Contributions of Robert T. O’Neill to the Evolution of Regulatory Statistical Science

References

• 33 Fed. Reg. 7762 . 1968 . “ May 28, ” .
   Google Scholar
• Balant , L. P. , Rowland , M. , Aarons , L. , Mentré , F. , Morselli , P. L. , Steimer , J.-L. and Vozeh , S. 1993 . New Strategies in Drug Development and Clinical Evaluation: The Population Approach . European Journal of Clinical Pharmacology , 45 : 93 – 94 .
   Google Scholar
• Brown , B. W. 1980 . The Crossover Experiment for Clinical Trials . Biometrics , 36 : 69 – 79 .
   PubMed Web of Science ®Google Scholar
• Carpenter , D. 2010 . Reputation and Power: Organizational Image and Pharmaceutical Regulation at the FDA , Princeton, NJ : Princeton University Press .
   Google Scholar
• Colton , T. 1974 . Statistics in Medicine , Boston , MA : Little, Brown & Company, Inc .
   Google Scholar
• Cornfield , J. 1956 . “ A Statistical Problem Arising from Retrospective Studies ” . In Proceedings of the Third Berkeley Symposium on Mathematical Statistics and Probability 4 , 135 – 148 . Berkeley , CA : University of California Press .
   Google Scholar
• Daemmrich , A. A. 2004 . Pharmacopolitics: Drug Regulation in the U.S. and Germany , Chapel Hill , NC : UNC Press .
   Google Scholar
• Daniel , C. , Tufte , E. R. and Kadane , J. A. 1971 . Statistics and Data Analysis in the Food and Drug Administration . Federal Statistics (Report of the President's Commission) , 2 : 65 – 95 .
   Google Scholar
• Davis , R. L. , Free , S. M. , Gulyas , S. W. , Hearron , M. S. , Lindborg , S. R. , O’Neill , R. and Sampson , C. B. The History of Biopharmaceutical Section of the American Statistical Association (ASA), 1966–1988 . Presented to the ASA Biopharmaceutical Executive Committee by the ASA Biopharm History Committee . Available at https://www.amstat.org/sections/sbiop/biopharmreport/bs_hist.pdf
   Google Scholar
• Dong , M. H. , Dubey , S. D. , O’Neill , R. T. and Tsong , Y. 1990 . Competing Risk Analysis of Life Table Data: Application to Lifetime Risk Computation . Journal of Clinical Epidemiology , 43 : 1351 – 1359 .
   Google Scholar
• Fisher , L. 1999 . Carvedilol and the Food and Drug Administrations (FDA) Approval Process: The FDA Paradigm and Reflections on Hypothesis Testing . Controlled Clinical Trials , 20 : 16 – 39 .
   PubMedGoogle Scholar
• Food and Drug Law Institute . 2012 . “ Commemorating the 50th Anniversary of the 1962 Amendments ” . Wednesday, October 17, 2012, Dupont Circle Hotel, Washington D.C. [unpublished transcript], Available at http://www.fdli.org/conferences/conference-pages/1962-amendments-reception/commemorating-the-50th-anniversary-of-the-1962-amendments
   Google Scholar
• Harris , R. 1964 . The Real Voice , New York : The MacMillan Co .
   Google Scholar
• Hecht , A. 1984 . A Long Reach Back to Assure Drug Quality: Drug Efficacy Study Implementation Program, FDA Consumer . : 14 – 15 .
   Google Scholar
• Hill , A. B. 1971 . Principles of Medical Statistics , 9th ed. , Edinburgh : Lancet Limited, R & R Clark .
   Google Scholar
• Hung , H. M. J. , O’Neill , R. T. , Bauer , P. and Köhne , K. 1997 . The Behavior of the P-Value When the Alternative Hypothesis is True . Biometrics , 53 : 11 – 22 .
   PubMed Web of Science ®Google Scholar
• Hung , H. M. J. , Wang , S.-J. and O’Neill , R. T. 2007 . Statistical Considerations for Testing Multiple Endpoints in Group Sequential or Adaptive Clinical Trials . Journal of Biopharmaceutical Statistics , 17 : 1201 – 1210 .
   PubMed Web of Science ®Google Scholar
• Hung , H. M. J. , Wang , S. J. , Tsong , Y. , Lawrence , J. and O’Neill , R. T. 2003 . Some Fundamental Issues with Non-Inferiority Testing in Active Controlled Trials . Statistics in Medicine , 22 : 213 – 225 .
