THURSDAY, December 8, 2005
Session 1: Bioethics and American Children
Norman Fost, M.D., M.P.H., Professor of Pediatrics and Director
of the Program in Medical Ethics, University of Wisconsin Hospital
In today's discussion, we enter a new field of inquiry for
the Council — the ethical and bioethical issues involved in
the care of infants, children, and young adults. They are, as has
so often been said, the future of our nation and the world. They
will confront the problems and the promise of biotechnology and
We can scarcely imagine today what the nature of those problems
and challenges and potentialities will be. Certainly, they will
involve such things as the uses of psychotrophic agents in children,
questions of human experimentation and clinical trials, issues of
consent, assent, and surrogate decisionmaking, as well as end-of-life
decisions, attitudes and values toward the disabled and the handicapped,
the questions of priority and the allocation of resources on the
basis of age and development, decisions of the utmost significance
during intrauterine and neonatal life, epidemic obesity, universal
vaccination, genetic testing. The list goes far beyond anything
we can fully envision at this time.
We have invited speakers whose expertise includes many of the
areas that I've mentioned, and many others they will bring to
our attention. We have asked them to be free to look at the issues
as individuals, from their own point of view, from their expertise,
and from their experience.
The second item, which we will discuss tomorrow, is a staff paper
on the subject of human dignity, a concept which is much used and
much abused in the bioethics literature today. It has been used
repeatedly by the Council itself, and we thought it wise perhaps
to reflect on some of the dimensions of this concept.
Before we begin, let me ask if you will express your appreciation
to Dr. Kass. He happily will be with us as a member of the Council,
and we will have continued access to his wisdom and his intelligence.
Leon, thank you very, very much.
Our first speaker this morning will be Dr. Norman Fost, who hardly
needs any introduction in the field of pediatric ethics. You have
a summarization of his career and curriculum vitae, and I will ask
him to address us — Norm, there you are. And I have asked
him very clearly to tell us what he thinks over the many, many years
of his cogitations on this subject we should be addressing and the
nation should be addressing.
DR.FOST: Thanks very much, Ed, for inviting me to this
Dr. Pellegrino's invitation reminded me of the famous exam
question, "Discuss the universe and give two examples."
I've decided instead to give three examples to be unduly ambitious,
and to talk about the three issues of the many that we could talk
about that seem to me most pressing.
Through the lens of someone who has been engaged in these issues
for 40 years — not as long as Dr. Pellegrino but I'm gaining
on you — as a pediatrician, as an investigator, as a human
subject, as Chair of an IRB for 28 years, Chair of the Hospital
Ethics Committee, someone who has written and taught about these
issues and had the opportunity to be involved in federal policy.
And let me say that I think ethics does matter, that Councils
and Commissions like this have had a — that there's been
a dramatic change. I'm going to try to make the point that
there have been dramatic changes in the well being of children as
a result of committees and councils and commissions like this, and
I think there is the opportunity to have more influence.
I'm going to talk about three issues, two of which will seemingly
overlap with other speakers, namely end-of-life decisions, research
issues, and issues about genetic screening. But I've talked
with all of them, and I think there will be minimal overlap and
what they say will sort of pick up where I leave off.
But before I do that, I want to just say a few words about two
overriding issues that seem to me to dwarf traditional bioethics
concerns with regard to children.
First is the continuing problem of 40-plus million Americans without
insurance, a third of them children, somewhere between eight and
ten million children without third party coverage, not eligible
for Medicaid, who die at a higher rate than others, who suffer,
who come into life with burdens, who leave the starting gate with
a 200-pound gorilla on their back, whether due to prematurity or
single mothers on drugs or lack of access to care.
And this is always mentioned as the most important problem involving
children and Americans, but not enough is done about it. It's
a big issue, and I'm not going to say any more about it, because
it is so complicated. But I hope the Council will take some cognizance
of it in a larger context.
Second, I want to just say a few words about child abuse, which
is a special interest of mine. I've been director of a child
protection team for 35 years, and, here again, it's a problem
that affects millions of children.
I mean, there are over a million reports of child abuse in the
United States, and we know that that is the tip of the iceberg.
Underreporting is somewhere in the five to ten to one range. That
is only five to — only 10 to 20 percent of cases get reported.
And in the area of physical abuse alone, we have known for 30
years how to prevent this. Dr. Henry Kempe did a landmark study
35 years ago, showed that we can predict 80 percent of cases of
physical child abuse in the delivery room. We know before they
go home who these children are.
And in a randomized, controlled study, showed that the simple
intervention of lay home visitors, lay people asked to spend an
average of five hours a week with these usually single mothers,
had dramatic effects in reducing the incidence of physical abuse,
permanent disability, and, in some cases, even death.
We are the only country in the world that I know of, certainly
one of the few, that does not have routine home visitors. It's
considered bizarre in other countries that we allow these children
to be born and go home, and nobody checks in, nobody offers to help.
It is extraordinary.
But Dr. Kempe's studies have been repeated many times, the
effectiveness of home visitors, professional or lay visitors, has
been shown in — when done correctly, to have very dramatic
effects. In fact, I would say the most important professional thing
I've ever done is to have the chance to be involved in starting
a nonprofit agency in Madison whose sole purpose is to recruit lay
volunteers for home visitors. It has been copied elsewhere around
the country, and it has been immensely gratifying.
So, again, it's a huge issue. It affects, logarithmically,
more children than the issues that we're going to be talking
about. And I hope the Council at some point can take that up.
I'd be happy to talk more about it when the occasion arises.
But I will concentrate on the three traditional issues. From
a historical perspective, what I'd like to do is say something
about how it was when I started out in this field, how it is now,
and, in my view, how it should be, because I think things were terrible.
I think there have been dramatic changes. But I think there is
still work to be done, and I'll suggest what that might be.
So first some comments about withholding and withdrawing life-sustaining
treatment, end-of-life decisions in children who are ill and handicapped
and in need of medical care. My entre into bioethics was facilitated
by a single patient, a child who has come to be known as the Hopkins
Mongol case, in the language of the day, a newborn with Down's
Syndrome and duodenal atresia, intestinal obstruction of a kind
that's very easily fixed in a simple operation that has virtually
100 percent success rate.
This was in 1971 when I was in my second year as Chief Resident
at Hopkins. The parents typically did not want surgery done. It
was withheld, an NPO signed, "Nothing by Mouth" was put
on the child's crib, and he was put into a room by himself and
allowed to die of dehydration over a 15-day period.
With the help of the Kennedy Foundation, we made a movie of that
case. That has been seen by more than a million people, and became
the centerpiece of a symposium at the Kennedy Center in Washington.
That was one of the first cases to bring bioethics into the public
sphere. It was one of the first widely-publicized cases that attracted,
appropriately, public scrutiny and discussion.
I went to the record room at Hopkins at the time and found six
other cases of the identical situation — that is, children
with Down's Syndrome and duodenal atresia who had been allowed
to die. In fact, a case just about three years before that had
been taken to court by Dr. Robert E. Cooke, the Chair of Pediatrics,
to try to get court intervention, without success. The court said
that the parents had the right to make that decision.
Shortly after that case and the publicity surrounding it, a national
survey was done of pediatricians and pediatric surgeons, and 70
percent of them agreed with what was done in that case. That is,
70 percent of American pediatricians polled said that, if confronted
with the same case, they would defer to the parents' wishes.
They would not go to court. They would not seek to override the
A similar survey was done in Massachusetts and produced the identical
result; 70 percent of pediatricians in Massachusetts said they thought
it was — what the parents had asked for was okay, and they
would go along with it.
An article was published in The New England Journal by
one of my teachers at Yale, Dr. Raymond Duff, reporting that one
in five deaths at the Yale New Haven nursery over a two-year period
was due to withholding of standard medical treatment. This included
children with Down's Syndrome and other relatively modest handicaps
or disabilities, and children with profound disabilities who had
little prospects for a long or meaningful life.
And in the last paragraph of that article Dr. Duff said, "If
what we did is illegal, then that shows that the law needs to be
The world 30 years later is dramatically different, I think in
part as a result of the work of a prior Presidential Commission
on Bioethics, work by the American Academy of Pediatrics, scholars
in the field, and so on. There has been a complete reversal of
The turning point came in the mid-1980s when President Reagan
implemented so-called Baby Doe regulations that prohibited discrimination
on the basis of handicap. And to the best of my knowledge, since
1985, there has not been a single case of a child who has —
with Down's Syndrome or spina bifida, the other common malformation,
who has died due to withholding of standard care simply on the basis
of having Down's Syndrome or spina bifida.
Needless to say, such children sometimes die, and sometimes due
to withholding of treatment, but because of some profound, often
untreatable, illness, not because they have Down's Syndrome.
