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

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  

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  

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  

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

Breast Cancer Nation

Why Not Brown?

Value Safety and

Efficacy

Our Current Model of Colon Cancer

Antoni van Leeuwenhoek (1632-1723)

Invented the microscope around 1668

10

The view from 35,000 feet

Everything looks the same from up here

What can we predict?

Standard  of  Care  

New  Pa>ent  

Diagnosis  /  staging  

Standard  Chemotherapy  

Hope  for  the  best  

30%  chance  of    any  response  

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.

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  

Hypothesis  

Prospec>ve  incorpora>on  of  molecular  profiling  will  

transform  global  cancer  care  

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  

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  

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