In recent years the number of innovative medicinal products and devices submitted and approved by regulatory bodies has declined dramatically. The medical product development process is no longer able to keep pace with increasing technologies, science and innovations and the goal is to develop new scientific and technical tools and to make product development processes more efficient and effective. Statistical Methods in Healthcare focuses on the application of statistical methodologies to evaluate promising alternatives and to optimize the performance and demonstrate the effectiveness of those that warrant pursuit is critical to success. Statistical methods used in planning, delivering and monitoring health care, as well as selected statistical aspects of the development and/or production of pharmaceuticals and medical devices are also addressed.
With a focus on finding solutions to these challenges, this book:
* Provides a comprehensive, in-depth treatment of statistical methods in healthcare, along with a reference source for practitioners and specialists in health care and drug development.
* Offers a broad coverage of standards and established methods through leading edge techniques.
* Uses an integrated, case-study based approach, with focus on applications.
* Looks at the use of analytical and monitoring schemes to evaluate therapeutic performance.
* Features the application of modern quality management systems to clinical practice, and to pharmaceutical development and production processes.
* Addresses the use of modern Statistical methods such as Adaptive Design, Seamless Design, Data Mining, Bayesian networks and Bootstrapping that can be applied to support the challenging new vision.
Practitioners in healthcare-related professions, ranging from clinical trials to care delivery to medical device design, as well as statistical researchers in the field, will benefit from this book.
Foreword xix
Preface xxi
Editors xxiii
Contributors xxv
Part One STATISTICS IN THE DEVELOPMENT OF PHARMACEUTICAL PRODUCTS
1 Statistical Aspects in ICH, FDA and EMA Guidelines 3
Allan Sampson and Ron S. Kenett
Synopsis 3
References 19
2 Statistical Methods in Clinical Trials 22
Telba Irony, Caiyan Li and Phyllis Silverman
Synopsis 22
References 54
Further Reading 54
3 Pharmacometrics in Drug Development 56
Serge Guzy and Robert Bauer
Synopsis 56
4 Interactive Clinical Trial Design 78
Zvia Agur
Synopsis 78
Adaptive Clinical Trial Design Methods 99
Acknowledgements 100
References 100
5 Stage-wise Clinical Trial Experiments in Phases I, II and III 103
Shelemyahu Zacks
Synopsis 103
References 120
6 Risk Management in Drug Manufacturing and Healthcare 122
Ron S. Kenett
Synopsis 122
References 152
7 The Twenty-first Century Challenges in Drug Development 155
Yafit Stark
Synopsis 155
References 166
Part Two STATISTICS IN OUTCOMES ANALYSIS
8 The Issue of Bias in Combined Modelling and Monitoring of Health Outcomes 169
Olivia A. J. Grigg
Synopsis 169
References 182
9 Disease Mapping 185
Annibale Biggeri and Dolores Catelan
Synopsis 185
References 210
10 Process Indicators and Outcome Measures in the Treatment of Acute Myocardial Infarction Patients 219
Alessandra Guglielmi, Francesca Ieva, Anna Maria Paganoni and Fabrizio Ruggeri
Synopsis 219
References 228
11 Meta-analysis 230
Eva Negri
Synopsis 230
References 247
Part Three STATISTICAL PROCESS CONTROL IN HEALTHCARE
12 The Use of Control Charts in Healthcare 253
William H. Woodall, Benjamin M. Adams and James C. Benneyan
Synopsis 253
Acknowledgements 265
References 265
13 Common Challenges and Pitfalls Using SPC in Healthcare 268
