CLINICAL POLICY Department: Medical Management Document Name: Opdivo Reference Number: CP.PHAR.121 Effective Date: 07/15



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Page: 1 of 6 IMPORTANT REMINDER This Clinical Policy has been developed by appropriately experienced and licensed health care professionals based on a thorough review and consideration of generally accepted standards of medical practice, peerreviewed medical literature, government agency/program approval status, and other indicia of medical necessity. The purpose of this Clinical Policy is to provide a guide to medical necessity. Benefit determinations should be based on the applicable contract provisions governing plan benefits ( Benefit Plan Contract ) and applicable state and federal requirements, as well as applicable plan-level administrative policies and procedures. To the extent there are any conflicts between this Clinical Policy and the Benefit Plan Contract provisions, the Benefit Plan Contract provisions will control. Clinical policies are intended to be reflective of current scientific research and clinical thinking. This Clinical Policy is not intended to dictate to providers how to practice medicine, nor does it constitute a contract or guarantee regarding payment or results. Providers are expected to exercise professional medical judgment in providing the most appropriate care, and are solely responsible for the medical advice and treatment of members. Subject Medical necessity criteria for Opdivo (nivolumab) Description The intent of the criteria is to ensure that patients follow selection elements established by Centene medical policy for Opdivo. FDA-Approved Indications and Limitations of Use 1 Unresectable or Metastatic Melanoma Opdivo is indicated for the treatment of patients with unresectable or metastatic melanoma and disease progression following ipilimumab and, if BRAF V600 mutation positive, a BRAF inhibitor. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. Metastatic Squamous n-small Cell Lung Cancer Opdivo is indicated for the treatment of patients with metastatic squamous non-small cell lung cancer (NSCLC) with progression on or after platinum-based chemotherapy. Limitations of Use: The safety and effectiveness of Opdivo have not been established in pediatric patients.

Page: 2 of 6 Policy/Criteria It is the policy of health plans affiliated with Centene Corporation that Opdivo is medically necessary for members meeting the following criteria: Figure 1: Opdivo Algorithm Figure 1: Opdivo Algorithm Currently receiving through a Centene benefit? Experiencing unacceptable toxicity (App. B) or disease progression? Approve for 6 months Age 18 years? Unresectable or metastatic melanoma What is the diagnosis? Other Metastatic Squamous NSCLC Tested for BRAF V600 mutation? Had progression on or after platinumbased chemotherapy (App. A)? BRAF V600 mutation positive? Experienced disease progression following ipilimumab therapy? Currently receiving prescribed regimen? Approve for 3 months Experienced disease progressoin following a BRAF inhibitor therapy (e.g., Zelboarf, Tafinlar)? Experiencing unacceptable toxicity (App. B) or disease progression? Approve for 6 months

Page: 3 of 6 Background Opdivo mechanism of action Binding of the PD-1 ligands, PD-L1 and PD-L2, to the PD-1 receptor found on T cells inhibits T- cell proliferation and cytokine production. 1 Upregulation of PD-1 ligands occurs in some tumors and signaling through this pathway can contribute to inhibition of active T-cell immune surveillance of tumors. 1 Opdivo is a human immunoglobulin G4 (IgG4) monoclonal antibody that binds to the PD-1 receptor and blocks its interaction with PD-L1 and PD-L2, releasing PD-1 pathway-mediated inhibition of the immune response, including the anti-tumor immune response. 1 In syngeneic mouse tumor models, blocking PD-1 activity resulted in decreased tumor growth. 1 Unresectable or metastatic melanoma Opdivo is indicated for the treatment of patients with unresectable or metastatic melanoma and disease progression following ipilimumab and, if BRAF V600 mutation positive, a BRAF inhibitor. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. Melanoma accounts for less than 2% of skin cancer cases but causes a large majority of skin cancer deaths. 2 The American Cancer Society estimates that in 2015 approximately 73,870 new melanomas will be diagnosed and 9,940 deaths will occur in the United States as a result of melanoma. 2 The median age at diagnosis of melanoma is 59 years. 3 The American Joint Committee of Cancer (AJCC) categorizes patients with melanoma into three groups: 1) localized disease with no evidence of metastases (stage I-II), 2) regional disease (stage III), and 3) distant metastatic disease (stage IV). 3 It is estimated that 82% to 85% of patients with melanoma present with localized disease, 10% to 13% with regional disease, and 2% to 5% with distant metastatic disease. Depending on the stage at presentation, five-year survival rates range from 10% to 90%. 3 Treatment options for melanoma include surgery, radiation and systemic therapy. 3 Surgical excision plays a prominent role in earlier stages of melanoma while systemic therapy and radiation become relatively more central for patients with unresectable or metastatic melanoma. 3 In its March, 2015, melanoma treatment guidelines, the National Comprehensive Cancer Network (NCCN) added Opdivo to its list of preferred regimens for advanced or metastatic melanoma. Additionally, Opdivo s FDA-approved indication for advanced melanoma includes cases that also are BRAF V600 mutation positive. 1 Approximately half of patients with metastatic melanoma have an activating mutation of the intracellular signaling kinase, BRAF. 3 BRAF V600 mutations account for the majority of BRAF mutations found in all patients with cancer. 4 Metastatic Squamous NSCLC Opdivo is indicated for the treatment of patients with metastatic squamous NSCLC with progression on or after platinum-based chemotherapy (e.g., cisplatin, carboplatin). 1

