Drug Pipeline Prez Final

Bench to Bedside:
The Drug Development Pipeline
Shannon McArdel
Immunology Program, Harvard University
Outline for this session
Introduction Illustration Innovations

•  The goal •  Multiple Sclerosis •  Tissue on chip
•  The pathway •  Natalizumab
•  The players
•  The challenges
What is a drug?
A product that alleviates, cures or prevents disease, or
is intended to affect the structure or function of the body.
Antibiotics
Insulin
(~100x bigger
than antibiotic)
Chemotherapeutic
agents
Antibody
(600x bigger
than antibiotic)
Tricyclic
antidepressants
Pennicillin: Public Domain

Gleevac: Public Domain
Antidepressants: Harbin, Public Domain
Insulin: Isaac Yonemoto, CC-BY
Antibody: Public Domain
What qualities do we want in a new drug?
Efficacy
Safety
Affordability
Convenience
or testing
ans.
IND Application 4
1
The sponsor submits an
20 - 80 P large-scale studies in Phase 3 will b

At the end of Phase 2, FDA and sponsors discuss how
What is the path to get3

DRUG Investigational New Drug
H
there?
SPONSOR
(IND) application to FDA
A
2 5
The typical number
IEW
based on the results from S
intial testing that include, E emphasizes safety. T
he IND to assure
sed studies,
red to as clinical
lace human
the drug’s
H
manufacturing,
A and
1000’s
P composition and
1 FDA
Approval
drug's most frequen
metabolized and exc
The typical number of patients used in Phase 3. These studies gather more
S a plan for testing
easonable risk of develops
Study of
o verifies that E information about safety and effectiveness, study different populations and
4
uate informed the drug on humans.
different dosages, and uses the drug in combination with other drugs.
Disease
uman subject U.S. Food and Drug Administration
3 What is a drug as defined by
Drug Approval Process
IND Application
100’s
A drug is any product that is intended for use in the diagnosis, cure mitigati
Efficacy
Animals Tested prevention of disease; and that tis intended to affect the structure or any func
P DRUG
The sponsor submits an H SPONSO
Sponsor must test new A The typical number
Safety
Investigational New Drug
(IND)
drug onapplication
animals forto FDA S effectiveness. This g
basedMultiple
Drug Sponsor’s Discovery and Screening Phase
on the results from IND REVIEW E in people who have
toxicity. species
Affordability receiving the drug a
areintial
usedtesting that include,
to gather basic
the drug’s composition and
FDA reviews the IND to assure
Convenience 2 treatment--usually a
information on the safety
manufacturing, and
and efficacy of the
that the proposed studies,
generally referred to as clinical 3
P
H
A
20 80
evaluated,-and short
2
The typical number of heal
develops a plan for testing trials, do not place human S
compound being subjects at unreasonable risk of E emphasizes safety. The goa
the drug on humans. drug's most frequent side e
investigated/researched. harm. FDA also verifies that 1 metabolized and excreted.
there are adequate informed Clinical

Pre-Clinical At the end of Phase 2, FDA and
Research
consent and human subject
protection.
DRUG Research
4
Flickr, Mycroyance, CC BY-NC 2.0
IND Application SPONSOR
P 100’s
1
DrugProcess,
Drug Approval Develope
fda.govd The sponsor submits an H
Barnheartowl, Remigho, egore911 open clip art Investigational New Drug A The typical number of pati
effectiveness. This goal is to
What are the challenges?
14 years 85 Failure
% rate $2.6 Billion
Average length of time From Phase I to FDA Cost per successful drug,
from target discovery to approval, during clinical when all failures are
approval of a new drug. trials. factored in.
Why do these challenges exist?
Dr. Francis Collins, “Tackling the bottlenecks in the drug development pipeline”, NIH Director’s Blog, January 2014
Hay et al, Nature Biotechnology, January 2014
PR, Tufts CSDD 2014 Cost Study, November 2014
Questions?
Example:
Multiple Sclerosis (MS) and natalizumab
Multiple Sclerosis (MS) Natalizumab

Immune cells enter the
•  Affects 90 per 100,000 brain, cause damage
•  Prevents immune
people in the US cells from entering
the brain
•  Due to damage to myelin,
the insulation for nerves
•  Thought to be caused by
immune cells
Carrie Hersh, “Multiple Sclerosis,” Cleveland Clinic Continuing Education

A.D.A.M. Inc, http://www.nlm.nih.gov/medlineplus/ency/imagepages/17089.htm
Multiple sclerosis:
Identified as disease in 1868
1868 1960 1970 1980 1990 2000 2010 2020

