Recent Publications


An adaptive population enrichment phase III trial of TRC105 and pazopanib versus pazopanib alone in patients with advanced angiosarcoma (TAPPAS trial)
Annals of Oncology
C R Mehta, L Liu, C Theuer

ADaM Intermediate Dataset: how to improve your analysis traceability
PhUSE US Connect 2018, Paper SD16
Tinazzi, A. Curto, T. and Aggarwal, A.

Confidence, credibility and prediction.
METRON, 76(2), pp.251-268
Aitkin, M. and Liu, C.


On Shapes of ADR Report Accumulation Data for Banned Drugs.
Current Science, 113(09), p.1664.
Ghubade, S., Asvalayan, K., Paranjpe, S., Gupta, K. and Gore, A.

Efficacy and Safety of MED2005, a Topical Glyceryl Trinitrate Formulation, in the Treatment of Erectile Dysfunction: A Randomized Crossover Study.
Sex Med
Ralph, DJ., Eardley, I., Taubel, J., Terrill, P. and Holland T.

Serial Measurement of High-Sensitivity Troponin I and Cardiovascular Outcomes in Patients With Type 2 Diabetes Mellitus in the EXAMINE Trial (Examination of Cardiovascular Outcomes With Alogliptin Versus Standard of Care)Clinical Perspective.
Circulation, 135(20), pp.1911-1921.
Cavender, M., White, W., Jarolim, P., Bakris, G., Cushman, W., Kupfer, S., Gao, Q., Mehta, C.R., Zannad, F., Cannon, C. and Morrow, D.

Relationship of glycated haemoglobin and reported hypoglycaemia to cardiovascular outcomes in patients with type 2 diabetes and recent acute coronary syndrome events: The EXAMINE trial.
Diabetes, Obesity and Metabolism, 19(5), pp.664-671.
Heller, S., Bergenstal, R., White, W., Kupfer, S., Bakris, G., Cushman, W., Mehta, C.R., Nissen, S., Wilson, C., Zannad, F., Liu, Y., Gourlie, N. and Cannon, C.

Topical safety and vasoconstrictive assay-based bioequivalence of a new reformulated mometasone cream.
Krishna, R., Horowitz, A., Larson, P., Bolognese, J. and Marcantonio, E.Mehta, C.R.

A gatekeeping procedure to test a primary and a secondary endpoint in a group sequential design with multiple interim looks.
Tamhane, A., Gou, J., Jennison, C., Mehta, C.R. and Curto, T.

Efficiency Considerations for Group Sequential Designs with Adaptive Unblinded Sample Size Re-assessment.
Statistics in Biosciences.
Liu, L., Hsiao, S. and Mehta, C.R.

Optimizing adaptive design for Phase 2 dose-finding trials incorporating long-term success and financial considerations: A case study for neuropathic pain.
Contemporary Clinical Trials, 57, pp.69-86.
Gao, J., Nangia, N., Jia, J., Bolognese, J., Bhattacharyya, J. and Patel, N.

The Case for a Bayesian Approach to Benefit-Risk Assessment.
Therapeutic Innovation & Regulatory Science
Costa, M., He, W., Jemiai, Y., Zhao, Y. and Casoli, C.

Design and monitoring of multi-arm multi-stage clinical trials. Recognized as a top 20 downloaded paper by Wiley
Biometrics. doi:10.1111/biom.12687
Ghosh, P., Liu, L., Senchaudhuri, P., Gao, P. and Mehta, C.R.


An objective re-evaluation of adaptive sample size re-estimation: Commentary on “Twenty-five years of confirmatory adaptive designs”.
Statistics in Medicine, 35(3), pp. 350–358.
Mehta, C.R. and Liu, L.

A randomized, phase 2 study investigating TRV130, a biased ligand of the μ-opioid receptor, for the intravenous treatment of acute pain.
Pain, 157, no. 1 264-272.
Viscusi, Eugene R., Bolognese, J., et al.

Cardiovascular mortality in patients with type 2 diabetes and recent acute coronary Syndromes from the EXAMINE trial.
Cardiovascular and Metabolic Risk, 39(7), pp. 1267–1273.
White, W.B., Zannad, F., Mehta, C.R., et al.

Angiotensin-converting enzyme inhibitor use and major cardiovascular outcomes in type 2 diabetes Mellitus treated with the Dipeptidyl Peptidase 4 inhibitor Alogliptin Novelty and significance.
Hypertension, 68(3), pp. 606–613.
White, W.B., Wilson, C.A., Mehta, C.R., Nissen, S.E., Zannad, F., et al.

