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What is Regulatory Science? Stanford Medicine 2017 Big Data in Biomedicine Conference |
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Sean Khozin, MD, MPH
Associate Director (Acting): FDA Oncology Center of Excellence
Founding Director: Information Exchange and Data Transformation (INFORMED), an incubator for driving innovations in agile technology, digital health, and data science to advance public health
Composer and Instrumentalist: wel.kins
Previously (no longer affiliated):
[Opinions my own]
Founding Director: Information Exchange and Data Transformation (INFORMED), an incubator for driving innovations in agile technology, digital health, and data science to advance public health
Composer and Instrumentalist: wel.kins
Previously (no longer affiliated):
- Cofounder, Hello Health
- Founder & Chief Medical Officer, SKMD: High-tech and high-touch patient-centered healthcare delivery network in New York City seamlessly incorporating telemedicine, remote motoring, and advanced analytics into clinical workflows. Dubbed by Health Affairs "an emblem of modern medicine."
[Opinions my own]
Information Exchange and Data Transformation (INFORMED) Initiative
Launched in collaboration with the U.S. Department of Health and Human Services’ Innovation, Design, Entrepreneurship and Action (IDEA) Lab, Information Exchange and Data Transformation (INFORMED) is an incubator for collaborative oncology regulatory science research focused on supporting innovations that enhance FDA’s mission of promotion and protection of public health. Drawing from the expertise of a diverse group of oncologists, data scientists, statisticians, and entrepreneurs-in-residence, INFORMED is expanding organizational and technical infrastructure for big data analytics and examining modern approaches in evidence generation to support regulatory decisions. Special emphasis is placed on systems thinking in oncology regulatory science research to facilitate the development and adoption of new solutions for improving efficiency, reliability, and productivity in a broad range of workflows related to oncology drug development and regulatory decision making.
The research portfolio of INFORMED includes investigations into the use of real world data for clinical evidence generation and prospective pragmatic clinical trials, testing the utility of biosensors and the internet of things to quantify intrinsic and extrinsic (e.g., environmental) factors influencing the patient’s experience, identifying opportunities for machine learning and artificial intelligence to improve existing practices, and exploring the utility of emerging technologies such as blockchain to enable secure exchange of health data at scale. In addition, INFORMED is actively pursuing the development of programs to support the creation of a knowledgeable, sustainable, and agile data science workforce capable of addressing the needs of cancer patients in today’s technology-driven and connected world.
Additional information:
Launched in collaboration with the U.S. Department of Health and Human Services’ Innovation, Design, Entrepreneurship and Action (IDEA) Lab, Information Exchange and Data Transformation (INFORMED) is an incubator for collaborative oncology regulatory science research focused on supporting innovations that enhance FDA’s mission of promotion and protection of public health. Drawing from the expertise of a diverse group of oncologists, data scientists, statisticians, and entrepreneurs-in-residence, INFORMED is expanding organizational and technical infrastructure for big data analytics and examining modern approaches in evidence generation to support regulatory decisions. Special emphasis is placed on systems thinking in oncology regulatory science research to facilitate the development and adoption of new solutions for improving efficiency, reliability, and productivity in a broad range of workflows related to oncology drug development and regulatory decision making.
The research portfolio of INFORMED includes investigations into the use of real world data for clinical evidence generation and prospective pragmatic clinical trials, testing the utility of biosensors and the internet of things to quantify intrinsic and extrinsic (e.g., environmental) factors influencing the patient’s experience, identifying opportunities for machine learning and artificial intelligence to improve existing practices, and exploring the utility of emerging technologies such as blockchain to enable secure exchange of health data at scale. In addition, INFORMED is actively pursuing the development of programs to support the creation of a knowledgeable, sustainable, and agile data science workforce capable of addressing the needs of cancer patients in today’s technology-driven and connected world.
Additional information:
Selected Publications
Real-World Evidence In Support Of Precision Medicine: Clinico-Genomic Cancer Data As A Case Study.