So it has been a dramatic change. The status of children with
disabilities and the medical treatment that they receive is 180
degrees different from what it was 30 years ago, and even 20 years
ago. But this long history of undertreatment, of inappropriate,
and I think what is now widely agreed to be inappropriate withholding
of treatment in such children, has been replaced by what many of
us consider overtreatment.
That is, there was a rebound effect from the Baby Doe regulations.
Dr. Kopelman, one of the later speakers, has written eloquently
about this, and what we have now is a country in which children
receive treatment regardless of whether it serves any interest of
theirs, children who have little or no prospects for a meaningful
or long life, but suffer in intensive care units or in intensive
care units in their homes, in part as a result of fear of legal
repercussions, in part due to what I consider misinterpretation
of the famous or infamous Baby Doe regulations.
Dr. Nelson I know will be saying a lot more about this subject,
but I think work is needed, and I hope the Council will contribute
to this discussion about finding a middle ground. One of the causes
of the transformation in this area is not just the Baby Doe regulations,
but the growth of hospital ethics committees, infant care review
committees as they were called at the time, which introduced for
the first time almost obligatory multidisciplinary/multidimensional
discussions in end-of-life decisions, not just for children but
for all hospital patients.
As you know, these committees are now required by the Joint Commission
on Accreditation of Hospitals, and it's my view there has been
a dramatic change in just the process by which these end-of-life
decisions are made. So the debate is over a much narrower ground
of cases, a much narrower band of cases.
But in my view, there still is undue overtreatment, and it is
now very difficult in many parts of our country for a child to die
when there's widespread agreement that that would be in that
child's interest. Dr. Nelson will be saying much more about
Second, I'd like to say something about research, and start
again with personal experience. In the 1960s, a local reporter
at WABC in New York, WABC TV in New York named Gerald Rivers lurched
into national prominence with a sensational series of reports of
alleged abuses of profoundly retarded children at the Willowbrook
Hospital in Long Island, a state institution for the profoundly
Newly-admitted children to Willowbrook, he discovered, were deliberately
infected with the Hepatitis virus using fecal extracts from other
children in the institution. The reporter soon acquired a new name
to better fit his celebrity status. Gerald Rivers became Geraldo
Rivera, which is perhaps the most serious legacy of Willowbrook.
It's beside the point that Willowbrook, in my view, has been
badly and consistently misreported over the decades, and was framed
by Geraldo Rivera and everybody since as a non-therapeutic research
study to learn about the epidemiology of Hepatitis. It was, in
fact, intended as an early vaccine study. That is, Dr. Krugman
thought what he was doing was trying to prevent the infection of
Hepatitis in these children, where it was endemic.
Dr. Krugman was vilified for what were called coercive recruitment
techniques and disregard for minimum standards of informed consent.
My own view is that the consent procedures used by Dr. Krugman exceeded
any in any study I've known since, and this, mind you, was before
IRBs existed, before there was any — there were any requirements
for research involving human subjects.
One last point in the category of personal disclosures. My senior
thesis at Yale involved a study of chromosome breaks in children
who had had viral infections, and my source of my patient material
were the children at Willowbrook. That is, I found a nurse there
who would call me when there was a new outbreak of some viral disease
— measles, rubella, or hepatitis.
I would drive down with my little bag, go into the main ward there.
The nurse would find an appropriate patient, yank his or her arm
through the crib rails, I would draw blood and go back to New Haven
and do my studies. No committees, no parents, no consent, no review,
Well, I'm not here today to discuss the pros and cons of the
Willowbrook studies, Dr. Krugman's or mine. The point is that
it was typical of the day — that is, research involving children
and most everyone else — was a free for all with no regulations,
no oversight, no committees, no review, and usually no standards
for consent from everybody — from anybody.
This was standard practice. One of my heroes at Yale, Dr. Robert
Cooke, elucidated with his mentor, Daniel Darrow, most of what we
know about the role of potassium in the body, in part by taking
infants out of orphanages in New Haven, bringing them into a clinical
research unit where they were exposed to heat stress.
Meticulous balance studies were done. These studies resulted
in what ultimately became WHO electrolyte solution, and Dr. Cooke
conservatively estimates he saved several billion lives through
this research on a very small number of children, but is just astonished
at what he did, looks back with sort of horror that he was able
to do that, and has said many times, "If there was just some
committee that I had — just any group of people that I had
to check this out with, it never would have happened. I could have
done the studies on sick patients with very minimal risk."
When I was an intern at Hopkins, one of my fellow interns did
a landmark study on hernias in children by taking infants who had
been admitted to the hospital for an elective herniorrhaphy the
next day. He was interested in the question of whether children
with a hernia at one side were at risk for a hernia on the other.
So at midnight when he was done with his regular intern duties,
he would pick these children out of their cribs, take them down
to the emergency department, inject their abdomens with high opaque,
radio-opaque dye, jiggle them around, put on a lead apron on himself,
sweet talk an emergency room X-ray tech to take a flat plate of
the abdomen, including the gonads, and published a landmark study
showing a high incidence of inguinal hernia.
To this day, those parents have no idea who their — that
their children had their gonads irradiated with very primitive X-ray
And, finally, these examples are by the dozens. But just to make
the point, Dr. Gross at the Boston Children's Hospital, the
Chief of Cardiovascular Surgery there in the 1970s, was interested
in the role of the thymus — an immunology poorly understood
at the time — and took children who were brought to the OR
from elective surgery for congenital heart disease, took out the
thymus on some and not others, and did heterologous skin transplants
to see what the role of the thymus was in rejection.
The children would come back to the ward with these funny patches
on their arms. The nurses didn't know what it was, couldn't
answer the parents' questions. Again, no permission from anybody.
So we've come a long way.
Three weeks ago Dr. Nelson and I spent a day and a half discussing
whether an investigator at the University of Chicago could admit
children to a hospital overnight to give them a single injection
of a probably harmless drug, and collect a few blood samples. It
took one year for this investigator to get permission from his local
institution and still required the permission of the Secretary of
Health and Human Services. And Dr. Nelson and I were part of a
committee to advise the Secretary on that.
So the kinds of egregiously unethical research involving children
and everybody else that was done in the 1960s, like starving mongols
to death, is ancient history. But just as the undertreatment of
handicapped infants has been replaced by overtreatment, in my view
the underregulation of research has been replaced by overregulation
and disregulation, with severe sanctions against institutions for
failure to document compliance with rules, many of which have little
or no relationship to protection of human subjects.
Let me just give one example, again, of dozens in the discussion.
We could expand on this if you like.
As you all know, Duke University some years ago was shut down
by the Office of Human Research Protections for alleged violations
in protection of human subjects. There were 25 different categories
cited by the Office of Human Research Protection. I'm just
going to mention one.
One was the failure to document a quorum in the conduct of the
IRB's business. The Chair of the IRB, whom I know well, showed
that they had documented a quorum at the beginning of the meetings
and used Roberts Rules, which state that a quorum is presumed to
exist unless it's challenged by somebody during the meeting.
The leader of the site visit from OHRP said, "No, sir, the
common rule — the federal regulations require that all business
conducted at the meeting must be approved by a quorum of those of
the committee. You had over 100 action items at each committee,
and you did not document a quorum, we know that there's a lot
of Brownian motion at these committee meetings, people leave to
take pages, to go to the bathroom, and what not, and so we have
suspended all 2,200 protocols because you have no documentation
that a quorum existed.
As a result of that suspension, Duke, and now everybody else,
documents a quorum 100 times during a meeting. The minutes from
— I'm Chair of our IRB. The minutes of our meetings are
150 single-spaced pages for a three-hour meeting. This is —
the quorum rule, of course, is only one of the many things we have
One last anecdote about the quorum rule. OHRP went on to shut
down eight consecutive academic medical centers. The University
of Wisconsin was ninth on the list. We fully assumed that we would
be shut down because we were guilty of all the transgressions, virtually
all, that have happened at Duke.
And while we had taken steps to correct them to be in compliance,
we knew that if our records were examined that they could show that
our 2,000 active protocols also had been approved without documentation
of a quorum and many other things for each one.
Miraculously, we were not shut down. We received a commendation
letter. And as a result of this, when accreditation of IRBs started,
one of the new accreditation agencies asked us to become their beta
site, their testing site, since we were held in such high esteem
by the federal agency.
We were glad to do this. It gave us a chance at essentially a
free look at accreditation. It was literally cost-free. If we
flunked, it would be confidential. If we passed, we would be accredited,
so it seemed like a no-brainer.
The accreditation manual had 270 items. We had six months to
prepare. There was a four-day site visit. We flunked 70 percent
of those items, 70 percent of 270 items. This was an IRB that had
received a commendation from the federal authorities.
Let me just mention the quorum rule as an anecdote. We, of course,
as a result of the Duke experience had learned to document a quorum
100 times at each meeting for each action item. But the site visitor
said, "You haven't documented whether a non-scientific
member of the IRB was in the room. The federal rules require that
each IRB must have a non-scientific member, somebody whose major
expertise is in non-scientific areas."
