Non-Discrimination Statement and Multi-Language Interpreter Services information are located at the end of this document.

Transcription

1 SOLIRIS (eculizumab) Non-Discrimination Statement and Multi-Language Interpreter Services information are located at the end of this document. Coverage for services, procedures, medical devices and drugs are dependent upon benefit eligibility as outlined in the member's specific benefit plan. This Medical Coverage Guideline must be read in its entirety to determine coverage eligibility, if any. This Medical Coverage Guideline provides information related to coverage determinations only and does not imply that a service or treatment is clinically appropriate or inappropriate. The provider and the member are responsible for all decisions regarding the appropriateness of care. Providers should provide BCBSAZ complete medical rationale when requesting any exceptions to these guidelines. The section identified as Description defines or describes a service, procedure, medical device or drug and is in no way intended as a statement of medical necessity and/or coverage. The section identified as Criteria defines criteria to determine whether a service, procedure, medical device or drug is considered medically necessary or experimental or investigational. State or federal mandates, e.g., FEP program, may dictate that any drug, device or biological product approved by the U.S. Food and Drug Administration (FDA) may not be considered experimental or investigational and thus the drug, device or biological product may be assessed only on the basis of medical necessity. Medical Coverage Guidelines are subject to change as new information becomes available. For purposes of this Medical Coverage Guideline, the terms "experimental" and "investigational" are considered to be interchangeable. BLUE CROSS, BLUE SHIELD and the Cross and Shield Symbols are registered service marks of the Blue Cross and Blue Shield Association, an association of independent Blue Cross and Blue Shield Plans. All other trademarks and service marks contained in this guideline are the property of their respective owners, which are not affiliated with BCBSAZ. Description: Soliris (eculizumab) is a humanized monoclonal antibody that binds to the human C5 complement protein. Soliris is indicated for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis; for the atypical hemolytic uremic syndrome (ahus) to inhibit complement-mediated thrombotic microangiopathy; and for the treatment of adult individuals with generalized Myasthenia Gravis (gmg) who are anti-acetylcholine receptor (AchR) antibody positive. Soliris is not indicated for the treatment of individuals with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS). O938.4.docx Page 1 of 14

2 Description: (cont.) Paroxysmal Nocturnal Hemoglobinuria (PNH): A rare disorder in which red blood cells are easily destroyed by certain immune system proteins. Symptoms include blood clots and red or brownish urine in the morning. Aplastic anemia (decreased production of blood cells) may lead to PNH, and people with PNH are at increased risk of acute myelogenous leukemia. Atypical Hemolytic Uremic Syndrome (ahus): A disease that causes abnormal blood clots to form in small blood vessels in the kidneys. These clots can lead to serious medical problems if they restrict or block blood flow, including hemolytic anemia, thrombocytopenia, and kidney failure. It can occur at any age and is often caused by a combination of environmental and genetic factors. Genetic factors involve genes that code for proteins that help control the complement system (part of your body s immune system). Environmental factors include certain medications (such as anticancer drugs), chronic diseases (e.g., systemic sclerosis and malignant hypertension), viral or bacterial infections, cancers, organ transplantation, and pregnancy. Most cases are sporadic. Less than 20 percent of all cases have been reported to run in families. When the disorder is familial, it can have an autosomal dominant or an autosomal recessive pattern of inheritance. ahus differs from a more common condition called typical hemolytic-uremic syndrome. The two disorders have different causes and different signs and symptoms. Myasthenia Gravis Foundation of America (MGFA) Clinical Classification Identifies subgroups of individuals with distinct clinical features or severity of disease Myasthenia Gravis Specific Activities of Daily Living (MG ADL) scale A categorical scale that assesses the impact on daily function of 8 signs or symptoms that are typically affected in gmg. Each item is assessed on a 0-3-point scale where a score of 0 represents normal function and a score of 3 represents loss of ability to perform that function (total score ranges 0-24) MGFA Quantitative Myasthenia Gravis (QMG) test A 13-item categorical scale that assesses muscle weakness. Each item is assessed on a 0-3-point scale where a score of 0 represents no weakness and a score of 3 represents severe weakness (total ranges score 0-39). O938.4.docx Page 2 of 14

