THE DOCTORNAUT ACT
A Proposal by FIM, the Foundation for Innovation in Medicine, To Accelerate Medical Discovery
October 1, 2003
Modern technology offers enormous promise in the battle against
disease, disability and other things that ail us. The barriers (costs
and risks) to evaluate this promise in clinical studies, the critical
step in medical discovery, have been and are increasingly prohibitive.
Attempts to remedy this situation have had minimal success.
It is urgent that a way be found to effectively diminish these
barriers to clinical research. The result would lead to accelerated
medical discovery, reduction of morbidity (suffering due to disease) as
well as the rate of mortality and help reduce health care costs both in
the short and long terms.
For these reasons, FIM, the Foundation for Innovation in Medicine,
proposes that the Congress enact the Doctornaut Act, which would permit
physicians to more freely volunteer for clinical studies than
non-physician volunteers. A Doctornaut is a physician who volunteers for
Phase II clinical research with fewer restraints than those of
non-physician volunteers, and who is permitted to take greater risks
than the former. Phase II is the early medical discovery phase in
clinical research, where a substance(s) is preliminarily evaluated for
efficacy and safety. It usually involves small numbers of volunteers.
Among the benefits that will result from Congressional passage of the
• Rapid acceleration of medical discovery followed by:
• Reduced morbidity (increased quality of life)
• Reduced rate of mortality from disease
• Timely application of innovative medical therapies to clinical practice
• Reduced health care costs
Among the issues that need to be addressed to insure the success of this proposal are:
• Costs and risks to clinical investigators
• Risks to Doctornauts
• Clinical investigator immunity from suit
• Special consideration for the independent clinical investigator
with the help of the hospitals of the Veterans Administration.
Congress has an opportunity to respond expeditiously to the needs
of patients and the nation to enact a new law that will establish a new
category of volunteers for clinical research, Doctornauts. As with
astronauts, soldiers, police, firemen and many others, it is time to
grant the right to physicians to take risks—even life-threatening
risks—to help rid us of unnecessary suffering and premature death.
If physicians are willing to become Doctornauts, what, then, could
be the objections? The Doctornaut Act would be an effective response to a
national health issue that demands immediate attention. The “reduction
of morbidity and cure approach” offers many more benefits to the
American people from increased quality of life to access to more
effective treatment than the predominately traditional national “care”
In addition, it is an effective way to reduce health care costs, both in the short and long terms.
THE PATH TO CLINICAL MEDICAL DISCOVERY AND OUR NATIONAL COMPLACENCY ABOUT IT
Both as a nation and as patients (we are all patients!), it is
critical to understand how medical discoveries are made. Potential
medical breakthroughs become real ones only when they are proven to work
in Phase II studies, the early phase of clinical research. Penicillin,
dialysis, and coronary artery bypass were only shown to have real
medical value after they were tested in people. And that’s where the
major problem is—clinical research. Costs and risks to conduct Phase II
clinical research have placed most of our creative clinical
investigators on the sidelines. Drs. Joel Goldstein and Michael Brown,
Nobel Prize winners in medicine, share the same concern, warning that
patient-oriented research is in serious jeopardy. To add to this
national medical tragedy, there is an ongoing alarming decline of U.S.
Generally speaking, creative minds in all walks of life function
either poorly or not at all in environments that are barrier-laden. When
DaVinci or Einstein had an inspirational moment, the former would walk
to his easel and begin to paint, while the latter would go to his
blackboard and write equations, with only space as a barrier to each for
fulfilling his moment of creativity. Not so in clinical research: The
barriers to the clinical researcher are costly, risky and
time-consuming, all powerful factors that profoundly discourage clinical
research in general. For example, the average cost to obtain one
successful FDA approval of a pharmaceutical exceeds $800,000,000 which
in itself reflects the enormous barriers to clinical research.
Much of our promising medical technology is now waiting in
laboratory lines in a preclinical bottleneck waiting to be evaluated by
clinical testing. And there are no signs that things will improve.
