鈥淚S IT ethical to let me die when there is a drug which would very likely
save my life?鈥� Elizabeth Rees is 47, and a mother of two. In February 1999 her
life changed completely when she discovered that she had a common form of
leukaemia called chronic myeloid leukaemia. The disease can be controlled for a
while with chemotherapy or immunotherapy, but not cured. Even if an appropriate
donor can be found, bone marrow transplants are fraught with danger and in most
cases fail to cure the condition. Once CML progresses to its 鈥渁ccelerated
phase鈥�, it kills most patients within a year.
Now though, there鈥檚 a new hope, and word is flying around the world via the
Internet. Key 鈥淪TI571鈥� into any search engine and you鈥檒l get a long list of
sites detailing miraculous recoveries. Elizabeth has already read other
patients鈥� excited endorsements of STI571 on the Web. 鈥淲e all have a long way to
go but the future for me and my family seems wonderful again,鈥� writes Sandy. 鈥淚
now have reasons to believe that there is ample hope for the future,鈥� says
Virginia. It鈥檚 not surprising Elizabeth wants some鈥攄esperately.
STI571 is a new drug that has achieved stunning results in preliminary
trials, sending 95 per cent of patients into remission. For Elizabeth, it
could be her only hope, but the drug is not yet licensed and she doesn鈥檛 qualify
for clinical trials at her treatment centre鈥擧ammersmith Hospital in
London鈥攂ecause she has yet to exhaust the range of (not very effective)
standard treatments. Her consultant John Goldman told 快猫短视频
that if enough of the drug were available he would offer it to all his patients
in Elizabeth鈥檚 position.
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Normally, any new cancer drug would undergo three phases of clinical tests,
lasting years, to assess its safety and efficacy, before a drugs company would
even apply for a licence. But that was before the Internet. Because thousands of
patients like Elizabeth are clamouring for access to the new 鈥渨onder drug鈥�, it
might be granted a licence much sooner, at least in the US.
Novartis, which developed STI571, is planning to apply for its US licence in
the next month or so. Currently, 1150 people around the world are testing the
drug. But because of its potential as a life-saver the US Food and Drug
Administration has already agreed to an 鈥渁ccelerated approval鈥� approach, which
involves assessing the drug based on just two rounds of tests, rather than
requiring extensive data from a phase III trial, as is the norm.
The explosion of interest in STI571, fuelled by the Web, caught Novartis by
surprise. While CML might be one of the more common kinds of leukaemia, in
disease revenues terms it鈥檚 small fry. No companies are keen to expand costly
production of the drug without being confident of a licence. Now all the
publicity means it doesn鈥檛 have much choice.
While this sort of publicity might be a triumph for patient power, the Web
doesn鈥檛 always work to their advantage. In April, Britain鈥檚 Medicines Control
Agency (MCA) reprimanded one drugs company, for setting up a company-funded
website masquerading as a patient support group to promote its own drugs and
lobby for wider availability. Currently, the MCA is investigating complaints by
the Multiple Sclerosis Society about another website called MS Voice set up by
Schering Healthcare. The complaint is that the website gives the impression of
being a genuine patient support group, when it is actually a company initiative,
according to an MCA spokeswoman.
Charles Medawar of the pressure group Social Audit is worried that such
incidents mark the beginning of a trend. 鈥淭he borderline between promotion and
rational information has become almost impossibly blurred,鈥� he says. He worries
that the proliferation of websites about new drugs will give patients false hope
and might compromise safety if it puts the regulators under pressure. The MCA
agrees there鈥檚 a problem. 鈥淪etting up websites about drugs is something that is
becoming more common. It鈥檚 a question of getting a balance between legitimate
information and company promotion,鈥� says a spokesperson.
While the FDA is moving towards faster licensing, Europe remains more
conservative in its approach. Neither the MCA nor the Europe-wide European
Medicines Evaluation Agency (EMEA) has yet adopted an accelerated approval
procedure for cancer medicines. 鈥淲e may get more letters but we can鈥檛 let it
have an impact because that would compromise public safety,鈥� says an MCA
spokeswoman. They view safety as the top priority. Licence applications are
tackled in the order they arrive鈥攖hough licence applications for
potentially life-saving treatments do tend to get processed quicker.
But even in Europe changes may be on the way. As a result of pressure from
the European AIDS Treatment Group, the EMEA is considering licensing drugs for
鈥渟alvage therapy鈥濃�攖o treat AIDS patients where all existing treatments
have failed鈥攂ased on clinical trials of just 16 weeks. The decision is
expected in the next few months. The move is part of wider EU review of drug
licensing. The notion of 鈥渃onditional licensing鈥� is top of the agenda. 鈥淭his
would mean that a product could be accepted at an earlier stage of development,
but the company would be subject to certain conditions鈥攆or example,
continuing clinical trials after licensing,鈥� says Patrick Le Courtois, head of
the Sector for New Chemicals and Disease at the EMEA. 鈥淚n certain therapeutic
fields such as HIV or oncology, I think it can be helpful.鈥�
Conditional licensing of drugs for certain life-threatening conditions is
already accepted at the FDA. In certain cases companies can use 鈥渟urrogate
markers鈥� such as tumour shrinkage or lowered blood pressure to show that a drug
works. So a drug may not have to save lives to get this kind of licence. The
dramatic impact of HIV and AIDS in the last 15 years paved the way for this sea
change in clinical research. Patients and their doctors thought the
effectiveness of drugs should be measured by their ability to slow the
destruction of the immune system, rather than whether or not they prolonged
life. A drug gets its conditional licence on the understanding that the company
will continue clinical trials after approval to show the drug鈥檚 benefit
conclusively.
But there鈥檚 a major flaw in the FDA鈥檚 approach: it has no recourse to law
that can make drugs companies honour their commitments. A US watchdog group
called Public Citizen published a study in April this year of pharmaceuticals
companies that had agreed to continue post-marketing clinical studies of newly
approved drugs as a condition of receiving an FDA licence. They found that of 88
drugs approved between 1990 and 1994, studies had only been completed for 11 of
them, even though the companies had had between 5 and 10 years to do the trials
(see Graph).
And for drugs approved between 1995 and December 1999, not even one
fulfilled its licence commitment.
An FDA spokeswoman says that in theory, if a company fails to keep its
commitments after licensing, then the licence could be withdrawn, but she admits
鈥渢hat has never happened鈥�. It鈥檚 not clear if or how the EMEA would police such
post-marketing surveillance.
The figures show that an estimated 100 000 Americans die every year from
adverse reactions to drugs, and many feel that bowing to pressure from patients
and/or drugs companies by lowering licensing requirements could in the end cause
more problems than it would solve. It鈥檚 a tough call for any licensing agency,
and the EMEA is expected to come up with proposals concerning conditional
licensing at the end of the year.
In the meantime, Elizabeth Rees is still waiting.