Ethical Issues in Resource Allocation, Research, and New Product Development

Chapter 14
Ethical Issues in Resource Allocation,
Research, and New Product Development
Dan W. Brock and Daniel Wikler
The ethical justification for developing and providing the
the production of health. Costs are measured in monetary
means to reduce the burden of disease in developing countries
terms; benefits are measured in health improvements. By divid-
is self-evident. Nevertheless, those who pursue these laudable
ing costs by benefits, one can obtain a cost-to-effectiveness ratio
ends encounter ethical dilemmas at every turn. The develop-
for each health intervention, and interventions can be ranked
ment of new interventions requires testing with human
by these ratios. Although a CEA is typically an economic analy-
subjects, an activity fraught with controversy since the dawn of
sis performed by health economists, it is also a measure of
scientific medicine and especially problematic with poor and
one ethical criterion for the evaluation of health programs.
vulnerable participants in developing countries. Ethical dilem-
Cost-effectiveness is not merely an economic concern, because
mas arising in setting priorities among interventions and
improving people’s health and well-being is a moral concern,
among individuals in need of care are most acute when needs
and an allocation of resources that is not cost-effective pro-
are great and resources few.
duces fewer benefits than would have been possible with a dif-
We address some of these concerns in this chapter, identify-
ferent allocation. Producing more rather than fewer benefits for
ing some of the principal ethical issues that arise in the devel-
people is one important ethical consideration in evaluating
opment and allocation of effective interventions for developing
actions and social policies.
countries and discussing some alternative resolutions. We omit
Second, the allocation should be equitable or just; equity is
discussion of two other aspects of these ethical decisions:
concerned with the distribution of benefits and costs to distinct
ensuring that the process of decision making is fair and
individuals or groups. The maximization of benefits, which is
involves the subject population (Daniels 2000; Holm 1998),
associated with the general philosophical moral theory of util-
and respecting legal obligations under international human
itarianism or consequentialism, however, is routinely criticized
rights treaties (Gruskin and Tarantola 2001).
for ignoring those considerations (Rawls 1971). Equity in
health care distribution is complex and embodies several dis-
tinct moral concerns or issues that this chapter delineates
HEALTH RESOURCE ALLOCATION
(Brock 2003a). There is no generally accepted methodology
comparable to CEA for determining how equitable a distribu-
Resource allocation in health and elsewhere should satisfy two
tion is; nevertheless, allocations are unsatisfactory if equity
main ethical criteria. First, it should be cost-effective—limited
considerations are ignored.
resources for health should be allocated to maximize the health
Efficiency and equity can sometimes coincide. In some of
benefits for the population served. A cost-effectiveness analysis
the world’s poorest countries, for example, health budgets
(CEA) of alternative health interventions measures their respec-
support tertiary care and travel to clinics abroad for the elite
tive costs and benefits to determine their relative efficiency in
and the well connected, even as the poor are denied effective,
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low-cost prevention or treatment for life-threatening diseases
undiminished function (or health) is generally determined by
(Birdsall and Hecht 1995). Moreover, because equity concerns
soliciting a group of individuals’ preferences for life in that state
the relative treatment of different individuals, CEA is largely
using standard gambles, time tradeoffs, visual analog scales, or
unobjectionable when it is used only for evaluating alternative
person tradeoffs. In all these methods, a common issue is
health interventions that would serve the same patients.
whose preferences to use for valuing health states. The main
However, considerations of equity may conflict with cost-
debate has been whether to use a randomly selected group of
effectiveness and so may provide moral reasons for an alloca-
citizens or to use people who have the particular disability or
tion that is not cost-effective. The discussion in this chapter
limitation in function being evaluated.
accepts that CEA identifies one important ethical criterion in
This issue matters because a number of studies have shown
evaluating health care interventions—producing the most ben-
that persons without disabilities generally evaluate the quality
efits possible for individuals served by those interventions—
of life with a particular disability as significantly worse than do
and then focuses on the other ethical criterion of ensuring
persons who have that same disability (Menzel and others
equitable distribution of those benefits.
2002). If the preferences of persons without disabilities are
This chapter considers two types of equity issues: first, those
used, their lower evaluation of quality of life with various dis-
that arise in the general construction of a CEA—that is, in
abilities will mean that fewer QALYs will be produced by life-
determining the form of a CEA; second, those that arise in the
saving interventions for persons with disabilities than if the
use of the results of a CEA for resource allocation in the health
preferences of persons with disabilities had been used.
sector. It is worth noting that, when applied appropriately and
However, if we use the preferences of persons with disabilities,
broadly to all social conditions and programs that significantly
then both prevention and rehabilitation will receive less value
influence health, CEA may often support using resources to
than if the preferences of persons without disabilities had been
affect the so-called social determinants of health—which
used.
largely affect the incidence of disease, disability, and premature
This difference in evaluations in part results from igno-
mortality—rather than using those resources on health care to
rance, prejudice, and stereotypes on the part of persons
treat disease. However, we shall focus largely on CEA in the
without disabilities about what it is like to live with various
evaluation of health care and public health programs.
disabilities. The difference results as well from the process of
adaptation to disability in which disabled persons adjust by
learning new skills, cope by adjusting their expectations to their
Issues in the Construction of a Cost-Effectiveness Analysis
new circumstances, and accommodate by substituting new
Cost-effectiveness analyses require decisions about which costs
aims and activities for ones made difficult or impossible by
to include, which if any financial gains should be counted as off-
their disabilities (Solomon and Murray 2002). They thus adopt
setting costs, whether to include benefits beyond the effects of
a new valuational perspective for making health and quality-
the intervention on health, and whether all health gains should
of-life evaluations. Because the adoption of this new perspec-
be valued alike. None of those decisions, in our view, is exclu-
tive resulted from a disability, it will represent a set of values for
sively a technical issue, and CEA results reflect the analysts’
making choices that reflects a restricted set of abilities.
ethical judgments on those issues.
