The document discusses orphan drugs and rare diseases in Latin America. It provides background on rare diseases, noting they are often chronic, life-threatening conditions without cures or treatments. It discusses how various nations have created incentives to encourage development of orphan drugs for rare diseases. The document argues the time is now for Latin America to work with stakeholders like patient groups, governments, and industry to accelerate adoption of orphan drugs in the region through initiatives. The value of orphan drugs is seen as higher than their costs.

1. La#n
America,
when
and
how
to
enter
the
orphan
drugs
markets
Fernando
Ferrer,
MBA
Mul/na/onal
Partnerships
LLC

2. Disclaimer
The
views
and
opinions
expressed
in
this
session
are
those
of
the
individual
presenters
and
should
not
be
aAributed
to
their
employers
or
RAPS.
Fernando
Ferrer,
MBA
Mul/na/onal
Partnerships
LLC
2

3. Abstract
• Orphan
Drugs
are
medicaments
for
Rare
Diseases
and
low
incidence
Oncological
Diseases.
They
are
developed
by
the
life
science
industry
in
order
to
treat,
improve
the
health
condi/on
and
quality
of
life
of
individuals
and
families
affected
by
those
diseases.
• Star/ng
with
the
US
Orphan
Drug
Act
of
1983
various
na/ons
have,
created
incen/ves
and
regula/ons
to
encourage
companies
to
develop
and
offer
orphan
drugs.
• The
UN
and
Interna/onal
Organiza/ons
have
launched
universal
declara/ons
to
grant
people
the
right
to
health,
and
to
guide
ethics
in
healthcare.
Countries
across
the
globe
include
pa/ents’
right
in
their
cons/tu/ons,
as
well
as
other
norms
to
grant
universal
access
to
beAer
health.
• La/n
America
is
aligned
with
this
interna/onal
trend
and,
liAle
by
liAle,
people
gaining
access
to
new
diagnoses,
devices
and
medicines
to
treat
rare
diseases
and
cancer.
• Pa/ent
&
associa/ons,
health
care
prac//oners
and
providers,
governments,
industries
and
interna/onal
organiza/ons
must
work
together
in
a
number
of
ini/a/ves
across
La/n
America
to
accelerate
the
broad
and
fast
adop/on
of
orphan
drugs
within
the
region
Fernando
Ferrer,
MBA
Mul/na/onal
Partnerships
LLC
3

4. Content
Orphan
Drugs
and
Rare
Diseases
in
La1n
America
①
the
1me
is
now
②
market
access
is
a
right
③
the
value
is
much
higher
than
the
cost
④
ini1a1ves
Conclusion
Fernando
Ferrer,
MBA
Mul/na/onal
Partnerships
LLC
4

5. “First
they
came
for
the
Socialists,
and
I
did
not
speak
out—
Because
I
was
not
a
Socialist.
Then
they
came
for
the
Trade
Unionists,
and
I
did
not
speak
out—
Because
I
was
not
a
Trade
Unionist.
Then
they
came
for
the
Jews,
and
I
did
not
speak
out—
Because
I
was
not
a
Jew.
Then
they
came
for
me—
and
there
was
no
one
leY
to
speak
for
me.”
-­‐
Friedrich
Gustav
Emil
Mar/n
Niemöller,
Lutheran
pastor
14
January
1892,
Lippstadt,
German
Empire
–
6
March
1984,
Wiesbaden,
West
Germany
① the
1me
is
now
Fernando
Ferrer,
MBA
Mul/na/onal
Partnerships
LLC
5

6. Rare
Diseases
are
• OYen
chronic
and
life-­‐threatening
• ~
7,000
diseases.
Nearly
95%
have
no
cures
or
treatments
yet.
– 80%
of
rare
diseases
have
iden/fied
gene/c
origins
whilst
others
are
the
result
of
infec/ons
(bacterial
or
viral),
allergies
and
environmental
causes,
or
are
degenera/ve
and
prolifera/ve.
– 50%
of
rare
diseases
affect
children.
– 30%
of
children
with
rare
disease
will
not
live
to
see
their
5th
birthday
– Rare
diseases
are
responsible
for
35%
of
deaths
in
the
first
year
of
life
• No
single
country
or
geographical/economic
region
is
providing
diagnosis
and
treatment
for
all
these
diseases.
Fernando
Ferrer,
MBA
Mul/na/onal
Partnerships
LLC
6