Pre-clinical research:
Is there a way to cure multiple sclerosis?
Pre-clinical research
1868 1960 1970 1980 1990 2000 2010 2020

How do immune cells enter inflamed brain tissue?
Blood
vessel
Brain
Receptors that Test antibodies that Result

might be involved block each one
22 2 E
1
S
emphasizes safety. The goal here in this phase is to determ
E emphasizes
drug's most frequent side effects
metabolized and excreted.
safety.
are and, often, how
drug's most freque
the d
1 metabolized and ex
Clinical research: 4
IND Application
100’s
Drug Developed
1 IND Application
Test a drug IND
in Application
humans
P
H
A 4 4
100’s
Investigational New Drug The typical number of patients used in Phase 2; this phase
Drug sponsor develops a (IND) application to FDA S effectiveness. This goal is to obtain preliminary data on wh
new drug compound and based on the results from E P

in people who have a certain disease or condition. For con
1 1 H
eloped Drug Develop
it approved ed
receiving the drug are compared with similar patients rece
seeks to have The sponsor submits
intial testing an
that include,
2 treatment--usually a placebo, or a different drug. Safety con
by FDA for sale in the
United States.
Investigational New
manufacturing, and
Drug Investigational New Drug evaluated, andAshort-term side effects
The typical number
are studied.
evelops a Drug sponsor develops a (IND) application to for

develops a plan FDAtesting (IND) application to FDA S effectiveness. This g
pound and new drug compound and

the drug on humans.
based on the results from based on the results from Clinical research E in people who have
1868 seeks to have 1960 1970 1980 1990
intial testing that include, 2000 2010 2020
receiving the drug
approved
ALUATIO
by FDA
in the FDA’s Center
N
forfor
it approved Animals
sale in the
Drug
Tested intial testing that include,
the drug’s composition and the drug’s composition and
DRUG
SPONSOR
At the end of Phase
2
2, FDA and sponsors discuss how large-scale stud
treatment--usually
AN
Sponsor must test new

Evaluation andStates.
Research evaluated, and sho
D RE ARCH
United manufacturing, and manufacturing, and

SE
drug on animals for

(CDER) evaluates new drugs
toxicity. Multiple species INDfor
develops a plan REVIEW
testing develops a plan for testing
5
before they can be sold.
1000’s
are used to gather basic FDA reviews the IND to assure the drug on humans.
the drug on humans. P
information on the safety that the proposed studies,
generally referred to as clinical
H
enter’s evaluation not only prevents quackery, but also
trials, do not place human A
compound being S The typical number of patients used in Phase 3. These stud
Animals Tested
des doctors and patients the information they need to
medicines wisely. CDER ensures that drugs, both Animals Tested
investigated/researched.
subjects at unreasonable risk of
harm. FDA also verifies that E
DRUG
At the end of Phase 2, FDA and
information about safety and effectiveness, study different
DRUG
d-name and generic, are effective and their health there are adequate informed different dosages, and uses the drug in combination with o
g FDA’s Center for Drug
fits outweigh their known risks. consent and human subject SPONSOR
3 SPONSOR
Sponsor must test new Sponsor must test new protection.
arch
Evaluation and Research
drug on animals for drug on animals for
w drugs
toxicity. Multiple species IND
toxicity. Multiple REVIEW
species IND REVIEW
5
old.before they can be sold.
ry, but also

uation
are used to gather basic
not only prevents quackery, but also
are used to gather

basic the IND to assure
FDA reviews
information onthat
thethe proposed studies,
safety
trials, do not place human
trials, do not place human
P
H
A
5
1000
ey need to compound being compound being subjects at unreasonable risk of S The typical number
s and patients the information they need to subjects at unreasonable risk of
oth CDER ensures that drugs,
wisely. both harm. FDA also verifies that
investigated/researched. harm. FDA also verifies that E information about
dealth
generic, are effective and their health there are adequate informed there are adequate informed different dosages, a
gh their known risks. consent and human subject
protection.
protection.
3

Drug Approval Process, fda.gov
Features of clinical trials
Randomization
Placebo
Blinded
Study size $ $$$
Remigho,, open clip art

