Publications from Office of Biostatistics Staff
The Office of Biostatistics (OB) is part of the Office of Translational Sciences (OTS). 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 2025:
- Woods AC, Norsworthy KJ, Choe M, Gehrke BJ, Chen H, Vallejo J, Pan L, Jiang X, Li H, Kraft J, Liu J, Charlab R, Okusanya OO, Booth B, Pazdur R, Theoret MR, Angelo de Claro R. FDA approval summary: Olutasidenib for adult patients with relapsed or refractory acute myeloid leukemia with an isocitrate dehydrogenase 1 mutation. Clin Cancer Res. 2025;31(1):12-17.
- Singh S, Bradford D, Chatterjee S, Li X, Aungst SL, Skinner AM, Miller CP, Kim-McOlash S, Fourie Zirkelbach J, Xiong Y, Bi Y, Wang YH, Yang Y, Sun J, Kraft J, Charlab R, Shord SS, Tang S, Scepura B, Bulatao I, Udoka O, Saber H, Rahman NA, Pazdur R, Singh H, Donoghue M, Drezner N. FDA approval summary: Tovorafenib for relapsed or refractory BRAF-altered pediatric low-grade glioma. Clin Cancer Res. 2025;31(8):1383-1389.
- Weberpals J, Shaw PA, Lin KJ, Wyss R, Plasek JM, Zhou L, Ngan K, DeRamus T, Raman SR, Hammill BG, Lee H, Toh S, Connolly JG, Dandreo KJ, Tian F, Liu W, Li J, Hernández-Muñoz JJ, Schneeweiss S, Desai RJ. High-dimensional multiple imputation for partially observed confounders including natural language processing–derived auxiliary covariates. American Journal of Epidemiology. 2026;195(1):10–20.
- Liu J, Yang Y, Gobburu J, Musante CJ, Klein M, Zhao L, Madabushi R, Zhu H. Considerations for Regulatory reusability of dynamic tools in the new drug development. Pharm Res. 2025;42(5):765-771.
- 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. Note on targeted learning with an undersmoothed Lasso propensity score model for large-scale covariate adjustment in health care database studies. Am J Epidemiol. 2025;194(5):1470-1472.
- Wang J, Campbell G, Lee JH, Hu M, Feng K, Chattopadhyay S, Zhao L, Peck CC. Bioequivalence of ANDA data using a non-informative Bayesian procedure (BEST) compared with the two one‑sided t‑tests (TOST). AAPS J. 2025;27(2):47.
- Chen J, Di J, Daizadeh N, Lu Y, Wang H, Shen YL, Kirk J, Rockhold FW, Pang H, Zhao J, He W, Potter A, Lee H. Decentralized clinical trials in the era of real-world evidence: A statistical perspective. Clin Transl Sci. 2025;18(2):e70117.
- Chen J, Gruber S, Lee H, Chu H, Lee S, Tian H, Wang Y, He W, Jemielita T, Song Y, Tamura R, Tian L, Zhao Y, Chen Y, van der Laan M, Nie L. Use of Real-world data and real-world evidence in rare disease drug development: A Statistical perspective. Clin Pharmacol Ther. 2025;117(4):946-960.
- Wang SV, Russo M, Glynn RJ, Bradley MC, He J, Concato J, Schneeweiss S. A benchmark, expand, and calibration (BenchExCal) trial emulation approach for using real‐world evidence to support indication expansions: Design and Process for a planned empirical evaluation. Clinical Pharmacology & Therapeutics. 2025;117(6):1820-8.
- Tsakalozou E, Fang L, Skoda E, Nicholas T, Kollipara S, Ren K, Grosser S, Rege B, Roy P, Zhu H, Zhao L. Considering opportunities and challenges when implementing the model master file framework - a meeting report. Pharm Res. 2025;42(5):737-746.
- Chen C, Tsong Y, Zhao X, Shen M. Statistical considerations for using tolerance interval to set product specification for normally distributed attribute. J Biopharm Stat. 2025;13:1-6.