Page: 4 of 6 Lung cancer is the leading cause of cancer death in the United States. 5 In 2015, an estimated 221,200 new cases of lung and bronchial cancer will be diagnosed, and 158,040 deaths are estimated to occur because of the disease. 5 Only 16.8% of all patients with lung cancer are alive five years or more after diagnosis. 5 Lung cancer is divided into two classes based on its biology, therapy and prognosis: NSCLC which accounts for 85% of all lung cancer cases, and small cell lung cancer. 5 NSCLC includes two major types: 1) non-squamous carcinoma (including adenocarcinoma, large-cell carcinoma, and other cell types); and 2) squamous cell (epidermoid) carcinoma. 5 Adenocarcinoma is the most common type of lung cancer seen in the U.S., including among non-smokers. 5 Areas of focus in the diagnosis and approach to lung cancer include minimally invasive techniques for diagnosis and treatment, advances in radiation therapy, and development of targeted therapies such as Opdivo. 1,5 Safety The Opdivo package insert lists no contraindications. 1 Examples of platinum-based chemotherapy regimens are presented at Appendix A. Conditions associated with Opdivo use for which permanent discontinuation is recommended are outlined in Appendix B. Opdivo warnings and precautions are listed in Appendix C. The package insert contain additional information on dose adjustments, drug/drug interactions and use in special populations. Appendices Appendix A: Examples of Platinum-based Chemotherapy Regimens for NSCLC 5 Carboplatin (platinum agent)/paclitaxel Cisplatin (platinum agent)/paclitaxel Appendix B: Immune Mediated Adverse Reactions for which Permanent Discontinuation is Recommended 1 Severe (Grade 3) or life-threatening (Grade 4) pneumonitis Grade 4 colitis or for recurrent colitis upon restarting Opdivo Severe (Grade 3) or life-threatening (Grade 4) immune-mediated hepatitis Life-threatening (Grade 4) serum creatinine elevation Severe (Grade 3) or moderate (Grade 2) serum creatinine elevation if worsening or no improvement after withholding Opdivo and administering corticosteroids Appendix C: Opdivo - Warnings and Precautions 1 Adverse Reaction Warning and Precautions Monitor patients for signs and symptoms of pneumonitis. Administer pneumonitis corticosteroids at a dose of 1 to 2 mg/kg/day prednisone equivalents for Grade 2 or greater pneumonitis, followed by corticosteroid taper. Permanently discontinue Opdivo for severe (Grade 3) or life-threatening

