Clinical trials: Around half of new treatments perform better than existing treatments
Researchers show that uncertainty about new treatments meets ethical requirements
On average, new treatments perform better in clinical trials only
slightly more often than existing treatments, according to a new
systematic review published in
The Cochrane Library . The fact
that experimental treatments are not more effective may seem
disappointing, but the authors of the review say their findings satisfy
an important
ethical requirement for clinical trials.
Randomised trials compare the effects of one treatment to another.
In a randomised trial patients are randomly allocated to different
treatment groups to ensure that like will be compared with like. When a
new treatment is being tested, it is hoped or even expected that it will
be better than the established treatment with which it is being
compared.
These expectations lead to an ethical dilemma. If the
researchers already know that one treatment is better, they would be
knowingly allocating some people to an inferior treatment. If randomised
trials are to be ethical, therefore, only half of new treatments should
turn out to be better than existing ones.
Cochrane researchers looked at evidence from 743 publicly funded
randomised trials involving 297,744 patients in total. The trials
included new, experimental treatments for cancer and neurological
disorders, as well as a range of other diseases. On average, only very
slightly more than half of new treatments proved to be better than
established treatments.
"When we compared the effects of new treatments to established ones,
the pattern was almost symmetrical.
This is good news, because it means
researchers genuinely don't know whether new treatments are going to be
any better," said lead researcher Benjamin Djulbegovic, who works at
USF Health Clinical Research, and the H. Lee Moffitt Cancer Center &
Research Institute, at the University of South Florida in Tampa,
Florida, US. "So, overall, what we show is that we can expect the new
treatments to perform better a little bit more often than established
treatments,
at least in publicly funded trials like the ones we
considered."
The researchers found the same pattern in trials going back five
decades. The results provide an answer to the question posed
15 years
ago in the British Medical Journal by Iain Chalmers, a founder of the
Cochrane Collaboration and one of the authors of the review. "In 1997,
in a letter published in the BMJ, I asked 'What is the prior probability
of a proposed new treatment being superior to established treatments?' I
think this review currently provides the best answer to that question,"
said Chalmers.