* Companies may do shorter, fewer trials for approval
* Proposal likely to pass as part of FDA user fee bill
By Anna Yukhananov
WASHINGTON, May 9 (Reuters) – Experimental drugs that show a
big effect early in development for treating serious or
life-threatening diseases would get a faster and cheaper path to
U.S. approval, under a proposal likely to become law this year.
U.S. drug regulators would be able to label such treatments
“breakthrough” therapies, and work with companies to speed up
clinical trials, for example by testing the drugs for a shorter
time or enrolling fewer patients.
The U.S. Food and Drug Administration has said it supports
the proposal, which is included in both versions of an FDA
“must-pass” funding bill currently working its way through
Congress and set to be passed by the end of the summer.
The plan fits with President Barack Obama’s aim to foster
innovation as a means of spurring job creation, and may pacify
some critics who say the FDA lags European countries in
approving new medicines or medical devices.
Dr. Janet Woodcock, head of the FDA’s drugs center, has said
the FDA needs more flexibility to bypass “business as usual”
when it sees unexpected effects, or when a new medicine can
greatly help patients.
“What happens when you have a breakthrough drug that shows
an effect that’s never been seen before?” she told reporters in
March, discussing the proposal.
“If we’d done business as usual during the AIDS epidemic, we
would have never controlled that epidemic,” Woodcock said.
During a spike in new cases of AIDS in the early 1990s, the
FDA created an “accelerated approval” process to get new AIDS
drugs to the market more quickly by allowing companies to show
indirect measures of how the drug helped people.
Jeff Allen, executive director of Friends of Cancer
Research, said the time was right for another avenue to speed
innovative treatments to patients.
Allen said new understanding of human biology and of
diseases meant companies could create more effective drugs, but
with fewer side effects, because they would be more targeted to
specific patients or disease types.
He said a good example was Roche Holding AG’s skin
cancer drug Zelboraf, approved last year; people taking the pill
were 63 percent less likely to die from melanoma than people on
standard chemotherapy.
There was also Pfizer Inc’s targeted lung cancer
drug Xalkori, which could shrink or eliminate tumors in 10 to 12
months for people with a specific genetic mutation.
“The most promising drugs show an effect early,” Allen said.
“(But) there’s a mentality among drug developers or FDA
reviewers that you have to go through this multi-step approach”
to get a drug to market.
“We’re hoping to encourage getting away from that,” he said.
Allen said the FDA would retain its power to require
companies to do post-approval studies, or withdraw drugs from
the market if initial evidence of benefit was not shown in
follow-up trials.
