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Helping our clients find solutions to their challenges is our top priority. This library of case studies, journal articles and other publications are an excellent resource for your team.
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Adaptive Design in early phase Clinical Trials
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Evolution of Estimand framework and use in Clinical Trials
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Bayesian Statistics with Applications in Clinical Trials in the New Era
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Precision Recruitment Forecasting with Simulation-Based Technologies
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Aligning Clinical Strategy with Regulatory and Market Objectives Through Modern Forecasting
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Empowered Trial Selection with Weighting and Prioritization Tools
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Strategic Clinical Trial Design Unlocks Innovative Funding Opportunity
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Optimizing Interim Analyses to Conserve Resources and Speed Strong Therapies to Market
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PSE RELEVANT PUBLICATIONS

  • Amyloid-Related Imaging Abnormalities in 2 Phase 3 Studies Evaluating Aducanumab in Patients With Early Alzheimer Disease (2022) pp. 13-21.
  • Salloway S, Chalkias S, Barkhof F, Burkett P, Barakos J, Purcell D, Suhy J, Forrestal F, Tian Y, Umans K, Wang G, Singhal P, Budd Haeberlein S, Smirnakis K. Effect of early treatment with fluvoxamine on risk of emergency care and hospitalisation among patients with COVID-19: the TOGETHER randomised, platform clinical trial (2022) pp. e42-e51
    Reis G, Dos Santos Moreira-Silva EA, Silva DCM, Thabane L, Milagres AC, Ferreira TS, Dos Santos CVQ, de Souza Campos VH, Nogueira AMR, de Almeida APFG, Callegari ED, de Figueiredo Neto AD, Savassi LCM, Simplicio MIC, Ribeiro LB, Oliveira R, Harari O, Forrest JI, Ruton H, Sprague S, McKay P, Glushchenko AV, Rayner CR, Lenze EJ, Reiersen AM, Guyatt GH, Mills EJ; TOGETHER investigators.
  • Optimal adaptive promising zone designs (2022) Mehta C, Bhingare A, Liu L, Senchaudhuri P.
  • Implementation of platform trials in the COVID-19 pandemic: A rapid review (2022)
    Vanderbeek AM, Bliss JM, Yin Z, Yap C.
  • Evaluating COVID-19 vaccines in the real world (2022) pp. 1205-1206.
    Mills EJ, Reis G.
  • Effect of Early Treatment with Ivermectin among Patients with Covid-19 (2022).
    Reis G, Silva EASM, Silva DCM, Thabane L, Milagres AC, Ferreira TS, Dos Santos CVQ, Campos VHS, Nogueira AMR, de Almeida APFG, Callegari ED, Neto ADF, Savassi LCM, Simplicio MIC, Ribeiro LB, Oliveira R, Harari O, Forrest JI, Ruton H, Sprague S, McKay P, Guo CM, Rowland-Yeo K, Guyatt GH, Boulware DR, Rayner CR, Mills EJ; TOGETHER Investigators.
  • Emulating a target trial of the comparative effectiveness of clomiphene citrate and letrozole for ovulation induction (2022) pp. 793-805.
    Yland JJ, Chiu YH, Rinaudo P, Hsu J, Hernán MA, Hernández-Díaz S.
  • The (Harsh) Reality of Real-World Data External Comparators for Health Technology Assessment (2022)
    Cox O, Sammon C, Simpson A, Wasiak R, Ramagopalan S, Thorlund K.
  • Efficacy of non-intensive therapies approved for relapsed/refractory acute myeloid leukemia: a systematic literature review (2022).
    Russell-Smith TA, Gurskyte L, Muresan B, Mamolo CM, Gezin A, Cappelleri JC, Heeg B.
  • Humanistic and Economic Burden of Conversion Therapy Among LGBTQ Youths in the United States (2022)
    Forsythe A, Pick C, Tremblay G, Malaviya S, Green A, Sandman K.
  • Fluvoxamine for the Early Treatment of COVID-19: A Meta-analysis of Randomized Clinical Trials (2022).
    Guo CM, Harari O, Chernecki C, Thorlund K, Forrest JI.
  • Robust group sequential designs for trials with survival endpoints and delayed response (2022) pp. 343-360.
