Subject: Will the FDA Revert to Type?
Date: Published: 12/12/90 (187 lines)
Source: Wall Street Journal. Copyright Dow Jones & Co. Inc.

    Will the FDA Revert to Type?
    ----
    By Daniel Henninger

    AIDS has become one of the most intriguing public-policy
 phenomena of our time, raising many questions about the way
 the U.S. conducts medical research, regulates that research
 and pays for it.
    The mere fact that AIDS muscled its way to the front of
 the research queue merits attention. In the absence of
 contrary evidence, it suggests that the most efficacious
 means of getting the public-sector Colossus to move at all is
 gross civil disobedience. A more troubling question persists,
 though. In response to AIDS, the bureaucratic Colossus has
 moved in unexpected ways and with remarkable speed, but will
 it revert to its usual sluglike state when AIDS is cured or,
 more likely, when the suffering from AIDS becomes as "normal"
 as that from cancer, Alzheimer's or stroke?
    After years of watching the way the Food and Drug
 Administration confronts extraordinary events, I'll offer a
 skeptical answer: Exercising its famously "flexible"
 interpretation of the statutes, the FDA will hold the door
 open while the politically protected AIDS effort passes
 through; then it will close the door slowly on researchers
 and advocates for other diseases. In other words, the
 remarkably radical war on AIDS may be a unique event. If the
 changes wrought by AIDS on medical research and regulation
 aren't institutionalized or codified, the heavy stone of
 accomplishment will roll down the mountain.
    These matters are worth considering now because it isn't
 widely appreciated just how far forward attitudes toward
 research and regulation have moved in the past several years.
 In December 1985, for instance, this newspaper editorialized:
    "We continue to wonder why the medical-research and
 regulatory establishment is so adamantly opposed to serving
 this population of {cancer} patients. What is wrong with
 letting dying or seriously ill patients gain
 physician-supervised access to promising therapies even
 before the very last `i' is dotted years hence by the Food
 and Drug Administration's official approvers? If the research
 and regulatory community doesn't consider some way of
 adapting itself to speed up patient-access to promising
 therapies, then cancer's curators shouldn't be surprised if
 sick people soon start assaulting a system that for all its
 achievements, prescribes little more than patience."
    AIDS, of course, has displaced cancer as the nation's most
 publicized disease, but something more substantial has also
 changed. That was apparent at a recent conference in
 Cambridge, Mass., sponsored by the American Society of Law
 and Medicine, the Center for the Study of Drug Development
 and the Harvard AIDS Institute. It included researchers,
 physicians, patient advocates, lawyers and, almost as an
 afterthought, FDA officials. To anyone who has followed the
 debate over patient access to experimental drugs, the
 gathering's discussion topics suggest a sea change:
    -- Clinical Trials and Patients' Right of Access to
 Investigational Drugs.
    -- Expediting Access: Reforming the Conventional Basis for
 Drug Approval.
    -- Are Controlled Clinical Trials Necessary?
    -- Alternatives to the Clinical Trial.
    -- The Conflict between Science, Autonomy and Civil
 Liberties.
    -- Risk and Regulation: The Patient's Perspective.
    The sentiments heard at this conference are hardly unique.
 Anyone who doubts the significance of the changed attitude
 should read the paper signed by 22 medical statisticians in
 the Nov. 8 New England Journal of Medicine that proposes
 alternative methods for assessing the results of clinical
 trials for AIDS drugs. Statisticians are the pontifical Curia
 of medical research.
    Several FDA officials came to Cambridge, and one told the
 conference she'd heard that the organizers had considered not
 bothering to invite the agency. But ironically it wasn't an
 AIDS advocate but another federal official, Daniel Hoth of
 the National Institutes of Health, who threw down the
 gauntlet and challenged the drug agency in the most direct
 terms. "Who is deciding?" he asked.
    "What constitutes a safe and effective drug depends on
 where you sit," said Dr. Hoth, who heads the AIDS effort at
 the National Institute of Allergy and Infectious Diseases.
 The next day, when the FDA's decision in July to withhold
 approval for the kidney-cancer drug Interleukin-2 (now widely
 approved in Europe) came up in discussion, Dr. Hoth
 elaborated: "There are a lot of points of view and they
 aren't always represented in the process now" -- a situation
 he called a "regulatory barrier." He concluded: "The key
 issue is who makes the risk-benefit judgment and should there
 be an appeals mechanism?"
    Historically, the FDA has been obsessed with risk. This is