Nevertheless, neither the nondisabled perspective nor the
adapted disabled perspective is mistaken; they are only different
Evaluation of Benefits. Evaluating health benefits within a
(Brock 1995). These differences create controversy in the litera-
CEA involves several issues. This chapter assumes that some
ture over which perspective is correct for cost-effectiveness
version of a quality-adjusted life year (QALY) is used to com-
evaluations in health care.
bine the two main benefits of health care—(a) protecting or
A second issue is whether, in evaluating interventions that
improving health or health-related quality of life and (b) pre-
preserve or extend life, we should use life years saved (as QALYs
serving life. Disability-adjusted life years (DALYs) are a variant
do) or lives saved. Certainly individuals offered two interven-
of QALYs in that they measure the losses from disability or pre-
tions that would preserve their lives for different lengths of time
mature death; a CEA will determine which interventions will
would prefer, all other things being equal, the alternative with
maximize QALYs or minimize DALYs. Calculating QALYs
the longer period of survival. Moreover, when the differences
requires a metric evaluating the effect of different states of lim-
are extreme—for example, extending group A’s lives by a week
itations in function on health-related quality of life, such as the
or extending an equally numerous group B’s lives by 10 years—
Health Utilities Index (Horsman and others 2003). The Disease
virtually everyone would judge this difference to support giving
Control Priorities Project uses the health state valuations or
priority to group B. This fact suggests that even the proponent
disability weights of the World Health Organization (WHO).
of counting lives saved should require that the lives saved for a
The relative value of any particular health state, typically on a
shorter period of time must still be saved for a significant
scale in which “0” represents death and “1” represents full,
period of time; what is significant will depend in part on the
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duration of lives saved by the alternative with which it is being
the moral urgency, grounded in fairness, of preventing the
compared. Some empirical studies indicate that ordinary peo-
death. The younger a person is, the greater is the moral value of
ple tend not to give much weight to differences in the duration
providing a QALY to him or her. This view leaves open to what
of health benefits to different groups of persons when priori-
extent the moral value of QALYs should decline with the age of
tizing between them, as long as the lesser duration benefits are
the recipient. This age weighting to favor the young has been
viewed as significant; this attitude suggests that they favor lives
attacked by some as unjust age discrimination, but because an
saved over life years saved (Nord and others 1996). The life
explicit moral justification in terms of fairness is offered for it,
years saved versus lives saved controversy remains unsettled.
critics must show why that justification is unsound.
Should Life Years Be Age Weighted? The standard assumption
What Costs Should Count in Health Cost-Effectiveness
in most CEAs using QALYs is that one QALY has the same
Analyses? No controversy surrounds the inclusion in a CEA of
social value, regardless of the age of the recipient (Gold and
direct costs of a health intervention program or direct health
others 1996). Thus, equality is adopted as the weighting for
benefits to the intervention’s recipients. Ethical issues do, how-
QALYs achieved by recipients at different ages, and that is the
ever, arise in other aspects of the cost calculation (Brock
approach adopted in this volume. The use of any age weighting
2003b). A full CEA of alternative health programs should take
that gives less value to benefits for the elderly than for younger
account of all the economic effects on public or private expen-
persons is often charged as unjust age discrimination. Even the
ditures of the alternative health interventions or programs
use of equally weighted QALYs is often charged as unjust age
under analysis. An example is provided in the consideration of
discrimination because, other things being equal, saving the
treatment for two alternative health conditions judged to have
lives of younger persons will produce more QALYs than saving
equally detrimental effects on patients’ health: the first condi-
the lives of older persons. The goal of lives saved, as opposed to
tion permits patients to continue working, and the second
life years saved, removes this disadvantage to the elderly from
interferes with regular work and so has large economic costs to
CEAs that use QALYs. However, if the relevant benefit is adding
the patients’ employers. Should the costs of treating the second
years to life, then standard CEA is neutral or impartial regard-
be reduced by the cost savings to the employers from returning
ing age, in the sense that it gives the same value to a year of life
the patients to work on a regular basis? If so, the second treat-
extension whatever the age of its recipient.
ment program will have a more favorable cost-effectiveness
WHO, in its burden-of-disease and resource prioritization
ratio than the first, even if it may be no better or worse without
studies that use DALYs, rejected the equal age weighting that is
consideration of those economic effects. The same issue arises
standard with QALYs. Instead, it gave less value to DALYs
in many other contexts.
prevented for infants, young children, and the elderly, in com-
From the moral perspectives of both a consequentialist and
parison with persons in their productive adult years. WHO jus-
a standard CEA, these indirect economic effects for others are
tified this weighting by the fact that the very young and the eld-
real benefits or cost reductions and should be part of the CEA.
erly both tend to be economically, socially, and psychologically
The fundamental moral objection to giving higher priority to
dependent on adults during those adults’ productive working
treating those who can be treated at lower net cost because of
and child-rearing years (Murray 1994). This justification is eth-
the economic savings to their employers is the same as that
ically problematic, however, because it assigns different value to
with WHO’s instrumental rationale for its age weighting. One
meeting people’s health needs on the basis of differences in the
condition or group of patients gets higher priority solely
instrumental value to others of meeting their needs. This
because treating it or them is a means to producing economic
approach differentiates people solely on whether they are a
benefits to others, thereby reducing the net social costs of their
means to benefiting others. The same reasoning would justify
treatment. This approach violates the Kantian injunction
giving priority to rich over poor patients with the same med-
against treating people solely as means—the first group has
ical needs because the rich are more socially productive than
lower priority for treatment solely because treating that group
the poor, a practice that would be widely regarded as unjust.