We as a nation are highly suspicious and fearful of clinical
research. The latter is often referred to as human experimentation,
which bespeaks of this suspicion and fear. This fear is utterly out of
touch with the results of empirical research, which have demonstrated
that it is much safer to be a research subject than it is to be a
patient. Yet, astronauts who volunteer to risk and sometimes lose their
lives for space exploration are admired. There was no national outcry
against space exploration following the deaths of seven astronauts. They
were revered. A recent USA Today CNN/Gallup Poll reports that three out
of four Americans find fatal astronaut missions acceptable.
Witness, however, the national media coverage and uproar as a
result of a single death of a volunteer at the University of
Pennsylvania undergoing clinical testing of a promising type of gene
therapy. Gene studies were suddenly halted and the physicians conducting
the study were criticized nationally. Though certain mistakes were
made, the public and political response was highly irrational and
ultimately damaging to patients. New rules were then initiated which
increased the already formidable costs and risks to clinical research.
After the University of Pennsylvania came Johns Hopkins. Because of a
single death, the FDA halted all clinical studies at this great
institution. The result? More costs and risks. This trend continues
unabated, with profound negative consequences on medical discovery.
Contrast this to the fact that there are many thousands of deaths
in the United States due to marketed drugs, a fact which escape national
Over and above our national fear and suspicion of clinical
research, we have an inexplicable national complacency about medical
discovery itself. For example, it is almost the rule to read or hear
such messages in the media as, “An effective new gene therapy has been
found but it will take years of testing,” or “New drugs fight aging in
animals but such developments for humans are years away,” or “New drugs
for Parkinson’s disease are expected to take years of testing,” or “A
new protein to treat skin cancer has been tested successfully and may be
ready for humans someday.”
Why does it take so long for promising scientific research to be
translated into effective medical treatments? Moreover, why doesn’t
anyone in the media ask the question, “Why?”
Congress, in a similar vein and puzzling as it may seem, has never
held a thorough Hearing on the “Why?” issue. Yes, the Congress
occasionally senses that something is wrong, as exemplified by the
Orphan Drug Act and Food and Drug Administration Modernization Act,
FDAMA. Such Acts, however, do not get to the heart of the problem.
If the Congress were to have a Hearing and ask patients what they
really want from our health system, they would say something similar to
the following, “If I have my disease or disability, get rid of it. If I
don’t have it, I don’t want to get it.” They want what we call the
prevention and treatment of disease. Patients’ first priority is not
free hospitalization or reimbursement for their drugs—they prefer to be
Paradoxically, the Congress and other partners in the health
community have primarily concentrated on the issue of care instead of
THE DOCTORNAUT ACT
The Act will primarily address three categories:
1. The rationale and ground rules under which a physician-patient, a
Doctornaut, can volunteer to be a physician-research subject.
2. Clinical investigator immunity from suit.
3. Special consideration for the individual clinical investigator and a unique role for Veterans Administration’s hospitals.
DOCTORNAUT RATIONALE AND GUIDELINES FOR VOLUNTEERING
Rationale: Without the certainty of mathematics and Divine
Revelation, moral/ethical issues are oftentimes controversial,
particularly when dealing with volunteering for clinical research. The
general good not uncommonly appears to conflict with the particular or
individual good. In addition, the issue of free will combined with the
Golden Rule underscores the debate of what is considered moral/ethical
behavior in a particular situation.
Cutting through all these ambiguities and turning to history and
tradition, which often times mirror what works in human behavior,
physicians have frequently experimented on themselves or volunteered for
clinical studies in order to advance medical discovery and, in so
doing, benefited patients.
The list of high-risk self-experimentation by physicians to advance
medical discovery is a long one. Forssmann threaded—in what many of his
colleagues thought would be a fatal event—a rubber tube in his vein and
guided it to his heart; Stapp tested the limits of human tolerance to
the forces of crushing collision; Garrè, by making a skin incision,
infected himself with potentially fatal staphylococci cultured from
osteomyelitic bones and almost died, and, more recently, Marshall
swallowed the H. pylori bacterium to determine its role in the
pathogenesis of gastritis and gastrointestinal ulcers.
