Cancer
Pharmacy/Prescription Drug

Diagnosis: Bilateral Optic Nerve Tumors
Treatment: Mekinist 0.5 MG OR TABS
The insurer denied Mekinist 0.5 MG OR TABS.
The health plan's determination is overturned.

The patient is a female with a diagnosis with optic pathway glioma and diencephalic syndrome. She has been treated with standard of care chemotherapy, including carboplatin/vincristine and weekly vinblastine. She has progressive disease effecting vision and pituitary function. Her tumor was positive for BRAF-KIAA1549 fusion, a common molecular abnormality in pediatric low-grade gliomas.

Mekinist 0.5MG OR TABS is likely to be more beneficial than any standard treatment or treatments for the insured's life threatening or disabling condition or disease.
There is no clear standard of care for multiple recurrent pediatric low-grade gliomas. MEK inhibitors have been tested in multiple prior and ongoing trials and have had very promising results, especially in tumors harboring BRAF fusion abnormalities. They are oral agents and very well tolerated. This patient has limited treatment options for a life-threatening disease. She has tried and failed all other more "standard" treatment options, and the use of Mekinist is supported by her tumor tissue and the current standards of care in the field. The use of Mekinist is supported by published data on its use as well as the ongoing clinical trial by Children's Oncology Group.