Victoria Jordan and James C. Benneyan
Synopsis 268
References 285
14 Six Sigma in Healthcare 286
Shirley Y. Coleman
Synopsis 286
References 307
15 Statistical Process Control in Clinical Medicine 309
Per Winkel and Nien Fan Zhang
Synopsis 309
Appendix A 328
15.A.1 The EWMA Chart 328
15.A.2 Logistic Regression 329
15.A.3 Autocovariance and Autocorrelation 330
Acknowledgements 330
References 330
Part Four APPLICATIONS TO HEALTHCARE POLICY AND IMPLEMENTATION
16 Modeling Kidney Allocation: A Data-driven Optimization Approach 335
Inbal Yahav
Synopsis 335
Acknowledgement 352
References 352
17 Statistical Issues in Vaccine Safety Evaluation 353
Patrick Musonda
Synopsis 353
References 362
18 Statistical Methods for Healthcare Economic Evaluation 365
Caterina Conigliani, Andrea Manca and Andrea Tancredi
Synopsis 365
References 383
19 Costing and Performance in Healthcare Management 386
Rosanna Tarricone and Aleksandra Torbica
Synopsis 386
References 403
Part Five APPLICATIONS TO HEALTHCARE MANAGEMENT
20 Statistical Issues in Healthcare Facilities Management 407
Daniel P. O'Neill and Anja Drescher
Synopsis 407
References 425
21 Simulation for Improving Healthcare Service Management 426
Anne Shade
Synopsis 426
References 444
22 Statistical Issues in Insurance/payor Processes 445
Melissa Popkoski
Synopsis 445
Reference 455
23 Quality of Electronic Medical Records 456
Dario Gregori and Paola Berchialla
Synopsis 456
References 475
Index 481
* Provides a comprehensive, in-depth treatment of statistical methods in healthcare. * Presents a reference source for practitioners and specialists in health care and drug development. * Offers a broad coverage of standards and established methods through leading edge techniques.
In recent years the number of innovative medicinal products and devices submitted and approved by regulatory bodies has declined dramatically. The medical product development process is no longer able to keep pace with increasing technologies, science and innovations and the goal is to develop new scientific and technical tools and to make product development processes more efficient and effective. Statistical Methods in Healthcare focuses on the application of statistical methodologies to evaluate promising alternatives and to optimize the performance and demonstrate the effectiveness of those that warrant pursuit is critical to success. Statistical methods used in planning, delivering and monitoring health care, as well as selected statistical aspects of the development and/or production of pharmaceuticals and medical devices are also addressed.
With a focus on finding solutions to these challenges, this book:
* Provides a comprehensive, in-depth treatment of statistical methods in healthcare, along with a reference source for practitioners and specialists in health care and drug development.
* Offers a broad coverage of standards and established methods through leading edge techniques.
* Uses an integrated, case-study based approach, with focus on applications.
* Looks at the use of analytical and monitoring schemes to evaluate therapeutic performance.
* Features the application of modern quality management systems to clinical practice, and to pharmaceutical development and production processes.
* Addresses the use of modern Statistical methods such as Adaptive Design, Seamless Design, Data Mining, Bayesian networks and Bootstrapping that can be applied to support the challenging new vision.
Practitioners in healthcare-related professions, ranging from clinical trials to care delivery to medical device design, as well as statistical researchers in the field, will benefit from this book.