Page: 5 of 6 Adverse Reaction colitis hepatitis nephritis and renal dysfunction hypothyroidism and hyperthyroidism Other immunemediated adverse Reactions Warning and Precautions (Grade 4) pneumonitis and withhold Opdivo until resolution for moderate (Grade 2) pneumonitis. Monitor patients for immune-mediated colitis. Administer corticosteroids at a dose of 1 to 2 mg/kg/day prednisone equivalents followed by corticosteroid taper for severe (Grade 3) or life-threatening (Grade 4) colitis. Administer corticosteroids at a dose of 0.5 to 1 mg/kg/day prednisone equivalents followed by corticosteroid taper for moderate (Grade 2) colitis of more than 5 days duration; if worsening or no improvement occurs despite initiation of corticosteroids, increase dose to 1 to 2 mg/kg/day prednisone equivalents. Withhold Opdivo for Grade 2 or 3 immune-mediated colitis. Permanently discontinue Opdivo for Grade 4 colitis or for recurrent colitis upon restarting Opdivo Monitor patients for abnormal liver tests prior to and periodically during treatment. Administer corticosteroids at a dose of 1 to 2 mg/kg/day prednisone equivalents for Grade 2 or greater transaminase elevations, with or without concomitant elevation in total bilirubin. Withhold Opdivo for moderate (Grade 2) and permanently discontinue Opdivo for severe (Grade 3) or life-threatening (Grade 4) immune-mediated hepatitis. Monitor patients for elevated serum creatinine prior to and periodically during treatment. Administer corticosteroids at a dose of 1 to 2 mg/kg/day prednisone equivalents followed by corticosteroid taper for lifethreatening (Grade 4) serum creatinine elevation and permanently discontinue Opdivo. For severe (Grade 3) or moderate (Grade 2) serum creatinine elevation, withhold Opdivo and administer corticosteroids at a dose of 0.5 to 1 mg/kg/day prednisone equivalents followed by corticosteroid taper; if worsening or no improvement occurs, increase dose of corticosteroids to 1 to 2 mg/kg/day prednisone equivalents and permanently discontinue Opdivo. Monitor thyroid function prior to and periodically during treatment. Administer hormone replacement therapy for hypothyroidism. Initiate medical management for control of hyperthyroidism. There are no recommended dose adjustments of Opdivo for hypothyroidism or hyperthyroidism. For any suspected immune-mediated adverse reactions, exclude other causes. Based on the severity of the adverse reaction, withhold Opdivo, administer high-dose corticosteroids, and if appropriate, initiate hormonereplacement therapy. Upon improvement to Grade 1 or less, initiate corticosteroid taper and continue to taper over at least 1 month. Consider restarting Opdivo after completion of corticosteroid taper based on the

Page: 6 of 6 Adverse Reaction Embryo fetal toxicity Warning and Precautions severity of the event. Based on its mechanism of action and data from animal studies, Opdivo can cause fetal harm when administered to a pregnant woman. In animal reproduction studies, administration of nivolumab to cynomolgus monkeys from the onset of organogenesis through delivery resulted in increased abortion and premature infant death. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with Opdivo and for at least 5 months after the last dose of Opdivo. References 1. Opdivo [package insert]. Princeton, NJ: Bristol-Myers Squibb; March 2015. 2. American Cancer Society. What are key statistics about melanoma skin cancer? Available at: http://www.cancer.org. Accessed June 2015. 3. The National Comprehensive Cancer Network. The NCCN Clinical Practice Guidelines in Oncology: Melanoma (Version 3.2015). Available at: http://www.nccn.org. Accessed June 2015. 4. Flaherty KT, Robert C, Hersey P, et al. Improved Survival with MEK Inhibition in BRAF- Mutated Melanoma. N Engl J Med.2012;367(2):107-114. 5. National Comprehensive Cancer Network. NCCN Clinical Practice Guidelines in Oncology: n-small Cell Lung Cancer (Version 7. 2015). Available at http://www.nccn.org. Accessed June 2015. Revision Log Date 2014 Centene Corporation. All rights reserved. All materials are exclusively owned by Centene Corporation and are protected by United States copyright law and international copyright law. part of this publication may be reproduced, copied, modified, distributed, displayed, stored in a retrieval system, transmitted in any form or by any means, or otherwise published without the prior written permission of Centene Corporation. You may not alter or remove any trademark, copyright or other notice contained herein. Centene and Centene Corporation are registered trademarks exclusively owned by Centene Corporation.