Indeed, before receiving his medical degree, a young Peruvian
medical student named Daniel Carriòn injected himself with an aspirate
from a Verruga peruana lesion, in order to prove that this lesion was
the precursor to Oroya fever. He proved the point, but died in the
The vast majority of such acts, be they self-experimentation or
otherwise, however, are hidden, escaping the pages of medical history.
For the record, it is interesting to note that some physicians who
support the Doctornaut concept (Appendix) have either experimented on
themselves or volunteered in clinical studies.
In conclusion, the general good has overwhelmingly benefited from
the particular acts of Doctornauts who have taken risks. Some have
legitimately questioned whether modern-day U.S. physicians are willing
to take the high risks to which earlier generations of doctors exposed
themselves. Despite the overwhelming indications that physicians are
willing to take risks in clinical research, be they small or great, to
advance medical discovery and help patients, this is a question that
should be addressed.
About twenty years ago, the Foundation for Innovation in Medicine
(FIM), which has a particular interest in stimulating clinical research
on natural substances, conducted a random mail survey of physicians to
determine if they were willing to assume the role of a Doctornaut in
clinical studies involving natural substances. A total of 3,100
inquiries were mailed to a cross-section of 2,100 male M.D.’s, 500
female M.D.’s and 500 Doctors of Osteopathy. Age was also considered.
Half of the male physicians surveyed were 50 years old or younger.
A total of 10.3%, a good response with this type of survey, of the
physicians responded. A simple “yes” or “no” was required for the
“Would you as a physician-patient want the privilege to volunteer
for clinical research of natural substances under the supervision of a
physician-clinical researcher without FDA, institutional or other
Yes % No %
Male M.D.’s Total 52.5 47.5
Male M.D.’s Under Age 50 56.6 44.4
Male M.D.’s Over Age 50 50.0 50.0
Male D.O.’s 60.0 40.0
Female M.D.’s 56.3 43.7
The results of the survey clearly support that some American
physicians are willing to be Doctornauts. Some will argue that modern
U.S. medicine has changed radically since then and currently physicians
may not be as willing to take risks. Let’s assume that this is correct,
and that only twenty-five percent—about one-half the number two decades
ago—would volunteer to be Doctornauts.
Today, there are approximately 700,000 physicians in the U.S., which would make available 175,000 Doctornauts!
At a later date, consideration could be made for international
Doctornauts to participate in clinical studies conducted in the United
Ground Rules for Volunteering: The Doctornaut system must be under
the control of physicians or medical peers only. Also, in order to avoid
creating another complicated structure to monitor the clinical trials, a
subgroup of existing IRBs should be formed, M.D.-IRBs, composed of
physicians only with separate ground rules.
The ground rules are as follows:
1. An IND is optional. FDA should, however, be informed of the existence and progress of the studies.
2. Protocol rejection: The protocol can be rejected on the basis of risk and/or design.
Regarding risk, there generally should be a proportionality between
risk and benefit. For example, a Doctornaut with a fatal disease can
take a greater risk, including a life-threatening one, than a healthy
physician. On the other hand, the principle of proportionality is not
strictly binding. For example, many healthy volunteers are organ donors,
placing themselves at risk if the remaining organ, such as a kidney,
Also, the Doctornaut must have the “capacity” to make a decision to
volunteer in the study which judgement is made by the physician
clinical researcher. Capacity includes the following: The Doctornaut can
make a reasoned judgement, can communicate, and understands the
risk-benefit of participating in the study, and what would happened if
he or she did not participate.
Regarding the clinical design, the standard of acceptance should be
that it can be “reasonably assumed” that the results of the Phase II
study are due to the substance(s), device(s) or process(es) evaluated.
This means that the clinical study designs can be much more flexible
than those set by the federal government.
For example, current FDA Guidelines require a separate evaluation
of each component of a combination therapy. This requirement may be
scientifically sound, but it is medically unnecessary, and often
prohibitively expensive. Individual components of a combination need not
be individually evaluated against the combination itself. The
evaluation of the risk-benefit of the total combination is sufficient.