A drug is any product that is intended for use in the diagnosis, cure mitigation, treatment , or that include, receiving the dru
seeks to have it approved intial testing
2
20 - 80
prevention of disease; and that tis intended to affect the structure or any function ofthe
thedrug’s
body.composition and treatment--usual
by FDA for sale in the P
United States.
3
manufacturing, andH
develops a plan forA
testing
evaluated, and sh
2
The typical number of healthy volunteers used in Phase 1; this phase
the drug on humans.S
Eemphasizes safety. The goal here in this phase is to determine what the
ALUATIO
Clinical trials are1run in phases
Animals Tested
drug's most frequent side effects are and, often, how the drug
Drug Sponsor’s Clinical metabolized
Studies/Trials
and excreted. DRUG
is end of Phase 2, FDA an
At the
N
FDA’s Center for Drug SPONSOR
AN

D RE ARCH
4
SE
drug on animals for

toxicity. Multiple species IND REVIEW
5
IND Application
3
P
H 20 - 80
P
that the proposed studies, 100’s P
H 1000
1
A H
The typical number of healthy volunteerstrials,
useddoinnot
PhaseA
place1; this phase
human A
The typical number of patients used in Phase 2; this phase emphasizes
Investigational S
New Drug
compound being S The typical numb
E
(IND) application to FDA emphasizes safety. The goal here in this phase S
subjects at unreasonable risk ofeffectiveness. This goal is to obtain preliminary data on whether the drug
is to determine what the works
medicines wisely. CDER ensures that drugs, both investigated/researched. harm. FDA also verifies that
drug's most frequent side effects are and,there
often, how E
the drug is in people who have a certain disease or condition. For E
controlled
information abou
trials, patients
based
d-name and generic, are effective and theiron the results from
health are adequate informed
1
fits outweigh their known risks. intial testing that include, metabolized and excreted.
protection. 2 3
consent and human subject receiving the drug are compared with similar patients receiving a different
different dosages
the drug’s composition and treatment--usually a placebo, or a different drug. Safety continues to be
manufacturing, and evaluated, and short-term side effects are studied.
4
develops a plan for testing
50 100100 1000’s
Healthy Patients Patients
’s
the drug on humans.
als Tested
participants P
H
’s DRUG
At the end of Phase 2, FDA and sponsors discuss how large-scale studies in Phase 3 will be done.
A The typical number of patients used in Phase 2; this phase emphasizes
must test new
animals for
Safety S
E Efficacy SPONSOR
effectiveness. This goal is to obtain preliminary data on whether the drug works
in people who have a certain disease or condition. For controlled trials, patients
Efficacy
IND REVIEW receiving the drug are compared with similar patients receiving a different
Multiple species
2
5 1000’s
to gather basic treatment--usually a placebo, or a different drug. Safety continues to be
evaluated, and short-term side effects are studied. P
ion on the safety that the proposed studies,
H
acy of the
nd being subjects at unreasonable risk of S The typical number of patients used in Phase 3. These studies gather more
ted/researched. harm. FDA also verifies that E information about safety and effectiveness, study different populations and
there are adequate informed
At the end of Phase 2, FDA and sponsors discuss how large-scale studies in Phase 3 will dosages,
different be done. and uses the drug in combination with other drugs.
DRUG
SPONSOR
protection. 3
Page
5 P
H
A
1000’s
S The typical number of patients used in Phase 3. These studies gather more
E information about safety and effectiveness, study different populations and
Drug Approval Process, fda.gov 3
2
20 - 80
thedrug’s
United States.
3
manufacturing, andH
testing
evaluated, and sh
2 each phase, many

ALUATIO
At 1 drugs fail
Animals Tested
Studies/Trials
and excreted. DRUG
At the
N
AN

D RE ARCH
4
SE
drug on animals for

5
IND Application
3
P
H 20 - 80
P
H 1000
1
A H
useddoinnot
PhaseA
place1; this phase
human A
Investigational S
New Drug
E
often, how E
controlled
information abou
trials, patients
based
1
protection. 2 3
different dosages
4
50 100100 1000’s
’s
the drug on humans.
als Tested
participants P
H
’s DRUG
must test new
animals for
Safety S
E Efficacy SPONSOR
Efficacy
Multiple species
2
5 1000’s
treatment--usually a placebo, or a different drug. Safety continues to be
33% 42%
to gather basic FDA reviews the IND to assure
H
acy of the
ted/researched. harm. FDA also verifies that E information about safety and effectiveness, study different populations and
Not safe Not effective

