Subject: REVIEW & OUTLOOK (Editorial):  Decision for the Dying
Date: Published: 8/2/88 102 lines
Source:  WALL STREET JOURNAL. Copyright Dow Jones & Co. Inc. 

   REVIEW & OUTLOOK (Editorial):  
   Decision for the Dying

   Americans dying of AIDS, Alzheimer's disease, cancer and
other scourges may be closer than ever to getting real help
from their government.  According to well-informed sources,
the Reagan administration is discussing whether it should
announce a significant initiative that would revise
regulatory requirements to speed the approval and marketing
of new drugs for desperately ill patients.  The proposal would
most likely call for lifting the lengthy "phase three"
efficacy tests for new drugs that are now required by the
Food and Drug Administration.
   The proposal reportedly comes from a meeting last Friday
of Vice President Bush's Regulatory Task Force, which was
attended by FDA Commissioner Frank Young.  Word of the meeting
has since spread through Washington's drug-policy community.
   A decision to suspend phase-three testing of new drugs for
desperately ill patients would be the most significant event
in U. S. drug regulation since the 1962 Kefauver amendments to
the Food and Drug Act.  Enacted as a response to the birth
defects caused by the sedative thalidomide, Kefauver's
requirements have since gone well past the point of public
protection and are now a disincentive to developing drugs to
treat America's sickest people.
   If the administration is prepared to make this commitment,
we hope Vice President Bush has reserved the opportunity to
 make the announcement.  It would be an appropriate rejoinder
to Teddy Kennedy's taunt, "Where was George?  "
   While the rest of Washington temporized or pontificated
about the needs of AIDS patients, Mr. Bush has tried to use
his Regulatory Task Force to prod the FDA toward speedier
delivery of new therapies to all seriously ill people.
   As roughly outlined, the proposal would work as follows.
The FDA would involve itself closely in the first two phases
of the current drug-testing process.  Phase one tests a new
drug for toxicity.  Phase two, usually carried out in up to
some 200 volunteer patients, tries to discover if the drug
actually treats the disease and also monitors for toxicity
and significant side effects.  If the drug passed the
phase-two trials, it would presumably be approved for sale to
this special class of seriously ill patients.  The drug's
effects would then be closely watched through post-marketing
surveillance.
   The phase-three tests that would be eliminated for this
extreme class of patients currently involve large numbers of
patients in a series of blinded, randomized,
placebo-controlled clinical trials.  This typically can add
two to three years to a drug's approval time, and it is the
phase that has attracted the most controversy.
   Questions have been raised about the ethics of giving
placebos to terminally ill patients and about requiring
further pre-approval tests of a drug that has shown in phase
two that it offers relief.  Phase three's defenders argue that
placebos and randomized trials are the most reliable way of
obtaining statistically meaningful results from efficacy
tests.  In general terms there is something to this point, but
it hardly overwhelms the existing ethical concerns and it
begs the question of whether phase-three trials are
necessary.  The FDA's own figures on drug approvals suggest
that patients may have been paying too high a price waiting
for phase three's elaborate protocols to lay "meaningful"
data at the feet of the statisticians.
   In a study of new drugs that were submitted to the agency
over a two-year period, the FDA discovered the following
rates of success across the three currently mandated testing
phases:  
   Phase One:  70%.
   Phase Two:  33%.
   Phase Three:  29%.
   Commissioner Young has drawn attention to what is
perfectly obvious from the mere 4% dropout rate after
completing phase three:  "The majority of new chemical
entities that enter phase three eventually get approved for
marketing."  Put more bluntly, if you're desperately or
terminally ill, phase-three testing is a waste of time.
   There is a question whether the administration's proposal
to lift the phase-three requirement would require
congressional approval.  Probably not, but the political
 argument for submitting a bill is that it would commit
Congress to supporting the new system.  Committee chairmen
such as Congressmen Henry Waxman and Ted Weiss have made
 careers from catching FDA officers in regulatory traps, but
with many AIDS sufferers in both their districts it may be
 time for them to support reform.
   It is clear in any event that with this proposal in the
balance the administration has arrived at a turning point in
the war on the country's worst diseases.  Patients, the
casualties in this war, already endure sufficient burdens.
The most compassionate decision would be to relieve them of
the weight of phase-three testing, and move their battle
forward.

[This article is made available here by Dow Jones Co. for the 
personal and non-commercial use of callers to this bbs, in the 
hope that it will be of some help to those who are suffering 
from the disease and others who are seeking to help them.]