Appeal type: Experimental/Investigational
Decision: Upheld

Diagnosis: Stage IIIc T3 N1 M0 (stage 3 tumor, in lymph node - stage 1, not metastasized) Rectal Adenocarcinoma
Treatment: Proton Beam Radiation Therapy
The insurer denied the Proton Beam Radiation Therapy.
The denial is upheld. 

This is a man with stageIIIc T3N1M0  (stage 3 tumor, in lymph node - stage 1, not metastasized) 4.9 cm adenocarcinoma of rectum at 6 cm from the anal verge. Proton beam therapy of 25 Gy (gray unit of radiation) in 5 fractions is being requested for pre-operative radiation therapy. 

No, the health plan should not cover Proton Beam Therapy.

The ASTRO (American Society for Radiation Oncology) Proton Beam Therapy Model Policy states that Proton beam therapy is an advanced type of external-beam radiation therapy that uses proton rather than photon beams to deliver radiation doses to a tumor. Proton therapy offers a high degree of precision, which allows radiation oncologists to target an escalated dose of radiation directly on a tumor and spare nearby healthy tissue. Proton therapy is particularly beneficial for patients with tumors that are near critical structures, such as lung tumors near the heart.

Based on new evidence published since the original policy was issued, the updated model policy outlines two categories of appropriate clinical indications, or diagnoses, for proton beam therapy. For indications in Group 1, coverage is recommended; for Group 2, coverage is recommended if additional requirements (outlined below) are met. Disease sites were assigned to one of the two categories based on evidence in published clinical data and current Medicare coverage.

Group 1 indications, or the clinical scenarios that frequently support the use of proton therapy based on medical necessity and published clinical data, were updated with five additions and one modification. Group 1 indications, with additions marked by asterisks, include:
1) Malignant and benign primary central nervous system (CNS) tumors* 2) Advanced (e.g., T4 [stage 4]) and/or unresectable head and neck cancers* 3) Cancers of the paranasal sinuses and other accessory sinuses* 4) Non-metastatic retroperitoneal sarcomas* 5) Reirradiation cases where cumulative critical structure dose would exceed tolerance dose* 6) Hepatocellular cancer (no longer required to be treated in a hypofractionated regimen*) 7) Ocular tumors, including intraocular melanomas 8) Tumors that approach or are located at the base of skull, including but not limited to chordoma and chondrosarcomas 9) Primary or metastatic tumors of the spine where the spinal cord tolerance may be exceeded with conventional treatment or where the spinal cord has previously been irradiated 10) Primary or benign solid tumors in children treated with curative intent and occasional palliative treatment of childhood tumors when one of the criteria noted above apply 11) Patients with genetic syndromes making total volume of radiation minimization crucial, such as but not limited to NF-1 (neurofibromatosis) patients and retinoblastoma patients.

The policy recommends coverage for Group 2 indications if the patient is enrolled in either an Institutional Research Board (IRB)-approved study or in a multi-institutional registry adhering to Medicare requirements for Coverage with Evidence Development (CED). 

These indications also represent the disease sites for which evidence is accumulating and may support future Group 1 coverage. While the policy specifies that no indications are deemed inappropriate for CED, it also specifies several systems for Group 2 indications:
1) Non-T4 (stage 4) and resectable head and neck cancers (previously all head and neck malignancies*) 2) Non-metastatic prostate cancer (previously grouped with genitourinary carcinomas*) 3) Breast cancer* 4) Thoracic malignancies, including non-metastatic primary lung and esophageal cancers 5) Abdominal malignancies, including non-metastatic primary pancreatic, biliary and adrenal cancers 6) Pelvic malignancies, including non-metastatic rectal, anal, bladder and cervical cancers.

Medical documents do not indicate that proton beam radiation therapy for this member's rectal cancer is provided on a registry trial or therapeutic clinical trial. Therefore, proton beam radiation therapy in this case does not meet the ASTRO (American Society for Radiation Oncology) policy for proton beam radiation therapy.

NCCN (National Comprehensive Cancer Network) guideline for rectal cancer does not include proton beam therapy as standard modality for pre-op radiation therapy of rectal cancer.

No, proton beam therapy is not likely is not likely to be more beneficial than any standard treatment or treatments for the insured's condition or disease.

Proton beam radiation therapy (PBT) is an emerging treatment modality for several types of cancer due to its advantage of physical dose distribution (Bragg Peak) over photon radiotherapy. Proton beam radiation therapy can deliver sharp dose gradient between target and normal tissues. However, most published studies on proton beam RT (radiation therapy) for rectal cancer are physics/dosimetric studies comparing proton beam radiation therapy to photon based 3-D (three dimensional) or intensity-modulated radiotherapy.

Currently, published clinical studies on proton beam radiation therapy for rectal cancer are limited to a few single institutional uncontrolled retrospective studies with a small number of heterogeneous patient population with no clinical data for local control, survival, toxicities relative to photon radiation therapy.

These publications do not provide high grade clinical outcome evidence from prospective trials to demonstrate that the proton beam radiation therapy (PBT) is more likely to benefit than photon radiation therapy for pre-operative RT (radiation therapy) for rectal cancer.