References and resources for clinical trials

References

A short list of publications related to clinical trials are given below. This is by no means an exhaustive list nor does it include all useful or important references. It is a list of sources written by others which I’ve found helpful in my professional life working in clinical trials for over 20 years or that I found made for an enjoyable read. Some of these publications were referenced in my book, but a number of others are not.

Listing here does not imply an endorsement of everything said in the respective work. The focus and technical level varies. This list will expand over time.

History of clinical trials

Armitage P. Fisher, Bradford Hill, and randomization. International Journal of Epidemiology. 2003;32(6):925–8.

Doll R. Clinical trials: retrospect and prospect. Statistics in Medicine. 1982;1(4):337–44.

Hill AB. The clinical trial. New England Journal of Medicine. 1952;247:113–19.

Meldrum ML. A brief history of the randomized controlled trial. From oranges and lemons to the gold standard. Hematology/ Oncology Clinics of North America. 2000;14(4):745–60, vii.

Study design

Armitage P. The role of randomization in clinical trials. Statistics in Medicine. 1982;1(4):345–52.

Cook JA, Julious SA, Sones W, Hampson LV, Hewitt C, Berlin JA, Ashby D, Emsley R, Fergusson DA, Walkters SJ, Wilson ECF, MacLennan G, Stallard N, Rothwell JC, Bland M, Brown L, Ramsay CR, Cook A, Armstrong D, Altman D, Vale LV. DELTA2 guidance on choosing the target difference and undertaking and reporting the sample size calculation for a randomised controlled trial. BMJ. 2018;363:k3750. doi:10.1136/bmj.k3750.

Julious S. Sample Sizes for Clinical Trials. Boca Raton, FL: Chapman and Hall/ CRC Press; 2010.

Kempthorne O. Why randomize? Journal of Statistical Planning and Inference. 1977;1(1):1–25.

Pallmann P, Bedding AW, Choodari- Oskooei B, Dimairo M, Flight L, Hampson LV, et al. Adaptive designs in clinical trials: why use them, and how to run and report them. BMC Medicine. 2018;16(1):29.

Peto R, Pike MC, Armitage P, Breslow NE, Cox DR, Howard SV, Mantel N, McPherson K, Peto J, Smith PG. Design and analysis of randomized clinical trials requiring prolonged observation of each patient. I. Introduction and design. British Journal of Cancer. 1976;34(6):585-612. doi: 10.1038/bjc.1976.220.

Senn S. Cross-over Trials in Clinical Research. 2nd ed. Chichester: Wiley; 2002.

Study conduct

Farrell B, Kenyon S, Shakur H. Managing clinical trials. Trials. 2010;11:78.

Statistical analysis

Peto R, Pike MC, Armitage P, Breslow NE, Cox DR, Howard SV, Mantel N, McPherson K, Peto J, Smith PG. Design and analysis of randomized clinical trials requiring prolonged observation of each patient. II. analysis and examples. Br J Cancer. 1977 Jan;35(1):1-39. doi: 10.1038/bjc.1977.1.

Pocock SJ, McMurray JJ, Collier TJ. Making Sense of Statistics in Clinical Trial Reports: Part 1 of a 4-Part Series on Statistics for Clinical Trials. J Am Coll Cardiol. 2015 Dec 8;66(22):2536-49. doi: 10.1016/j.jacc.2015.10.014.

Pocock SJ, McMurray JJV, Collier TJ. Statistical Controversies in Reporting of Clinical Trials: Part 2 of a 4-Part Series on Statistics for Clinical Trials. J Am Coll Cardiol. 2015 Dec 15;66(23):2648-2662. doi: 10.1016/j.jacc.2015.10.023.

Pocock SJ, Clayton TC, Stone GW. Design of Major Randomized Trials: Part 3 of a 4-Part Series on Statistics for Clinical Trials. J Am Coll Cardiol. 2015 Dec 22;66(24):2757-2766. doi: 10.1016/j.jacc.2015.10.036.

Pocock SJ, Clayton TC, Stone GW. Challenging Issues in Clinical Trial Design: Part 4 of a 4-Part Series on Statistics for Clinical Trials. J Am Coll Cardiol. 2015 Dec 29;66(25):2886-2898. doi: 10.1016/j.jacc.2015.10.051.

Stone GW, Pocock SJ. Randomized trials, statistics, and clinical inference. Journal of the American College of Cardiology. 2010;55(5):428– 31.

Useful websites

• Cluster Randomised Trials including Stepped Wedge: Resources to support good quality research in cluster randomised trials and stepped wedge designs
• CONSORT and SPIRIT Statements: Reporting guidelines for clinical trials and their protocols
• DELTA2: Guidance on treatment effects for trials
• EQUATOR Network: Reporting guidelines for health research and resources on writing up health research 
• ICH guidelines: The International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use (ICH) brings together regulatory authorities and the pharmaceutical industry to discuss scientific and technical aspects of drug registration. ICH's mission is to achieve greater harmonisation worldwide to ensure that safe, effective, and high quality medicines are developed and registered in the most resource-efficient manner.
• James Lind Library: Illustrating the development of fair tests of treatments in health care
• PANDA - Home (shef.ac.uk) A practical adaptive & Novel Designs and Analysis toolkit
• Society for Clinical Trials
• TwiCs | Trials within Cohorts

SHORT COURSES

• RANDOMISED CONTROLLED TRIALS COURSE: A Guide to Design, Conduct, Analysis, Interpretation and Reporting — Nuffield Department of Orthopaedics, Rheumatology and Musculoskeletal Sciences (ox.ac.uk)
• Clinical Trial Management | Oxford University Department for Continuing Education