Publications from Office of Biostatistics Staff

The Office of Biostatistics (OB) conducts independent research on statistical methodologies relevant to CDER’s scientific mission and regulatory review process for publication. OB research spans such broad areas as clinical trial design, Bayesian statistics, signal detection, causal inference, meta-analysis, bioequivalence, benefit-risk assessment, patient focused drug development, psychometrics, and product quality assessment and control.

The following is a select list of recent peer-reviewed scientific journal articles that include OB statisticians:

• Wang X, Schuette P, Kam M. FDA experiences with a centralized statistical monitoring tool. Journal of Biopharmaceutical Statistics. 2024;34(6):986–992.
• Lu R, Li Y, Yao W. Semiparametric mixture regression for asynchronous longitudinal data using multivariate functional principal component analysis. Biostatistics. 2025;26(1):kxaf008.
• Wang Y, Tu W, Koh W, Travis J, Abugov R, Hamilton K, Zheng M, Crackel R, Bonangelino P, Rothmann M. Bayesian hierarchical models for subgroup analysis. Pharmaceutical Statistics. 2024.
• Cambon A, Travis J, Sun L, Idokoji J, Kettermann A. Optimizing sample size determinations for phase 3 clinical trials in type 2 diabetes. Pharmaceutical Statistics. 2024.
• Higgins KM, Levin G, Busch R. Considerations for open-label randomized clinical trials: Design, conduct, and analysis. Clinical Trials. 2024.
• Raghu G, Ghazipura M, Fleming TR, et al. Meaningful endpoints for idiopathic pulmonary fibrosis (IPF) clinical trials: Emphasis on 'Feels, Functions, Survives'. American Journal of Respiratory and Critical Care Medicine. 2024;209(6):647–669.
• Kinamon T, Waack U, Needles M, Rubin D, et al. Exploration of a potential DOOR endpoint for hospital-acquired bacterial pneumonia and ventilator-associated bacterial pneumonia using six registrational trials for antibacterial drugs. Clinical Infectious Diseases. 2024.
• Cortese I, Norato G, Harrington PR, et al. Biomarkers for progressive multifocal leukoencephalopathy: Emerging data for use of JC virus DNA copy number in clinical trials. The Lancet Neurology. 2024.
• Mulkey F, et al. Equipoise lost? Trial conduct challenges in an era of breakthrough therapies. Journal of Clinical Oncology. 2024.
• Xu J, et al. False-positive circulating tumor DNA results do not explain lack of efficacy for PARP inhibitors in patients with castration-resistant prostate cancer harboring ATM and CHEK2 mutations. Journal of Clinical Oncology. 2024.
• Zhao X, Sun J, Huang D. Meta-analysis application to hERG safety evaluation in clinical trials. Journal of Biopharmaceutical Statistics. 2024;35(3):343–355.
• Liu S, Cai X, Shen M, Tsong Y. In vitro dissolution profile comparison using bootstrap bias corrected similarity factor, f2. Journal of Biopharmaceutical Statistics. 2023;34(1):78–89.
• Bird ST, Smith ER, Gelperin K, et al. Severe Hypocalcemia With Denosumab Among Older Female Dialysis-Dependent Patients. JAMA. 2024;331(6):491–499.
• He, J., Zhang, D., & Li, F. Statistical methods to control for confounders in rare disease settings that use external control. Journal of Biopharmaceutical Statistics. 2024. 35(4), 531–549.
• Desai, R. J., Bradley, M. C., Lee, H., Eworuke, E., Weberpals, J., Wyss, R., Schneeweiss, S., Ball, R. Simulation-based bias analysis to assess the impact of unmeasured confounding when designing nonrandomized database studies. American Journal of Epidemiology. 2024.
• Leon, S., Rantou, E., Kim, J., Choi, S. and Choi, N.H. Comparative analyses of bioequivalence assessment methods for in vitro permeation test data. Pharmaceutical Research. 2024.