- Chen J, Nie L, Lee S, Chu H, Tian H, Wang Y, He W, Jemielita T, Gruber S, Song Y, Tamura R, Tian L, Zhao Y, Chen Y, van der Laan M, Lee H. Challenges and possible strategies to Address them in rare disease drug development: A statistical perspective. Clin Pharmacol Ther. 2025;118(1):62-73.
- Chen Y, Lawrence J, Stockbridge N. Corrigendum to "Days alive out of hospital in heart failure: Insights from the PARADIGM-HF and CHARM trials" [Am Heart J 241 (2021) 108-119]. Am Heart J. 2025;285:105. Erratum for: Am Heart J. 2021;241:108-119.
- Shinde M, Cosgrove A, Lyons JG, Kempner ME, Mosley J, Cole D, Hoffman E, Messenger-Jones E, Hernández-Muñoz JJ, Stojanovic D, Wong BHW, Zhao Y, Sahin L, Andrade SE, Toh S, Hua W. Characteristics and medication use Patterns of pregnancies with COVID-19 ending in live-birth in the sentinel system. Pharmacoepidemiol Drug Saf. 2025;34(4):e70121.
- Kunkoski E, Saha A, Kam MM. FDA perspective on the importance of digital health technologies in clinical trials. JAMA Health Forum. 2025;6(4):e250165.
- Boudreau DM, Pocobelli G, Bobb JF, Cheetham C, Dublin S, Andrade SE, Albertson-Junkans L, Griffin MR, Li DK, Raebel MA, Hechter RC, Pawloski PA, Toh S, Hua W, Ouellet-Hellstrom R, Horn P, Dinatale M, Ceresa C, Soukup M, Ma Y, Trinidad JP. Neural tube defect risk in relation to opioid analgesic exposure during early pregnancy. Am J Epidemiol. 2025:kwaf079.
- Zhai J, Smith F, Soon G. Comparison of continuous, binary, and ordinal endpoints. J Biopharm Stat. 2025;35(6):1143-1160.
- Chen K, Chen TY, Zhang Y, Lin R, Yuan Y. Practical considerations for using the TITE-BOIN Design to handle late-onset toxicity or fast accrual in phase I trials. Clin Cancer Res. 2025;31(13):2573-2580.
- Dilawari A, Krantz MJ, Bulatao I, Joeng HK, Neilson M, Wedam S, Gao X, Fiero MH, Nair A, Theoret M, Amiri-Kordestani L. Cardiac Adverse Events in Patients Receiving Immune Checkpoint Inhibitors in the Adjuvant Setting: An FDA Pooled Analysis. Ann Noninvasive Electrocardiol. 2025;30(3):e70087.
- Fleming TR, Carroll KJ, Wittes JT, Emerson SS, Rothmann MD, Collins S, Levin G. A perspective on the appropriate implementation of ICH E9(R1) addendum strategies for handling intercurrent events. Stat Med. 2025;44(10-12):e70104.
- Higgins KM, Zalkikar J, Zhang J, Wang Y, Chiu R, Soukup M, Chen YF, Levin G. The whys, whens, and hows of futility monitoring. Stat Med. 2025;44(10-12):e70117.
- Shah M, Chen TY, Ison G, Fiero MH, Zhang H, Gao X, Neilson M, Goldberg KB, Nair A, Ricks TK, Pierce WF, Gormley N, Theoret MR, Tang S, Pazdur R, Kluetz PG, Amiri-Kordestani L. Overall Survival and the Evolving Benefit-Risk Assessment for Poly (ADP-ribose) Polymerase Inhibitors in Advanced Ovarian Cancer. J Clin Oncol. 2025;43(19):2218-2227.
- Norsworthy KJ, Rivera DR, Wynne J, Zhao J, Konicki R, Kuzucan A, Vallejo J, Leong R, Okusanya OO, Booth B, Kluetz PG, Pazdur R, Angelo de Claro R. FDA approval summary: Abatacept for the prophylaxis of acute GVHD. Clin Cancer Res. 2025;31(15):3118-3123.
