November 27, 2013
The globalization of clinical research is demonstrated by the exponential increase in clinical trials conducted outside of the United States. These have been submitted to the US Food and Drug Administration (FDA) for marketing approval of new drug applications. An interesting article published in Nature written by FDA employees, discuss their experience with submissions of results from clinical trials performed outside the US, in specific therapeutic areas, including orphan drugs. They also discuss the “extent of this practice, differences between the effectiveness and safety outcomes of studies conducted inside and outside the United States and the FDA’s approach to acceptance of these trials“.
According to the authors, in the area of orphan drugs, the FDA exercises “greater regulatory flexibility” due to the rarity of the diseases in question and the difficulty of finding adequate number of patients to perform an efficient clinical trial, thus making FDA approval for orphan drugs based entirely on foreign data more likely. The authors provide a several examples where the FDA has approved drugs based completely on data gathered from countries other than US. These examples include the approval of velaglucerase alfa for type 1 Gaucher disease for which the clinical trials were conducted in five countries – Argentina, Paraguay, Israel, Russia, and Tunisia. The FDA has also approved carglumic acid for the treatment of hyperammonemia in patients with N-acetyl-glutamate synthase deficiency of the urea cycle was also based on data from France, Germany, and the United Kingdom. Thus, the authors confirm the FDA’s eagerness on the use of cross border data from clinical trials for orphan drugs.