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General medicine
Early experience with the FDA’s regulatory review of novel gene therapies

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  • Contributors JP and JR drafted the manuscript. JP and ACE acquired and analysed the data. All authors participated in the conception and design of the study, interpretation of the data, critical revision of the article for important intellectual content and final approval of the version to be published.

  • Funding The authors have not declared a specific grant for this research from any funding agency in the public, commercial or not-for-profit sectors.

  • Competing interests In the past 36 months, ACE and JR received research support through Yale University from the Laura and John Arnold Foundation for the Collaboration for Research Integrity and Transparency at Yale. ACE and JR currently receive support from the Food and Drug Administration (FDA) for the Yale-Mayo Clinic Center for Excellence in Regulatory Science and Innovation (CERSI) program (U01FD005938). JR received research support through Yale University from Medtronic, Inc and the FDA to develop methods for postmarket surveillance of medical devices (U01FD004585) and from the Centers of Medicare and Medicaid Services to develop and maintain performance measures that are used for public reporting (HHSM-500-2013-13018I). JR currently receives research support through Yale University from Johnson and Johnson to develop methods of clinical trial data sharing, from the Medical Device Innovation Consortium as part of the National Evaluation System for Health Technology, from the Agency for Healthcare Research and Quality (R01HS022882), from the National Heart, Lung and Blood Institute of the National Institutes of Health (NIH) (R01HS025164, R01HL144644), and from the Laura and John Arnold Foundation to establish the Good Pharma Scorecard at Bioethics International. In the past 36 months, NS has received research support through Mayo Clinic from the FDA to establish Yale-Mayo Clinic CERSI program (U01FD005938); the Centers of Medicare and Medicaid Innovation under the Transforming Clinical Practice Initiative; the Agency for Healthcare Research and Quality (R01HS025164; R01HS025402; R03HS025517; K12HS026379); the National Heart, Lung and Blood Institute of the NIH (R56HL130496; R01HL131535; R01HL151662); the National Science Foundation; and the Patient Centered Outcomes Research Institute to develop a Clinical Data Research Network (LHSNet). RR is an employee of the Veterans Health Administration, the views expressed in this article are that of the authors and do not necessarily reflect those of the US Department of Veteran Affairs or the US government.

  • Provenance and peer review Not commissioned; externally peer reviewed.

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