Adding flexibility to clinical trial designs: an example-based guide to the practical use of adaptive designs

Burnett, T., Mozgunov, P., Pallmann, P., Villar, S. S., Wheeler, G. M. & Jaki, T., 19/11/2020, In : BMC Medicine. 18, 1, 21 p., 352.

Research output: Contribution to journal › Journal article

An information-theoretic approach for selecting arms in clinical trials

Mozgunov, P. & Jaki, T. F., 10/11/2020, In : Journal of the Royal Statistical Society: Series B (Statistical Methodology). 82, 5, p. 1223-1247 25 p.

Research output: Contribution to journal › Journal article

A Benchmark for Dose Finding Studies with Unknown Ordering

Mozgunov, P., Paoletti, X. & Jaki, T., 9/11/2020, (Accepted/In press) In : Biostatistics.

Research output: Contribution to journal › Journal article

Using a Dose-Finding Benchmark to Quantify the Loss Incurred by Dichotomisation in Phase II Dose-Ranging Studies

Mozgunov, P., Jaki, T. & Paoletti, X., 1/11/2020, In : Biometrical Journal. 62, 7, p. 1717-1729 13 p.

Research output: Contribution to journal › Journal article

Using a dose-finding benchmark to quantify the loss incurred by dichotomization in Phase II dose-ranging studies

Mozgunov, P., Jaki, T. & Paoletti, X., 1/11/2020, In : Biometrical Journal. 62, 7, p. 1717-1729 13 p.

Research output: Contribution to journal › Journal article

Assessing goodness-of-fit for evaluation of dose-proportionality

Wolfsegger, M. J., Bauer, A., Labes, D., Schütz, H., Vonk, R., Lang, B., Lehr, S., Jaki, T. F., Engl, W. & Hale, M. D., 15/10/2020, In : Pharmaceutical Statistics. 10 p.

Research output: Contribution to journal › Journal article

A comparison of phase I dose-finding designs in clinical trials with monotonicity assumption violation

Abbas, R., Rossoni, C., Jaki, T., Paoletti, X. & Mozgunov, P., 1/10/2020, In : Clinical Trials. 17, 5, p. 522-534 13 p.

Research output: Contribution to journal › Journal article

A surface-free design for phase I dual-agent combination trials

Mozgunov, P., Gasparini, M. & Jaki, T., 1/10/2020, In : Statistical Methods in Medical Research. 29, 10, p. 3093-3109 17 p.

Research output: Contribution to journal › Journal article

Efficient Adaptive Designs for Clinical Trials of Interventions for COVID-19

Stallard, N., Hampson, L., Benda, N., Brannath, W., Burnett, T., Friede, T., Kimani, P. K., Koenig, F., Krisam, J., Mozgunov, P., Posch, M., Wason, J., Wassmer, G., Whitehead, J., Williamson, S. F., Zohar, S. & Jaki, T., 1/10/2020, In : Statistics in Biopharmaceutical Research. 12, 4, p. 483-497 15 p.

Research output: Contribution to journal › Journal article

Endpoints for randomized controlled clinical trials for COVID-19 treatments

Dodd, L. E., Follmann, D., Wang, J., Koenig, F., Korn, L. L., Schoergenhofer, C., Proschan, M., Hunsberger, S., Bonnett, T., Makowski, M., Belhadi, D., Wang, Y., Cao, B., Mentre, F. & Jaki, T., 1/10/2020, In : Clinical Trials. 17, 5, p. 472-482 11 p.

Research output: Contribution to journal › Journal article

A Critical Review of Graphics for Subgroup Analyses in Clinical Trials

Ballarini, N. M., Chiu, Y-D., Koenig, F., Posch, M. & Jaki, T., 1/09/2020, In : Pharmaceutical Statistics. 19, 5, p. 541-560 20 p.

