University of South Florida Distinguished Professor Benjamin Djulbegovic, MD, PhD, has studied the ethics of randomized clinical trials and their effectiveness in evaluating the outcomes of new treatments for decades.
Now, in a paper published Aug. 22 in the top journal Nature, Dr. Djulbegovic and colleagues report that on average new treatments work better than existing ones just over half the time. On scientific and ethical grounds, they say, the randomized controlled trial (RCT) system's little more than 50-50 success rate over the past half century is evidence that the system is working as intended.
The researchers analyzed 860 phase III published and unpublished RCTs performed by academic institutions or pharmaceutical companies. These trials collectively involved more than 350,000 patients.
"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," the researchers conclude. "These trials may need tweak and polish, but they're not broken."
People who consent to participate RCTs are willing to be randomly allocated to new or existing treatments. While RCTs are considered the gold standard for comparing the effects of one treatment to another, the gradual progress they yield can seem frustratingly slow -- particularly for patients with poor standard treatment options.