TIME: Almanac 1993/TIME Almanac 1993.iso/time/101292/10129930.000

Text File | 1993-04-08 | 13.6 KB | 267 lines

                                                                                SOCIETY, Page 66

Your Money or Their Lives


Patient advocates are learning from AIDS activists how to work
the system

By DICK THOMPSON/WASHINGTON


    With $150 in the bank, the Families for Alzheimer's
Rights Association lacks the muscle of the American Cancer
Society, whose budget totaled $346 million this year. But then
again, the American Cancer Society doesn't have George
Rehnquist. The 69-year-old retired electrical engineer created
the tiny lobby to fight for government approval of the
experimental drug THA. The treatment has yet to meet the Food
and Drug Administration's standards for safety or effectiveness.
But ever since THA eased his wife's Alzheimer's symptoms,
Rehnquist has been crusading to get the drug into patients'
hands -- legally or not. One day he is instructing people on how
to smuggle the drug from the Bahamas. The next day he may be
speaking, invited or not, at FDA hearings, sending out a barrage
of letters and organizing other families to do the same. Later
this month Rehnquist will lead a demonstration outside the FDA's
offices. "I have to fight for my wife and my daughters," he
says, "and all those people who call me and are hurting."

    If the Muscular Dystrophy Association and the March of
Dimes and the American Cancer Society built the model for
patient groups, advocates like Rehnquist are busy shattering it.
They are, for a start, less polite about their demands on the
medical establishment and more combative in their tactics.
"We're becoming nasty, and I love it," says Abbey Meyers,
executive director of the National Organization for Rare
Disorders. The new generation of patient advocates has borrowed
the medical activism first mastered by the AIDS lobby, in an
effort to educate patients, inform physicians, influence
politicians and pressure the pharmaceutical industry over
everything from development to government approval. They are
responsible for redirecting entire branches of biomedical
research -- prompting some scientists to warn of wasteful,
dangerous detours that will end up hurting the very patients the
advocates aim to save.

    Patient pressure groups have been a potent force in
American medicine for decades. The 13 specialty centers that
make up the National Institutes of Health, the epicenter of
America's biomedical-research enterprise, are monuments to
special-interest advocacy. Polio vaccines grew out of pivotal
research funded primarily by the March of Dimes. But as the
organizations grew, under the leadership of professional
lobbyists and fund raisers, they opened themselves to the charge
that they had lost touch with their grass roots and become a
part of the system they were supposed to be influencing.

    The new patient groups are often run not by professionals
but by victims of the disease or their close relatives. "It's
not your typical eight-hour-a-day job," says former beer
salesman Ron Brazeal, executive director of the United
Leukodystrophy Foundation, who lost two sons to the disease,
which is a rare genetic neurological disorder. Such groups
devote little time to the kind of sympathy campaigns that have
characterized the March of Dimes or the Jerry Lewis Telethon for
Muscular Dystrophy. Instead they depend on their own highly
committed members for operating funds.

    Unlike the established advocates, these groups are rarely
linked to the scientific establishment. They not only lobby
Congress for money, they tell scientists how to spend it.
"What's special about these groups," says National Institutes
of Health director Bernadine Healy, "is that they are primarily
driven by the patients and their loved ones. It makes them more
independent from scientific and medical communities and less
under their control."

    The main target of the groups is a federal research
enterprise perceived as being more interested in pursuing
intriguing scientific questions than easing pain or finding the
cures for afflicted patients. "The National Institutes of Health
is a government institution that is supposed to be responding
to the needs of its people," says Tom Sheridan, a lobbyist who
began working with AIDS groups and now has other
patient-advocacy clients, "and it hasn't always done that."

    The AIDS lobby led the crusade to force the NIH to change
its ways. Fully 10% of the NIH's $8.9 billion budget currently
goes into AIDS research. The FDA created the Office of Special
Populations just to handle the demands of the AIDS lobby.
Activists have become so well informed that they now sit on many
government advisory panels. Such recognition came only after
years of confrontation: demonstrating outside the White House,
shouting down the President at campaign stops and draping the
home of conservative Senator Jesse Helms in a condom-shaped
balloon. ACT UP members blockaded Wall Street and broke into the
headquarters of Burroughs-Wellcome to demand, and get, a
reduction in the price of the AIDS drug AZT.

    Women's health groups have been especially effective in
following the ACT UP model. They pressured the NIH to create an
Office of Research on Women's Health and forced a re-examination
of the practice of excluding women from clinical trials. Last
spring breast-cancer activists deluged Congress with 650,000
letters from women demanding more research into the causes of
the disease. Funding has jumped from $60.9 million five years
ago to $132.7 today. "Women saw what could be done," says Fran
Visco, a trial attorney in Philadelphia and president of the
National Breast Cancer Coalition. "The more noise they made, the
more successful they became. We decided we just couldn't be
polite anymore."

    Other groups of cancer patients have also broken out from
the umbrella of the American Cancer Society. Lloyd Ney, a
73-year-old retired engineer, learned in 1984 that he was dying
of prostate cancer. Told he had just months to live, Ney went
to Canada in search of an experimental treatment -- and found
the hospital waiting room filled with Americans. From that
encounter came not only the treatment that may have added years
to his life but also a mailing list for PAACT, or Patient
Advocates for Advanced Cancer Treatments. Eight years later, Ney
continues to work out of his basement, communicating with a
membership of 12,000 and managing a budget of $750,000. He is
credited with helping get the once experimental treatment
approved in the U.S., and now spends his time lobbying for new
therapies and responding to the hundreds of inquiries he gets
every week about prostate-cancer treatment.