Foreword xix
Preface xxi
Editors xxiii
Contributors xxv
Part One STATISTICS IN THE DEVELOPMENT OF PHARMACEUTICAL PRODUCTS
1 Statistical Aspects in ICH, FDA and EMA Guidelines 3
Allan Sampson and Ron S. Kenett
Synopsis 3
References 19
2 Statistical Methods in Clinical Trials 22
Telba Irony, Caiyan Li and Phyllis Silverman
Synopsis 22
References 54
Further Reading 54
3 Pharmacometrics in Drug Development 56
Serge Guzy and Robert Bauer
Synopsis 56
4 Interactive Clinical Trial Design 78
Zvia Agur
Synopsis 78
Adaptive Clinical Trial Design Methods 99
Acknowledgements 100
References 100
5 Stage-wise Clinical Trial Experiments in Phases I, II and III 103
Shelemyahu Zacks
Synopsis 103
References 120
6 Risk Management in Drug Manufacturing and Healthcare 122
Ron S. Kenett
Synopsis 122
References 152
7 The Twenty-first Century Challenges in Drug Development 155
Yafit Stark
Synopsis 155
References 166
Part Two STATISTICS IN OUTCOMES ANALYSIS
8 The Issue of Bias in Combined Modelling and Monitoring of Health Outcomes 169
Olivia A. J. Grigg
Synopsis 169
References 182
9 Disease Mapping 185
Annibale Biggeri and Dolores Catelan
Synopsis 185
References 210
10 Process Indicators and Outcome Measures in the Treatment of Acute Myocardial Infarction Patients 219
Alessandra Guglielmi, Francesca Ieva, Anna Maria Paganoni and Fabrizio Ruggeri
Synopsis 219
References 228
11 Meta-analysis 230
Eva Negri
Synopsis 230
References 247
Part Three STATISTICAL PROCESS CONTROL IN HEALTHCARE
12 The Use of Control Charts in Healthcare 253
William H. Woodall, Benjamin M. Adams and James C. Benneyan
Synopsis 253
Acknowledgements 265
References 265
13 Common Challenges and Pitfalls Using SPC in Healthcare 268
Victoria Jordan and James C. Benneyan
Synopsis 268
References 285
14 Six Sigma in Healthcare 286
Shirley Y. Coleman
Synopsis 286
References 307
15 Statistical Process Control in Clinical Medicine 309
Per Winkel and Nien Fan Zhang
Synopsis 309
Appendix A 328
15.A.1 The EWMA Chart 328
15.A.2 Logistic Regression 329
15.A.3 Autocovariance and Autocorrelation 330
Acknowledgements 330
References 330
Part Four APPLICATIONS TO HEALTHCARE POLICY AND IMPLEMENTATION
16 Modeling Kidney Allocation: A Data-driven Optimization Approach 335
Inbal Yahav
Synopsis 335
Acknowledgement 352
References 352
17 Statistical Issues in Vaccine Safety Evaluation 353
Patrick Musonda
Synopsis 353
References 362
18 Statistical Methods for Healthcare Economic Evaluation 365
Caterina Conigliani, Andrea Manca and Andrea Tancredi
Synopsis 365
References 383
19 Costing and Performance in Healthcare Management 386
Rosanna Tarricone and Aleksandra Torbica
Synopsis 386
References 403
Part Five APPLICATIONS TO HEALTHCARE MANAGEMENT
20 Statistical Issues in Healthcare Facilities Management 407
Daniel P. O'Neill and Anja Drescher
Synopsis 407
References 425
21 Simulation for Improving Healthcare Service Management 426
Anne Shade
Inhaltsverzeichnis
Foreword xix
Preface xxi
Editors xxiii
Contributors xxv
Part One STATISTICS IN THE DEVELOPMENT OF PHARMACEUTICAL PRODUCTS
1 Statistical Aspects in ICH, FDA and EMA Guidelines 3
Allan Sampson and Ron S. Kenett
Synopsis 3
References 19
2 Statistical Methods in Clinical Trials 22
Telba Irony, Caiyan Li and Phyllis Silverman
Synopsis 22
References 54
Further Reading 54
3 Pharmacometrics in Drug Development 56
Serge Guzy and Robert Bauer
Synopsis 56
4 Interactive Clinical Trial Design 78
Zvia Agur
Synopsis 78
Adaptive Clinical Trial Design Methods 99
Acknowledgements 100
References 100
5 Stage-wise Clinical Trial Experiments in Phases I, II and III 103
Shelemyahu Zacks
Synopsis 103
References 120
6 Risk Management in Drug Manufacturing and Healthcare 122
Ron S. Kenett
Synopsis 122
References 152
7 The Twenty-first Century Challenges in Drug Development 155
Yafit Stark
Synopsis 155
References 166
Part Two STATISTICS IN OUTCOMES ANALYSIS
8 The Issue of Bias in Combined Modelling and Monitoring of Health Outcomes 169
Olivia A. J. Grigg
Synopsis 169
References 182
