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:

The Role of FDA CDER Statisticians in Response Efforts to the COVID-19 Pandemic. Collins, S. H., Price, D., & Johnson, L. L. Statistics in Biopharmaceutical Research (2022), Vol. 14(1), 3-4.
Statement on P-values. Gwise, T., Rothmann, M. D., James Hung, H. M., Amatya, A., Rothwell, R., Wang, S. J., Wu, Y., Smith, F., Weng, Y., Andraca-Carrerra, E., Grosser, S., Chattopadhyay, S., & Collins, S. H. Statistics in Biopharmaceutical Research (2021), Vol. 13(1), 57-58.
Composite Endpoints in Cardio-Renal Clinical Outcome Trials. Hung, H. J., & Lawrence, J. Statistics in Biopharmaceutical Research (2023), Vol. 15(2), 318-322.
Bayesian Methods in Human Drug and Biological Products Development in CDER and CBER. Ionan, A. C., Clark, J., Travis, J., Amatya, A., Scott, J., Smith, J. P., Chattopadhyay, S., Salerno, M.J., & Rothmann, M. (2022). Therapeutic Innovation & Regulatory Science (2023), 57, 436-444.
Concept End Points Informing Design Considerations for Confirmatory Clinical Trials in Osteoarthritis. Kim, Y., Levin, G., Nikolov, N. P., Abugov, R., & Rothwell, R. Arthritis Care & Research (2022), Vol. 74(7), 1154-1162.
Biostatistical Considerations When Using RWD and RWE in Clinical Studies for Regulatory Purposes: A Landscape Assessment. Levenson, M., He, W., Chen, J., Fang, Y., Faries, D., Goldstein, B. A., Ho, M., Lee, K., Mishra-Kalyani, P., Rockhold, F., Wang, H., & Zink, R. C. Statistics in Biopharmaceutical Research (2023), Vol. 15(1), 3-13.
External Control Arms in Oncology: Current Use and Future Directions. Mishra-Kalyani, P. S., Kordestani, L. A., Rivera, D. R., Singh, H., Ibrahim, A., DeClaro, R. A., Shen, Y., Tang, S., Sridhara, R., Kluetz, P.G., Concato, J., Pazdur, R., & Beaver, J. A. Annals of Oncology (2022), Vol. 33(4), 376-383.
The U.S. Food and Drug Administration’s Complex Innovative Trial Design Pilot Meeting Program: Progress to date. Price, D., & Scott, J. Clinical Trials (2021), 18(6), 706-710.
FDA Approval of Remdesivir—A Step in the Right Direction. Rubin, D., Chan-Tack, K., Farley, J., & Sherwat, A. New England Journal of Medicine (2020), 383(27), 2598-2600.

See a list of all publications by OB statistical review staff published in 2022:

2022

FDA approval summary: pembrolizumab for neoadjuvant and adjuvant treatment of patients with high-risk early- stage triple negative breast cancer.

Shah M, Osgood CL, Amatya AK, Fiero MH, Pierce WF, Nair A, Herz J, Robertson KJ, Mixter BD, Tang S, Pazdur R, Beaver JA, Amiri-Kordestani L. Clin Cancer Res 2022 Dec 15;28(24):5249-53

FDA approval summary: nivolumab for adjuvant treatment of adults with completely resected esophageal/ gastroesophageal junction cancer and residual pathologic disease.

Horiba MN, Casak SJ, Mishra-Kalyani PS, Roy P, Beaver JA, Pazdur R, Kluetz PG, Lemery SJ, Fashoyin-Aje LA. Clin Cancer Res 2022 Dec 15;28(24):5244-8

Bayesian methods in human drug and biological products development in CDER and CBER.

Ionan AC, Clark J, Travis J, Amatya A, Scott J, Smith JP, Chattopadhyay S, Salerno MJ, Rothmann M. Ther Innov Regul Sci 2022 Dec 2

FDA approval summary: lutetium Lu 177 vipivotide tetraxetan for patients with metastatic castration-resistant prostate cancer.

Fallah J, Agrawal S, Gittleman H, Fiero MH, Subramaniam S, John C, Chen W, Ricks TK, Niu G, Fotenos A, Wang M, Chiang K, Pierce WF, Suzman DL, Tang S, Pazdur R, Amiri-Kordestani L, Ibrahim A, Kluetz PG. Clin Cancer Res 2022 Dec 5

FDA approval summary: ripretinib for advanced gastrointestinal stromal tumor (GIST).

Kumar V, Doros L, Thompson M, Mushti SL, Charlab R, Spehalski EI, Zhao H, Thompson MD, Tang S, Pazdur R, Lemery SJ, Theoret MR, Fashoyin-Aje LA. Clin Cancer Res 2022 Dec

Why are there not more Bayesian clinical trials? Ability to interpret Bayesian and conventional statistics among medical researchers.

Medical Outreach Team of the Drug Information Association Bayesian Scientific Working Group, Bray R, Hartley A, Wenkert D, Muehlemann N, Natanegara F, Harrell FE Jr, Wang F, Clark J. Ther Innov Regul Sci 2022 Dec 10

FDA approval summary for lonafarnib (Zokinvy) for the treatment of Hutchinson-Gilford progeria syndrome and processing-deficient progeroid laminopathies.