    Ghosh P, Ristl R, König F, Posch M, Jennison C, Götte H, Schüler A, Mehta C.
  • Effectiveness of crizotinib versus entrectinib in ROS1-positive non-small-cell lung cancer using clinical and real-world data (2022).
    Tremblay G, Groff M, Iadeluca L, Daniele P, Wilner K, Wiltshire R, Bartolome L, Usari T, Cappelleri JC, Camidge DR.
  • Solutions to problems of nonexistence of parameter estimates and sparse data bias in Poisson regression (2022) pp. 253-266.
    Joshi A, Geroldinger A, Jiricka L, Senchaudhuri P, Corcoran C, Heinze G.
  • Treatment Outcomes of High-Risk Non-Muscle Invasive Bladder Cancer (HR-NMIBC) in Real-World Evidence (RWE) Studies: Systematic Literature Review (SLR) (2022) pp. 35-48.
    Musat MG, Kwon CS, Masters E, Sikirica S, Pijush DB, Forsythe A.
  • How to Use and Interpret the Results of a Platform Trial: Users' Guide to the Medical Literature (2022) pp. 67-74. 
    Park JJH, Detry MA, Murthy S, Guyatt G, Mills EJ.
  • Helicobacter pylori infection treatment in the United States: clinical consequences and costs of eradication treatment failure (2022) pp. 1-17.
    Shah S, Hubscher E, Pelletier C, Jacob R, Vinals L, Yadlapati R.
  • Corrigendum: Pre-conceptional Maternal Vitamin B12 Supplementation Improves Offspring Neurodevelopment at 2 Years of Age: PRIYA Trial (2022).
    D'souza N, Behere RV, Patni B, Deshpande M, Bhat D, Bhalerao A, Sonawane S, Shah R, Ladkat R, Yajnik P, Bandyopadhyay SK, Kumaran K, Fall C, Yajnik CS.
  • ISPE-Endorsed Guidance in Using Electronic Health Records for Comparative Effectiveness Research in COVID-19: Opportunities and Trade-Offs (2022).
    Sarri G, Bennett D, Debray T, Deruaz-Luyet A, Soriano Gabarró M, Largent JA, Li X, Liu W, Lund JL, Moga DC, Gokhale M, Rentsch CT, Wen X, Yanover C, Ye Y, Yun H, Zullo AR, Lin KJ.
  • Epidemiology and treatment of patients with Chronic rhinosinusitis with nasal polyps in Germany-A claims data study (2022).
    Starry A, Hardtstock F, Wilke T, Weihing J, Ultsch B, Wernitz M, Renninger M, Maywald U, Pfaar O.
  • Defining Incidence of Acute Compartment Syndrome in the Research Setting: A Proposed Method From the PACS Study pp. S26-S32.
    Leroux A, Frey KP, Crainiceanu CM, Obremskey WT, Stinner DJ, Bosse MJ, Karunakar MA, O'Toole RV, Carroll EA, Hak DJ, Hayda R, Alkhoury D, Schmidt AH; METRC.
  • Bridging the gap between oncology clinical trials and real-world data: evidence on replicability of efficacy results using German claims data (2022).
    Ghiani M, Maywald U, Wilke T, Heeg B.
  • Durable remission for four pediatric patients with high-risk relapsed classical Hodgkin lymphoma treated with brentuximab vedotin plus gemcitabine but without autologous stem cell transplantation: A report from the Children's Oncology Group (2022).
    Buhtoiarov IN, Mba NI, Santos CDL, McCarten KM, Metzger ML, Pei Q, Bush R, Baker K, Kelly KM, Cole PD.
  • Burden of Pertussis in Individuals with a Diagnosis of Asthma: A Retrospective Database Study in England (2022) pp. 35-51.
    Bhavsar A, Aris E, Harrington L, Simeone JC, Ramond A, Lambrelli D, Papi A, Boulet LP, Meszaros K, Jamet N, Sergerie Y, Mukherjee P.
  • Evaluating the Economic Burden of Relapses in Neuromyelitis Optica Spectrum Disorder: A Real-World Analysis Using German Claims Data (2022).
    Knapp RK, Hardtstock F, Wilke T, Maywald U, Deiters B, Schneider S, Mouchet J.
