Appeal type: Experimental/Investigational
Decision: Upheld

Diagnosis: Medulloblastoma

Treatment: Ibrance 75 milligram (mg) tab (tablet)

The insurer denied coverage for Ibrance 75 milligram (mg) tab.

The denial is upheld.

The patient is a child who has been diagnosed with medulloblastoma. The patient's medulloblastoma is a recurrent disease after chemotherapy, radiation, and surgical resection. Molecular testing of the tumor predicts response to Ibrance (palbociclib). The treating provider is requesting plan approval for treatment with Ibrance (palbociclib) 75 milligram (mg) tablets.

According to the clinical documentation provided for review, the patient has been diagnosed with medulloblastoma. The patient's medulloblastoma is a recurrent disease after chemotherapy, radiation, and surgical resection. Molecular testing of the tumor predicts response to Ibrance (palbociclib). The patient had a large posterior fossa mass with obstructive hydrocephalus that was discovered on magnetic resonance imaging (MRI).

Ibrance (palbociclib) 75 milligram (mg) tablets is not Food and Drug Administration (FDA) approved for the treatment of medulloblastoma. 

Millard et al. state, "Medulloblastoma accounts for nearly 10% (percent) of all childhood brain tumors. These tumors occur exclusively in the posterior fossa and have the potential for leptomeningeal spread. Treatment includes a combination of surgery, radiation therapy (in patients > 3 years old). Patients > 3 years old are stratified based on the volume of postoperative residual tumor and the presence or absence of metastases into "standard risk" and "high risk" categories with long term survival rates of approximately 85% and 70%, respectively. Outcomes are inferior in infants and children younger than 3 years with exception of those patients with the MBEN [ Medulloblastoma with extensive nodularity] histologic subtype. Treatment for medulloblastoma is associated with significant morbidity, especially in the youngest patients. Recent molecular subclassification of medulloblastoma has potential prognostic and therapeutic implications. Future incorporation of molecular subgroups into treatment protocols will hopefully improve both survival outcomes and post-treatment quality of life."                

Ibrance 75 milligram (mg) tab for the treatment of medulloblastoma needs final approval from the appropriate regulatory bodies for this diagnosis. It is not FDA approved for the treatment of medulloblastoma.     

This patient is not a good candidate for Ibrance 75 milligram (mg) tab for the treatment of medulloblastoma.  Ibrance 75 milligram (mg) tab for the treatment of medulloblastoma is not the best available treatment for this patient at this time.  There are adverse risks increased over standard treatment and/or services.

Ibrance for the treatment of this patient's clinical circumstance is not likely to be more beneficial than any other standard treatments as the literature does not yet support a result of improvement in health outcome. Ibrance, a selective cyclin-dependent kinase 4/6 (CDK4/6) inhibitor in pediatric patients with progressive/refractory brain tumors with intact retinoblastoma protein has been studied in a phase I trial in pediatrics. A total of 21 patients were enrolled. Although the drug was well tolerated, there were no objective responses.      

Based on the above, Ibrance 75 milligram (mg) tab for the treatment of medulloblastoma  is upheld.