Using Real World Data (RWD) to Assess the Value of New Technology for Patients April 12, 2012 Diana Brixner, RPh, PhD Professor and Chair, College of Pharmacy Director, Pharmacotherapy Outcomes Research Center firstname.lastname@example.org
Key Points • Definition of Value • The pros and cons of Real World Data • Development of Patient-Centered Retrospective Research Registries (PCR3s) within our system • Application to value assessment of new technology
Cost VALUE Outcome UHC Technology ReimbursementConsiderations under HCR • Improvinghealthcareefficiency • Improving health care quality • Allocation of scare healthcare resources ═
Information is Needed Beyond RCTs . . . Efficacy and safety in a small population with a restricted study protocol RCT Randomized Clinical Trials GAP Patient Population Real world information to make health care decisions for large populations within defined budgets Real World Data Garrison LP Jr, Neumann PJ, Erickson P, Marshall D, Mullins CD. Using real-world data for coverage and payment decisions: the ISPOR Real- World Data Task Force report. Value Health. 2007;10(5):326-35.
Efficacy vs. Effectiveness • Efficacy • RCT • High internal validity • Limited generalizability • Effectiveness • Observational studies • High external validity • Lack of Controls Holtorf AP, Watkins JB, Mullins CD, Brixner D. Incorporating obser- vational data into the formulary decision-making process-summary of a roundtable discussion. J Manag Care Pharm. 2008;14(3):302-08.
UU Patient Centered Retrospective Research Registries Collaboration with University of Utah: Information Technology and Bioinformatics Utah Population Database HCI/UHOSP investigators and clinicians Enterprise Data Warehouse (EDW) Contain longitudinal data on patient cohorts from 1995 to current including clinical, survival and charge data. Used to develop models to predict cost-effective outcomes of new pharmaceuticals and diagnostic tests
4 Cancer Cohorts in Development • Cohort for HCC, Breast Cancer and CML already completed • Prostate cancer cohort is next planned cohort for development • Developing one for Atrial Fibrillation in collaboration with CARMA
We can determine survival by stage… Stage I Stage II Stage III Stage IV
We can determine charges by stage… * * Inpatient and outpatient charges for one year post staging across the Huntsman Cancer Institute and the University Healthcare system
The Promise of Personalized Medicine • Pharmacogenomic tests can fine tune treatment pathways for patients with breast cancer after surgery • Stratify patients as to whether they should receive treatment based on risk • Stratify patients by which treatment would provide the best response • However these tests come at a cost that need to be weighed against the benefit they can bring to patients
The Value of Personalized Medicine 75 Chemo $10,000 Not Tested 90% 5 year survival $750/patient $0 25 No Chemo $0 40 Chemo 100 women after breast cancer surgery $10,000 Tested 90% 5 year survival $8100/patient $4100 60 No Chemo $0 • Assist researchers in development • Assist payers in reimbursement decisions • Assist patients in treatment decisions
Summary • Development of Patient-Centered Retrospective Research Registries (PCR3s) are in process within our system • These PCR3s can be applied to value assessment of new technology for Health Care Reform