#### 2017

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. *Biometrics.*

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. *Biometrics.* doi:10.1111/biom.12687

Ghosh, P., Liu, L., Senchaudhuri, P., Gao, P. and Mehta, C.R.

#### 2016

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.

#### 2015

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.

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.

#### 2014

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.