I 285

Tobias Polak

Introduction 10
Chapter 1 Data collection in expanded access: real-world examples Pritelivir for treatment-resistant herpes simplex infections 20
Chapter 2 Expanded access in the Netherlands: prescribing unregistered medicine 30
Chapter 3 The clinical value of drugs under development in hematological The predictive value of a positive phase II ASH abstract for peer reviewed publication and progression to phase III 48
Chapter 4 Incremental benefits of novel pharmaceuticals in the United Kingdom: A cross-sectional analysis of NICE technology appraisals from 2010 – 2020 56
Chapter 5 Expanded access as a source of real-world data: An overview of FDA and EMA approvals 82
Chapter 6 Real-world data from expanded access programs in health technology assessments: a review of NICE technology appraisals 100
Chapter 7 Results from expanded access programs: a review of academic literature 124
Chapter 8 Data collection for the sake of data collection Response to Open Peer Commentaries on “Making It Count: Extracting Real World Data from Compassionate Use and Expanded Access Programs” 168
Chapter 9 Augmenting treatment arms with external data through propensity-score weighted power-priors with an application in expanded access 184
Chapter 10 The DRUG Access Protocol: access inequality and European harmonization 220
Chapter 11 Generating Evidence from Expanded Access Use of Rare Disease Medicines: Challenges and Recommendations 224
Chapter 12 The ethics of expanded access research 236
Summary 248
Discussion 253
Nederlandse samenvatting 266
Bibliography 272
Portfolio 275
About the author 279
Acknowledgements (dankwoord) 280

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