there are adequate informed
different be done. and uses the drug in combination with other drugs.
DRUG
SPONSOR
protection. 3
Page
5 P
H
A
1000’s
Drug Approval Process, fda.gov different dosages, and uses the drug in combination with other drugs.
Data from Hay et al, Nature Biotechnology, January 20143
P 100’s 4
100’s
H
IND Application A The typical number of patients used in Phase 2; this phase emphasizes
S effectiveness. This goal is to obtain preliminary data on whether the drug works P
E
receiving the drug are compared with similar patients receiving a different
H
2
60%
of trials in Phase 3 are treatment--usually a placebo, or a different drug. Safety continues to be
evaluated, and short-term side effects are studied.
suspended
A
S
The typical number of patients use
effectiveness. This goal is to obtain
based on the results from E in people who have a certain disea
receiving the drug are compared w
intial testing that include,
DRUG
2
treatment--usually a placebo, or a d
SPONSOR Reasons'for'suspension'during'phase'III
evaluated, and short-term side effe
manufacturing, and
the drug on humans.
5 H
A
P
1000’s 54%
At the end of Phase 2, FDA and sponsors discu
DRUG
3 SPONSOR
1000’s Patients
IND REVIEW
Page 1
18% 19%
Efficacy
in
specific
trials, dopopulations
not place human
5 P 9%
H
A
1000’s
subjects at unreasonable risk of S The typical number of patients use
harm. FDA also verifies that Efficacy'''''''Safety''''Commercial'''Unknown
E information about safety and effec
there are adequate informed different dosages, and uses the dru
protection. 3
Reason for suspension

Through)Phases)of)Clinical)Trials
Why are safety and efficacy hard to predict?
67%
39%
15.3%
Phase:))))))1))))))))))))))))))))))))))2)))))))))))))))))))))))))))3)))))))))))))))))))))Approval
•  Research models of disease are not the same as humans

•  Studies of humans are limited by available materials
•  A new drug needs to be better than current drugs

Questions?
Hay et al, Nature Biotechnology, January 2014

2
20 - 80
thedrug’s
United States.
3
manufacturing, andH
testing
evaluated, and sh
2
ALUATIO
Natalizumab had very1promising results
Animals Tested
Studies/Trials
and excreted. DRUG
At the
N
AN

D RE ARCH
4
SE
drug on animals for

5
IND Application
3
P
H 20 - 80
P
H 1000
1
A H
useddoinnot
PhaseA
place1; this phase
human A
Investigational S
New Drug
E
often, how E
controlled
information abou
trials, patients
based
1
protection. 2 3
different dosages
4
50 100100 1000’s
’s
the drug on humans.
als Tested
participants P
H
’s DRUG
must test new
animals for
Safety S
E Efficacy SPONSOR
Efficacy
Multiple species
2
5 1000’s
to gather basic treatment--usually a placebo, or a different drug. Safety continues to be
H
acy of the
ted/researched.
1997 - 1999 harm. FDA also verifies that
there are adequate informed 1999 - 2003 E
different 2003 -
information about safety and effectiveness, study different populations and
be done. and uses the drug in combination with other drugs.
DRUG
SPONSOR
protection. 3
Page
5 P
H
A
1000’s
Drug Approval Process, fda.gov different dosages, and uses the drug in combination with other drugs.
Steinman, Nat Rev Drug Disc, 2005.
3
22 2 E
1
S
E emphasizes
safety.
are and, often, how
drug's most freque
the d
4
100’s
IND Application
Drug Developed Expedited FDA approval,

1 IND Application IND Application in 4
2004
P
H
A 4
100’s
Investigational New Drug The typical number of patients used in Phase 2; this phase
Drug sponsor develops a (IND) application to FDA S effectiveness. This goal is to obtain preliminary data on wh
new drug compound and based on the results from E P

in people who have a certain disease or condition. For con
1 1 H
eloped Drug Develop
it approved ed
intial testing an
that include,
United States.
Investigational New
manufacturing, and
Drug Investigational New Drug evaluated, andAshort-term side effects
The typical number
are studied.


the drug on humans.
1868 seeks to have 1960 1970 1980 1990
receiving the drug
approved
ALUATIO
by FDA
N
forfor
it approved Animals
sale in the
Drug
DRUG
SPONSOR
At the end of Phase
2
treatment--usually
AN

