Climate change is a long-term change in the average weather patterns that have come

todefine Earth’s local, regional, and global climates. The term interchanged with global
warming but they mean different. Global warming refers to “the long-term heating of the
Earth’s climate system observed since the pre-industrial period (between 1850 and 1900)
due to human activities, primarily fossil fuel burning.

The Philippines is highly vulnerable to climate change. In fact, it ranked first as the country
most hit by climate change in 2013 due to the damages brought by Typhoon
Yolanda (Haiyan). In the recent Global Climate Risk Index 2020, it appeared second to
Japan in the list of the top ten country's most hit by climate change. The country's location
and risk factors such as the rise in sea levels, extreme rainfall events, extreme heating
events, increased ocean temperatures, and a disturbed water budget make it vulnerable to
climate change. Examine the Philippine Exposure Map on Climate Change and see the risk
factors to which each region in the country predispose to.

How do we know that the climate of the world is changing?

“Warming of the climate system is unequivocal, and since the 1950s, many of the observed
changes are unprecedented from decades to millennia.”

- Intergovernmental Panel for Climate Change

The National Aeronautics and Space Administration (NASA) gathered pieces of evidence on
the occurrence of climate change such as:

1. Global temperature rise

Scientists from various institutions around the world agree to the fact that global surface
temperature has increased since 1880 (Fig.1).

Fig. 1. Temperature data showing rapid warming in the past few decades, the latest data
going up to 2019. According to NASA data, 2016 was the warmest year since 1880,
continuing a long-term trend of rising global temperatures. The 10 warmest years in the
140-year record all have occurred since 2005, with the six warmest years being the six most
recent years.

The global average surface temperature increased by about 0.6 to 0.9 oC (1.1 to
1.6oF) between 1906 to 2005. Most of the warming took place in the past 35 years, with the
six warmest years on record, 2015 to 2019. June 2019 was the hottest June ever recorded
in 140 years according to U.S National Oceanic and Atmospheric Administration (NOAA).
The average global temperature in June 2019 was 1.71 oF higher than the 20th century
average of 59.9 oF. The year 2016 sets the world record of being the hottest year for the
entire planet. The average global temperatures across land and ocean surface areas in 2016
reached the highest 0.94oC (1.69oF) above the 20th century average of 13.9oC (57.0oF).

2. Warming oceans

The oceans are so vast and it can absorb a large amount of heat without large
changes in its temperature. That protects life in it. According to the State of the Climate
2018 report, however, “averaged over Earth's surface, the 1993–2018 heat-gain rates were
0.36 to 0.4 (±0.06) watts per square meter for depths from 0–700 meters and 0.14 (±0.0) to
0.32 (±0.03) for depths of 700–2,000 meters. For depths between 2000–6000 m, the
estimated increase was 0.07 (±0.04) watts per square meter for the period between
September 1992 to May 2011. Summing the three layers (with their slightly different time
periods), the full-depth ocean heat gain rate ranges from 0.57 to 0.81 watts per square meter
roughly applicable to 1993–2018."

Do you know how ocean heat is measured? If you are interested you may
visit https://www.climate.gov/news-features/understanderiod.ing-climate/climate-change-oce
an-heat-content

3. Shrinking ice sheets

The Greenland and Antarctic ice sheets have decreased in mass as shown in ice mass
measurement by NASA’s GRACE satellite since 2002. Data from NASA's Gravity Recovery
and Climate Experiment show Greenland lost an average of 286 billion tons of ice per year
between 1993 and 2016, while Antarctica lost about 127 billion tons of ice per year during
the same time period. The rate of Antarctica ice mass loss has tripled in the last decade.

Interested to know more about the shrinking ice sheets? Here is an 11-minute
read The Big Thaw.

4. Glacial retreat

Glaciers are large masses of ice that flows slowly downhill. Most of the world's
glacial mountains are retreating since the 1850s.

Over the last 25 years, for instance, the Gangotri Glacier in Himalaya has retreated more
than 850 meters, with a recession of 76 meters from 1996 to 1999 alone.
5. Decreased snow cover

Land snow cover in the Northern Hemisphere averaged 24.8 million square kilometers
in 2019. This measurement is 0.3 million square kilometers less than the 50-year average.
Evidence of diminishing snow cover was recorded by satellite images and revealed that in
the last five decades, on average, spring snow is disappearing earlier in the year than in the
past.