Well, we had several such members, and so we were quite confident
that there always was at least one such person in the room. But
the site visitor said, "You haven't documented it. All
you've said is that you have a quorum, but how do we know if
the quorum included a non-scientific member?"
Well, virtually all our votes are unanimous or unanimous minus
one. We just keep talking until we reach consensus. And I asked,
"Has there ever in the history of the world been a study which
involved humans, a research study, in which the vote was tipped
due to the vote of the non-scientific member? Do you know of any
Well, of course, nobody knew of such an instance, but that was
beside the point. A rule is a rule. As a result of that requirement
by that agency, Hopkins, I was told, in one of their IRBs began
passing a clipboard around at their IRB meetings where all 24 members
signed the clipboard for each of 100 items at the committee —
2,400 signatures at one meeting — to make sure that the non-scientific
member and other IRB requirements were met.
This is silly, of course, but if it were just the only example
I wouldn't bother you with it. In my view, there are a dozen
similar kinds of rules, and the problem is that research which used
to be too easy is now too hard. That it is now very difficult
to get senior faculty to participate in IRBs.
We had a very valued member storm out of a meeting and quit permanently
because of discussions like this, saying, "This is not what
I'm here for. I'm happy to donate 150 hours a year of my
time at no reimbursement to protect human subjects, but not for
this kind of nonsense."
It is harder for investigators, of course. Clinical research
is dying in my view, as a field of inquiry. That is, there is a
dramatic decline in the number of young American physicians going
into this area. If you look at the lead author of New England
Journal articles over the last decade, less than 50 percent
are now American authors.
If you look at the number of M.D. principal investigators of NIH
funding, it is also in almost a straight line decline. Children
are the most serious victims of this decline in clinical research.
That is, children, unlike adults, are almost always treated with
what is euphemistically called innovative therapy.
My colleague Paul Lietman at Hopkins says innovative therapy means
if you don't want to learn any — if you promise not to
learn anything from what you're doing, you don't have to
go through an IRB. Eighty percent of all drugs prescribed for children
in America have never been approved or tested for safety or efficacy
And as we know, children are not little adults. History is littered
with examples of large numbers of children who have died or suffered
because of treatments that were quite okay in adults, but turned
out to be toxic and even lethal in children — chlorenphenicol,
sulfonamides, oxygen given indiscriminately to children for 80 years
before anyone asked the question whether there might be a dose-response
Even simple bicarbonate, used as a buffer for patients with acidosis,
shown by Dr. Michael Simmons to cause death and profound brain damage
in infants with hyaline membrane disease. And on and on.
That is, children need research more than adults. The problem
is not that there's too much research in children, or too much
unregulated research as went on 30 years ago, but not enough. The
problem is that children are now "experimented on" but
without — and no systematic way and without any collection
That said, the regulations for research need some refining. Sara
Goldkind will say more about this. I just want to mention briefly
a couple of elements of the regulations that seem to be problematic
and possibly an example of persistent underregulation. Dr. Goldkind
will say much more about this.
In my view, the justification for non-therapeutic research on
children has never been made. The brilliant work of the National
Commission for the Protection of Human Subjects never really, in
my view, made the case for allowing non-therapeutic intrusions into
children without children who were incapable of consent.
The argument that was made was a utilitarian one, namely that
it's good for children as a class. We wouldn't want to
live in a world in which no non-therapeutic research is done on
children. And it's good for them to have such studies done
One of the restrictions was it's okay as long as the research
is of minimal risk. Realize, of course, that there is no such exception
for adults. That is, the utilitarian argument is equally true.
We could — knowledge could advance much more efficiently with
adults, if it weren't for this pesky consent problem.
If we could do more non-therapeutic research on adults without
their consent, we could learn a lot more a lot faster, but this
is absolutely taboo and appropriately so, but yet the argument is
allowed for children.
The minimal risk rule, defined in the federal rules as the probability
and severity of harm, comparable to what would happen on a routine
visit to the doctor, is interpreted widely — in a widely and
wildly variable way. Published studies have shown that IRB chairs
and IRB members interpret this to include everything from venapunctures
to non-therapeutic bronchoscopies and small bowel biopsies.
Investigators make the claim either that this is minimal risk
in my hands or this is what happens on a routine visit to my office.
I do a bronchoscopy on everybody I see. And the variation in IRB
acceptance of this kind of argument is quite remarkable.
So more work is needed in this area. Dr. Goldkind may say some
more about it, but I think we do not have enough conceptual work
or even practical, pragmatic definitions of the minimal risk rule.
The other area that, in my view, needs much more work is the notion
of assent. Legally valid informed consent is not possible with
young children, and so this term "assent" was invented
as a surrogate — the requirement that children at least be
told in language that's meaningful to them what is proposed,
and essentially that they don't need to do it if they don't
I don't think this is taken seriously. I think if it were,
it's hard to imagine why any seven-year-old would let himself
or herself be stuck with a needle if it had no relationship to his
or her health care, and they didn't want to do it. Moreover,
there has never been an adequate discussion as to why the age of
seven is used as a boundary for this requirement.
Three-year-olds know perfectly well what a needle stick is all
about. They know perfectly well that they don't want it, for
the same reason that adults don't want it, because it's
annoying, because it hurts, and they would just rather not do it.
But there is no respect for three-year-olds or infants for such
Well, I don't want to have non-therapeutic research on children
stopped, but I think, as I say, conceptual work is needed, and this
Council is very qualified to either do that work or facilitate it.
Finally, let me just say a few words about genetic testing, which
Dr. Pellegrino referred to, which in my view is the most serious
of these three areas that is affecting the most number of children.
And, again, I'd like to start with a historical example and
a personal experience, and then express my concern about where we
are today, and then I'll close.
In 1960, PKU, Phenylketonuria, was known to be an admittedly rare,
but a well understood, cause of profound mental retardation. It
affects approximately 1 in 10,000 live-born children. The biochemistry
of it was fairly well understood. It's due to inability to
metabolize an amino acid named phenylalanine, which is ubiquitous
It was known that if you could diagnose this early enough and
get a child started on a restricted diet that you could ameliorate,
and in some cases prevent completely, the profound brain damage
that uniformly occurs.
But the test that was available at the time, a urine test, was
inconvenient to get. It was obtained at the first well baby visit
after the child had been on a normal diet for a while and the horse
was out of the barn. That is, by the time children were diagnosed,
brain damage had already occurred.
The diet was expensive and unpalatable. Parents had an awful
time getting children to cooperate with it.
There were three breakthroughs in 1960 — the discovery by
Dr. Robert Guthrie of a simple test, making it cheap and efficient
to diagnose this condition on all newborns on a single drop of blood;
second, the development by Mead Johnson of Lofenalac, a low phenylalanine
milk that was reasonably affordable and palatable; and, three, the
election of John Kennedy.
President Kennedy, because of his profound interest in mental
retardation, his family's interest, with Dr. Guthrie formed
a so-called PKU lobby and arranged for laws to be passed in all
states requiring PKU testing, realizing correctly that doctors in
offices would be unlikely to adopt a test for a disease that affected
1 in 10,000 children — something that a pediatrician might
never see in his or her entire career. So mandatory newborn screening
for PKU became the national policy.
In 1973, I was invited by Dr. Barton Childs to be part of a commission
— a committee at the Institute of Medicine to look into the
PKU story. This was my first exposure to national policy.
The problem was, it turned out, that the PKU test was the worst
test in the history of the world. It had a sensitivity and specificity
that have not been matched to the best of my knowledge. That is,
the test had a five percent true positive rate. It had a 95 percent
false positive rate. That is, a child with a positive test, confirmed
by a whole blood assay, had a 20 to 1 chance of being normal.
This was not appreciated for many years. So many normal children,
we now know, were started on a restricted diet, and it turned out
that a phenylalanine-restricted diet was as harmful, or more harmful,
as a diet with excess of phenylalanine. That is, this essential
amino acid, when withheld from normal children, resulted in brain
damage due to starving of brain cells, and every other cell in the
body, because phenylalanine is a part of so many proteins.
So many children — we don't know how many — were
made retarded by this program. Some were killed. In fact, kwashiorkor
developed in America in the PKU program in children who had profound
protein malnutrition because of the restricted diet.
Well, if that happened to a child with PKU, you might say nothing
ventured, nothing gained, these children had little to lose. They
were — had terrible prognoses anyway. But when this happened
in a normal child, it's obviously a major tragedy.
In 1965, the American Academy of Pediatrics sent a letter to the
Secretary of DHHS urging that the mandatory PKU screening programs
be stopped, because we didn't understand the significance of
the test, and we didn't know how to regulate the diet.
