In an editorial published in the Journal of the American Medical Association (JAMA), Drs. Hu, Laviana, and Sedrakyan suggest that new technologies should be used cautiously until rigorous scientific evidence of safety and efficacy is available. This position is fully endorsed by the Focused Ultrasound Foundation.
As a case in point, the authors cite the example of high intensity focused ultrasound (HIFU) to treat prostate disease. Although HIFU has been used around the world for more than a decade to treat more than 50,000 patients with prostate disease, and the US Food and Drug Administration (FDA) approved SonaCare’s Sonablate and EDAP’s Ablatherm systems to ablate prostate tissue last fall, there is still no definitive evidence as to which patients will benefit and what grades of disease are most appropriate to treat.
Questions also remain surrounding the ideal monitoring technology and whether the transurethral or transrectal route will yield best results. The answers to these questions are not likely to be achieved through randomized controlled clinical trials. Such trials are not practical because of the number of patients required, the length of time for follow-up, reluctance of patients to randomize, and the fact that technology evolves over time.
As the authors conclude, the best approach is to create registries that will help assess which treatment yields optimal results while also generating data to obtain specific indications for use. These registries could even be extended to serve as comparative effectiveness trials, evaluating the utility of HIFU and other therapies for all stakeholders, particularly if standardized data can be collected prospectively with outcomes evaluated by individuals independent of the management of the patient.
In fact, the FDA has invited Foundation staff and representatives from industry, academia, and the patient community to attend a meeting this week to discuss the development of such a registry. We look forward to participating in the process to generate robust data to enable a better understanding of the benefits, risks, and side effects of all prostate treatment technologies and guide physicians and patients in choosing their best option. These types of tasks are a fundamental part of our role in advocating for the technology.