- Ma Y, Haddad J, Liu W, Snyder E, Bennett D, Mayo S. Review of recent pharmacoepidemiologic post-market safety studies through the lens of the Estimand framework. Ther Innov Regul Sci. 2025;59(5):971-980. Erratum in: Ther Innov Regul Sci. 2025;59(5):981.
- Janiczak S, Tanveer S, Tom K, Zhang R, Ma Y, Wolf L, Muñoz MA. An evaluation of duplicate adverse event reports characteristics in the Food and Drug Administration Adverse Event Reporting System. Drug Saf. 2025;48(10):1119-1126. Erratum in: Drug Saf. 2025;48(12):1661.
- Mateja A, Chu E, Murray TA, Bramante CT, Moser C, Givens N, Abdelghany M, Blair C, Chen S, Lat PK, Harari O, Kallewaard NL, Macpherson LF, Boulware DR, Suñer C, Mitjà O, Adam SJ, De Gruttola V, Hughes MD, Rubin D, Smith DM, Potter GE. The choice of viral load end point in early phase trials of COVID-19 treatments aiming to reduce 28-day hospitalization and/or death. J Infect Dis. 2025;232(1):60-68.
- Gao W, Liu J, Musante CJ, Zhu H, Thompson M, Shah M, Cao Y, Ivaturi V, Conaway MR, Bottino D, Yin D, Semiond D, Oganesian A, Ratain MJ, Li C, Zhu L, Ou YC, Jiang X, Vallejo J, Madabushi R, Liu Q, Theoret M, Rahman A, Booth B, Okusanya O, Johnson-Williams BE, Shord SS. Using quantitative approaches to optimize dosages for new combinations and subsequent indications for oncology drugs. Clin Pharmacol Ther. 2025;118(4):772-777.
- Ma Y, Haddad J, Liu W, Snyder E, Bennett D, Mayo S. Correction: Review of recent pharmacoepidemiologic post-market safety studies through the lens of the Estimand framework. Ther Innov Regul Sci. 2025;59(5):981. Erratum for: Ther Innov Regul Sci. 2025;59(5):971-980.
- Rivera DR, Cutler TL, McShane L, Meeker-O'Connell A, Marston HD, Califf RM, Bertagnolli MM; FDA-NIH MoRE Glossary Working Group; Avilés-Santa L, Ayalew K, Bayha R, Chetta J, Concato J, Culp M, Dodd L, Facey N, Forshee R, Freedman A, Friedman P, Golfinopoulos E, Grandinetti C, Jarman-Miller H, Jorgenson L, Karesh A, Kline L, Kluetz PG, Kraus S, Lee JJ, Lerro CC, Levenson M, Li J, Lu Y, Moore C, Ordóñez AE, Potter G, Prindiville S, Rosenberg Y, Sarosy G, Shapley S, Smith ER, Talley A, Tamburro R, Viviano C, Yongpravat C. Modernizing research and evidence consensus definitions: A Food and Drug Administration-National Institutes of Health collaboration. JAMA Netw Open. 2025;8(6):e2516674.
- Pottackal G, Travis J, Nie L, Chiu R, Neuner R, Levin G, Niu J, Marathe A, Mulugeta LY, Nikolov NP. Application of Bayesian statistics to support approval of intravenous belimumab in children with systemic lupus erythematosus in the United States. Lupus. 2025;34(9):943-952.
- Woods AC, Pulte ED, Wang X, Vallejo J, Chadda R, Zheng N, Blanco JG, Dorff SE, Li H, Liu J, Okusanya OO, Pazdur R, Theoret MR, Angelo de Claro R, Norsworthy KJ. FDA approval summary: Ivosidenib for treatment of adult patients with relapsed/refractory myelodysplastic syndrome with an IDH1 mutation. Clin Cancer Res. 2025;31(17):3613-3617.
- Kleppinger C, Ivy D, Stockbridge N, Bates A, Handler S, Krishnan US, Mullen MP, Yung D, Hopper RK, Varghese NP, Fineman J, Austin ED, Avitabile CM, Freire G, Clark J, Sun H. Procedural variation may contribute to 6-minute walk distance variability in real-world pediatric pulmonary arterial hypertension study. AAPS J. 2025;27(5):114.
