Feasibility of assessing comparative long-term safety for gene therapy products by creating an external control arm using real-world data sources

被引：0
|
作者：
Vossen, Carla Y. [1 ]
Madaj, Keely [1 ]
Menon, Jyothi [1 ]
Albano, Jessica D. [1 ]
机构：
[1] Syneos Hlth, Bridgewater, NJ USA
来源：
PHARMACOEPIDEMIOLOGY AND DRUG SAFETY | 2023年 / 32卷
关键词：
D O I：
暂无
中图分类号：
R1 [预防医学、卫生学];
学科分类号：
1004 ; 120402 ;
摘要：
655
引用
收藏
页码：307 / 308
页数：2
相关论文
共 50 条
  • [1] Identification of appropriate safety metrics in long-term studies using real-world data
    Soukup, Mat
    Kim, Clara
    Jung, Taehyun
    PHARMACOEPIDEMIOLOGY AND DRUG SAFETY, 2021, 30 : 241 - 241
  • [2] LONG-TERM AND REAL-WORLD SAFETY AND EFFICACY OF RETROVIRAL GENE THERAPY FOR ADENOSINE DEAMINASE DEFICIENCY
    Migliavacca, Maddalena
    Barzaghi, Federica
    Fossati, Claudia
    Rancoita, Paola M. V.
    Gabaldo, Michela
    Dionisio, Francesca
    Giannelli, Stefania
    Salerio, Federica Andrea
    Ferrua, Francesca
    Tucci, Francesca
    Valeria, Calbi
    Gallo, Vera
    Recupero, Salvatore
    Consiglieri, Giulia
    Pajno, Roberta
    Garella, Vittoria
    Monti, Ilaria
    Silvani, Paolo
    Darin, Silvia
    Casiraghi, Miriam
    Corti, Ambra
    Zancan, Stefano
    Levi, Margherita
    Cesana, Daniela
    Pituch-Noworolska, Anna
    AbdElaziz, Dalia
    Baumann, Ulrich
    Finocchi, Andrea
    Cancrini, Caterina
    Ladogana, Saverio
    Meinhardt, Andrea
    Meyts, Isabelle
    Montin, Davide
    Notarangelo, Lucia Dora
    Porta, Fulvio
    Pasquet, Marlene
    Speckmann, Carsten
    Stepensky, Polina
    Tommasini, Alberto
    Rabusin, Marco
    Karakas, Zeynep
    Galicchio, Miguel
    Leonardi, Lucia
    Duse, Marzia
    Guner, Sukru Nail
    Di Serio, Clelia
    Ciceri, Fabio
    Bernardo, Maria Ester
    Aiuti, Alessandro
    Cicalese, Maria Pia
    BONE MARROW TRANSPLANTATION, 2024, 59 : 64 - 65
  • [3] Long-term and real-world safety and efficacy of retroviral gene therapy for adenosine deaminase deficiency
    Maddalena Migliavacca
    Federica Barzaghi
    Claudia Fossati
    Paola M. V. Rancoita
    Michela Gabaldo
    Francesca Dionisio
    Stefania Giannelli
    Federica Andrea Salerio
    Francesca Ferrua
    Francesca Tucci
    Valeria Calbi
    Vera Gallo
    Salvatore Recupero
    Giulia Consiglieri
    Roberta Pajno
    Maria Sambuco
    Alessio Priolo
    Chiara Ferri
    Vittoria Garella
    Ilaria Monti
    Paolo Silvani
    Silvia Darin
    Miriam Casiraghi
    Ambra Corti
    Stefano Zancan
    Margherita Levi
    Daniela Cesana
    Filippo Carlucci
    Anna Pituch-Noworolska
    Dalia AbdElaziz
    Ulrich Baumann
    Andrea Finocchi
    Caterina Cancrini
    Saverio Ladogana
    Andrea Meinhardt
    Isabelle Meyts
    Davide Montin
    Lucia Dora Notarangelo
    Fulvio Porta
    Marlène Pasquet
    Carsten Speckmann
    Polina Stepensky
    Alberto Tommasini
    Marco Rabusin
    Zeynep Karakas
    Miguel Galicchio
    Lucia Leonardi
    Marzia Duse
    Sukru Nail Guner
    Clelia Di Serio
    Nature Medicine, 2024, 30 : 488 - 497
  • [4] Long-term and real-world safety and efficacy of retroviral gene therapy for adenosine deaminase deficiency
    Migliavacca, Maddalena
    Barzaghi, Federica
    Fossati, Claudia
    Rancoita, Paola M. V.