Suzuki M, Jeng LJB, Chefo S, Wang Y, Price D, Li X, Wang J, Li RJ, Ma L, Yang Y, Zhang X, Zheng N, Zhang K, Joseph DB, Shroff H, Doan J, Pacanowski M, Smpokou P, Donohue K, Joffe HV. Genet Med 2022 Dec 12

Use of single-arm trials for US Food and Drug Administration drug approval in oncology, 2002-2021. Agrawal S, Arora S, Amiri-Kordestani L, de Claro RA, Fasho- yin-Aje L, Gormley N, Kim T, Lemery S, Mehta GU, Scott EC, Singh H, Tang S, Theoret MR, Pazdur R, Kluetz PG, Beaver JA. JAMA Oncol 2022 Dec 29

FDA approval summary: belzutifan for von Hippel-Lindau disease associated tumors.

Fallah J, Brave MH, Weinstock C, Mehta GU, Bradford D, Gittleman H, Bloomquist EW, Charlab R, Hamed SS, Miller CP, Dorff SE, Chambers WA, Mixter BD, Dinin J, Pierce WF, Ricks TK, Tang S, Donoghue M, Pazdur R, Amiri-Kordestani L, Ibrahim A, Beaver JA. Clin Cancer Res 2022 Nov 14;28(22):4843-8

FDA approval summary: belantamab mafodotin for patients with relapsed or refractory multiple myeloma.

Baines AC, Ershler R, Kanapuru B, Xu Q, Shen G, Li L, Ma L, Okusanya OO, Simpson NE, Nguyen W, Theoret MR, Pazdur R, Gormley NJ. Clin Cancer Res 2022 Nov 1;28(21):4629-33

Prostate cancer theranostics: concurrent approvals by the Food and Drug Administration of the first diagnostic imaging drug indicated to select patients for a paired radioligand therapeutic drug.

Hofling AA, Fotenos AF, Niu G, Fallah J, Agrawal S, Wang SJ, Marzella L. J Nucl Med 2022 Nov;63(11):1642-3

Comments on “Statistical methodology for highly variable compounds: a novel design approach for the Ofatumumab phase 2 bioequivalence study”.

Sun W, Schuirmann DJ, Grosser S. Pharm Stat 2022 Nov;21(6):1366-7

Improving methods of identifying anaphylaxis for medical product safety surveillance using natural language processing and machine learning

Carrell DS, Gruber S, Floyd JS, Bann MA, Cushing-Haugen KL, Johnson RL, Graham V, Cronkite DJ, Hazlehurst BL, Felcher AH, Bejan CA, Kennedy A, Shinde M, Karami S, Ma Y, Stojanovic D, Zhao Y, Ball R, Nelson J. Am J Epidemiol 2022 Nov

Social media for early characterization of pandemic symptoms: a qualitative analysis of patient-reported COVID-19 experiences.

Khashei M, Janiczak S, Clair CS, Liu W, Song JJ, Hua W, Falconer M, Eworuke E. Pharmacoepidemiol Drug Saf 2022 Nov 5

Validation of claims-based algorithms to identify non-live birth outcomes.

Zhu Y, Bateman BT, Hernandez-Diaz S, Gray KJ, Straub L, Reimers RM, Manning-Geist B, Yoselevsky E, Taylor LG, Ouellet-Hellstrom R, Ma Y, Qiang Y, Hua W, Huybrechts KF. Pharmacoepidemiol Drug Saf 2022 Nov 24

Should responder analyses be conducted on continuous outcomes?

Abugov R, Clark J, Higginbotham L, Li F, Nie L, Reasner D, Rothmann M, Yuan X, Sharretts J. Pharm Stat 2022 Nov 23

The FDA Oncology Center of Excellence scientific collaborative: charting a course for applied regulatory science research in oncology.

Schneider JA, Gong Y, Goldberg KB, Kluetz PG, Theoret MR, Amiri-Kordestani L, Beaver JA, Fashoyin-Aje L, Gormley NJ, Jaigirdar AA, Lemery SJ, Mishra-Kalyani PS, Reaman GH, Rivera DR, Rubinstein WS, Singh H, Sridhara R, Pazdur R. Clin Cancer Res 2021 Oct 1;27(19):5161-7

FDA approval summary: cabozantinib for differentiated thyroid cancer.

Duke ES, Barone AK, Chatterjee S, Mishra-Kalyani PS, Shen YL, Isikwei E, Zhao H, Bi Y, Liu J, Rahman NA, Wearne E, Leighton JK, Stephenson M, Ojofeitimi I, Scepura B, Nair A, Pazdur R, Beaver JA, Singh H. Clin Cancer Res 2022 Oct 1;28(19):4173-7

Accounting for delayed entry into observational studies and clinical trials: length-biased sampling and restricted mean survival time.

Lee MT, Lawrence J, Chen Y, Whitmore GA. Lifetime Data Anal 2022 Oct; 28(4):637-58

FDA/Arthritis Foundation osteoarthritis drug development workshop recap: assessment of long-term benefit.

Kim JS, Borges S, Clauw DJ, Conaghan PG, Felson DT, Fleming TR, Glaser R, Hart E, Hochberg M, Kim Y, Kraus VB, Lapteva L, Li X, Majumdar S, McAlindon TE, Mobasheri A, Neogi T, Roemer FW, Rothwell R, Shibuya R, Siegel J, Simon LS, Spindler KP, Nikolov NP. Semin Arthritis Rheum 2022 Oct; 56:152070

Improving dose-optimization processes used in oncology drug development to minimize toxicity and maximize benefit to patients.