- Le RQ, Godder K, Wang J, Collazo JS, Konicki R, Choe M, Feng M, Przepiorka D, Vallejo J, Shah A, Liu J, Gehrke BJ, Wilson W, Siegel A, Wu Y, Kuo CY, Ray M, Pazdur R, Theoret MR, Angelo de Claro R. FDA approval summary: Axatilimab for adult and pediatric patients weighing at least 40 kilograms with chronic gvhd after two prior lines of systemic therapy. Clin Cancer Res. 2025;31(17):3607-3612.
- Zhong Z, Ren A, Doluisio N, Smith W, Qin B, Wang Y, Zhang F. Correlation between material, structure, and drug release of Nexplanon®: an ethylene vinyl acetate (EVA) based long-acting implant. Int J Pharm. 2025;682:125971.
- Chang E, Gittleman H, Song C, Bloomquist E, Fernandes L, Weinstock C, Agrawal S, Gormley N, Tang S, Suzman DL, Amiri-Kordestani L, Pazdur R, Kluetz PG, McDermott DF, Regan MM, Rini BI. The Food and Drug Administration pooled analysis of overall survival according to depth of response in frontline advanced immune-oncology renal cell carcinoma trials. JNCI Cancer Spectr. 2025;9(4):pkaf069.
- Loingeville F, Rakez M, Thuy Nguyen T, Donnelly M, Fang L, Feng K, Zhao L, Grosser S, Sun G, Sun W, Mentré F, Bertrand J. Model-based approach for two-stage group sequential or adaptive designs in bioequivalence studies using parallel and crossover designs. Stat Methods Med Res. 2025;34(10):1968-1981.
- Maguire WF, Chi DC, Agrawal S, Joeng HK, Kim-McOlash S, Manheng W, Bulatao I, Sanchez-Solana B, Pathak A, Lee A, Han Y, Ricks TK, Hamed SS, Fiero MH, Pierce WF, Kalavar S, Ghosh S, Leighton JK, Tang S, Amiri-Kordestani L, Kluetz PG, Suzman DL. FDA approval summary: Niraparib plus abiraterone acetate fixed-dose combination for BRCA-mutated metastatic castration-resistant prostate cancer. Clin Cancer Res. 2025;31(18):3823-3829.
- Shives E, Gurmu Y, Lee W, Morris E, Wang Y. Novel clinical trial design with stratum-specific endpoints and global test methods for rare diseases with heterogeneous clinical manifestations. Stat Med. 2025;44(18-19):e70206.
- Dilawari A, Zhang H, Shah M, Gao X, Fiero M, Bhatnagar V, Pierce W, Mixter B, Pazdur R, Amiri-Kordestani L. US Food and Drug Administration approval summary: Trastuzumab deruxtecan for the treatment of adult patients with hormone receptor-positive, unresectable or metastatic human epidermal growth factor receptor 2-low or human epidermal growth factor receptor 2-ultralow breast cancer. J Clin Oncol. 2025;43(26):2942-2951.
- Lin LA, Hammad TA, Liu W, Ma Y, Whalen E, Sinvhal R, Munsaka M, Wang W. Leveraging multi-national observational study in post-marketing safety assessment: Challenges and strategies. Ther Innov Regul Sci. 2025;59(6):1526-1536. Erratum in: Ther Innov Regul Sci. 2025;59(6):1537.
- Howard-Anderson J, Doernberg SB, Dixon D, Gopinath R, Rubin D, Kinamon T, Kim P, Collyar D, Cosgrove SE, Fowler VG, Chambers HF, Evans SR, Holland TL, King HA, Boucher HW. Interagency collaboration for patient-centered antibacterial drug development. Clin Infect Dis. 2025;81(Supplement_1):S11-S15.
- Thanarajasingam G, Bhatnagar V, Noble BN, Chen TY, Fiero MH, Hoffman R, Jeffery M, Mazza GL, Mascarenhas J, Mesa R, Murugappan M, Ross J, Sidana S, Warsame R, Kluetz PG, Dueck AC. Longitudinal graphics of patient-reported physical function in patients treated for hematologic malignancies. BMC Med Res Methodol. 2025;25(1):189.
