PI Perspective #14
June 1994 View PDF
AIDS Research & Politics in 1994 -
Are You Better Off Today … ?
Sixteen months into the new Administration in Washington, it is
clear that the change of political stewardship has not made a major
difference for people living with AIDS. High hopes that the sympathetic
tone of the new Administration would translate into better policy,
faster research, or an improved prevention effort have come crashing
back to reality. Whether this is due to any fault of the Administration,
or just a consequence of unrealistic expectations, is open for debate.
At the very least, there is little evidence that this Administration
has made AIDS any more of a national priority than the previous
one. And, depending on how it addresses five key issues facing AIDS
research today, it could eventually earn the reputation of doing
more harm than good.
The nightly news, and the President’s attention, is dominated
by crime, the economy, foreign and military affairs and general
health care concerns. While the Administration is struggling to
convince the public that a few more million dollars for CARE and
research programs is a big step forward, we learn that $750 million
in new money has been allocated to support U.S. companies manufacturing
lightweight color computer screens, that billion-dollar military
spy satellites are lining up on the launch pad, and that the 1995
budget for nuclear weapons development exceeds $24 billion. There
has been no Bill Clinton “Town Meeting on AIDS.” The
President has appeared twice on MTV, but has yet to take questions
at an AIDS service organization. Leaders of the automotive industry
have been escorted into the Oval Office to collaborate with the
government on making more fuel efficient cars, and Hollywood stars
have been given phone lines and office space in the White House,
but AIDS researchers have had no such access and no such invitation.
At best, AIDS has provided an occasional photo-opportunity for a
few people to pose with the President.
After collecting big money in the campaign on the promise of important
things to come in AIDS research, the Administration has since spent
an inordinate amount of time explaining why Clinton really didn’t
mean it when he called for a “Manhattan Project” on
AIDS.
We do have a new “White House” AIDS policy coordinator,
but her first several months in office suggest that she has been
given little if any authority to take action on anything, let alone
carry the authority of the White House in her job. Similarly, we
have a newly appointed Assistant Secretary of Health and Human Services
(who controls both the NIH and the FDA), a new head of the National
Institutes of Health, and a new director of the Office of AIDS Research—all
fine men (yes, once again they are all men, white ones at that).
New committees, task forces and advisory bodies are being formed,
taking over where the last bunch of committees, task forces and
advisory bodies left off. One bright spot is a new enthusiasm for
strategic planning—largely a response to activist pressure
and apparently a new idea in medical research—but few people
engaged in the process have ever seen, let alone studied or written
a strategic plan before. Some reports from the first budget and
planning meeting on AIDS research suggest that, because there is
still no real process for setting priorities, the effort was little
more than a review of last year’s expenditures.
AIDS research is reeling and in serious need of detoxification.
Because of change in personnel, combined with growing public pressures
and uncertainties, five key issues are up for grabs. Each represents
both an opportunity for positive change as well as a risk of making
major missteps. It is a situation ripe for a five-step program.
Problem 1
The overall AIDS research effort is now being led by people who
have neither AIDS experience nor expertise in management skills
required or called for in the Office of AIDS Research reorganization.
Solution Step 1: The Office of AIDS Research should
seek the advice of proven experts in strategic planning, project
management, efficiency and quality assurance. As new people have
been given responsibilities for managing the AIDS research effort,
they have been selected on the basis of scientific resumes, not
their knowledge of AIDS or skills in major enterprise management.
If they get the proper professional support and guidance, they might
be effective and bring fresh thinking to the effort—hopefully
with a minimum of time lost while “getting up to speed.”
If they assume they know how to write and pursue a strategic plan,
establish balanced, long-range priorities, assure process efficiency
and quality and effectively oversee the management of research,
we are headed for trouble. These skills simply are not part of a
scientist’s background and are all too often looked down upon
as “process skills” by “real” scientists.
On the contrary, the ability to plan and manage effectively in the
thicket of AIDS research is fundamental to success.