Health Aff (Millwood). 2018 May;37(5):765-772.
INFORMED: an incubator at the US FDA for driving innovations in data science and agile technology.
Nat Rev Drug Discov. 2018 Apr 6. doi: 10.1038/nrd.2018.34.
Characteristics of Real-World Metastatic Non-Small Cell Lung Cancer Patients Treated with Nivolumab and Pembrolizumab During the Year Following Approval.
Oncologist. 2018 Jan 9. pii: theoncologist.2017-0353
Core Clinical Data Elements for Cancer Genomic Repositories: A Multi-stakeholder Consensus.
Cell , Volume 171 , Issue 5 , 982 - 986
Real-world Data for Clinical Evidence Generation in Oncology.
J Natl Cancer Inst. 2017 Nov 1;109(11).
Biometric monitoring devices for assessing end points in clinical trials: developing an ecosystem.
Nat Rev Drug Discov. 2017 Oct;16(10):736.
Milestone Analyses of Immune Checkpoint Inhibitors, Targeted Therapy, and Conventional Therapy in Metastatic Non-Small Cell Lung Cancer Trials: A Meta-analysis.
JAMA Oncol. 2017 Jun 15:e171029.
U.S. Food and Drug Administration Approval Summary: Atezolizumab for Metastatic Non-Small Cell Lung Cancer.
Clin Cancer Res. 2017 Jun 13. pii: clincanres.0540.2017.
Data-Sharing Models.
N Engl J Med. 2017 Jun 8;376(23):2305-2306.
Advantages of a Truly Open-Access Data-Sharing Model
N Engl J Med. 2017 Mar 23;376(12):1178-1181.
From big data to smart data: FDA's INFORMED initiative
Nat Rev Drug Discov. 2017 Feb 24. doi: 10.1038/nrd.2017.26
Osimertinib for the Treatment of Metastatic Epidermal Growth Factor T970M Positive Non-Small Cell Lung Cancer
Clin Cancer Res. 2016 Dec 6. pii: clincanres.1773.2016
Expanded Access of Investigational Drugs: The Experience of the Center of Drug Evaluation and Research Over a 10-Year Period
Ther Innov Regul Sci. 2016 Nov;50(6):705-709.
Evaluating the potential for digital submission of expedited premarket safety reports to the FDA
Nat Rev Drug Discov. 2016 Sep 29;15(10):670-1.
Selumetinib with and without erlotinib in KRAS mutant and KRAS wild-type advanced nonsmall-cell lung cancer
Ann Oncol. 2016 Apr;27(4):693-9.
Clinician Perspectives on Current Issues in Lung Cancer Drug Development
J Thorac Oncol. 2016 Sep;11(9):1387-96.
The Majority of Expedited Investigational New Drug Safety Reports Are Uninformative.
Clin Cancer Res. 2016 Jan 18
Selumetinib with and without erlotinib in KRAS mutant and KRAS wild-type advanced non-small cell lung cancerBenefit-Risk Summary of Nivolumab for Patients With Metastatic
Ann Oncol. 2016 Jan 22.
Squamous Cell Lung Cancer After Platinum-Based Chemotherapy: A Report From the US Food and Drug Administration.
JAMA Oncol. 2016 Jan 1;2(1):118-22.
Why do oncology drugs fail to gain US regulatory approval?
Nat Rev Drug Discov. 2015 May 22.
Ceritinib for the treatment of metastatic anaplastic lymphoma kinase-positive non-small cell lung cancer.
Clin Cancer Res. 2015 Jun 1;21(11):2436-9
Overall response rate, progression-free survival, and overall survival with targeted and standard therapies in advanced non-small-cell lung cancer: US Food and Drug Administration trial-level and patient-level analyses.
J Clin Oncol. 2015 Mar 20;33(9)
Erlotinib for the First-Line Treatment of Metastatic Non-Small Cell Lung Cancer With Epidermal Growth Factor Receptor Exon 19 Deletions or Exon 21 (L858R) Substitution Mutations.