This letter was suppressed. People were called Luddites who were
against newborn screening. The PKU lobbying was very powerful,
and testing went on until 1971 when a political scientist named
Joseph Cooper uncovered this story through the Freedom of Information
Act and led to the appointment of the IOM Committee, whose report
was published in 1975 articulating principles for ethically responsible
newborn screening, particularly genetic screening or screening for
These guidelines published by the Institute of Medicine in 1975
have been essentially photocopied by a dozen committees, commissions,
councils, professional groups, lay groups. There is virtual unanimity
on the principles of responsible genetic screening, and newborn
screening in particular.
It is not a controversy, and it represents another marvelous example
of the good work of ethics, of thoughtful people and ethics, law,
public policy, patients, parents, and so on, agreeing on guidelines.
The only problem is that the guidelines are systematically ignored.
That is, newborn screening has expanded like topsy, with the same
mistakes that beleaguered the PKU program happening over and over
That is, numerous screening and treatment programs have been implemented
without testing, evaluation of the tests, without any systematic
study of the sensitivity, specificity, or predictive value of the
test, or of the interventions.
This happens in part because genetic testing and treatment falls
outside of the regulations of the FDA. That is, there is no toll
gate through which an investigator or an innovator has to go to
get these kinds of programs approved. He or she only needs to persuade
existing committees and state health departments to simply add another
test onto the drop of blood or the drops of blood that now exist
for virtually every newborn in America.
I won't take time to rehearse for you other examples of newborn
screening gone awry, and the large number of children in my view
who have been killed, normal children in some cases, by screening
and treatment programs that have never been adequately evaluated.
Not enough research.
The new technologies, such as tandem mass spectrometry, now make
it possible to test for hundreds of conditions on this single drop
of blood. And, indeed, a committee of the American College of Medical
Genetics has persuaded the Secretary's Advisory Committee on
Genetic Testing to recommend to the Secretary national implementation
of a uniform standard for testing of newborns using tandem mass
These recommendations include over 50 conditions, half of which
have no known association with human disease. That is, approximately
half of the tests on the committee's recommended list are abnormalities
that have been observed whose relationship to clinical manifestations
are unknown or uncertain, and the other half roughly involve serious
diseases but diseases for which the sensitivity and specificity
and predictive value of the test is unknown, and in which the interventions
have never been systematically tested.
It is telling, in my view, that the UK equivalent of the FDA has
recommended implementation of only one of these 50 conditions.
Even worse, multi-array DNA testing — that is, the ability
to test for a thousand genetic variations using recombinant DNA
techniques on a single drop of blood is also now upon us, and work
is proceeding rapidly to add multi-array genetic testing to the
newborn PKU spots.
So we now already have many states, including Wisconsin, that
does routine testing without consent, without prior research, for
dozens of conditions using tandem mass spectrometry. And I predict,
unless there is some dramatic change in the way we think about these
things, the way we do these things, that multi-array DNA testing
will occur within the next few years, as soon as the cost comes
down to make it efficient to do it.
This, to me, is a calamity involving every child in America, the
amount of mischief. The amount of harm, psychosocial harm that
will occur to families and children, not to mention medical harm,
is, in my view, going to be quite extensive.
And, worse, 20 years from now we won't know what harm has
been done, because in the absence of systematic studies we won't
know which children were helped and which were harmed, because we
won't know whether like — in the PKU program, we won't
know if a positive test meant that that was a child who was destined
to become brain damaged or dead, or whether it was a false positive
test that had poor predictive value.
In summary, where have we been? Where are we? Where are we going?
In the area of end-of-life decisions, there has been a transformation
from egregiously unethical undertreatment, withholding of simple
treatment from children with excellent prospects for long, happy
lives, to serious overtreatment.
A thoughtful kind of undertreatment has been replaced, in my view,
by a thoughtless kind of overtreatment. In the area of research,
egregiously unethical research with no oversight or regulation has
been replaced by too little research, with excessive and inappropriate
regulation. The result has been an expansion of innovative therapy,
a decline in physician investigators or funds to support them.
And, finally, in the area of genetic screening, the mistakes of
the PKU program has resulted in guidelines about which there is
little dispute, but practices which have not changed at all and
which arguably may become much worse due to the advent of multi-array
I could tell many other stories in all these categories, and there
are, of course, other issues that we haven't talked about.
But these seem to me worthy of Council's attention.
Thank you for the opportunity to present to you.
CHAIRMAN PELLEGRINO: Thank you very much, Dr. Fost. Dr.
Fost is going on 35 years in bioethics. Bioethics itself was invented
or baptized in 1972, according to some people. You were there at
the beginning. And thank you very, very much for your reflections
on pediatrics and pediatric ethics since then.
I'd like to open to Dr. Fost's paper. The members of the
Council put on their red light, indicating that they'd like
to talk, to save me from being a traffic cop.
DR. KASS: Thank you very much for a really lucid and illuminating
paper. I have I guess — and I appreciate and feel the force
of each of these issues. But if I could draw you out on sort of
the — some of the tacit ethical suggestions that you made
along the way.
First, the second point on the research issues, you begin by saying
the case for non-therapeutic research on children hasn't been
made. You concluded by saying you don't want this to be shut
down, and, therefore, I assume you think the case can be made.
And I wonder — on non-utilitarian grounds, I assume, and I
wonder if you would — if you have any sort of beginning inklings
as to how you could do this.
Our mutual friend Paul Ramsey thought it was impossible to make
such a case, and I'm not sure I agree with you that the case
hasn't been made. But if you think that there is a way to make
it, I would be interested to hear at least the outline.
And, second, I wonder whether, if we follow your suggestion that
there is egregious — well, you didn't say egregious overtreatment,
but that there has been an excess — that there has been a
turn in the direction of excess treatment, and I'm not exactly
sure how you describe what constitutes the excess.
At one point, you talked about cases in which it would be in the
child's interest to die, and I wonder whether we might, 10 or
15 years from now, if such a formulation were made the standard
of care, whether we would say the case — we regret the way
in which that case was made. In other words, could — are
you able to articulate, for yourself at least, and suggest how we
might articulate the principles for deciding that this kind of treatment
is really excessive.
The Council has dealt with this with respect to end-of-life issues
in the adults, and we've, in a way, affirmed the old standards
where the treatment is excessively burdensome or where it's
I take it you might think that there are some treatments that
would be efficacious and not excessively burdensome, but that might
still be excessive under the present circumstances. I don't
know that you mean that, but if you could say a little bit more
on the ethical side of what you think constitutes too much, or how
we would recognize it.
DR.FOST: Thanks, Leon. First, I'm glad you mentioned
Paul Ramsey, who was maybe more than anyone else responsible for
my entering this field as an undergraduate at Princeton. He was
immensely influential in getting me interested in these issues and
stimulating me to get involved, and an important mentor to me throughout
my life, a really wonderful man.
Paul Ramsey did not quite say "impossible." In his
famous debates with Richard McCormick, he said for a number of years
it was impossible to find an ethically coherent justification for
non-therapeutic research in children. But I'm sure, as you
remember, Leon, near the end he said that perhaps we had to sin
bravely, as he put it. That is, we have to do this kind of research.
We have to admit that it's wrong, but we have to do it.
What's the ethical rationale for that? In my view, a better
rationale than the simple utilitarian one would be what some have
called a constructive consent. That is, to have a reasonable inquiry
as to what a child in that circumstance would do if he or she understood
all the ramifications.
That is, if an infant had a moment of lucidity and could understand
everything that we understood, might he or she agree to allow a
mere venipuncture in the interest of the class to which he belongs,
the class of children or children with a certain disease, and so
That seems to me at least a more plausible basis. I think the
bottom line is that, like Paul Ramsey, I don't want to see this
— to see a shutdown, a prohibition of non-therapeutic research.
I think that the minimal risk rules and the assent rules are useful
boundaries, but need to be, in the case of minimal risk, better
I think that it has gotten way out of control. I was part of
the National Commission deliberations, not as a member but as a
consultant. And I know that what was meant by that rule was things
that happen on a visit to a general pediatrician for a routine well
baby visit, and that has gotten way out of hand. So I think that
needs to be reined in, and I think if it were reined in I'd
be comfortable with it.
And I just think assent needs to be taken more seriously. That
is, children really — somebody really needs to say to them,
and we need to have more monitoring of it, "Junior, let me
make something clear. We want to stick a needle and get some blood
from you. You don't have to do this if you don't want to."
Now, would everybody say no? I don't think so. As Will Gaylin
famously said, "A parent has a right to decide that their children
are not going to grow up to be a selfish little bastard."
So that is some parents, and many parents in fact, would persuade
their children that they want them to volunteer for this kind of
research, because they think it's good for them to be a volunteer,
and it's important to be altruistic.
That does happen, and I think it's appropriate. I don't
think it's abusive. Parents do more serious things in the name
of altruism for their children.
So it's a long-winded way of saying I think the common rule
is adequate to protect children in this regard, if minimal risk
were defined more rigorously and if assent were taken more seriously.
