Opinion ID: 655549
Heading Depth: 1
Heading Rank: 1

Heading: facts

Text: 3 New medicines go through several phases of clinical evaluation before general release onto the market. HEM conducted a clinical trial with 92 patients designed to evaluate the effectiveness, side effects, and risks of Ampligen, classified by the FDA as an investigational new drug not yet permitted to be sold freely as a prescription medication. The study was double blind, which means that some patients got Ampligen, some got a placebo (saline solution), and neither the doctor nor the patient knew who was getting which. The Ampligen was administered as a liquid by slow injection into a vein. All the patients signed consent forms warning of the experimental status of Ampligen and possible side effects. Although the patients were free to withdraw at any time, if they remained in the study they were required to accept the risks of treatment, forgo other drugs, not become pregnant, and submit to uncomfortable testing. 4 The arrangement with the experimental subjects was that they would participate in the double-blind study for a year. This was to facilitate evaluation of the safety and effectiveness of Ampligen. After the double-blind phase of testing ended, they would be entitled to receive Ampligen for a full year at no charge. The consent forms included a conditional promise of additional Ampligen after the double-blind study was completed: 5 If statistical analysis of the endpoints show that Ampligen TM shows efficacy compared to placebo, then following completion of all termination procedures, you understand that if you received placebo on study, you will be offered Ampligen TM and will reenter and follow the same protocol as if you had been randomized to receive Ampligen on study. If you received Ampligen TM on study, you understand that you will be offered continuation on Ampligen TM and will re-enter and follow the same protocol. 6 After the double-blind study, HEM applied for permission to proceed with what the FDA calls a treatment IND. IND is an FDA acronym for investigational new drug application. 21 C.F.R. § 312.3(b). The application, had it been granted, would have allowed the use of Ampligen in the treatment of patients not in the clinical trials. 21 C.F.R. § 312.34(a). This procedure can be used for an experimental new drug if the disease is serious and there is no satisfactory alternative treatment. 21 C.F.R. § 312.34(b). The FDA rejected the application for safety reasons, but allowed the next phase in clinical trials, an open label study, where the doctor and patient know that the patient is receiving Ampligen. 7 FDA has completed its review of the treatment IND and has concluded that the data do not support expansion of ampligen treatment at this time. Therefore, the application has been placed on clinical hold until outstanding issues and concerns can be resolved. 8 In addition to numerous deficiencies in the application, the agency is concerned particularly about the serious and potentially life-threatening reactions that were observed during the study, and the conduct of the study in general. These serious reactions included acute hepatic (liver) toxicity, severe abdominal pain and irregular heartbeat. 9 In a letter to the company ... FDA Commissioner David A. Kasslar, M.D., said that HEM's study results involving 92 patients are preliminary and that data submitted thus far to FDA are incomplete and inadequate for FDA to assess safety and effectiveness in CFS. He emphasized that it was important for CFS sufferers to know that the significant side effects associated with Ampligen would have to be weighed against any claimed benefit before the drug could be approved for wide-spread use. FDA will continue to work closely with the manufacturer in an effort to resolve all issues. 10 . . . . . 11 An open label study of Ampligen in patients with CFS will be allowed to continue. FDA Talk Paper T91-63 (Oct. 4, 1991) 1 12 Thus, the FDA prohibited use of Ampligen on patients not in the clinical trials, but expressly allowed it for those who were. 13