is not a means to the economic savings to employers. It fails to
Writers in this field have provided different reasons for giv-
give equal moral concern to the health needs of each group of
ing greater value to QALYs for younger patients, however, that
patients because it discriminates against the less socially valu-
are not subject to this moral objection and that are specifically
able patients. Conversely, at the macro level of the allocation of
grounded in fairness. For example, Alan Williams has devel-
resources to health instead of other social goods, the WHO
oped an argument to the effect that fairness requires that indi-
Commission on Macroeconomics and Health has supported
viduals should each receive “fair innings” of QALYs in their
increasing health investments in developing countries because
lives (Williams 1997). In this view, the earlier a preventable
such investments often more than pay for themselves in their
death could occur and the worse a person’s past health is, the
economic and development benefits (CMH 2001). Using a
greater is the unfairness the person suffers—so the greater is
“separate spheres” view, only the health benefits and health
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costs of alternative health interventions should determine
The ethical issue about discounting is whether, after taking
their priority for obtaining resources, but this view remains
account of such considerations, a health benefit of the same
controversial.
size has progressively less social value the farther into the future
Another aspect of cost calculation concerns whether future
that it occurs. To make the issue more concrete, suppose we
health care and other costs, such as old-age payments, that will
must decide between two programs: one will save 100 lives
be incurred as a result of a person’s life being saved should be
now, and the other, say a hepatitis vaccination program, will
added to the costs of treating that person now. Persons who do
save 200 lives in 30 years. The vaccination program will save
not die now because of a life-saving intervention will typically
twice as many lives, but if we apply even a 3 percent discount
go on to incur future health costs that would not have been
rate to the future lives saved, they are equivalent to only 78 lives
incurred had they died now. The U.S. Public Health Service
saved now, and we should prefer the first program. This exam-
Panel on cost-effectiveness recommended that inclusion of
ple illustrates not only the theoretical issue, but its practical
these costs be optional in CEAs (Gold and others 1996). Others
import, too, because discounting future health benefits will sys-
have argued that, if CEA is designed to maximize lifetime util-
tematically tend to disadvantage prevention programs that
ity, the future costs should be included (Meltzer 1997). These
must be undertaken now but whose benefits occur only at
are costs that would not be incurred if the patient was not
some point in the future. This reasoning applies not only to
saved, but virtually no one would argue that, because of those
many vaccination programs, but also to most programs to
costs, we should judge a life-saving intervention as not cost-
change unhealthy behaviors in which the benefits generally
effective and thus deserving of lower priority than interven-
occur at some later time.
tions that do not have those effects. What does this thinking
Arguments for discounting health benefits at the same rate
show? That we are not prepared to allocate health resources on
as costs have included consistency arguments (Weinstein and
the basis of a full CEA that accounts for all the costs incurred
Stason 1997), avoidance of paradoxes in allocation concerning
and saved by those interventions—that is, that some should be
research and deferral of spending (Keeler and Cretin 1983),
disregarded on ethical grounds.
individual or social rates of time preference, and so forth.
Those arguments cannot be reviewed here, but whether to dis-
Should Health Benefits and Costs Be Discounted in count health benefits is squarely an ethical question about the
Cost-Effectiveness Analyses? As standard practice in CEAs,
valuing of health benefits over time and should be explicitly
both health care costs and benefits are discounted at the same
addressed as such in allocating resources.
rate, for example, 3 percent or 5 percent, and the Disease
Control Priorities Project applies a 3 percent discount rate to
costs and benefits (Gold and others 1996). Little controversy
Issues in the Use of Cost-Effectiveness Analysis
surrounds the idea that future monetary costs and benefits
for Resource Allocation
should be discounted to their present value in a CEA. The same
It is now widely recognized that CEA alone is not a satisfactory
amount of money is worth more if received today than in
guide to resource allocation in all cases. CEA, as customarily
10 years because it can be invested at the market rate of inter-
formulated, measures the sum of costs and benefits and largely
est if received today. For the same reason, costs that can be
ignores the pattern of their distribution across the affected
deferred require fewer present dollars to meet them.
population. In some cases, the resulting allocation will strike
The controversial issue is whether health benefits should be
most observers as unfair. Health resource allocators need to
discounted—that is, whether the same magnitude of health
take distributional issues into account along with cost-
benefit has progressively less social value the farther into the
effectiveness.
future it occurs. This issue is complex and has engendered an
extensive literature that cannot be reviewed here, but we can at
Priority to the Worst Off. Justice requires a special concern for
least try to focus the issue. It is appropriate to discount for the
the worst off, as is reflected in aphorisms such as “you can tell
uncertainty about whether potential beneficiaries will survive
the justice of a society by how it treats its least well-off mem-
to receive a future health benefit and to discount for any
bers,” in the well-known Difference Principle in John Rawls’s
increased uncertainty about whether a benefit will occur
theory of justice, and by the special concern for the poor with-
because it is more distant. However, these uncertainties are
in many religious traditions (Brock 2002; Rawls 1971). This
reflected in the calculation of expected future benefits and do
concern is often understood to reflect a concern for equality—
not require that future benefits be discounted. Likewise, if indi-
in particular, equality in outcomes or welfare between people.
viduals receive a health benefit (such as regaining mobility)
In the health context, it takes the form of a concern for reduc-
sooner rather than later, their total lifetime benefit may be
ing inequalities in health between persons or groups. A variety
greater, but this fact, too, is reflected in the estimation of the
of ethical bases underpin a concern for equality in general and
total benefit without discounting.
for equality in health in particular, and they cannot be explored
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here. It is important, however, to understand that concern
Finally, how much priority should the worst off receive?
for the worst off is different from a concern for equality,
Giving absolute priority to the worst off is implausible because
because the two can be and often are confused. Raising the
it faces the bottomless pit problem—using very great amounts
position of the worst off will typically reduce inequality, but it
of resources to produce very limited or marginal gains in the
need not always do so. Sometimes improving the position of
health-related quality of life of the severely ill or disabled.
the worst off may unavoidably improve the position of those
However, there is no apparent principled basis for determining
who are better off even more and thereby increase inequality.
how much priority the worst off should receive.
