Evaluating promising new treatments for life-threatening disease: implications of the HDC/ABMT experience for treating breast cancer.

threatening diseases often inspire hope, patient demand, and physician enthusiasm before evaluation. In the late 1980s, highdose chemotherapy/autologous bone marrow transplantation (HDC/ABMT) emerged as a promising medical procedure for treating metastatic and high-risk breast cancer. But procedures, unlike new drugs, face no requirement for evaluation by randomized clinical trials (RCTs). In the early 1990s, HDC/ABMT began diffusing rapidly and widely into clinical practice concurrent with much slower evaluation by RCTs. By the decade’s end, four trials reported “no benefit” to the experimental procedure compared to standard treatment. Two trials reporting positive benefit, when audited, were found to be fraudulent.