• Hinds, D. and Sun, W. An adaptive three-arm comparative clinical endpoint bioequivalence study design with unblinded sample size re-estimation and optimized allocation ratio. Pharmaceutical Research. 2024; e2439.
• Rodriguez, L. R., Gormley, N. J., Lu, R., Amatya, A. K., Demetri, G. D., Flaherty, K. T., Mesa, R. A., Pazdur, R., Sekeres, M. A., Shan, M., Snapinn, S., Theoret, M. R., Umoja, R., Vallejo, J., Warren, N. J. H., Xu, Q., Anderson, K. C. Improving collection and analysis of overall survival data. Clinical Cancer Research. 2024;30(18):3974–3982.

See a list of all publications by OB statistical review staff published in 2024:

2024

• Gong Y, Barretto FX, Tsong Y, Mousa Y, Ren K, Kozak D, Shen M, Hu M, Zhao L. Development of Quantitative Comparative Approaches to Support Complex Generic Drug Development. AAPS J. 2024 Jan 24;26(1):15
• Fang L, Gong Y, Hooker AC, Lukacova V, Rostami-Hodjegan A, Sale M, Grosser S, Jereb R, Savic R, Peck C, Zhao L. The Role of Model Master Files for Sharing, Acceptance, and Communication with FDA. AAPS J. 2024 Feb 27;26(2):28
• Sun W, Alosh M, Schuirmann DJ, Grosser S. Letter to the Editor on "Group-by-Treatment Interaction Effects in Comparative Bioavailability Studies". AAPS J. 2024 Sep 9;26(5):101
• Yang, Y., Wang, J., Wanasathop, A. et al. Evaluation of in vitro Skin Permeation of Clascoterone From Clascoterone Topical Cream, 1% (w/w). AAPS PharmSciTech 25, 186 (2024)
• Wyss R, van der Laan M, Gruber S, Shi X, Lee H, Dutcher SK, Nelson JC, Toh S, Russo M, Wang SV, Desai RJ, Lin KJ. Targeted learning with an undersmoothed LASSO propensity score model for large-scale covariate adjustment in health-care database studies. Am J Epidemiol. 2024 Nov 4;193(11):1632-1640
• Desai, R. J., Bradley, M. C., Lee, H., Eworuke, E., Weberpals, J., Wyss, R., Schneeweiss, S., & Ball, R. (2024), A simulation-based bias analysis to assess the impact of unmeasured confounding when designing nonrandomized database studies, American Journal of Epidemiology, 193(11), 1600–1608
• Thai TN, Winterstein AG, Suarez EA, He J, Zhao Y, Zhang D, Stojanovic D, Liedtka J, Anderson A, Hernández-Muñoz JJ, Munoz M, Liu W, Dashevsky I, Messenger-Jones E, Siranosian E, Maro JC. Triple challenges-small sample size in both exposure and control groups to scan rare maternal outcomes in a signal identification approach: a simulation study. Am J Epidemiol. 2024 Dec 2;193(12):1805-1813
• Zhu, Y., Hernandez-Diaz, S., Bateman, B. T., Gray, K. J., Alt, E. M., Straub, L., Taylor, L. G., Ouellet-Hellstrom, R., Ma, Y., Qiang, Y., Vine, S., Mogun, H., Hua, W., & Huybrechts, K. F. (2025). Development and validation of claims-based algorithms for estimating gestational age of spontaneous abortion and termination. American Journal of Epidemiology, 194(7), 2069–2074
