Appeal type: Experimental/Investigational
Decision: Overturned

Physician 1: The patient is a 45-year-old woman who has had diabetes since the age of 10 with long standing symptoms of nausea and vomiting from gastroparesis, particularly at night. She also has hypertension. Currently, she is maintained on an insulin pump. She has failed to have improvement with conventional therapy and has used domperidone without improvement. The patient underwent endoscopy (which did not demonstrate an anatomic cause for symptoms) with injection of Botox into the pylorus (which did resolve her symptoms). She has had an abnormal, delayed gastric emptying study on two occasions and an electrogastrogram performed in February 2004 was abnormal. A trial of Enterra therapy has been recommended. The Health Plan has denied authorization and coverage for the requested therapy on the basis it is considered experimental.The patient has documented childhood diabetes with complications including peripheral neuropathy and long-standing symptoms of gastroparesis. The abnormal gastric physiology has been documented by gastric emptying study. The proposed therapy is insertion of a gastric electrical stimulator, known as Enterra.The gastric electrical stimulator used in the WAVESS trial was submitted for review by the Food and Drug Administration. Because of the lack of a controlled trial showing clear-cut efficacy, the FDA did not approve the device for unrestricted marketing for the treatment of gastroparesis. However, because of the promise shown in the uncontrolled trials, the device was approved as a humanitarian use device (HUD). The device received humanitarian device exemption approval in March 2000 to be used for the treatment of refractory diabetic and idiopathic gastroparesis under FDA conditions of approval. A second sham-controlled trial ultimately will need to be performed before the Enterra device is formally approved for open, unrestricted marketing. Though there is only a small body of literature attesting to its efficacy and it has not yet been accepted for general use by the medical community, the difficulties in obtaining unequivocal data proving efficacy in a rare condition were recognized by the FDA in granting a HUD exemption. This approval does show the judgment of the approving panel that the device is likely to be helpful. In the case of this patient, other alternative treatments have been exhausted without success. Based upon the available literature, the evaluation by the FDA, and the use of this treatment in selected cases by experts in gastrointestinal motility disorder, Enterra therapy is likely to be more beneficial for this patient than any available standard therapy. Therefore, the Health Plans denial should be overturned.Physician 2: The patient is a 45-year-old woman who has had diabetes since the age of 10. She is symptomatic every day. She has responded partially to domperidone. Her 120 min. gastric emptying study was 71% and she had a four-hour retention of 37%. Pyloric Botox was also used, which did not help this patient. An electrogastrogram also showed evidence of gastroparesis. She had also failed therapy with promotility agents. The patients provider has recommended Enterra therapy, which has been denied by the Health Plan.The Enterra device was approved by the FDA in March 2000. This has been studied in the multicenter Worldwide Anti-Vomiting Electrical Stimulation Study (WAVESS) trial. Though the trial involved a low number of patients, the 12-month improvement in symptoms and quality of life were greater than 50%. In addition, this form of therapy reduced the rate of hospitalization 30% in the first year after implantation. Benefits from this form of therapy have also been reported in the GEMS study group in 1997 and 1999. Furthermore, in an article by McCallum, et al., the authors concluded gastric pacing improves emptying and symptoms in patients with gastroparesis. In the case of this patient, she meets the entry criteria for use of the device including symptomatic gastroparesis for greater than one year, refractory or intolerant to at least two anti-emetic and prokinetic drug classes, frequency of vomiting greater than 60% retention at two hours and greater than 10% at four hours. The patient has been symptomatic for quite some time with multiple failures to medical therapy. There is sufficient evidence of efficacy to warrant use of the Enterra device in the case of this patient.Based upon the information set forth above, I have determined the requested therapy is likely to be more beneficial for treatment of the patients medical condition than any available standard therapy. Therefore, the Health Plans denial should be overturned.Physician 3: The patient is a 45-year-old female with type I diabetes mellitus since the age of 10. She has been vomiting since the age of 25. The patient has had several hospitalizations for hydration throughout the years. There has been a decrease in her hospitalizations since she has been on domperidone. However, the patient continues to have nausea and vomiting every day that wakes her up at night. Her last hospitalization was approximately one year ago.The patient has had extensive workup for her intractable symptoms. She had two gastric emptying studies, both of which were abnormal in delayed gastric emptying. The last one showed a two-hour retention of 71% (normal being 60%) and a four-hour retention of 37% (normal being less than 10%). She also had an upper endoscopy with pyloric manometry (pyloric pressure increased to 59.5) and Botox injection of pylorus in December 2004 with no change in her symptoms.It has been recommended the patient receive Enterra therapy. The Health Plan has denied coverage for Enterra therapy citing a lack of proven efficacy.Enterra therapy has been approved by the Food and Drug Administration, and its safety and efficacy have been established in peer-reviewed literature. It offers a safe and effective alternative for patients with intractable symptomatic gastroparesis. The patient has intractable nausea and vomiting secondary to diabetic gastroparesis that has been refractory to medical therapy. Enterra therapy is medically indicated and an appropriate intervention at this point to improve her symptoms and quality of life. Furthermore, it would improve her diabetes with better glycemic control, and reduce hospitalizations. Therefore, 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.