Moreover, the concern for equality in outcomes is subject to the
“leveling down” objection, in which equality is achieved by
Aggregation and Cost Differences. The aggregation problem
making the better-off members worse off, even when doing so
occurs when determining at what point small benefits to a large
in no way benefits those who are worst off. In the face of that
number of persons should take priority over very large benefits
objection, many have rejected equality in outcomes in favor of
to a few, because the former result in greater aggregate or total
a prioritarian view, according to which benefiting people has
benefits (Daniels 1993; Kamm 1993). The issue can be illus-
greater moral value the worse off those people are (Parfit 1991).
trated by the initial effort to prioritize different treatment-
A number of possible lines of reasoning support prioritari-
condition pairs in the Medicaid program in the U.S. state of
anism. For example, the worse off that people are, the greater is
Oregon by what was essentially a cost-effectiveness standard.
the relative improvement that a given size of benefit will pro-
As was widely reported, capping teeth for exposed pulp was
vide them, so the more the benefit may matter to them.
ranked just above performing appendectomies for acute
Alternatively, the greater the undeserved health deprivation or
appendicitis, even though appendicitis is a life-threatening
need that an individual suffers, the greater is the moral claim to
condition. A variety of methodological problems affected
have it alleviated or met.
Oregon’s analysis, but this kind of result is to be expected from
However priority to the worst off is justified, an important
CEA. The Oregon Health Services Commission estimated that
issue is who the worst off are. In the context of resource alloca-
it was possible to provide a tooth capping for more than
tion in health care, the worst off might be those who are glob-
100 patients for the cost of one appendectomy, so the aggregate
ally worst off, those with the worst overall well-being (such as
benefits of the many tooth cappings were estimated to exceed
the poor), or those with the worst health (that is, the sickest).
the benefit of one appendectomy. As a consequence of results
General theories of justice usually focus on people’s overall
of this sort, the commission fundamentally changed its priori-
well-being, often allowing a lower level in one domain of well-
tization methodology to largely ignore cost differences, except
being to be compensated for by a higher level in another
in the case of roughly equally beneficial interventions. The
domain. However, there are both moral and pragmatic reasons
commission essentially adopted what might be called a relative
for what has been called a separate spheres view, according to
effectiveness or benefit standard (Hadorn 1991).
which the worst off for the purpose of health resource alloca-
What Oregon’s experience shows is that most people’s sense
tion should be considered to be those with worse health.
of priorities is determined by a one-to-one comparison of the
Morally, for example, Scanlon has argued that “for differences
benefits of different interventions, in which case appendec-
in level to affect the relative strength of people’s claims to help,
tomies are clearly a higher priority than tooth capping. That
these differences have to be in an aspect of welfare that the
ignores the great differences in costs between different health
help in question will contribute to” (Scanlon 1997, 227).
interventions that a CEA will reflect. Is it then simply a mistake
Pragmatically, it may generally be too difficult, costly, intrusive,
to ignore those cost differences in allocating health resources?
and controversial, as well as too subject to mistake and abuse,
At least two moral considerations suggest not. First, empirical
to have to inquire into all aspects of people’s overall levels of
studies have shown that many people ignore the cost differ-
well-being.
ences because they believe that patients should not be at a
Even if health allocation to the worst off should be based on
disadvantage in priority for treatment simply because their
levels of health, other issues remain. For example, are those
condition happens to be more expensive to treat than are other
with worse health those who are sickest now, at the time a
patients’ conditions (Nord and others 1995). Second, according
health intervention would be provided for them, or those with
to many moral theories, individuals should confront other
worse health over time, taking into account past and perhaps
competitors for scarce resources as individuals, and their prior-
expected future health? The latter would give special weight to
ity for treatment should be determined by the urgency of their
meeting the health needs of those with long-term chronic dis-
individual claims to treatment (Scanlon 1997).
eases and disabilities. Separate spheres would still include past
Then again, most people and most moral theories do not
and future health. Should special priority also be given to those
reject all aggregation of different sizes and costs of health ben-
whose health is not worse now but is especially vulnerable to
efits in setting priorities and allocation, although there is no
becoming worse?
consensus either on when aggregation should be permitted or
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for what reasons. However, at a minimum, we suggest that indi-
groups are treated. In the extreme case, some live and others
viduals should not be denied very great health benefits—in the
die. The better outcome is produced by funding program A
extreme case, life-saving interventions—merely to provide
rather than program B, but that additional good is insufficient
small health benefits to a large number of other persons.
to justify morally the huge difference in the way the two groups
of patients are treated. The conflict between fair chances and
Fair Chances and Best Outcomes. The thesis that resources
best outcomes can arise in a variety of contexts (Kamm 1993).