• Raghu G, Ghazipura M, Fleming TR, Aronson KI, Behr J, Brown KK, Flaherty KR, Kazerooni EA, Maher TM, Richeldi L, Lasky JA, Swigris JJ, Busch R, Garrard L, Ahn DH, Li J, Puthawala K, Rodal G, Seymour S, Weir N, Danoff SK, Ettinger N, Goldin J, Glassberg MK, Kawano-Dourado L, Khalil N, Lancaster L, Lynch DA, Mageto Y, Noth I, Shore JE, Wijsenbeek M, Brown R, Grogan D, Ivey D, Golinska P, Karimi-Shah B, Martinez FJ. Meaningful Endpoints for Idiopathic Pulmonary Fibrosis (IPF) Clinical Trials: Emphasis on 'Feels, Functions, Survives'. Report of a Collaborative Discussion in a Symposium with Direct Engagement from Representatives of Patients, Investigators, the National Institutes of Health, a Patient Advocacy Organization, and a Regulatory Agency. Am J Respir Crit Care Med. 2024 Mar 15;209(6):647-669
• Bird ST, Gelperin K, Smith ER, Jung TH, Lyu H, Thompson A, Easley O, Naik KB, Zhao Y, Kambhampati R, Wernecke M, Niak A, Zemskova M, Chillarige Y, Kelman JA, Graham DJ. The Effect of Denosumab on Risk for Emergently Treated Hypocalcemia by Stage of Chronic Kidney Disease: A Target Trial Emulation. Ann Intern Med. 2025 Jan;178(1):29-38
• Le RQ, Przepiorka D, Chen H, Shen YL, Pulte ED, Norsworthy K, Theoret MR, De Claro RA. Complete remission with partial hematological recovery as a palliative endpoint for treatment of acute myeloid leukemia. Blood. 2024 Jul 11;144(2):206-215
• Raman SR, Hammill BG, Shaw PA, Lee H, Toh S, Connolly JG, Dandreo KJ, Nalawade V, Tian F, Liu W, Li J, Hernández-Muñoz JJ, Glynn RJ, Desai RJ, Weberpals J. Analyzing missingness patterns in real-world data using the SMDI toolkit: application to a linked EHR-claims pharmacoepidemiology study. BMC Med Res Methodol. 2024 Oct 19;24(1):246
• Shi, Amy & Zhou, Heng & Nie, Lei & Lin, Lifeng & Li, Hongjian & Chu, Haitao. (2024). Robustness Assessment of Oncology Dose-Finding Trials Using the Modified Fragility Index. Cancers. 16. 3504. 10.3390/cancers16203504
• Woods A, Norsworthy KJ, Wang X, Vallejo J, Chiu Yuen Chow E, Li RJ, Sun J, Charlab R, Jiang X, Pazdur R, Theoret MR, de Claro RA. FDA Approval Summary: Ivosidenib in Combination with Azacitidine for Treatment of Patients with Newly Diagnosed Acute Myeloid Leukemia with an IDH1 Mutation. Clin Cancer Res. 2024 Apr 1;30(7):1226-1231
• Telaraja D, Kasamon YL, Collazo JS, Leong R, Wang K, Li P, Dahmane E, Yang Y, Earp J, Grimstein M, Rodriguez LR, Theoret MR, Gormley NJ. FDA Approval Summary: Pirtobrutinib for Relapsed or Refractory Mantle Cell Lymphoma. Clin Cancer Res. 2024 Jan 5;30(1):17-22
• Singh S, Bradford D, Li X, Mishra-Kalyani PS, Shen YL, Wang L, Zhao H, Xiong Y, Liu J, Charlab R, Kraft J, Khasar S, Miller CP, Rivera DR, Kluetz PG, Pazdur R, Beaver JA, Singh H, Donoghue M. FDA Approval Summary: Alpelisib for PIK3CA-Related Overgrowth Spectrum. Clin Cancer Res. 2024 Jan 5;30(1):23-28
• Patel TH, Brewer JR, Fan J, Cheng J, Shen YL, Xiang Y, Zhao H, Lemery SJ, Pazdur R, Kluetz PG, Fashoyin-Aje LA. FDA Approval Summary: Tremelimumab in Combination with Durvalumab for the Treatment of Patients with Unresectable Hepatocellular Carcinoma. Clin Cancer Res. 2024 Jan 17;30(2):269-273