  • Why are not There More Bayesian Clinical Trials? Perceived Barriers and Educational Preferences Among Medical Researchers Involved in Drug Development (2022).
    Medical Outreach Subteam of the Drug Information Association Bayesian Scientific Working Group, Clark J, Muhlemann N, Natanegara F, Hartley A, Wenkert D, Wang F, Harrell FE Jr, Bray R.
  • Effect of early treatment with metformin on risk of emergency care and hospitalization among patients with COVID-19: The TOGETHER randomized platform clinical trial (2022).
    Reis G, Dos Santos Moreira Silva EA, Medeiros Silva DC, Thabane L, Cruz Milagres A, Ferreira TS, Quirino Dos Santos CV, de Figueiredo Neto AD, Diniz Callegari E, Monteiro Savassi LC, Campos Simplicio MI, Barra Ribeiro L, Oliveira R, Harari O, Bailey H, Forrest JI, Glushchenko A, Sprague S, McKay P, Rayner CR, Ruton H, Guyatt GH, Mills EJ.
  • Developing a Prediction Model for Pathologic Complete Response Following Neoadjuvant Chemotherapy in Breast Cancer: A Comparison of Model Building Approaches (2022).
    Basmadjian RB, Kong S, Boyne DJ, Jarada TN, Xu Y, Cheung WY, Lupichuk S, Quan ML, Brenner DR.
  • Comparison of Mortality Risk With Different Surgeon and Hospital Operative Volumes Among Individuals Undergoing Pancreatectomy by Emulating Target Trials in US Medicare Beneficiaries (2022).
    Madenci AL, Wanis KN, Cooper Z, Subramanian SV, Haneuse S, Hofman A, Hernán M.
  • Assessment of the Cow's Milk-related Symptom Score (CoMiSS) as a diagnostic tool for cow's milk protein allergy: a prospective, multicentre study in China (MOSAIC study) (2022).
    Vandenplas Y, Zhao ZY, Mukherjee R, Dupont C, Eigenmann P, Kuitunen M, Ribes Koninckx C, Szajewska H, von Berg A, Bajerová K, Meyer R, Salvatore S, Shamir R, Järvi A, Heine RG; MOSAIC Study Investigator Group.
  • Assessment of Clinical Outcomes Among Children and Adolescents Hospitalized With COVID-19 in 6 Sub-Saharan African Countries (2022).
    Nachega JB, Sam-Agudu NA, Machekano RN, Rabie H, van der Zalm MM, Redfern A, Dramowski A, O'Connell N, Pipo MT, Tshilanda MB, Byamungu LN, Masekela R, Jeena PM, Pillay A, Gachuno OW, Kinuthia J, Ishoso DK, Amoako E, Agyare E, Agbeno EK, Martyn-Dickens C, Sylverken J, Enimil A, Jibril AM, Abdullahi AM, Amadi O, Umar UM, Sigwadhi LN, Hermans MP, Otokoye JO, Mbala-Kingebeni P, Muyembe-Tamfum JJ, Zumla A, Sewankambo NK, Aanyu HT, Musoke P, Suleman F, Adejumo P, Noormahomed EV, Deckelbaum RJ, Fowler MG, Tshilolo L, Smith G, Mills EJ, Umar LW, Siedner MJ, Kruger M, Rosenthal PJ, Mellors JW, Mofenson LM; African Forum for Research and Education in Health (AFREhealth) COVID-19 Research Collaboration on Children and Adolescents.
  • Efficacy and Safety of TRC105 Plus Pazopanib vs Pazopanib Alone for Treatment of Patients With Advanced Angiosarcoma: A Randomized Clinical Trial (2022).
    Jones RL, Ravi V, Brohl AS, Chawla S, Ganjoo KN, Italiano A, Attia S, Burgess MA, Thornton K, Cranmer LD, Cheang MCU, Liu L, Robertson L, Adams B, Theuer C, Maki RG.
  • Twins in Guinea-Bissau have a 'thin-fat' body composition compared to singletons (2022) pp. 1-7.
    Wagh R, Bjerregaard-Andersen M, Bandyopadhyay S, Yajnik P, Prasad RB, Otiv S, Byberg S, Hennild DE, Gomes GM, Christensen K, Sodemann M, Jensen DM, Yajnik CS.
