Subject:
Romiplostim (Nplate)
Description:
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IMPORTANT NOTE:
The purpose of this policy is to provide general information applicable to the administration of health benefits that Horizon Blue Cross Blue Shield of New Jersey and Horizon Healthcare of New Jersey, Inc. (collectively “Horizon BCBSNJ”) insures or administers. If the member’s contract benefits differ from the medical policy, the contract prevails. Although a service, supply or procedure may be medically necessary, it may be subject to limitations and/or exclusions under a member’s benefit plan. If a service, supply or procedure is not covered and the member proceeds to obtain the service, supply or procedure, the member may be responsible for the cost. Decisions regarding treatment and treatment plans are the responsibility of the physician. This policy is not intended to direct the course of clinical care a physician provides to a member, and it does not replace a physician’s independent professional clinical judgment or duty to exercise special knowledge and skill in the treatment of Horizon BCBSNJ members. Horizon BCBSNJ is not responsible for, does not provide, and does not hold itself out as a provider of medical care. The physician remains responsible for the quality and type of health care services provided to a Horizon BCBSNJ member.
Horizon BCBSNJ medical policies do not constitute medical advice, authorization, certification, approval, explanation of benefits, offer of coverage, contract or guarantee of payment.
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Romiplostim (Nplate) was FDA approved in August 2008 for the treatment of thrombocytopenia in patients with chronic immune (idiopathic) thrombocytopenic purpura (ITP) who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy. In October 2019, the indication was expanded to allows use in adults with ITP (chronic and acute) and in pediatric patients with chronic ITP. ITP is an autoimmune disorder in which antiplatelet antibodies cause accelerated destruction of platelets as well as impair production of platelets. ITP is defined as isolated thrombocytopenia in a patient with no clinically apparent associated conditions or factors that can cause thrombocytopenia, such as HIV. Clinical presentation primarily includes minor bleeding symptoms, such as epistaxis, petechiae, and bruising. Intracranial hemorrhage, protracted epistaxis, hematuria, hemoptysis and gastrointestinal bleeding are more severe bleeding events that may occur with ITP. Current therapeutic options include corticosteroids, intravenous immunoglobulins, intravenous anti-D, splenectomy and romiplostim.
Romiplostim, a member of the thrombopoietin (TPO) mimetic class, is an Fc-peptide fusion protein (peptibody) that activates intracellular transcriptional pathways leading to increased platelet production via the TPO receptor. The peptibody molecule contains two identical single-chain subunits, each consisting of human immunoglobulin IgG1 Fc domain, covalently linked at the C-terminus to a peptide containing two thrombopoietin receptor-binding domains. Romiplostim has no amino acid sequence homology to endogenous TPO.
On December 6, 2011, the FDA removed the REMS program requirements. Healthcare professionals, hospitals, specialty care facilities, and patients are no longer required to be enrolled in the Nplate NEXUS (Network of Experts Understanding and Supporting Nplate and Patients) Program to prescribe, dispense, or receive these products. Healthcare professionals also will no longer be required to complete periodic safety forms for patients receiving romiplostim.
The modified REMS programs will include a communication plan that will inform healthcare professionals about the changes to the REMS and the safety risks associated with each product.
Policy:
(Note: For Medicare Advantage, please refer to the Medicare Coverage Section below for coverage guidance.)
The requirements of the Horizon BCBSNJ Romiplostim (Nplate) Program may require a precertification/prior authorization via MagellanRx Management. These requirements are member-specific: please verify member eligibility and requirements through the Horizon Provider Portal (www.horizonblue.com/provider). Ordering clinicians should request pre-certification from MagellanRx Management at ih.magellanrx.com or call 1-800-424-4508 (when applicable).
I. Romiplostim (Nplate) is medically necessary for the following FDA-approved indications:
- Treatment of chronic immune (idiopathic) thrombocytopenic purpura (ITP), in adult and pediatric patients (1 years or older) who meet the following criteria:
A. Has thrombocytopenia demonstrated by platelet count less than 30,000/mm3 (30x109/L) and clinical condition that increase the risk for bleeding; and
B. Insufficient response to corticosteroids, immunoglobulins, or splenectomy; and
C. Nplate is not being used to attempt to normalize platelet count.; and
D. Member is not receiving therapy with any other thrombopoietin agonist or mimetic (e.g., lusutrombopag, eltrombopag, avatrombopag, etc.) and
E. If the request is for a pediatric member (ages 1-17), member has had ITP for at least 6 months
- Treatment of acute immune (idiopathic) thrombocytopenic purpura (ITP), in adult patients (18 years or older) who meet the following criteria:
A. Has thrombocytopenia demonstrated by platelet count less than 30,000/mm3 (30x109/L) and clinical condition that increase the risk for bleeding; and
B. Insufficient response to corticosteroids, immunoglobulins, or splenectomy; and
C. Nplate is not being used to attempt to normalize platelet count; and
D. Member is not receiving therapy with any other thrombopoietin agonist or mimetic (e.g., lusutrombopag, eltrombopag, avatrombopag,
II. When romiplostim (Nplate) is medically necessary, initial therapy will be approved for a period of 12 weeks at the following FDA recommend doses:
- Initial dose of 1 mcg/kg based on actual body weight once weekly as a subcutaneous injection. Do not exceed the maximum weekly dose of 10 mcg/kg. Intended to maintain a platelet count of ≥50 x 109 (50,000/mm3). Do not dose if platelet count is >400 x 109/L.