That would still leave the problem of infants and children who can't
speak at all a little bit out there, but I invite you and the others
on this Council to help us. I think with some more work we might
be able to come up with an appropriate rationale.
With regard to your second question, I think if — the problem
is that even for children who meet the parameters that you describe
for adults, that it's treatment that is excessively burdensome.
And without compensating benefit, it is difficult in many settings
to allow these children to die a dignified death.
The Schiavo case is an adult case, of course, but there are many
children similar to Terri Schiavo, or not quite in a persistent
vegetative state which is harder to diagnose in infants and children,
but with profound brain damage in which there is unanimity among
the medical people that this child has little prospect for any kind
of social interaction, any opportunity to experience any of the
pleasures of life, even those of a profoundly retarded person, and
in which hospital attorneys or neonatologists, fearing legal consequences,
or the kind of public maelstrom that surrounded the Schiavo case,
even the involvement of the United States Senate, are reluctant
to pull the plug or to stop treatment.
Skip Nelson will be saying much more about this later, and I don't
want to intrude on his territory and where he has a lot of clinical
experience. But I think it is very difficult to let children die
in many settings, because of a very defensive attitude by lawyers,
In fact, Loretta Kopelman is the co-author of a famous study in
The New England Journal with her husband Arthur, a neonatologist,
showing that after the Baby Doe regulations large numbers of neonatologists
in America stopped using their judgment about what was in the interests
of their patients — that is, shifted towards overtreatment,
which was defined as treating children who they thought had no interest
in being children, as a result of the Baby Doe regulations, as a
result of, in my view, a false fear that the Baby Doe regulations
prohibited them from acting in the interests of their children.
So, in summary, again, a complicated area and a long-winded answer,
but I think the sorts of guidelines that apply to adults are applicable
to children. We can't even — it has become increasingly
difficult to even apply those. I think the use of ethics committees
provides a really reasonable procedural safeguard against the kind
of egregiously unethical withholding and withdrawing that occurred
30 years ago.
I think it's hard to find cases of really what we would call
egregiously unethical withholding of treatment that has been processed
through an appropriately-constituted ethics committee. And, as
always, the courts are, and should be, available to people who disagree
with ethics committees or with decisions that are being made in
I hope that's responsive.
CHAIRMAN PELLEGRINO: Any other questions? Gil, and then
PROF. MEILAENDER: Yes. I want to raise several questions,
several areas. I'm going to pass on the end-of-life questions
for the moment, not because I think they're unimportant, but
just to try to get at a few deeper issues in some of the other areas.
First, with respect to the research, just as an aside, there is
probably no way we could settle this without getting out the text,
but I don't think Ramsey changed his mind, by the way. The
sin boldly stuff was, at least on my recollection, what Ramsey loved
to do. That is, play with possible justifications that one might
come up with for various things.
It was not, I think, his preferred proposal on the matter, but
we'd have to get the text to decide that one. But on the research,
I want to sort of get at a couple of what seem to me to be deeper
questions, and then also on the genetic testing.
The examples you gave were wonderful in a certain way of the requirements,
the hoops that you are forced to jump through, and which do seem
ridiculous to probably almost anybody sitting there listening.
Raise a deeper question, though, exactly what — what's
the larger concern here? The larger concern is, whom shall we trust?
And how do we structure circumstances in such a way that we think,
you know, trustworthy people are making decisions that are wise.
And we find ourselves in a circumstance, not just here but in
many areas of our life, where the only way we can seem to find to
do it is regulation/overregulation to the point of absurdity. But
one of the reasons we find ourselves in that circumstance has to
do precisely with the history you recounted, in a way.
So I'd be interested if you could say just a little more about
that kind of question about exactly how we satisfy ourselves if
there are people whom we can trust.
And then, one — second question on the research. This follows
up on Leon's question. On the one hand, you don't want
to shut down non-therapeutic research on children. On the other
hand, you don't think a moral justification for it, up until
now, that's satisfactory has been provided.
Would you be willing to say that to take up the question in a
non-frivolous manner would require leaving open the possibility,
as one possible answer, that it should stop? That is to say, your
answer to Leon was simply you're confident that an appropriate
rationale can be found if we work on it.
Well, and that may be true, but wouldn't — if we're
really serious and not frivolous in taking up the question, wouldn't
we, from the start, have to have at least as one possibility the
idea that maybe there wasn't any justification, and we just
couldn't say that?
And then, on the genetic testing, if I can just put one more question
before you, there must be deeper impulses or urges at work that
lead us to want to know, with respect to these newborns, not just
about PKU but about a couple hundred or a thousand possible abnormalities,
even if, as in many cases you've said, not for now at least
directly connected to any known disease.
What's going on there? In other words, is there some larger
cultural power or impulse at work about what we want from a baby
or our need to know something if we can know it. In other words,
are we going to solve these problems just by a recommendation, or
are there some sort of deeper issues at work that push us in certain
directions? And that may be very much harder to get a hand on than
just a regulation.
I'd be interested if you could say something about that.
DR.FOST: Thanks. Those are all great questions.
First, with regard to who to trust in the research area, it's
my view that IRBs have worked extraordinary well. That is, that's
— that's the main thing that has changed since the '60s
and the '70s, the common rule and local IRBs to implement them.
It's very difficult to find, in the last 20 years —
and there have been several studies to support this — an example
of the kind of egregiously unethical research that was common and
ubiquitous in the '70s. There is a lot of conceptual basis
for IRBs. I myself am very enamored of what's called ideal
observer theory. Put in simple language — many heads are
better than one.
And I think it's so hard to find an example of, really, the
kind of research that was common back then that whenever something
happens that's controversial it becomes a cause celebre. So
the death of Ellen Roche at Hopkins, the death of Jesse Gelsinger,
stay with us for years and years and years, probably decades, and
in my view don't even themselves constitute egregiously unethical
That is, I think it's very difficult. I think there is a
case to be made, both sides of those cases, and yet they've
acquired — they've become poster children as research
Research has not gone wild. The Roche case and the Gelsinger
case are remarkable, because they are exceptionally uncommon, and
even those cases I think have been — there has been demagoguery
about them, and they've been overstated. So in my view IRBs
have done an amazing job.
Have they produced a zero-risk situation, a situation in which
no human subject ever gets mistreated? No, of course not. Nothing
can. But it's remarkably rare for — to have unanticipated
complications of those sort, but, more importantly, to have research
that would attract widespread condemnation.
So can you trust them? No, I wouldn't trust investigators.
I think investigators, left to their own devices, will run through
the stop signs for all the reasons that they did 30 years ago, not
because they are evil or mercenary, but because they — their
eye is on curing cancer in our lifetime and advancing science and
So, no, you need committees. I don't think we need the kind
of micromanagement at the federal level. I think the local IRBs
have generally done a good job. I think the sanctions are way too
punitive, and for all the wrong reasons. So that's my response
With regard to non-therapeutic research, is it possible that a
serious inquiry will lead to the conclusion that there's no
justification for it? Sure. Where would that lead? Would that
lead to — lead me to recommend the policy of no more non-therapeutic
research involving children? No, I'd come out somewhere where
Paul Ramsey came at, and we — I'd be happy to dig out
the text with you.
By the way, it was sin bravely, I think, not sin boldly.
But, obviously, national policy should not hinge on what Paul
Ramsey thought. It should hinge on what a consensus of people in
America think today.
So it may be that there is no — it may be one of these unresolvable
issues in which there is no real compelling moral argument for it,
but there is not a horrendous argument against it either, if we
keep it — if we keep the noise level down and keep it to truly
And your last issue, what drives this mania for testing? I had
notes on that, and I'm glad you gave me a chance to expand.
I didn't want to use up too much of the air time.
There are several factors that are quite common in these —
in this repetition compulsion. One is that the PKU lobby has been
duplicated over and over again. That is, there almost always is
a very zealous lobby that forms, consisting of parents of children
who have died or have become profoundly — you know, if your
child died of a rare disease, you understandably don't want
to see this happen again to anybody ever.
And so — but you're not a scientist, and so you say
it's just unimaginable to me that every child in America isn't
tested for this very rare disorder, and so that something can be
done about it. So parents are very much out in front of this.
Second, they are put out in front by the testers, some of whom
have, frankly, commercial interests, so there is serious financial
conflicts of interest in some cases. That wasn't the case in
PKU, and it's not the case in all screening programs.
But there are commercial testing laboratories who would love to
have this happen, and are funding publicity campaigns, as happens
in other areas of medicine. So there is just frank commercial conflicts
Third, there are empires that don't make people rich, but
that make people more powerful or influential. Let me tell you
a personal story there. I've been interested in newborn screening
for cystic fibrosis. I was co-investigator of what is I think the
largest clinical trial ever done. Dr. Farrell at University of
Wisconsin randomized over 600,000 infants over a 10-year period,
to see if early diagnosis and treatment for cystic fibrosis was
Fifteen years ago, before that trial was even done, was even started,
I was invited by WHO to attend a technical assistance conference
to help other countries begin cystic fibrosis newborn screening.