Research output: Contribution to journal › Journal article

Exposure–response modelling approaches for determining optimal dosing rules in children

Wadsworth, I., Hampson, L. V., Bornkamp, B. & Jaki, T., 1/09/2020, In : Statistical Methods in Medical Research. 29, 9, p. 2583-2602 20 p.

Research output: Contribution to journal › Journal article

AGILE-ACCORD: A Randomized, Multicentre, Seamless, Adaptive Phase I/II Platform Study to Determine the Optimal Dose, Safety and Efficacy of Multiple Candidate Agents for the Treatment of COVID-19: A structured summary of a study protocol for a randomised platform trial

UK NIHR Community, 19/06/2020, In : Trials. 21, 1, 3 p., 544.

Research output: Contribution to journal › Journal article

The adaptive designs CONSORT extension (ACE) statement: a checklist with explanation and elaboration guideline for reporting randomised trials that use an adaptive design

Dimairo, M., Pallmann, P., Wason, J., Todd, S., Jaki, T., Julious, S. A., Mander, A. P., Weir, C. J., Koenig, F., Walton, M. K., Nicholl, J. P., Coates, E., Biggs, K., Hamasaki, T., Proschan, M. A., Scott, J. A., Ando, Y., Hind, D., Altman, D. G. & Group, ACE. C., 17/06/2020, In : Trials. 21, 1, 34 p., 528.

Research output: Contribution to journal › Journal article

The Adaptive designs CONSORT Extension (ACE) statement: a checklist with explanation and elaboration guideline for reporting randomised trials that use an adaptive design

Dimairo, M., Pallmann, P., Wason, J., Todd, S., Jaki, T., Julious, S. A., Mander, A. P., Weir, C. J., Koenig, F., Walton, M. K., Nicholl, J. P., Coates, E., Biggs, K., Hamasaki, T., Proschan, M. A., Scott, J. A., Ando, Y., Hind, D., Altman, D. G. & Group, ACE. C., 17/06/2020, In : BMJ. 369, 31 p., m115.

Research output: Contribution to journal › Journal article

Evaluation of the efficacy and safety of intravenous remdesivir in adult patients with severe COVID-19: Study protocol for a phase 3 randomized, double-blind, placebo-controlled, multicentre trial

Wang, Y., Zhou, F., Zhang, D., Zhao, J., Du, R., Hu, Y., Cheng, Z., Gao, L., Jin, Y., Luo, G., Fu, S., Lu, Q., Du, G., Wang, K., Lu, Y., Fan, G., Zhang, Y., Liu, Y., Ruan, S., Liu, W. & 5 others, Jaki, T., Hayden, F. G., Horby, P. W., Cao, B. & Wang, C., 24/05/2020, In : Trials. 21, 1, 11 p., 422.

Research output: Contribution to journal › Journal article

Estimation of treatment effects following a sequential trial of multiple treatments

Whitehead, J., Desai, Y. & Jaki, T., 20/05/2020, In : Statistics in Medicine. 39, 11, p. 1593-1609 17 p.

Research output: Contribution to journal › Journal article

Remdesivir in adults with severe COVID-19: a randomised, double-blind, placebo-controlled, multicentre trial

Wang, Y., Zhang, D., Du, G., Du, R., Zhao, J., Jin, Y., Fu, S., Gao, L., Cheng, Z., Lu, Q., Hu, Y., Luo, G., Wang, K., Lu, Y., Li, H., Wang, S., Ruan, S., Yang, C., Mei, C., Ding, D. & 25 others, Wu, F., Tang, X., Ye, X., Ye, Y., Liu, B., Yang, J., Yin, W., Wang, A., Fan, G., Zhou, F., Liu, Z., Gu, X., Xu, J., Shang, L., Zhang, Y., Cao, L., Guo, T., Wan, Y., Qin, H., Jiang, Y., Jaki, T., Hayden, F. G., Horby, P. W., Cao, B. & Wang, C., 16/05/2020, In : The Lancet. p. 1569-1578 10 p.

