Cell and Gene Therapy Trials: Are We Facing an 'Evidence Crisis'?

被引：25

作者：

Abou-El-Enein, Mohamed ^{[1
,2
]}

Hey, Spencer Phillips ^{[3
,4
]}

机构：

[1] Charite Univ Med Berlin, Berlin Brandenburg Ctr Regenerat Therapies BCRT, Berlin, Germany

[2] Charite Univ Med Berlin, Berlin Ctr Adv Therapies BeCAT, Berlin, Germany

[3] Brigham & Womens Hosp, Program Regulat Therapeut & Law PORTAL, 1620 Tremont St, Boston, MA 02120 USA

[4] Harvard Med Sch, Ctr Bioeth, Boston, MA 02115 USA

来源：

ECLINICALMEDICINE | 2019年 / 7卷

关键词：

Clinical trials; Evidence synthesis; Cell and gene therapy; Meta-analysis; Data visualization; Regulatory approval;

D O I：

10.1016/j.eclinm.2019.01.015

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

引用

页码：13 / 14

页数：2

共 10 条

[1] Registry Contributions to Strengthen Cell and Gene Therapeutic Evidence [J].

Abou-El-Enein, Mohamed ;

Grainger, David W. ;

Kili, Sven .

MOLECULAR THERAPY, 2018, 26 (05) :1172-1176

[2] Overcoming Challenges Facing Advanced Therapies in the EU Market [J].

Abou-El-Enein, Mohamed ;

Elsanhoury, Ahmed ;

Reinke, Petra .

CELL STEM CELL, 2016, 19 (03) :293-297

[3] Barriers to the conduct of randomised clinical trials within all disease areas [J].

Djurisic, Snezana ;

Rath, Ana ;

Gaber, Sabrina ;

Garattini, Silvio ;

Bertele, Vittorio ;

Ngwabyt, Sandra-Nadia ;

Hivert, Virginie ;

Neugebauer, Edmund A. M. ;

Laville, Martine ;

Hiesmayr, Michael ;

Demotes-Mainard, Jacques ;

Kubiak, Christine ;

Jakobsen, Janus C. ;

Gluud, Christian .

TRIALS, 2017, 18

[4] Clinical Trial Evidence Supporting FDA Approval of Novel Therapeutic Agents, 2005-2012 [J].

Downing, Nicholas S. ;

Aminawung, Jenerius A. ;

Shah, Nilay D. ;

Krumholz, Harlan M. ;

Ross, Joseph S. .

JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION, 2014, 311 (04) :368-377

[5] Enhancing patient-level clinical data access to promote evidence-based practice and incentivize therapeutic innovation [J].

Fortunato, Alice ;

Grainger, David W. ;

Abou-El-Enein, Mohamed .

ADVANCED DRUG DELIVERY REVIEWS, 2018, 136 :97-104

[6] What is an evidence map? A systematic review of published evidence maps and their definitions, methods, and products [J].

Miake-Lye I.M. ;

Hempel S. ;

Shanman R. ;

Shekelle P.G. .

Systematic Reviews, 5 (1)

[7] Diversity in Clinical and Biomedical Research: A Promise Yet to Be Fulfilled [J].

Oh, Sam S. ;

Galanter, Joshua ;

Thakur, Neeta ;

Pino-Yanes, Maria ;

Barcelo, Nicolas E. ;

White, Marquitta J. ;

de Bruin, Danielle M. ;

Greenblatt, Ruth M. ;

Bibbins-Domingo, Kirsten ;

Wu, Alan H. B. ;

Borrell, Luisa N. ;

Gunter, Chris ;

Powe, Neil R. ;

Burchard, Esteban G. .

PLOS MEDICINE, 2015, 12 (12)

[8]

U.S. Food and Drug Administration, 2018, YESCARTA AX CIL

[9]

U.S. Food and Drug Administration, 2017, CTL019 US FDA

[10]

US Food and Drug Administration, 2018, KYMRIAH TIS

← 1 →