   PubMed Web of Science ®Google Scholar
• Hutt , P. B. 1973 . Views on Supreme Court/FDA Decisions . Food, Drug, and Cosmetic Law Journal , 28 : 662 – 675 .
   Google Scholar
• Inman , W. H. and Vessey , M. P. 1968 . Investigation of Deaths from Pulmonary, Coronary, and Cerebral Thrombosis and Embolism in Women of Child-Bearing Age . British Medical Journal , 2 : 193 – 211 .
   PubMed Web of Science ®Google Scholar
• Ioannidis , J. P. A. , Evans , S. J. W. , Gøtzche , P. C. , O’Neill , R. T. , Altman , D. G. , Schulz , K. , Moher , D. and CONSORT Group . 2004 . Better Reporting of Harms in Randomized Trials: An Extension of the CONSORT Statement . Annals of Internal Medicine , 141 : 781 – 788 .
   PubMed Web of Science ®Google Scholar
• Junod , S. W. and Marks , L. 2002 . Women's Trials: The Approval of the First Oral Contraceptive Pill in the United States and Great Britain . Journal of the History of Medicine and Allied Sciences , 57 : 117 – 160 .
   PubMed Web of Science ®Google Scholar
• Junod , S. W. and Tucker , R. A. 2011a . “ History of the Food and Drug Administration, Interview with Robert T. O’Neill ” . Available at http://www.fda.gov/downloads/AboutFDA/WhatWeDo/History/OralHistories/SelectedOralHistoryTranscripts/UCM289034.pdf
   Google Scholar
• Junod , S. W. and Tucker , R. A. 2011b . “ Oral History Interview with Walter M. Batts, December 13 and December 20, 2011 ” . Audiotape/Transcript, Food and Drug Administration .
   Google Scholar
• 1962 . “ Kefauver-Harris Drug Amendments (also known as the 1962 Drug Amendments), P.L. 87–781, 87th Congress, §102d ” .
   Google Scholar
• Knapp , D. E. , Zax , B. B. , Rossi , A. C. and O’Neill , R. T. 1980 . A Method for Post Marketing Screening of Adverse Reactions to Drugs, Initial Results . Drug Intelligence and Clinical Pharmacy , 14 : 23 – 27 .
   Google Scholar
• Lumpkin , M. 2012 . “ FDA Perspectives on International Clinical Trials ” . In FDA Clinical Trials Workshop Silver Spring , MD unpublished transcript, http://www.fda.gov/downloads/Training/ClinicalInvestigatorTrainingCourse/UCM283376.pdf
   Google Scholar
• Mainland , D. 1954 . The Rise of Experimental Statistics and the Problems of a Medical Statistician . Yale Journal of Biology and Medicine , 27 : 123 – 124 .
   Google Scholar
• Marks , H. 2000 . Progress of Experiment: Science and Therapeutic Reform in the United States, 1900–1990 , Cambridge : Cambridge University Press .
   Google Scholar
• Meinert , C. and Tonascia , S. 1986 . Clinical Trials: Design, Conduct and Analysis , New York : Oxford University Press .
   Google Scholar
• Mele , J. (2010) . “ Study Data Specifications and ADaM Review and Comparison ” . FDA, CDER, Available at http://www.fda.gov/downloads/Drugs/NewsEvents/UCM209127
   Google Scholar
• National Academy of Science. 1969 . Final Report to the Commissioner of Food and Drugs: Food and Drug Administration from Division of Medical Sciences , Washington, DC : National Academy of Sciences .
   Google Scholar
• O’Neill , R. T. 1974 . Discussion of “Crossover Designs Involving Two Treatments” by A. Varma and N. Chilton . Journal of Periodontal Research , Supplement 14 : 166 – 170 .
   Google Scholar
• O’Neill , R. T. 1984 . Sample Sizes for Estimation of the Odds ratio in Unmatched Case-Control Studies . American Journal of Epidemiology , 120 : 145 – 153 .
   PubMedGoogle Scholar
• O’Neill , R. T. 1985 . “ Sampling to a Pre-Specified Precision of the Log Odds Ratio Estimator: A Sequential Approach ” . In Proceedings of the Biopharmaceutical Section , 118 – 122 . Alexandria , VA : American Statistical Association .
   Google Scholar
• O’Neill , R. T. 1988 . On Sample Sizes to Estimate the Protective Efficacy of a Vaccine . Statistics in Medicine , 7 : 1279 – 1288 .
   PubMed Web of Science ®Google Scholar
• O’Neill , R. T. 1989 . Comments on “Multistage Testing with Adaptive Designs” by P. Bauer . Biometrie und INformatik in Medizin und Biologie , 20 : 142 – 144 .