Research output: Contribution to journal › Journal article

TAILoR (TelmisArtan and InsuLin Resistance in Human Immunodeficiency Virus [HIV]): An Adaptive-design, Dose-ranging Phase IIb Randomized Trial of Telmisartan for the Reduction of Insulin Resistance in HIV-positive Individuals on Combination Antiretroviral Therapy

Pushpakom, S., Kolamunnage-Dona, R., Taylor, C., Foster, T., Spowart, C., García-Fiñana, M., Kemp, G. J., Jaki, T., Khoo, S., Williamson, P. & Pirmohamed, M., 15/05/2020, In : Clinical infectious diseases : an official publication of the Infectious Diseases Society of America. 70, 10, p. 2062-2072 11 p.

Research output: Contribution to journal › Journal article

A comparison of stochastic programming methods for portfolio level decision-making

Graham, E., Jaki, T. & Harbron, C., 1/05/2020, In : Journal of Biopharmaceutical Statistics. 30, 3, p. 405-429 25 p.

Research output: Contribution to journal › Journal article

Creating a framework for conducting randomized clinical trials during disease outbreaks

Dean, N. E., Gsell, P-S., Brookmeyer, R., Crawford, F. W., Donnelly, C. A., Ellenberg, S. S., Fleming, T. R., Halloran, E. M., Horby, P., Jaki, T., Krause, P. R., Longini, I. M., Mulangu, S., Muyembe-Tamfum, J-J., Nason, M. C., Smith, P. G., Wang, R., Henao-Restrepo, A. M. & De Gruttola, V., 2/04/2020, In : New England Journal of Medicine. 382, 14, p. 1366-1369 4 p.

Research output: Contribution to journal › Editorial

A benchmark for dose finding studies with continuous outcomes

Mozgunov, P., Jaki, T. F. & Paoletti, X., 1/04/2020, In : Biostatistics. 21, 2, p. 189–201 13 p.

Research output: Contribution to journal › Journal article

Designing and evaluating dose-escalation studies made easy: The MoDEsT web app

Pallmann, P., Wan, F., Mander, A. P., Wheeler, G. M., Yap, C., Clive, S., Hampson, L. V. & Jaki, T., 1/04/2020, In : Clinical Trials. 17, 2, p. 147-156 10 p.

Research output: Contribution to journal › Journal article

Repeated measures regression mixture models

Kim, M., Van Horn, M. L., Jaki, T., Vermunt, J., Feaster, D., Lichstein, K. L., Taylor, D. J., Riedel, B. W. & Bush, A. J., 1/04/2020, In : Behavior Research Methods. 52, 2, p. 591-606 16 p.

Research output: Contribution to journal › Journal article

Improving safety of the continual reassessment method via a modified allocation rule

Mozgunov, P. & Jaki, T., 30/03/2020, In : Statistics in Medicine. 39, 7, p. 906-922 17 p.

Research output: Contribution to journal › Journal article

A randomised controlled trial of rosuvastatin for the prevention of aminoglycoside-induced kidney toxicity in children with cystic fibrosis

McWilliam, S. J., Rosala-Hallas, A., Jones, A. P., Shaw, V., Greenhalf, W., Jaki, T., Smyth, A. R., Smyth, R. L. & Pirmohamed, M., 4/02/2020, In : Scientific Reports. 10, 1, 9 p., 1796.

Research output: Contribution to journal › Journal article

A quantitative framework to inform extrapolation decisions in children

Wadsworth, I., Hampson, L. V., Jaki, T., Sills, G. J., Marson, A. G. & Appleton, R., 1/02/2020, In : Journal of the Royal Statistical Society: Series A Statistics in Society. 183, 2, p. 515-534 20 p.

Research output: Contribution to journal › Journal article

Methods for Non-Compartmental Pharmacokinetic Analysis With Observations Below the Limit of Quantification

Barnett, H. Y., Geys, H., Jacobs, T. & Jaki, T., 21/01/2020, In : Statistics in Biopharmaceutical Research.