Fourie Zirkelbach J, Shah M, Vallejo J, Cheng J, Ayyoub A, Liu J, Hudson R, Sridhara R, Ison G, Amiri-Kordestani L, Tang S, Gwise T, Rahman A, Pazdur R, Theoret MR. J Clin Oncol 2022 Oct 20;40(30):3489-500

Impact of model misspecification on model-based tests in PK studies with parallel design: real case and simulation studies.

Guhl M, Mercier F, Hofmann C, Sharan S, Donnelly M, Feng K, Sun W, Sun G, Grosser S, Zhao L, Fang L, Mentré F, Comets E, Bertrand JJ. Pharmacokinet Pharmacodyn 2022 Oct;49(5):557-77

FDA approval summary: pemigatinib for previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with FGFR2 fusion or other rearrangement.

Patel TH, Marcus L, Horiba MN, Donoghue M, Chatterjee S, Mishra-Kalyani PS, Schuck RN, Li Y, Zhang X, Fourie Zirkelbach J, Charlab R, Liu J, Yang Y, Lemery SJ, Pazdur R, Theoret MR, Fashoyin-Aje LA. Clin Cancer Res 2022 Oct

Benchmarking the quality practices of global pharmaceutical manufacturing to advance supply chain resilience.

Fellows M, Friedli T, Li Y, Maguire J, Rakala N, Ritz M, Bernasconi M, Seiss M, Stiber N, Swatek M, Viehmann A. AAPS J 2022 Oct 20;24(6):111

Performance of LTMLE in the presence of missing data in control-matched longitudinal studies.

Wang SJ, Huang ZP, Zhu H. Stat Biopharm Res 2022 Oct 3

Statistical consideration for fit-for-use real-world data to support regulatory decision making in drug development.

Levenson M, He WL, Chen L, Dharmarajan S, Izem R, Meng ZL, Pang H, Rockhold F. Stat Biopharm Res 2022 Oct 7

Standardization of continuous and categorical covariates in sparse penalized regressions.

Li X, Ma Y, Pan Q. Stat Methods Med Res 2022 Oct 3

Developing a targeted learning-based statistical analysis plan.

Gruber S, Lee H, Phillips R, Ho M, van der Laan M Stat Biopharm Res 2022 Oct 3

Qualitative versus quantitative treatment-by-subgroup interaction in equivalence studies with multiple subgroups. Sun WJ, Schuirmann D, Grosser S. Stat Biopharm Res 2022 Oct 31

Evaluation of treatment effect in underrepresented population in cancer trials: discussion with international regulators.

Sridhara R, Marchenko O, Jiang Q, Barksdale E, Chen J, Dreyer N, Fashoyin-Aje L, Garrett-Mayer E, Gormley N, Gwise T, Hess L, Mandrekar S, Pignatti F, Rantell K, Raven A, Shen YL, Singh H, Tendler CL, Theoret M, Pazdur R. Stat Biopharm Res 2022 Oct 31

Data-adaptive selection of the propensity score truncation level for inverse probability weighted and targeted maximum likelihood estimators of marginal point treatment effects.

Am J Epidemiol 2022 Sep;191(9):1640-51 Gruber S, Phillips RV, Lee H, van der Laan MJ

Assessing microstructural critical quality attributes in PLGA microspheres by FIB-SEM analytics.

Clark AG, Wang R, Qin Y, Wang Y, Zhu A, Lomeo J, Bao Q, Burgess DJ, Chen J, Qin B, Zou Y, Zhang S

J Control Release 2022 Sep;349:580-91

Changes in physical activity and sedentary time in United States adults in response to COVID-19.

Matthews CE, Saint-Maurice P, Fulton JE, Patel S, Loftfield E, Sampson JN, Keadle SK, Berrigan D

PLoS One, 2022 Sep 9;17(9):e0273919

FDA approval summary: mobocertinib for metastatic non-small cell lung cancer with EGFR exon 20 insertion mutations.

Duke ES, Stapleford L, Drezner N, Amatya AK, Mishra-Kalyani PS, Shen YL, Maxfield K, Fourie Zirkelbach J, Bi Y, Liu J, Zhang X, Wang H, Yang Y, Zheng N, Reece K, Wearne E, Glen JJ, Ojofeitimi I, Scepura B, Nair A, Bikkavilli RK, Ghosh S, Philip R, Pazdur R, Beaver JA, Singh H, Donoghue M. Clin Cancer Res, 2022 Sep 16

Retrieved-dropout-based multiple imputation for time-to- event data in cardiovascular outcome trials.

He J, Crackel R, Koh W, Chen LW, Li F, Zhang J, Rothmann M J. Biopharm Stat, 2022 Sep 19

The use of machine learning in regulatory drug safety evaluation.

Zhang D, Song J, Dharmarajan S, Jung TH, Lee H, Ma Y, Zhang RM, Levenson M. Stat Biopharm Res, 2022 Sep 23

Cancer clinical trials beyond pandemic: report of an American Statistical Association Biopharmaceutical Section Open Forum discussion.

