The government's own watchdog will soon be sniffing around FDA drug approvals and FDA-ordered post-marketing studies. Prompted by Sen. Charles Grassley, the Government Accountability Office plans to investigate the practice of using biomarkers such as blood sugar in diabetics and cholesterol levels among those at risk of heart disease, in evaluating efficacy. These measures are used in drug trials in part because assessing actual outcomes like heart attacks and strokes requires years-long studies that cost megabucks. If the FDA required pre-approval outcomes studies, new drugs would come to market even more slowly than they do now. And development costs would skyrocket.
Lately, though, biomarkers have become controversial as safety and effectiveness questions arose about meds such as GlaxoSmithKline's Avandia. That drug was approved because it lowered blood sugar, which docs believe will in turn cut the risk of heart attack. But a study last year showed that Avandia might actually increase heart-attack risk. Then, early this year, a major trial of Merck and Schering-Plough's cholesterol med Vytorin showed that it did a better job of lowering bad cholesterol than a generic statin did--but it did no better at preventing plaque buildup in patients' arteries. So in these cases, long-trusted biomarkers were called into question, and some experts contended that the FDA shouldn't OK a product without real outcomes studies.
Now, the GAO has agreed to weigh in on the subject. Sen. Charles Grassley (R-IA) asked the watchdog to investigate. His point: If the FDA uses biomarkers to approve a drug, then it needs to enforce its own calls for post-marketing outcomes studies, so-called Phase IV trials. Is the FDA doing so? According to a 2006 Health and Human Services investigation, no; at the time, the FDA couldn't even readily identify which companies had made progress on those studies and if so, how much. Recently, the FDA said that of 1,200 of the studies it had required 900 hadn't even begun.
Under a new law, FDA can fine companies up to $1 million for failing to perform those studies. But is $1 million an adequate punishment? Grassley has argued for higher fines, and in his request to the GAO, asked whether FDA needs more authority to compel the studies. All we can say is, if the FDA got all the power and money its critics say it needs these days, it would be formidable indeed.
- check out Grassley's release and letter to the GAO
- read the AP report