7. FAQs
About
Rare
Diseases
Rare
diseases
became
known
as
orphan
diseases
because
drug
companies
were
not
interested
in
adop#ng
them
to
develop
treatments.
The
Orphan
Drug
Act
(ODA)
created
financial
incen/ves
to
encourage
companies
to
develop
new
drugs
for
rare
diseases.
Fernando
Ferrer,
MBA
Mul/na/onal
Partnerships
LLC
7

8. 0
250
500
Requested
Designa/on
Approved
…and
the
industry
responded!
516
FDA
Orphan
Drugs
1983
–
2015e
Vs.
Requested
in
average:
70%
received
Orphan
Designa/on
status,
and
12%
were
Approved
References are available upon request
Fernando
Ferrer,
MBA
Mul/na/onal
Partnerships
LLC
8

9. Rare
Diseases
should
not
be
mistaken
for
neglected
diseases
• Neglected
tropical
diseases
(NTDs)
– are
a
group
of
parasi/c
and
bacterial
infec/ous
diseases(*)
– are
a
diverse
group
of
communicable
diseases
that
prevail
in
tropical
and
subtropical
condi/ons
in
149
countries
and
affect
more
than
one
billion
people,
cos/ng
developing
economies
billions
of
dollars
every
year.
They
mainly
affect
popula/ons
living
in
poverty,
without
adequate
sanita#on
and
in
close
contact
with
infec/ous
vectors
and
domes/c
animals
and
livestock.
(**)
18
neglected
tropical
diseases
Sources:
(*)The
END
Fund.
(**)
World
Health
Organiza/on
(WHO)
Buruli
ulcer
Lympha/c
filariasis
Chagas
Mazza
disease
Mycetoma
Dengue
and
Chikungunya
Onchocerciasis
(river
blindness)
Dracunculiasis
(guinea-­‐worm
disease)
Rabies
Echinococcosis
Schistosomiasis
Foodborne
trematodiases
Soil-­‐transmiAed
helminthiases
Human
African
trypanosomiasis
(sleeping
sickness)
Taeniasis/Cys/cercosis
Leishmaniasis
Trachoma
Leprosy
(Hansen's
disease)
Yaws
(Endemic
treponematoses)
Fernando
Ferrer,
MBA
Mul/na/onal
Partnerships
LLC
9

10. Pa/ents
with
Rare
Diseases
in
La/n
America
Popula/on:
625
Million
• Rare
Disease
6%
–
8%
=
37
-­‐
50
million
SA,
415,
66%
CA&C,
87,
14%
NA,
123,
20%
446
-­‐
600
million
people
with
a
Rare
Disease
in
the
world
EU
30
–
40
Million
USA
25
–
30
Million
19
La/n
American
587
m
popula/on
:
Argen/na,
Brazil,
Chile,
Colombia,
Ecuador,
Mexico,
Peru,
Puerto
Rico,
Venezuela
Cuba,
PR,
DR,
T&T,
Guatemala,
El
Salvador,
Honduras,
Nicaragua,
CR,
Panama
19
top
La#n
American
countries:
47
million
Fernando
Ferrer,
MBA
Mul/na/onal
Partnerships
LLC
10

11. Access
to
orphan
drugs
is
granted
in
La/n
America
by
diverse
norms
interna/onal
trea/es
and
conven/ons
na/onal
cons/tu/on
federal
law
statutes
/
codes
decrees
② market
access
is
a
right
Fernando
Ferrer,
MBA
Mul/na/onal
Partnerships
LLC
11

12.
The
Universal
Declara/on
of
Human
Rights
(1)
Everyone
has
the
right
to
a
standard
of
living
adequate
for
the
health
and
well-­‐being
of
himself
and
of
his
family,
including
food,
clothing,
housing
and
medical
care
and
necessary
social
services,
and
the
right
to
security
in
the
widowhood
event
of
unemployment,
sickness,
disability,,
old
age
or
other
lack
of
livelihood
in
circumstances
beyond
his
control.
(2)
Motherhood
and
childhood
are
en/tled
to
special
care
and
assistance.
All
children,
whether
born
in
or
out
of
wedlock,
shall
enjoy
the
same
social
protec/on.
Fernando
Ferrer,
MBA
Mul/na/onal
Partnerships
LLC
12

13. Interna/onal
Covenant
on
Economic,
Social
and
Cultural
Rights
(ICESCR)
Par/es
and
signatories
to
the
ICESCR:
signed
and
ra/fied
(164)
signed
but
not
ra/fied
(6)
neither
signed
nor
ra/fied
(27)
Ar#cle
12
1.
The
States
Par/es
to
the
present
Covenant
recognize
the
right
of
everyone
to
the
enjoyment
of
the
highest
aTainable
standard
of
physical
and
mental
health.
It
commits
its
par/es
to
work
toward
the
gran/ng
of
economic,
social,
and
cultural
rights
(ESCR)
Fernando
Ferrer,
MBA
Mul/na/onal
Partnerships
LLC
13