• Barbato MI, Nashed J, Bradford D, Ren Y, Khasar S, Miller CP, Zolnik BS, Zhao H, Li Y, Bi Y, Shord SS, Amatya AK, Mishra-Kalyani PS, Scepura B, Al-Matari RA, Pazdur R, Kluetz PG, Donoghue M, Singh H, Drezner N. FDA Approval Summary: Dabrafenib in Combination with Trametinib for BRAFV600E Mutation-Positive Low-Grade Glioma. Clin Cancer Res. 2024 Jan 17;30(2):263-268
• Chang E, Zhou J, Song C, Gittleman H, Fernandes L, Weinstock C, Atkins MB, Agrawal S, Sridhara R, Gormley N, Tang S, Suzman DL, Amiri-Kordestani L, Kluetz PG, Pazdur R, Rini BI, McDermott DF, Regan MM. A Pooled Analysis of Treatment-Free Survival in Advanced Renal Cell Carcinoma. Clin Cancer Res. 2024 Aug 1;30(15):3282-3286
• Maguire WF, Lee D, Weinstock C, Gao X, Bulik CC, Agrawal S, Chang E, Hamed SS, Bloomquist EW, Tang S, Pazdur R, Kluetz PG, Amiri-Kordestani L, Suzman DL. FDA Approval Summary: Enfortumab Vedotin plus Pembrolizumab for Cisplatin-Ineligible Locally Advanced or Metastatic Urothelial Carcinoma. Clin Cancer Res. 2024 May 15;30(10):2011-2016
• Barbato MI, Bradford D, Ren Y, Aungst SL, Miller CP, Pan L, Zirkelbach JF, Li Y, Bi Y, Fan J, Grimstein M, Dorff SE, Amatya AK, Mishra-Kalyani PS, Scepura B, Schotland P, Udoka O, Ojofeitimi I, Leighton JK, Rahman NA, Pazdur R, Singh H, Kluetz PG, Drezner N. FDA Approval Summary: Repotrectinib for Locally Advanced or Metastatic ROS1-Positive Non-Small Cell Lung Cancer. Clin Cancer Res. 2024 Aug 15;30(16):3364-3370
• Sandra J. Casak, Vaibhav Kumar, Chi Song, Mengdie Yuan, Anup K. Amatya, Joyce Cheng, Pallavi S. Mishra-Kalyani, Shenghui Tang, Steven J. Lemery, Doris Auth, Gina Davis, Paul G. Kluetz, Richard Pazdur, Lola A. Fashoyin-Aje; FDA Approval Summary: Durvalumab and Pembrolizumab, Immune Checkpoint Inhibitors for the Treatment of Biliary Tract Cancer. Clin Cancer Res 2024
• Fusco MJ, Casak SJ, Mushti SL, Cheng J, Christmas BJ, Thompson MD, Fu W, Wang H, Yoon M, Yang Y, Moore JN, Bi Y, Nan Y, Long CE, Auth D, Rahman NA, Tang S, Pazdur R, Fashoyin-Aje LA, Kluetz PG, Lemery SJ. FDA Approval Summary: Fruquintinib for the Treatment of Refractory Metastatic Colorectal Cancer. Clin Cancer Res. 2024 Aug 1;30(15):3100-3104
• Rodriguez LR, Gormley NJ, Lu R, Amatya AK, Demetri GD, Flaherty KT, Mesa RA, Pazdur R, Sekeres MA, Shan M, Snapinn S, Theoret MR, Umoja R, Vallejo J, Warren NJH, Xu Q, Anderson KC. Improving Collection and Analysis of Overall Survival Data. Clin Cancer Res. 2024 Sep 13;30(18):3974-3982
• Pamuk GE, Chow ECY, Ionan AC, Chen H, Lee SL, Hsu V, Grimstein M, Zheng N, Sun J, Charlab R, Gehrke BJ, Vallejo J, Ehrlich LA, de Claro RA, Theoret MR. FDA Approval Summary: Asciminib for Ph+ CML in Chronic Phase Treated with Two or More Tyrosine Kinase Inhibitors and for the T315I Mutation. Clin Cancer Res. 2024 Oct 1;30(19):4266-4271
• Brave MH, Maguire WF, Weinstock C, Zhang H, Gao X, Li F, Yu J, Fu W, Zhao H, Pierce WF, Chang E, Dinin J, Fiero MH, Rahman NA, Tang S, Pazdur R, Kluetz PG, Amiri-Kordestani L, Suzman DL. FDA Approval Summary: Enfortumab Vedotin plus Pembrolizumab for Locally Advanced or Metastatic Urothelial Carcinoma. Clin Cancer Res. 2024 Nov 1;30(21):4815-4821
