THE SECTION ON Evidence Based Health Care OF THE NEW YORK ACADEMY OF MEDICINE 2014 TEACH Workshop Simple. Scientifically Informed Medical Practice and Learning Suzana Alves da Silva, MD, MSc, PhD, FNYAM
Clinical Problems P I C O What we know? Chatterji, M; Graham, M; Wyer, P. Mapping Cognitive Overlaps Between Practice-Based Learning and Improvement and Evidence-Based Medicine: An Operational Definition for Assessing Resident Physician Competence.2009
Frequency of outcome Prediction of outcome Impact on outcome What is new? Silva, S and Wyer P. The Roadmap: a blueprint for evidence literacy within a Scientifically Informed Medical Practice and Learning Model. 2012
Likelihood issue on therapy This scenario converges on an issue of the absolute probability of survival given maximal, evidence-based, interventions. An elderly man is brought to the emergency room from a nursing home. He is febrile, hypotensive, anuric, minimally responsive. Medical and nursing staffs are preparing to implement an advanced protocol for sepsis care when the patient’s daughter arrives. The daughter wants to know “So, if you give him the most intense treatment, how likely is it that he will survive? It doesn’t make sense if he is likely to die anyway!”
Utility issue on prognosis This scenario involves a trade-off between risks versus benefits of sharing the results of a prognostic information. A 40 year old woman is seeing her primary care physician. Her mother has recently been diagnosed with Alzheimer’s disease. She is concerned about the possibility that it could happen to her. The physician advises that there is a genetic test that could eliminate that likelihood. However, the woman expresses ambivalence, saying, “I am not sure I want to know.”
Performance issue on therapy This scenario involves predicting the likelihood of benefit from a treatment if the predictor is present. It calls for information regarding the predictive performance of criteria such as a biomarker in modifying the probability of benefit from a therapeutic option. A medical director of a health care organization is considering inclusion of BRCA1 within a panel of molecular biomarkers for optimization of individualized care of women with diagnosed breast cancer with respect to hormonal therapy, immune-therapy or chemo-therapy. His first question is what is the accuracy of BRCA1 mutation to predict that a women with cancer will in fact respond to a specific therapeutic regimen.
Levels of efficacy of Dx imaging tests Phases of novel cardiac markers diagnostic accuracy, sensitivity, and specificity associated with interpretation of the test. whether the information produces change in the referring physician’s diagnostic thinking. effect of the information on the patient management plan and on patient outcomes. societal costs and benefits of a diagnostic imaging technology. Performance Utility Likelihood Performance Utility • 1. Proof of concept—Do novel marker levels differ between subjects with and without outcome? • 2. Prospective validation—Does the novel marker predict development of future outcomes? • 4. Clinical utility—Does the novel risk marker change predicted risk sufficiently to change recommended therapy and to improve clinical outcomes? • 6. Cost-effectiveness—Does use of the marker justify additional costs of testing and treatment? Fryback and Thornbury. 1991 Hlatky. AHA 2009
“My doctors estimated that I had an 87 percent risk of breast cancer and a 50 percent risk of ovarian cancer… Once I knew that this was my reality, I decided to be proactive and to minimize the risk as much as I could.”
HMOs being pressured for the inclusion of BRCA mutation testing for screening of women with high risk of breast cancer. Chief Medical Executive Physician Healthy Patient Many patients with family history of cancer want to be tested for BRCA mutation. Many wonder about preventive surgery or chemotherapy on top of surveillance. Many primary care physicians are ordering BRCA mutation testing to their patients or referring them for genetic counseling without knowing exactly what to do with it. Scenario
Breast Cancer || Ovarian Cancer Women in general Preventive surgery will drop the risk of cancer by ~ 90% Little info about the effect of chemoprevention or close surveillance Little info about impact on death Source: 1. Senkus. European Society of Medical Oncology.2014. 2. Risk Assessment, Genetic Counseling, and Genetic Testing for BRCA-Related Cancer: U.S. Preventive Services Task Force Recommendation. 2013. 3. Berliner. National Society of Genetic Counselors 2013
Why this is so important? Recognition of questions of importance to patients, managers and policy makers in a fashion that facilitates the use of clinical research to inform the corresponding decisions. Provide guidance regarding how to explore and interpret patient and stakeholder expressions of the concerns that will maximally inform decisions. Categorization of clinical research designs in relationship to different types of clinical questions in a way that embraces the full spectrum of clinically relevant research.
Therapy Utility Therapy Frequency
Prognosis Utility Prognosis Frequency