Appeal type: Medical necessity
Decision: Overturned

Diagnosis:  Cancer.
Treatment:  Lynparza.
 
The insurer denied Lynparza.  The denial was overturned.

This is a patient who has diagnoses of pancreatic cancer stage IV, chronic lymphocytic leukemia of B-cell type in remission, type 1 diabetes mellitus, and iron deficiency anemia.  After treatment with Oxaliplatin, irinotecan, and Xeloda, Lynparza was prescribed based on FoundationOne.  According to FoundationOne results the patient has presence of an ATM mutation.

Per Golan T, et al. (2019), "Of the 3315 patients who underwent screening, 154 underwent randomization and were assigned to a trial intervention (92 to receive olaparib and 62 to receive placebo).  The median progression-free survival was significantly longer in the olaparib group than in the placebo group (7.4 months vs. 3.8 months; hazard ratio for disease progression or death, 0.53; 95% confidence interval [CI], 0.35 to 0.82; P =0.004).  An interim analysis of overall survival, at a data maturity of 46%, showed no difference between the olaparib and placebo groups (median, 18.9 months vs. 18.1 months; hazard ratio for death, 0.91; 95% CI, 0.56 to 1.46; P = 0.68).  There was no significant between-group difference in health-related quality of life, as indicated by the overall change from baseline in the global quality-of-life score (on a 100-point scale, with higher scores indicating better quality of life) based on the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (between-group difference, -2.47 points; 95% CI, -7.27 to 2.33).  The incidence of grade 3 or higher adverse events was 40% in the olaparib group and 23% in the placebo group (between-group difference, 16 percentage points; 95% CI, -0.02 to 31); 5% and 2% of the patients, respectively, discontinued the trial intervention because of an adverse event.  Conclusions: Among patients with a germline BRCA mutation and metastatic pancreatic cancer, progression-free survival was longer with maintenance olaparib [Lynparza] than with placebo."

Orphan Drug Designation (ODD) status was granted for the treatment of ovarian cancer in October 2013.  Earlier this year an amended ODD status was granted to include both fallopian tube and primary peritoneal cancers following the expanded US approval of Lynparza in August 2017 for the maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube or primary peritoneal cancer, who are in a complete or partial response to platinum-based chemotherapy.  The FDA grants ODD status to medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US. (AstraZeneca., 2018)

Phase III POLO trial of Lynparza in pancreatic cancer was reported in July 2019.  It showed a statistically significant and clinically meaningful improvement in progression-free survival (PFS) as first-line maintenance therapy alone in patients with germline BRCA-mutated (gBRCAm) metastatic adenocarcinoma of the pancreas in patients who haven't responded to platinum-based chemotherapy (Kindler HL, et al., 2019).                  

The health care plan did not act reasonably and with sound medical judgment and in the best interest of the patient.      

In this case, the patient has pancreatic cancer stage IV.  He has tried Oxaliplatin, irinotecan, and Xeloda. He has a ATM mutation.  The last magnetic resonance imaging (MRI) showed progression. Lynparza is appropriate in this clinical scenario.  Therefore, Lynparza is considered medically necessary for this patient.
                                   
Based on the above, the medical necessity for coverage for Lynparza is substantiated.  The insurer's denial is overturned.