For renal studies, the downstream effect of trials conducted in developing countries is even more complex and far-reaching. At the individual patient level, overseas research could decrease U.S. patients’ access to experimental treatments. As to why this is important, consider the situation in oncology, where experimental therapies frequently offer better and longer life to patients not responding to conventional therapies.
On a trial level, the issues include the necessary limitations related to generalizability and safety. Our population is so exceptionally heterogeneous that we are just beginning to be able to evaluate the role of concomitant conditions, such as inflammation (Kidney Int.1999;55:648-658). The trend toward conducting trials in undeveloped countries could leave important clinical issues largely unaddressed.
One such issue is how treatment efficacy and safety are affected by practice differences in the management of complications, such as hypertension and anemia. Practice differences vary significantly between countries and likely affect outcomes. There are other notable differences to be considered in overseas trials, e.g., race.
Finally, this movement toward conducting trials abroad comes at a time of decreased federal funding. The shifting of research to other countries will undoubtedly affect the ability of young trainees in the United States to stay in academic medicine and begin their research careers. This, in turn, could decrease the number and morale of future physician-scientists.
The nephrology community needs to look at solutions that have been successful with other relatively small patient populations. For example, recognizing the disproportionately small amount of research being conducted in children in 1997, Congress enacted the Food and Drug Administration Modernization Act. This act rewards companies that perform studies in the pediatric population both financially and with extended patent life for the medication tested.
That extension enables companies to recoup research costs, making this contribution to evidence-based care of this relatively underserved population at least financially neutral, if not financially beneficial. Given our similar need in nephrology, a parallel offering may well be a viable solution to increase pharmaceutical interest in the treatment of kidney disease. This, however, will require significant lobbying of the FDA by a unified nephrology community.
Overcoming the hurdles that face us will require major contributors to nephrology research (e.g., physician-scientists, pharmaceutical companies, dialysis providers) to stop thinking and functioning in isolation. If efforts of these disparate groups were coordinated, I have no doubt we would see more substantial advancements in therapeutics and in the understanding of renal disease.
Dr. Szczech is associate professor of medicine at Duke University Medical Center in Durham, N.C., and a member of the Renal & Urology News editorial advisory board.