should be targeted to interventions in which they will do the
Preferring the most cost-effective program can also seem
most good ascribes a higher priority to those who can be helped
unfair because it compounds existing unfair inequalities. For
more easily or cheaply. This thinking, in turn, implies that some
example, screening slum-dwelling black men for hypertension
patients will lose out simply because their needs are more diffi-
targets the group with the highest incidence and greatest risk of
cult or expensive to meet. Consider, for example, a ward with
premature death. However, it is more cost-effective to
100 patients,50 of whom require one pill and 50 of whom require
target well-to-do suburban white men, because they have more
two pills to recover. The patients are otherwise similar. The clinic
ordered lives, comply better, have personal doctors and
has 50 pills and must decide how to distribute them. To achieve
the means to obtain medical services, are more educated,
the best outcome, all 50 pills should be given to the patients who
and are more likely to modify their lifestyles wisely. However, if
need only one to recover. However, to give each patient an equal
the poor black men are not screened for this reason, it only
chance to recover, entitlement to treatment should be awarded
compounds their existing unjust deprivation and, of course, is
randomly. Seventeen fewer cures would result.
also in conflict with giving priority to the worst off.
Limited surveys indicate a sharp difference between health
If those who need a less cost-effective program deserve a fair
professionals and the general public in their responses to this
chance to have their needs met, what would be a fair chance?
conflict. Most health professionals favor distribution to one-
Some argue that a fair chance is an equal chance, so some ran-
pill patients only, and most members of the general public
dom method of selecting which program to fund should be
insist that people should not be penalized for needing two pills
used (Broome 1991). Others suggest proportional chances or a
(Nord 1999). This division of opinion goes to the heart of CEA,
weighted lottery, in which the chance of each program being
which is precisely a guide to identifying the route to the best
selected is proportional to the amount of health benefit each
outcomes that can be hoped for with existing resources. It also
would produce, as a way of balancing fair chances against best
creates a dilemma for those health professionals who maintain
outcomes (Brock 1988). Alternatively, some resources might go
that health policy should be based on values most frequently
to each program (which is usually possible at the macro level),
endorsed by the population affected.
thereby benefiting some patients in each group—at least if
The conflict between fair chances and best outcomes arises
their relative benefits are not strikingly dissimilar—instead of
not only from differences in the costs of treating otherwise sim-
all going to the most cost-effective programs.
ilar groups of patients, but also when one group of patients will
Another consideration supports spreading some resources
receive somewhat greater benefits than another at the same
to less cost-effective programs instead of devoting them all to
cost. The appeal of a fair-chances solution is greater when the
the most cost-effective: to give all—or at least more—patients
difference in cost-effectiveness between the two programs is
a reason to hope that their health needs will be met. This con-
relatively small compared with the potential gain or loss to
sideration may be especially important in developing countries
individual patients. Suppose that health program A will pro-
where resource scarcity is more severe and adhering strictly to
duce 5,000 QALYs while program B will produce 4,500 QALYs
cost-effectiveness criteria could result in large numbers of
and that the effect on the health or life of each patient served is
patients with serious—or even life-threatening—health needs
large—in the extreme, life saving. Patients who would be served
having no hope that their needs will be met.
by program B could complain that it is not fair that all the
resources go to program A and none to B when they have
Discrimination against Persons with Disabilities. The use of
nearly as pressing health needs and would be benefited by
CEA in resource allocation to maximize the QALYs produced
treatment nearly as much as the patients served by program A.
by available resources will often discriminate against persons
If all cannot be treated, they might go on to argue, they deserve
with disabilities. Many persons with disabilities such as cystic
a fair chance to have their needs met rather than having no
fibrosis, HIV/AIDS, and chronic pulmonary or heart disease
chance for treatment only because treating them would pro-
have reduced life expectancies or health-related quality of life
duce slightly less benefit than treating the patients served by
as a result of their disabilities. Life-extending health care for
program A. The small difference in benefits produced for the
those people will produce fewer QALYs than for people with-
two groups—for example, a slightly greater life expectancy or
out them, all else being equal.
more serious disability averted in program A—they argue, is
When health interventions are aimed at improving quality
too small to justify the tremendous difference in how the two
of life rather than extending life, similar discrimination can
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arise. The presence of disabilities can act as comorbidities,
the practice or norms of medicine, which have the goal of
making treatment less effective or more expensive (or both)
meeting patients’ medical needs.
than it would otherwise be, thereby worsening its cost-
There are strong moral reasons for considerable caution in
effectiveness ratio relative to comparable treatment for persons
letting health resource allocation depend on individuals’
without disabilities. These effects of treatment can result from
responsibility for their health needs (Wikler 2002). For that
a disability that exists before treatment and is unrelated to the
practice to be fair to those whose needs receive lower priority
treatment provided. So it seems that a cost-effectiveness stan-
because of behavior, (a) the needs must have been caused by the
dard for resource allocation discriminates against such persons
behavior, (b) the behavior must have been voluntary, and (c)
specifically because of their disabilities. Moreover, this effect
the persons must have known that the behavior would cause the
will arise not only in the case of preexisting disabilities, but also
health needs and that if they engaged in it their health needs
in the case of patients who become disabled as a result of treat-
resulting from it would receive lower priority. Smoking shows
ment that is only partially effective.
that these conditions are not easily satisfied. Smoking is one
Several strategies to avoid this discrimination in resource
causal factor in much cancer and heart disease, but many smok-
allocation have been suggested. Perhaps the most plausible, at
ers do not get those diseases, indicating that other factors, no
least for the case of life-sustaining treatment, is to ignore dif-
doubt in part genetic differences for which individuals are not
ferences in patients’ posttreatment quality of life as long as each
responsible, also play an important causal role. Smoking is typ-
patient accepts and values that quality of life and to ignore dif-
ically begun when individuals are young adolescents, and as dis-
ferences in life expectancy after treatment as long as each will
cussed in chapter 46, it is highly addictive, which undermines
receive a significant gain in life extension; obviously, what
the voluntariness of continuing to smoke. Individuals in indus-
counts as significant is vague and needs finer definition.
trial countries are now generally familiar with the health risks of
Ignoring differences in life expectancy posttreatment fits with
smoking, but this is less true among less educated populations
empirical evidence that individuals give little weight to dura-
in developing countries, where smoking is an increasing prob-
tion of benefits in prioritizing between health interventions
lem. No one anywhere has been informed before they smoke
that serve different individuals.
that, if they do, their health needs from smoking will receive
lower priority for treatment than will other health needs.
