BlueCross and BlueShield of Montana Medical Policy/Codes
Soliris (eculizumab)
Chapter: Drugs - Medical Benefit
Current Effective Date: August 27, 2013
Original Effective Date: August 27, 2013
Publish Date: May 27, 2013
Description

Paroxysmal nocturnal hemoglobinuria (PNH), which usually develops in adults, is a disease characterized by red blood cells that develop abnormally.  Once the abnormal cells are present in the bloodstream, naturally occurring proteins (called the complement system), designed to destroy bacteria and other infection-causing organisms, break these cells down.  This leads to abnormally darkened urine and, more importantly, causes anemia.  Depending upon the severity of the disorder, patients with PNH may have pain, fatigue and debilitating weakness, the need for frequent blood transfusions, blood clots, and life-threatening or fatal strokes, heart attacks and intestinal disease.  Soliris does not cure PNH, but treats the breakdown of red blood cells, the most common characteristic of PNH.  Soliris acts to block the complement system activity, including the destruction of PNH red blood cells.  Terminal complement inhibition with Soliris increases the susceptibility to serious meningococcal infections.   

The U.S. Food and Drug Administration (FDA) approved Soliris on March 16, 2007.  Soliris is classified as an orphan drug product.  Orphan products are developed to treat rare diseases or conditions that affect fewer than 200,000 people in the United States.  The Orphan Drug Act provides a seven-year period of exclusive marketing to the first manufacturer who obtains marketing approval for a designated orphan product.  About one person out of a million people will be diagnosed with PNH.

Atypical Hemolytic Uremic Syndrome (aHUS)

A rare and chronic blood disease that can lead to kidney (renal) failure and is also associated with increased risk of death and stroke.  Atypical HUS accounts for 5 to 10 percent of all cases of hemolytic uremic syndrome. The disease disproportionately affects children. Soliris will continue to be available only through a restricted program, and prescribers must enroll in a registration program and provide a medication guide to patients who receive the drug. (7)

Soliris is given through intravenous (IV) infusion.  The recommended dose of Soliris is dependent on the indication for which the drug is administered and the age of the patient (adult or child).  Soliris is manufactured by Alexion Pharmaceuticals, Inc.

Policy

Prior authorization is recommended. To authorize, call Blue Cross and Blue Shield of Montana (BCBSMT) Customer Service at 1-800-447-7828 or fax your request to the Medical Review Department at 406-441-4624. A retrospective review will be performed if services are not prior authorized.

Medically Necessary

BCBSMT may consider Soliris™ (eculizumab) medically necessary to reduce hemolysis for patients who have paroxysmal nocturnal hemoglobinuria (PNH) AND meet the following criteria:

  • Documentation that the patient has received a meningococcal vaccine at least two weeks prior to receiving the first dose of Soliris; and
  • Documentation of diagnosis of PNH through analysis by:
    • Flow cytometry of erythrocytes for CD59 deficiency; or
    • Granulocytes for either CD59 or CD55

Soliris (eculizumab) may be considered medically necessary to treat patients with atypical hemolytic uremic syndrome (aHUS) with documentation that the patient has received a meningococcal vaccine at least two weeks prior to receiving the first dose of Soliris.

Investigational

BCBSMT considers Soliris experimental, investigational and unproven for all other indications, including but not limited to treatment of patients with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS).

Federal Mandate

Federal mandate prohibits denial of any drug, device, or biological product fully approved by the FDA as investigational for the Federal Employee Program (FEP). In these instances coverage of these FDA-approved technologies are reviewed on the basis of medical necessity alone. Call the BCBSMT FEP Customer Service Department at 1-800-634-3569 for benefit information.

Rationale for Benefit Administration

This medical policy was developed through consideration of peer reviewed medical literature, FDA approval status, accepted standards of medical practice in Montana, Technology Evaluation Center evaluations, and the concept of medical necessity. BCBSMT reserves the right to make exceptions to policy that benefit the member when advances in technology or new medical information become available.

The purpose of medical policy is to guide coverage decisions and is not intended to influence treatment decisions. Providers are expected to make treatment decisions based on their medical judgment. BCBSMT recognizes the rapidly changing nature of technological development and welcomes provider feedback on all medical policies.

When using this policy to determine whether a service, supply or device will be covered, please note that member contract language will take precedence over medical policy when there is a conflict.