- Gao JJ, Prowell TM, Gittleman H, Cheng J, Fiero M, Bulatao I, Ching-Jey Chang G, Ricks TK, Green F, Wu H, Yu J, Grimstein M, Yang Y, Zhao H, Liu Q, Kolhatkar R, Gao T, Tu CM, Hou S, Redwood S, Fuller B, Griffiths L, Ajua-Alemanji M, Leach C, Woods S, Akinboro O, Narayan P, Shah M, Osgood C, Raghavachari R, Rahman A, Tang S, Bhatnagar V, Gormley N, Kim T, Pierce WF, Pazdur R, Kluetz PG, Amiri-Kordestani L. US Food and Drug Administration approval summary: Ribociclib with an aromatase inhibitor in the adjuvant hormone receptor-positive, human epidermal growth factor receptor 2-negative stage II and III high-risk early breast cancer treatment setting. J Clin Oncol. 2025;43(30):3312-3320.
- Mateja A, Chu E, Murray TA, Bramante CT, Moser C, Givens N, Abdelghany M, Blair C, Chen S, Lat PK, Harari O, Kallewaard NL, Macpherson LF, Boulware DR, Suñer C, Mitjà O, Adam SJ, De Gruttola V, Hughes MD, Rubin D, Smith DM, Potter GE. Evaluating viral load sampling schemes and swabbing methods for optimal surrogacy. J Infect Dis. 2025:jiaf443.
- Anania FA, Hager R, Higgins K, Makar GA, Siegel J, Tran TT. Non-invasive tests: Establishing efficacy for metabolic dysfunction-associated steatohepatitis beyond the biopsy-current perspectives from the Division of Hepatology and Nutrition, US Food and Drug Administration. Hepatology. 2025.
- Huang D. Dos and don'ts for concentration - QTc analysis as primary analysis for assay sensitivity assessment. J Pharmacokinet Pharmacodyn. 2025;52(5):47.
- Zhao X, Sun J, Huang D. Meta-Analysis of Moxifloxacin Concentration-QTc Effects with Application to Assay Sensitivity Assessment. Statistics in Biopharmaceutical Research. 2025;17(3):379–389.
- Wedam S, Narayan P, Gittleman H, Cheng J, Bhatnagar V, Sabit H, Price LSL, Rahman NA, Chiu HJ, Biel N, Ricks T, Fiero M, Tang S, Osgood C, Pierce W, Pazdur R, Kluetz PG, Amiri-Kordestani L. US Food and Drug Administration approval summary: Inavolisib With palbociclib and fulvestrant for endocrine-resistant, PIK3CA-mutated, hormone receptor-positive, human epidermal growth factor receptor 2-negative, locally advanced or metastatic breast cancer. J Clin Oncol. 2025;43(28):3123-3131.
- Russo M, Sreedhara SK, Smith J, Davis SE, Maro JC, Deramus T, Lii J, Yang J, Desai RJ, Hernández-Muñoz JJ, Ma Y, Wang Y, Jones JT, Wang SV. Electronic health record-enhanced signal detection using tree-based scan statistic methods. Am J Epidemiol. 2026;195(1):178-187.
- Zarmpi P, Tsikritsis D, Watson AC, Vorng JL, Tyagi V, Belsey NA, Rantou E, Ghosh P, Bunge AL, Woodman TJ, Delgado-Charro MB, Guy RH. Raman spectroscopy as a tool to accelerate development of complex medicinal products. J Control Release. 2025;387:114190.
- Wang H, Daizadeh N, Shen YL, Chen J, Rockhold FW, Pang H, Lee H. Decentralized clinical trials in the era of real-world evidence: A critical assessment of recent experiences. Clin Transl Sci. 2025;18(9):e70328.
- Tu D, Lemberger NS, Wallmeier K, Riseman J, Kuzma BA, Wei Y, Khoo TC, Rantou E, Ghosh P, Luke MC, Raney SG, Fallnich C, Evans CL. Improved dual-modality bioequivalence evaluation of topical formulations within human skin using stimulated Raman scattering Microscopy. Pharmaceutics. 2025;17(9):1193.