It is believed that no “magic bullet” for the treatment of many
cancers is on the near horizon. In the meantime, for example, a
combination pharmaceutical approach, along with other possible
therapeutic modalities such as gene therapy, would be more productive.
Another example supporting the “total combination approach” would
be to evaluate a nutraceutical (primarily dietary supplements with
medical-health benefits) that contains twenty ingredients of vitamins,
minerals and other dietary substances in a clinical study to determine
if it could significantly improve the immune system. If all of the
possible combinations of this nutraceutical were to be evaluated in
1,000 volunteers at a cost of $10,000 per volunteer, then the total cost
would be $24,300,000,000,000,000,000,000,000, exceeding the total
annual gross product of all the nations of the Earth for all time!
We seemed to have forgotten that a meal, the greatest combination
in the world, no matter where its origins, keeps us alive fueling our
body’s defenses that fight off disease. Do we need to study each
individual component of all the diets in the world to prove this point?
Also, the pervasive current government-academic cultural emphasis
on the need for double- blind studies should not play a dominant role in
the MD-IRB decision making process. Historical controls are often
sufficient. For example, a double blind study in not necessary to
demonstrate that insulin can reverse diabetic coma, penicillin cure
bacterial lobar pneumonia and that a stem cell implant creates new
arterial pathways around a blocked coronary artery.
3. For the aforementioned reasons, guidelines should be carefully applied on a case by case basis.
4. The studies can be terminated by the M.D.-IRB, the FDA or the
sponsoring party for reasons of negligence, willful misconduct,
significant failure to adhere to the protocol, and the occurrence of
unexpected, unacceptable adverse effects during the study.
CLINICAL INVESTIGATOR IMMUNITY TO SUIT
Generally speaking, to date, suing by patient volunteers has not
been commonplace. One reason is that in the informed consent document
that is signed, there is an explanation to the volunteer about the
degree of coverage for research-induced injury. Regarding
pharmaceuticals, for example, the coverage generally relates to any drug
adverse effects whether the sponsor is a pharmaceutical company or an
individual clinical investigator.
Another reason is that the clinical research process takes place in
patients, and it can be difficult to distinguish what is the cause of
the adverse effect, if something goes wrong.
Recent happenings, however, indicate that suing in clinical
research may become commonplace, as it is in other sectors of American
society. For this reason, it is imperative that the clinical
investigator—as with astronauts, military personnel and others—be immune
to suit from any quarter, except for intentional wrongdoing or gross
SPECIAL CONSIDERATION FOR THE INDIVIDUAL CLINICAL
INVESTIGATOR AND A UNIQUE ROLE FOR THE
VETERANS ADMINISTRATION HOSPITALS
There is a large, untapped reservoir of potential individual
clinical investigators with medical-breakthrough ideas that cannot see
the light of day because of cost-risk factors. Large companies,
foundations and government bodies generally provide sufficient coverage
not only to cover the costs of the clinical study but also insurance in
case something goes awry. Individual researchers can be covered by their
medical institutions, but high-risk clinical research is discouraged
because of its accompanying risk.
The Veterans Administration hospital system is a unique institution
which can not only help veterans, but also broaden its role in the
health system by partnering with individual clinical researchers, both
within and outside of the Veteran Administration System, whose
innovative medical ideas not only permit them to conduct clinical
research at their facilities, but also to cover all costs.
These hospitals are experienced in matters of clinical research and
have, in large part, fixed operating costs or overhead, an important
factor in substantially reducing the additional costs of clinical
research. FIM believes that this is an extremely cost-effective way for
the government to help achieve the goals of The Doctornaut Act.
TIMELY APPLICATION OF INNOVATIVE MEDICAL THERAPIES TO CLINICAL PRACTICE
New innovative medical discoveries will follow two paths, each of
which will expedite the addition of successful therapies to the
The paths are: a) One that does not require government approval, and b) one that does require government approval.
The paths not requiring government approval, for example, will
involve off-label use of FDA-approved drugs or medical devices and also
new surgical techniques that standard IRBs would not approve for
non-Doctornaut volunteers because of unacceptable risk.
Innovative therapies requiring government approval, such as
pharmaceuticals, can be placed in a “fast track” approval system such as
stipulated under FDAMA.
