Study: Amazing early-stage R&D results rarely hold up in big trials

Any regular reader of FierceBiotech knows just how rare it is for a promising drug that produced stellar results in small early studies to march all the way through the clinic without losing its luster or getting snuffed out by an adverse event or lackluster results. Now there's an in-depth trial review that shows clearly just how bad the odds of continued success are.

A new study that has examined the results of thousands of clinical trials has determined that 90% of the early winners failed to continue to produce the same clinical effect in later studies. A whopping 98% of the follow-on studies that did see a big effect failed to continue to wow investigators in subsequent trials.

So why is disappointment the ultimate rule for drug developers? Dr. John Ioannidis at Stanford says the big reason is trial size. If you study a drug in a small trial, random positive responses tend distort the results, skewing the data in a way that wows developers.

"I think some healthy skepticism and a conservative approach may be warranted if only a single study is available--even more so if that study is small and/or had obvious problems and biases," Ioannidis tells HealthDay. "Most of the time, waiting for some better, larger, more definitive evidence is a good idea. No need to rush."

- read the Reuters story
- here's the HealthDay report

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