EFFICACY OF TREATMENT

[摘要] The "gospel" of randomization is based on a pyramid [of six steps] conveying the hierarchy of strength of evidence concerning the efficacy of treatment: (1) anecdotal case reports, (2) series without controls, (3) series with literature controls, (4) series with historical controls, (5) single randomized clinical trials, and (6) confirmed randomized clinical trials. The spectrum of evidence ranges from individual anecdotal case reports to findings confirmed in multiple randomized trials. It is certainly true that efficacious treatments can be identified in the lower numbered steps of the pyramid. If an investigator happens to "hit a home run" in such a manner and discovers a cure for a previously untreatable disease, then it may not be necessary to initiate further studies. Smaller gains, however, require more stringent proof. I would argue that without steps 5 and 6 (ie, randomized trials), it is impossible truly to convince the scientific community about the efficacy of treatment.

[学科分类] 儿科学