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 overturned.

This patient has a diagnosis of medulloblastoma. The patient was enrolled on Head Start 4. She received chemotherapy and radiation, and underwent craniectomy and resection. She has had recurrence, and underwent organotypic slice culture which is an unproven program. Palbociclib treatment showed the strongest drug-response. The plan is to start treatment with Irinotecan, temozolomide, and palbociclib/Ibrance. She received chemotherapy and radiation, and underwent craniectomy and resection. She has had recurrence and underwent organotypic slice culture. It was noted that Palbociclib treatment showed the strongest drug-response, and the treatment plan is to start treatment with Irinotecan, temozolomide, and palbociclib/Ibrance. 

As noted by the treating provider, given that there is no available standard of care in this current setting, it is ethical to provide the best treatment proposition based on the informed decision available data.  It is unknown if there will be adverse risks increased over standard treatment/services. However, given the patient's clinical situation, it is the best available treatment for this patient at this time.

"While leukemias are the most common type of malignancy to afflict the pediatric population, brain tumors are the most common solid tumors in this age group. Medulloblastoma is the most common malignant brain tumor in children constituting nearly 20% (percent) of all pediatric brain tumors. It is categorized as an embryonal neuroepithelial tumor of the cerebellum."

This is a high-grade tumor that has a propensity to spread via the cerebrospinal fluid.
Within the first few years of diagnosis, mortality approximates 15%; however, cure rates can reach as high as 60% with current therapeutic modalities. Surgical resection preceded and/or followed by radiation and chemotherapy is the mainstay of therapy, with five-year survival rates of between 50% to 90%. This wide range is multifactorial, owing in part to age at diagnosis, the presence of metastases at diagnosis, and a histologic variant of medulloblastoma. Regardless of long-term survival, treatment-related cognitive, neurologic, and endocrinologic effects remain a debilitating concern and an impetus for the search for further therapeutic modalities." (Mahapatra, S., & Amsbaugh, M. J. 2021).                                                    

There is supportive medical literature for the use of Ibrance 75 mg in this scenario.      Ibrance 75 milligram (mg) tab does not have final approval from the appropriate regulatory bodies for this diagnosis. However, even though it is not approved in this setting, there are supportive medical literature for its use, as there is no established standard of care in this advanced stage.     

Based on this data, the patient a good candidate for Ibrance 75 milligram (mg) tab for the treatment of medulloblastoma.

In this scenario, the patient a good candidate for Ibrance 75 milligram (mg) tab for the treatment of her condition, medulloblastoma.  Ibrance 75 milligram (mg) tab for the treatment of medulloblastoma is the best available treatment for this patient at this time. The requested treatment is likely to be more beneficial than any standard treatments.

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