Sridhara R, Barksdale E, Marchenko O, Jiang Q, Ando Y, Bloomquist E, Coory M, Crouse M, Degtyarev E, Framke T, Freidlin B, Gerber DE, Gwise T, Josephson F, Hess L, Kluetz P, Li D, Mandrekar S, Posch M, Rantell K, Ratitch B, Raven A, Roes K, Rufibach K, Sarac SB, Simon R, Singh H, Theoret M, Thomson A, Zuber E, Li Shen Y, Pazdur R. Stat Biopharm Res 2022, Sep 22

FDA approval summary: decitabine and cedazuridine tablets for myelodysplastic syndromes.

Kim N, Norsworthy KJ, Subramaniam S, Chen H, Manning ML, Kitabi E, Earp J, Ehrlich LA, Okusanya OO, Vallejo J, Gehrke BJ, de Claro RA, Pazdur R. Clin Cancer Res 2022, Aug 15;28(16):3411-6

FDA approval summary: crizotinib for pediatric and young adult patients with relapsed or refractory systemic anaplastic large cell lymphoma.

Merino M, Kasamon Y, Li H, Ma L, Leong R, Zhou J, Reaman G, Chambers W, Richardson N, Theoret M, Pazdur R, Gormley N. Pediatr Blood Cancer 2022, Aug;69(8):e29602

Mortality among Parkinson’s disease patients treated with pimavanserin or atypical antipsychotics: an observational study in Medicare beneficiaries.

Mosholder AD, Ma Y, Akhtar S, Podskalny GD, Feng Y, Lyu H, Liao J, Wei Y, Wernecke M, Leishear K, Nelson LM, MaCurdy TE, Kelman JA, Graham DJ. Am J Psychiatry, 2022 Aug;179(8):553-61

Global landscape of benefit-risk considerations for medicinal products: current state and future directions.

Waschbusch M, Rodriguez L, Brueckner A, Lee KJ, Li X, Mokliatchouk O, Tremmel L, Yuan SS. Pharmaceut Med 2022 Aug; 36(4):201-13

Bayesian and frequentist approaches to sequential monitoring for futility in oncology basket trials: a comparison of Simon’s two-stage design and Bayesian predictive probability monitoring with information sharing across baskets.

Kaizer A, Zabor E, Nie L, Hobbs B PLoS One, 2022 Aug 2;17(8):e0272367

Bayesian sample size planning tool for phase I dose-finding Trials.

Lin X, Lyu J, Yuan S, Bi D, Wang SJ, Ji Y. JCO Precis Oncol 2022 Aug; 6:e2200046

Improving traditional registrational trial endpoints: development and application of a desirability of outcome ranking (DOOR) endpoint for complicated urinary tract infection clinical trials.

Howard-Anderson J, Hamasaki T, Dai W, Collyar D, Rubin D, Nambiar S, Kinamon T, Hill C, Gelone SP, Mariano D, Baba T, Holland TL, Doernberg SB, Chambers HF, Fowler VG, Evans SR, Boucher HW, Antibacterial Resistance Leadership Group. Clin Infect Dis, 2022 Aug 29

Concept endpoints informing design considerations for confirmatory clinical trials in osteoarthritis.

Kim Y, Levin G, Nikolov NP, Abugov R, Rothwell R. Arthritis Care Res 2022 Jul;74(7):1154-62

Type I error considerations in master protocols with common control in oncology trials: report of an American Statistical Association Biopharmaceutical Section Open Forum discussion.

Sridhara R, Marchenko O, Jiang Q, Pazdur R, Posch M, Redman M, Tymofyeyev Y, Li XY, Theoret M, Shen YL, Gwise T, Hess L, Coory M, Raven A, Kotani N, Roes K, Josephson F, Berry S, Simon R, Binkowitz B. Stat Biopharm Res, 2022; 14(3):349-52

Use of nonconcurrent common control in Master Protocols in oncology trials: report of an American Statistical Association Biopharmaceutical Section open forum discussion.

Sridhara R, Marchenko O, Jiang Q, Pazdur R, Posch M, Berry S, Theoret M, Shen YL, Gwise T, Hess L, Raven A, Rantell K, Roes K, Simon R, Redman M, Ji Y, Lu C. Stat Biopharm Res, 2022; 14(3):353-7

Plasma protein markers in advanced or metastatic colorectal cancer patients receiving chemotherapy with bevacizumab or cetuximab: results from CALGB 80405 (Alliance).

Nixon AB, Sibley AB, Liu Y, Hatch AJ, Jiang C, Mulkey F, Starr MD, Brady JC, Niedzwiecki D, Venook AP, Baez-Diaz L, Lenz HJ, O’Neil BH, Innocenti F, Meyerhardt JA, O’Reilly EM, Owzar K, Hurwitz HI. Clin Cancer Res, 2022 Jul 1;28(13):2779-88

Clinical and statistical perspectives on the ICH E9(R1) estimand framework implementation.

Ionan AC, Paterniti M, Mehrotra DV, Scott J, Ratitch B, Collins S, Gomatam S, Nie L, Rufibach K, Bretz F. Stat Biopharm Res, 2022 Jul 6

FDA approval summary: ivosidenib for the treatment of patients with advanced unresectable or metastatic, chemotherapy refractory cholangiocarcinoma with an IDH1 mutation.

Casak SJ, Pradhan S, Fashoyin-Aje LA, Ren Y, Shen YL, Xu Y, Chow ECY, Xiong Y, Zirklelbach JF, Liu J, Charlab R, Pierce WF, Fesenko N, Beaver JA, Pazdur R, Kluetz PG, Lemery SJ. Clin Cancer Res, 2022 Jul 1; 28(13):2733-7

FDA approval summary: oral azacitidine for continued treatment of adults with acute myeloid leukemia unable to complete intensive curative therapy.