[INFORMATIONAL NOTE: As per the FDA labeled package insert, for all patients, use the lowest dose of Nplate to achieve and maintain a platelet count ≥ 50 × 109 /L as necessary to reduce the risk for bleeding. Administer Nplate as a weekly subcutaneous injection with dose adjustments based upon the platelet count response.
For adult patients with ITP, future dose adjustments are based on changes in platelet counts only. Adjust the weekly dose of Nplate by increments of 1 mcg/kg until the patient achieves a platelet count ≥ 50 × 109 /L as necessary to reduce the risk for bleeding; do not exceed a maximum weekly dose of 10 mcg/kg. In clinical studies, most adult patients who responded to Nplate achieved and maintained platelet counts ≥ 50 × 109 /L with a median dose of 2 mcg/kg. Adjust the dose as following for adult patients:
-If the platelet count is < 50 × 109 /L, increase the dose by 1 mcg/kg.
-If platelet count is > 200 × 109 /L and ≤ 400 × 109 /L for 2 consecutive weeks, reduce the dose by 1 mcg/kg.
-If platelet count is > 400 × 109 /L, do not dose. Continue to assess the platelet count weekly. After the platelet count has fallen to < 200 × 109 /L, resume Nplate at a dose reduced by 1 mcg/kg.
For pediatric patients with ITP, future dose adjustments are based on changes in platelet counts and changes in body weight. Reassessment of body weight is recommended every 12 weeks. Adjust the weekly dose of Nplate by increments of 1 mcg/kg until the patient achieves a platelet count ≥ 50 × 109 /L as necessary to reduce the risk for bleeding; do not exceed a maximum weekly dose of 10 mcg/kg. In a pediatric placebo-controlled clinical study, the median of the most frequent dose of Nplate received by patients during weeks 17 through 24 was 5.5 mcg/kg. Adjust the dose as follows for pediatric patients:
-If the platelet count is < 50 × 109 /L, increase the dose by 1 mcg/kg.
-If platelet count is > 200 × 109 /L and ≤ 400 × 109 /L for 2 consecutive weeks, reduce the dose by 1 mcg/kg.
-If platelet count is > 400 × 109 /L, do not dose. Continue to assess the platelet count weekly. After the platelet count has fallen to < 200 × 109 /L, resume Nplate at a dose reduced by 1 mcg/kg]
III. Continued therapy will be approved every 6 months after the initial 12 week period, if :
- Member had disease response indicated by the achievement and maintenance of a platelet count of at least 50 × 109/L (not exceeding 400 X 109/L); AND
- Absence of unacceptable toxicity from the drug. (e.g.: progression of MDS to AML; thrombotic/thromboembolic complications; bone marrow reticulin formation; severe hypersensitivity reactions)
IV. Romiplostim (Nplate) is considered medically necessary for the off-label indications that are 2A or better recommendations on National Comprehensive Cancer Network (NCCN) compendium. Refer to National Comprehensive Cancer Network: Drugs and Biologics Compendium - Romiplostim (Nplate). Available at: https://www.nccn.org/professionals/drug_compendium/content/.
V. Other uses of romiplostim (Nplate) are considered investigational.
Medicare Coverage
There is no National Coverage Determination (NCD) or Local Coverage Determination (LCD) for jurisdiction JL specific to this drug. Romiplostim (Nplate) is administered subcutaneously. Per Local Coverage Article A53127, Drugs ADMINISTERED subcutaneously are considered to be usually SELF-ADMINISTERED. If a drug is SELF-ADMINISTERED by more than 50 percent of Medicare beneficiaries, the drug is excluded from coverage. For members with a Medicare drug plan (Part D) Romiplostim (Nplate) may be covered under that plan.
