Diagnosis: Atypical Teratoid Rhabdoid Teratoma (AT/RT).
treatment: Tazverik (tazemetostat).

The insurer denied Tazverik (tazemetostat). The health plan's determination is overturned.

The patient is a male who presented for care with a 5 centimeter (cm) cystic/solid minimally enhancing mass in the 4th ventricle/left foramen of Luschka with heterogeneous restricted diffusion, and multifocal leptomeningeal dissemination. He had a stereotactic guided subtotal resection and the pathology was consistent with atypical teratoid rhabdoid tumor (ATRT). He was started on treatment as per the Children's Oncology Group (COG) protocol. Stem collection happened and she started the second induction. A lumbar puncture was performed that showed continued rare tumor cells, and the magnetic resonance imaging (MRI) of the brain and spine showed increased ventriculomegaly with diffuse leptomeningeal spread but no definite tumor progression at the primary site. The auto-transplantation was completed. The provider is now recommending treatment with Tazverik (Tazemetostat).

The health plan, in its determination of medical necessity, did not act reasonably, with sound medical judgment, and in the best interest of the patient.

Yes, the requested health service/treatment of Tazemetostat is medically necessary for this patient. This patient has a likely terminal brain tumor with her ATRT with leptomeningeal spread. There are currently no clear Food and Drug Administration (FDA) approved treatments for this type of cancer, and historically, this disease has almost been uniformly fatal. The provider has recommended treatment with Tazemetostat, a selective enhancer of zeste homolog 2 (EZH2) inhibitor, noting that it has previously shown at least some antitumor activity in ATRT cases. As per a recent review of this topic, epigenetic altering drugs such as Histone Deacetylase (HDAC) inhibitors, Panobinostat and Tazemetostat and Ribavirin may restore epigenetic balance to ATRT. There is in vivo mouse data to support this therapy, and the dosing of this product has previously been determined in pediatric patients.

Given the rarity of this likely terminal disease using the current therapies, and no FDA approved medications for this type of cancer, the use of Tazemetostat is clinically appropriate and has a possibility of being at least somewhat effective. There are no other standard treatment options at this time. Therefore, this request should be approved as medically necessary at this time.

The health plan's determination of medical necessity is overturned in whole.