Subject: REVIEW & OUTLOOK (Editorial):  New Ideas for New Drugs
Date: Published: 12/28/88 100 lines
Source:  Wall Street Journal.  Copyright Dow Jones & Co. Inc. 

    REVIEW & OUTLOOK (Editorial):  
    New Ideas for New Drugs

    The federal Food, Drug and Cosmetic Act is 50 years old,
 but suddenly a number of voices in the American medical
 establishment are saying the time has come to go back to the
 drawing board.  We've been calling for a better, faster way of
 getting new drugs into the hands of patients for some three
 years.  It's heartening that the cause of reforming the
 drug-approval system is being joined by some of the people
 who have worked most closely with it.
    Jere E. Goyan, who was FDA Commissioner in the Carter
 presidency, recently published an editorial in the Journal of
 the American Medical Association titled, "Drug Regulation:  
 Quo Vadis?" The question Dr. Goyan put to his colleagues is
 whether they should fundamentally rethink the relationship
 between medical researchers and sick patients.  "What I am
 suggesting is the need for a re-examination of all of the
 assumptions on which the scientific requirements of the
 present system are based," Dr. Goyan wrote.  "In particular,
 we need to consider alternative study designs that allow the
 patient maximum hope for cure and the opportunity for some
 control over his or her destiny."
    President-elect Bush has put in motion one effort to
 address the concerns Dr. Goyan raises.  He has created the
 National Committee to Review Current Procedures for Approval
 of New Drugs for Cancer and AIDS. The committee will hold its
 first public meeting Jan. 4 at the National Institutes of
 Health.  One of the committee's members is Gertrude Elion.
 Earlier this year, Dr. Elion, a drug researcher with
 Burroughs-Wellcome, was awarded the Nobel Prize in Medicine
 along with her Burroughs co-worker, George Hitchings.  Dr. 
 Hitchings and Dr. Elion have some well developed opinions
 about the FDA. 
    "My estimate is that the FDA has killed 100,000, maybe a
 million people," Dr. Hitchings said at a company facility in
 Greenville, N. C., shortly after being named a Nobel winner.
 He was referring to delay in the FDA's approval in the 1970s
 of an antibacterial agent called Septra.  "We were five years
 behind the rest of the world," he said.  "We could have been
 saving lives."  His estimate, reported by the AP, is that the
 drug had saved a half-million lives elsewhere before it was
 available in the U. S. 
    Commenting on an anti-leukemia drug they developed, Dr. 
 Hitchings said:  "We went from synthesis to the commercial
 drug in three years.  That is absolutely impossible today."
 Dr. Elion thinks AIDS is shaking up the system:  "It may be
 the first break in the armor of federal regulations, and I
 believe it could lead to quicker release for drugs against
 cancer."  Ironically, the agency cites its fast approval of
 Burroughs's AIDS drug, AZT, as proof of its commitment to
 reform.
    Until recently, the medical-research establishment cringed
 when critics of the system talked like Dr. Hitchings about
 FDA delays "killing" people.  Well, since writing his JAMA
 editorial, Dr. Goyan has said that any reform effort should
 draw some of the agency's critics into the process.  The
 "critics" we'd nominate to any such panel would be patients
 and their families.  Let defenders of the status quo explain
 to people with cancer, Alzheimer's or AIDS why redundant
 efficacy testing, in which half the patients get a placebo,
 doesn't constitute "killing" in the name of FDA-mandated
 medical statistics.  Controlled trials of new AIDS drugs in
 New York City are becoming virtually impossible because
 patients refuse to risk getting the placebo.
    Dr. Samuel Thier, president of the Institute of Medicine
 (which is an arm of the National Academy of Sciences), puts
 it bluntly:  "We are at the stage where many agree something
 needs to be done."  Citing the fact that beta blockers (for
 hypertension and myocardial infarction) and calcium channel
 blockers (for angina), were available overseas first, Dr. 
 Thier said recently, "We pursued our conservative caution,
 but it meant that our population did not have very useful and
 important drugs."
    When the FDA announced in October that in the case of
 life-threatening diseases it would consider dropping the
 third phase of its current testing requirement, it also for
 the first time formally laid down what could be the
 cornerstone of any new drug-approval regime.  That cornerstone
 is the concept of risk vs.  benefit.
    Risk-benefit, a phrase from the dry language of economics,
 is instinctively understood by anyone in pain or whose life
 is in danger.  AIDS patients have driven home to the U. S. 
 medical and political establishment what enormous risks human
 beings in death's grip will take to gain relief or respite.
 George Hitchings and Gertrude Elion understood 40 years ago
 that offering benefit to patients at risk was the core
 purpose of their skills, and this year the Nobel committee
 recognized that effort.  Now the evidence mounts that the U. S. 
 research establishment and its regulators have also begun to
 rediscover that their first purpose is to serve the sick.

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