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BLA multidisciplinary review and evaluation: BLA 761049. Bavencio (Avelumab)
Dreyer NA
Advancing a framework for regulatory use of real-world evidence: When real is reliable
Stephen Calderwood, Barbara Murray, Henry Chambers (2015)
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L. Martin, Melissa Hutchens, C. Hawkins, Alaina Radnov (2017)
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Prescription Drug User Fee Act, 571
Ali AK, Hartzema AG (eds)
Enriched Studies in Post-Authorization Safety Studies of Medicinal Products: The PASS Book (ed 1)
Miksad Employment: Flatiron Health Stock and Other Ownership Interests: Flatiron Health, Roche Consulting or Advisory Role: Advanced Medical, Grand Rounds (I)
US Food and Drug Administration
PDUFA reauthorization performance goals and procedures fiscal years 2018 through 2022
(2018)
US Food and Drug Administration: PDUFA reauthorization performance goals and procedures fiscal years
(2018)
Enriched Studies
(2018)
Hartzema AG (eds): Enriched Studies in Post-Authorization Safety Studies of Medicinal Products: The PASS Book (ed
Guanghong Huang, Xin Sun, Dapeng Liu, Yunfeng Zhang, Boxiang Zhang, G. Xiao, Xiang Li, Xiao Gao, Chenhao Hu, Meng Wang, H. Ren, S. Qin (2017)
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US Food and Drug Administration
US Food and Drug Administration
N. Dreyer (2018)
Advancing a Framework for Regulatory Use of Real-World EvidenceTherapeutic Innovation & Regulatory Science, 52
S. Okun, M. Overhage, R. Platt (2017)
A FRAMEWORK FOR REGULATORY USE OF REAL-WORLD EVIDENCE
Emily Valice Data analysis and interpretation: All authors Manuscript writing: All authors Final approval of manuscript
J. Franklin, S. Schneeweiss (2017)
When and How Can Real World Data Analyses Substitute for Randomized Controlled Trials?Clinical Pharmacology & Therapeutics, 102
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G. Carrigan, S. Whipple, Michael Taylor, Aracelis Torres, A. Gossai, B. Arnieri, Melisa Tucker, Philip Hofmeister, P. Lambert, S. Griffith, W. Capra (2019)
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US Food and Drug Administration
Use of Electronic Health Record Data in Clinical Investigations: Guidance for Industry
PURPOSE: This pilot study examined the ability to operationalize the collection of real-world data to explore the potential use of real-world end points extracted from data from diverse health care data organizations and to assess how these relate to similar end points in clinical trials for immunotherapy-treated advanced non-small-cell lung cancer. PATIENTS AND METHODS: Researchers from six organizations followed a common protocol using data from administrative claims and electronic health records to assess real-world end points, including overall survival (rwOS), time to next treatment, time to treatment discontinuation (rwTTD), time to progression, and progression-free survival, among patients with advanced non-small-cell lung cancer treated with programmed death 1/programmed death-ligand 1 inhibitors in real-world settings. Data sets included from 269 to 6,924 patients who were treated between January 2011 and October 2017. Results from contributors were anonymized. RESULTS: Correlations between real-world intermediate end points (rwTTD and time to next treatment) and rwOS were moderate to high (range, 0.6 to 0.9). rwTTD was the most consistent end points as treatment detail was available in all data sets. rwOS at 1 year post-programmed death-ligand 1 initiation ranged from 40% to 57%. In addition, rwOS as assessed via electronic health records and claims data fell within the range of median OS values observed in relevant clinical trials. Data sources had been used extensively for research with ongoing data curation to assure accuracy and practical completeness before the initiation of this research. CONCLUSION: These findings demonstrate that real-world end points are generally consistent with each other and with outcomes observed in randomized clinical trials, which substantiates the potential validity of real-world data to support regulatory and payer decision making. Differences observed likely reflect true differences between real-world and protocol-driven practices.
JCO Clinical Cancer Informatics – Wolters Kluwer Health
Published: Jul 23, 2019
Keywords: ALK, EGFR, CALR, DCX, CD274
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