Appeal type: Experimental/Investigational
Decision: Upheld

Physician 1: The patient is a 44-year-old premenopausal female. Shortly after the birth of her last child she experienced increasing weight loss, abdominal pain and increasing night sweats. Evaluation of her symptoms revealed Stage IV disease believed to be of gastrointestinal (GI) origin, though the exact primary was debatable. The patient sought treatment through her Health Plan and was advised that treatment was palliative in nature. The patient then sought treatment at the Burzynski clinic in May 2004. She was advised to consider a combination of agents approved for the treatment of GI primary, agents not FDA approved for the treatment of a GI malignancy (i.e., Herceptin), and nutritional supplements plus the utilization of sodium phenylbutyrate.The patient has experienced a questionable response, but has experienced significant thrombocytopenia. A request has been made for coverage for treatment with Dr. Burzynski. The Health Plan has denied the request on the basis that the requested therapy is considered experimental. There is no evidence to support the combination of agents utilized in this treatment regimen. While Xeloda, oxaliplatin, and Avastin are utilized in the management of GI cancer, the combination of these agents plus Herceptin is not conventional. Though there is a study investigating the combination of Herceptin and Avastin in breast cancer patients and the data seems promising, there is insufficient data to determine the efficacy of this therapy. Furthermore, the utilization of sodium phenylbutyrate in treatment of colorectal cancer is not considered standard treatment and remains of unproven efficacy in some studies. There is no data supporting the efficacy of the combination of drugs that has been utilized in the treatment of this patient. Thus, there is no evidence the requested therapy is likely to result in any benefit. Therefore, I have determined that the requested therapy is not likely to be more beneficial for treatment of the patients medical condition than any available standard therapy. The Health Plans denial should be upheld. Physician 2: The patient is a 44-year-old female who delivered her fourth child in November 2003. In early 2004, she developed progressive tiredness and weight loss. In May 2004, an ultrasound and then a CT scan of the abdomen revealed metastases in the liver and questionable prominence in the head of the pancreas. On 5/17/04, the patient had a liver biopsy revealing adenocarcinoma, most consistent with a colon primary. It was noted that she had very high levels of CEA, CA-125, and CA 19-9. There has never been a definitive primary identified.The patient apparently saw a local oncologist, but eventually sought consultation with Dr. Burzynski. His clinic began her on a chemotherapy program soon thereafter, which included oxaliplatin, Xeloda, Avastin, Herceptin, and sodium phenylbutyrate. A follow-up CT scan in October 2004 showed slight decrease in the size of the liver lesions. However, another scan in January 2005 revealed no further change in the liver lesions and a new finding of multiple lung metastases.A request has been made for coverage for Dr. Burzynskis treatment protocol. The Health Plan has denied this request on the basis that the requested therapy is considered experimental. Dr. Burzynskis treatment protocol includes several drugs that are considered standard for the treatment of metastatic colon cancer. Specifically, Xeloda, oxaliplatin and Avastin are often used in the treatment of patients such as this patient and are readily available. The additional elements at issue here, specifically, Herceptin and sodium phenylbutyrate, have thus far not been shown to have efficacy in colon cancer. Furthermore, it is very likely that the mild degree of improvement noted in the October CT scan was due to the use of the conventional and accepted drugs; there is no reason to believe that the addition of the other drugs added any benefit. In any case, the disease is clearly now progressive and the overall treatment has not been successful. There has been no convincing data to prove clinical benefit from this approach. Accordingly, I have determined that the requested therapy is not likely to be more beneficial for treatment of the patients medical condition than any available standard therapy. The Health Plans denial should be upheld. Physician 3: There is a lack of consensus regarding standard therapy for this clinical scenario. Combination chemotherapy is often used or recommended in patients with good performance status, and the chemotherapy regimen is often physician-tailored to the best guess primary or for very broad coverage. Existing studies are very difficult to interpret on a patient-to-patient basis due to the acknowledged heterogeneity of presentation and tumor origin in patients with AUP. Several regimens containing newer drugs appear to have more antitumor activity than older regimens. However, the best regimen has not been defined in prospective randomized trials. Based upon the information provided, this patients primary is quoted throughout the medical record as likely colonic in origin. The elevated markers could also be consistent with an upper GI origin, and the Her-2 expression (again presumed positive) could argue for occult breast cancer or for a tumor driven by unregulated Her-2 neu expression. The patients regimen includes agents known to be active in each of these scenarios, and, although an unusual combination, fits with the principal of very broad coverage in terms of potential activity. Though use of sodium phenylbutyrate and the vitamins and nutritional supplements does not have established efficacy in this setting, on the whole, the immuno-chemotherapy regimen prescribed for this young patient who currently has a good performance status appears to be beneficial. There is no generally accepted standard of care in this setting other than a trial of empirical therapy. As such, I have determined that the requested therapy is likely to be more beneficial for treatment of the patients medical condition than any available standard therapy. The Health Plans denial should be overturned.