Aging, Frailty, and Medical Therapy for Colorectal Cancer: Who Should Be Treated and How? Martine Extermann M.D., Ph.D. Moffit Cancer Center Daniel Sargent Ph.D. Mayo Clinic Cancer Center Richard M. Goldberg M.D. University of North Carolina Lineberger Comprehensive Cancer Center
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Aging, Frailty, and Medical Therapy for Colorectal Cancer: Who Should Be Treated and How?
Aging, Frailty, and Medical Therapy for Colorectal Cancer: Who Should Be Treated and How?. Martine Extermann M.D., Ph.D. Moffit Cancer Center Daniel Sargent Ph.D. Mayo Clinic Cancer Center Richard M. Goldberg M.D. University of North Carolina Lineberger Comprehensive Cancer Center. - PowerPoint PPT Presentation
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Aging, Frailty, and Medical Therapy for Colorectal Cancer: Who Should Be Treated and How?
Martine Extermann M.D., Ph.D.Moffit Cancer Center
Daniel Sargent Ph.D.Mayo Clinic Cancer Center
Richard M. Goldberg M.D.University of North Carolina
Lineberger Comprehensive Cancer Center
Elderly and/ or frail patients with colorectal cancer - a clinician's approach
• Limited numbers of patients >75 are accrued to Phase II or III studies
• These patients are carefully selected• There is only so much data out there• We need other sources of information
Comparative Effectiveness Research
Health Care Quality
• IOM:– The degree to which health services for individuals
and populations increase the likelihood of desired health outcomes and are consistent with current professional knowledge.
– Underuse, overuse, misuse
Outcomes – Comparative Effectiveness
• Comparative effectiveness research (CER) is the generation and synthesis of evidence that compares the benefits and harms of alternative methods to prevent, diagnose, treat, and monitor a clinical condition or to improve the delivery of care.
• The purpose of CER is to assist consumers, clinicians, purchasers, and policy makers to make informed decisions that will improve health care at both the individual and population levels.
(IOM, 2009)
Federal Funding Mechanisms
• AHRQ:– Agency for Health Research and Quality Quality
• improvement and patient safety.• Outcomes and effectiveness of care.• Clinical practice and technology assessment.• Health care organization and delivery systems. • Primary care (including preventive services).• Health care costs and sources of payment.
AHRQ Cancer Comparative Effectiveness
• Medicare Modernization Act: – “…conduct research to improve the quality, effectiveness, and efficiency of
Medicare, Medicaid, and State Children Health Insurance (SCHIP) programs.”
• Increasing emphasis on patient-level attributes (rather than “the average patient”) that may modify the balance of benefits or harms can lead to more personalized medicine, reducing the pressure to try alternatives found to be ineffective in similar subgroups.
• DEcIDE : Decisions about effectiveness research : – Expeditiously develop valid scientific evidence about the outcomes,
comparative clinical effectiveness, safety, and appropriateness of health care items and services
AHRQ Cancer DEcIDE Comparative Effectiveness
• Clinical trials– Relatively homogeneous population
• Younger, healthier, more likely Caucasian– Randomization to control for unmeasured (and
unmeasurable) heterogeneity
• CER– Examinations prioritizing the context of heterogeneity
• Better representativeness• Examination of subpopulations
ExamplesStage II/III Colorectal cancer Chemotherapy
Trials– NEJM, 2004– JCO, 2007
vs.
SEER:
II III IVMean Age 71.4 69.0 67.7
Race White 83.7% 81.2% 79.3% Black 9.0% 10.6% 13.5% Other/Unk 7.4% 8.3% 7.2%
Gender Female 52% 52% 51% Male 48% 48% 49%
*Source: SEER, 2004-2005 data.
Stage at Diagnosis
Generalizable, results by sub-population
NCDB study: n=86,000; hospitals=560(Jessup et al, JAMA, 2005)
CER and Outcomes Research
• CER has value, maximizing our understanding with observational data– Fast– Inexpensive– Can be very large databases
• CER will not replace clinical trials• Larger future of outcomes research: Moving
from studies of “what” to understanding “why”
CER and Outcomes Research:Emerging Directions
• Application of advanced methods using secondary data, including the development of new methods– AHRQ-sponsored White papers:
• “Registries for Evaluating Patient Outcomes”– July 2009 DEcIDE RFTO (~$500,000 x 1 year):
• “Methods to Study the Heterogeneity of Treatment Effects in Comparative Effectiveness Research”
– Fall 2009: 10 x $10 million awards• Clinical and Health Outcomes Initiative in Comparative
Effectiveness
CER and Outcomes Research:Emerging Directions
• Development and application of advanced data– Developing new data sources
• Retrospective studies• Prospective studies
– Fall 2009: $48 million• New Registries for CER
• Data needs:– Sample size, generalizability of claims-based studies– Richness, depth of measures of survey / interview-based
studies– Clinical detail, follow-up of registries
Why new data and models?A prevailing model of cancer, comorbidity, and outcomes:
• Current models are linear, simply specified, and fairly simple• Randomization controls for many relevant factors• Intent-to-treat is dominant
Source: Geraci, JM, et al. (2005). “Comorbid Disease and Cancer: The Need for More Relevant Conceptual Models in Health Services Research.” Journal of Clinical Oncology. 23(30):7399-404.
Why new data and models?
• Because its just not that simple• We are increasingly interested in other outcomes,
including Patient Reported Outcomes (PROS)• With observational data, you can’t randomize-out
confounders and effect modifiers– Before we can make assumptions with them, we need to study
them• Need new data that allow rich characterization of factors
at multiple levels, with a substantial sample size for generalizability of findings and sub-population analysis
What Data Sources Might We Use to Evaluate Effectiveness?
1. SEER-Medicare
2. NYSCR/CCR-Medicaid
3. CanCORS
4. NCCN
– SEER • For purposes of anchoring comparison for overall mortality
Conclusions
• Elderly colorectal cancer patients are underrepresented in clinical trials
• Surgery (including hepatic resections) and RT for rectal cancers can be done safely and effectively but leads to higher early death rates
• CER can help answer some of the questions in this subpopulation