References

This sections lists the references used in the course - it will be updated as the notes are updated. Some of the more accessible (dare I say ‘interesting’) resources are linked from the notes. If you want to read any of these articles, the easiest way is to copy the title into Google scholar.

Altman, Douglas G. 1990. Practical Statistics for Medical Research. CRC press.

———. 1998. “Confidence Intervals for the Number Needed to Treat.” Bmj 317 (7168): 1309–12.

Altman, Douglas G, and J Martin Bland. 1999. “Treatment Allocation in Controlled Trials: Why Randomise?” Bmj 318 (7192): 1209–9.

Armitage, Peter, and Michael Hills. 1982. “The Two-Period Crossover Trial.” Journal of the Royal Statistical Society: Series D (The Statistician) 31 (2): 119–31.

Arnold, Benjamin F, Daniel R Hogan, John M Colford, and Alan E Hubbard. 2011. “Simulation Methods to Estimate Design Power: An Overview for Applied Research.” BMC Medical Research Methodology 11 (1): 1–10.

Berger, James O, José M Bernardo, and Dongchu Sun. 2009. “The Formal Definition of Reference Priors.”

Berger, James O, and Donald A Berry. 1988. “Statistical Analysis and the Illusion of Objectivity.” American Scientist 76 (2): 159–65.

Berry, Donald A. 2006. “Bayesian Clinical Trials.” Nature Reviews Drug Discovery 5 (1): 27–36.

Borm, George F, Jaap Fransen, and Wim AJG Lemmens. 2007. “A Simple Sample Size Formula for Analysis of Covariance in Randomized Clinical Trials.” Journal of Clinical Epidemiology 60 (12): 1234–38.

Collett, David. 2003a. Modelling Binary Data. 2nd ed. Texts in Statistical Science. Chapman & Hall.

———. 2003b. Modelling Survival Data in Medical Research. 2nd ed. Texts in Statistical Science. Chapman & Hall.

Cottingham, Marci D, and Jill A Fisher. 2022. “Gendered Logics of Biomedical Research: Women in US Phase i Clinical Trials.” Social Problems 69 (2): 492–509.

Cox, David R. 1972. “Regression Models and Life-Tables.” Journal of the Royal Statistical Society: Series B (Methodological) 34 (2): 187–202.

Dickinson, L Miriam, Brenda Beaty, Chet Fox, Wilson Pace, W Perry Dickinson, Caroline Emsermann, and Allison Kempe. 2015. “Pragmatic Cluster Randomized Trials Using Covariate Constrained Randomization: A Method for Practice-Based Research Networks (PBRNs).” The Journal of the American Board of Family Medicine 28 (5): 663–72.

Edmonson, John H, Thomas R Fleming, David G Decker, George D Malkasian, Edward O Jorgensen, John A Jefferies, Maurice J Webb, and Larry K Kvols. 1979. “Prognosis in Advanced Ovarian Carcinoma Versus Minimal Residual.” Cancer Treatment Reports 63 (2): 241–47.

Efron, Bradley. 1971. “Forcing a Sequential Experiment to Be Balanced.” Biometrika 58 (3): 403–17.

Elmunzer, B Joseph, James M Scheiman, Glen A Lehman, Amitabh Chak, Patrick Mosler, Peter DR Higgins, Rodney A Hayward, et al. 2012. “A Randomized Trial of Rectal Indomethacin to Prevent Post-ERCP Pancreatitis.” New England Journal of Medicine 366 (15): 1414–22.

Fentiman, Ian S, Robert D Rubens, and John L Hayward. 1983. “Control of Pleural Effusions in Patients with Breast Cancer a Randomized Trial.” Cancer 52 (4): 737–39.

Freedman, LS, and Susan J White. 1976. “On the Use of Pocock and Simon’s Method for Balancing Treatment Numbers over Prognostic Factors in the Controlled Clinical Trial.” Biometrics, 691–94.

Gelman, Andrew, Jennifer Hill, and Aki Vehtari. 2021. Regression and Other Stories. Cambridge University Press.

Giardiello, Francis M, Stanley R Hamilton, Anne J Krush, Steven Piantadosi, Linda M Hylind, Paul Celano, Susan V Booker, C Rahj Robinson, and G Johan A Offerhaus. 1993. “Treatment of Colonic and Rectal Adenomas with Sulindac in Familial Adenomatous Polyposis.” New England Journal of Medicine 328 (18): 1313–16.

