DO FAIR AND JUST SYSTEMS REQUIRE COMPENSATION
FOR THE DISADVANTAGES OF THE NATURAL LOTTERY?
A DISCUSSION ON SOCIETY’S DUTIES ON THE
PROVISION OF GENE THERAPY
Ekmekci PE1,*, Güner MD2
*Corresponding Author: Perihan E. Ekmekci, M.D., Ph.D., Department of History of Medicine and Ethics,
TOBB Economics and Technology University Medical School, Sogutozu Cad. No: 43, 06560 Ankara, Turkey.
Tel: +90-532-262-0350. Fax: +90-312-292-4432. E-mail: p.ekmekci@etu.edu.tr; drpelifek@gmail.com
page: 69
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INTRODUCTION
Case 1. Three-year-old Baby X is brought to an emergency
service with severe abdominal pain. Her physical
examination show fat deposits under her skin and severe
hepatosplenomegaly. The doctors’ first diagnosis was acute
pancreatitis and further tests showed that she had lipoprotein
lipase deficiency (LPLD), a genetic metabolic disorder
that disrupts the normal breakdown of fats in the body due
to a deficiency of the lipoprotein lipase enzyme. Lipoprotein
lipase deficiency is inherited in an autosomal recessive
pattern, which means that the parents of an LPLD patient
carry one copy of the mutated gene, but they typically do
not show signs and symptoms of the condition. The disorder
in Baby X’s fat metabolism results in her blood having
a milky appearance and causes a massive accumulation of
fat in her tissues and increases plasma triglycerides. The
doctors tell the family that if Baby X remains untreated,
she will experience several acute pancreatitis attacks that
would damage her pancreas and may be life-threatening.
A lifetime of adhering to a diet restricted of fats was the
only advice doctors could provide for Baby X. However,
doctors warned the family to be aware of abdominal pain,
as dietary restrictions usually fall short of avoiding acute
pancreatitis attacks, as well as other health risks such as
depression, memory loss, mild intellectual decline and
dementia, which Baby X may suffer from in the near future.
Case 2. Six-month-old Baby Y has been admitted to
a pediatric service due to repeated and severe infections.
First it was pneumonia, accompanied by a high fever and
cyanosis that turned Baby Y purple due to oxygen deficiency.
He developed a widespread skin rash accompanied
by chronic diarrhea. Doctors also observed developmental
delays, which alerted them regarding a possible genetic
disorder. Several tests were administered and finally Baby Y was diagnosed with adenosine deaminase (ADA) deficiency,
a genetically inherited disorder that causes severe
combined immunodeficiency (SCID) by destroying the
immune system. Babies with a lack of immune protection
from any kind of microorganism are susceptible to
repeated and persistent infections that can be very serious
or life-threatening. Doctors said that without treatment,
these babies usually do not survive past the age of 2. The
treatment options include early diagnosis and treatment for
the infections and intravenous immunoglobulin injections.
Bone marrow and stem cell transplantations are investigated
with high prospects of effectiveness. However, the
long term prognosis depends on the severity of the disease
and the timing of the diagnosis. Severe and underdiagnosed
babies typically do not survive past the age of 2.
Patients such as Baby X and Baby Y are destined for
very restricted lifestyles, if not premature death. Research
on genetic disorders such as LPLD and ADA-SCID aim
to develop treatments for these kinds of genetic diseases
that have high rates of morbidity and mortality. Alipogene
tiparvovec (brand name Glybera), is a gene therapy product
indicated for the treatment of LPLD, which was approved
for use in the European Union on March 8, 2004 [1], and
to date has only successfully treated one patient. After
Glybera, Strimvelis was the second gene therapy approved
by European Medicines Agency (EMEA) for the treatment
of ADA-SCID [2]. It was the first instance of marketing
approval for an ex vivo gene therapy in Europe, paving
the way for the next generation of advanced medicinal
therapy products [3]. Following these interventions, the
number of approved gene therapy medicines increased in
a short period time. By the end of December 2017, there
were four gene therapies approved by the Food and Drug
Administration (FDA) in the United States: Imlygic (talimogen
elaherparepvec), a genetically modified oncolytic
viral therapy indicated for the local treatment of melanoma
[4]; Kymriah (tisagenlecleucel), the first gene therapy drug
approved to treat leukemia [5]; Yescarta (axicabtageneciloleucel),
a gene therapy for lymphoma [6]; and Luxturna
(voretigeneneparvovec-rzyl), the first gene therapy to treat
a rare inherited disease, retinal dystrophy [7].
There is no doubt that Glybera and subsequently approved
gene therapies have made giant strides forward
for patients such as Baby X and Baby Y to have a normal
life-span, which was only a remote possibility before.