Rationale

U.S. Food and Drug Administration (FDA)  approval of Soliris was based on the results of three multi-national clinical studies:

  • TRIUMPH, a placebo-controlled 26 week Phase 3 study involving 87 PNH patients,
  • SHEPHERD, an open-label 52 week Phase 3 trial involving 97 PNH patients, and
  • E05-001, a long term extension study.

These studies showed that Soliris reduced hemolysis in every treated patient.  Hemolysis was dramatically reduced from a baseline LDH of 2,032 U/L to 239 U/L at week 26 (p<0.001).  The reductions in hemolysis occurred within one week of initiating treatment and were sustained for periods of up to 54 months with continued dosing of Soliris.  The reduction in hemolysis expands the number of circulating PNH cells and, thereby, increases the hemoglobin level.  Hemoglobin stabilization and the number of transfused packed red blood cell units, the pivotal study's co-primary endpoints, were both achieved.  Half of the Soliris-treated patients achieved hemoglobin stabilization compared with none of the patients in the placebo group; the median number of transfusions was reduced from 10 units/patient to 0 units/patient, respectively (p < 0.001 in both cases).  Soliris patients reported less fatigue and improved health-related quality of life.  There were fewer thrombotic events with Soliris treatment than during the same period of time prior to treatment. (1,2,3,4,5)

2010 Update

A search of peer reviewed literature through October 2010 identified no new clinical trial publications or any additional information that would change the coverage position of this medical policy.

2012 Update

Atypical hemolytic uremic syndrome (aHUS)

Soliris’ safety and effectiveness for aHUS were established in two single-arm trials in 37 adults and adolescent patients with aHUS and one retrospective study in 19 pediatric patients and 11 adult patients with aHUS.  Patients treated with Soliris in these studies experienced a favorable improvement in kidney function, including elimination of the requirement for dialysis in several patients with aHUS that did not respond to plasma therapy. Patients treated with Soliris also exhibited improvement in platelet counts and other blood parameters that correlate with aHUS disease activity.

There are no other FDA-approved treatments for aHUS, and the safety and effectiveness of current standard treatment, plasma therapy (plasma exchange or fresh frozen plasma infusion), have not been studied in well controlled trials.

The effectiveness of Soliris in aHUS is based on the effects on thrombotic microangiopathy (TMA) and renal function. Prospective clinical trials in additional patients are ongoing to confirm the benefit of Soliris in patients with aHUS. (7)

Coding

Disclaimer for coding information on Medical Policies

Procedure and diagnosis codes on Medical Policy documents are included only as a general reference tool for each policy. They may not be all-inclusive.

The presence or absence of procedure, service, supply, device or diagnosis codes in a Medical Policy document has no relevance for determination of benefit coverage for members or reimbursement for providers. Only the written coverage position in a medical policy should be used for such determinations.

Benefit coverage determinations based on written Medical Policy coverage positions must include review of the member’s benefit contract or Summary Plan Description (SPD) for defined coverage vs. non-coverage, benefit exclusions, and benefit limitations such as dollar or duration caps.

ICD-9 Codes

283.11, 283.2

ICD-10 Codes

D595, D596, D598

Procedural Codes: J1300
References
  1. Hillmen, P., Young, N., et al.  The Complement Inhibitor Eculizumab in Paroxsysmal Nocturnal Hemoglobinuria.  New England Journal of Medicine (2006 September 21) 355(12):1233- 43.
  2. Young, N., Antonioli, E., et al.  Safety and efficacy of the terminal complement inhibitor eculizumab in patients with paroxysmal nocturnal hemoglobinuria: SHEPHERD phase III clinical study results. Blood (2006) 108:971.
  3. Hillmen, P., Muus, P. et al.  The terminal complement inhibitor eculizumab reduces thrombosis in patients with paroxysmal nocturnal hemoglobinuria.  Blood (2006) 108:123.
  4. Soliris(TM) (eculizumab) prescribing information.  Alexion Pharmaceuticals, Inc., 2006.
  5. Product Information: Soliris solution for IV infusion, eculizumab solution for IV infusion. Alexion Pharmaceuticals, Inc, Cheshire, CT, 2009.
  6. Thomson Reuters - Micromedex Healthcare Series. Drugdex Evaluations: Soliris - www.thomsonhc.com .   Assessed November 18, 2010.
  7. FDA – Department of Health and Human Services – Soliris® (eculizumab) – Approval Labeling (9/23/2011). www.FDA.gov  Accessed October 2012.
History
May 2013  New BCBSMT medical policy.  Possibly investigational.
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Soliris (eculizumab)