There were 25 countries there — Bahrain, the Soviet Union,
Argentina. They all wanted to start newborn CF screening, and my
thought was, why? I don't know of any evidence that it's
I know of lots of reasons why it might be harmful. Our study
explicated and documented many of these harms, medical as well as
psychosocial. You have far more serious problems in your country
than cystic fibrosis. You don't even know what the incidence
of cystic fibrosis is in your countries.
My sense of what was going on is that the people at the meeting
were the equivalent of state lab directors who, you know, it was
another machine. It was another couple of people on their staff
whose expanded budget — it was getting more information, possibly
some research interest. I don't think they were getting rich
off of it, but they — technicians like to do things. Doctors
like to do things. Testers like to test. So that's part of
And part of it is very poorly thought out moral claims of justice
and unfairness. If Wisconsin is screening for 30 conditions, it's
unfair that a child in Iowa — and this has been the argument
used driving the Secretary's Advisory Committee as far as I
can tell. We need a uniform policy.
Well, if it's a bad policy, there's no virtue in it being
uniform. There is no unfairness in not distributing something that
has no known value or whose harms may exceed the benefits.
So those are some of the factors that have driven this, and they
have been repetitive.
CHAIRMAN PELLEGRINO: Dr. Lawler?
PROF. LAWLER: Yes. Thanks for the great presentation.
Just one point of clarification. The Baby Doe regulations were
a great advance in terms of protection of the rights of people with
Down's Syndrome. And that protection is more urgent than ever
as there are fewer and fewer people with Down's Syndrome around.
Now, you said that has led to a regime of overregulation, over
— of overtreatment — overtreatment, right. Now, the
only thing I didn't understand fully is whether that overtreatment
is based upon a misinterpretation of — a fearful misinterpretation
of the existing regulations, or if the regulations, as now exist,
need to be mended in some way, need to be changed in some way.
What's the bottom line on that?
DR.FOST: Thank you, again. I mean, it's as if I've
planted these questions. First, I agree with you completely that
the Baby Doe regulations were absolutely essential, or something
like them. That is, I hope I've conveyed my personal view that
it — I think I used the word "egregiously unethical"
withholding of treatment from children with Down's Syndrome,
spina bifida, and many other children, with excellent prospects
for meaningful, happy lives.
The overreaction, in my view — and Dr. Kopelman and Dr.
Nelson may disagree with this — my view is that overtreatment
that's based on Baby Doe regulations is based on a false understanding
of them. That is, I think the Baby Doe regulations have been widely
misinterpreted and misapplied.
For example, to whom do the regulations apply? Any regulation
has to say who it applies to. It applies to state health departments.
It's a regulation requiring state health departments to respond
in a certain way if there's a report of medical neglect, of
refusal to provide medically necessary treatment.
Absent a report, the state has no duties. And, in fact, the Inspector
General under President Reagan found all 50 states to be in compliance,
even though thousands of babies were still being allowed to die
in seeming violation of the substance of the rules.
The rules do not apply to doctors, hospitals, or parents. So
they don't, in my view, impose a single requirement on physicians,
families, and hospitals making local decisions. They're as
free as they ever were at — I mean, they are subject to tort
law and criminal law and all of the other laws that always did exist,
but in my view the Baby Doe regulations have actually no legal implications
for treatment decisions.
So the overtreatment that has been predicated on them is misstated
and is a misinterpretation of them. Is that responsive to your
PROF. LAWLER: Exactly.
DR. ROWLEY: Thank you very much, Norman, and it's
nice to see you again.
I have a different question, because you really raised three issues,
of which you dealt with only one. Our Council meets every few months,
and I think I've been a proponent of dealing with ethical issues
related to children. We have actually in the past had several people
come and speak with us about children, particularly early childhood
development and brain development in young infants and children,
which were very helpful for me.
I think that, in fact, we are going to have to make a choice,
because we can't deal with the three issues that you raised.
And you made a point that the issue you were actually going to discuss
affected far fewer children than the two that you did not discuss.
And so from your perspective of the Council and where —
I hate to use the term "where it could have most effect,"
because I'm all in favor of tilting at windmills if only —
if that's the major thing we should be doing. But I would appreciate
your opinion as to whether — which of the issues that you
raised, such as our dreadful neglect of children born into poverty,
infants born into poverty, or child abuse, as compared with those
that you spent the time discussing.
DR.FOST: Janet, thank you for bringing me back to what
really matters, as you always do. Yes. I didn't talk at length
about the problems of poverty or the solutions to it or the problem
of the uninsured out of sheer diffidence. I mean, because I don't
have any particular expertise, certainly compared to other members
of the panel or other experts who you could bring in.
I mean, I have an ordinary person's notion of what needs to
be done. But those are overwhelmingly the problems that oppress
and afflict American children — being born into poverty, into
situations in which access to health care are constrained.
And child abuse I would put third on the list, because that only
— but that only involves a few million children as compared
with the tens of millions — I mean, these numbers are just
absurd — who start off life with no fair opportunity to really
So those are the overwhelming problems. And, yes, if this Council
could have an influence on the President's policies or the country's
policies on something that really affected children, I would much
rather you spent all your time on doing something about problems
of poverty and access to health care and child abuse than these
other three angels on the head of a pin issue, which in the final
analysis affect very few children.
So, and part of it, I talked about things. There's a story
Robert Morley, the great British comic actor, tells that —
if you'll indulge me — he was explaining why he quit school
at age 12, and he said it was an exam question. The exam question
was, "Describe and discuss the Cape of Good Hope." "And
I wrote in my exam booklet, "I don't have the foggiest
idea where or what the Cape of Good Hope is, but I do know the 12
And he wrote the names of the 12 apostles and said something about
each. And he said, "They flunked me," and it showed me
once and for all they weren't interested in what I did know
but only in what I didn't know.
So I talked about what I do know, because it's comfortable,
and it seemed to me overreaching to go into any more detail about
things that I really am not very expert in.
DR. ROWLEY: No. But I — that was not the purpose.
The purpose was really to say if we as a group have to make a decision,
which will be in a sense a group decision, what kind of advice can
you, as a consultant, give us on that? So that was the purpose
of the question.
DR.FOST: My advice would be that you spend what time and
resources you have in making recommendations that affect children
who were born into poverty and children who are born without reasonable
access to health care. If you can make some recommendations that
might influence national policy in those areas, it would be time
PROF. DRESSER: I have a related question, perhaps to push
you in this area, because I think one of the reasons we haven't
done as much as we should in bioethics on these issues is because
we don't know how to approach it. It seems so overwhelming.
And I know that some of us on the Council do want to get into these
social justice questions.
So I guess one way I think about it is with limited resources,
how would you allocate them differently from the way they are allocated
now? And I'd be interested in what thoughts you might have,
not just about allocation of health care resources and how that
might better affect children, but also research resources.
So if you were thinking about taking the NIH budget and making
it more child friendly, how would you spend your money?
DR.FOST: You've caught me off guard, Rebecca, which
is good. So I don't have well thought out responses to that
question. So I'll think aloud.
First, let me just say with regard to resources in health care,
I've gotten into big trouble with the American Academy of Pediatrics
for suggesting that the way we deliver pediatric health care should
be dismantled. That is, having a pediatrician for every child is
an extremely expensive way of providing mega hours of service that
has no known relationship to health.
Roughly half of a pediatrician's time is spent doing health
supervision, which has no known relationship to the health of children.
And these are — everything that's done on those visits
could be done far more cheaply and efficiently by a nurse or someone
with even less training. I mean, all of the immunizations, all
of the weighing and the measuring and the eye checks, and all of
that, you don't need a doctor to do that. The doctor spends
almost no time doing anything that you need a medical degree or
So we could expand access of children if it were — if they
didn't have to be funneled through pediatric offices, if we
had more of a public health program with well baby stations as occurred
50 and 75 years ago, where immunizations were given and/or even
acute illnesses were managed by a non-M.D. So I would change that,
if I could.
I would change the allocation of health resources away from acute
illness towards more preventive services, of which child abuse is
simply the most important example I know of. That is, prevention
through home visitor programs rather than the $5 million treatment
of every kid who gets whacked in the head and then we take care
of him or her the rest of their life.
With regard to research, I would greatly shift the emphasis away
from biomedical research into more epidemiologic and public health
research. My M.P.H., my one year getting a master's in public
health, showed me just how much more powerful epidemiologic approach
is to health care, both in research and implementation. I could
give dozens of examples that are probably familiar to you of how
— I'll just give one just to make — make the point.
My colleague Murray Katcher, a pediatrician in Madison, discovered
that were 100,000 patients admitted to hospitals each year for severe
scald burns, almost all infants and the elderly. If the hot water
heaters were set at 130 instead of 140 — at 140 it takes two
seconds to get a third degree burn. At 130, it takes 45 seconds.