    Other radicalized patients arrived at their activism by
similar routes. Most of them suffer from diseases that are rare
or difficult to diagnose, which means that they were shuttled
from specialist to specialist, leaving a trail of money and test
results but no answers or cures. By banding together and sharing
information, patients become informed consumers. Many groups
have a Rolodex of scientists and physicians who can be called
upon to educate a local doctor confronting his first case of a
rare disease. Information campaigns help improve diagnosis,
which in turn gives public health officials a more accurate
sense of the impact that even rare diseases have.

    Perhaps the most successful awareness campaign has been
conducted by the Lyme Borreliosis Foundation. In 1988 Lyme
disease was scarcely known even in the area around Lyme,
Connecticut. Karen Forschner, who had the Lyme-disease symptom
of a bull's-eye rash during pregnancy, started the foundation
with her husband Tom shortly after the birth of their only
child, a son who was finally diagnosed with the disease. "Using
the media is definitely a part of our strategy," says Tom
Forschner. The husband-and-wife team has captured the attention
of all the daytime talk shows and most newspapers and magazines.
By 1990 a Gallup poll found that Lyme disease was recognized by
88% of the U.S. population. "We lived 40 miles away from Lyme,
and Karen's physicians didn't recognize her rash," says Tom.
Their son died last year of the disease. "Awareness would have
made a huge difference."

    Information campaigns are just the starting point. Medical
researchers acknowledge that their work very often proceeds
without anyone taking a broad view. "The approach to diseases
in general has been sort of haphazard," says Donna Brogan,
chairperson of the biostatistics division at Emory University's
School of Public Health and a member of the research task force
for the National Breast Cancer Coalition. By organizing their
own scientific meetings, advocates help assess the state of
research for a particular disease and look for areas that need
strengthening. "That's unique to them," says NIH director Healy.
"They are setting bold, far-reaching goals."

    The drug industry has also felt the heat from new pressure
groups. Abbey Meyers of the National Organization for Rare
Disorders is acknowledged as the force behind creation of the
Orphan Drug Act. This federal statute provides incentives for
companies to develop drugs for rare diseases that might
otherwise be overlooked by firms seeking more lucrative markets.
Meyers is now back in Washington lobbying for a revision in the
law. She wants to close a loophole that has allowed companies
to reap windfall profits and leaves the orphan drugs so
expensive that a new treatment can be out of the reach of most
American families.

    The most profound impact of the advocacy movement has come
within the Food and Drug Administration. The agency has for
decades held to the rigid standard that new drugs must be
unequivocally proved to be both safe and effective. But in the
wake of intense lobbying, the FDA will now consider granting
conditional approval to experimental treatments for terminal
diseases for which there are few or no medical alternatives.
This is a radical shift for the nation's pharmaceutical
watchdog. Drugs conditionally approved will be closely monitored
and withdrawn if they prove to be too toxic or ineffective. "We
may be wrong," says FDA Commissioner David Kessler, "but with
life-threatening illnesses, those risks are acceptable."

    Some scientists are worried about the growing influence of
patient groups, particularly their success in persuading
Congress to micromanage research. In the past, elected officials
were reluctant to give detailed directions to scientists. But
for aggressive patient lobbyists, the lawmakers were an easy
target. Activism on the front lines of medical research plays
well to the hometown constituency. Now attached to every check
from Congress is a growing list of what the NIH must do -- even
while the total allocation remains roughly the same. The only
way for the NIH to follow Congress's orders is to eliminate
existing programs and transfer the resources. This year's budget
for the National Cancer Institute, for example, contains an
order to increase by more than one-third spending on breast-,
cervical- and prostate-cancer research. Yet congressional
funding for the institute did not even keep pace with inflation.

    Many NIH watchers are now concerned that responding to
congressional whims will undermine America's biomedical-research
structure. "We're seeing the pie being split up into smaller and
smaller segments," says David Moore, an official with the
Association of American Medical Colleges. "Some of these groups
have to be cautious," says John Seffrin, CEO of the American
Cancer Society. "They could advocate for major shifts in funding
in ways that on the surface makes sense but in the long haul do
great violence to the scientific effort. It raises the prospect
that these precious resources can be wasted."

    Researchers complain that junk science is interfering with
real science and threatens to disrupt progress toward actual
cures. Durland Fish, a Lyme-disease expert at New York Medical
College, has been appalled at the studies put forward as science
by clinicians funded by the groups. He recalls a recent meeting
at which a doctor claimed evidence of transmission of Lyme
disease through blood transfusion -- without ever establishing
that the recipient even had Lyme disease. The AIDS advocacy
group Project Inform ran its own trial of an experimental drug
called Compound Q, a purified protein derived from a
cucumber-like Chinese plant. At least one of the test subjects,
a man with AIDS, died from toxicity associated with the effort.
"It's very sad and it's scary," says Fish. "These groups have
a lot of political power now and a low appreciation of the
scientific method." Agrees Peter Scardino, chairman of the
urology department at Baylor College of Medicine: "These groups
have a very dangerous side to them when they take the place of
careful scientific investigations. They very deliberately say
to their members, `Don't let your doctor talk you into this or
that.' It takes a long time for us to talk these people down and
re-educate the patient."

    It is too soon to judge how much junk science will be
funded to appease these powerful groups. But the danger is that
allocation of scarce research resources will devolve into a kind
of political mud wrestling, with the spoils going to the most
powerful and not the most needy. George Rehnquist knows one
thing about the future. Two weeks ago, after he led Alzheimer's
family members in a demonstration outside the offices of the
FDA, Commissioner Kessler invited Rehnquist to come back this
month for talks. It's another step toward the day when THA will
be fully approved.