Adaptive designs for clinical trials.
New England Journal of Medicine, 375(1), pp. 65–74.
Bhatt, D.L. and Mehta, C.R.

Addressing challenges and opportunities of “Less Well-Understood” Adaptive Designs.
Therapeutic Innovation & Regulatory Science.
He, W., Gallo, P., Miller, E., Jemiai, Y., et al.

Comments on: “Some Challenges with Statistical Inference in Adaptive Designs”, by Hung, Wang, and Yang.".
Journal of Biopharmaceutical Statistics, 26, no. 2 402-404.
Mehta, C.R.

DIA’s Adaptive Design Scientific Working Group (ADSWG) Best Practices Case Studies for “Less Well-understood” Adaptive Designs.
Therapeutic Innovation & Regulatory Science.
Miller, Eva, Gallo, Paul, Paul, He, Weili, Jemiai, Y., et al.

Methodological extensions of phase 2 trial designs based on program-level considerations: Further development of a case study in Neuropathic pain.
Therapeutic Innovation & Regulatory Science.
Bolognese, J., Bhattacharyya, J., Assaid, C. and Patel, N.R. 


Efficacy and safety of repeated use of ulipristal acetate in uterine fibroids.
Fertil Steril. 2015;103(2):519-527
Donnez, J., Hudecek, R., Donnez, O., Matule, D., Arhendt, HJ., Zatik, J., Kasilovskiene, Z., Dumitrascu, MC., Fernandez, H., Barlow, DH., Bouchard, P., Fauser, BCJM., Bestel, E., Terrill, P., Osterloh, I. and Loumaye, E.

Dynamically optimizing budget allocation for phase 3 drug development portfolios incorporating uncertainty in the pipeline.
Patel, N.R. and Ankolekar, S.

Drug development and the cost of capital.
Chapters from: Optimization of Pharmaceutical R&D Programs and Portfolios pp. 35-47.

Schulz, Kraig F., Sarah T. Bobulsky, Frank S. David, Patel, N.R., and Antonijevic, Z.Bhattacharyya, J.

Vosaroxin plus cytarabine versus placebo plus cytarabine in patients with first relapsed or refractory acute myeloid leukaemia (VALOR): A randomised, controlled, double-blind, multinational, phase 3 study.
The Lancet Oncology, 16, no. 9 1025-1036.
Ravandi, Farhad, Ellen K. Ritchie, Hamid Sayar, Jeffrey E. Lancet, Michael D. Craig, Mehta, C.R., et al.

Heart failure and mortality outcomes in patients with type 2 diabetes taking alogliptin vs. placebo in EXAMINE: a multicentre, randomised, double-blind trial.
The Lancet, 385, no. 9982 2067-2076.
Zannad, Faiez, Christopher P. Cannon, Mehta, C.R., et al.

A randomized, controlled trial of oral propranolol in infantile hemangioma.
New England Journal of Medicine, 372, no. 8 735-746.
Léauté-Labrèze, Christine, Peter Hoeger, Mehta, C.R., et al.

Impact of phase 2b strategies on optimization of drug development programs.
Optimization of Pharmaceutical R&D Programs and Portfolios, pp. 103–83.
Antonijevic, Z., Bolognese, J., Burman, C., Chuang-Stein, C., Jennison, C., Patel, N.R., et al.

An intra-articular, extended-release formulation of triamcinolone acetonide prolongs and amplifies analgesic effect in patients with osteoarthritis of the knee.
The Journal of Bone & Joint Surgery, 97, no. 11 877-888.
Bodick, Neil, Joelle Lufkin, Bolognese, J., Schoonmaker, C., et al.

Evaluation of an adaptive maximizing design study based on clinical utility versus morphine for TRV130 proof-of-concept and dose-regimen finding in patients with Postoperative pain after Bunionectomy.
Therapeutic Innovation & Regulatory Science, 49(5), pp. 756–766. 
Bolognese, J., Subach, R.A., and Skobieranda, F.

Creating value with financially adaptive clinical trials.
Nature Reviews Drug Discovery, 14(8), pp. 523–524.
David, F.S., Bobulsky, S., Schulz, K. and Patel, N.R.


Individualized vaginal bleeding experience of women with uterin fibroids in the PEARL I randomized controlled trial comparing the effects of ulipristal acetate or placebo.
Hum Reprod, 2014; 29(3):480-9 
Barlow, DH., Lumsden, MA., Fauser, BCJM., Terrill, P. and Bestel, E.

