Illinois-based Northfield Laboratories delivered some unpleasant news to medical professionals and investors this week, announcing that its blood substitute, PolyHeme, did not prove to be effective in late-stage clinical trials. However, the company is claiming that “discrepancies in the initial data” and “protocol violations” may invalidate the results and they intend to re-examine the data before certifying the findings.
In its announcement of the findings, Northfield said that 712 patients were included in the randomized study: 349 in the PolyHeme group and 363 in the control group (which received the standard treatment of blood and saline). In the study population, there were 46 deaths (13.2 percent) in the PolyHeme group, compared with 35 deaths (9.6 percent) in the control group. These results fell short of the standard necessary to deem the product safe and effective.
Yet, the company is maintaining that large numbers of patients–20 percent of the PolyHeme group and 15 percent of the control group–should be considered protocol violations. When these patients are removed from the calculations, the trial appears to be more successful: 30 deaths (10.8 percent) in the PolyHeme group and 28 deaths (9.1 percent) in the control. Protocol violations include mistakes having to do with eligibility and treatment regimen.
Northfield describes its PolyHeme product as a “human hemoglobin-based oxygen-carrying red blood cell substitute [used] in the treatment of severely injured and bleeding patients when blood is needed but not immediately available.” The failure of the trial means that it is highly unlikely that the U.S. Food and Drug Administration will recommend approval of PolyHeme based on available data. Northfield will probably have to conduct a new study before it asks the FDA for approval.