   Google Scholar
• O’Neill , R. T. 1993 . Discussion of “Some FDA Perspectives on Data Monitoring in Clinical Trials in Drug Development” . Statistics in Medicine , 12 : 601 – 608 .
   PubMed Web of Science ®Google Scholar
• O’Neill , R. T. 1994 . Early Stopping of Prevention Trials when Multiple Outcomes are of Interest: A Discussion: Conclusion 2 . Statistics in Medicine , 12 : 1493 – 1499 .
   Google Scholar
• O’Neill , R. T. 1995 . Statistical Concepts in the Planning and Evaluation of Drug Safety from Clinical Trials in Drug Development: Issues of International Harmonization . Statistics in Medicine , 14 : 117 – 127 .
   PubMed Web of Science ®Google Scholar
• O’Neill , R. T. 1997 . “Secondary Endpoints Cannot be Validly Analyzed if the Primary Endpoint does not Demonstrate Clear Statistical Significance” (with discussion) . Control Clinical Trials , 6 : 550 – 567 .
   Google Scholar
• ——— . 1998 . Biostatistical Considerations in Pharmacovigilance and Pharmacoepidemiology: Linking Quantitative Risk Assessment in Pre-Market Licensure Application Safety Data, Post-Market Alert Reports and Formal Epidemiological Studies . Statistics in Medicine , 17 : 1851 – 1858 .
   Google Scholar
• O’Neill , R. T. A Perspective on the Development and Future of Statistics at the FDA . Presented at the 2002 Marvin Zelen Leadership Award in Statistical Science on May 31, Harvard University .
   Google Scholar
• O’Neill , R. T. 2008 . A Perspective on Characterizing Benefits and Risks Derived from Clinical Trials: Can We Do More? . Drug Information Journal , 42 : 235 – 245 .
   Web of Science ®Google Scholar
• O’Neill , R. T. 2012 . Reflections on Professor Jerome Cornfield's Contributions to the U.S. Food and Drug Administration . Clinical Trials , 10 : 332 – 336 .
   Google Scholar
• O’Neill , R. T. , Beninger , P. and Wykoff , R. 1992 . Comment on “Statistical Issues Arising in AIDS Clinical Trials” . Journal of the American Statistical Association , 87 : 579 – 581 .
   Google Scholar
• O’Neill , R. T. and Chen , C. W. 1978 . A Statistical Procedure Useful in Evaluating Time of Onset and Termination of Response in Clinical Trials . Biometrics , 34 : 411 – 420 .
   Google Scholar
• O’Neill , R. T. and Idanpaan-Heikkila , J. 1983 . “ Assessing Adverse Event Rates in Clinical Trials As a Function of Duration of Treatment Exposure ” . Rockville, MD : National Center for Drugs and Biologics . Appendix I, A Review of Safety Information Obtained from Phases 1 and II and Phase II Clinical Investigations of Sixteen Selected Drugs
   Google Scholar
• O’Neill , R. T. and Szarfman , A. 1999 . Discussion of “Bayesian Data Mining in Large Frequency Tables with Applications to FDA Spontaneous Reporting System” by W. Dumouchel . The American Statistician , 53 : 190 – 196 .
   Google Scholar
• O’Neill , R. T. , Temple , R. , Fairweather , W. and Glocklin , V. 1988 . The Case for Blinded Slide Readings . Comments on Toxicology , 2 : 99 – 109 .
   Google Scholar
• Podolsky , S. H. 2010 . Antibiotics and the Social History of the Controlled Clinical Trial, 1950–1970 . Journal of the History of Medicine , 65 : 327 – 336 .
   Google Scholar
• Rossi , A. C. , Knapp , D. E. , Anello , C. E. , O’Neill , R. T. , Graham , C. F. , Mendelis , P. S. and Stanley , G. R. 1983 . Discovery of Adverse Drug Reactions: A Comparison of Selected Phase IV Studies With Spontaneous Reporting Methods . Journal American Medical Association , 249 : 2226 – 2228 .
   PubMed Web of Science ®Google Scholar
• Rossiter , M. W. 1995 . Women in Science: Before Affirmative Action, 1940–1972: Volume II , Baltimore , MD : Johns Hopkins University Press .
   Google Scholar
• Segreti , A. C. , Leung , H. M. , Koch , G. G. , Davis , R. L. , Mohberg , N. R. and Peace , K. E. 2001 . Statistics in a Pharmaceutical Regulated Environment: Past, Present, and Future . Journal of Biopharmaceutical Statistics , 11 : 347 – 372 .
