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

Mohamed Abou-El-Enein^{1

2}, Spencer Phillips Hey^{3

4}

Affiliations

¹ Berlin-Brandenburg Center for Regenerative Therapies (BCRT), Charité-Universitätsmedizin Berlin, Berlin, Germany.
² Berlin Center for Advanced Therapies (BeCAT), Charité-Universitätsmedizin Berlin, Berlin, Germany.
³ Program on Regulation, Therapeutics, and Law (PORTAL), Brigham and Women's Hospital, 1620 Tremont Street, Boston, MA 02120, USA.
⁴ Center for Bioethics, Harvard Medical School, Boston, MA, USA.

PMID: 31193634
PMCID: PMC6537560
DOI: 10.1016/j.eclinm.2019.01.015

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

Mohamed Abou-El-Enein et al. EClinicalMedicine. 2019.

. 2019 Feb 2:7:13-14.

doi: 10.1016/j.eclinm.2019.01.015. eCollection 2019 Jan.

Authors

Mohamed Abou-El-Enein^{1

2}, Spencer Phillips Hey^{3

4}

Affiliations

¹ Berlin-Brandenburg Center for Regenerative Therapies (BCRT), Charité-Universitätsmedizin Berlin, Berlin, Germany.
² Berlin Center for Advanced Therapies (BeCAT), Charité-Universitätsmedizin Berlin, Berlin, Germany.
³ Program on Regulation, Therapeutics, and Law (PORTAL), Brigham and Women's Hospital, 1620 Tremont Street, Boston, MA 02120, USA.
⁴ Center for Bioethics, Harvard Medical School, Boston, MA, USA.

PMID: 31193634
PMCID: PMC6537560
DOI: 10.1016/j.eclinm.2019.01.015

No abstract available

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

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References

1. U.S. Food and Drug Administration YESCARTA (axicabtagene ciloleucel) 2018. https://www.fda.gov/BiologicsBloodVaccines/CellularGeneTherapyProducts/A...
1. U.S. Food and Drug Administration KYMRIAH (tisagenlecleucel) 2018. https://www.fda.gov/BiologicsBloodVaccines/CellularGeneTherapyProducts/A...
1. U.S. Food and Drug Administration FDA advisory committee briefing document: Tisagenlecleucel (CTL019) 2017. https://www.fda.gov/downloads/advisorycommittees/committeesmeetingmateri...
1. Abou-El-Enein M. Overcoming challenges facing advanced therapies in the EU market. Cell Stem Cell. 2016;19(3) - PubMed
1. Downing N.S. Clinical trial evidence supporting FDA approval of novel therapeutic agents, 2005–2012. JAMA. Jan 22, 2014;311(4):368. - PMC - PubMed

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

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

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