  • Sustained and improved guselkumab response in patients with active psoriatic arthritis regardless of baseline demographic and disease characteristics: pooled results through week 52 of two phase III, randomised, placebo-controlled studies (2022).
  • Ritchlin CT, Mease PJ, Boehncke WH, Tesser J, Schiopu E, Chakravarty SD, Kollmeier AP, Xu XL, Shawi M, Jiang Y, Sheng S, Wang Y, Xu S, Merola JF, McInnes IB, Deodhar A.
  • Effectiveness of a third dose of the BNT162b2 mRNA COVID-19 vaccine for preventing severe outcomes in Israel: an observational study (2021) pp. 2093-2100.
    Barda N, Dagan N, Cohen C, Hernán MA, Lipsitch M, Kohane IS, Reis BY, Balicer RD.
  • Correlates of protection against symptomatic and asymptomatic SARS-CoV-2 infection (2021) pp. 2032-2040.
    Feng S, Phillips DJ, White T, Sayal H, Aley PK, Bibi S, Dold C, Fuskova M, Gilbert SC, Hirsch I, Humphries HE, Jepson B, Kelly EJ, Plested E, Shoemaker K, Thomas KM, Vekemans J, Villafana TL, Lambe T, Pollard AJ, Voysey M; Oxford COVID Vaccine Trial Group.
  • Global incidence and prevalence of idiopathic pulmonary fibrosis (2021) pp. 197.
    Maher TM, Bendstrup E, Dron L, Langley J, Smith G, Khalid JM, Patel H, Kreuter M.
  • Integrated Safety and Efficacy Among Patients Receiving Benralizumab for Up to 5 Years (2021) pp. 4381-4392
    Korn S, Bourdin A, Chupp G, Cosio BG, Arbetter D, Shah M, Gil EG.
  • Randomised trials at the level of the individual (2021) pp. e691-e700.
    Park JJH, Ford N, Xavier D, Ashorn P, Grais RF, Bhutta ZA, Goossens H, Thorlund K, Socias ME, Mills EJ.
  • How COVID-19 has fundamentally changed clinical research in global health (2021) pp. e711-e720.
    Park JJH, Mogg R, Smith GE, Nakimuli-Mpungu E, Jehan F, Rayner CR, Condo J, Decloedt EH, Nachega JB, Reis G, Mills EJ.
  • A comparison of confidence distribution approaches for rare event meta-analysis (2021) pp. 5276-5297.
    Zabriskie BN, Corcoran C, Senchaudhuri P.
  • The role and challenges of cluster randomised trials for global health (2021) pp. e701-e710.
    Dron L, Taljaard M, Cheung YB, Grais R, Ford N, Thorlund K, Jahan F, Nakimuli-Mpungu E, Xavier D, Bhutta ZA, Park JJH, Mills EJ.
  • A permutation-based approach for heterogeneous meta-analyses of rare events (2021) pp. 5587-5604.
    Zabriskie BN, Corcoran C, Senchaudhuri P.
  • The Ci3+3 design for dual-agent combination dose-finding clinical trials (2021) pp. 1-20.
    Yuan S, Zhou T, Lin Y, Ji Y.
  • Urgently seeking efficiency and sustainability of clinical trials in global health (2021) pp. e681-e690.
    Park JJH, Grais RF, Taljaard M, Nakimuli-Mpungu E, Jehan F, Nachega JB, Ford N, Xavier D, Kengne AP, Ashorn P, Socias ME, Bhutta ZA, Mills EJ.
  • Changes in Eating Behaviour during SARS-CoV-2 Pandemic among the Inhabitants of Five European Countries (2021) pp. 1624. 
    Górska P, Górna I, Miechowicz I, Przysławski J.
  • Practical Impact of Modern Dose-Finding Designs When Compared With the 3 + 3 Design: An Editorial (2021) pp. 1584-1587.
    Ji Y, Yuan S.
  • Gaining Efficiency in Clinical Trials With Cardiac Biomarkers: JACC Review Topic of the Week (2021) pp. 1922-1933.