Problem 2
Key players at the NIH seem convinced that a return to the laboratory
to pursue “basic research” rather than clinical questions
is needed. If they are wrong, research and spending priorities could
be misdirected for years to come. Currently, both clinical research
programs and drug discovery programs are being targeted for possible
funding reductions.
Solution Step 2: Make no changes in the balance
of priorities until scientists, policymakers and advocates agree
upon a clear set of terms and definitions and an in-depth, independent
and honest assessment of the real needs of AIDS research takes place.
No field is more rife with buzzwords and fashion-frenzy than AIDS
research. One of the current fashions rushing through the NIH is
a call for a return to “basic research.” Wise scientists,
bureaucrats and activists nod gravely in agreement. But each person
in seeming agreement actually has conflicting definitions of “basic
research”—definitions remarkably consistent with each
one’s own field of interest:
- To the new NIH director and other cell biologists, “basic
research” means more on fundamental laboratory exploration
to tied to any specific disease and not geared to development
of any particular therapy. They argue that this is the font of
real innovation. Perhaps so, though critics argue this perspective
may be motivated more by self interest regarding funding streams
than by science.
- When virologists applaud “basic research” they envision
investigation into the molecular biology of HIV (the study of
the virus itself). This, they feel, is where we will learn to
cripple the disease. Possibly, but aren’t these the same
people who have told us for years that we have learned more about
the biology of HIV than any other organism in history? How much
more do we need to know and where will it get us?
- Other scientists argue that “basic research” is
the study of the immune-pathogenesis of HIV, how the virus interacts
with and breaks down the immune system. Indeed, this is very important,
but it’s possible that the answers will be found most quickly
through clinical studies involving people, rather than through
basic laboratory explorations. The opportunity for such clinical
studies will be lost if the pendulum swings unduly in favor of
more laboratory, and less clinical, work. Another view is that
giving excessive attention to the study of pathogenesis might
itself be unproductive, since history records that most diseases
which have been conquered were beaten long before scientists fully
understood their workings.
- When some of the community voices call for “basic science,”
their description sounds suspiciously like a demand for more rapid
preclinical drug development. Important indeed, but definitely
not what the others are talking about when they use the term.
So far, the call for “basic research” sounds more like
confusion than consensus. The outcome of the debate will determine
the priorities, spending, and thus the programs, of the next several
years of research. No substantive change of direction should be
taken, especially in an environment of limited resources, without
first conducting a critical, independent and impartial assessment
of the state of knowledge and the true needs of AIDS research. If
the balance shifts too far in favor of “cell biology”
benchwork or HIV molecular virology, we will see less emphasis on
developing and testing new therapies for the next 5 to 10 years,
and consequently fewer new emerging options for people struggling
against AIDS. This could spell disaster for the generation of people
who will face the greatest need for therapy in the next five years.
People with AIDS will be best served by a program which seeks to
maintain a reasonable balance between bench science, pathogenesis
and clinical research.
Problem 3
Some voices in and outside of government believe that no therapeutic
approach currently in development is likely to be a cure for AIDS.
Thus, they argue for a de-emphasis of drug development in favor
of developing completely new solutions.
Solution Step 3: Before de-emphasizing therapy
development, let’s try a concentrated effort to speed the
development of the most promising drugs already in the labs. Recent
data from protease inhibitor studies are so intriguing that some
scientists believe what’s needed is an all out engineering
effort to maximize the benefits of this new class of drugs, along
with an accelerated effort to test them in combinations. The striking,
if time-limited, response seen to one of the protease inhibitors
may provide the first hint that antiviral research is indeed on
the right track, a notion many had begun to lose faith in. If so,
then the greatest benefits in the near term are likely to come from
developing this model, rather than looking for completely new approaches.
Similarly, many avenues of therapy development are floundering
not for lack of promise, but for lack of sponsorship by government
or industry. These include products which target cellular, rather
than viral mechanisms critical to viral replication, integrase inhibitors,
LTR inhibitors and other approaches targeting the virus in novel
ways. Moreover, there is a backlog of interesting products in the
field of immune-based therapies which receive little or no attention
or funding. There is an unmet need for new forms of collaboration
between government, industry and academia in developing new avenues
of therapy—and little evidence that the Administration is
moving to meet it.