6. Sea level rise

The latest report from the Intergovernmental Panel on Climate Change concluded that sea
level rose around 0.07 inches (1.7 millimeters) per year between 1901 and 2010 and that
between 1993 and 2010, the rate was very likely higher, more like 0.13 inches (3.2 mm) per
year.

7. Extreme events

More intense typhoons and extreme flooding in some areas of the world while other
areas experience wildfires, extended droughts, and heatwaves. Some of the extreme events
are Super Typhoon Haiyan or (Yolanda in the Philippines) in 2013, drought in Africa in
2019, the recent Arizona bush fires (June 17, 2020). The occurrence of these extreme
events was studied and mapped. Explore the map.

12.4 Causes of Climate Change

Human influence on the climate system is clear, and recent anthropogenic emissions of
greenhouse gases are the highest in history.

- Intergovernmental Panel for Climate Change

IPCC AR5 reiterated that human activities, particularly the burning of fossil fuels, are the
major cause of climate change. This activity poured a significant amount of greenhouse
gases (GHG) into the atmosphere.

1. Greenhouse gases and human activity

 Greenhouse gases are naturally present in the atmosphere, trapping and absorbing the
heat, creating a greenhouse effect protecting us from extreme heat and cold. Greenhouse
gasses include water vapor (H2O); carbon dioxide (CO2); nitrous oxide (N2O), methane
(CH4), and chlorofluorocarbons (CFC).

Water vapor is the most abundant greenhouse gas. The amount of water vapor in the
atmosphere increases as the Earth's atmosphere warms. This creates the possibility of
clouds and precipitation.

Carbon dioxide (CO2) is present in small amounts in the atmosphere. It comes from natural
sources such as decomposition, respiration, weathering of rocks, and volcano eruptions. It is
also added to the atmosphere through human activities such as the burning of fossil fuels
and deforestation.

Nitrous oxide is a greenhouse gas introduced into the atmosphere through the use
of fertilizers, combustion in motor vehicles, and production of nitric acid and
charcoal. Burning of the forest, agricultural, and domestic wastes also contributes nitrous
oxide to the atmosphere.

Methane is present in a small amount in the atmosphere. It is produced through natural

sources such as the decomposition of wastes, wetlands, and rice paddies. Human activities
such as biomass burning, rice cultivation, manure management in livestock, exploration, and
transport of fossil fuels.

Chlorofluorocarbons (CFCs) are synthetic compounds from industrial sources used in a

number of applications. Its production, however, now largely regulated due to its
known ability to contribute to the destruction of the ozone layer.

Increased demand for economic growth and development coupled with increasing
population growth generated a greater need for energy and food production. These human
activities resulted in more greenhouse gas (GHG) emissions that made the earth hotter than
ever before. The burning of fossil fuel such as coal, oil, and gas for electricity, heat, and
transportation is the primary source of human-generated emissions.

Data show that the primary sources of global GHG emissions are electricity and heat (31%),
agriculture (11%), transportation (15%), forestry (6%), and manufacturing (12%). Energy
production of all types accounts for 72 % of all emissions. United States, Russia, and Japan
top the per capita greenhouse gas emissions and China toppled others of the seven major
global emitters which are responsible for nearly two-thirds of the global emissions.

2. Natural causes
 There are natural factors that affect the climate system of the earth: solar
variation, volcanic eruption, ocean current, and internal variability such as La Niña and La
Niño.

Impacts of Climate Change

Recent climate change have had widespread impacts on human and natural systems.

- Intergovernmental Panel for

Climate Change

Global climate will continue to change in the coming century. The magnitude of change
depends primarily on the amount of GHGs emitted. If this continues, then the following are
the projected effects of climate change:

The temperature will continue to riseThe growing season will lengthenChanges in

precipitation patternsMore droughts and heatwavesCyclone (typhoon and hurricanes) will
become strongerSea level will rise by 1-8 feet in 2100The Arctic likely to become
ice-freeCoral bleachingDegradation of ecosystems

These effects will bring about the greatest threats to biodiversity, human health, food
security, livelihood, water supply, and economy, among others. If global temperature
continues at a current rate, it is likely to reach 1.5oC in 2030 to 2050. The Paris
Agreement in 2015 aims to keep the overall increase in global temperatures below 2
degrees Celsius, with the hope of limiting it to 1.5 degrees Celsius. Scientists believe that
these are the largest increases in global temperature that we could experience without
causing catastrophic changes to the Earth’s climate.