3 Description: (cont.) Commonly used therapies for myasthenia gravis include the following: Immunotherapies: - Prednisone - Azathioprine - Mycophenolate mofetil - Cyclosporine and tacrolimus - Intravenous immune globulin Other agents include: - Rituximab - Etanercept - Methotrexate - Cyclophosphamide Definitions: Adult: Age 18 years or older Risk Evaluation and Mitigation Strategies (REMS): Use of Soliris is subject to a Risk Evaluation and Mitigation Strategies (REMS) program that requires provider, patient, and dispensing pharmacy be enrolled into the program. Only providers and Pharmacies enrolled into the REMS may prescribe and dispense the drug, respectively, to individuals who are also in the program. A REMS program attempts to manage known or potentially serious risks associated with a drug product and is required by the Food and Drug Administration (FDA) for some drugs to ensure that the benefits of a drug outweigh its risks. O938.4.docx Page 3 of 14

4 Criteria: SOLIRIS IS AVAILABLE ONLY THROUGH RESTRICTED DISTRIBUTION UNDER A RISK EVALUATION AND MITIGATION STRATEGY (REMS) PROGRAM CALLED SOLIRIS REMS PROGRAM. Effective 03/01/18: For site of service requirements for Soliris (eculizumab), see BCBSAZ Medical Coverage Guideline #O1008, Site of Service Requirements for Certain Medications. See Resources section for FDA-approved dosage and MGFA Clinical Classification index table, MG-ADL Scale index table, MGFA QMG Test index table. Soliris for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis in adults with no evidence of an unresolved serious Neisseria meningitidis infection is considered medically necessary. Soliris for the treatment of atypical hemolytic uremic syndrome (ahus) to inhibit complementmediated thrombotic microangiopathy in an individual with no evidence of an unresolved serious Neisseria meningitidis infection is considered medically necessary. Soliris for the treatment of adults with generalized Myasthenia Gravis (gmg) is considered medically necessary with documentation of ALL of the following: 1. Positive serologic test for anti-achr antibodies 2. No evidence of an unresolved serious Neisseria meningitides infection 3. Myasthenia Gravis Foundation of America (MGFA) Clinical Classification is Class II-IV 4. Myasthenia Gravis activities of daily living (MG-ADL) total score is > 6 5. Individual has failed treatment over 1 year or more with ONE of the following: Use of 2 or more immunosuppressive therapies either in combination or as monotherapy Use of 1 immunosuppressive therapy and required ANY of the following: a. Chronic plasmapheresis b. Plasma c. Intravenous immunoglobulin (IVIG) Continuation of Soliris for the treatment of adults with gmg is considered medically necessary with documentation of ALL of the following: 1. MD-ADL of 3-points or more over baseline is achieved or maintained 2. Quantitative Myasthenia Gravis (QMG) test for muscle weakness of 5-points or more over baseline is achieved or maintained O938.4.docx Page 4 of 14

5 Criteria: (cont.) Soliris for all other indications not previously listed or if above criteria not met is considered experimental or investigational based upon: 1. Lack of final approval from the Food and Drug Administration, and 2. Insufficient scientific evidence to permit conclusions concerning the effect on health outcomes, and 3. Insufficient evidence to support improvement of the net health outcome, and 4. Insufficient evidence to support improvement of the net health outcome as much as, or more than, established alternatives, and 5. Insufficient evidence to support improvement outside the investigational setting. These indications include, but are not limited to: Treatment with dosing or frequency outside the FDA-approved dosing and frequency Resources: Literature reviewed 03/20/18. We do not include marketing materials, poster boards and nonpublished literature in our review. 1. Jaretzki A, 3rd, Barohn RJ, Ernstoff RM, et al. Myasthenia gravis: recommendations for clinical research standards. Task Force of the Medical Scientific Advisory Board of the Myasthenia Gravis Foundation of America. Neurology. Jul ;55(1): Muppidi S, Wolfe GI, Conaway M, Burns TM, Mg C, Mg-Qol15 Study G. MG-ADL: still a relevant outcome measure. Muscle Nerve. Nov 2011;44(5): Myasthenia Gravis Foundation of America Inc., Barohn RJ, Herbelin L. The Quantitative Myasthenia Gravis (QMG) Test: The Manual. 03/25/ National Cancer Institute. Paroxysmal nocturnal hemoglobinuria. Accessed 03/16/ National Institutes of Health. Atypical hemolytic uremic syndrome. Accessed 03/16/2015. O938.4.docx Page 5 of 14

6 Resources: (cont.) Soliris Package Insert: - FDA-approved indication and dosage: Indication Paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis Recommended Dose For intravenous use only. 600 mg weekly for the first 4 weeks, followed by 900 mg for the fifth dose 1 week later, then 900 mg every 2 weeks thereafter Soliris should be administered at the recommended dosage regimen time points, or within two days of these time points. The safety and effectiveness of Soliris for the treatment of PNH in pediatric patients have not been established. O938.4.docx Page 6 of 14

