Subject: REVIEW & OUTLOOK (Editorial):  Relief From Suffering
Date: Published: 9/19/88 101 lines
Source:  Wall Street Journal.  Copyright Dow Jones & Co. Inc. 

    REVIEW & OUTLOOK (Editorial):  
    Relief From Suffering

    The government's effort to change the Food and Drug
 Administration's status quo for the release of promising
 drugs to terminally ill patients is at a critical stage.  The
 status quo is a system that announces breakthroughs in drug
 treatment, and then makes patients wait years for final
 government approval.  If federal officials get the revisions
 right, the new, speedier system could be a great source of
 hope for America's sickest citizens.
    A draft of the proposed reforms is circulating through the
 FDA bureaucracy, HHS Secretary Otis Bowen's office, OMB and
 Vice President Bush's Task Force on Regulatory Relief, which
 set the initiative in motion.  FDA Commissioner Frank Young
 has briefed interested private parties on the proposal.  One
 participant, Dr. Robert J. Beall of the Cystic Fibrosis
 Foundation, told us his group is "very much in support" of
 the initiative.  "This can only help those of us dealing with
 a life-threatening disease every day."  Briefly, under current
 practice, the FDA requires virtually all new drugs to pass
 through a three-stage approval system.  With the new system,
 the long third stage would be dropped if a drug for
 life-threatening diseases showed safety and benefit after the
 second testing phase.  All patients, not just research
 subjects, would gain access to the drug sooner.
    Abbey Meyers, of the National Organization for Rare
 Disorders, also supports the proposal but has doubts born of
 past experiences.  "Some of the FDA's division directors will
 resist this," she says, "and it may still be difficult to get
 drugs through those divisions."  A draft copy of the proposal,
 obtained by the Journal, suggests that concerns about the FDA
 bureaucracy are justified.
    On one hand, it shows that the FDA intends to take the
 virtually unprecedented step of officially sanctioning the
 concept of risk-benefit analysis.  The draft says, "The agency
 recognizes that safety is not absolute (i.e., no drug is free
 of risk), but must be assessed in light of what condition the
 drug treats.  This is particularly true in the case of drugs
 to treat life-threatening diseases, where drugs that are
 quite toxic may nevertheless be considered safe under the
 circumstances."
    Those words suggest that the agency intends to use a
 flexible standard of judgment in approving drugs for
 life-threatening diseases.  However, the draft also states
 that it will require "controlled trials that are capable of
 providing definitive data on the drug's safety and efficacy."
 If in fact the standard of approval is going to be
 "definitive data," the FDA will definitively destroy a lot of
 raised hopes.
    In deciding over the years which drugs work and which
 don't, the FDA and most of the medical research community
 have set common sense on the shelf and put their faith in the
 arcane discipline of statistics.  The reason this new proposal
 is on the table, however, is that the agency and its allies
 allowed "chi-square tests" and "probability values" to
 overrun patient needs and simple human values.  The rigid
 worship of "definitive data" is why AIDS victims have
 virtually laid siege to the federal medical establishment.
    These tortuous (and often misused) statistical standards
 may make sense in approving drugs to treat less severe
 conditions, such as anxiety or common infections.  The issue
 here is, will the FDA bureaucracy recognize a world in which
 people live entirely without hope as incurably ill patients
 for whom "efficacy" isn't a statistically elegant study but
 respite from their daily suffering?  
    Up to now, a drug's sponsor has always carried the
 overwhelming obligation to prove that a drug does little harm
 or "definitively" works.  But for this new category of
 extremely destructive or life-threatening diseases, with the
 quality of life supremely important to the victims and their
 families, the rules should put the burden of proof on the
 agency.  Substitute "relief" for "benefit" as the regulation's
 explicit goal, and the whole point of this exercise becomes
 immediately clear.  Relief is currently the "benefit" from the
 drug AZT, which has enormously improved the quality of life
 for fatally ill AIDS victims.  The FDA should be required to
 justify why a drug that may offer only symptomatic benefit,
 such as the Alzheimer's drug THA, shouldn't be put on the
 market.
    In a survey earlier this year, the American Medical
 Association asked doctors and the public the following
 question:  "A new federal regulation allows drugs that have
 not been fully tested to be used in treating patients with
 life-threatening diseases for which there is no effective
 treatment.  Do you favor or oppose this regulation?  "
    The percentage of public support was 68%; among doctors it
 was 88%.  It remains to be seen in the coming weeks if the
 Food and Drug Administration can for once offer a system that
 plainly and unequivocally provides the help desired by
 doctors who try to treat these destructive diseases and by
 the people who now must endure them.

[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.]