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0:00
My name is Kasem Akhras.
I am the Co-Founder and CEO
of a consulting company
called VantEdge Access LLC.
We are in Oak Brook,
Illinois, just outside Chicago.
Today, I will be talking
about Gene Therapies:
the Economics,
Access Barriers, and
Commercial Models.
0:20
The topics we will cover today
are outlined on this slide.
We're going to go through
a brief introduction
to gene therapies and rare
diseases to highlight
a few things related to the
humanistic economic impact of
rare diseases and the value of
gene therapies from a payer
and societal perspective.
We're going to talk about
pricing and
affordability challenges
facing gene therapies.
We're going to focus quite a bit
of details on
providing insights,
learning practical aspects of
developing gene therapies from
a development and
commercialization perspective,
and concluding with some
summary and remarks.
1:01
As an introduction,
gene therapies
and rare diseases:
by definition,
gene therapy is applicable
or more relevant to
rare diseases given
that the majority of
rare diseases are
caused by single-gene mutations,
which we call monogenic.
Most of the gene
therapies that are
in development now or
approved are monogenic.
This makes gene therapy ideal
for tackling rare diseases.
Unfortunately, children are most
impacted by rare diseases.
While we're talking
about rare disease,
it's almost a misnomer because
there are more than 7,000
rare diseases impacting
about 25-30 million
Americans and,
globally, well over 300
million people worldwide.
It's a big impact even though
we call it rare disease.
It's also unfortunate
that due to
the limited number of approved
therapies for rare diseases,
the true clinical, economic,
and humanistic burden
is not well understood.
Not well understood by people
who develop or work on
the development of gene
therapy for rare diseases,
as well as by stakeholders
in the healthcare ecosystem.