- Baum LVM, Soulos PR, Kc M, Jeffery MM, Ruddy KJ, Lerro CC, Lee H, Graham DJ, Rivera DR, Liberatore M, Leapman MS, Jairam V, Dinan MA, Gross CP, Park HS. Opioid prescribing trends and pain scores among adult patients with cancer in a large health system. Cancer. 2025;131(19):e70027.
- Crackel R, Ji Y, Kim Y, Travis J, Tu W, Wang Y, Kim S, Bonangelino P. Application of Bayesian borrowing methods in clinical trials for children with type ii diabetes mellitus. Ther Innov Regul Sci. 2026;60(1):210-222.
- Bhatnagar V, Lee Johnson L, Fiero MH, Mulkey F, Kluetz P. Assessing tolerability of cancer therapeutics: A regulatory perspective from the FDA Oncology Center of Excellence. Cancer. 2025;131:Suppl 2(Suppl 2):e35949.
- Kim N, Pulte ED, Ehrlich LA, Ionan AC, Haupert S, Vallejo J, Green F, Zheng N, Wang Y, Liu J, Blanco JG, Dorff SE, Booth B, Choe M, Gehrke B, Bhatnagar V, Theoret M, Pazdur R, Angelo De Claro R, Norsworthy KJ. FDA approval summary: Imetelstat for treatment of selected patients with low- to intermediate-1 risk myelodysplastic syndromes with transfusion-dependent anemia. J Clin Oncol. 2025;43(35):3760-3768.
- Shah M, Fiero MH, Cheng J, Chen TY, Ison G, Pazdur R, Amiri-Kordestani L. Reply to: Decoding the end points of poly (ADP-ribose) polymerase inhibitor trials in ovarian cancer. J Clin Oncol. 2025;43(36):3847-3849.
- Park SY, Ahn J, Lee JH, Kwon J, Lee H. Overlap weights for binary outcomes: A performance assessment. Pharmacoepidemiol Drug Saf. 2025;34(11):e70253.
- Sun W, Schuirmann D, Makhlouf F, Donnelly M, Jiang W, Zhang H, Mehta MU, Zhang L, Tampal N, Grosser S. A Proposal for Alternative FDA Bioequivalence Criteria for Narrow Therapeutic Index Drug Products to Support Future Harmonization. Pharm Res. 2025;42(10):1677-1690.
- Kettermann A, Dandi G, Clark J, Kim Y, Song J, Pucino F, Navarro Almario E. Navigating the future: Considerations for use of continuous glucose monitoring in diabetes trials. Ther Innov Regul Sci. 2025.
- Perez-Vilar S, Hua W, Kwist A, Ma Y, Dong D, Zhang R, Ma Y, Chavez A, Huang N, Sheikh V, Chiang Y, Salone C, Pica N, Zhu Y, Pratt N, Nadimpalli G, Pershing S, Wernecke M, Cunningham FE, Graham DJ. Validation of the US Food and Drug Administration's COVID-19 disease severity categorization for use in real-world data. Pharmacoepidemiol Drug Saf. 2025;34(11):e70252.
- Weng YT, Huang D. Statistical approaches to evaluate the positive control drug using the hERG assay. J Biopharm Stat. 2025:1-17.
- Turner S, Floden L, Simmatis L, Fromy P, Langford J, Daza EJ, Potter A, Troeger K; STAGES cohort investigator group. Methods for analytical validation of novel digital clinical measures: Implementation feasibility evaluation using real-world datasets. J Med Internet Res. 2025;27:e70314.
- Kim Y, Crackel R, Cho HS, Tu W, Wang Y. Beyond time in range: Hidden statistical challenges of continuous glucose monitoring data in diabetes drug development. J Diabetes Sci Technol. 2026;20(1):229-230. Epub 2025 Nov 17.