Oncologist. 2014 Jul;19(7):774-9
Cixutumumab for patients with recurrent or refractory advanced thymic epithelial tumours: a multicentre, open-label, phase 2 trial.
Lancet Oncol. 2014 Feb;15(2):191-200
A phase I/II study of sepantronium bromide (YM155, survivin suppressor) with paclitaxel and carboplatin in patients with advanced non-small-cell lung Cancer
Ann Oncol. 2013 Oct;24(10):2601-6
Concurrent molecular alterations in tumors with germ line epidermal growth factor receptor T790M mutations.
Clin Lung Cancer. 2013 Jul;14(4):452-6
18F-fluorodeoxyglucose positron emission tomography in the management of patients with thymic epithelial tumors.
Clin Cancer Res. 2013 Mar 15;19(6):1487-93
Hepatoid carcinoma of the lung with anaplastic lymphoma kinase gene rearrangement
J Thorac Oncol. 2012 Nov;7(11):e29-31
Response to crizotinib in ROS1-rearranged non-small-cell lung cancer.
J Clin Oncol. 2012 Sep 20;30(27):3425-6; author reply 3426
Integrating a Physical Office with an Online Platform to Improve Continuity of Care and Expand Patient-Physician Communication.
J Participat Med. 2011 Jan 12; 3:e5.
Using the hierarchy of control technologies to improve healthcare facility infection control: lessons from severe acute respiratory syndrome.
J Occup Environ Med. 2004 Jul;46(7):613-22
The effects of adrenalectomy and aldosterone replacement in transgenic mice expressing antisense RNA to the type 2 glucocorticoid receptor.
Physiol Behav. 2002 Nov;77(2-3):417-23
Lifetime exposure to a soluble TGF-beta antagonist protects mice against metastasis without adverse side effects.
J Clin Invest. 2002 Jun;109(12):1607-15
Health Aff (Millwood). 2018 May;37(5):765-772.
INFORMED: an incubator at the US FDA for driving innovations in data science and agile technology.
Nat Rev Drug Discov. 2018 Apr 6. doi: 10.1038/nrd.2018.34.
Characteristics of Real-World Metastatic Non-Small Cell Lung Cancer Patients Treated with Nivolumab and Pembrolizumab During the Year Following Approval.
Oncologist. 2018 Jan 9. pii: theoncologist.2017-0353
Core Clinical Data Elements for Cancer Genomic Repositories: A Multi-stakeholder Consensus.
Cell , Volume 171 , Issue 5 , 982 - 986
Real-world Data for Clinical Evidence Generation in Oncology.
J Natl Cancer Inst. 2017 Nov 1;109(11).
Biometric monitoring devices for assessing end points in clinical trials: developing an ecosystem.
Nat Rev Drug Discov. 2017 Oct;16(10):736.
Milestone Analyses of Immune Checkpoint Inhibitors, Targeted Therapy, and Conventional Therapy in Metastatic Non-Small Cell Lung Cancer Trials: A Meta-analysis.
JAMA Oncol. 2017 Jun 15:e171029.
U.S. Food and Drug Administration Approval Summary: Atezolizumab for Metastatic Non-Small Cell Lung Cancer.
Clin Cancer Res. 2017 Jun 13. pii: clincanres.0540.2017.
Data-Sharing Models.
N Engl J Med. 2017 Jun 8;376(23):2305-2306.
Advantages of a Truly Open-Access Data-Sharing Model
N Engl J Med. 2017 Mar 23;376(12):1178-1181.
From big data to smart data: FDA's INFORMED initiative
Nat Rev Drug Discov. 2017 Feb 24. doi: 10.1038/nrd.2017.26
Osimertinib for the Treatment of Metastatic Epidermal Growth Factor T970M Positive Non-Small Cell Lung Cancer
Clin Cancer Res. 2016 Dec 6. pii: clincanres.1773.2016
Expanded Access of Investigational Drugs: The Experience of the Center of Drug Evaluation and Research Over a 10-Year Period
Ther Innov Regul Sci. 2016 Nov;50(6):705-709.
