Subject: (Editorial):  Finally, the Patients Benefit
Date: Published: 10/20/88 95 lines
Source:  Wall Street Journal.  Copyright Dow Jones & Co. Inc. 

    REVIEW & OUTLOOK (Editorial):  
    Finally, the Patients Benefit

    Exactly two years ago this week, we published an editorial
 titled, "AIDS Research:  Who Benefits?  " The government had
 just announced that a new drug called AZT had delayed death
 and reduced suffering in AIDS patients.  A federal telephone
 hotline was set up to handle calls from the AIDS community,
 and they came in at the rate of 1,000 a day.  The editorial
 commented:  "We assume that all those AIDS patients phoning in
 fully understand that the drug's success is preliminary, that
 it has toxic side effects, and that other side effects may
 show up later.  Knowing all this, they, like people with so
 many other life-destroying diseases, want to brave the risks
 and take the drug.  Many, however are told they can't have the
 drug.  The alliance of researchers and regulators says only it
 will decide who may benefit now from AZT and who may not."
    Yesterday, the U. S. Food and Drug Administration took the
 biggest step to date toward putting the American system of
 drug development back in the business of serving the nation's
 patients -- not itself, not the drug companies, not
 researchers, not some disembodied deity called "science."  The
 FDA announced a major revision of its rules for approving
 drugs developed to treat diseases that are life-threatening
 or severely debilitating.  A partial list of potential
 beneficiaries would include victims of cancer, AIDS, stroke
 and heart attack, Alzheimer's, Parkinson's, blindness,
 osteoporosis, and rheumatoid arthritis.
    The goal is simple:  to ensure that the discoveries of
 modern medical research reach the victims of such diseases as
 quickly as possible.  The current approval system requires
 three stages of tests for all drugs.  Under the new system,
 drugs intended for these serious diseases won't have to pass
 through the long third phase of tests if they show real
 benefit after phase-two tests.  Instead, they will be in
 hospitals and doctors' offices.
    Perhaps most important, the new rules explicitly recognize
 a concept that patients and their attending physicians have
 always understood -- balancing risks against benefits.  What
 this means is that a drug's real risks should not be allowed
 to overpower its benefits and prevent approval.  In practice,
 it should allow patients in the most extreme and desperate
 circumstances to take a long shot.  Ask almost any patient
 facing death or no prospect of gain, and he or she will say
 that a long shot is all they want.
    That is the promise of the FDA's new rules.  The reality
 remains to be seen.  In the past the FDA staff has relied on a
 system that invariably demanded that a drug show clear and
 convincing benefit, based on "definitive data," for a large
 percentage of research subjects.  The agency now has the
 challenge of turning into reality the official recognition
 that this standard is simply inappropriate for the most dire
 diseases.
    Pharmaceutical companies also carry a burden to make the
 new rules work for patients.  They will have to bring their
 most promising discoveries into the approval system
 expeditiously.  Some smaller companies with less experience in
 getting drugs approved may be reluctant to risk early
 rejection even under the new rules.  Both they and the FDA
 staff will have to show good faith in designing clear and
 honest tests for these drugs.
    FDA Commissioner Frank Young and the agency's officials
 deserve credit for committing themselves to such a
 significant change in longstanding procedures.  The nature of
 a bureaucracy, public or private, is to resist change as an
 unwarranted challenge to its competence and authority.  Even
 as late as last week, it wasn't clear that the agency's
 career officials would support the proposal.  By finally
 accepting a new approval system for this special category of
 drugs, the FDA's bureaucracy has the tools to pull American
 medicine forward, rather than hold it back.  The new rules, in
 effect, pledge the agency's officials to think more like the
 world's best-informed patients -- aware of all the scientific
 shortcomings and risks that any powerful therapeutic agent
 possesses, but also aware of the good it may do for some
 people.
    No doubt patients always have figured somewhere in the
 decisions of the triumvirate of the agency, the
 pharmaceutical industry and the research community.  But over
 the past 25 years the system lost sight of its main priority.
 Perhaps now, instead of being preoccupied with research
 statistics, reputation-building and constant fears of attack
 from publicity-seeking House investigations, the participants
 should be able to return to the purpose laid out succinctly
 by Hippocrates:  "I will exercise my art solely for the cure
 of my patients."

[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.]