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” The American Journal of Bioethics 2020 168 Epilogue 172 Part III ♥ Statistical inclusion of expanded access data 178 Prologue 180 Chapter 9 Augmenting treatment arms with external data through propensity-score weighted power-priors with an application in expanded access Statistics in Medicine: Under Revision. Preprint: Arxiv 2023 184 Part IV ♠ Policy implications and ethical considerations 216 Prologue 218 Chapter 10 The DRUG Access Protocol: access inequality and European harmonization Lancet Oncology 2022 220 Chapter 11 Generating Evidence from Expanded Access Use of Rare Disease Medicines: Challenges and Recommendations Frontiers in Pharmacology 2022 224 Chapter 12 The ethics of expanded access research JAMA 2023 236 Postlude 246 Summary 248 Discussion 253 Appendices 264 Nederlandse samenvatting 266 Bibliography 272 Portfolio 275 About the author 279 Acknowledgements (dankwoord) 280
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