Goldacre, B. 2012. Bad Pharma: How Medicine Is Broken, and How We Can Fix It. HarperCollins Publishers. https://books.google.co.uk/books?id=4amY1Q6Id4QC.

Grambsch, Patricia M, and Terry M Therneau. 1994. “Proportional Hazards Tests and Diagnostics Based on Weighted Residuals.” Biometrika 81 (3): 515–26.

Greenwood, Major. 1926. “The Natural Duration of Cancer.” Reports on Public Health and Medical Subjects 33: 1–26.

Hayes, Richard J, and Lawrence H Moulton. 2017. Cluster Randomised Trials. CRC press.

Health, National Institute of. 2023. “History of Women’s Participation in Clinical Research.” Office of Research on Women’s Health. https://orwh. od. nih. gov/toolkit …. https://orwh.od.nih.gov/toolkit/recruitment/history.

Hjalmas, Hanson, and Kruse Hellstrom. 1998. “Long‐term Treatment with Desmopressin in Children with Primary Monosymptomatic Nocturnal Enuresis: An Open Multicentre Study.” British Journal of Urology.

Hommel, EHEBMJ, Hans-Henrik Parving, Elisabeth Mathiesen, Berit Edsberg, M Damkjaer Nielsen, and Jørn Giese. 1986. “Effect of Captopril on Kidney Function in Insulin-Dependent Diabetic Patients with Nephropathy.” Br Med J (Clin Res Ed) 293 (6545): 467–70.

Hotelling, Harold et al. 1931. “The Generalization of Student’s Ratio.”

Hulley, Stephen B, Steven R. Cummings, Warren S. Browner, Deborah G. Grady, and Thomas B. Newman. 2013. Designing Clinical Research, Fourth Edition. Lippincott Williams & Wilkins.

Kallis, P, JA Tooze, S Talbot, D Cowans, DH Bevan, and T Treasure. 1994. “Pre-Operative Aspirin Decreases Platelet Aggregation and Increases Post-Operative Blood Loss–a Prospective, Randomised, Placebo Controlled, Double-Blind Clinical Trial in 100 Patients with Chronic Stable Angina.” European Journal of Cardio-Thoracic Surgery: Official Journal of the European Association for Cardio-Thoracic Surgery 8 (8): 404–9.

Kaplan, Edward L, and Paul Meier. 1958. “Nonparametric Estimation from Incomplete Observations.” Journal of the American Statistical Association 53 (282): 457–81.

Kar, Sumit, Ajay Krishnan, Preetha K, and Atul Mohankar. 2012. “A Review of Antihistamines Used During Pregnancy.” Journal of Pharmacology and Pharmacotherapeutics 3 (2): 105–8.

Kassambara, Alboukadel. 2019. Datarium: Data Bank for Statistical Analysis and Visualization. https://CRAN.R-project.org/package=datarium.

Kendall, John. 2003. “Designing a Research Project: Randomised Controlled Trials and Their Principles.” Emergency Medicine Journal: EMJ 20 (2): 164.

Le-Rademacher, Jennifer G, Ryan A Peterson, Terry M Therneau, Ben L Sanford, Richard M Stone, and Sumithra J Mandrekar. 2018. “Application of Multi-State Models in Cancer Clinical Trials.” Clinical Trials 15 (5): 489–98.

Little, Roderick JA, and Donald B Rubin. 2019. Statistical Analysis with Missing Data. Vol. 793. John Wiley & Sons.

Marshall, Geoffrey. 1948. “STREPTOMYCIN TREATMENT OF PULMONARY TUBERCULOSIS a MEDICAL RESEARCH COUNCIL INVESTIGATION.” British Medical Journal. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2091872/pdf/brmedj03701-0007.pdf.

Matthews, John NS. 2006. Introduction to Randomized Controlled Clinical Trials. CRC Press.

Moertel, Charles G, Thomas R Fleming, John S Macdonald, Daniel G Haller, John A Laurie, Phyllis J Goodman, James S Ungerleider, et al. 1990. “Levamisole and Fluorouracil for Adjuvant Therapy of Resected Colon Carcinoma.” New England Journal of Medicine 322 (6): 352–58.