So you would end burns forever if the heaters were set at 130.
He set out to do that. And to make a long story short, he did
it. So there is an intervention that — all new water heaters
in America now are set at 130. You can't get people to turn
the old ones down. So it will be 40 years before they all expire.
There is a piece of research and implementation that cost zero,
didn't cost one cent, that is going to prevent hundreds of thousands,
millions of cases of suffering and death due to hot water burns,
child-resistant containers — another virtually free intervention
that has prevented tens of hundreds of thousands.
So I would shift the whole national research budget to more public
health epidemiologic study and interventions of that sort. And
I myself would spend less on hard science, assuming fixed dollars.
CHAIRMAN PELLEGRINO: Dr. Hurlbut?
PROF. HURLBUT: You mentioned the massive experimentation
that is being done through off-label use of drugs, and then you
also said that clinical research is dying, and that we're looking
at a whole new set of potential interventions that are quasi-therapeutic
related to genetic screening. And it seems by implication those
could go backwards, too, into fetal development eventually. In
other words, you might end up administering drugs to pregnant women
to — to deal with some things eventually.
Well, looking at it post-natally, is there a way we can enter
into this difficult arena through the current therapeutically justified
trials, research, therapeutically? In other words, given the difficulty
of defining and justifying non-therapeutic interventions, can we
get a better handle on some of the research that needs to be done
through the therapeutic trials?
DR.FOST: Yes, definitely. If the PKU program had been
subject to a — even a randomized clinical trial, which is
sometimes hard to justify for what you think is a uniformly horrendous
disorder, some children with PKU would have been lost — that
is, would fail to have been treated. But many hundreds —
we don't know the number — of normal children would have
been saved. That is, we would have found out in a year or two,
instead of 10 or 15 years, what we were doing.
So that's point number one is if these new technologies were
studied systematically, I think in the long run we're less likely
to harm and more likely to help.
Even for conditions in which it is hard to justify a randomized
trial, even a simple registry — I mean, we presently don't
have a requirement for states that are implementing all of these
programs to maintain a registry, a state or a national registry.
That alone would at least allow for some observational collection
of data, so that 10 years from now we know what the outcomes of
all these children are.
So, in my view, that's urgently needed — a national
registry for all children that are screened and treated as a result
of these new technologies.
Third, I think having the laboratories of the states that —
let me say that differently. Having variation among the states
I think is a good idea, not a bad idea. That is, right now some
states like Wisconsin are doing them. Others are holding off.
I hope it stays that way for a time, because it's not quite
a prospective randomized trial, but it's a reasonably controlled
trial. So that 10 years from now we'll be able to look at children
in Idaho and with — who have these conditions, and we can
always diagnose the conditions, and we can always get a sample of
blood and find out who has these chemical abnormalities and who
So I think in the absence of better knowledge having some variation
in the way states approach this is a healthy thing. I worry a lot
about a national standard being implemented. I hope that's
CHAIRMAN PELLEGRINO: Other comments?
DR. KASS: Norm, I wonder — again, this may be to
ask something outside of your area of expertise. But none of the
things that you have spoken about deal with the question of the
mental health of children. And I wonder if — I mean, child
abuse is one area, and the consequences of poverty and deprivation
imply that part of the cost is in terms of mental well being broadly
But do you have any suggestions along these lines? And not just
concerning, let's say, the health of the identifiable abused
children, but more generally whether there are things for this Council
to be thinking about in that area.
DR.FOST: Yes. In fact, I've written a couple of papers,
which I'd be happy to provide to you, on essentially the perils
of innovative therapy in emotional and behavioral disorders of children.
That is, the inadequate amount of prospective randomized, controlled
trials, the difficulty in getting funding is a big part there, but
also in getting investigators and some fighting off what I consider
to be somewhat demagogic opposition or resistance to experimenting
on children, or using them as research subjects, in treatment trials,
in therapeutic trials.
So I think mental health is especially forlorn as an area for
systematic research for interventions, pharmacologic, behavioral,
and others. And there's way too much innovative therapy that
is, as you probably know and other members of the Council, millions
— tens of millions of American children get SSRIs prescribed
for everything from depression to behavior problems to anxiety to
obsessive-compulsive disorder, with almost no data. And we now
know that these drugs have serious adverse effects, that suicidal
ideation is now — and suicidal behavior has been unequivocally
shown to be increased in children.
Well, that may be worth it if you're prescribing it for something
that's a real problem and for something for which there's
a real benefit, but we don't know that. And I could cite more
specific data, and I can send you the article that shows it.
So the general answer is there is a tremendous shortfall in the
amount of research that should be done on children with mental,
emotional, and behavioral disorders. There is way too much innovative
therapy going on, especially in that area.
DR. KASS: May I? This was welcomed, but let me —
let me just put it a little harder. I talked about mental health
and well being. The answer came back in terms of the medical treatment
of disorders. And this may be what Gil was hinting at when he was
asking about the drive toward testing.
Should we be concerned about what in previous discussions —
we've had a few discussions of this so-called problem of medicalization,
of the — the revision of the understanding of child development
and child-rearing in medical terms such that the turn is immediately
for some kind of medical testing, medical intervention.
Do we need — I mean, is this something that ought to concern
us? How one actually conceives normal child development, child-rearing
practices, and to what extent the desire to see these increasingly
either through neuropsychiatric terms or medical terms is itself
something to be worried about, or is it — are we going in
the right direction, but we just don't have enough of it?
DR.FOST: Okay. I think I understand your question better.
Well, let's take ADHD as an example. That often arises in these
discussions — attention deficit hyperactivity disorder. Explosion
in medical approach to that disorder, millions of prescriptions
a year for children — for treatment of a disorder which has
no gold standard test, for which diagnosis is uncertain, and criticism
by many people who think that whatever problem a child has is something
that would be better if it were understood as an environmental problem,
behavioral problem, a parenting problem, a school problem, and so
I think there's truth on both sides of that debate. That
is, I think there's no reason to be phobic about medicalization
of a problem, if medicine helps — if it's safe and effective,
and if we're sure it's getting to the right children, and
if we have systematic studies and have reasonable non-toxic drugs.
So the fact that ten million children are getting a drug that
may help them perform better in school, if it's a safe drug
and if it does help them perform better in school and makes their
lives better for that reason — I mean, hypothetically, if
the drug had no side effects and were free, I don't see any
reason, just because it's a drug, that we should be against
it. Nor would I see any reason to be against better schools with
better teacher-child ratios or better parenting or environmental
or behavioral approaches or nurturing that would produce the same
But the problem, as you know, is it's easier and cheaper to
find a drug that will solve the problem than to figure out how to
get the teacher-student ratio down from 30 to 1 to 10 to 1. So
I don't see any a priori reason to be for or against any of
Obviously, we should be doing research on all of these fronts,
and we should be looking for the most effective, most efficient,
safest, best way to help children. I don't see any reason to
be for or against drugs just because they're drugs.
CHAIRMAN PELLEGRINO: Dr. Meilaender?
PROF. MEILAENDER: This is a question that's related
in a way, though coming from a very different angle. And I can
only come at it — I mean, I'm not a practitioner, so I
just have to go on my own tiny little experience.
But it was interesting to me before, in an answer to an earlier
question, when you said that — I think you were talking about
allocation of resources or something and you said that — that
pediatricians spend a great deal of their time doing things that
you don't need medical training for. I mean, you could have
other people do them.
But that's connected to the medicalization question, because
I would have said — again, now just off — just off my
own personal experience, that pediatrics is the one area of medicine
where there is still a good bit of talk that goes on between doctors
Now, that's not something maybe that you need certain kinds
of medical training for, but I wouldn't want to call it an unimportant
part of medical care. And, indeed, I think from the lay person's
perspective one of the problems with — I mean, a lot of the
jokes that people make about doctors — there's no talk
that goes on. You've got to kind of yank him back in the door
So it just — the question that interests me is sort of how
we ought to try to think about these questions, and whether we need
to think about them in — well, I was going to say larger,
but that prejudges the question — just what the right frame
of reference is to think about these.
You see, I would say that it's good that pediatricians spend
a lot of time just talking, even if it doesn't require some
sort of hyperspecialized training. And I wonder if you could just
think about that a little bit.
DR.FOST: I think it would be good if pediatricians spent
a lot of time talking, but they don't. No, there are numerous
studies that show —
PROF. MEILAENDER: Mine do, but —
DR.FOST: Yes. Well, it has been studied. Maybe it's
not the same everywhere. I'm sure it's not the same everywhere.
But the studies that have been done show — first of all, I
think what — the main thing that parents want out of that
well baby visit is — is talk.
And to talk about the long list of concerns about child-rearing
and child behavior and what to do about drugs and alcohol, and all
of the other things that beset parents and children these days,
the problem is empirically that the amount of time that a typical
pediatrician spends talking about those things in a well child visit
is about 90 seconds. That's the average.