REDUCTION OF HEALTH CARE COSTS
Many argue, with good reason, that although medical discovery often
times improves the quality of life of patients, it significantly
increases health care costs. The latter is particularly alarming when
the demand for health care in the United States is increasing rapidly.
As examples, the use of dialysis devices in the treatment of End Stage
Renal Disease, and high technology pharmaceuticals are frequently cited.
Dialysis treatment costs the federal government billions of dollars,
and the annual cost of some high technology products, particularly for
rare diseases, is in the tens of thousands of dollars.
We have entered the era of “creeping technology,” high-cost
products with either promising or proven medical benefits that can often
cost billions of dollars for each individual medical condition. For
example, a new surgical technique for the treatment of emphysema can
cost Medicare up to 15 billion dollars annually. Also, with the aging
population, congestive heart failure is becoming commonplace. When the
heart, despite the use of pharmaceuticals, begins to fail, a new medical
device, L.V.A.D., or Left Ventricular Assist Device, can help the heart
to pump more efficiently. It is estimated that this device could cost
Medicare up to 12 billion dollars annually, and many more such “creeping
technology” therapies are clearly on the near horizon.
Many of the new therapies are directed toward chronic diseases.
Currently it is estimated that the annual cost for chronic diseases is
$750 billion. With “creeping technology,” plus the aging baby boomer
population that will soon become Medicare recipients, coupled with
medical prescription reimbursement and increasing patient demand, health
care costs will climb to exceedingly high levels.
Must we then assume that increased medical innovation will inexorably lead to astronomically increased costs? Of course not!
There are two ways to reduce such costs. The first is to reduce
demand, which would be difficult to implement, because it would deprive
Americans of the availability of effective medical technology. The
second, more practical and desirable way is to discover relatively
inexpensive therapies that can prevent, treat or cure disease, including
For example, a significant percentage of patients who are on
dialysis have heart attacks and strokes and are also diabetic. A well
thought-out pharmaceutical-nutraceutical combination that could even
partially prevent the blockage of large and small arteries would
significantly reduce the need for dialysis as well as the number of
heart attacks and strokes.
It is important to note that one principal reason behind high
health care costs is the high cost of conducting clinical research,
particularly when combined with the high costs of government approval
such as an NDA. High costs to arrive at the marketplace frequently leads
to high-cost products.
Doctornauts will lead to the discovery of both high and low cost
therapies the latter of which can be used to reduce or at least spread
out overall health care costs.
WHY DOCTORNAUTS ONLY?
As a group, physicians best understand the nature of disease and
the therapies used in the medical armamentarium. They undergo, more or
less, the same medical training and share a consistent fundamental
knowledge base. In addition, physicians have a historical, moral
obligation to help their patients and, by participating in Phase II
clinical trials, which will inevitably benefit their patients, they
fulfill part of that obligation. Once a promising new therapy is
discovered in such trials, however, non-physician volunteers can then
have the opportunity to participate in subsequent clinical studies.
CAN DOCTORNAUTS HELP CHILDREN?
A mother of a child stricken by a severe disability commented that
Doctornauts are adults and questioned whether they can help children.
The answer is, “Yes”! For example, positive studies on ways to
transplant central nervous system tissue in adults can rapidly be
applied to children with cerebral palsy or other central nervous system
diseases. Also, one major factor in delivering effective gene therapy is
the difficulty in finding carriers to deliver genetic material into
cells. Once they are tested and discovered in Doctornaut clinical
studies, the gene carriers can then be used for treatment in children.
Most important pharmaceuticals such as steroids and antibiotics were first tested in adults before being given to children.
As with adults, Doctornauts will both accelerate the discovery and delivery of new medical therapies to children.
MONITORING THE DOCTORNAUT ACT
The federal government, in order to assess the medical-health
impact of The Doctornaut Act, should fund an independent,
non-governmental organization to identify Doctornaut clinical studies
and periodically report its findings.
It is anticipated that other organizations will also monitor some
aspect of Doctornaut clinical studies including the establishment of
lists of physicians who are willing to become Doctornauts for specific
types of clinical studies.
