Randomized controlled trials are effective, research shows

In just about 50 percent of randomized controlled trials, new treatments work better than existing approaches, making these trials both ethical and effective, according to a new research report published in the journal Nature.

By researching 860 randomized controlled trials involving more than 350,000 patients performed in the last 50 years, lead author Benjamin Djulbegovic, a professor of medicine and oncology at University of South Florida, determined that randomized controlled trials are effective.

"Our retrospective review of more than 50 years of randomized trials shows that they remain the 'indispensable ordeals' through which biomedical researchers' responsibility to patients and the public is manifested," Djulbegovic and his co-researchers said in a statement. "These trials may need tweak and polish, but they're not broken."

Although randomized controlled trials, which assign new or existing treatments to participants, can generate very slow results comparing treatments' effects, Djulbegovic said it's the uncertainty that makes these clinical research trials so effective.

That's because if there was enough of a chance that one treatment was more effective than another, it actually would be unethical for researchers to deny some participants the superior treatment. What's more, Djulbegovic said, well-informed patients wouldn't want to participate in these studies.

This research report contrasts a study published in Mayo Clinic Proceedings that found 146 contemporary medical interventions are ineffective or actually cause more harm than good. Researchers in that study analyzed 1,344 original articles published in the New England Journal of Medicine between 2001 and 2010, determining that of the 363 articles that tested what doctors are doing today, 40 percent found the practices to be ineffective, 38 percent reaffirmed the value of existing practice while 22 percent were inconclusive.

To learn more:
- here's the research report
- read the University of South Florida statement