Jen EY, Wang X, Li M, Li H, Lee SL, Ni N, Przepiorka D, Vallejo J, Leong R, Ma L, Gehrke BJ, McLamore S, Theoret MR, de Claro RA. Clin Cancer Res, 2022 Jul 15; 28(14):2989-993

Analysis of association of radiation therapy with risk of adverse events in patients receiving immunotherapy using pooled trial data matched by propensity score--reply.

Weinstock C, Amatya A, Beaver. JA JAMA Oncol, 2022 Jul;8(7):1073

Acute pain pathways: protocol for a prospective cohort study.

Jeffery MM, Ahadpour M, Allen S, Araojo R, Bellolio F, Chang N, Ciaccio L, Emanuel L, Fillmore J, Gilbert GH, Koussis P, Lee C, Lipkind H, Mallama C, Meyer T, Moncur M, Nuckols T, Pacanows- ki MA, Page DB, Papadopoulos E, Ritchie JD, Ross JS, Shah ND, Soukup M, St Clair CO, Tamang S, Torbati S, Wallace DW, Zhao Y, Heckmann R. BMJ Open, 2022 Jul 5;12(7): e058782

PROPHETIC EU: prospective identification of pneumonia in hospitalized patients in the intensive care unit in European and United States cohorts.

Bergin SP, Calvert SB, Farley J, Sun JL, Chiswell K, Dieperink W, Kluytmans J, Lopez-Delgado JC, Leon-Lopez R, Zervos MJ, Kollef MH, Sims M, Kabchi BA, Rubin D, Santiago J, Natarajan M, Tenaerts P, Fowler VG, Holland TL, Bonten MJ, Hullegie SJ. Open Forum Infect Dis, 2022 Jul;9(7): ofac231

Novel methods for pregnancy drug safety surveillance in the FDA Sentinel System.

Suarez EA, Nguyen M, Zhang D, Zhao Y, Stojanovic D, Munoz M, Liedtka J, Anderson A, Liu W, Dashevsky I, Cole D, DeLuccia S, Menzin T, Noble J, Maro JC. Pharmacoepidemiol Drug Saf, 2022 Jul 24

Rejoinder to the commentaries on “The current landscape in biostatistics of real-world data and evidence: label expansion”.

Levenson M, He WL. Stat Biopharm Res, 2022 Jul 18

FDA public workshop summary — Coccidioidomycosis (Valley Fever): considerations for development of antifungal drugs.

O’Shaughnessy E, Yasinskaya Y, Dixon C, Higgins K, Moore J, Reynolds K, Ampel NM, Angulo D, Blair JE, Catanzaro A, Galgiani JN, Garvey E, Johnson R, Larwood DJ, Lewis G, Purdie R, Rex JH, Shubitz LF, Stevens DA, Page SJ, Shukla SJ, Farley JJ, Nambiar S. Clin Infect Dis, 2022 Jun 1;74(11):2061-6

FDA approval summary: pembrolizumab, atezolizumab, and cemiplimab-rwlc as single agents for first-line treatment of advanced/metastatic PD-L1 high NSCLC.

Akinboro O, Larkins E, Pai-Scherf LH, Mathieu LN, Ren Y, Cheng J, Fiero MH, Fu W, Bi Y, Kalavar S, Jafri S, Mishra-Kalyani PS, Fourie Zirkelbach J, Li H, Zhao H, He K, Helms WS, Chuk MK, Wang M, Bulatao I, Herz J, Osborn BL, Xu Y, Liu J, Gong Y, Sick- afuse S, Cohen R, Donoghue M, Pazdur R, Beaver JA, Singh H Clin. Cancer Res, 2022 Jun 1;28(11):2221-8

Bayesian basket trial design with false-discovery rate control.

Zabor EC, Kane MJ, Roychoudhury S, Nie L, Hobbs BP. Clin Trials 2022 Jun;19(3):297-306

FDA approval summary: belumosudil for adult and pediatric patients 12 years and older with chronic GVHD after two or more prior lines of systemic therapy. Przepiorka D, Le RQ, Ionan A, Li RJ, Wang YH, Gudi R, Mitra S, Vallejo J, Okusanya OO, Ma L, Yang Y, Patel P, Mezaache D, Shah R, Banerjee A, McLamore S, Maung AN, Goldberg KB, Pazdur R, Theoret MR, de Claro RA. Clin Cancer Res 2022 Jun 15;28(12):2488-92

Group sequential design for randomized trials using “first hitting time” model.

Chen Y, Lawrence J, Lee MT. Stat Med 2022 Jun 15;41(13):2375-402

FDA approval summary: ruxolitinib for treatment of chronic graft-versus-host disease after failure of one or two lines of systemic therapy.

Le RQ, Wang X, Zhang H, Li H, Przepiorka D, Vallejo J, Leong R, Ma L, Goldberg KB, Pazdur R, Theoret MR, De Claro A Oncologist 2022 Jun;27(6):493-500

Timing is everything: the importance of patient-reported outcome assessment frequency when characterizing symptomatic adverse events.