Adaptive Sequential testing for multiple comparisons.
Journal of Biopharmaceutical Statistics, 24(5), pp. 1035–1058. 
Gao, P., Liu, L. and Mehta, C.R.

Clinical development approaches and statistical methodologies to prospectively assess the cardiovascular risk of new Antidiabetic therapies for type 2 diabetes.
Therapeutic Innovation & Regulatory Science, 49(1), pp. 50–64. 
Geiger, M.J., Mehta, C.R., Gaydos, B, et al.

Research designs for proof-of-concept chronic pain clinical trials: IMMPACT recommendations.
Pain, 155(9), pp. 1683–1695.
Gewandter, J.S., Dworkin, R.H., Mehta, C.R., et al.

Biomarker driven population enrichment for adaptive oncology trials with time to event endpoints.
Statistics in Medicine, 33(26), pp. 4515–4531.
Mehta, C.R., Schäfer, H., Daniel, H. and Irle, S.

Early clinical development planning via Biomarkers, clinical endpoints, and simulation: A case study to optimize for phase 3 dose selection.
Therapeutic Innovation & Regulatory Science, 49(3), pp. 405–414. 
Musser, B., Bolognese, J., Fayad, G.N., Bhattacharyya, J., Patel, N.R., et al.

Approaches for clinical supply modeling and simulation.
Practical Considerations for Adaptive Trial Design and Implementation, pp. 273–297.
Patel, N.R., Ankolekar, S. and Senchaudhuri, P.

Key Publications

Authors’ response to “Comment on adaptive increase in sample size when interim results are promising”.
Statistics in Medicine, 30(28), pp. 3302–3303.
Mehta, C.R. and Pocock, S.J.

Adaptive increase in sample size when interim results are promising: A practical guide with examples.
Statistics in Medicine, 30(28), pp. 3267–3284.
Mehta, C.R. and Pocock, S.J.

Efficient Monte Carlo methods for conditional logistic regression.
Journal of the American Statistical Association, 95(449), p. 99.
Mehta, C.R., Patel, N.R. and Senchaudhuri, P.

Exact logistic regression: Theory and examples.
Statistics in Medicine, 14(19), pp. 2143–2160.
Mehta, C.R. and Patel, N.R.

Exact Permutational tests for group Sequential clinical trials.
Biometrics, 50(4), p. 1042.
Mehta, C.R., Patel, N.R., Senchaudhuri, P. and Tsiatis, A.

A network algorithm for performing Fisher’s exact test in r × c contingency tables.
Journal of the American Statistical Association, 78(382), p. 427.
Mehta, C.R. and Patel, N.R.

Repeated confidence intervals for adaptive group sequential trials.
Statistics in Medicine, 26, 5422-5433.
Mehta, C.R. Bauer, P., Posch, M. and Brannath, W.

Optimizing trial design; sequential, adaptive and enrichment strategies.
Statistical Primer for Cardiovascular Research, 119:597-605.
Mehta, C.R. Gao, P, Bhatt, DL, Harrington, RA, Skerjanec, S and Ware JH.

Exact inference for adaptive group sequential designs.
Statistics in Medicine, volume 32, 3991-4005.
Gao, P., Liu, L., and Mehta, C.R.

Biomarker driven population enrichment for adaptive oncology trials with time to event endpoints.
Statistics in Medicine, 33, 4515-4531.
Mehta, C.R., Schäfer, H., Daniel, H. and Irle, S.


Multiplicity: discussion points from the Statisticians in the Pharmaceutical Industry multiplicity expert group.
Pharmaceut. Statist., 2013, 12 255–259
Phillips, A., Fletcher, C., Atkinson, G., Channon, E., Douiri, A., Jaki, T., Maca, J., Morgan, D., Roger, JH. and Terrill, P.


PEARL I Study Group. Ulipristal acetate versus placebo for fibroid treatment before surgery.
N Engl J Med., 2012; 366(5):409-20.
Donnez J, Tatarchuk TF, Bouchard P, Puscasiu L, Zakharenko NF, Ivanova T, Ugocsai G, Mara M, Jilla MP, Bestel E, Terrill, P., Osterloh I and Loumaye E.

PEARL II Study Group. Ulipristal acetate versus leuprolide acetate for uterine fibroids.
N Engl J Med., 2012; 366(5):421-32.
Donnez J, Tomaszewski J, Vázquez F, Bouchard P, Lemieszczuk B, Baró F, Nouri K, Selvaggi L, Sodowski K, Bestel E, Terrill, P., Osterloh I and Loumaye E.