Research output: Contribution to journal › Journal article

Toward Accelerated Authorization and Access to New Medicines for Juvenile Idiopathic Arthritis

Schanberg, L. E., Ramanan, A. V., De Benedetti, F., Beukelman, T., Eakin, G. S., Del Gaizo, V., Ringold, S., Vesely, R., Schrandt, S., Jaki, T., Bili, A., Chung, J. B., De Bono, S., Douglass, W., Enejosa, J. V., Kanik, K. S., Knobe, K., Kunder, R., Leite-Schnell, J. C., Suehiro, R. M. & 6 others, Wong, R. L., Mieszkalski, K. L., Marrow, L. C., Siebenaler, K., Fraulo, E. & Kimura, Y., 1/12/2019, In : Arthritis and Rheumatology. 71, 12, p. 1976-1984 9 p.

Research output: Contribution to journal › Journal article

A flexible design for advanced Phase I/II clinical trials with continuous efficacy endpoints

Mozgunov, P. & Jaki, T., 1/11/2019, In : Biometrical Journal. 61, 6, p. 1477-1492 16 p.

Research output: Contribution to journal › Journal article

Subgroup analysis of treatment effects for misclassified biomarkers with time-to-event data

Wan, F., Titman, A. & Jaki, T., 1/11/2019, In : Journal of the Royal Statistical Society: Series C (Applied Statistics). 68, 5, p. 1447-1463 17 p.

Research output: Contribution to journal › Journal article

Loss Functions in Restricted Parameter Spaces and Their Bayesian Applications

Mozgunov, P., Jaki, T. F. & Gasparini, M., 3/10/2019, In : Journal of Applied Statistics. 46, 13, p. 2314-2337 24 p.

Research output: Contribution to journal › Journal article

A novel measure of drug benefit–risk assessment based on Scale Loss Score

Saint-Hilary, G., Robert, V., Gasparini, M., Jaki, T. F. & Mozgunov, P., 1/09/2019, In : Statistical Methods in Medical Research. 28, 9, p. 2738-2753 16 p.

Research output: Contribution to journal › Journal article

A Bayesian model to estimate the cutoff and the clinical utility of a biomarker assay

Vradi, E., Jaki, T. F., Vonk, R. & Brannath, W., 1/08/2019, In : Statistical Methods in Medical Research. 28, 8, p. 2538-2556 19 p.

Research output: Contribution to journal › Journal article

Bayesian sequential integration within a preclinical pharmacokinetic and pharmacodynamic modeling framework: Lessons learned

La Gamba, F., Jacobs, T., Geys, H., Jaki, T., Serroyen, J., Ursino, M., Russu, A. & Faes, C., 1/07/2019, In : Pharmaceutical Statistics. 18, 4, p. 486-506 21 p.

Research output: Contribution to journal › Journal article

Model-Based dose escalation Designs in R with crmPack

Bové, D. S., Yeung, W. Y., Palermo, G. & Jaki, T., 13/06/2019, In : Journal of Statistical Software. 89, 10, 22 p.

Research output: Contribution to journal › Journal article

Response to comments on Jaki et al., A proposal for a new PhD level curriculum on quantitative methods for drug development. Pharm Stat 17(5):593-606, Sep/Oct 2018., DOI https://doi.org/10.1002/pst.1873

Jaki, T., Gordon, A., Forster, P., Bijnens, L., Bornkamp, B., Brannath, W., Fontana, R., Gasparini, M., Hampson, L. V., Jacobs, T., Jones, B., Paoletti, X., Posch, M., Titman, A., Vonk, R. & Koenig, F., 1/05/2019, In : Pharmaceutical Statistics. 18, 3, p. 284-286 3 p.

Research output: Contribution to journal › Letter

The effects of sample size on the estimation of regression mixture models

Jaki, T. F., Kim, M., Lamont, A. E., George, M., Feaster, D. & Van Horn, M. L., 1/04/2019, In : Educational and Psychological Measurement. 79, 2, p. 358-384 27 p.

