Treatment: It’s no longer “one size fits all” John Marshall, MD, Georgetown University Medical Center

1. Fighting a Smarter War On Colon Cancer:
John L. Marshall, MD
Value as a new endpoint?
Tel: (202) 444-0275
Fax: (202) 444-1229
http://lombardi.georgetown.edu/GI

2. Stakeholder
Mo-va-on
Stakeholders
• FDA
• CMS/Payers
• NCI/CTEP
• PhRMA
• Community
Onc
• Academic
Onc
• Pa>ents
Priority/Agenda
• Safety
and
Efficacy
• Cost
Control/Value
• Cure
Cancer
• Markets,
ROI
• Efficient/Quality
Care
• Clinical
Trial
Accrual
• Cure/Benefit/Altruism

3. Stakeholder
Mo-va-on
Stakeholders
• FDA
• CMS/Payers
• NCI/CTEP
• PhRMA
• Community
Onc
• Academic
Onc
• Pa>ents
Priority/Agenda
• Safety
and
Efficacy
• Cost
Control/Value
• Cure
Cancer
• Markets,
ROI
• Efficient/Quality
Care
• Clinical
Trial
Accrual
• Cure/Benefit/Altruism

4. Worldwide
Cancers
Sta>s>cs
F.
Bray
et
al,
Lancet
Oncol
2012;
13:
790–801
and
h"p://www.cancer.org/aboutus/globalhealth.
2030
values
are
es>mated
using
projected
incidence
and
mortality
rates
from
2008
to
2030
and
weigh>ng
for
prevalence
in
developed
compared
to
developing
countries.
GI
2008
0
1000000
2000000
3000000
4000000
5000000
6000000
7000000
0
1000000
2000000
3000000
4000000
5000000
6000000
7000000
2030
Breast
Prostate
Lung
Lung
Prostate
Breast
GI

5. Gastrointestinal (GI) Cancers Facts
v GI cancers represent the most common and fatal cancers in the world
v 2009: 275,720 new diagnosis of GI Cancers and 135,830 deaths in the
US alone
v Anal Cancer
v Colorectal Cancer
v Esophageal Cancer
v Gallbladder Cancer
v Liver Cancer
v Pancreatic Cancer
v Small Intestine Cancer
v Stomach/Gastric Cancer
v No two cancers are alike and treatments must be selected based on an
individual’s tumor characteristics, by personalized medicine

6. Breast Cancer Nation

7. Why Not Brown?

8. Value
Safety and
Efficacy

9. Our Current Model of Colon Cancer

10. Antoni van Leeuwenhoek
(1632-1723)
Invented the microscope
around 1668
10

11. The view from 35,000 feet
Everything looks the same from up here

12. What can we predict?

13. Standard
of
Care
New
Pa>ent
Diagnosis
/
staging
Standard
Chemotherapy
Hope
for
the
best
30%
chance
of
any
response

16. Cancer is driven by hyperactive or defective protein circuits
The components of these circuits contain the drug targets
of the future.
Patient A Patient B
Each patient’s cancer is different. A drug that works for
one patient may not work for another patient with the
same cancer.

17. Ruesch
Symposia:
• 2010
Biomarkers:
Can
we
measure
them?
• 2011
Defining
Value
in
Cancer
• 2012
Clinical
Research:
Engaging
the
97%
• 2013
Molecular
Profiling:Research/
Prac>ce

18. Hypothesis
Prospec>ve
incorpora>on
of
molecular
profiling
will
transform
global
cancer
care

19. Personalized
Medicine
New
Pa>ent
Diagnosis
/
staging
HER2 ↑
TS ↓
TOP1 ↑
KRAS ↑
ERCC1 ↓
SPARC ↑
B-RAF ↑
PGP ↓
CURE?
Targeted
Therapy
Personalized
Therapy
↑
Response
Molecular-­‐
Tailored
Therapy

20. GI
Cancer
Pa>ents
Smart Centers
Profile
All
Treatment
Outcomes
Unified
CRF/EMR
BioBank
Team
Central
Imaging
Central
Consent
Data
Cloud:
Shared
&
IP
Protected
Managed
by
DSM,
STATS
HIPPA
compliant,
Regulatory Review
Cancer
Centers
Profilers
Pharma
+
Guidelines

21. Fundamental
Shies
In
Cancer
Care
Yesterday
• Consump>on
• Individual
Prac>ces
• Rich
Countries
• Microscope
• Safety
and
Efficacy
• Large
trials
• 1.4
months
• QOL
• Pa>ent
as
a
“Subject”
• Chao>c
Data
Collec>on
• Ins>tu>onal
IRBs
• Na>onal
Approvals
Tomorrow
• Outcomes
• Healthcare
Systems
• All
Countries
• Gene
Profile
• Value
• Small
trials
• “Substan>al
Improvement”
• Pa>ent
Reported
Outcomes
• Pa>ent
as
a
“Partner”
• Standard
Data
Collec>on
• Central/Na>onal
IRBs
• Global
Approvals