7 Resources: (cont.) Soliris Package Insert: (cont.) - FDA-approved indication and dosage: (cont.) Indication Atypical hemolytic uremic syndrome (ahus) to inhibit complement-mediated thrombotic microangiopathy Recommended Dose For intravenous infusion use only. For patients 18 years of age and older, Soliris therapy consists of: 900 mg weekly for the first 4 weeks, followed by 1200 mg for the fifth dose 1 week later, then 1200 mg every 2 weeks thereafter. For patients less than 18 years of age, administer Soliris based upon body weight, according to the following schedule: Patient Body Induction Weight 40 kg and over 900 mg weekly x 4 doses 30 kg to less than 40 kg 20 kg to less than 30 kg 10 kg to less than 20 kg 5 kg to less than 10 kg 600 mg weekly x 2 doses 600 mg weekly x 2 doses 600 mg weekly x 1 dose 300 mg weekly x 1 dose Maintenance 1200 mg at week 5; then 1200 mg every 2 weeks 900 mg at week 3; then 900 mg every 2 weeks 600 mg at week 3; then 600 mg every 2 weeks 300 mg at week 2; then 300 mg every 2 weeks 300 mg at week 2; then 300 mg every 3 weeks Soliris should be administered at the recommended dosage regimen time points, or within two days of these time points. Soliris is not indicated for the treatment of patients with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS). The safety and effectiveness of Soliris for the treatment of ahus have been established in pediatric patients. O938.4.docx Page 7 of 14

8 Resources: (cont.) Soliris Package Insert: (cont.) - FDA-approved indication and dosage: (cont.) Indication Atypical hemolytic uremic syndrome (ahus) to inhibit complement-mediated thrombotic microangiopathy (cont.) Recommended Dose For intravenous infusion use only. Supplemental dosing of Soliris is required in the setting of concomitant support with PE/PI (plasmapheresis ; or fresh frozen plasma infusion) Type of Intervention Plasmapheresis Fresh frozen plasma infusion Most Recent Soliris Dose Supplemental Soliris Dose with Each PE/PI Intervention 300 mg 300 mg per each plasmapheresis session 600 mg 600 mg per each plasmapheresis session 300 mg or more 300 mg per infusion of fresh frozen plasma Timing of Supplemental Soliris Dose Within 60 minutes after each plasmapheresis 60 minutes prior to each infusion of fresh frozen plasma Soliris is not indicated for the treatment of patients with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS). The safety and effectiveness of Soliris for the treatment of ahus have been established in pediatric patients. O938.4.docx Page 8 of 14

9 Resources: (cont.) Soliris Package Insert: (cont.) - FDA-approved indication and dosage: (cont.) Indication Adult individuals with generalized Myasthenia Gravis (gmg) who are antiacetylcholine receptor (AchR) antibody positive. Recommended Dose For intravenous infusion use only. For patients with generalized Myasthenia Gravis, Soliris therapy consists of: 900 mg weekly for the first 4 weeks, followed by 1200 mg for the fifth dose 1 week later, then 1200 mg every 2 weeks thereafter. Soliris should be administered at the recommended dosage regimen time points, or within two days of these time points. Supplemental dosing of Soliris is required in the setting of concomitant support with PE/PI (plasmapheresis ; or fresh frozen plasma infusion) Type of Intervention Plasmapheresis Fresh frozen plasma infusion Most Recent Soliris Dose Supplemental Soliris Dose with Each PE/PI Intervention 300 mg 300 mg per each plasmapheresis session 600 mg or more 300 mg or more 600 mg per each plasmapheresis session 300 mg per infusion of fresh frozen plasma Timing of Supplemental Soliris Dose Within 60 minutes after each plasmapheresis 60 minutes prior to each infusion of fresh frozen plasma O938.4.docx Page 9 of 14