Problem 4
AIDS research is increasingly characterized by rapid swings in belief
about clinical trial design, diagnostic markers and drug development
strategies. The first time a belief or concept fails to produce
significant results, it falls from fashion, leaving patients, physicians
and drug developers in a state of confusion. Currently, that state
of confusion is threatening the hard-won right of people to early
access to promising new therapies.
Solution Step 4: Understand that uncertainty may
remain a hallmark of AIDS for many years to come. Adhere to logical,
common sense approaches to research, at least until they are proven
conclusively wrong—stop changing direction with each shift
in the scientific breeze. When small changes in CD4+ counts in a
single study failed to predict the benefit of therapy, a lemming-like
herd rushed for the cliffs declaring that CD4+ counts were meaningless,
and that drugs shouldn’t be approved merely on the basis that
they improve CD4+ counts and quality of life. In another startling
turnaround, mechanisms like parallel track and accelerated approval
are under attack by voices urging restricted access to new therapies
and demanding long, survival-based studies before further therapies
are licensed for sale. While anyone is welcome to choose such beliefs
to guide his or her own therapy, many people with AIDS want the
opportunity to access promising experimental therapies while they’re
still around to try them. If scientists or regulators continue to
place emphasis on quantifying small differences between today’s
at best modestly useful drugs, no one will benefit and the costs
will drive industry away from AIDS drug development. Instead, such
drugs should simply be available for those who choose to use them,
while the profits gathered from their sale must be invested in developing
a better generation of therapies and new diagnostic markers.
Long-term studies which seek to prove survival benefits of mediocre
drugs have proven all but impossible in the rapidly-changing field
of AIDS research. People refuse to stay in such studies for more
than a year or two. Care providers are unwilling to encourage patients
to remain on a therapy which is failing just to provide data for
these studies. Ethics demands that people be permitted to switch
therapies when the existing regimen fails. Long-term studies measuring
survival will only become practiced when we have a generation of
drugs capable of producing long-term beneficial activity without
major side effects. We need no further studies to prove that we
are not yet at this point.
Over time, the restructuring of the Office of AIDS research will
almost certainly lead to greater focus and less duplication of effort—but
that change will not be noticed quickly. While the train moves ever
so slowly forward, we must make sure that people with AIDS continue
to have the earliest reasonable access to therapies which might
help them. The fundamental right of freedom of choice, won in hard
battles fought in the late 1980’s, must not be lost in a debate
over the conflicting results of studies conducted on today’s
mediocre generation of drugs. No bureaucrat, whether inside or outside
the community of people with AIDS, should be permitted to threaten
this right.
Problem 5
AIDS activism is dead, dying or increasingly entwined within the
bureaucracy. The healthy spirit of criticism which produced so much
progress in the past has grown quiet as individual activists have
passed away, become caregivers or burned out. There is a sense of
complacency driven at least in part by the mistaken assumption that
our interests are being taken care by the new Administration.
Solution Step 5: Renew the aggressive spirit of
AIDS activism which was so productive in the past. AIDS research
is at a crossroads. A year ago, people with AIDS hoped that the
start of a new Administration would lead to newly invigorated, better-managed
and more aggressive programs of AIDS research. That moment of opportunity
has come and gone with only modest changes to show for it. There
will be no new major programs or initiatives for at least another
year. Regardless of its more supportive rhetoric, the Clinton Administration
should feel no less pressure and demand from the streets than the
Bush and Reagan Administrations. More, not fewer people are dying
of AIDS today than at any time in history. Will Clinton be content
to be remembered as the President on whose watch we saw the greatest
number of deaths from AIDS? Kind words are not enough, nor are modest
improvements in funding. To paraphrase a line from candidate Clinton,
we will never see a cure for AIDS until people find the will and
the courage to implement change. Change, in this case, begins with
recognizing that the current effort is still not good enough.
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