14. Physician-­‐Pa/ent
rela/onship
is
the
cornerstone
of
medical
prac/ce
“The
health
of
my
pa/ent
will
be
my
first
considera/on”
-­‐ Declara/on
of
Geneva,
World
Medical
Associa/on
“A
physician
shall
owe
his/her
pa/ents
complete
loyalty
and
all
the
scien/fic
resources
available
to
him/her”
-­‐ Interna/onal
Code
of
Medical
Ethics
Fernando
Ferrer,
MBA
Mul/na/onal
Partnerships
LLC
14

15. American
College
of
Physicians
(ACP)
Ethics
Manual
Physicians
have
an
obliga/on
to
promote
their
pa/ents'
welfare
in
an
increasingly
complex
health
care
system.
This
entails
forthrightly
helping
pa/ents
to
understand
clinical
recommenda/ons
and
make
informed
choices
among
all
appropriate
care
op/ons.
It
includes
management
of
the
conflicts
of
interest
and
mul/ple
commitments
that
arise
in
any
prac/ce
environment,
especially.
It
also
includes
stewardship
of
finite
health
care
resources
so
that
as
many
health
care
needs
as
possible
can
be
met,
whether
in
the
physician's
office,
in
the
hospital
or
long-­‐term
care
facility,
or
at
home.
Fernando
Ferrer,
MBA
Mul/na/onal
Partnerships
LLC
15

16. Rare
Disease
regula/ons
in
La/n
America
Argen/na
Brazil
Chile
Colombia
Mexico
ANMAT
ANVISA
ISP
INVIMA
COFEPRIS
Law
26689,
EPF/
2011
Na/onal
law
for
rare
diseases.
Disposi/on
4622
August
2012
Decree
2577/2006
–
768/2006,
RDC
28/2007
RDC
16/2008
allows
for
priority
review-­‐
Ricate
Soto
Law
January,
2015;
medica/ons
for
rare
diseases
and
cancer.
Law
19966
AUGES-­‐GES
Law
1751
of
2015.
Law
1392
of
2010
and
Law
1438
of
2011;
orphan
designa/on
Decree
481
of
2004
Ar/cle
224
Bis
Mexican
Health
Law,
Official
GazeAe,
January
2012;
orphan
drug
designa/on
Fernando
Ferrer,
MBA
Mul/na/onal
Partnerships
LLC
16

17.
Prevalence
and
Incidence
of
rare
diseases:
Bibliographic
data
These
low
incidence
diseases
are
being
treated
in
La/n
America.
Why?
Source:
Orphanet
report
series
,
March
2016
Fernando
Ferrer,
MBA
Mul/na/onal
Partnerships
LLC
17

18. The
stakeholders
needs
must
be
addressed
independently
of
market
size
1. Pa/ents,
caregivers
and
pa/ent
advocacy
organiza/ons
2. Non-­‐governmental
(NGO)
and
Inter-­‐governmental
(IGO)
Organiza/ons
3. Clinicians,
nurses,
pharmacists,
biochemists,
other
health-­‐related
professionals
and
their
professional
associa/ons
(HCPs)
4. Life
science-­‐related
industries
and
services,
and
their
associa/ons
5. Employers
and
unions
6. Na/onal
legisla/ve,
execu/ve,
and
judiciary
powers
7. Government
agencies
8. Civil
society
9. Ins/tu/onal
health
care
providers,
such
as
hospital
systems
and
medical
clinics
10. Purchasers
and
payers,
such
as
employers
and
public
and
private
insurers
11. Health
care
industry
representa/ves
12. Healthcare
policy
makers
at
the
federal,
state
and
local
levels
13. Healthcare
researchers
and
research
ins/tu/ons
14. Interna/onal
and
suprana/onal
organiza/ons
15. Media
and
communica/on
channels.
③
the
value
is
much
higher
than
the
cost
Fernando
Ferrer,
MBA
Mul/na/onal
Partnerships
LLC
18

19. Diagnosis
and
treatment
for
few
pa/ents
are
more
costly
Who
is
gonna
pay
the
bill
!?
Source:
The
Telegraph,
UK,
CREDIT:
REUTERS
Fernando
Ferrer,
MBA
Mul/na/onal
Partnerships
LLC
19

