• Adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy.
• Pediatric patients 1 year of age and older with ITP for at least 6 months who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy.
• In adults and pediatric patients (including term neonates) to increase survival when acutely exposed to myelosuppressive doses of radiation (Hematopoietic Syndrome of Acute Radiation Syndrome [HS-ARS]).

Romiplostim should only be used in patients with ITP whose degree of thrombocytopenia and clinical condition increases the risk for bleeding. It should not be used in an attempt to normalize platelet counts. Romiplostim is not indicated for the treatment of thrombocytopenia due to myelodysplastic syndrome (MDS) or any cause of thrombocytopenia other than chronic ITP.

Thrombotic/thromboembolic complications may result from an increase in platelet counts with use of romiplostim. Portal vein thrombosis has been reported in patients with chronic liver disease receiving romiplostim.

Definitions:

Lower risk myelodysplastic syndrome is defined by the International Prognostic Scoring System (IPSS) and identifies the prognosis of primary untreated adult patients with MDS. Lower risk classifications are IPSS-R (Very Low, Low, Intermediate), IPSS (Low/Intermediate-1), and WPSS (Very Low, Low, Intermediate).

Note: This policy does not address romiplostim for use in patients with hematopoietic syndrome of acute radiation syndrome (HS-ARS) due to the urgency of the required treatment.

I.   Initial and Renewal Review for Oncologic Indications

Romiplostim may be considered MEDICALLY NECESSARY AND APPROPRIATE when ALL of the following criteria are met:

• Diagnosis of lower risk myelodysplastic syndrome (MDS) with severe or refractory thrombocytopenia following disease progression or no response to hypomethylating agents, immunosuppressive therapy, or clinical trial*; AND
• No FDA labeled contraindications to therapy (see table 1 below); AND
• The dose is supported in the literature for the indication.

*NOTE: The FDA approved prescribing information for romiplostim includes the following limitation of use: Romiplostim is not indicated for the treatment of thrombocytopenia due to myelodysplastic syndrome (MDS) or any cause of thrombocytopenia other than chronic ITP.

II.  Initial Review for Non-Oncologic Indications

Romiplostim may be considered MEDICALLY NECESSARY AND APPROPRIATE when ALL of the following criteria are met:

• ONE of the following:
  • Adult patients (age ≥18 years) with a diagnosis of chronic immune thrombocytopenia (ITP); OR
  • Pediatric patients (age ≥1 year) with a diagnosis of ITP and child has had ITP for ≥6 months; AND
• For patients not currently receiving romiplostim, platelet count is less than 30,000/mm³; AND
• ONE of the following:
  • Inadequate response to splenectomy, glucocorticoid therapy, or immune globulin therapy; OR
  • Documented intolerance, FDA-labeled contraindication, or hypersensitivity to glucocorticoid therapy and immune globulin therapy; AND
• No FDA-labeled contraindications to romiplostim (see table 1 below); AND
• The dose is within the FDA-labeled dose for the indication (see table 2 below); AND
• For commercial health plan members only, romiplostim is administered in accordance with site of service criteria (see policy XI-06); AND
  
• For commercial health plan members only, step therapy supplement criteria may apply for select conditions (see policy II-242: Step Therapy Supplement).
  

III. Renewal Review for Non-Oncologic Indications

Romiplostim may be considered MEDICALLY NECESSARY AND APPROPRIATE when ALL of the following criteria are met:

• Previously approved for romiplostim through the initial review process; AND
• Demonstrated positive clinical response to romiplostim therapy as demonstrated by increased platelet count ≥ 50,000/mm; AND
• No FDA labeled contraindications to romiplostim (see table 1 below); AND
• The dose is within the FDA labeled dose for the indication (see table 2 below); AND
• For commercial health plan members only, romiplostim is administered in accordance with site of service criteria (see policy XI-06).
  

IV.  Experimental/Investigative Uses

All other uses of romiplostim are considered EXPERIMENTAL/INVESTIGATIVE due to the lack of clinical evidence demonstrating an impact on improved health outcomes.

Table 1. FDA Labeled Contraindications

Table 2. Dosing for Non-Oncologic Indications

NOTE: See documentation submission requirements below if the requested dose is higher or more frequent that the dosing criteria provided in this table.

Documentation Submission:

Documentation supporting the medical necessity criteria described in the policy must be included in the prior authorization, when prior authorization is required. In addition, the following documentation must also be submitted:

Initial Review for Non-Oncologic Indications

1. Clinical notes describing the diagnosis and clinical features of the diagnosis.
2. Laboratory reports documenting the patient’s platelet count is less than 30,000/mm³.
3. Clinical notes describing the current and past medications for the diagnosis, including response to the medications.
4. The dose being requested, including the patient's weight. If the requested dose is higher or more frequent than the dosing guidelines provided in the table above, a clear explanation for the medical necessity of the requested dose MUST be submitted, including prior dosing (strength and frequency) associated with inadequate response.
5. For commercial health plan members only, the site of service for romiplostim administration is specified, including CMS place of service code (see policy XI-06). If romiplostim is administered in a hospital outpatient facility, a clear explanation for the medical necessity of the site of service MUST be submitted, including documentation for one or more of the site of service criteria provided in policy XI-06.
   
6. For commercial health plan members only, when step therapy requirements apply for the requested indication, documentation for one or more of the step therapy supplement criteria MUST be provided (see policy II-242).
   

Renewal Review for Non-Oncologic Indications

1. Documentation of prior approval for romiplostim through the initial review process.
2. Documentation supporting the positive clinical response (i.e., platelet count ≥ 50,000/mm³).
3. Clinical notes describing current and past medications for the diagnosis, including response to the medications.
4. The dose being requested, including the patient's weight. If the requested dose is higher or more frequent than the dosing guidelines provided in the table above, a clear explanation for the medical necessity of the requested dose MUST be submitted, including prior dosing (strength and frequency) associated with inadequate response.
5. For commercial health plan members only, the site of service for romiplostim administration is specified, including CMS place of service code (see policy XI-06). If romiplostim is administered in a hospital outpatient facility, a clear explanation for the medical necessity of the site of service MUST be submitted, including documentation for one or more of the site of service criteria provided in policy XI-06.