These interventions showed that gene therapy, once considered
too risky, is safe and can save the lives of patients
with previously untreatable lethal diseases. However, the
positive expectations from gene therapy are overshadowed
by their extremely high prices. The unit price of Glybera
was approximately ˆ1 million per patient [8] and it has
only been used to treat one patient [9]. Glybera’s market
authorization holder did not apply for the renewal of its
license and in only 5 years, Glybera’s status has changed
from being a miracle cure to a commercial disaster, at least
for the company.
The incredible potential of gene therapy implies that
the share of gene therapy drug spending in total pharmaceutical
budgets will likely increase even more in the
coming years. These economic concerns drive discussions
about society’s duty to provide these treatments to those in
need. These discussions mostly focus on how to meet the
needs of genetic disease patients without violating the notion
of justice and fairness in society. Disorders or diseases
of a genetic origin have been considered a result of bad
luck in the natural lottery, and therefore, have been excluded
from the domain of justice for centuries. However,
the novel genetic knowledge that we have acquired and
the interventions that gene therapy make possible, require
that we reconsider the appropriateness of this exclusion.
This study discusses the theoretical grounds for society’s
duty to provide available gene therapies to patients in
need in order to compensate for their disadvantages. The
main question is whether a fair and a just system requires
that society provide available lifesaving gene therapies to
those in need. This article discusses the plausible answers
to this question in the domain of justice and fairness. The
discussion is constructed around the crucial role of the notion
of the fair equal opportunity principle in a just system
and the plausibility of including disadvantages emerging
from bad luck in the natural lottery in the domain of justice.
The discussion proceeds with particular interest in the distinction
between genetic diseases and other consequences
of the natural lottery, such as personal traits and features.
Justice and the Fair Equal Opportunity Principle.
Theories on justice set forth principles in order for society
to have a fair fundamental structure. This fair structure
enables individuals in that society to make and realize
their own life plans depending on their own concepts of
the “good life.” Individuals require some primary goods
in order to fulfill this end. These primary goods can be
natural or social in source. Income, welfare and equal
opportunity constitute some of the primary social goods,
whereas intelligence, vigor, and imagination are among the
primary natural goods. John Rawls’ [10] theory of justice
is one of the most influential contemporary theories in this
domain. His theory states that a fair social system requires
the establishment of institutions to distribute the social
primary goods due to principles of justice and fairness,
and these principles are determined in a hypothetical situation.
In this hypothetical situation, rational individuals are
behind a veil of ignorance and do not have any information
regarding their social status, gender, age, ethnicity, abilities,
level of intelligence, and level of education. The veil of ignorance guarantees the objectivity and impartiality of
fundamental principles of justice. Rawls [10] puts forth
that rational individuals will agree on two fundamental
principles of justice in the hypothetical original position:
1) Every individual should have equal liberty and fundamental
rights. Political liberties, liberty of conscience,
freedom of speech and gathering, freedom of expression,
self-respect, the right to personal integrity, the right to
property, the right to not be arrested arbitrarily, and freedom
of thought are addressed in this principle. 2) Social
inequalities should be avoided by the difference principle
and every individual should have an equal opportunity to
apply for positions in institutions with responsibility and
authority for the administration. Rawls [10] thinks that
the difference principle, which frankly states that “the
inequalities of income and welfare are considered to be
fair if and only if these inequalities are for the benefit of the
worst off,” would ensure fairness in society. The difference
principle implies that the social primary goods do not have
to be distributed in absolute equality; on the contrary, they
should be allocated in a way that maximizes the benefit of
the worst-off groups in society [11].
In addition to John Rawls [10], many other contemporary
theorists have considered establishing equal opportunity
as a crucial factor for a just system and to require
compensation for the disadvantages hindering individuals
from having this equal opportunity. However, the discussion
regarding the extent of this compensation has not
reached an end [12]. It is plausible to say that there is
unanimity regarding the elimination of legal barriers to
provide equal opportunity for people with similar talents or
qualifications, and that there is a high degree of consensus
about avoiding informal barriers such as discrimination
based on ethnic identities, gender orientations, or religions.
The discussion aggravates when compensation for disadvantages
resulting from bad luck in the social or natural
lottery is suggested. Equal opportunity clearly demands
that positions and posts should be open to all applicants
and that the selection of applicants should be based on appropriate
criteria relevant to performance, and that the most
qualified applicant should be offered the position [13].
However, this perspective does not necessarily ensure that
all people enjoy equal opportunity in practice, as acquiring
the merits and capabilities required for a particular position
or post greatly depends on an individual’s initial starting
point in life. The social, cultural, and economic factors
in which an individual is born may enhance or impair
their chances to develop the required talents or abilities.