20. The
ques/on
should
be:
Who
is
gonna
pay
the
bill
if
we
do
nothing?
More
people…
more
needs
Humankind
took
millions
of
years
to
grow
to
1
billion
people
…
and
only
113
years
to
reach
2
billion
…
and
95
years
to
become
8
billion
!
• Fernando
Ferrer,
MBA
Mul/na/onal
Partnerships
LLC
20

21. Who
should
be
diagnosed
and
treated?
Individual
Society
.
.
• Fernando
Ferrer,
MBA
Mul#na#onal
Partnerships
LLC
• 21

22.
The
common
good
must
include
the
individual
The
need
of
the
individual
must
be
properly
addressed
It
is
not
about
the
person
the
society.
It
is
about
the
person
AND
the
society
Fernando
Ferrer,
MBA
Mul/na/onal
Partnerships
LLC
22

23. La/n
America
is
the
place
for
Orphan
Drugs
ü We
have
50
millions
pa/ents
ü The
global
industry
has
developed
/
is
developing
diagnos/cs,
medicines,
devices
and
treatments
that
are
ready
for
LA
ü The
HCPs
are
well
prepared
and
trained
ü Health
Care
centers
have
the
ability
/
resources
to
treat
pa/ents
ü There
are
interna/onal
and
na/onal
regula/ons
and
codes
ü Pa/ents
are
being
treated
using
local
budgets
Nothing
is
missing
but
you
!
Fernando
Ferrer,
MBA
Mul/na/onal
Partnerships
LLC
23

24. Challenge
To
bring
beAer
health
and
quality
of
life
for
individuals
and
families
affected
by
rare
diseases
and
cancer
④
ini1a1ves
Fernando
Ferrer,
MBA
Mul/na/onal
Partnerships
LLC
24

25. Ini/a/ves
(1)
Centers
for
Rare
Disorders
Areas
in
health
care
facili/es
for
the
diagnosis,
treatment
and
supply
of
Orphan
Drugs
– Mul/disciplinary
team
of
Health
Care
Prac//oners
(HCPs),
in
situ
and
remote
– Collabora/on
with
local
and
interna/onal
health
care
systems,
public
and
private
organiza/ons
including
academic
ins/tu/ons,
and
KOLs
– Treatments
with
Approved
Orphan
Drugs
and
with
Orphan
Drugs’
Designa/on
status
granted
by
US/PR
and
EU
Fernando
Ferrer,
MBA
Mul/na/onal
Partnerships
LLC
25

26. Ini/a/ves
(2)
Newborn
Screening
&
Pa/ent
Registry
Homogenize/Harmonize
and
expand
the
implementa/on
of
– newborn
screening
(NBS)
programs
– pa/ent
registry
to
allow
beAer
monitoring
of
the
disease
incidence
and
prevalence,
which
is
essen/al
to
make
reliable
predic/ons
for
disease
management
across
La/n
American
Fernando
Ferrer,
MBA
Mul/na/onal
Partnerships
LLC
26

27. Ini/a/ves
(3)
The
year
of
Rare
Diseases
in
La/n
America
Following
the
European
Year
for
Rare
Diseases
2019
• Pa/ents/rela/ves/associa/ons,
healthcare
prac//oners
and
providers,
governments,
industries,
na/onal
and
suprana/onal
organiza/ons,
any
and
all
stakeholders
are
invited
to
support
the
Year
of
Rare
Disease
in
La/n
America.
Fernando
Ferrer,
MBA
Mul/na/onal
Partnerships
LLC
27

28. Conclusions
① 50
million
of
pa/ents
to
receive
Orphan
Drugs
in
La/n
America
② There
are
regula/ons
in
place.
The
right
of
pa/ents
to
have
access
has
being
granted
and
the
medical
ethic
supports
it
③ The
way
is
open;
pa/ents
are
receiving
high
cost
treatments
already
④ New
ini/a/ves
will
help
accelerate
the
adop/on
of
Orphan
Drugs
in
La/n
America.
There
are
strategies
and
knowledge
suitable
for
each
market
What
are
you
wai/ng
for
to
offer
your
products
in
La/n
America?
Fernando
Ferrer,
MBA
Mul/na/onal
Partnerships
LLC
28

29. Thank
you
Muchas
Gracias
Muito
Obrigado
Fernando
Ferrer
Mul/na/onal
Partnerships
LLC
+1
908
219
9291
fferrer@mul/na/onalpartnerships.com
Orphan
Drugs
and
Rare
Diseases
La/n
America