- Amdal CD, Falk RS, Alanya A, Schlichting M, Roychoudhury S, Bhatnagar V, Wintner LM, Regnault A, Ingelgård A, Coens C, le Cessie S, Holzner B, Chang J, Taphoorn M, Cislo P, Giesinger JM, Cappelleri JC, Pawar V, Ten Seldam S, Papadopoulos EJ, Calvert MJ, Joseph KL, Bottomley A, Griebsch I, Arraras JI, Astrup GL, Basch E, Belančić A, Brundage M, Campbell A, Rerhou Rantell K, Cocks K, Cherny N, Eremenco S, Ferrer M, Fiero MH, Gerlinger C, Goetghebeur E, Grouven U, Lauer A, Aiyegbusi OL, Machingura A, Mizusawa J, Molenberghs G, Aa Petersen M, Reijneveld JC, Ringash J, Rumpold G, Rutherford C, Quinten C, Sail K, Sasseville M, Sauerbrei W, Schiel A, Smith AW, Snyder C, Velikova G, Wang XS, Bjordal K, Pe M; SISAQOL-IMI Consortium. SISAQOL-IMI consensus-based guidelines to design, analyse, interpret, and present patient-reported outcomes in cancer clinical trials. Lancet Oncol. 2025;26(12):e683-e693.
- Janiczak S, Tanveer S, Tom K, Zhang R, Ma Y, Wolf L, Muñoz MA. correction: an evaluation of duplicate adverse event reports characteristics in the Food and Drug Administration Adverse Event Reporting System. Drug Saf. 2025;48(12):1661. Erratum for: Drug Saf. 2025;48(10):1119-1126.
- Lin LA, Hammad TA, Liu W, Ma Y, Whalen E, Sinvhal R, Munsaka M, Wang W. Publisher correction: leveraging multi-national observational study in post-marketing safety assessment: Challenges and strategies. Ther Innov Regul Sci. 2025;59(6):1537. Erratum for: Ther Innov Regul Sci. 2025;59(6):1526-1536.
- Rabik CA, Wang S, Chadda R, Przepiorka D, Vallejo J, Jiang X, Theoret MR, Angelo de Claro R. FDA approval summary: Blinatumomab for the treatment of B-cell precursor acute lymphoblastic leukemia in the consolidation phase of multiphase chemotherapy. Clin Cancer Res. 2025;31(20):4230-4238.
- Heiss BL, Chang E, Joeng HK, Fiero MH, Wang L, Hamed SS, Chiu HJ, Ricks TK, Koh EH, Dellibovi-Ragheb TA, Wang M, Lee C, Pierce WF, Leighton JK, Rahman NA, Tang S, Pazdur R, Amiri-Kordestani L, Kluetz PG, Suzman DL. FDA approval summary: Nogapendekin alfa inbakicept-pmln with BCG for BCG-unresponsive carcinoma in situ. Clin Cancer Res. 2025;31(20):4223-4229.
- Royce M, Shah M, Zhang L, Cheng J, Bonner MK, Pegues M, Miller CP, Leu L, Price LSL, Qiu J, Yu J, Truong TM, Dorff SE, Yang Y, Zhang N, Gutierrez-Lugo M, Ricks TK, Pierce WF, Luo Z, Kappel D, Goldberg KB, Shord SS, Tang S, Bhatnagar V, Pazdur R, Kluetz PG, Amiri-Kordestani L. FDA approval summary: Datopotamab deruxtecan-dlnk for treatment of patients with unresectable or metastatic, HR-positive, HER2-negative breast cancer. Clin Cancer Res. 2025;31(21):4405-4411.
- Barbato MI, Barone AK, Aungst SL, Miller CP, Ananthula S, Bi Y, Yang Y, Li X, Xiong Y, Fan J, Dorff SE, Zhao H, Zhou H, Pradhan S, Scepura B, Sinha AK, Stephenson M, Bhatnagar V, Saber H, Rahman NA, Tang S, Pazdur R, Kluetz PG, Larkins E, Drezner N. FDA approval summary: Vorasidenib for IDH-mutant grade 2 Astrocytoma or oligodendroglioma following surgery. Clin Cancer Res. 2025;31(21):4412-4418.