Translating Science into Improved Health Care:

Cancer as a Model

William S. Dalton, Ph.D., M.D.Center Director

H. Lee Moffitt Cancer Center & Research Institute

The Future of Medicine

• Where we are today:Where we are today: Sequencing of the human genome has dramatically improved molecular technology and our understanding of disease

• The potential is HUGE!The potential is HUGE! New technology will ultimately improve molecular diagnosis, identify new targets for therapy, create personalized therapies, and identify populations at risk of disease, especially cancer.

• The challengeThe challenge: How do we translate new How do we translate new molecular technologies into clinical benefits for molecular technologies into clinical benefits for our patients?our patients?

The Scenario

• The Answer: Develop a new approach to deliver cancer care that integrates research at every step of a patient’s journey dealing with cancer, and create alliances with academia, the cancer care community, and patients.

• The Opportunity: Develop a novel delivery system to incorporate new technologies and define their value in the care of cancer patients.

Barriers• Lack of platform/grids for development and

exchange of information: Create eco-system for discovery and data

generation Fragmentation of information/ Need Integration

of datasets Need for real-time access of information Need for clinical outcomes analysis/affect of

intervention• Lack of standards:

Performance of assays Databases No common lexicon

Barriers

• Lack of collaboration and inter-disciplinary approach Create cross-culture environment Improve entrepreneurial culture in

academia• Gap between “the Ivory Towers” and

the point of impact: the community where vast majority of patients are treated

Two Translational Roadblocks on the

Way Toward Improved Public Health

JAMA. 2004;291:1120-1126

…”whereas the National Institutes of Health has been consistently targeting the bench-to-bedside block, no one is taking responsibility for the second, which is integrally tied with the funding of the health care delivery system”

The Future of Medicine

• The future of medicine is based upon new molecular insights into cancer and other diseases.

• Current Model of Delivery: develop technology at research institutions and “carry the tablets to the masses” or “build it and they will come”.

• Proposed Model of Delivery: early involvement of the community in assessing the value of new technologies. Capitalize on economy of scale and “real world” experience from the beginning.

From Vision To Reality:

Cancer as a Model

The Vision

“The right treatment, for the right patient, at the right time and the

right place”

The Requirements• Enrollment of large patient populations in

prospective efforts to collect clinical histories and molecular data;

• Expansion of information technology infrastructure to integrate large data sets from multiple sources;

• Experienced multi-disciplinary research teams to perform data-mining and modeling;

• New healthcare networks capable of reducing new methods to standard practice;

• Federal, State and private health insurance payers to embrace new health care models.

The Challenges – Cost

• Infrastructure cost Bio repositories Large integrated and interoperable

databases Human Resources

Researchers, clinicians, biostatisticians, bioinformaticians

Clinical trials staff, IRB, consenters, abstractors

• Public-private partnerships needed

The Challenges – Patient Understanding

• Patient participation in clinical trials Consenting & patient survey logistics Understanding timing of developing

personalized cancer care Overcoming concerns

Tissue use – Honest Broker systemTissue & data ownershipPrivacy“Genetics” Access by collaborators (NFP & FP)

Challenges – Data Collection

• Interoperability - limited• Robust data collection without interfering in clinical

care process• Precious little data available in a discrete formatPrecious little data available in a discrete format• Few electronic sources of data for direct accessFew electronic sources of data for direct access• Data often manually abstracted retrospectivelyData often manually abstracted retrospectively• Data may be in the MD’s office paper chart rather Data may be in the MD’s office paper chart rather

than the hospital’s electronic medical recordthan the hospital’s electronic medical record• High investment in capital • Navigating HIPAA & Human Subject Research

requirements

Other Challenges

• Reimbursement• Conducting the research• Integration into clinical practice• Training future clinicians and

researchers

The ApproachThe Approach

DataInformationKnowledgeWisdom

Improved medical practiceImproved medical practice

Create a delivery system that will integrate new technologies into the standard of care and develop evidence-based guidelines for

the treatment of cancer.

Total Cancer Care• Will provide an approach to identify the best treatment for

individual patients based on clinical and biological characteristics of a patient’s cancer.

• Integrates new technologies into the standard of care in an evidence-based fashion to personalize treatment and improve individual outcomes.

• Addresses cancer as a public health issue and seeks to improve access, affordability and quality of care by creating an information system to assist in making clinical decisions based on outcomes and comparative effectiveness.

• Creates a network of health care providers and researchers who contribute and share information from individual patients to ultimately improve care of patients from Florida and beyond.

•

Progress

• Total Cancer Care Protocol 33k+ consented patients 9500+ tissues collected since 2006:6500+

profiled 17 contracted consortium sites contributing

70% of tumors Database & data collection enhanced

• Data Warehouse

Total Cancer Care: The Future

•Further expansion of TCC Consortium Sites (FL, US, Internationally)

•Development of a dynamic information-learning system

• Integrated model for personalized medicine Personalized Health Record Cancer Biomarker & Drug

Target Discovery Patient Treatment Matching Comparative Effectiveness

Research

Why Personalized Medicine?

“…when doctors can truly prescribethe right treatment, to the right person, at the right time, we

will have a new levelof precision and effectiveness that will provide the

knowledge-driven power thatis necessary to achieve our highest goals in healthcare

reform—including moreeffective disease prevention and early disease detection.”

HHS Secretary Kathleen SebeliusWritten testimony given during Senate confirmation hearings, April 2, 2009