The US Food and Drug Administration
has traditionally been the body that injects caution
into the drug discovery process. The FDA has historically
put its job of protecting patients from dangerous drugs
ahead of any responsibility to accelerate access to
unproven new drugs.
But now the FDA is proposing throwing
caution to the wind by vastly expanding access to drugs
that have never been approved. Under new proposals,
the exemptions given to groups and individuals with
life-threatening diseases are to be given far more liberally.
No longer will the FDA bend its
rules only for people at death's door. Under proposals
currently open for public comment on the internet (http://www.fda.gov/cder/regulatory/applications/IND_PR.htm),
early access to experimental drugs may be granted for
conditions whose burden may be defined as "persistent
or recurrent morbidity." That's a far cry from the current
standard of "imminent risk of death."
And patients will be able to get
in at an earlier stage of development. In the case of
really dangerous diseases, even drugs in phase I basic
safety trials will be accessible. Only about 12% of
such drugs ever go on to be approved.
In the case of phase II and III
drugs, which are generally evaluated in placebo-controlled
trials, patients granted access under the FDA's proposed
new rules will be sure they are getting the real deal,
and not a sugar pill.
The new rules won't distinguish
between Canadian and American patients. It's the drugmaker
who must apply, and all the FDA wants to know about
trial subjects is how many, and how sick they are. Canadians
who today cross the border for drugs in US clinical
trials will be eligible for this program too.
The FDA has always run various
programs to allow early access to investigational new
drugs, but knowledge of the ins and outs of the system
was generally limited to drug companies and hospital
specialists. The FDA's Deputy Commissioner for Operations,
Dr Janet Woodcock, acknowledged while announcing the
new proposals that the arcane nature of the old rules
made their compassionate use program "one of the best
kept secrets around." In the FDA's entire history, only
about 100,000 patients have been able to jump the gun
in this way.
Dr Woodcock said the proposed changes
were partly "a response to people who felt there was
a lack of clarity." Cynical observers might suggest
that it's actually a response to being sued. A dormant
lawsuit from two American patients' rights groups, demanding
that the FDA relax its regulatory standards, has recently
been reanimated by court decision. The hearing is set
for March 2007, which coincidentally is also the end
of the public consultation period on the new rules.
Like Canada's special access program,
the FDA insists that the new process can't be a substitute
for approval. All drug developers must be sincerely
aiming for market approval. Applications will still
be dealt with on a case by case basis — though
in future the FDA's rules and reasoning will be less
obscure.
A real concern is that the new
accessibility might interfere with recruitment for real
drug trials. No patient is going to take a 50% chance
of placebo in a double-blinded trial if they could be
sure of getting the active drug another way.
The FDA itself often comments on
the tension between its role as a regulator and its
natural desire to see patients get useful treatments.
Its original access program arose partly from a 1979
lawsuit launched by patients demanding pre-approval
cancer drug access. The FDA won that case, when the
Supreme Court ruled that its regulatory duties trumped
all others. But faced with endless demands from angry
patients, the agency decided that discretion was the
better part of valour.
In days of yore, if dangerous treatments
were suggested, nobody would have dreamed of denying
the patient — a paying customer — his or her
right to try.
But the growth of real professional
medical knowledge altered that balance of power. By
the time antibiotics were introduced, the doctor was
firmly in charge, and the patient's role was to take
what they were given — even if they were given
nothing.
Now the times have changed again,
and today's patient is a quick study. Across North America,
thousands of very sick people are glued to their computers,
frantically searching for any new drug that might offer
them an outside chance of improvement, cure or survival.
They can't know how safe an experimental drug is —
but then neither can the FDA. The patient will know
there are risks, and they will also know the likely
cost of doing nothing.
In deciding to treat patients like
adults, the FDA is simply recognizing the changed situation.
Informed consent is key to all medical treatment. It's
only logical that better-informed patients should be
able to consent to more things.
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