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CAR T-Cell Therapy Horizon Scanning CAR T-Cell Therapy Horizon Scanning EBEU Report No. 11/2020 AIHTA Policy Brief No. 006a CAR T-Cell Therapy Horizon Scanning EBEU Report No. 11/2020 AIHTA Policy Brief No. 006a Innsbruck, August 2020 Project Team Project leader: Dr. MMag. Sabine Geiger-Gritsch, Evidenzbasierte Entscheidungsunterstützung (EBEU) - Medizinisches Projektmanagement, Tirol Kliniken GmbH, Innsbruck Project support: Univ.-Prof. Dr. Christian J. Wiedermann, Medizinischer Geschäftsführer, Tirol Kliniken GmbH, Innsbruck External Review: PD Dr. Claudia Wild, HTA Austria - Austrian Institute for Health Technology Assessment GmbH, Vienna Correspondence: Sabine Geiger-Gritsch, [email protected] Conflict of Interest All authors declared they have no conflicts of interest according to the Uniform Requirements of Manuscripts Statement of Medical Journal Editors (www.icmje.org). This report should be referenced as follows: Geiger-Gritsch S, Wiedermann C. CAR T-Cell Therapy, Horizon Scanning. EBEU Report No. 11/2020; AIHTA Policy Brief No. 6a/2020, Innsbruck/Vienna. Tirol Kliniken GmbH Innsbruck in Collaboration with the Austrian Institute for Health Technology Assessment GmbH Die AIHTA Policy Briefs erscheinen unregelmässig und dienen der Veröffentlichung der Forschungsergebnisse des Austrian Institute for Health Technology Assessment. Die AIHTA Policy Briefs erscheinen in geringer Auflage im Druck und werden über den Dokumentenserver "https://eprints.aihta.at/view/types/policy=5Fbrief.html" der Öffentlichkeit zur Verfügung gestellt AIHTA Policy Brief Nr.: 006a ISSN 2710-3234 ISSN online 2710-3242 1 Content List of Figures ....................................................................................................................................... 3 List of Tables ........................................................................................................................................ 3 List of Abbreviations ............................................................................................................................ 4 Deutsche Zusammenfassung ................................................................................................................... 6 1 Introduction ........................................................................................................................................ 10 Cancer Immunotherapy & CAR T-cells ............................................................................................... 10 CAR T-cell production for clinical application .................................................................................... 13 Regulatory requirements ................................................................................................................... 14 2 Scope .................................................................................................................................................. 15 3 Methods ............................................................................................................................................. 16 Approach ........................................................................................................................................... 16 Search strategy .................................................................................................................................. 16 Eligibility Criteria ............................................................................................................................... 17 Data extraction .................................................................................................................................. 17 4 Results ................................................................................................................................................ 18 Search results .................................................................................................................................... 18 CAR T-cell technologies under advanced stage development ........................................................... 19 Extension of indications for Kymriah, Yescarta and Tecartus ........................................................... 32 Allogeneic CAR T-cell therapy ............................................................................................................ 35 In-hospital production of CAR T-cell therapies .................................................................................. 36 5 Discussion ........................................................................................................................................... 38 Additional oncological indications ..................................................................................................... 39 Allogeneic CAR T-cell therapy ............................................................................................................ 41 In-hospital production of CAR T-cell therapy ..................................................................................... 42 Outlook .............................................................................................................................................. 43 Limitation of the report ..................................................................................................................... 43 6 Conclusion & Recommendation ......................................................................................................... 44 7 References .......................................................................................................................................... 46 8 Appendix ............................................................................................................................................. 51 Search strategy for clinical trial registries ......................................................................................... 51 Search results from clinical trial registries ..................................................................................... 52 2 List of Figures Figure 1: Cancer Immunotherapy: Checkpoint inhibitors (anti-PD-1 / anti-PD-L1) and CAR T-cell trials in 2019 (ClinicalTrials.gov) ..................................................................................................................... 10 Figure 2: Approaches to improve CAR T-cell therapy ........................................................................... 12 Figure 3: Schematic representation of CAR T cell therapy (Source: National Cancer Institute) ........... 13 Figure 4: Flow chart of clinical trial identification and CAR T-cell technologies inclusion .................... 18 List of Tables Table 1: Inclusion and exclusion criteria ............................................................................................... 17 Table 2: Overview of identified CAR T-cell technologies (sorted by target) ......................................... 19 Table 3: Overview of CAR T technologies at the most advanced development stage .......................... 25 Table 4: List of ongoing clinical trials for Tisagenlecleucel (Novartis) ................................................... 32 Table 5: List of ongoing clinical trials for Axicabtagen-ciloleucel (Kite/Gilead) .................................... 33 Table 6: List of ongoing clinical trials for brexucabtagene autoleucel (Kite/Gilead)............................. 34 Table 7: Allogeneic CAR T-cell therapies under development .............................................................. 35 Table 8: Examples of clinical trials with point-of-care CAR T-cell production ....................................... 36 Table 9: Comparison between autologous and allogeneic CAR T-cells (by Depil et al.) ....................... 41 Table 10: Overview of CAR-T Cell products in clinical trials: results from clinicaltrials.gov* ................ 52 Table 11: Overview of CAR-T Cell products in clinical trials: results from https://apps.who.int/trialsearch/* ....................................................................................................... 80 Table 12: Overview of CAR-T Cell products in clinical trials: results from EU Clinical Trials Register (Trials with EudraCT Protocol)* ............................................................................................................. 92 3 List of Abbreviations AIHTA Austrian Institute for Health Technology Assessment GmbH ALL Acute Lymphoblastic Leukaemia AML Acute Myeloid Leukaemia ASH American Society of Haematology ATMP Advanced Therapy Medicinal Products BCMA B-Cell Maturation Antigen BLA Biologics License Application BPDCN Blastic Plasmacytoid Dendritic Cell Neoplasm CAR Chimeric Antigen Receptor CEA Carcinoembryonic Antigen CLL Chronic Lymphocytic Leukaemia CMC Chemistry, Manufacturing and Control CPIS Checkpoint Inhibitors CRS Cytokine Release Syndrome DLBCL Diffuse-Large B-Cell Lymphoma EGFRVIII Epidermal Growth Factor Receptor Variant III EMA European Medicines Agency EAAS Early Awareness and Alert System EU European Union EWS Early Warning System 4SCART 4th Generation Chimeric Antigen Receptor gene-modified T-cells FDA Food and Drug Administration FL Follicular Lymphoma GMP Good Manufacturing Practice GTMP Gene Therapy Medicinal Products 4 GVHD Graft-versus-Host Disease HL Hodgkin’s Lymphoma HSS Horizon Scanning System ICC Intrahepatic Cholangiocarcinoma IND Investigational New Drug INHL indolent Non-Hodgkin Lymphoma ITCRP International Clinical Trials Registry Platform
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