This target is possible but will require enormous emission reductions and far-reaching
changes in all aspects of society. Various sectors can decide on how to reduce consumption
and emissions. Suggestions include the eliminate use of fertilizer, restrict meat diet and
advocate vegetarian diet, invest in renewable resources, give emphasis on forest
management and reforestation, restore soil and water protection, choose green roofing,
green architecture, green technology, innovate electric and fuel cell transportation, efficient,
ubiquitous public transportation.

Despite global efforts the world does not seem to progress in terms of climate change
efforts. A number of political leaders in progressive countries hesitate to comply with the
Paris Agreement. The current pandemic even made global efforts toward the attainment of
United Nations Sustainable Goal #13 Climate Change Action more difficult. But do not lose
hope. Persevere in what you think is correct. The magnitude of your contribution does not
matter. If we do this collectively, then the impact matters!

In the Philippines, Climate Change Commission hurdled the task of being the lead agency to
ensure a “resilient and climate-smart Philippines”. It is currently pursuing significantly
increased action towards disaster risk reduction and climate change adaptation.

How can we help?

On a personal level, one can engage in the following activities that reduce carbon footprint:
compost organic garbage, avoid plastics, participate in sharing economy (share a ride, share
tools for brief usage, share your unused clothes and things instead of throwing them), shrink
your digital footprint (download rather stream videos and music playlist). Further, the
Climate Change Commission encourages us to practice Low Emission Development
Strategies (LEDS). Watch the video.

- There is a global consensus on the occurrence of climate change as evidenced by

global temperature increase, warming oceans, melting ice sheets, decreasing snow
cover, sea level rise, glacial retreat, extreme events, and ocean acidification.
- The major causes of climate change are greenhouse gases, the production of which
was hastened by human activity.Climate change has widespread impacts on human
and natural systems.
- There are many ways by which we can reduce our carbon footprint and contribute to
climate change mitigation

Roboethics

In the 1940s, American writer Isaac Asimov developed the Three Laws of Robotics arguing
that intelligent robots should be programmed in a way that when facing conflict they should
remit and obey the following three laws:

A robot may not injure a human being, or, through inaction, allow a human being to come to
harm

A robot must obey the orders given it by human beings except where such orders would
conflict with the First Law

A robot must protect its own existence as long as such protection does not conflict with the
First or Second Law
Fast-forward almost 80 years into the present, today, Asimov's Three Laws of Robotics
represent more problems and conflict to roboticists than they solve.

Roboticists, philosophers, and engineers are seeing an ongoing debate on machine ethics.
Machine ethics, or roboethics, is a practical proposal on how to simultaneously engineer and
provide ethical sanctions for robots.

- The onward progress of science and technology is a movement towards the good
life.
- Technology empowers us to use our rights and provide us opportunity to widen our
knowledge beyond our immediate surrounding.
- As we contemplate on the issues on science and technology, we need to have a
constant practice of the good.

Module 8 8.1 The Information Society

The invention of computers and the internet ushered the global community to evolve into the
current society where usage, creation, and distribution of information, communication, and
knowledge drive global functions. These inventions facilitated the transformation of the
global community to an “information society”.

Key Points:

- In an information society (a) information is used as a resource, (b) it is possible to

identify greater use of information among the general public, and (c) information
sector is developed within the economy.
- The global community has transformed into an information society, has enjoyed the
benefits of such transformation but has to deal with the economic, social, and ethical
issues associated with it.
- We have numerous means to access information in our society, like radio, television,
smartphone and the sophisticated one the social media.
- Social media is a complex interactions of people in which they can create, share and
exchange information and ideas in a virtual communities.
- Social media has numerous forms like Facebook, Instagram, twitters, and
YouTube.Social media has a positive and negative sides to people.

What is Biodiversity?

The food you eat, the water you drink, and the air you breathe all depend on biodiversity.
Biodiversity is short for "biological diversity" which was first coined by Walter Rosen when
organizing the National Forum on Biodiversity. The proceedings were later published as part
of a book called Biodiversity by E.O. Wilson in 1988 that cemented the term in both
scientific communications and public awareness (Neuman, 2009).

Biodiversity is the measure of variety and variability of life (Faith, 2007).

Biodiversity is comprised of several levels: genetic diversity, species diversity, and

ecosystem diversity (Australian Museum, 2020).

Genetic diversity is the variety of genes present among individuals of the same species
(Australian Museum, 2020). Genes are made up of DNA and are the building blocks that
determine how an organism will develop (eg. variation of eye color) and what traits and
abilities will be (eg. ability to metabolize).

