Advanced Issues in CR

in preparation. For suggestions, contact sergio.uribe@rsu.lv

Advanced Issues in Clinical Research

How many patients?

Jones SR, Carley S and Harrison M (2003) An introduction to power and sample size estimation. Emergency medicine journal: EMJ 20(5): 453–458.

Cook JA, Julious SA, Sones W, et al. (2018) DELTA2 guidance on choosing the target difference and undertaking and reporting the sample size calculation for a randomised controlled trial. BMJ 363: k3750.

Cook JA, Julious SA, Sones W, et al. (2017) Choosing the target difference (’effect size') for a randomised controlled trial - DELTA2 guidance protocol. Trials 18(1): 271.

Small trials: Althouse AD, Yabes JG and Abebe KZ (2021) Issues in designing and interpreting small clinical trials. The Canadian journal of cardiology. Elsevier BV. DOI: 10.1016/j.cjca.2021.03.013.

Bias

Flanagin A, Frey T, Christiansen SL, et al. (2021) The Reporting of Race and Ethnicity in Medical and Science Journals: Comments Invited. JAMA: the journal of the American Medical Association 325(11): 1049–1052.

The analysis of the data from clinical trials

• The essential: Exploratory Data analysis. Check for the "Mastering your data" course from the Doctoral School RSU and r4ds

• Bayesian analysis of clinical trials

• Frequentist analysis of clinical trials

• R package for the statistical report of clinical trials: hreport, gtsummary

Repeated measures

Guo Y, Logan HL, Glueck DH, et al. (2013) Selecting a sample size for studies with repeated measures. BMC medical research methodology 13(1): 100.

Dealing with missing data

Donders ART, van der Heijden GJMG, Stijnen T, et al. (2006) Review: a gentle introduction to imputation of missing values. Journal of clinical epidemiology 59(10): 1087–1091.

Little RJ, D’Agostino R, Cohen ML, et al. (2012) The Prevention and Treatment of Missing Data in Clinical Trials. The New England journal of medicine 367(14): 1355–1360.

Groenwold RHH, Donders ART, Roes KCB, et al. (2012) Dealing with missing outcome data in randomized trials and observational studies. American journal of epidemiology 175(3): 210–217.

Survival analysis

Bewick V, Cheek L and Ball J (2004) Statistics review 12: survival analysis. Critical care / the Society of Critical Care Medicine 8(5): 389–394.

Personalized Medicine and Adaptative Clinical trials

Pallmann P, Bedding AW, Choodari-Oskooei B, et al. (2018) Adaptive designs in clinical trials: why use them, and how to run and report them. BMC medicine 16(1): 29.

Garralda E, Dienstmann R, Piris-Giménez A, et al. (2019) New clinical trial designs in the era of precision medicine. Molecular oncology 13(3): 549–557.

Razzouk S and Termechi O (2013) Host genome, epigenome, and oral microbiome interactions: toward personalized periodontal therapy. Journal of periodontology 84(9): 1266–1271.

Kent DM, Steyerberg E and van Klaveren D (2018) Personalized evidence based medicine: predictive approaches to heterogeneous treatment effects. BMJ 363: k4245.

Predictive and Prognostic modelling

Prediction Research Janssens

Hemingway H, Croft P, Perel P, et al. (2013) Prognosis research strategy (PROGRESS) 1: a framework for researching clinical outcomes. BMJ 346: e5595.

Riley RD, Hayden JA, Steyerberg EW, et al. (2013) Prognosis Research Strategy (PROGRESS) 2: prognostic factor research. PLoS medicine 10(2): e1001380.

Steyerberg EW, Moons KGM, van der Windt DA, et al. (2013) Prognosis Research Strategy (PROGRESS) 3: prognostic model research. PLoS medicine 10(2): e1001381.

Hingorani AD, Windt DA van der, Riley RD, et al. (2013) Prognosis research strategy (PROGRESS) 4: stratified medicine research. BMJ 346: e5793.

Rules for interim analysis

Schulz KF and Grimes DA (2005) 20 Multiplicity in randomised trials II: subgroup and interim analyses. The Lancet 365(9471): 1657–1661.

Pilot and feasibility trials

Lancaster GA and Thabane L (2019) Guidelines for reporting non-randomised pilot and feasibility studies. Pilot and feasibility studies 5: 114.

Lancaster GA, Dodd S and Williamson PR (2004) Design and analysis of pilot studies: recommendations for good practice. Journal of evaluation in clinical practice 10(2): 307–312.

Moore CG, Carter RE, Nietert PJ, et al. (2011) Recommendations for planning pilot studies in clinical and translational research. Clinical and translational science 4(5): 332–337.

Harms

Phillips R, Cro S, Wheeler G, Bond S, Morris TP, Creanor S, et al. Visualising harms in publications of randomised controlled trials: consensus and recommendations. BMJ. 2022;377:e068983.

Other issues

Institute of Medicine (US) Roundtable on Value & Science-Driven Health Care (2011) Redesigning the Clinical Effectiveness Research Paradigm: Innovation and Practice-Based Approaches: Workshop Summary (L Olsen and JM McGinniseds ). Washington (DC): National Academies Press (US).

Del Paggio JC, Berry JS, Hopman WM, et al. (2021) Evolution of the Randomized Clinical Trial in the Era of Precision Oncology. JAMA Oncology. DOI: 10.1001/jamaoncol.2021.0379.

Clinical National Trials, see https://www.recoverytrial.net/