TABLE OF CONTENTS Prelude 08 Introduction Background and outline of this thesis 10 Chapter 1 Data collection in expanded access: real-world examples Pritelivir for treatment-resistant herpes simplex infections Poster at SSAI/ICHS 2022 and Oral at STI & HIV 2023 (adapted) Tabelecleucel for Epstein-Barr Virus-driven diseases Poster at EHA 2022 and ASCO 2022 (adapted) 20 Chapter 2 Expanded access in the Netherlands: prescribing unregistered medicine Nederlands Tijdschrift voor Geneeskunde 2021 30 Part I ♣ The clinical merit of expanded access 44 Prologue 47 Chapter 3 The clinical value of drugs under development in hematological The predictive value of a positive phase II ASH abstract for peerreviewed publication and progression to phase III Blood 2022 (adapted) Two decades of targeted therapies in acute myeloid leukemia Leukemia 2021 (adapted) 48 Chapter 4 Incremental benefits of novel pharmaceuticals in the United Kingdom: A cross-sectional analysis of NICE technology appraisals from 2010 – 2020 BMJ Open 2022 56 Epilogue 74 Part II ♦ The usage of expanded access data 78 Prologue 80 Chapter 5 Expanded access as a source of real-world data: An overview of FDA and EMA approvals British Journal of Clinical Pharmacology 2020 82 Chapter 6 Real-world data from expanded access programs in health technology assessments: a review of NICE technology appraisals BMJ Open 2022 100 Chapter 7 Results from expanded access programs: a review of academic literature Drugs 2023 124
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