The amount of genetic diversity is important because it represents the raw materials for
evolution and adaptation. It serves as a way for the population to adapt to changing
environments (Lumen Learning, n.d.).

Less diversity leads to uniformity which would lead to less adaptability to a changing
environment. The offspring can be exposed to a handful of environmental factors which
could lead to furthering biodiversity or its stagnation depending on which factor it is exposed
(California Academy of Sciences, 2014).

Genetic diversity often increases with environmental variability because many genes are in
the population at any given time, however, inbreeding, genetic drift (founder effect and
bottleneck effect), restricted gene flow (eg. by physical barriers), and small population size
all contribute to a reduction in genetic diversity. Fragmented and threatened populations are
typically exposed to these conditions, which is likely to increase their risk of extinction
(Furlan et al., 2012).

Species diversity is the variety of species within a habitat or a region (Australian Museum,
2020). A species is a group of organisms that is able to interbreed freely under natural
conditions to produce viable offspring. Each species can be considered to have a particular
"role" in the ecosystem thus, any addition or subtraction of single species may have
consequences for the system as a whole.

Species diversity is measured by species richness and species evenness (Launchbaugh,

2011) The two different communities can have the same species richness, but a different
relative abundance (evenness). A community with an even species abundance is more
diverse (Figure A) than one in which one or two species are abundant and the remainder are
rare (Figure B).

Worldwide there are more than 1.7 million species were identified, but scientists believe that
it is estimated to reach up to 14 million. Some species importance can be out of line with
their members such as the Keystone species where their absence can cause an ecosystem
to be dramatically different or cease to exist altogether.

Ecosystem diversity is the variety of ecosystems in a given space. The basic principle of
biodiversity applies here as well, but the scope is much larger such as with the community of
organisms and their physical environment interacting together. It is at this level that
the interaction and links among species and the consequences of those are evident
(Australian Museum, 2020).

Why is Biodiversity important?

We are all intertwined in a massive system called Earth, and each element supports and
enables all of us to thrive. For the planet to stay healthy, nature needs to be stable and able
to cope with change which requires the interaction of a large variety of animals and plants.

If we allow the Earth to flourish, we can benefit from the resources it can produce for the
food we eat, the water we drink, and the medicine we rely on. The benefits of what
biodiversity brings are called ecosystem services which are commonly divided into four
categories: provisioning, regulating, cultural, and supporting services (National Wildlife
Federation, n.d.).

Human health ultimately depends upon ecosystem products and services that are
required for good human health and productive livelihoods such as the use of medicinal
products and biomedical research that relies on plants, animals, and microbes to understand
human physiology and to understand and treat human diseases (World Health Organization,
2015).

Biodiversity loss can have significant direct human health impacts if ecosystem services
are no longer adequate to meet social needs. Indirectly, changes in ecosystem services
affect livelihoods, income, local migration, and sometimes political conflict. (Watch the videos
below)

Interaction of organisms through the food web

Types of ecosystem

Unlike species diversity, ecosystem diversity is very difficult to examine and measure
because there is no globally agreed classification of ecosystems, defining and demarcating
ecosystems can be problematic, and species contain within that ecosystem vary over time.
Regions contain a great variety of ecosystems are rich in biodiversity but individual
ecosystems contain endemic species also make a significant contribution to global
biodiversity. Check on the list of endemic and endangered plants and animals in the
Philippines.

Philippine Policies in Relation to GMOs

1990 – Executive Order 430 (EO 430): -- Created the National Committee on Biosafety of
the Philippines (NCBP)

1991 – First Edition of the Philippine Biosafety Guidelines (PBG) - with a revised edition
dated 2014

2002 – Department of Agriculture- Administrative Order 8 (DAO 8) -- appoints its Bureau of

Plant Industry (DA-BPI) to regulate field trials and propagation and commercial release of
GMOs

2006 – Executive Order 514 (EO 514): National Biosafety Framework

2010 – Republic Act 10068 or the Organic Agriculture Act

2013 – Republic Act 10611 or the Food Safety Act Relevant Laws

The advent of technology that can increase and optimize food production such
as biotechnology can support our increasing demand for resources. The question arises
particularly on Genetically Modified Organisms (GMOs) whether it can be detrimental to
human health (production of allergens) and even with the environment (ie. genetic
pollution, resistant pests/diseases, upset healthy ecological function).