D RE ARCH

SE
drug on animals for

5
1000’s
H
Animals Tested
DRUG
DRUG
3 SPONSOR
arch
w drugs
(CDER) evaluates new drugs In clinical trials 1997 - 2004
species IND REVIEW
5
are used to gather basic the IND to assure
FDA reviews
information onthat
safety
P
H
A
5
1000
D
ry, but also trials, do not place human
E
uation not only prevents quackery, but also trials, do not place human
ey need
s and
wisely.
to
patients
compound being
the information they need to
oth CDER ensures that drugs, both
compound being subjects at unreasonable risk of
harm. FDA also verifies that
investigated/researched. AP
harm. FDA also verifies that P R OV S
E
The typical number
information about
dealth
gh their known risks. consent and human subject
protection.
protection.
3

Natalizumab suspended 3 months later:
Two cases of fatal brain inflammation
(PML: Progressive multifocal leukoencephalopathy)
During multiple During infection

sclerosis: Natalizumab with JC virus:
prevents immune
cells from entering
Immune cells enter
the brain
Immune cells enter the
brain, cause brain, stop the spread
damage of virus, and protect
brain tissue
A.D.A.M. Inc, Medline, National Library of Medicine

Steinman, Nat Rev Drug Disc, 2005.
22 2 E
1
S
E emphasizes
safety.
are and, often, how
drug's most freque
the d
Reapproved 1 year later 4

100’s
IND Application
Drug Developed
1 IND Application
Stringent restrictions for use 4 P
P
H
A 4
100’s
The typical number of patients used in Phase 2; this phase
Drug sponsor develops a

IND Application
(IND) application to FDA S effectiveness. This goal is to obtain preliminary data on wh
new drug compound and based on the results from E in people who have a certain disease or condition. For con
1 1 H
eloped Drug Develop
it approved ed
intial testing an
that include,
United States.
Investigational New
manufacturing, and
Drug Investigational New Drug A The typical number
evaluated, and short-term side effects are studied.


the drug on humans.
1868 seeks to have 1960 1970 1980 1990
receiving the drug
approved
ALUATIO
by FDA
N
forfor
it approved Animals
sale in the
Drug
DRUG
SPONSOR
At the end of Phase
2
treatment--usually
AN

D RE ARCH

SE
drug on animals for

5
1000’s
H
Animals Tested
DRUG
DRUG
3 SPONSOR
arch
w drugs
species IND REVIEW
5
are used to gather
information onthat
basic the IND to assure
FDA reviews
safety
P
H 5
1000
ry, but also
uation
not only prevents quackery, but also
trials, do not place human trials, do not place human SU S E D
A
ey need
s and
wisely.
to
patients
compound being
the information they need to
oth CDER ensures that drugs, both
compound being subjects at unreasonable risk of
P O
E V The typical number
APPR NDED information about

S
E
dealth
gh their known risks. consent and human subject consent and human subject 3 OVED
REAP R P
protection. protection.

Key points from the natalizumab story
Discoveries in different fields led to the

development of natalizumab
Pre-clinical studies could not predict

complications due to JC virus infection
SU S ED
The FDA wants to make SAFE APPPREONVDED
drugs available to patients P P R OV ED
REA
or testing
ans.
IND Application 4
1
20 - 80
P large-scale studies in Phase 3 will b
At the end of Phase 2, FDA and sponsors discuss how
3
DRUG Investigational New Drug
H
Summary
SPONSOR
A
2 5
The typical number
IEW
based on the results from S
intial testing that include, E emphasizes safety. T
he IND to assure
sed studies,
red to as clinical
lace human
the drug’s
H
manufacturing,
A and
1000’s
P composition and
1 FDA
Approval
drug's most frequen
metabolized and exc
The typical number of patients used in Phase 3. These studies gather more
S a plan for testing
easonable risk of develops
Study of
o verifies that E information about safety and effectiveness, study different populations and
4
uate informed the drug on humans.
Disease
uman subject U.S. Food and Drug Administration
3 What is a drug as defined by
Drug Approval Process
IND Application
100’s
A drug is any product that is intended for use in the diagnosis, cure mitigati
Efficacy
Animals Tested prevention of disease; and that tis intended to affect the structure or any func
P DRUG
The sponsor submits an H SPONSO
Sponsor must test new A The typical number
Safety
(IND)
drug onapplication
animals forto FDA S effectiveness. This g
basedMultiple
Drug Sponsor’s Discovery and Screening Phase
on the results from IND REVIEW E in people who have
toxicity. species
Affordability receiving the drug a
areintial
usedtesting that include,
to gather basic
Convenience 2 treatment--usually a
manufacturing, and
generally referred to as clinical 3
P
H
A
20 80
evaluated,-and short
2
The typical number of heal
develops a plan for testing trials, do not place human S
compound being subjects at unreasonable risk of E emphasizes safety. The goa
the drug on humans. drug's most frequent side e
investigated/researched. harm. FDA also verifies that 1 metabolized and excreted.
there are adequate informed Clinical