Thus, it would generally be unfair to give smokers lower pri-
Cutoffs for Cost per Quality-Adjusted Life Year. It is not
ority for treatment of smoking-related diseases on the grounds
uncommon in health care allocation to suggest the use of cut-
that they were morally responsible for those health needs,
offs tied to cost per QALY, although the cutoffs suggested vary
although there may be other behaviors for which individuals
substantially depending on the overall wealth of the country
could more justifiably be held responsible. Moreover, attempt-
and on the amount that it spends on health care. The cutoffs
ing to make those judgments in individual cases would be
can be of some value in identifying health interventions that
extremely difficult and controversial. Given the difficulty of
are either good or poor buys, given the society’s overall wealth
instituting a fair practice that allocates health resources accord-
and overall level of health spending. However, it is important to
ing to people’s moral responsibility for their health needs, we
be clear that such cutoffs should never function as anything
generally have good moral reason to preserve the egalitarian
more than a rough initial guide in health resource allocation.
feature of the practice of medicine that looks to patients’ needs
The various equity considerations discussed briefly above can
for care rather than to whether they deserve care.
serve as justification for departing from or violating any cutoffs
related to cost per QALY.
ETHICS IN RESEARCH AND NEW
Responsibility for Health Needs. Some have suggested that
PRODUCT DEVELOPMENT
health needs for which individuals are morally responsible
should have lower priority than health needs for which indi-
All new drugs and other medical products must be tested on
viduals are not responsible (Moss and Siegler 1991). If individ-
human subjects before they are sold. Although participation
uals are responsible for their health needs and could have taken
in health research is often a valuable opportunity for partici-
steps to avoid them, they have weaker claims on social
pants, what happens to them is determined not only by their
resources to meet those needs than do individuals whose health
clinicians’ therapeutic intent (if any) but also by the need to
needs are no fault of their own and could not have been pre-
ensure that the research yields useful information. Managing
vented. Smoking and substance abuse are two of the most
the potential conflict between those motivations is often an
prominent examples of behaviors often cited. However, differ-
ethically challenging task, and the issues become particularly
entiating patients by whether they deserve care on the basis of
contentious when research is conducted in developing
whether they are responsible for their health needs does not fit
countries.
Ethical Issues in Resource Allocation, Research, and New Product Development | 265

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Developing Consensus on Ethics and Human
scientists must explore the range of reasonable therapeutic
Subjects Research
alternatives with the patient. Potential subjects must under-
The central ethical question in health research that involves
stand that their participation is completely voluntary and that
human subjects is what may be asked of some individuals so
they may withdraw at any time and for any reason. Because
that others may benefit. The question arises in any research in
they cannot voluntarily shoulder risks, further protection must
which human subjects are asked to participate, but is most
be provided to those who cannot give consent. Such people
pressing if the care that is offered to subjects provides no ther-
include, among others, mentally incompetent or immature
apeutic benefit or if that care is compromised by the require-
participants and those involved in research (chiefly in social
ments of the study design. Informed consent, while in most
psychology) that requires initial deception. Consent, however,
cases a requirement for ethical justification of research involv-
is not sufficient to ensure fairness; there should be additional
ing risk, does not relieve the scientist of responsibility. The eth-
safeguards against unfair distribution of the burdens and ben-
ical question is what potential subjects may be recruited for,
efits of research. Finally, all research that involves potential risks
even if they do consent.
should be reviewed by an IRB acting on the basis of interna-
A rough consensus exists worldwide on the elements of
tionally recognized ethical principles.
research ethics and, increasingly, on the central role of the eth-
The global acceptance of these principles and the rapid
ical review committee, or institutional review board (IRB).
development of capacity for ethical review attest to the per-
This consensus can be traced back to the post–World War II
ceived validity of this system of rules and procedures of ethi-
international determination to ensure that the kind of barbaric
cal review. However, there has been relatively little research on
research practiced by Nazi scientists would not again stain the
how IRBs actually perform. Many IRBs in smaller institutions
good name of medical science.1 Three advisory documents
lack the necessary expertise to review novel or complex pro-
have been particularly influential. The Nuremberg Tribunal
posals, and their institutional setting creates a potential con-
that conducted the postwar Doctors’ Trial promulgated a code
flict of interest. Government investigations of the adequacy of
of conduct for medical research that stressed the requirement
IRBs for the tasks that are now assigned to them have often
of informed consent. The World Medical Association issued the
been critical (for example, Office of the Inspector General
first version of its Declaration of Helsinki in 1964 and has
1998, 2000). IRBs are often overworked and understaffed,
revised it several times. A further set of guidelines, issued in
resulting in ever-lengthening delays between initial submis-
1993 and revised a decade later, was published by the Geneva-
sion of protocols and final approval. Regardless of the value of
based Council for International Organizations of Medical
IRBs, predicting what will pass through them and what might
Sciences. Although they lack the force of law, these documents
provoke delay or rejection has become a significant concern
are widely acknowledged as international standards. Indeed,
for medical researchers. The system thus has costs as well as
the World Medical Association’s periodic revisions of the
benefits, a fact that lends additional gravity to the controversies
Declaration of Helsinki have become focal points for interna-
that it must resolve.
tional debates over outstanding issues in research ethics.