In the Philippines, the introduction of Genetically Modified Organisms (GMOs) started in the
late 1990s. Currently, there are 120 GMO crops approved for food, feed, or for processing
including Alfalfa, canola, cotton, corn, potato, rice, soybean, and sugar beet

Human-induced Threats

As our numbers increase, resource utilization also increases, and this poses a threat not
only to the quality of life in terms of social and economic status, but on biodiversity as well.

Human-induced threats are the main drivers of biodiversity loss which can either
be local or global factors. Local factors include land-use changes, pollution, resource
exploitation, and the introduction of exploited species, all of which can also overlap. Global
factors such as climate change will be discussed on the succeeding modules.

Humans are dependent upon healthy biodiversity, for good health and welfare.Biodiversity is
threatened by human-induced factors.Biotechnology can support our increasing demand for
resources, however, can also be detrimental to biodiversity with the utilization of Genetically
Modified Organisms (GMOs)Individuals must be aware of the implications of GMOs to
provide a sustainable strategy for food security and promoting biodiversity conservation.

These viruses are engineered such that they replicate only (or predominantly) in cancer cells
causing the cancer cells to lyse. The released viruses during the cell lyses will now infect
other cancerous cells but not normal cells. Thus this types of viruses are known as oncolytic.

How are viruses used in gene therapy?

Viruses are used in gene therapy as gene delivery vectors and as oncolytic viruses:

Viruses as gene delivery vectors

In order to deliver the genes to the cell, only genetically engineered vectors such as viruses
are used in gene therapy. These newly engineered virus protects the new gene it carries
from degrading enzymes in the blood. The virus being an obligate intracellular organism
forces the cell to take up the new gene, and goes into the nucleus. Once in the nucleus, or,
for some, once integrated into the chromosome, the new gene starts to perform its function
to synthesize a new improved or functional protein. Furthermore, these viral vectors are
designed such that their ability to replicate uncontrollably inside the cells are removed.
However there must a room for a new gene.

Can viral vectors make you sick?

No, because these viruses used in Gene Therapy are designed to reduce the risk of adverse
effects. These viral vectors are tested in animals and in human trials to make sure that
human cells are not harmed. However, like all treatments or therapy, it has its risks.
Therefore anyone availing of this therapy must discuss with their physician and other health
advocates the pros and cons of Gene Therapy.

Risks Associated with Gene Therapy

"Risks of any medical treatment depend on the exact composition of the therapeutic agent
and its route of administration. Different types of administration, whether intravenous,
intradermal or surgical, have inherent risks.

Risks include the outcome that gene therapy or cell therapy will not be as effective as
expected, possibly prolonging or worsening symptoms, or complicating the condition with
adverse effects of the therapy. The expression of the genetic material or the survival of the
stem cells may be inadequate and/or may be too short-lived to fully heal or improve the
disease. Their administration may induce a strong immune response to the protein in the
case of replacing proteins from genetic diseases. This immune response may become
uncontrolled and lead to normal proteins or cells being attacked, as in autoimmune diseases.
On the other hand, in the case of cancer or viral/fungal/bacterial infections, there may be an
insufficient immune response, or the targeted cell or microorganism may develop resistance
to the therapy. With the current generation of vectors in clinical trials, there is no way to “turn
off” gene expression, if it seems to be producing unwanted effects.

In the case of retroviral or lentiviral vectors, integration of the genetic material into the
patients’ DNA may occur next to a gene involved in cell growth regulation and the insertion
may induce a tumor over time by the process called insertional mutagenesis.

High doses of some viruses can be toxic to some individuals or specific tissues, especially if
the individuals are immune compromised.

Gene therapy evaluation is generally carried out after birth. There is little data on what
effects this therapeutic approach might have on embryos, and so pregnant women are
usually excluded from clinical trials.

Risks of cell therapy also include the loss of tight control over cell division in the stem cells.
Theoretically, the transplanted stem cells may gain a growth advantage and progress to a
type of cancer or teratomas. Since each therapy has potential risks, patients are strongly
encouraged to ask questions of their investigators and clinicians until they fully understand
the risks."

Characteristics of diseases which can be treated with gene therapy

"Characteristics of diseases amenable to gene therapy and cell therapy include those for
which there is no effective treatment, those with a known cause (such as a defective gene),
those that have failed to improve or have become resistant to conventional therapy, and/or
cases where current therapy involves long term administration of an expensive therapeutic
agent or an invasive procedure.