Pre-Clinical At the end of Phase 2, FDA and
Research
protection.
DRUG Research
4
IND Application SPONSOR
P 100’s
1
DrugProcess,
Drug Approval Develope
fda.govd The sponsor submits an H
Barnheartowl, Remigho, egore911, Open clip art Investigational New Drug A The typical number of pati
effectiveness. This goal is to
To address the challenges
Repurposing of Improve laboratory New technology:

approved drugs models of disease do more with less
à  Use drugs already à  Better understand à  More data from
approved for safety the current small clinical
models samples
à  Test in other
candidate diseases à  New models à  Allows better study
of human biopsies,
blood
Steve Perrin, Nature, March 2014, “Preclinical research: Make mouse studies work”
Innovative approach:
Tissue chip for drug screening
Tissue chips Pre-Clinical
•  Mimic human tissues
Tissue Chips
•  Screen drugs before
testing in humans
Clinical
Dr. Francis Collins, “Tackling the bottlenecks in the drug development pipeline”, NIH Director’s Blog, January 2014
Tissue Chip, NCATS. http://www.ncats.nih.gov/tissuechip/chip/disease
Summary
The drug development pipeline
•  The goals
–  Safety, Efficacy, Affordability
•  The pathway
–  Long, expensive, challenging
•  The players
–  Scientists…Pharma…FDA…Patients
•  The challenges
–  Difficult to understand how the body works
–  Difficult to predict how a drug will act in humans
•  Innovations
–  Facilitated by growing scientific knowledge
–  Facilitated by new technology
Thank you!
SITN would like to acknowledge the following
organizations for their generous support of this event.

Drug Pipeline Prez Final

Enviado por

Dados do documento

Direitos autorais

Formatos disponíveis

Compartilhar este documento

Compartilhar ou incorporar documento

Opções de compartilhamento

Você considera este documento útil?

Este conteúdo é inapropriado?

Direitos autorais:

Formatos disponíveis

Drug Pipeline Prez Final

Enviado por

Direitos autorais:

Formatos disponíveis

Bench to Bedside:

The Drug Development Pipeline

Introduction Illustration Innovations

Pennicillin: Public Domain

20 - 80 P large-scale studies in Phase 3 will b

What is the path to get3

there are adequate informed Clinical

Why do these challenges exist?

Multiple Sclerosis (MS) Natalizumab

Carrie Hersh, “Multiple Sclerosis,” Cleveland Clinic Continuing Education

1868 1960 1970 1980 1990 2000 2010 2020

A.D.A.M. Inc, http://www.nlm.nih.gov/medlineplus/ency/imagepages/17089.htm

Flickr, Mycroyance, CC BY-NC 2.0

Receptors that Test antibodies that Result

new drug compound and based on the results from E P

evelops a Drug sponsor develops a (IND) application to for

pound and new drug compound and

Sponsor must test new

United manufacturing, and manufacturing, and

drug on animals for

ry, but also

and efﬁcacy of the

Flickr, Mycroyance, CC BY-NC 2.0

Study size $ $$$

Remigho,, open clip art

Sponsor must test new

drug on animals for

2 each phase, many

Sponsor must test new

drug on animals for

Not safe Not effective

Drug Approval Process, fda.gov

Why are safety and efficacy hard to predict?

• Research models of disease are not the same as humans

Data from Hay et al, Nature Biotechnology, January 2014

Hay et al, Nature Biotechnology, January 2014

Sponsor must test new

drug on animals for

Drug Developed Expedited FDA approval,

new drug compound and based on the results from E P

evelops a Drug sponsor develops a (IND) application to for

pound and new drug compound and

Sponsor must test new

United manufacturing, and manufacturing, and

drug on animals for

Flickr, Mycroyance, CC BY-NC 2.0

During multiple During infection

A.D.A.M. Inc, Medline, National Library of Medicine

Reapproved 1 year later 4

Drug sponsor develops a

evelops a Drug sponsor develops a (IND) application to for

pound and new drug compound and

Sponsor must test new

United manufacturing, and manufacturing, and

drug on animals for

APPR NDED information about

Flickr, Mycroyance, CC BY-NC 2.0

Discoveries in different fields led to the

Pre-clinical studies could not predict

there are adequate informed Clinical

Repurposing of Improve laboratory New technology:

Você também pode gostar

•  Research models of disease are not the same as humans