9 Disease Mapping 185
Annibale Biggeri and Dolores Catelan
Synopsis 185
References 210
10 Process Indicators and Outcome Measures in the Treatment of Acute Myocardial Infarction Patients 219
Alessandra Guglielmi, Francesca Ieva, Anna Maria Paganoni and Fabrizio Ruggeri
Synopsis 219
References 228
11 Meta-analysis 230
Eva Negri
Synopsis 230
References 247
Part Three STATISTICAL PROCESS CONTROL IN HEALTHCARE
12 The Use of Control Charts in Healthcare 253
William H. Woodall, Benjamin M. Adams and James C. Benneyan
Synopsis 253
Acknowledgements 265
References 265
13 Common Challenges and Pitfalls Using SPC in Healthcare 268
Victoria Jordan and James C. Benneyan
Synopsis 268
References 285
14 Six Sigma in Healthcare 286
Shirley Y. Coleman
Synopsis 286
References 307
15 Statistical Process Control in Clinical Medicine 309
Per Winkel and Nien Fan Zhang
Synopsis 309
Appendix A 328
15.A.1 The EWMA Chart 328
15.A.2 Logistic Regression 329
15.A.3 Autocovariance and Autocorrelation 330
Acknowledgements 330
References 330
Part Four APPLICATIONS TO HEALTHCARE POLICY AND IMPLEMENTATION
16 Modeling Kidney Allocation: A Data-driven Optimization Approach 335
Inbal Yahav
Synopsis 335
Acknowledgement 352
References 352
17 Statistical Issues in Vaccine Safety Evaluation 353
Patrick Musonda
Synopsis 353
References 362
18 Statistical Methods for Healthcare Economic Evaluation 365
Caterina Conigliani, Andrea Manca and Andrea Tancredi
Synopsis 365
References 383
19 Costing and Performance in Healthcare Management 386
Rosanna Tarricone and Aleksandra Torbica
Synopsis 386
References 403
Part Five APPLICATIONS TO HEALTHCARE MANAGEMENT
20 Statistical Issues in Healthcare Facilities Management 407
Daniel P. O'Neill and Anja Drescher
Synopsis 407
References 425
21 Simulation for Improving Healthcare Service Management 426
Anne Shade
Synopsis 426
References 444
22 Statistical Issues in Insurance/payor Processes 445
Melissa Popkoski
Synopsis 445
Reference 455
23 Quality of Electronic Medical Records 456
Dario Gregori and Paola Berchialla
Synopsis 456
References 475
Index 481
Klappentext
In recent years the number of innovative medicinal products and devices submitted and approved by regulatory bodies has declined dramatically. The medical product development process is no longer able to keep pace with increasing technologies, science and innovations and the goal is to develop new scientific and technical tools and to make product development processes more efficient and effective. Statistical Methods in Healthcare focuses on the application of statistical methodologies to evaluate promising alternatives and to optimize the performance and demonstrate the effectiveness of those that warrant pursuit is critical to success. Statistical methods used in planning, delivering and monitoring health care, as well as selected statistical aspects of the development and/or production of pharmaceuticals and medical devices are also addressed.
With a focus on finding solutions to these challenges, this book:
* Provides a comprehensive, in-depth treatment of statistical methods in healthcare, along with a reference source for practitioners and specialists in health care and drug development.
* Offers a broad coverage of standards and established methods through leading edge techniques.
* Uses an integrated, case-study based approach, with focus on applications.
* Looks at the use of analytical and monitoring schemes to evaluate therapeutic performance.
* Features the application of modern quality management systems to clinical practice, and to pharmaceutical development and production processes.
* Addresses the use of modern Statistical methods such as Adaptive Design, Seamless Design, Data Mining, Bayesian networks and Bootstrapping that can be applied to support the challenging new vision.
Practitioners in healthcare-related professions, ranging from clinical trials to care delivery to medical device design, as well as statistical researchers in the field, will benefit from this book.