King-Kallimanis BL, Bhatnagar V, Horodniceanu EG, Chen TY, Kluetz PG. Clin Trials 2022 Jun;19(3):267-73

A targeted simulation-extrapolation method for evaluating biomarkers based on new technologies in precision medicine. Wang D, Wang SJ, Xu J, Lababidi S. Pharm Stat 2022 May;21(3):584-98

Rates of, and factors associated with, switching among generic levothyroxine preparations in commercially insured American adults.

Brito JP, Deng Y, Ross JS, Choi NH, Graham DJ, Qiang Y, Rantou E, Wang Z, Zhao L, Shah ND, Lipska KJ Endocrine 2022 May;76(2):349-58

Time to deterioration of symptoms or function using patient-reported outcomes in cancer trials.

Fiero MH, Roydhouse JK, Bhatnagar V, Chen TY, King- Kallimanis BL, Tang S, Kluetz PG. Lancet Oncol 2022 May;23(5):e229-34

Application of modeling and simulation to identify a shortened study duration and novel bioequivalence metric for a long-acting intrauterine system.

Sharan S, Choi S, Zou Y, Wang Y, Kim MJ, Fang L, Choi S, Makhlouf F, Grosser SC, Zhang X, Zhao L. AAPS J, 2022 May 2;24(3):63

Consideration of the adaptive randomization allocation ratio in the presence of treatment group heteroscedasticity in clinical trials.

Lu J, Chen YF. J Biopharm Stat 2022 May 4;32(3):511-26

Statistical learning in preclinical drug proarrhythmic assessment.

Xi NM, Hsu YY, Dang Q, Huang DP. J Biopharm Stat, 2022 May 4;32(3):450-473

A structured framework for adaptively incorporating external evidence in sequentially monitored clinical trials.

Kwiatkowski E, Andraca-Carrera E, Soukup M, Psioda MA. J Biopharm Stat, 2022 May 4;32(3):474-95

A Bayesian model with application for adaptive platform trials having temporal changes.

Wang C, Lin M, Rosner GL, Soon G. Biometrics, 2022 Apr 27

Effects of compounded nonnormality of residuals in hierarchical linear modeling.

Man KW, Schumacker R, Morell M, Wang YR. Educ Psychol Meas, 2022 Apr;82(2):330-55

A trans-agency workshop on the pathophysiology of radiation-induced lung injury.

Cassatt DR, Gorovets A, Karimi-Shah B, Roberts R, Price PW, Satyamitra MM, Todd N, Wang SJ, Marzella L. Radiat Res, 2022 Apr;197(4):415-33

FDA approval summary: sotorasib for KRAS G12C-mutated metastatic NSCLC.

Nakajima EC, Drezner N, Li X, Mishra-Kalyani PS, Liu Y, Zhao H, Bi Y, Liu J, Rahman A, Wearne E, Ojofeitimi I, Hotaki LT, Spillman D, Pazdur R, Beaver JA, Singh H. Clin Cancer Res, 2022 Apr 15;28(8):1482-6

FDA approval summary: margetuximab plus chemotherapy for advanced or metastatic HER2-positive breast cancer.

Royce M, Osgood CL, Amatya AK, Fiero MH, Chang CJG, Ricks TK, Shetty KA, Kraft J, Qiu J, Song P, Charlab R, Yu J, King KE, Rastogi A, Janelsins B, Weinberg WC, Clouse K, Bor- ders-Hemphill V, Brown L, Gomez-Broughton C, Li Z, Nguyen TT, Qiu Z, Ly AT, Chang S, Gao T, Tu CM, King-Kallimanis B, Pierce WF, Chiang K, Lee C, Goldberg KB, Leighton JK, Tang S, Pazdur R, Beaver JA, Amiri-Kordestani L. Clin Cancer Res, 2022 Apr 15;28(8):1487-92

External control arms in oncology: current use and future directions.

Mishra-Kalyani PS, Amiri Kordestani L, Rivera DR, Singh H, Ibrahim A, DeClaro RA, Shen Y, Tang S, Sridhara R, Kluetz PG, Concato J, Pazdur R, Beaver JA. Ann Oncol, 2022 Apr;33(4):376-83

FDA approval summary: abemaciclib with endocrine therapy for high-risk early breast cancer.

Royce M, Osgood C, Mulkey F, Bloomquist E, Pierce WF, Roy A, Kalavar S, Ghosh S, Philip R, Rizvi F, Mixter BD, Tang S, Pazdur R, Beaver JA, Amiri-Kordestani L. J Clin Oncol 2022 Apr 10;40(11):1155-62

Association between generic-to-generic levothyroxine switching and thyrotropin levels among US adults.

Brito JP, Deng Y, Ross JS, Choi NH, Graham DJ, Qiang Y, Rantou E, Wang Z, Zhao L, Shah ND, Lipska KJ. JAMA Intern Med, 2022 Apr;182(4):418-25

US Food and Drug Administration analysis of patient-reported diarrhea and its impact on function and quality of life in patients receiving treatment for breast cancer.

Chen TY, King-Kallimanis BL, Merzoug L, Horodniceanu EG, Fiero MH, Gao JJ, Beaver JA, Bhatnagar V, Kluetz P. Value Health, 2022 Apr;25(4):566-70

Clinical pharmacology considerations for the approval of belimumab for the treatment of adult patients with active lupus nephritis: a regulatory perspective.

Liu T, Neuner R, Thompson A, Pottackal G, Petullo D, Liu J, Nikolov N, Sahajwalla C, Doddapaneni S, Chen J. Lupus 2022 Apr;31(4):424-32

Informatics in medical product regulation: the right drug at the right dose for the right patient.