10 Resources: (cont.) Initial Approval Duration: 6 months Renewal Approval Duration: 12 months Myasthenia Gravis Foundation of America (MGFA) Clinical Classification: Class I II IIa IIb III IIIa IIIb IV IVa IVb V Clinical symptoms Any ocular muscle weakness May have weakness of eye closure All other muscle strength is normal Mild weakness affecting other than ocular muscles May also have ocular muscle weakness of any severity Predominantly affecting limb, axial muscles, or both May also have lesser involvement of oropharyngeal muscles Predominantly affecting oropharyngeal, respiratory muscles, or both May also have lesser or equal involvement of limb, axial muscles, or both Moderate weakness affecting other than ocular muscles May also have ocular muscle weakness of any severity Predominantly affecting limb, axial muscles, or both May also have lesser involvement of oropharyngeal muscles Predominantly affecting oropharyngeal, respiratory muscles, or both May also have lesser or equal involvement of limb, axial muscles, or both Severe weakness affecting other than ocular muscles May also have ocular muscle weakness of any severity Predominantly affecting limb, axial muscles, or both May also have lesser involvement of oropharyngeal muscles Predominantly affecting oropharyngeal, respiratory muscles, or both May also have lesser or equal involvement of limb, axial muscles, or both Intubation, with or without mechanical ventilation, except when employed during routine post-operative management. The use of a feeding tube without intubation places the individual in Class IVb O938.4.docx Page 10 of 14

11 Resources: (cont.) Myasthenia Gravis Specific Activities of Daily Living Scale (MG-ADL): Grade Score 1. Talking Normal Intermittent slurring of nasal speech Constant slurring or nasal, but can be understood Difficult to understand speech 2. Chewing Normal Fatigue with solid Fatigue with soft food Gastric tube food 3. Swallowing Normal Rare episodes of choking 4. Breathing Normal Shortness of breath with exertion 5. Impairment of ability to brush teeth or comb hair 6. Impairment of ability to arise from chair None None Extra effort, but no rest periods needed Mild, sometimes uses arms 7. Double vision None Occurs, but not daily 8. Eyelid droop None Occurs but not daily Frequent choking necessitating changes in diet Shortness of breath at rest Rest periods needed Moderate, always uses arms Daily, but not constant Daily but not constant Gastric tube Ventilator dependence Cannot do one of the functions Severe, requires assistance Constant Constant Total ADL Score from 1-8: Range is 0-24 O938.4.docx Page 11 of 14

12 Resources: (cont.) Myasthenia Gravis Foundation of America (MGFA) Quantitative Myasthenia Gravis (QMG) Test: Test item weakness None Mild Moderate Severe Item Score: Score 1. Double vision (lateral gaze) sec Spontaneous 2. Ptosis (upward gaze) sec Spontaneous 3. Facial muscles Normal lid closure Complete, weak, some Complete, without resistance Incomplete resistance 4. Swallowing 4 oz water Normal Minimal coughing or throat clearing Severe coughing, choking or nasal regurgitation Cannot swallow, test not done 5. Speech following counting aloud None at Dysarthria at Dysarthria at #10-29 Dysarthria at #9 from 1-50 (onset of dysarthria) #50 # Right arm outstretched ( sitting) sec. 7. Left arm outstretched ( sitting) sec. 8. Forced vital capacity > 80% 65-79% 50-64% < 50% 9. Right hand grip (kg) Male Female 10. Left hand grip (kg) Male Female > 45 > 30 > 35 > Head, lifted (45% supine) sec Right leg outstretched (45-50% supine) sec 13. Left leg outstretched (45-50% supine) sec Total item score from 1-13: Range is O938.4.docx Page 12 of 14

13 Non-Discrimination Statement: Blue Cross Blue Shield of Arizona (BCBSAZ) complies with applicable Federal civil rights laws and does not discriminate on the basis of race, color, national origin, age, disability or sex. BCBSAZ provides appropriate free aids and services, such as qualified interpreters and written information in other formats, to people with disabilities to communicate effectively with us. BCBSAZ also provides free language services to people whose primary language is not English, such as qualified interpreters and information written in other languages. If you need these services, call (602) for Spanish and (877) for all other languages and other aids and services. If you believe that BCBSAZ has failed to provide these services or discriminated in another way on the basis of race, color, national origin, age, disability or sex, you can file a grievance with: BCBSAZ s Civil Rights Coordinator, Attn: Civil Rights Coordinator, Blue Cross Blue Shield of Arizona, P.O. Box 13466, Phoenix, AZ , (602) , TTY/TDD (602) , crc@azblue.com. You can file a grievance in person or by mail or . If you need help filing a grievance BCBSAZ s Civil Rights Coordinator is available to help you. You can also file a civil rights complaint with the U.S. Department of Health and Human Services, Office for Civil Rights electronically through the Office for Civil Rights Complaint Portal, available at or by mail or phone at: U.S. Department of Health and Human Services, 200 Independence Avenue SW., Room 509F, HHH Building, Washington, DC 20201, , (TDD). Complaint forms are available at Multi-Language Interpreter Services: O938.4.docx Page 13 of 14

14 Multi-Language Interpreter Services: (cont.) O938.4.docx Page 14 of 14