Gene therapy and cell therapy have the potential for high therapeutic gain for a broad range
of diseases. An example would be those caused by a mutation in a single gene where an
accessible tissue is available, such as bone marrow, and with the genetically modified cell
ideally having a survival advantage. However, patients with similar symptoms may have
mutations in different genes involved in the same biological process. For example, patients
with hemophilia A have a mutation in blood clotting Factor VIII whereas patients with
hemophilia B have a mutation in Factor IX. It is important to know which gene is mutated in a
particular patient, as well as whether they produce an inactive protein which can help to
avoid immune rejection of the normal protein.
Gene therapy and cell therapy also offer a promising alternative or adjunct treatment for
symptoms of many acquired diseases, such as cancer, rheumatoid arthritis, diabetes,
Parkinson’s disease, Alzheimer’s disease, etc. Cancer is the most common disease in gene
therapy clinical trials. Cancer gene therapy focuses on eliminating the cancer cells, blocking
tumor vascularization and boosting the immune response to tumor antigens. Many gene and
cell therapy approaches are being explored for the treatment of a variety of acquired
diseases. More details are listed in the ASGCT.org disease information page."

What is a fair price for gene therapy?

author: Kevin Curran PhD

updated: 1-15-2019

It has finally happened, we now have a gene therapy approved in the US with a price-tag
over 1 million…actually, well over 1 million.

Zolgensma, a gene therapy for spinal muscular atrophy, was approved by the FDA in 2019.
Novartis has priced this therapy at $2.1 million per patient. The science behind this new drug
is ground-breaking. The value it brings to patients is also phenomenal. The cost of
manufacturing this viral vector based therapy is not cheap. But, the question remains…does
$2.1 M represent a fair price? Maybe…maybe not. Many folks in the healthcare industry are
trying to make sense of these numbers.

First of all, when discussing gene therapy, people often use the word treatment, however if
these therapies eliminate the medical condition, then by definition – we are talking about
a cure.

If gene therapy delivers a cure…how much is that cure worth?

This is a challenging question that yields no quick answers. The healthcare industry doesn’t
have a lot of experience delivering or pricing cures. Most drugs on the market offer
treatments, not cures.

Organ transplants provide an interesting comparison as they’re considered a curative

procedure. So… what’s the bill for a new organ? In the U.S. market, a heart transplant
costs about 1.38 million, a liver is 812K and a new kidney runs 414K. Organ transplants
are not cheap.

Are organ transplant prices indicative of the cost of future gene therapies?

Below, I’ll attempt to tackle this question by analyzing three different drug pricing paradigms.
Before we get into that, let’s take a few items into consideration.

Much of the enormous price for gene therapies will be paid for by either private insurance
companies or Medicare. This means the general population (all of us) will ultimately share
the tab via federal taxes or through higher premium rates/deductibles on our insurance
policies. Therefore, we are all stakeholders in this decision.
That said, a drug company is free to price their gene therapy at any number they desire.
Getting a drug approved by the FDA is incredibly difficult, although once the FDA approves a
drug, there’s no government agency that regulates the price.

Drug companies often price their drug based on the dollar amount they think they can get
from the market. This leads to supply and demand economics.

The problem is…supply and demand economics is a blunt and ugly tool to use when
considering life and death situations.

In this regard, healthcare is fundamentally different than other industries.

A car company may price a Mercedes Benz at 50K … and that’s fine. If you don’t care to
spend 50K on a car, then walk around the corner and test drive a Honda Civic. In contrast, if
your child is born with a life-threatening genetic disorder, then you (and your insurance
company) will feel compelled/obligated to pay any price to save that human life.

The element of consumer choice is severely reduced in healthcare decisions. If a drug

company is free to charge exorbitant costs for a life-saving treatment, the patient will
justifiably feel that their life is held at ransom. Nobody wants that. Nobody wants a drug’s
price tag to take on the tone of a ransom note.

Yet, at the same time…drug manufacturers must be incentivized.

Breakthroughs in science come at a high cost. Pushing a drug through the FDA pipeline is
astronomically expensive. One report estimates an average cost of 2.5 billion to finance the
FDA approval of a single drug. Innovators deserve a reward if they develop a successful
gene therapy.

So, where does this leave us?

Is there an equitable process to use when determining the fair price for an effective gene
therapy?

Below, I examine this question from three different paradigms.

Determine the cost of not treating the genetic disorder. Price your gene therapy under that
number.Price your gene therapy as competitive to a preexisting treatment for the same
disease.Use a cost-effective ratio to set price based on the number of quality-adjusted life
years a therapy delivers.