The most elaborate codification of research ethics is the so-
called Common Rule of Conduct of the U.S. Code of Federal
Goals of Ethical Review of Research
Regulations (title 45, section 46), which derived from the work
Although the overall purpose of ethical review is to ensure that
of the National Commission for the Protection of Human
research with human subjects is ethically defensible, the inter-
Subjects of Biomedical and Behavior Research of the mid
national consensus specifies several distinct goals that are
1970s. In addition to proposing rules governing many aspects
sometimes in tension with each other:
of research with human subjects, the commission proposed
that the IRB be given the central role in research ethics and be
• Protection. Ethical review committees can protect subjects
responsible for prior review of research proposals.2 The IRB
by alerting investigators to unforeseen hazards and by sug-
was a compromise, granting a measure of self-regulation to sci-
gesting research designs that can avoid unnecessary risk or
entists and an assurance of ethical conduct to the government
reduce the number of subjects exposed to risk. By insisting
and the public for publicly funded investigations.
that a clear explanation of risks and benefits be provided to
The basic elements of research ethics engender little dis-
potential participants, ethical review committees also help
agreement. The research must never be brutal or inhumane,
potential participants to protect themselves. Ethical review
and all unnecessary risks should be eliminated. Any net risks to
committees often take the name “Committee for the
subjects must be justified by the prospect of potential benefits
Protection of Human Subjects,” reflecting a central preoccu-
to others. Prospective participants must be told that they are in
pation of research ethics today.
a study and must be informed of its nature and its risks and
• Assurance that participation is voluntary. Some research can-
benefits. In the case of research that offers therapeutic benefit,
not be conducted without asking some participants to
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endure discomfort or pain, to delay relief from symptoms of
Current Controversies in Research Ethics
their disease, or to risk other harm so that future patients
Some of the most sharply disputed issues have arisen in inter-
may benefit. Permitting investigators to approach potential
national collaborative research involving scientists and spon-
subjects in these cases requires an ethical judgment. In
sors from wealthy countries conducting experiments in devel-
approving such a proposal, the function of the ethical
oping countries. Some of the problems are procedural. For
review committee is not, strictly speaking, only to protect
example, U.S. agencies have insisted on the same kind of
the subjects (the goal of protection would often be served
recordkeeping for IRBs in developing countries that is required
more effectively by declining to do the research), but also to
of IRBs in U.S. research institutions. IRBs in developing coun-
permit them to be enlisted in the effort to improve health
tries may accept the same principles of accountability, but they
care for others. Thus, a second function of ethical review is
do not have the elaborate staffs and budgets that leading IRBs
to ensure that those who agree to participate do so volun-
rely on.
tarily and freely and that they understand what is being
The most difficult disputes involving the ethics of research
asked of them.
in developing countries are, however, substantive rather than
• Equality and fairness. Although research ethics committees
procedural.
have little authority to address persistent social injustices, a
third concern of research ethics is that the benefits and bur-
Standard of Care. The international guidelines used in navi-
dens of health research be distributed fairly. This function
gating the ethical dilemmas of research in developing countries
receives relatively little attention in the literature of research
were created for the very different purpose of ensuring that
ethics, despite its prominence in such well-known docu-
what happened at Dachau and Auschwitz would not recur. It is
ments as the Belmont Report of the National Commission
not clear whether those rules usefully resolve the kinds of
for the Protection of Human Subjects (1979). Many of the
dilemmas that arise in, say, Uganda or Peru.
most notorious abuses of research subjects, including the
The Declaration of Helsinki, following the Nuremberg
Nazi investigations in the concentration camps, the Japanese
Tribunal, requires informed consent of all competent research
biowarfare experiments on Chinese and other civilians,
subjects, and in section 29 states that “the benefits, risks, bur-
and the Tuskegee research on African Americans suffer-
dens, and effectiveness of a new method should be tested
ing from syphilis, were committed on subjects chosen
against those of the best current prophylactic, diagnostic, and
exclusively from disadvantaged groups.
therapeutic methods.”
To its supporters, any departure from the letter of the
Those three goals of ethical review—protecting subjects;
Declaration of Helsinki that would permit an experiment in a
ensuring voluntary, informed participation; and reviewing the
poor country that would be forbidden in a rich one would
fairness of recruitment—are promulgated in the international
constitute a double ethical standard. In their view, this clause of
guidelines and in the Common Rule (and in the regulations of
the Declaration of Helsinki affirms the equal importance of
other countries), but they do not always point in the same
human lives, regardless of wealth or nationality, and stands as a
direction. For example, a research project that asks participants
safeguard against exploitation of those made vulnerable by
to endure a burden or risk—thus failing to offer full
poverty, sickness, and absence of governmental protection.
protection—can still meet the requirement of equality if the
Opponents, however, argue that this position seems to rule
burden is equally shared.
