Medical research: Trial unpredictability yields predictable therapy gains
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Extract: The effects of a new treatment are hard to predict. In the 1940s, pathologist Sidney Farber theorized that folate might help children with acute lymphoblastic leukaemia by stimulating blood cells; instead, he was surprised to find that the leukaemia cells proliferated. That ‘failure’ led him to try anti-folate drugs. These did lead to remissions, and were the first example of successful chemotherapy for cancer. In the 2000s, physicians thought that corticosteroids might help to reduce brain swelling following trauma, but randomized trials showed that they actually increased mortality. Surprisingly, however, corticosteroids were found to reduce the death rate in meningitis1. These are just a few examples of the steady therapeutic advances delivered over the past half century by the randomized controlled trial (RCT) system. But these improvements can feel frustratingly slow for patients and physicians. There is much soul searching in the drug-discovery community about how progress could be made more quickly.
This document has been peer reviewed.