Research output: Contribution to journal › Journal article

Instrumental variable estimation in semi-parametric additive hazards models

Brueckner, M., Titman, A. C. & Jaki, T. F., 1/03/2019, In : Biometrics. 75, 1, p. 110-120 11 p.

Research output: Contribution to journal › Journal article

The R package MAMS for designing multi-arm multi-stage clinical trials

Jaki, T. F., Pallmann, P. S. & Magirr, D., 31/01/2019, In : Journal of Statistical Software. 88, 4, 25 p.

Research output: Contribution to journal › Journal article

How to design a dose-finding study using the continual reassessment method

Wheeler, G. M., Mander, A. P., Bedding, A., Brock, K., Cornelius, V., Grieve, A. P., Jaki, T., Love, S. B., Odondi, L., Weir, C. J., Yap, C. & Bond, S. J., 18/01/2019, In : BMC Medical Research Methodology. 19, 1, 15 p., 18.

Research output: Contribution to journal › Journal article

Editorial for the MCP 2017 Special Issue

Cui, X., Hsu, J. C. & Jaki, T., 11/01/2019, In : Biometrical Journal. 61, 1, p. 7 1 p.

Research output: Contribution to journal › Editorial

Confidence regions for treatment effects in subgroups in biomarker stratified designs

Wan, F., Kunz, C. U. & Jaki, T. F., 1/01/2019, In : Biometrical Journal. 61, 1, p. 27-39 13 p.

Research output: Contribution to journal › Journal article

An alternative method to analyse the Biomarker-strategy design

Kunz, C. U., Jaki, T. F. & Stallard, N., 30/12/2018, In : Statistics in Medicine. 37, 30, p. 4636-4651 16 p.

Research output: Contribution to journal › Journal article

Design and estimation in clinical trials with subpopulation selection

Chiu, Y-D., Koenig, F., Posch, M. & Jaki, T. F., 20/12/2018, In : Statistics in Medicine. 37, 29, p. 4335-4352 18 p.

Research output: Contribution to journal › Journal article

Multi-arm multi-stage trials can improve the efficiency of finding effective treatments for stroke: a case study

Jaki, T. & Wason, J. M. S., 27/11/2018, In : BMC Cardiovascular Disorders. 18, 1, 8 p., 215.

Research output: Contribution to journal › Journal article

Development process of a consensus-driven CONSORT extension for randomised trials using an adaptive design

Dimairo, M., Coates, E., Pallmann, P., Todd, S., Julious, S. A., Jaki, T., Wason, J., Mander, A. P., Weir, C. J., Koenig, F., Walton, M. K., Biggs, K., Nicholl, J., Hamasaki, T., Proschan, M. A., Scott, J. A., Ando, Y., Hind, D. & Altman, D. G., 16/11/2018, In : BMC Medicine. 16, 1, 20 p.

Research output: Contribution to journal › Journal article

Randomized dose-escalation designs for drug combination cancer trials with immunotherapy

Mozgunov, P., Jaki, T. F. & Paoletti, X., 23/10/2018, In : Journal of Biopharmaceutical Statistics. 29, 2, p. 359-377 19 p.

Research output: Contribution to journal › Journal article

Subgroup identification in clinical trials via the predicted individual treatment effect

Ballarini, N. M., Rosenkranz, G. K., Jaki, T., König, F. & Posch, M., 18/10/2018, In : PLoS ONE. 13, 10, 22 p., e0205971.

Research output: Contribution to journal › Journal article

Performance of different clinical trial designs to evaluate treatments during an epidemic

Brueckner, M., Titman, A. C., Jaki, T. F., Rojek, A. & Horby, P. W., 11/09/2018, In : PLoS ONE. 13, 9, 19 p., e0203387.

Research output: Contribution to journal › Journal article