• Fallah J, Heiss BL, Joeng HK, Weinstock C, Gao X, Pierce WF, Chukwurah B, Bhatnagar V, Fiero MH, Amiri-Kordestani L, Pazdur R, Kluetz PG, Suzman DL. FDA Approval Summary: Belzutifan for Patients with Advanced Renal Cell Carcinoma. Clin Cancer Res. 2024 Nov 15;30(22):5003-5008
• Sarraf Yazdy M, Kasamon YL, Gu W, Rodriguez LR, Jin S, Bhatnagar V, Richardson NC, Theoret MR, Pazdur R, Gormley NJ. FDA Approval Summary: Polatuzumab Vedotin in the First-Line Treatment of Select Large B-Cell Lymphomas. Clin Cancer Res. 2024 Dec 16;30(24):5521-5526
• Weberpals J, Raman SR, Shaw PA, Lee H, Russo M, Hammill BG, Toh S, Connolly JG, Dandreo KJ, Tian F, Liu W, Li J, Hernández-Muñoz JJ, Glynn RJ, Desai RJ. A Principled Approach to Characterize and Analyze Partially Observed Confounder Data from Electronic Health Records. Clin Epidemiol. 2024 May 21;16:329-343
• Howard-Anderson J, Hamasaki T, Dai W, Collyar D, Rubin D, Nambiar S, Kinamon T, Leister-Tebbe H, Hill C, Geres H, Holland TL, Doernberg SB, Chambers HF, Fowler VG Jr, Evans SR, Boucher HW; Antibacterial Resistance Leadership Group. Moving Beyond Mortality: Development and Application of a Desirability of Outcome Ranking (DOOR) Endpoint for Hospital-Acquired Bacterial Pneumonia and Ventilator-Associated Bacterial Pneumonia. Clin Infect Dis. 2024 Feb 17;78(2):259-268
• Jjingo, C. J., Bala, S., Waack, U., Needles, M., Bensman, T. J., McMaster, O., Smith, T., Blakely, B., Chan, I. Z., Puthawala, K., Dixon, C., Kim, Y., Lim, R., Colangelo, P., St. Clair, C., Nambiar, S., Moss, R. B., Botgros, R., Bazaz, R., Denning, D. W., Marr, K. A., Husain, S., Berman, L., Christensen, D. J., Keywood, C., Clayton, R. G., Walsh, T. J., Song, H. S. E., Shukla, S. J., & Farley, J., Food and Drug Administration Public Workshop Summary—Addressing Challenges in Inhaled Antifungal Drug Development, Clinical Infectious Diseases, Volume 78, Issue 6, 15 June 2024, Pages 1564–1570
• Kinamon T, Waack U, Needles M, Rubin D, Collyar D, Doernberg SB, Evans SR, Hamasaki T, Holland TL, Howard-Anderson J, Chambers H, Fowler VG Jr, Nambiar S, Kim P, Boucher HW, Gopinath R. Exploration of a Potential DOOR Endpoint for Hospital-acquired Bacterial Pneumonia and Ventilator-associated Bacterial Pneumonia Using Six Registrational Trials for Antibacterial Drugs. Clin Infect Dis. 2024 Jul 19;79(1):60-69
• Hiruy, H., Bala, S., Byrne, J. M., Roche, K. G., Jang, S. H., Kim, P., Nambiar, S., Rubin, D., Yasinskaya, Y., Bachmann, L. H., Bernstein, K., Botgros, R., Cammarata, S., Chaves, R. L., Deal, C. D., Drusano, G. L., Duffy, E. M., Eakin, A. E., Gelone, S., Hiltke, T., Hook, E. W., Jerse, A. E., McNeil, C. J., Newman, L., O’Brien, S., Perry, C., Reno, H. E. L., Romaguera, R. A., Sato, J., Unemo, M., Wi, T. E. C., Workowski, K., O’May, G. A., Shukla, S. J., & Farley, J. J., US Food and Drug Administration, Centers for Disease Control and Prevention, and National Institutes of Health Co-Sponsored Public Workshop Summary—Development Considerations of Antimicrobial Drugs for the Treatment of Gonorrhea, Clinical Infectious Diseases, 2024;, ciae386