We have a unique, highly promising and doable opportunity to
rapidly advance medical discovery within a relatively short period of
time. There are, unfortunately, strong forces impeding the fruition of
this opportunity—and they are growing.
For example, most of the worldwide profits made by U.S. and
international pharmaceutical companies with a United States presence are
earned in the United States market which, in turn, support most of the
costly research and development on drugs.
Many countries, including Europe, have price controls where
profitability is minimum. The result? The citizens of these countries
benefit from the profits made in the United States for drugs paid for by
Americans: And there are no signs that this international policy will
But it appears that profitability in the United States may now be
in jeopardy, as exemplified by Medicare prescription drug reimbursement
and importation of drugs from foreign countries, which, if they occur,
could lead to significant drops in profits critical to fuel all medical
research, from the laboratory to clinical studies. The highly promising
high-risk biotechnology industry would be particularly threatened.
This possibility augurs poorly for the patient.
In conclusion, FIM holds the firm conviction that the benefits of the Doctornaut
Act will be:
• Rapid acceleration of medical discovery followed by:
• Reduced morbidity (increased quality of life)
• Reduced rate of mortality from disease
• Timely application of innovative medical therapies to clinical practice
• Reduced health care costs
Stephen L. DeFelice, M.D.
The Foundation for Innovation in Medicine
Physicians In Support of Doctornauts
FIM, The Foundation for Innovation in Medicine, contacted physician
leaders to discuss their thoughts on the importance of Doctornauts. All
of the following agreed:
David P. Carbone, M.D., Ph.D.
Professor of Medicine and Cancer Biology
Vanderbilt School of Medicine
Director of Experimental Therapeutics Program
Vanderbilt-Ingram Cancer Center
Joseph R. DiPalma, M.D.
Dean Emeritus and Professor of Pharmacology
MCP/Hahnemann University School of Medicine
Elaine B. Feldman, M.D.
Professor Emeritus of Medicine, Physiology and Endocrinology
Medical School of Georgia
Former Director of The Georgia Institute of Human Nutrition
Louis Lasagna, M.D.*
Dean, Sackler School of Graduate Biomedical Sciences
Founder and Chairman
The Center for the Study of Drug Development
Robert J. Levine, M.D.
Co-chair of the Executive Committee
Yale University Interdisciplinary Project in Bioethics
Director, Law, Policy and Ethics Core
Center for Interdisclipinary Research on AIDS
Professor of Medicine and Lecturer in Pharmacology
D. Ted Lewers, M.D.
Chairman of the Board
Medical Mutual Liability Insurance Society of Maryland
Past Chair, Board of Trustees, American Medical Association
Donald B. Louria, M.D.
Professor and Chairman Emeritus
Department of Preventive Medicine
New Jersey Medical School
Chairman, The Advisory Board to the New Jersey Health, Wellness Promotion Act
Thomas F. O’Donnell, Jr., M.D.
President and CEO
New England Medical Center
Past President, The Society for Vascular Surgery
Michael J. Painter, M.D.
Professor of Neurology and Pediatrics
University of Pittsburgh School of Medicine
Past President, Child Neurology Society
Edmund D. Pellegrino, M.D.
Professor Emeritus of Medicine and Medical Ethics
Founder, Center for Clinical Bioethics
Richard S. Rivlin, M.D.
Senior Vice President, Medical Affairs
Naylor Dana Chair, Nutrition
Institute for Cancer Prevention
Past President, the American Society of Clinical Nutrition
George E. Schreiner, M.D.
Distinguished Professor of Medicine
Georgetown University School of Medicine
Shepherded the Medicare Amendment for Renal Dialysis Reimbursement
William E. Thornton, M.D.
Professor of Cardiology
University of Texas Medical Branch
Former NASA Physician-Astronaut Involved in Clinical Experimentation in Space
Raymond L. Woosley, M.D., Ph.D.
Vice President for Health Sciences
Arizona Health Sciences
Former Candidate for FDA Commissioner
*Dr. Lasagna, one of America’s greatest patient advocates, has recently passed on.