LONDON (Reuters)
— Children with cancer are
being denied access to potentially lifesaving medicines because
European Union rules allow drug firms to waive the need to test some
drugs in pediatric trials, researchers said on Monday.
Calling for a loophole in EU legislation to be
closed, scientists and specialists in childhood oncology led by
Britain's Institute for Cancer Research (ICR) said changing the
rules could extend or save many young lives.
"Many cancer drugs developed for adults could be effective in
children if we were able to test them in clinical trials," Louis
Chesler, an ICR researcher and a pediatric oncologist at London's
Royal Marsden hospital, told reporters at a briefing.
Currently, pharmaceutical companies developing new cancer medicines
can gain exemptions from carrying out expensive testing of those
drugs in patients under age 18, even where a drug's mechanism of
action suggests it could work in children.
As a result, campaigners and scientists say, there are often long
delays before new drugs are available for children, and some are
never licensed for pediatric use at all.
"Kids get hand-me-downs in many areas of life," said Chesler. "But
cancer drugs should not be an area where kids get hand-me-downs."
Experts say the system is out of date because it is based on
previous thinking about cancer as a disease specific to the organ — such as lung, breast or bowel cancer — rather than on latest
scientific knowledge showing cancers are driven by genetic changes
that can happen in many parts of the body and in various types and
ages of people.
Drugs have been approved for treating adult lung and other cancers
driven by mutations in ALK or EGFR genes, for example, but the
companies have been granted waivers from testing them in children — even though ALK and EGFR mutations have been shown to play a role in
some childhood cancers.
"The current system is failing to provide children with access to
new treatments that could add years to their lives," Alan Ashworth,
ICR's chief executive, told the briefing.
"We're not blaming the EU legislation for this — but it needs
rapidly on be modernized," he added.
Research carried out by the ICR together with the European
Consortium for Innovative Therapies for Children with Cancer (ITCC),
based in France, found that of 28 cancer drugs approved for adult
marketing authorization in Europe since 2007, 26 work in a way that
is relevant for childhood cancers.
Yet 14 have been exempted from being tested in children because the
specific adult condition for which the drug is developed does not
occur in children.
The ICR and other international cancer researchers and campaigners
want the EU to abolish drug makers' right to an opt-out — a change
that would compel them to test a new drug's potential to treat
children's cancers as well as adults'.
Ashworth also said he'd like to see stronger financial incentives to
ensure a decent return for drugmakers who take on the challenge of
developing new drugs that are only likely to be able to be used in a
small number of patients.
