Non-US Clinical Trial Data and the Food and Drug Administration

Commercial requirements and the use of indigenous science for advertising purposes indicate that at least some studies will always be done in each country where medications are marketed. However, drug development is a transnational phenomenon where data from one country will be used to obtain marketing approval in another. Yet local regulatory and scientific requirements preclude the use of one set of data for all countries, which increases the cost of an already expensive process. In the US the FDA has historically expressed concerns about accepting foreign data as the sole basis for approval of an NDA. Despite the timolol case, foreign data are still used infrequently and only on an ad hoc basis. Data quality, verification, authentication, and inspection continue to be the most important problems in this area. Standardized protocols, and strict adherence to them; the use of investigators with a proven track record, ability, and appropriate training; frequent monitoring by United States drug sponsors; appropriate statistical analyses; a rigorous internal critique of the study; the use of anthropologists to deal with perceived and cryptic cross-cultural problems; and avoidance of “found” data will ameliorate problems with non-US data.