UP FRONT
Pulling Back the Curtain: Peter Marks, MD, PhD
What strikes you as the
biggest differences among
the fields you’ve been
involved in?
One way to understand the differ-
ences between clinical medicine,
the pharmaceutical industry, and
government is thinking about
whom you’re helping in each role.
In clinical medicine, you have a
devotion to the individual patient
and your focus is on the care of
that individual.
In industry, you are developing
products that will hopefully help
groups of patients, as well as bring
value to the company because, ob-
viously, pharmaceutical companies
don’t just make drugs out of the
goodness of their hearts.
In government, you are work-
ing on behalf of the entire popu-
lation and do so in a variety of
ways. We are committed to serving
public health, from addressing
the needs of individuals in need
of access to investigational medi-
cines to working to prevent global
pandemics, and everything in
between.
What advice would you
give trainees or younger
hematologists who are
interested in a “dream job”
in the public sector?
First, I would recommend you
take the time to find excellent
mentors and explore all your
options.
Second – although saying this
might not make me too popular
with some of my colleagues at
the agency – I think it is helpful
to spend a few years caring for
patients, doing research, or work-
ing on other public health–related
projects. The experiences and per-
sonal development that you gain
there can be invaluable in subse-
quent work in the public sector
and beyond. That’s not to say that
you can’t go directly from a resi-
dency or fellowship to a job in the
public sector, but life experience
in patient care, clinical medicine,
or research serves one well, parti-
cularly if you want to advance
to a leadership or management
position.
Third, follow your heart. You
need to feel a strong commitment
to the advancement of public
health. Compensation in the
public sector is reasonable, but
money usually isn’t the primary
reason that people are drawn to
it. Seeing forward progress in
public health is really the greatest
reward that you can have in such
positions.
16
ASH Clinical News
“All the
different
positions
in my
career have
provided me
with a wealth
of experience
... [and]
prepared me
very well for
my current
position,
which I
view as my
personal
dream job.”
What has been the biggest
accomplishment in your
career?
I’ve been fortunate to work with
magnificent colleagues and to
contribute to many successes in
the different sectors I’ve worked in,
but as CBER director, I can think
of two accomplishments. Also, I’d
like to emphasize that these are
team successes, rather than my
personal successes.
One is an administrative
achievement of bringing together
groups of talented people to help
execute our mission at CBER.
Having a deep pool of talented
individuals allows us to conduct
the scientific and regulatory work
that we need to do to ensure the
safety and effectiveness of blood
products, vaccines, and cellular
and gene therapies.
The other is related to policy
issues: During my time at the
CBER, we have released a fair
number of guidance documents
that have helped people develop
products and deal with various
outbreaks, such as Zika or Ebola.
But, of those, the one that stands
out is establishing the Regenera-
tive Medicine Advanced Therapy
Designation for cell therapies or
therapeutic tissue-engineering
products, or human cell and tissue
products, as directed under the
21st Century Cures Act.
We worked as a team to roll
out that implementation effectively
and efficiently, and, within a few
weeks of the act’s passage, we had a
process in place for receiving ap-
plications and reviewing them in a
timely manner. That program has
been quite successful: In the just-
over two years of its existence, we
have received nearly 100 requests
for this designation.
What have been the biggest
changes in the work of CBER
since you started as deputy
director?
The introduction of cellular and
gene therapies is causing us to take
a renewed look at manufacturing
processes and clinical development
timelines. Certain challenges and
questions have come to light be-
cause we are dealing with smaller
patient populations and products
that are more difficult to manufac-
ture consistently. For example, how
will we adapt to products that aim
to address different mutations in a
gene using a specific gene-editing
approach?
On the clinical side of things,
we are at a place where we must
look for the most relevant end-
points for many uncommon or
rare diseases to understand what
matters most to patients. The clini-
cal trials of investigational agents
for rare diseases are conducted
with relatively small groups of in-
dividuals and within clinical areas
where we perhaps don’t have ex-
tensive natural history studies.
Biologic therapies also are rap-
idly evolving, and we need to learn
more about their optimal manu-
facturing and their critical quality
attributes to understand how to
evaluate them most efficiently.
That’s a real challenge: We want to
get these products out to patients
in need and we don’t want to re-
quire unnecessary information for
regulatory approval that overbur-
dens investigators and industry, yet
we still need enough information
to ensure that the products are safe
and effective.
Our responsibility is to ensure
that these novel products are held
to the same reasonable standards
for safety and efficacy to which
other medical products are held.
Just because something can be
treated by a cellular product
or a gene therapy rather than a
conventional drug doesn’t mean
that the cellular product or gene
therapy is the better treatment.
That can be difficult to under-
stand for people who are perhaps
overenthusiastic about these new
treatment modalities. At the end
of the day, we want to enable the
best treatment for patients – it
doesn’t matter what type of treat-
ment that is.
Does a change in
administration affect your
work at the FDA?
Some priorities may change with
a new presidential administra-
tion, but our priority at the FDA
remains the same: protecting and
promoting public health. When
there’s an administration change
we must brief a whole new group
of people, but we’ve been fortunate
that people on both sides of the
aisle have recognized the impor-
tance of our work. Administration
changes can affect different people
in different ways; my way of deal-
ing with it – and I think that many
of my colleagues have the same
approach – is to stay focused on
our public health mission.
With that in mind, what do
you think people should
be enthusiastic about?
What types of therapies or
approaches do you think
will be considered for
approval in the near future?
Research is advancing so rapidly
that what I say is all speculation,
but my sense is that, five years from
now, we’ll probably be approving
gene therapies for a wide variety
of genetic diseases. We also might
be evaluating novel approaches to
streamline gene therapy approvals,
like use of a single vector with a
range of different inserts tailored to
target different mutations causing a
particular condition – analogous to
a razor and razor blades.
Ten years from now, I suspect
that we will have an array of molec-
ularly targeted medicines and gene
therapies that will address both rare
and common diseases. And we also
may be approving new generations
of vaccines that provide broad pro-
tection against classes of viruses,
such as influenza.
If these predictions come true,
they will be game-changers for
population health. ●
February 2019