Rawls’ [10] difference principle endorses this perspective
and requires that society compensate for disadvantages
resulting from socioeconomic status, religion, ethnicity,
or any other factor beyond individuals’ control or choice.
Apart from Rawls’ [10] theory of justice and the difference
principle, there is another argument that supports
compensation for disadvantages resulting from bad luck
in the social lottery. This argument emphasizes the core
position of individual responsibility and puts forth that if a
disadvantage is as a result of an individual’s own behavior,
then it is not society’s responsibility to compensate for that
disadvantage [14]. However, if the disadvantages happen
to find an individual due to bad luck in the natural lottery,
then justice requires that society eliminate these disadvantages.
The natural lottery is beyond individuals’ control.
No one can choose their nationality, religion, ethnicity, or
socio-cultural environment [15]. The individual responsibility
argument is endorsed by the sheer bad luck perspective,
which implies that equal opportunity can be achieved
if the inequalities in talents and abilities as well as means
resulting from an initial social position are compensated
for. This perspective suggests establishing a compensation
mechanism to allocate resources in such a system in order
to provide fair distribution, and that fairness is realized if
every individual has access to resources that they require
in order to achieve equal opportunity [12].
Can We Use the Same Arguments to Justify the
Plausibility of Compensating for Natural Primary
Goods? It is apparent that the results of the natural lottery
are not limited to social disadvantages. Primary natural
goods such as intelligence, imagination, vigor, and health
are also gifted or deprived by the same lottery. Unlike social
primary goods, the inequalities between human beings
originating from natural factors have been excluded from
the domain of justice since Plato. There can be two reasons
for this. First, theories of justice aim to establish a just
system through structures and terms of implementation for
institutions. Social institutions and social structures have
no impact on the inequalities emerging from the natural
lottery of primary natural goods; if the social system and
institutions have no effect on the occurrence of a disadvantage,
it is beyond the scope of justice.
The second reason relies on the fact that knowledge
about how the natural lottery occurs and how the results of
the natural lottery lead to disadvantageous consequences
have been lacking until recently. The multifactorial unknown
etiology of the disadvantages emerging from the
natural lottery leaves us ignorant about how to annihilate
or correct them. Therefore, the lack of knowledge about
the causes and means to rectify these disadvantages could
have contributed to excluding the disadvantages arising
from the natural lottery from the domain of justice.
Contemporary science has changed this ignorance to
some extent and has proven that some personal qualifications
and diseases do result from genetic factors. A low
intelligence quotient, defects in phenotype, or even some social behavior disorders have been proven to emerge
from our genes. As a result, knowledge of the inner workings
of the natural lottery are no longer a mystery. Thanks
to genetics, our knowledge of diseases with genetic origins
has increased. For example, we now know that Down
syndrome is a result of trisomy 21 and that LPLD is
a rare autosomal recessive inherited disease caused by
mutations in the lipoprotein lipase gene. Moreover, the
increase in our genetic knowledge has provided medical
means to intervene in the natural lottery and to avoid
or undo the results of sheer bad luck by means of gene
therapies.
Although today, gene therapy is still considered experimental,
the prospects of replacing a mutated gene that
causes disease with a healthy one, deactivating a mutated
gene, introducing a new gene into the body to combat a
disease, or scanning intrauterine fetuses for gene disorders,
are more promising than ever. This new idea of knowing
how the natural lottery works and having the means to
remove the disadvantages of a genetic origin urges us to
reconsider the on-going assumption of excluding natural
factors from the domain of justice, and to provide a new
status for curable or preventable disadvantages stemming
from the natural lottery. Referring to the discussion in the
previous section, this new status can be defended on two
grounds. The first is fair equal opportunity. As it has been
established that equal opportunity is considered crucial
for justice [12] and that some form of compensation for
disadvantages hindering individuals from having equal
opportunity is required, and since it is indubitable that
genetic diseases cause disadvantages for individuals that
prohibit them from enjoying equal opportunities, justice
requires compensation for such disadvantages. It is plausible
to think that this argument was not developed when
equal opportunity was first discussed because it was not
possible to intervene in the consequences of the natural
lottery. The only option was to accept them as bad luck and
to focus on the disadvantages that we are able to mitigate
or compensate for.
The second ground is individual responsibility. Since
genetic disease is a consequence of bad luck in the natural
lottery and an individual has no inducements to have a genetic
disorder, and as justice requires that society eliminate
the disadvantages emerging from bad luck in the natural
lottery in which the individual has no inducement, then
it follows that genetic diseases should be considered in
the purview of justice. Interventions to prevent or cure
genetic diseases should be considered as part of the compensation
mechanism to allocate resources to provide fair
distribution, and that fairness is only realized once every
individual has access to the resources that they require to
achieve equal opportunities [12].