   PubMedGoogle Scholar
• Seigel , D. and Corfman , P. 1963 . Epidemiological Problems Associated with Studies of the Safety of Oral Contraceptives . Journal of the American Medical Association , 203 : 950 – 954 .
   Google Scholar
• Siddiqui , O. , Hung , H. M. and O’Neill , R. T. 2009 . MMRM vs. LOCF: A Comprehensive Comparison Based on Simulation Study and 25 NDA Datasets . Journal of Biopharmaceutical Statistics , 19 : 227 – 246 .
   PubMed Web of Science ®Google Scholar
• Szarfman , A. , Machado , S. G. and O’Neill , R. T. 2002 . Use of Screening Algorithms and Computer Systems to Efficiently Signal Higher-Than- Expected Combinations of Drugs and Events in the US FDA's Spontaneous Reports Database . Drug Safety , 25 : 381 – 392 .
   PubMed Web of Science ®Google Scholar
• Temple , R. 1995 . “ Development of Drug Law, Regulations, and Guidance in the U.S ” . In Principles of Pharmacology: Basic Concepts and Clinical Applications , Edited by: Breese , G. R. 1643 – 1664 . New York : Chapman and Hall .
   Google Scholar
• Temple , R. and Pledger , G. W. 1980 . FDA's Critique of the Anturane Reinfarction Trial . New England Journal of Medicine , 303 : 1488 – 1492 .
   PubMed Web of Science ®Google Scholar
• Tobbell , D. 2011 . Pills, Power and Policy: Struggle for Drug Reform in Cold War America and Its Consequences , Berkeley, CA : University of California Press .
   Google Scholar
• U.S. Department of Health, Education, and Welfare, Public Health Service, Food and Drug Administration . 1977 . “ General Considerations for the Evaluation of Drugs ” . HEW Publication No. (FDA) 77–3040, Available at http://www.fda.gov/downloads/ScienceResearch/SpecialTopics/WomensHealthResearch/UCM131196.pdf
   Google Scholar
• U.S. Department of Health and Human Services, Center for Drug Evaluation and Research, and Food and Drug Administration. 1988 . “ Guidance for the Format and Content of the Clinical and Statistical Sections of an Application ” . Available at http://www.fda.gov/downloads/Drugs/Guidances/UCM071665.pdf
   Google Scholar
• U.S. Department of Health and Human Services, Food and Drug Administration, Center for Drug Evaluation and Research Center for Biologics Evaluation and Research. 1998a . “ Guidance for Industry E9 Statistical Principles for Clinical Trials ” . Available at http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm073137.pdf
   Google Scholar
• U.S. Department of Health and Human Services, Food and Drug Administration, Center for Drug Evaluation and Research Center for Biologics Evaluation and Research. 1998b . “ Guidance for Industry Providing Clinical Evidence of Effectiveness for Human Drug and Biologic Products ” . Available at http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm078749.pdf
   Google Scholar
• U.S. Department of Health and Human Services, Food and Drug Administration, Center for Drug Evaluation and Research Center for Biologics Evaluation and Research. 2001 . “ Guidance for Industry E10 Choice of Control Group and Related Issues I Clinical Trials ” . Available at http://www.fda.gov/downloads/RegulatoryInformation/Guidances/ucm129460.pdf
   Google Scholar
• U.S. Department of Health and Human Services, Food and Drug Administration, Center for Drug Evaluation and Research Center for Biologics Evaluation and Research. 2010 . “ Guidance for Industry Non-Inferiority Clinical Trials ” . Available at http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM202140.pdf
   Google Scholar
• Wang , S. J. , O’Neill , R. T. and Hung , H. M. J. 2007 . Approaches to Evaluation of Treatment Effect in Randomized Clinical Trials with Genomic Subset . Pharmaceutical Statistics , 6 : 227 – 244 .
   PubMed Web of Science ®Google Scholar
• Wilson , S. E. and Zhou , F. A Statistical Reviewer's Perspective . 42nd Annual Meeting, Drug Information Association, Philadelphia, PA .
   Google Scholar
• Woodcock , J. and Junod , S. W. 2012 . “ PDUFA Lays the Foundation: Launching into the Era of User Fee Acts ” . In PDUFA & the Expression of FDA User Fees: Lessons from Negotiators, edited by the Food and Drug Law Institute , 1 – 26 . Washington , DC : Food and Drug Law Institute . Available at http://www.fda.gov/AboutFDA/WhatWeDo/History/Overviews/ucm305697.htm
   Google Scholar