Medicaid Coverage
For Horizon NJ Health members, please follow this link for the corresponding HNJH drug policy https://services3.horizon-bcbsnj.com/ddn/NJhealthWeb.nsf
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Horizon BCBSNJ Medical Policy Development Process:
This Horizon BCBSNJ Medical Policy (the “Medical Policy”) has been developed by Horizon BCBSNJ’s Medical Policy Committee (the “Committee”) consistent with generally accepted standards of medical practice, and reflects Horizon BCBSNJ’s view of the subject health care services, supplies or procedures, and in what circumstances they are deemed to be medically necessary or experimental/ investigational in nature. This Medical Policy also considers whether and to what degree the subject health care services, supplies or procedures are clinically appropriate, in terms of type, frequency, extent, site and duration and if they are considered effective for the illnesses, injuries or diseases discussed. Where relevant, this Medical Policy considers whether the subject health care services, supplies or procedures are being requested primarily for the convenience of the covered person or the health care provider. It may also consider whether the services, supplies or procedures are more costly than an alternative service or sequence of services, supplies or procedures that are at least as likely to produce equivalent therapeutic or diagnostic results as to the diagnosis or treatment of the relevant illness, injury or disease. In reaching its conclusion regarding what it considers to be the generally accepted standards of medical practice, the Committee reviews and considers the following: all credible scientific evidence published in peer-reviewed medical literature generally recognized by the relevant medical community, physician and health care provider specialty society recommendations, the views of physicians and health care providers practicing in relevant clinical areas (including, but not limited to, the prevailing opinion within the appropriate specialty) and any other relevant factor as determined by applicable State and Federal laws and regulations.
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Index:
Romiplostim (Nplate)
Idiopathic (Immune) Thrombocytopenic Purpura
ITP Treatment with Romiplostim (Nplate)
Nplate (Romiplostim)
References:
1. Nplate prescribing information. Amgen Inc. Thousand Oaks, CA. 10/2019.
2. Kuter DJ, Bussel JB, Lyons RM, et al. Efficacy of romiplostim in patients with chronic immune thrombocytopenic purpura: a double-blind randomized controlled trial. Lancet 2008;371:395-403.
3. George JN, Woolf SH, Raskob GE, et al. Idiopathic thrombocytopenic purpura: a practice guideline developed by explicit methods for the American Society of Hematology. Blood 1996;88:3–40.
4. Psaila B, Bussel JB. Immune Thrombocytopenic Purpura. Hematol Oncol Clin N Am 2007;21:743-59.
5. Two New Drugs for Chronic ITP. Med Lett Drugs Ther 2009;51(1305):10-11.
6. Nplate(R) Approved in the European Union for the Treatment of Chronic Immune (Idiopathic) Thrombocytopenic Purpura (ITP); http://www.amgen.com/media/media_pr_detail.jsp?releaseID=1253720; . February 6, 2009. Accessed October 16, 2009.
7. Cines DB and Blanchette VS. Immune thrombocytopenic purpura. N Eng J Med. 2002; 346(13): 995.
8. Kantarjian H, Fenaux P, Sekeres MA, et al. Safety and efficacy of romiplostim in patients with lower-risk myelodysplastic syndrome and thrombocytopenia. J Clin Oncol. 2010;28(3):437-44.
9. Bussel JB, Kuter DJ, Pullarkat V, et al. Safety and efficacy of long-term treatment with romiplostim in thrombocytopenic patients with chronic ITP. Blood. 2009;113(10):2161-71.
10. MICROMEDEX® 2.0 (Healthcare Series). DRUGDEX® Evaluations. Romiplostim. Updated February 14, 2014. Available at: http://www.thomsonhc.com. Accessed Feburary 18, 2015.
11. Clinical Pharmacology. Available at: www.clinicalpharmacology.com. Accessed February 28, 2020.
12. British Committee for Standards in Haematology General Haematology Task Force. Guidelines for the investigation and management of idiopathic thrombocytopenic purpura in adults, children and in pregnancy. Br J Haematol. 2003;120:574-596.
13. The American Society of Hematology 2011 evidence-base practice guideline for immune thrombocytopenia. Blood. 2011;117(16):4190-4207.
14. American Hospital Formulary Service® (AHFS). AHFS Drug Information 2013®. Bethesda, MD: American Society of Health-System Pharmacists®, 2013.
15. Nplate. Clinicaltrial.gov. Accessed on 2/26/20. Available at: https://clinicaltrials.gov/ct2/results?cond=&term=nplate&cntry=&state=&city=&dist=
Codes:
(The list of codes is not intended to be all-inclusive and is included below for informational purposes only. Inclusion or exclusion of a procedure, diagnosis, drug or device code(s) does not constitute or imply authorization, certification, approval, offer of coverage or guarantee of payment.)
CPT*
HCPCS
* CPT only copyright 2020 American Medical Association. All rights reserved. CPT is a registered trademark of the American Medical Association.
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Medical policies can be highly technical and are designed for use by the Horizon BCBSNJ professional staff in making coverage determinations. Members referring to this policy should discuss it with their treating physician, and should refer to their specific benefit plan for the terms, conditions, limitations and exclusions of their coverage.
The Horizon BCBSNJ Medical Policy Manual is proprietary. It is to be used only as authorized by Horizon BCBSNJ and its affiliates. The contents of this Medical Policy are not to be copied, reproduced or circulated to other parties without the express written consent of Horizon BCBSNJ. The contents of this Medical Policy may be updated or changed without notice, unless otherwise required by law and/or regulation. However, benefit determinations are made in the context of medical policies existing at the time of the decision and are not subject to later revision as the result of a change in medical policy
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