Almario EN, Kettermann A, Popat V. Methods Mol Biol 2022;2486:277-314

Exploring racial disparities in treatment patterns and outcomes for patients with multiple myeloma using real world data.

Maignan K, Fashoyin-Aje LA, Torres AZ, Fernandes LL, Gwise T, Baxi SB, Roose JP, Rivera DR, Shen YL, Kluetz PG, Gormley NJ. Blood Cancer J, 2022 Apr 19;12(4):65

Bayesian adaptive design for pediatric clinical trials incorporating a community of prior beliefs.

Wang Y, Travis J, Gajewski B. BMC Med Res Methodol, 2022 Apr 21;22(1):118

aphBO-2GP-3B: a budgeted asynchronous parallel multi-acquisition functions for constrained Bayesian optimization on high-performing computing architecture.

Tran A, Eldred M, Wildey T, McCann S, Sun J, Visintainer RJ. Struct Multidiscipl Optim, 2022 Apr 1;65:132

U.S. FDA drug approvals for gynecological malignancies: a decade in review.

Arora S, Narayan P, Ison G, Berman T, Suzman DL, Wedam S, Prowell TM, Ghosh S, Philip R, Osgood CL, Gao JJ, Shah M, Krol D, Wahby S, Royce M, Brus C, Bloomquist EW, Fiero MH, Tang S, Pazdur R, Ibrahim A, Amiri-Kordestani L, Beaver JA. Clin Cancer Res, 2022 Mar 15;28(6):1058-71

U.S. FDA drug approvals for breast cancer: a decade in review.

Arora S, Narayan P, Osgood CL, Wedam S, Prowell TM, Gao JJ, Shah M, Krol D, Wahby S, Royce M, Ghosh S, Philip R, Ison G, Berman T, Brus C, Bloomquist EW, Fiero MH, Tang S, Pazdur R, Ibrahim A, Amiri-Kordestani L, Beaver JA. Clin Cancer Res, 2022 Mar 15;28(6):1072-86

Potential role of heme oxygenase-1 in the resolution of experimentally induced colitis through regulation of macrophage polarization.

Gwak SY, Kim SJ, Park J, Kim SH, Joe Y, Lee HN, Kim W, Muna IA, Na HK, Chung HT, Surh YJ. Gut Liver, 2022 Mar 15;16(2):246-58

Response rate, event-free survival, and overall survival in newly diagnosed acute myeloid leukemia: US Food and Drug Administration trial-level and patient-level analyses.

Norsworthy KJ, Gao X, Ko CW, Pulte ED, Zhou J, Gong Y, Shen YL, Vallejo J, Gwise TE, Sridhara R, Deisseroth AB, Farrell AT, de Claro RA, Blumenthal GM, Pazdur R. J Clin Oncol, 2022 Mar 10;40(8):847-54

Corrigendum to “Bringing safe and effective therapies to premenopausal women with breast cancer: efforts to broaden eligibility criteria”. [Annals of Oncology 32 (2021) 950-953]

Gao JJ, Krol D, Narayan P, Cardoso F, Regan MM, Goetz MP, Hurvitz SA, Mauro L, Hodgdon C, Miller CP, Booth B, Bloomquist E, Ison G, Osgood C, Bhatnagar V, Fashoyin-Aje L, Pazdur R, Amiri-Kordestani L, Beaver JA. Ann Oncol, 2022 Mar;33(3):356

Comments on the evaluation of suicidality risk of newer antiseizure medications.

Apata J, Mosholder A, Levenson M. JAMA Neurol, 2022 Mar;79(3):309-10

Analysis of racial and ethnic disparities in multiple myeloma U.S. FDA drug approval trials.

Kanapuru B, Fernandes LL, Fashoyin-Aje LA, Baines AC, Bhatnagar V, Ershler R, Gwise T, Kluetz P, Pazdur R, Pulte E, Shen YL, Gormley N. Blood Adv, 2022 Mar 22;6(6):1684-91

Theranostics approach in drug development: is there study efficiency when the prevalence of the molecular target is very high?

Wang SJ, Fotenos A, Masters SC, Marzella L. Theranostics, 2022;12(7):3079-83

FDA approval summary: tivozanib for relapsed or refractory renal cell carcinoma.

Chang E, Weinstock C, Zhang L, Fiero MH, Zhao M, Zahalka E, Ricks TK, Fourie Zirkelbach J, Qiu J, Yu J, Chen XH, Bhatnagar V, Goldberg KB, Tang S, Kluetz PG, Pazdur R, Ibrahim A, Beaver JA, Amiri-Kordestani L. Clin Cancer Res, 2022 Feb;28(3):441-5

FDA approval summary: nivolumab in combination with ipilimumab for the treatment of unresectable malignant pleural mesothelioma.

Nakajima EC, Vellanki PJ, Larkins E, Chatterjee S, Mishra- Kalyani PS, Bi Y, Qosa H, Liu J, Zhao H, Biable M, Hotaki LT, Shen YL, Pazdur R, Beaver JA, Singh H, Donoghue M. Clin Cancer Res, 2022 Feb;28(3):446-51

Considerations to address missing data when deriving clinical trial endpoints from digital health technologies.

