Appeal type: Experimental/Investigational
Decision: Overturned

Physician 1: The patient is a 62-year-old man with advanced sarcoma status post multiple chemotherapeutic agents. He is not a candidate for radiation therapy. A request has been made for approval for participation in a clinical trial of ET 743 (Yondelis). The Health Plan has denied this request on the basis that there is insufficient scientific evidence supporting the efficacy of Yondelis in the treatment of soft tissue sarcoma.Given that this patient has exhausted all standard chemotherapeutic agents and given the published data available in well-respected journals, enrollment in this clinical trial is likely to be more beneficial than standard therapy, which is non-existent for this patient. This clinical trial is the only treatment option remaining for this patient. Given the published data, the patients participation in the trial is medically reasonable and appropriate. 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.Physician 2: The patient is a 62-year-old male with progressive soft tissue sarcoma without response to anthracycline infosfamide and darcarbazine. He remains in good condition and is an appropriate candidate for additional chemotherapy. The patients provider has recommended the patient be enrolled in a clinical trial of ET 743 (Yondelis). The Health Plan has denied this request on the basis that Yondelis is considered experimental.There is adequate evidence that the proposed investigational treatment has definite activity in refractory soft tissue sarcomas. There are no available conventional chemotherapy agents with any reasonable likelihood of benefit. The patient is an appropriate candidate for the requested trial. Given the scientific data available to date and because of the lack of alternative options, the proposed clinical trial is appropriate for this patient and should be authorized. 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.Physician 3: This patient is a 62-year-old male who has advanced soft tissue sarcoma. He has failed all standard treatment modalities. The patients provider believes a clinical trial of Yondelis (trabectedin) is the only appropriate treatment option that remains for the patient. The Health Plan considers the proposed therapy experimental and therefore has denied authorization and coverage for the clinical trial.This patient has soft tissue sarcoma, has failed standard of care treatment, and appears to be eligible for the requested clinical trial as listed at the NCI clinical trials website. The study drug, trabectedin, has been presented at American Society of Clinical Oncology meetings in several promising Phase I and Phase II clinical trials. Given the scientific evidence currently available, the requested clinical trial is a reasonable and promising step for this patient. Accordingly, 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.