Appeal type: Medical Necessity
Decision: Overturned

Nature of Statutory Criteria/Case Summary: The patient is a 63-year-old female who has been diagnosed with a hepatocellular mass and chronic hepatitis B. The provider noted that the patient underwent hepatocellular mass resection in 2009. She was treated with transarterial chemoembolization (TACE). The provider noted the patient did well until 2018 when a computed tomography (CT) scan showed a two cm right upper lobe mass, a lytic lesion, and multiple enlarged mediastinal lymph nodes. The provider noted a positron emission tomography (PET) scan obtained in July 2019 confirmed metastatic disease. She was found to have an epidermal growth factor receptor (EGFR) exon 19 mutation and was started on gefitinib in September 2019. An esophagogastroduodenoscopy (EGD) with biopsies of a duodenal mass obtained revealed a poorly differentiated adenocarcinoma. The cancer type identification from a liver biopsy was noted to be 89% cholangiocarcinoma. She was treated with afatinib on 2/15/20. A PET scan revealed significant progression of disease with a new right upper lobe lung nodule, increased mediastinal lymphadenopathy, and worsening liver metastases. Although the patient has been treated for unknown primary cancer and non-small cell lung cancer, the provider noted the patients clinical picture is more consistent with a cholangiocarcinoma, and treatment with pemigatinib was recommended based on her fibroblast growth factor receptor 2 (FGFR2) fusion. The patient has requested authorization and coverage for Pemazyre (pemigatinib, oral) 13.5 mg tablets. The Health Plan denied the request and reported the requested medication is not medically necessary for the treatment of this patient. At issue in this case is whether Pemazyre (pemigatinib, oral) 13.5 mg tablets are medically necessary for the treatment of the patients medical condition. The U.S. Food and Drug Administration (FDA) noted, Pemazyre is a kinase inhibitor indicated for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement as detected by an FDA-approved test. Researchers reported, FGFR 2 gene alterations are involved in the pathogenesis of cholangiocarcinoma. Pemigatinib is a selective, potent, oral inhibitor of FGFR1, 2, and 3. This study evaluated the safety and antitumor activity of pemigatinib in patients with previously treated, locally advanced or metastatic cholangiocarcinoma with and without FGFR2 fusions or rearrangements. The authors concluded, These data support the therapeutic potential of pemigatinib in previously treated patients with cholangiocarcinoma who have FGFR2 fusions or rearrangements. In this case, the patient has a complex oncological history. She has had at least two prior lines of therapy in terms of EFGR therapy. It is likely that she does have cholangiocarcinoma based on her prior pathology and molecular profiling. Therefore, the use of pemigatinib is reasonable. The medical evidence supports the requested medication in this clinical setting. Thus, Pemazyre (pemigatinib, oral) 13.5 mg tablets are medically necessary for the treatment of this patient.Final Result: The reviewer determined that the requested medication is medically necessary for treatment of the patients medical condition. Therefore, the Health Plans denial should be overturned.Credentials/Qualifications: The reviewer is board certified in internal medicine with sub-specialty certification in medical oncology and hematology and is actively practicing. The reviewer is an expert in the treatment of the enrollees medical condition and knowledgeable about the proposed treatment through recent or current actual clinical experience treating those with the same or a similar medical condition.