The Limits of Compensation: Genetic Diseases
and Personal Variations. The disadvantages of the natural
lottery are not limited to genetic diseases. Being shorter
than the average person, extreme shyness, an introverted
personality, or even having ears that stick out, are consequences
of the natural lottery. It can be argued that these
features cause disadvantages when applying for positions
or posts. A person who is extremely shy and has difficulty
speaking in front of a jury or significantly short when
compared to other candidates, would face difficulties in
enjoying equal opportunity. In such cases, it is not only
genetic diseases but also several negative features or personality
traits that have the potential to endanger equal
opportunity. Additionally, individuals with these features
or personal traits do not have any responsibility in having
them. Therefore, it is plausible to say that the two theoretical
grounds, equal opportunity and individual responsibility,
apply to all disadvantages emerging from the natural
lottery and that they should also be compensated for in
a just system. If we are justifying the right to access to
health services, gene therapy in our case, to ameliorate the
genetic disadvantages of an individual so that they may
enjoy a normal opportunity range and fair equal opportunity,
then we should also agree to provide these services
to, for example, shy people.
Although the broad implications of our discussion
seem plausible on theoretical grounds, there are strong
counter arguments against them. First, variations in phenotype
and personality together with talents and abilities are
among the features that make a person unique. These variations
should be considered normal and necessary for the
genetic plurality of the human species. This variation and
uniqueness might restrain an individual’s opportunities to
be appointed to some positions or posts, but may enhance
their chances in others. For example, an introverted person
might be disadvantaged if seeking a career in the media
sector, but may enjoy a good career in writing. Another
example would be that your physical appearance might
not be a huge disadvantage if your brain functions like
that of Stephen Hawking.
The second argument is that the disadvantageous nature
of these features or traits is not absolute or universal.
Their value in terms of being advantageous varies over
time and according to different cultures. For example, a
big body with strong muscles was certainly an advantage
in the Stone Age, but contributes little to a career as a nuclear
scientist. Likewise, being humble and taciturn might
be considered a sign of self-containment and maturity in
Eastern cultures, but people with these features might be
considered too obscure and insignificant in Western societies.
On the other hand, genetic diseases form health conditions
with severe morbidity and mortality risks regardless of the time and culture they occur in. There is no occasion
that ADA or LPLD would be considered an advantage.
Moreover, the genetic disorder behind genetic diseases
cannot be considered as variations in normal human genomes,
as their consequences are incompatible with life.
The third argument comes from science. The direct
causal relationship between genetics and the presence of
these features or traits is not defined. On the contrary,
our current knowledge suggests that several phenotypic
variations as well as personality traits result from multiple
factors. Hereditary factors together with environment, nutrition,
education, and cultural and religious customs play
a crucial role in their presence. Therefore, it is not possible
to predetermine their occurrence in the future. Moreover,
the means to “cure” them is limited and has no resemblance
to the certainty and scientificity of gene therapy. Thus, it is
not plausible to form a comparison between them.
Conclusions. The vast improvements in genetics
have enabled us to intervene in genetic diseases, something
that was considered to be a result of bad luck in the natural
lottery, and was excluded from the domain of justice in
this respect. Contemporary theories on justice require that
society compensate for the disadvantages of individuals
so that they may enjoy equal opportunities, which is considered
crucial for a fair and just system. The high rates of
mortality and morbidity of genetic diseases create concrete
disadvantages for these patients to enjoy fair equal opportunities.
From this perspective, the fair equal opportunity
principle requires that society provide available genetic
treatment to those in need in order to compensate for the
disadvantages that result from genetic disease. This view
brings a new context to the domain of justice by including
disadvantages resulting from bad luck in the natural
lottery. However, genetic diseases are not the sole element
in the set of disadvantageous results of the natural lottery.
Personal traits and features that might have some negative
impact on enjoying fair equal opportunity are also included
in this set. Thus, we need to draw a line in order to avoid a
slippery slope and to hold society responsible for correcting
any variation in human beings that has the potential of
hindering the realization of a person’s life plans. The high
rates of mortality and morbidity of genetic diseases create
an absolute and universal disadvantage for any individual
who is unlucky enough to have them, whereas the disadvantageous
qualification of personal traits or features are
relative to certain times and cultures. Moreover, personal
traits and features are considered as normal variations in
the human species with no requirement for a cure, whereas
genetic diseases are disorders that are not compatible with
life unless cured. Thus, it is plausible to say that society’s
duty to compensate for genetic diseases can be justified due
to the fair equal opportunity principle; however, this does
not apply to the compensation of any variation emerging
from the natural lottery.
Declaration of Interest. The authors report no conflicts
of interest. The authors alone are responsible for the
content and writing of this article.
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