Di J, Demanuele C, Kettermann A, Karahanoglu FI, Cappelleri JC, Potter A, Bury D, Cedarbaum JM, Byrom B. Contemp Clin Trials, 2022 Feb;113:106661

Association of radiation therapy with risk of adverse events in patients receiving immunotherapy: a pooled analysis of trials in the US Food and Drug Administration Database.

Anscher MS, Arora S, Weinstock C, Amatya A, Bandaru P, Tang C, Girvin AT, Fiero MH, Tang S, Lubitz R, Amiri-Kordestani L, Theoret MR, Pazdur R, Beaver JA. JAMA Oncol, 2022 Feb;8(2):232-40

NK cells require immune checkpoint receptor LILRB4/gp49B to control neurotropic Zika virus infections in mice.

Lee HN, Manangeeswaran M, Lewkowicz AP, Engel K, Chowdhury M, Garige M, Eckhaus MA, Sourbier C, Ireland DD, Verthelyi D. JCI Insight, 2022 Feb 8;7(3):e151420

Comment on “Estimands for recurrent event endpoints in the presence of a terminal event”.

Hung HMJ, Wang SJ. Stat Biopharm Res, 2022 Feb 24

FDA approval summary: revised indication and dosing regimen for ponatinib based on the results of the OPTIC trial.

Pulte ED, Chen HY, Price LSL, Gudi R, Li HS, Okusanya OO, Ma L, Rodriguez L, Vallejo J, Norsworthy KJ, de Claro RA, Theoret MR, Pazdur R. Oncologist, 2022 Feb;27(2):149-57

Efficient model-based bioequivalence testing.

Mollenhoff K, Loingeville F, Bertrand J, Nguyen TT, Sharan S, Zhao L, Fang L, Sun G, Grosser S, Mentre F, Dette H Biostatistics 2022 Jan;23(1):314-27

The role of FDA CDER statisticians in response efforts to the COVID-19 pandemic.

Collins SH, Price D, Johnson LL. Stat Biopharm Res, 2022;14(1):3-4

FDA approval summary: capmatinib and tepotinib for the treatment of metastatic NSCLC harboring MET exon 14 skipping mutations or alterations.

Mathieu LN, Larkins E, Akinboro O, Roy P, Amatya AK, Fiero MH, Mishra-Kalyani PS, Helms WS, Myers CE, Skinner AM, Aungst S, Jin R, Zhao H, Xia H, Fourie Zirkelbach J, Bi Y, Li Y, Liu J, Grimstein M, Zhang X, Woods S, Reece K, Abukhdeir AM, Ghosh S, Philip R, Tang S, Goldberg KB, Pazdur R, Beaver JA, Singh H. Clin Cancer Res, 2022 Jan;28(2):249-54

Refining neoadjuvant therapy clinical trial design for muscle-invasive bladder cancer before cystectomy: a joint US Food and Drug Administration and Bladder Cancer Advocacy Network workshop.

Chang E, Apolo AB, Bangs R, Chisolm S, Duddalwar V, Efstathiou JA, Goldberg KB, Hansel DE, Kamat AM, Kluetz PG, Lerner SP, Plimack E, Prowell T, Singh H, Suzman D, Yu EY, Zhang H, Beaver JA, Pazdur R, Weinstock C, Galsky MD. Nat Rev Urol, 2022 Jan;19(1):37-46

The Friends of Cancer Research real world data (RWD) collaboration pilot 2.0: methodological recommendations from oncology case studies.

Rivera DR, Henk HJ, Garrett-Mayer E, Christian JB, Belli AJ, Bruinooge SS, Espirito JL, Sweetnam C, Izano MA, Natanzon Y, Robert NJ, Walker MS, Cohen AB, Boyd M, Enewold L, Hansen E, Honnold R, Kushi L, Mishra Kalyani PS, Pe Benito R, Sakoda LC, Sharon E, Tymejczyk O, Valice E, Wagner J, Lasiter L, Allen JD. Clin Pharmacol Ther, 2022 Jan;111(1):283-92

Heart failure population with therapeutic response to sacubitril/valsartan, spironolactone and candesartan: FDA perspective.

Gandotra C, Clark J, Liu Q, Senatore FF, Rose M, Zhang J, Stockbridge NL. Ther Innov Regul Sci, 2022 Jan;56(1):4-7

Why are not there more Bayesian clinical trials? Perceived barriers and educational preferences among medical researchers involved in drug development.

Medical Outreach Subteam of the Drug Information Association Bayesian Scientific Working Group, Clark J, Muhlemann N, Na- tanegara F, Hartley A, Wenkert D, Wang F, Harrell FE Jr, Bray R. Ther Innov Regul Sci, 2022 Jan 3

Challenges and potential strategies utilizing external data for efficacy evaluation in small-sized clinical trials.

Jiao F, Chen YF, Min M, Jimenez S. J Biopharm Stat, 2022 Jan 2;32(1):21-33

Exploring the potential of external control arms created from patient level data: a case study in non-small cell lung cancer.

Yin X, Mishra-Kalyan PS, Sridhara R, Stewart MD, Stuart EA, Davi RC. J Biopharm Stat, 2022 Jan 2;32(1):204-18

Nonproportional hazards—an evaluation of the MaxCombo test in cancer clinical trials.

Shen YL, Wang X, Sirisha M, Mulkey F, Zhou JX, Gao X, Zhang LJ, Gwise T, Tang SH, Theoret M, Pazdur R, Sridhara R. Stat Biopharm Res, 2022 Jan 4