Newcombe, Robert G. 1998. “Interval Estimation for the Difference Between Independent Proportions: Comparison of Eleven Methods.” Statistics in Medicine 17 (8): 873–90.

Pocock, Stuart J, and Richard Simon. 1975. “Sequential Treatment Assignment with Balancing for Prognostic Factors in the Controlled Clinical Trial.” Biometrics, 103–15.

Rubin, Donald B. 1976. “Inference and Missing Data.” Biometrika 63 (3): 581–92.

Ruetzler, Kurt, Michael Fleck, Sabine Nabecker, Kristina Pinter, Gordian Landskron, Andrea Lassnigg, Jing You, and Daniel I Sessler. 2013. “A Randomized, Double-Blind Comparison of Licorice Versus Sugar-Water Gargle for Prevention of Postoperative Sore Throat and Postextubation Coughing.” Anesthesia & Analgesia 117 (3): 614–21.

Schoenfeld, David. 1982. “Partial Residuals for the Proportional Hazards Regression Model.” Biometrika 69 (1): 239–41.

Smith, AC, JF Dowsett, RCG Russell, ARW Hatfield, and PB Cotton. 1994. “Randomised Trial of Endoscopic Steriting Versus Surgical Bypass in Malignant Low Bileduct Obstruction.” The Lancet 344 (8938): 1655–60.

Spiegelhalter, David J, Keith R Abrams, and Jonathan P Myles. 2004. Bayesian Approaches to Clinical Trials and Health-Care Evaluation. Vol. 13. John Wiley & Sons.

Spiegelhalter, David J, Laurence S Freedman, and Mahesh KB Parmar. 1994. “Bayesian Approaches to Randomized Trials.” Journal of the Royal Statistical Society: Series A (Statistics in Society) 157 (3): 357–87.

Stan Development Team. 2024. “RStan: The R Interface to Stan.” https://mc-stan.org/.

Syriopoulou, Elisavet, Tove Wästerlid, Paul C Lambert, and Therese M-L Andersson. 2022. “Standardised Survival Probabilities: A Useful and Informative Tool for Reporting Regression Models for Survival Data.” British Journal of Cancer 127 (10): 1808–15.

Taves, Donald R. 1974. “Minimization: A New Method of Assigning Patients to Treatment and Control Groups.” Clinical Pharmacology & Therapeutics 15 (5): 443–53.

Teerenstra, Steven, Sandra Eldridge, Maud Graff, Esther de Hoop, and George F Borm. 2012. “A Simple Sample Size Formula for Analysis of Covariance in Cluster Randomized Trials.” Statistics in Medicine 31 (20): 2169–78.

Thall, Peter F, and Stephen C Vail. 1990. “Some Covariance Models for Longitudinal Count Data with Overdispersion.” Biometrics, 657–71.

Therneau, Terry M. 2024. A Package for Survival Analysis in r. https://CRAN.R-project.org/package=survival.

Treasure, Tom, and Kenneth D MacRae. 1998. “Minimisation: The Platinum Standard for Trials?: Randomisation Doesn’t Guarantee Similarity of Groups; Minimisation Does.” Bmj. British Medical Journal Publishing Group.

Villar, Jesús, Carlos Ferrando, Domingo Martı́nez, Alfonso Ambrós, Tomás Muñoz, Juan A Soler, Gerardo Aguilar, et al. 2020. “Dexamethasone Treatment for the Acute Respiratory Distress Syndrome: A Multicentre, Randomised Controlled Trial.” The Lancet Respiratory Medicine 8 (3): 267–76.

Vitale, Cristiana, Massimo Fini, Ilaria Spoletini, Mitja Lainscak, Petar Seferovic, and Giuseppe MC Rosano. 2017. “Under-Representation of Elderly and Women in Clinical Trials.” International Journal of Cardiology 232: 216–21.

Wei, LJ. 1978. “An Application of an Urn Model to the Design of Sequential Controlled Clinical Trials.” Journal of the American Statistical Association 73 (363): 559–63.

Wilson, Edwin B. 1927. “Probable Inference, the Law of Succession, and Statistical Inference.” Journal of the American Statistical Association 22 (158): 209–12.

Zhong, Baoliang. 2009. “How to Calculate Sample Size in Randomized Controlled Trial?” Journal of Thoracic Disease 1 (1): 51.