Evaluating the potential for digital submission of expedited premarket safety reports to the FDA
Nat Rev Drug Discov. 2016 Sep 29;15(10):670-1.
Selumetinib with and without erlotinib in KRAS mutant and KRAS wild-type advanced nonsmall-cell lung cancer
Ann Oncol. 2016 Apr;27(4):693-9.
Clinician Perspectives on Current Issues in Lung Cancer Drug Development
J Thorac Oncol. 2016 Sep;11(9):1387-96.
The Majority of Expedited Investigational New Drug Safety Reports Are Uninformative.
Clin Cancer Res. 2016 Jan 18
Selumetinib with and without erlotinib in KRAS mutant and KRAS wild-type advanced non-small cell lung cancerBenefit-Risk Summary of Nivolumab for Patients With Metastatic
Ann Oncol. 2016 Jan 22.
Squamous Cell Lung Cancer After Platinum-Based Chemotherapy: A Report From the US Food and Drug Administration.
JAMA Oncol. 2016 Jan 1;2(1):118-22.
Why do oncology drugs fail to gain US regulatory approval?
Nat Rev Drug Discov. 2015 May 22.
Ceritinib for the treatment of metastatic anaplastic lymphoma kinase-positive non-small cell lung cancer.
Clin Cancer Res. 2015 Jun 1;21(11):2436-9
Overall response rate, progression-free survival, and overall survival with targeted and standard therapies in advanced non-small-cell lung cancer: US Food and Drug Administration trial-level and patient-level analyses.
J Clin Oncol. 2015 Mar 20;33(9)
Erlotinib for the First-Line Treatment of Metastatic Non-Small Cell Lung Cancer With Epidermal Growth Factor Receptor Exon 19 Deletions or Exon 21 (L858R) Substitution Mutations.
Oncologist. 2014 Jul;19(7):774-9
Cixutumumab for patients with recurrent or refractory advanced thymic epithelial tumours: a multicentre, open-label, phase 2 trial.
Lancet Oncol. 2014 Feb;15(2):191-200
A phase I/II study of sepantronium bromide (YM155, survivin suppressor) with paclitaxel and carboplatin in patients with advanced non-small-cell lung Cancer
Ann Oncol. 2013 Oct;24(10):2601-6
Concurrent molecular alterations in tumors with germ line epidermal growth factor receptor T790M mutations.
Clin Lung Cancer. 2013 Jul;14(4):452-6
18F-fluorodeoxyglucose positron emission tomography in the management of patients with thymic epithelial tumors.
Clin Cancer Res. 2013 Mar 15;19(6):1487-93
Hepatoid carcinoma of the lung with anaplastic lymphoma kinase gene rearrangement
J Thorac Oncol. 2012 Nov;7(11):e29-31
Response to crizotinib in ROS1-rearranged non-small-cell lung cancer.
J Clin Oncol. 2012 Sep 20;30(27):3425-6; author reply 3426
Integrating a Physical Office with an Online Platform to Improve Continuity of Care and Expand Patient-Physician Communication.
J Participat Med. 2011 Jan 12; 3:e5.
Using the hierarchy of control technologies to improve healthcare facility infection control: lessons from severe acute respiratory syndrome.
J Occup Environ Med. 2004 Jul;46(7):613-22
The effects of adrenalectomy and aldosterone replacement in transgenic mice expressing antisense RNA to the type 2 glucocorticoid receptor.
Physiol Behav. 2002 Nov;77(2-3):417-23
Lifetime exposure to a soluble TGF-beta antagonist protects mice against metastasis without adverse side effects.
J Clin Invest. 2002 Jun;109(12):1607-15
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