Subject: (Editorial):  The FDA for Itself
Date: Published: 10/13/88 103 lines
Source:  Wall Street Journal.  Copyright Dow Jones & Co. Inc. 

    REVIEW & OUTLOOK (Editorial):  
    The FDA for Itself

    It has become a battle between people who have all the
 time in the world and people who have little time left in
 their lives.  On Tuesday, AIDS demonstrators assaulted the
 headquarters building of the Food and Drug Administration
 outside Washington.  They lay on the ground with hand-painted
 tombstones at their heads.  "I Died for the Sins of the FDA.  "
 "I Got the Placebo."
    It won't work.
    The current effort by the Reagan administration to
 telescope the three phases of new-drug testing into two,
 thereby speeding the availability of drugs for
 life-destroying diseases, is on the verge of collapsing.
 Career bureaucrats inside the FDA have dug in their heels to
 insist on provisions that essentially would eviscerate the
 initiative's goal of faster approvals of drugs to treat
 life-threatening diseases.  Moreover, FDA Commissioner Frank
 Young, though he personally supports the change, is said to
 have concluded that he can't overcome opposition within his
 own bureaucracy.
    Unless this standoff is resolved, it is likely that the
 Presidential Task Force on Regulatory Relief, which is
 chaired by George Bush and which asked the FDA to make these
 changes, will drop the initiative.  That would be a victory
 for the FDA bureaucracy and its supporters.  It would be a
 stunning defeat for AIDS sufferers and victims of myriad
 other awful diseases.
    The argument for the Bush initiative is strong.  Drugs now
 pass through three FDA testing phases:  the first to prove
 safety, the last two to prove efficacy.  The FDA did a study
 to track the record of approvals through these three stages.
 Some 70% of all submitted drugs proved to be safe for human
 use in phase one.  The most revealing finding, though, was
 that almost 82% of the drugs that displayed efficacy in the
 relatively short phase-two tests also survived the
 much-longer phase-three trials that are the main cause of the
 much-criticized delays.  The Vice President's task force
 concluded that if a drug is for a disease that is killing
 people, a successful phase-two trial would suffice.  It said
 that a drug proved safe in phase one and showing hope in
 phase two should be sold to these desperate patients.
 Surveillance and testing of the drug would continue after
 marketing.
    Now, however, instead of simply relying on a drug-approval
 record already established by existing FDA procedures, the
 agency's bureaucrats are using the initiative as a pretext to
 "improve" the testing system.  The department's memorandum on
 the proposal shows that the bureaucracy wants to be involved
 more deeply and earlier in the design of research:  "The FDA
 will consult early with the sponsor on the design of animal
 studies"; "the FDA will actively consult at the end of early
 phase-one clinical testing, on the proper design of phase two
 controlled clinical trials"; "FDA will participate in focused
 research efforts in such areas as pharmacokinetics where the
 agency has special expertise."  And so on.
    So in return for letting go of the redundant phase-three
 requirement, the bureaucrats demand greater power over those
 parts of the earliest research they don't fully control now.
 This is the unacceptably Faustian deal the initiative's
 supporters believe they cannot make with the bureaucracy.
 Observers close to the testing initiative believe the
 resistance resides mainly in the Office of New Drug
 Evaluation, led by Dr. Robert Temple.  While claiming to
 support the change, Dr. Temple has said that drug approvals
 would depend on a "meaningful endpoint," a phrase with no
 standard definition and which has caused delays in the past.
    It is unfortunate that the full weight of this issue has
 fallen onto one beleaguered FDA commissioner, Dr. Frank
 Young, who needs to understand more clearly whom he and his
 agency work for.  FDA bureaucrats do not work for themselves,
 their own ideas of "science," the drug industry or the two
 Congressmen from Hollywood and Greenwich Village who rule the
 agency like some personal fiefdom.  The FDA's obligations are
 to people who are sick right now or soon will be.
    While there has been much effort to refocus the FDA toward
 patients the past eight years, there has been relative
 silence from one man:  Ronald Reagan.  President Reagan
 displayed considerable compassion for handicapped infants
 during the Baby Doe controversy.  The group that is suffering
 because of the FDA status quo is much larger -- the elderly
 who are spending their last years in the fog of Alzheimer's,
 the always-desperate victims of cancer, Tuesday's
 demonstrators and, yes, Baby Does with rare, wasting
 diseases.
    These people are a President's constituents too.  His Vice
 President is within an eyelash of significantly enhancing
 their hope of benefiting from American medicine and science.
 With a phone call to Dr. Young or Health and Human Services
 Secretary Otis Bowen, Mr. Reagan could help an extraordinary
 number of Americans who deserve better than to have come this
 close during his presidency, only to be left behind in the
 hands of the FDA's bureaucracy.

[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.]