• Coroller, T., Sahiner, B., Amatya, A., Gossmann, A., Karagiannis, K., Moloney, C., Samala, R.K., Santana-Quintero, L., Solovieff, N., Wang, C., Amiri-Kordestani, L., Cao, Q., Cha, K.H., Charlab, R., Cross, F.H., Jr, Hu, T., Huang, R., Kraft, J., Krusche, P., Li, Y., Li, Z., Mazo, I., Paul, R., Schnakenberg, S., Serra, P., Smith, S., Song, C., Su, F., Tiwari, M., Vechery, C., Xiong, X., Zarate, J.P., Zhu, H., Chakravartty, A., Liu, Q., Ohlssen, D., Petrick, N., Schneider, J.A., Walderhaug, M. and Zuber, E. (2024), Methodology for Good Machine Learning with Multi-Omics Data. Clin Pharmacol Ther, 115: 745-757
• Liu, Q., Nair, R., Huang, R., Zhu, H., Anderson, A., Belen, O., Tran, V., Chiu, R., Higgins, K., Chen, J., He, L., Doddapaneni, S., Huang, S.-M., Nikolov, N.P. and Zineh, I. (2024), Using Machine Learning to Determine a Suitable Patient Population for Anakinra for the Treatment of COVID-19 Under the Emergency Use Authorization. Clin Pharmacol Ther, 115: 890-895
• Donnelly M, Fang L, Madabushi R, Zhu H, Luke M, Canterbury C, Lerman B, Kaur P, Patel D, Sun W, Schuirmann D, Dinsmore S, Mehta M, Zhao L. Narrow Therapeutic Index Drugs: FDA Experience, Views, and Operations. Clin Pharmacol Ther. 2025 Jan;117(1):116-129
• Higgins KM, Levin G, Busch R. Considerations for open-label randomized clinical trials: Design, conduct, and analysis. Clin Trials. 2024 Dec;21(6):681-688
• Muñoz MA, Dal Pan GJ, Wei YJ, Xiao H, Delcher C, Giffin A, Sadiq N, Winterstein AG. Sociodemographic Characteristics of Adverse Event Reporting in the USA: An Ecologic Study. Drug Saf. 2024 Apr;47(4):377-387
• Simonetti A, Colilla S, Edwards B, Kübler J, Lackey L, Rodriguez L, Talbot S, Yang H, Wang W, Williams D, Higginson JM. Key Opinion Leaders' Interviews to Inform the Future of Benefit-Risk Planning in the Medical Total Product Life Cycle of Global Pharmaceutical and Medical Device Organizations. Drug Saf. 2024 Sep;47(9):853-868
• Apata J, Pennap D, Ma Y, Mosholder A. Suicide poisoning mortality: a comparison of the national poison data system and centers for disease control national dataset. Inj Prev. 2024 Jan 25;30(1):81-83
• Chai G, Xu J, Goyal S, Woods C, Ho A, Song J, Dal Pan G. Trends in Incident Prescriptions for Behavioral Health Medications in the US, 2018-2022. JAMA Psychiatry. 2024 Apr 1;81(4):396-405
• Weberpals, J., Raman, S. R., Shaw, P. A., Lee, H., Hammill, B. G., Toh, S., Connolly, J. G., Dandreo, K. J., Tian, F., Liu, W., Li, J., Hernández-Muñoz, J. J., Glynn, R. J., & Desai, R. J.. Severe Hypocalcemia With Denosumab Among Older Female Dialysis-Dependent Patients. JAMA. 2024 Feb 13;331(6):491-499
• Janick Weberpals, Sudha R Raman, Pamela A Shaw, Hana Lee, Bradley G Hammill, Sengwee Toh, John G Connolly, Kimberly J Dandreo, Fang Tian, Wei Liu, Jie Li, José J Hernández-Muñoz, Robert J Glynn, Rishi J Desai, smdi: an R package to perform structural missing data investigations on partially observed confounders in real-world evidence studies, JAMIA Open, Volume 7, Issue 1, April 2024, ooae008
