2006 CTOS Changing Role of Patient Organizations In Cancer Research

2006 CTOS

Changing Role

of

Patient Organizations

In Cancer Research

Announcement

A cure for all cancers has just been discovered!

GO HOME !

Just kidding

On the other hand,

One has to wonder

For patients with GIST (Gastrointestinal Stromal

Tumor)

Life Raft Life Raft GroupGroup

Our

Mission

Is Survival

Keeping GIST Patients Alive By Ensuring

• That no one dies because they cannot access treatment

• That no one dies because of their ignorance or that of their physician

• That research is coordinated to urgently find a cure

LRG Research Has Two Parallel Tracks.

1. We conduct our own research using medical updates provided by patients and caregivers

2. We direct a coordinated strategy by the LRG world class research team to find a cure for GIST that will serve as a model for other cancers

We conduct our own research using medical

updates provided by patients and caregivers

LRG’s New Medical Database

Why The Life Raft Group Conducts Its Own Research

• Timeliness

• Patient Relevance

• Information not available in clinical trials

• Survival

History Of Life Raft Group Research

• Gleevec Resistance:– Introduced evaluation of actual dosage as

opposed to intended (starting) dosage– Evaluated relapse amongst metastatic GIST

patients– Determined that there was a relationship

between dosage and relapse

• Growing medical data-bases for adult and pediatric GIST

History Of Life Raft Group Research

• Gleevec Response Rates: First to report Gleevec Effectiveness for GIST patients

• Gleevec Side Effects: – Designed Severity Rating Scale for Patient

Self Evaluation– First to report that Side Effects Got Better

Over Time– First to report Gender Differences in Side

Effects

The LRG ability to collect patient driven data and publish it on our website and in our newsletters had unintended consequences

• Turned the flow of clinical trial information upside down: patient and patient’s doctor get the information first

• Increased the leverage of the LRG regarding clinical trials

Time does not permit a more detailed discussion of the research we conduct using medical updates provided by patients and caregivers

Instead I would direct my presentation to:

We direct a coordinated strategy by the LRG world

class research team to find a cure for GIST that will serve as a model for other cancers

We have not made enough progress with the traditional

approach to research

Too many are still dying

Change in the US Death Rates by Cause, 1950 & 2001

* Age-adjusted to 2000 US standard population.Sources: 1950 Mortality Data - CDC/NCHS, NVSS, Mortality Revised.2001 Mortality Data–NVSR-Death Final Data 2001–Volume 52, No. 3. http://www.cdc.gov/nchs/data/nvsr/nvsr52/nvsr52_03.pdf

CerebrovascularDiseases

21.8

193.9

586.8

48.1

180.7

245.8

194.4

57.5

0

100

200

300

400

500

600

1950

2001

HeartDiseases

Pneumonia/Influenza

Cancer

Rate Per 100,000

Traditional Research Approach

• Donate money to an institution in the name of research, perhaps with a specific disease designation

or

• General call for proposals from the research community: Individual projects are selected for funding (typical government approach)

What’s wrong with the traditional research approach?

• High overhead costs: from 50 to 75%

• No overall strategic plan; lack of coordination

• Little accountability

• Lack of urgency

How Does the Life Raft Group Strategy Address the

Shortcomings of The Traditional Research

Approach?

We Reduced High Overhead Costs

The LRG reduced overhead costs in our research grants from between 50 to 75% down to no more than 10%

We Addressed A Lack of Strategy and Coordination

• We assumed a leadership role

• We brought together the best scientists in the world

• We told them that if they created a coordinated strategic plan, and agreed to cooperate, that we would fund it

• They did

• We did

Our Own Research Team Includes World Class Scientists

From

• Catholic University, Leuven Belgium• Cleveland Clinic• Dana-Farber Cancer Center/Brigham &

Women’s Hospital, Boston• Memorial Sloan Kettering• Oregon Health Sciences University/VA

Hospital• Stanford University

Our Research Team Members Have Dual Roles

• To Effectively Implement Their Own Institution’s Research Component of Our Strategic Plan

• To Coordinate a Priority Research Area Across Institutional and National Boundaries

Our Research Team Created A Strategic Plan

• We committed two year grant awards to implement it

• We published the plan on our website: www.liferaftgroup.org

We Funded Two Tissue BanksTo Support The Search For A

Cure

–At Memorial Sloan Kettering for pediatric GIST

–At Stanford University for adult GIST

We Introduced Accountability to the Research Grant Process

• We created six month funding cycles and required a satisfactory progress report to justify further funding

• We are prepared to shift funds from less promising to more promising areas

We introduced a sense of urgency to the search for a cure for GIST

• We created incentives to reward and accelerate progress by building in accelerated and supplemental funding of promising breakthroughs

President Nixon declared war on cancer in 1971, 35 years ago.

Our evaluation so farApril 18, 2006

Dear Norman,

In the Life Raft Group Research plan of action, one finds both true dedication and conviction that can only emerge from one’s heart as well as efficiency and professionalism… It seems to me that this combination is the best ground for successful research and I would like to express my sincere respect and admiration for your efforts and the efforts of your group. I have no doubt that you will succeed in making a significant contribution to GIST research …

Yours truly,

Dan Vasella, M.D., CEO, Novartis

GIST Is The Perfect Model For Demonstrating How To Cure

Other Cancers

• Relatively simple and increasingly understood mechanism of cancer mutations

• Growing list of targeted drugs to address these mutations

• Innovative research strategy• Unprecedented collaboration between

researchers, pharmaceutical companies and patients

Patient Roles in Cancer Research Have Evolved

• Passive Participant

• Some Input

• Seat at the Decision Making Table

• Seat at the Head of the Decision Making Table

Food for Thought

Can we distinguish between acts of outrage that make us feel good and

acts of intervention that actually change something ?

ThanksFor Listening

The Life Raft Group