 so because over the past 75 years occasional, highly
 publicized accidents involving drugs have given congressmen a
 pretext to announce, with the cameras rolling, that the
 agency is too sloppy with risk. Whether the general public
 ever feared risk as much as the politicians claimed during
 these controversies is a good question. Suffice to say that
 for at least 20 years the subject has been Naderized:
 Nader-like groups would identify risks from particular drugs,
 and supportive newspaper reporters in Washington would vilify
 the FDA and offending drug companies for "negligence." For
 years, no one, least of all dying patients, could overcome
 this risk-based demagoguery. The assumptions supporting an
 absolutist view of risk, however, are being re-examined.
    A former FDA commissioner, Arthur Hull Hayes, told the
 Cambridge conference: "A risk-free society would so restrict
 choices that there would be no choices at all." He wondered
 whether the FDA could be changed to not only protect the
 public "but to benefit the public." As recently as three
 years ago, anyone who went before such a conference and
 suggested a distinction in the FDA's role between protecting
 and benefiting the public -- the fulcrum of the argument over
 risk -- would be derided for not knowing "how the system
 works." No one derided Dr. Hayes at this conference. He also
 noted sardonically that the media had done a "flip-flop," now
 criticizing anyone who stands in the way of getting
 experimental drugs into the hands of patients, or at least
 AIDS patients.
    The whole system of AIDS-related research is now canted
 toward providing benefit to patients as quickly as possible.
 The design of clinical trials, participation rates for those
 trials, opposition to placebos, various "fast-track" or
 "parallel-track" regulatory schemes -- all are now thought of
 primarily in terms of benefiting the sick. The speed of the
 changes inevitably will create some problems. So-called
 community-based trials, for instance, which are conducted
 outside traditional academic research, often give patient
 groups a role in designing the program. This potentially
 could cause a too-many-cooks problem. "Empowerment"
 overwhelms science.
    If the FDA really wanted the status quo to prevail over
 these trends, its best strategy probably would be to give
 them free rein. The odds are that another accident would
 occur, perhaps years hence when passions have cooled, and the
 congressional demagogues would reimpose the old paternalism.
 The FDA's performance in Cambridge certainly suggested no
 inclination to assist a fundamental change in the agency's
 mission.
    Ann Witt, associate counsel for the FDA, said the agency
 maintains "a bias toward protecting the community as a whole
 rather than access for individuals." She said the FDA statute
 "doesn't give individual patients the right of access."
 Indeed, "the statute requires the FDA to be the gatekeeper."
 She cited Laetrile.
    This is an unapologetic -- and in the political context of
 AIDS, maybe even bold -- defense of the status quo. It also
 suggests how the operating philosophy of a bureaucracy can
 become largely immune from intellectual challenge. On its
 own, the FDA will never change substantially, no matter how
 loud the protests of patients, because its flawed logic
 assumes that the immediate needs of seriously ill
 "individuals" conflict with those of "society." This cold
 conclusion is familiar to people with AIDS, cancer,
 Alzheimer's or any other socially burdensome disease, which,
 they discover, erects a wall between them and "society."
    We have arrived at a critical juncture in the debate over
 drugs, patients and the FDA. Judging from the sentiments
 heard at the Cambridge conference, which generally reflected
 the thinking that has developed around this subject for the
 past two years, the intellectual debate is essentially over.
 The dominant view of the 1990s is that in matters of drug
 therapy, questions of benefit now deserve equal standing with
 risk. The key issue outstanding is, Who decides?
    The answer to that is political. And since the FDA won't
 act on its own and since Congress created the risk-obsessed
 status quo, the lead responsibility falls either to Health
 and Human Services Secretary Louis Sullivan or to President
 Bush. Indeed the reform movement has reached this point in no
 small part because Vice President Bush quietly encouraged it
 during the Reagan presidency.
    Absent political intervention, it's likely that the sands
 of the status quo will wipe away most of the gains of the
 AIDS movement. If that happens, other patients who are deeply
 or hopelessly ill may decide to discuss with AIDS activists
 the methods of redress necessary to focus the drifting
 attention of their government.
    ---
    Mr. Henninger is deputy editor of the Journal's editorial
 page.

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