• Jaleh Fallah et al. False-Positive Circulating Tumor DNA Results Do Not Explain Lack of Efficacy for PARP Inhibitors in Patients With Castration-Resistant Prostate Cancer Harboring ATM and CHEK2 Mutations. JCO Precis Oncol 8, e2400354(2024)
• Wang XT, Schuette P, Kam M. FDA experiences with a centralized statistical monitoring tool. J Biopharm Stat. 2024 Oct;34(6):986-992. doi: 10.1080/10543406.2024.2330210. Epub 2024 Mar 29. Erratum in: J Biopharm Stat. 2024 Oct;34(6):vii-viii
• He J, Zhang D, Li F. Statistical methods to control for confounders in rare disease settings that use external control. J Biopharm Stat. 2025;35(4):531-549
• Zhao, X., Sun, J., & Huang, D. (2024). Meta-analysis application to hERG safety evaluation in clinical trials. Journal of Biopharmaceutical Statistics, 35(3), 343–355
• Lautier JP, Grosser S, Kim J, Kim H, Kim J. Clustering plasma concentration-time curves: applications of unsupervised learning in pharmacogenomics. J Biopharm Stat. 2025;35(4):678-696
• Xi NM, Huang DP. Drug safety assessment by machine learning models. J Biopharm Stat. 2024 Jun 18:1-12
• Hu, J., Mei, X., Pepper, S., Wang, Y., Zhang, B., Cernik, C., & Gajewski, B. (2024). PROpwr: a Shiny R application to analyze patient-reported outcomes data and estimate power. Journal of Biopharmaceutical Statistics, 35(5), 969–980
• Shi X, Liu Z, Zhang M, Hua W, Li J, Lee JY, Dharmarajan S, Nyhan K, Naimi A, Lash TL, Jeffery MM, Ross JS, Liew Z, Wallach JD. Quantitative bias analysis methods for summary-level epidemiologic data in the peer-reviewed literature: a systematic review. J Clin Epidemiol. 2024 Nov;175:111507
• Mirat Shah et al. US Food and Drug Administration Approval Summary: Elacestrant for Estrogen Receptor–Positive, Human Epidermal Growth Factor Receptor 2–Negative, ESR1-Mutated Advanced or Metastatic Breast Cancer. JCO 42, 1193-1201(2024)
• Jaleh Fallah et al. Efficacy of Poly(ADP-ribose) Polymerase Inhibitors by Individual Genes in Homologous Recombination Repair Gene-Mutated Metastatic Castration-Resistant Prostate Cancer: A US Food and Drug Administration Pooled Analysis. JCO 42, 1687-1698(2024)
• Brian L. Heiss et al. US Food and Drug Administration Approval Summary: Talazoparib in Combination With Enzalutamide for Treatment of Patients With Homologous Recombination Repair Gene-Mutated Metastatic Castration-Resistant Prostate Cancer. JCO 42, 1851-1860(2024).
• Elizabeth S. Duke et al. US Food and Drug Administration Approval Summary: Eflornithine for High-Risk Neuroblastoma After Prior Multiagent, Multimodality Therapy. JCO 42, 3047-3057(2024)
• Ashley S. Margol et al. Use of External Control Cohorts in Pediatric Brain Tumor Clinical Trials. JCO 42, 1340-1343(2024).
• Asma Dilawari et al. US Food and Drug Administration Approval Summary: Capivasertib With Fulvestrant for Hormone Receptor–Positive, Human Epidermal Growth Factor Receptor 2–Negative Locally Advanced or Metastatic Breast Cancer With PIK3CA/AKT1/PTEN Alterations. JCO 42, 4103-4113(2024).