    Gabaldo, Michela
    Dionisio, Francesca
    Giannelli, Stefania
    Salerio, Federica Andrea
    Ferrua, Francesca
    Tucci, Francesca
    Calbi, Valeria
    Gallo, Vera
    Recupero, Salvatore
    Consiglieri, Giulia
    Pajno, Roberta
    Sambuco, Maria
    Priolo, Alessio
    Ferri, Chiara
    Garella, Vittoria
    Monti, Ilaria
    Silvani, Paolo
    Darin, Silvia
    Casiraghi, Miriam
    Corti, Ambra
    Zancan, Stefano
    Levi, Margherita
    Cesana, Daniela
    Carlucci, Filippo
    Pituch-Noworolska, Anna
    Abdelaziz, Dalia
    Baumann, Ulrich
    Finocchi, Andrea
    Cancrini, Caterina
    Ladogana, Saverio
    Meinhardt, Andrea
    Meyts, Isabelle
    Montin, Davide
    Notarangelo, Lucia Dora
    Porta, Fulvio
    Pasquet, Marlene
    Speckmann, Carsten
    Stepensky, Polina
    Tommasini, Alberto
    Rabusin, Marco
    Karakas, Zeynep
    Galicchio, Miguel
    Leonardi, Lucia
    Duse, Marzia
    Guner, Sukru Nail
    Di Serio, Clelia
    NATURE MEDICINE, 2024, 30 (2) : 488 - 497
  • [5] A Framework for Extension Studies Using Real-World Data to Examine Long-Term Safety and Effectiveness
    Mehmet Burcu
    Cyntia B. Manzano-Salgado
    Anne M. Butler
    Jennifer B. Christian
    Therapeutic Innovation & Regulatory Science, 2022, 56 : 15 - 22
  • [6] A Framework for Extension Studies Using Real-World Data to Examine Long-Term Safety and Effectiveness
    Burcu, Mehmet
    Manzano-Salgado, Cyntia B.
    Butler, Anne M.
    Christian, Jennifer B.
    THERAPEUTIC INNOVATION & REGULATORY SCIENCE, 2022, 56 (01) : 15 - 22
  • [7] Design and Evaluation of an External Control Arm Using Prior Clinical Trials and Real-World Data
    Ventz, Steffen
    Lai, Albert
    Cloughesy, Timothy F.
    Wen, Patrick Y.
    Trippa, Lorenzo
    Alexander, Brian M.
    CLINICAL CANCER RESEARCH, 2019, 25 (16) : 4993 - 5001
  • [8] SAFETY OF LONG-TERM STATIN THERAPY: DATA FROM REAL-WORLD OBSERVATION IN SUBJECTS AT VERY HIGH RISK
    Benczur, Bela
    Herczeg, Bela
    ATHEROSCLEROSIS, 2017, 263 : E246 - E246
  • [9] Long term comparative safety of ARNi verses ACEi/ARB: A real-world data analysis
    Elsobky, Yasmin
    Hassan, Mona
    Seger, Diane L.
    Bates, David
    PHARMACOEPIDEMIOLOGY AND DRUG SAFETY, 2023, 32 : 367 - 368
  • [10] Investigating long-term complications of gestational diabetes using real-world data
    Goldet, G.
    Tam, F.
    Frankel, A.
    Ul-Haq, Z.
    Dattani, R.
    Kamalati, T.
    DIABETOLOGIA, 2023, 66 (SUPPL 1) : S193 - S193
12345