Second, those things could all be very well done, as good or better,
by a qualified and trained nurse, nurse's aid, social worker,
somebody who could be equally knowledgeable. You don't need
to go to medical school and take — in fact, there is very
little in medical school or in residency training on those issues.
Pediatricians get remarkable little training in all of that, in
some programs better than others. But the point is you don't
need a pediatrician to do it.
So if I just had access to the dollars that go into pediatric
care in America, defined as care that children get in pediatrician's
offices, I could get a much bigger bang for that buck, including
much more talk — and I don't mean to belittle talk at
all. I agree with you, I could get much more talk by reallocating
it to less expensive, equally qualified practitioners.
PROF. MEILAENDER: Yes. I don't really know exactly
what got me to this, or how we come to it from where you started
us. But I don't think it's just the amount of talk. It's
a question of who you're talking to. See, I mean, I want to
— when I'm sick, there are certain people I want to talk
Get out of the medical realm entirely, the large congregation,
of which I'm a member, sends all sorts of people to the hospital
to talk to members of the congregation. And as I once told my pastor
a while back, "When I'm in there, you'd better be there
to talk to me."
It's not just a question of amount of talk. It's the
question of to whom you're speaking. And, once again, that's
got something to do with the way we conceive of what's important
in medicine. I just — I don't want to lose that in the
focus on specific specialized technical details.
DR.FOST: You're referring or suggesting that the authority
of the doctor may have some added value that would not accrue to
a less prestigious person?
PROF. MEILAENDER: Well, that may be true. And it's
— you know, I take — I spent a lot of time and money
going to see this person, and I expect something other than just
DR.FOST: Right. I mean, I think when you're sick
or when your child is sick there's a lot of truth to what you
say, and validity to it. But if your child is bed-wetting, I don't
know that the kind of advice that you need, and the kind of remedies
that are available, and where do I start, and what do I do about
this, I don't know that there's much added value in having
somebody with an M.D. after his or her name as compared with somebody
who knows the field, knows what's available, knows what to do,
can get you started on the right track in an empathic way.
CHAIRMAN PELLEGRINO: Alfonso?
DR.GÓMEZ-LOBO: This is just a brief reflection
from someone looking at all of these things completely from the
outside. And I'm very much impressed by the — your reference
to poverty and to the deprivation of access to health of young children,
because of the social condition.
And it seems to me that that seems to be sort of a grounding reality,
and my reflection is simply this. As a Council, we should consider
that surely as part of bioethics, as part of social justice issues.
And I just hope that in the final report on issues relating to childhood
we go into that as deeply as we can.
Now, I think that there's an intersection with politics and
social policy that it's very hard for us to tackle. But, still,
it seems to me it — it seems to be so central, so deeply rooted
in virtually everything that happens afterwards that we should really
as a Council consider it, I think.
DR.FOST: Yes. Let me just give you one fact that ties
some of this together, some of my comments together. One-third
of all abused children in America are ex-prematures. One-third
of all abused children in America are ex-premature children.
Prematurity accounts for about six percent of American births.
It accounts for about 33 percent of child abuse in America. Who
gets born — the reasons for that are multiple. Prematures
are born, many of them, to teenage mothers. They are children who
are notoriously difficult to take care of. They have disabilities
and handicaps. There's a lack of bonding. In the newborn period
they're in an intensive care unit for weeks or months, and the
parents take this stranger home, and so on.
Who has premature children? Teenage mothers, poor mothers, mothers
without prenatal care, mothers who have health and drug problems
of their own. That is, poverty, lack of prenatal care, prematurity,
child abuse, if you could just reduce the incidence of premature
— our problems of prematurity dwarf. I mean, you know, it's
one of those many areas of American health where we're near
the bottom of the list of industrialized countries.
If something could be done to reduce that, there would be reduced
CHAIRMAN PELLEGRINO: Dr. McHugh? We have time for one
DR. McHUGH: Just one question. I enjoyed your presentation,
Dr. Fost, and I particularly appreciated some of those old stories
that I remember about how, really, the doctors lost the trust of
families who had given their children into hospitals and discovered
that — some of them discovered directly, others indirectly,
that the doctors and even the nurses themselves had lost some interest
in the loved one that they had submitted there.
And the loss of trust or the betrayed trust has produced what
it always does — namely, this increase in regulation, the
regulations that are completely out of control now and are interfering
with our capacity to work it.
But, you know, the blame does fall back upon us. We did it, and
now we are paying a terrible price. And to win back the trust of
the American people of this sort requires not only the presence
of IRBs but the particular demonstrations, as you've demonstrated
today, of the — of what we are losing.
Now, I'd just like you to look ahead now, since, you know,
we have — this world we're in now and we ourselves aren't
perfect — what kinds of things do you think we are going to
think in 10 or 15 years from now represent egregious betrayals of
trust in hospitals and places of that sort, to the families, that
bring our children — their children to them?
I wonder whether, for example, you might think in terms of whether
managed care in the great shortage of time now presented to —
in-hospital to patients and families that might lead — mean
longer time, or investments of more resources for the care of them,
even with chronic illnesses like the AIDS epidemic in young children,
and whether we are prepared to carry the concerns and services that
those kids need.
And you have mentioned in this little discussion with Dr. Meilaender
how little talk gets done. And maybe little talk gets done by pediatricians
and other M.D.'s because they don't know any psychology, and
we don't teach psychology in most — to most medical students.
It's the only basic science they don't know anything about.
And, eventually, even empirical psychology would help them in
this, and this neglect might be something that people will come
to regret and ultimately find, once again, imposed upon them. Do
you have any predictions from what we're doing now?
DR.FOST: Well, I'll accept your invitation to wax
political for a moment. If I — if you asked me to guess what
will most embarrass us, or make us ashamed of ourselves 25 years
from now, it would be the massive reallocation of funds from the
lower and middle classes to the upper classes.
That is, in a country that has these problems that we've been
talking about, the continuing widening of the gap between the rich
and the poor and the reallocation of funds in the — from those
who are least well off to those who are best off. That, to me,
should be an embarrassment, and I think will be. And the effect
that that will have on our children will become clearer as time
DR. McHUGH: Are we doctors contributing to that? Are
we — in the way we run our hospitals and insist on the lengths
of stay and things? Is that really — by the way, the care
of the poor is certainly something we all agree on, and those of
us who live and work in hospitals of — in the inner city see
this all the time.
But the pressure is not simply in relationship to whether the
government is going to take a concern for the poor, but whether
the hospitals themselves are contributing to the abuse of the poor
and the kind of regulations that they are prepared to enforce upon
their young physicians.
In our hospital, the doctors are told that they have to have a
certain short length of stay to be darn sure that they get the money
from — and these are for the poor.
DR.FOST: Well, you know, Justice Cardozo said laws aren't
written until they're first broken. So I think things like
managed care and length of stay rules, and so on, were necessary.
That is, I think the waste in the American health care system was,
and still is, prodigious, the inefficiency.
And given the finitude of funds that are available, I think squeezing
inefficiency out of that was unavoidable and necessary. And doctors
weren't doing it on their own, so payers had to hire managed
care companies to do it for them.
So I think that was unavoidable, necessary, and the studies that
I read — not all that bad in the final analysis. That is,
there are efficient managed care systems, like Kaiser Permanente,
where satisfaction rates are as high or higher in fee-for-service
systems and where patients are actually quite pleased with the care
they get, even though there has been a great squeezing down, and
I think another thing that we at least should be embarrassed about
25 years from now — whether we will be or not, obviously,
I don't know — is this constant ability to find new technologies
to spend our money on when we — we just — we're
just out of money when it comes to solving problems like the uninsured.
So it seems to me remarkable that the State of California has
$3 billion to spend on stem cell research. Forget the ethics of
stem cell research, about which you've dealt in great depth.
But that the citizens of that state could decide that they have
$3 billion for a technology that has as yet no known payoff, and
even when it does the number of people who it's likely to help
are going to be measured in a state that has prodigious problems
with education, with access to health care, with poverty, and so
We always can come up with a billion here and a billion there
for some new technical thing that is fun and fascinating. But we're
just out of money. We just can't afford to have basic access
to simple, ordinary, primary health care for 40 million people.
CHAIRMAN PELLEGRINO: Thank you very much, Dr. Fost. I'm
afraid the schedule requires that we intervene at this point and
have our break. I'll take the Chairman's prerogative of
extending it beyond the time in the agenda, which is supposed to
be at 10:45. And if you'll return at 10:50, we'll start
Thank you very much, Norman, for drawing on your extensive experience.
And let me thank the Council members for their close questioning
on a very important topic.
(Whereupon, the proceedings in the foregoing
matter went off the record at 10:39 a.m. and went back on the record
at 10:59 a.m.)