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

The Scenario The Answer: Develop a new approach to deliver cancer care that integrates research at every step of a patients 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: …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 system Tissue & data ownership Privacy 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 MDs office paper chart rather than the hospitals electronic medical recordData may be in the MDs office paper chart rather than the hospitals 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 Approach Data Information Knowledge Wisdom Improved 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 patients 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 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 prescribe the right treatment, to the right person, at the right time, we will have a new level of precision and effectiveness that will provide the knowledge-driven power that is necessary to achieve our highest goals in healthcare reformincluding more effective disease prevention and early disease detection. HHS Secretary Kathleen Sebelius Written testimony given during Senate confirmation hearings, April 2, 2009