• Jaleh Fallah et al. Equipoise Lost? Trial Conduct Challenges in an Era of Breakthrough Therapies. JCO 42, 3783-3787(2024).
• Iliopoulos F, Tu D, Pence IJ, Li X, Ghosh P, Luke MC, Raney SG, Rantou E, Evans CL. Determining topical product bioequivalence with stimulated Raman scattering microscopy. J Control Release. 2024 Mar;367:864-876
• Wang Y, Otte A, Park H, Park K. In vitro-in vivo correlation (IVIVC) development for long-acting injectable drug products based on poly(lactide-co-glycolide). J Control Release. 2025 Jan 10;377:186-196
• Perez-Vilar S, Mosholder AD, Smith ER, Lee HS, Lo AC, Stone M, Brehm A, Zhao Y, Avagyan A, Leishear K, Wernecke M, MaCurdy TE, Kelman JA, Graham DJ. Antipsychotics and Risk of Acute Respiratory Failure in U.S. Medicare Beneficiaries with Chronic Obstructive Pulmonary Disease. J Gen Intern Med. 2025 Feb;40(3):587-594
• Smith HL, Bensman TJ, Mishra S, Li X, Dixon CA, Sheikh J, McMaster OG, Joshi A, Rubin DB, Goodwin A, Miller TJ, Danielsen ZY, Syed I, Shukla SJ, Iarikov D, Kim PW, Farley JJ. Regulatory Considerations in the Approval of Rezafungin (Rezzayo) for the Treatment of Candidemia and Invasive Candidiasis in Adults. J Infect Dis. 2024 Aug 16;230(2):505-513
• Van Metre Baum, L., KC, M., Soulos, P. R., Jeffery, M. M., Ruddy, K. J., Lerro, C. C., Lee, H., Graham, D. J., Rivera, D. R., Leapman, M. S., Jairam, V., Dinan, M. A., Gross, C. P., & Park, H. S., Trends in new and persistent opioid use in older adults with and without cancer, JNCI: Journal of the National Cancer Institute, Volume 116, Issue 2, February 2024, Pages 316–323
• Fallah J, Gittleman H, Weinstock C, Chang E, Agrawal S, Tang S, Pazdur R, Kluetz PG, Suzman DL, Amiri-Kordestani L. Cytoreductive nephrectomy in the era of immune checkpoint inhibitors: a US Food and Drug Administration pooled analysis. J Natl Cancer Inst. 2024 Jul 1;116(7):1043-1050
• Fallah J, Gittleman H, Weinstock C, Chang E, Agrawal S, Tang S, Pazdur R, Kluetz PG, Suzman DL, Amiri-Kordestani L. RE: Is it time to reconsider the role of upfront cytoreductive nephrectomy in metastatic renal cell carcinoma? J Natl Cancer Inst. 2024 Sep 1;116(9):1534
• Jagust WJ, Mattay VS, Krainak DM, Wang SJ, Weidner LD, Hofling AA, Koo H, Hsieh P, Kuo PH, Farrar G, Marzella L. Quantitative Brain Amyloid PET. J Nucl Med. 2024 May 1;65(5):670-678
• Cortese, I., Norato, G., Harrington, P. R., Usher, T., Mainardi, I., Martin-Blondel, G., Cinque, P., Major, E. O., & Sheikh, V. (2024). Biomarkers for progressive multifocal leukoencephalopathy: Emerging data for use of JC virus DNA copy number in clinical trials. The Lancet Neurology, 23(5), 534–544.
• Goulart BHL, Mushti SL, Chatterjee S, Larkins E, Mishra-Kalyani PS, Pazdur R, Kluetz PG, Singh H. Correlations of response rate and progression-free survival with overall survival in immunotherapy trials for metastatic non-small-cell lung cancer: an FDA pooled analysis. Lancet Oncol. 2024 Apr;25(4):455-462
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