With ever-rising healthcare costs, evidence generation through Health Economics and Outcomes Research (HEOR) plays an increasingly important role in decision-making about the allocation of resources. Accordingly, it is now customary for health technology assessment and reimbursement agencies to request for HEOR evidence, in addition to data from clinical trials, to inform decisions about patient access to new treatment options.
While there is a great deal of literature on HEOR, there is a need for a volume that presents a coherent and unified review of the major issues that arise in application, especially from a statistical perspective. Statistical Topics in Health Economics and Outcomes Research fulfils that need by presenting an overview of the key analytical issues and best practice. Special attention is paid to key assumptions and other salient features of statistical methods customarily used in the area, and appropriate and relatively comprehensive references are made to emerging trends. The content of the book is purposefully designed to be accessible to readers with basic quantitative backgrounds, while providing an in-depth coverage of relatively complex statistical issues.
The book will make a very useful reference for researchers in the pharmaceutical industry, academia, and research institutions involved with HEOR studies. The targeted readers may include statisticians, data scientists, epidemiologists, outcomes researchers, health economists, and healthcare policy and decision-makers.
Table of Contents
1. Data Sources for Health Economics and Outcomes Research
Kelly H. Zou, Christine L. Baker, Joseph C. Cappelleri, and Richard B. Chambers
2. Patient-Reported Outcomes: Development and Validation
Joseph C. Cappelleri, Andrew G. Bushmakin, and Jose Ma. J. Alvir
3. Observational Data Analysis
Demissie Alemayehu, Marc Berger, Vitalii Doban, and Jack Mardekian
4. Predictive Modeling in HEOR
Birol Emir, David C. Gruben, Helen T. Bhattacharyya, Arlene L. Reisman, and Javier Cabrera
5. Methodological Issues in Health Economic Analysis
Demissie Alemayehu, Thomas Mathew, and Richard J. Willke
6. Analysis of Aggregate Data
Demissie Alemayehu, Andrew G. Bushmakin, and Joseph C. Cappelleri
7. Health Economics and Outcomes Research in Precision Medicine
Demissie Alemayehu, Joseph C. Cappelleri, Birol Emir, and Josephine Sollano
8. Best Practices for Conducting and Reporting Health Economics and Outcomes Research
Kelly H. Zou, Joseph C. Cappelleri, Christine L. Baker, and Eric C. Yan
Demissie Alemayehu, PhD, is Vice President and Head of the Statistical Research and Data Science Center at Pfizer Inc. He earned his Ph.D. in Statistics from the University of California at Berkeley. He is a Fellow of the American Statistical Association, has published widely, and has served on the editorial boards of major journals, including the Journal of the American Statistical Association and the Journal of Nonparametric Statistics. Additionally, he has been on the faculties of both Columbia University and Western Michigan University. He has co-authored Patient-Reported Outcomes: Measurement, Implementation and Interpretation, published by Chapman & Hall/CRC Press.
Joseph C. Cappelleri, PhD, MPH, MS is Executive Director in the Statistical Research and Data Science Center at Pfizer Inc. He earned his M.S. in Statistics from the City University of New York, Ph.D. in Psychometrics from Cornell University, and M.P.H. in Epidemiology from Harvard University. As an adjunct professor, he has served on the faculties of Brown University, University of Connecticut, and Tufts Medical Center. He has delivered numerous conference presentations and has published extensively on clinical and methodological topics, including regression-discontinuity designs, meta-analyses, and health measurement scales. He is lead author of the monograph Patient-Reported Outcomes: Measurement, Implementation and Interpretation. He is a Fellow of the American Statistical Association.
Birol Emir, PhD, is Senior Director and Statistics Lead in the Statistical Research and Data Science Center at Pfizer Inc. In addition, he is an Adjunct Professor of Statistics at Columbia University in New York and External PhD Committee Member in the Graduate School of Arts and Sciences at Rutgers, The State University of New Jersey. Recently, his primary focuses have been on big data, predictive modeling and genomics data analysis. He has numerous publications in refereed journals and authored a book chapter in A Picture Is Worth a Thousand Tables: Graphics in Life Sciences. He has taught several short courses and has given invited presentations.
Kelly H. Zou, PhD, PStat®, is Senior Director and Analytic Science Lead at Pfizer Inc. She is a Fellow of the American Statistical Association and an Accredited Professional Statistician. She has published extensively on clinical and methodological topics. She has served on the editorial board of Significance, as an Associate Editor for Statistics in Medicine and Radiology, and as a Deputy Editor for Academic Radiology. She was Associate Professor of Radiology, Director of Biostatistics, and Lecturer of Health Care Policy at Harvard Medical School. She was Associate Director of Rates at Barclays Capital. She has co-authored Statistical Evaluation of Diagnostic Performance: Topics in ROC Analysis and Patient-Reported Outcomes: Measurement, Implementation and Interpretation, both published by Chapman and Hall/CRC. She authored a book chapter in Leadership and Women in Statistics by the same publisher. She was the theme editor on statistics books entitled Mathematical and Statistical Methods for Diagnoses and Therapies.