out the possibility of testing cheap new products that may be
Ethical review, thus, is not a matter of applying a checklist,
effective, although perhaps not as effective as other products
but it imposes an obligation of substantial ethical judgment. A
that the population could not afford. If so, it would be difficult
key challenge for IRBs is to earn and retain the trust of partic-
to understand whom the single-standard-of-care position
ipants and of the public, a task made more difficult by the
would be protecting, for surely it is better for a seriously ill per-
unavoidable absence of explicit criteria for approval. This prob-
son to receive a good drug, even if it is not the best, than to
lem is exacerbated by the institutional conflict of interest
receive no drug at all.3
inherent in the placement of the IRB within the research
Both points of view deserve respect. The single-standard
institution, which prompts concern that the committees will
approach is consistent with the postwar consensus on princi-
downplay risks to subjects for projects that profit or benefit the
ples of research ethics, and it offers a bright line between
institution or its influential staff members. Conversely, IRBs
research that amply respects human subjects and that which
that are fearful of institutional embarrassment or legal sanction
might result if sponsors and scientists were tempted to roam the
in the event of any harm befalling research participants might
globe in search of human subjects who could be used as exper-
lean too far in the direction of overprotection of subjects, at the
imental material with a minimum of expense or trouble.
expense of important scientific research initiatives. Both con-
Opponents of the universal-standard view, however, chal-
cerns have been raised about the IRB system.
lenge its premise. It made sense to insist on a single, universal
Ethical Issues in Resource Allocation, Research, and New Product Development | 267

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standard when the problem was Nazi barbarity, because the
beneficiaries of the research and possibly against the immedi-
prevailing standard was high and the medical criminals in the
ate interests of the women in the control group.
death camps denied it to the imprisoned minority—people
unjustly stripped of their entitlements. In Uganda or Nepal,
Rights of Host Communities. Ideally, research involving
however, care at the highest world standard is available, if at all,
human subjects would be a cooperative endeavor for mutual
to only a small elite.
advantage among free citizens who understand and endorse
A full reconciliation of those points of view may not be pos-
the need for research and who expect to share both in the bur-
sible. The authors suggest that a relativized standard should be
den of serving as research subjects and in the eventual benefit
considered only when the beneficiaries will include the impov-
of improved health care. Societies that recruit subjects prima-
erished, sick population. Even in those cases, however, the local
rily from lower socioeconomic strata fall short of this ideal;
standard of care could be adopted in the experiment only if it
those that do not offer new advances in care to all of their citi-
met or exceeded the standard provided by other countries at
zens fall even further short, raising serious questions about
similar levels of development.
fairness. Furthest of all from this ideal are some instances of the
increasingly common practice of recruiting research subjects
Placebo Controls and Other Issues Involving Research
among the poorest people in the poorest countries. The means
Design. For certain purposes, scientists use a placebo control
for protecting human subjects in these countries are often non-
even though a proven treatment exists. Patients in these control
existent. Most of their citizens will be unable to afford new
groups thus receive care that is inferior to what they would expe-
drugs developed by firms in industrial countries. It is not clear
rience in good clinical care. Until very recently, the Declaration
that these subjects participate voluntarily. Their lack of scien-
of Helsinki flatly condemned this practice (its current language
tific education or even literacy limits their ability to understand
is somewhat less restrictive), but the U.S. Food and Drug
the terms of the proposed agreement with the scientists and
Administration (FDA) accepted results of these trials in applica-
sponsors (particularly when consent forms, on legal advice, run
tions for approval of new drugs. The FDA’s justification for this
to 20 dense pages), and poverty often deprives them of any
acceptance rests on two claims, one scientific and one ethical.
alternative means of recovering their health.
The first is that in certain contexts (for example, for conditions
Despite these potential ethical shortcomings, international
such as depression, in which eligibility criteria and outcomes are
collaborative research is assured of continued growth. Some of
subjective, to an appreciable extent, and in which symptoms
this research targets diseases affecting mainly poor people, who
fluctuate in both treated and untreated patients), active controls
as a group suffer more from too little research on their popula-
may produce misleading indications of equivalency, yielding
tions than from too much. Even research intended to develop
seemingly positive results that may be spurious. The second is
therapies that will be affordable only to much wealthier
that when only placebo controls can be informative, it is some-
patients can be defended. Individual participants may receive
times justifiable to ask participants to be randomized with
better care than they would otherwise, and visiting scientists
placebo and thereby to risk discomfort and distress (but not any
offer employment and technical training.
appreciable risk of death or long-term impairment).
To right the perceived imbalance in what is asked of research
Debates over placebo controls are often joined in the con-
subjects in poor countries and the value that is obtained by sci-
text of disputes over the appropriate standard of care that arise
entists who experiment on them, some have proposed that
in the case of research in developing countries, but placebo
sponsors of research in the poorest countries compensate their
controls are controversial in trials in high-income countries,
hosts by offering a supplementary benefit (Glantz and others
too. Placebo controls are one instance of a large category of
1998). One much discussed option is access following the end
ethical issues in research that require weighing the importance
of the study to any drugs or other therapies whose effectiveness
of a scientifically ideal research design against the well-being of
is confirmed in the research. The most limited proposals would
participants. For example, a study of long-term chemotherapy
restrict this entitlement to individuals who were enrolled in the
to prevent the recurrence of breast cancer was halted before the
study (those who received placebo as members of a control
designated endpoints had been reached after the study’s Data
group, for instance), and time limits (such as three years) have
Safety and Monitoring Board decided that continuing the
been proposed in the case of chronic diseases such as
study after a strong trend had been established favoring the
HIV/AIDS. More expansive community benefit proposals have
chemotherapy would be unfair to the control group. It is
called for lifetime access to the treatments by all participants,
notable that in this instance severe criticism of this decision
their families, other members of the local community, or even
was voiced by an organization representing women at risk for
all citizens of the country. Other proposed benefits include a
breast cancer, as well as by the editorial board of the New York
specified amount of technology transfer, including scientific
Times. Critics of the early termination of the trial were,
training and the construction of clinics and laboratories, and
in effect, aligning themselves with the interests of future
cash payments earmarked for health care. A moderate proposal
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