    Januzzi JL Jr, Canty JM, Das S, DeFilippi CR, Gintant GA, Gutstein DE, Jaffe A, Kaushik EP, Leptak C, Mehta C, Pina I, Povsic TJ, Rambaran C, Rhyne RF, Salas M, Shi VC, Udell JA, Unger EF, Zabka TS, Seltzer JH.
  • Keeping it real: implications of real-world treatment outcomes for first-line immunotherapy in metastatic non-small-cell lung cancer (2021) pp. 1453-1456.
    Hsu GG, MacKay E, Scheuer N, Ramagopalan SV.
  • Open Science to Address COVID-19: Sharing Data to Make Our Research Investment Go Further (2021) pp. 558-560.
    Zariffa N, Haggstrom J, Rockhold F.
  • REDUCE-IT INTERIM: accumulation of data across prespecified interim analyses to final results (2021) pp. e61-e63.
    Olshansky B, Bhatt DL, Miller M, Steg PG, Brinton EA, Jacobson TA, Ketchum SB, Doyle RT Jr, Juliano RA, Jiao L, Granowitz C, Tardif JC, Mehta C, Mukherjee R, Ballantyne CM, Chung MK; REDUCE-IT Investigators.
  • A Review of Current Approaches to Defining and Valuing Innovation in Health Technology Assessment (2021) pp. 1773-1783.
    Hofmann S, Branner J, Misra A, Lintener H.
  • Using supervised machine learning approach to predict treatment outcomes of vedolizumab in ulcerative colitis patients (2021) pp. 1-16.
    Chen J, Girard M, Wang S, Kisfalvi K, Lirio R.
  • Growth in Infants with Cow's Milk Protein Allergy Fed an Amino Acid-Based Formula (2021) pp. 392-402.
    Vandenplas Y, Dupont C, Eigenmann P, Heine RG, Høst A, Järvi A, Kuitunen M, Mukherjee R, Ribes-Koninckx C, Szajewska H, von Berg A, Zhao ZY; Mosaic Study Investigator Group.
  • Child mortality associated with maternal HIV status: a retrospective analysis in Rwanda, 2005-2015 (2021).
    Remera E, Chammartin F, Nsanzimana S, Forrest JI, Smith GE, Mugwaneza P, Malamba SS, Semakula M, Condo JU, Ford N, Riedel DJ, Nisingizwe MP, Binagwaho A, Mills EJ, Bucher H.
  • A simulation-based comparison of estimation methods for adaptive and classical group sequential clinical trials (2021).
    Nelson BS, Liu L, Mehta C.
  • Evobrutinib, a covalent Bruton's tyrosine kinase inhibitor: Mass balance, elimination route, and metabolism in healthy participants (2021) pp. 2420-2430.
    Scheible H, Dyroff M, Seithel-Keuth A, Harrison-Moench E, Mammasse N, Port A, Bachmann A, Dong J, van Lier JJ, Tracewell W, Mitchell D.
  • Comparative Effectiveness of Atezolizumab, Nivolumab, and Docetaxel in Patients With Previously Treated Non-Small Cell Lung Cancer (2021).
    Ramagopalan S, Gupta A, Arora P, Thorlund K, Ray J, Subbiah V.
  • What Does the Economic Burden of Acute Myeloid Leukemia Treatment Look Like for the Next Decade? An Analysis of Key Findings, Challenges and Recommendations (2021) pp. 245-255.
    Forsythe A, Sandman K.
  • Patterns of undertreatment among patients with acute myeloid leukemia (AML): considerations for patients eligible for non-intensive chemotherapy (NIC) (2021) pp. 3359-3368.
    Hubscher E, Sikirica S, Bell T, Brown A, Welch V, Russell-Smith A, D'Amico P.
  • Red cell distribution width in patients with diabetes and myocardial infarction: An analysis from the EXAMINE trial (2021) pp. 1580-1587.
    Ferreira JP, Lamiral Z, Bakris G, Mehta C, White WB, Zannad F.
  • Pre-conceptional Maternal Vitamin B12 Supplementation Improves Offspring Neurodevelopment at 2 Years of Age: PRIYA Trial (2021).
    D'souza N, Behere RV, Patni B, Deshpande M, Bhat D, Bhalerao A, Sonawane S, Shah R, Ladkat R, Yajnik P, Bandyopadhyay SK, Kumaran K, Fall C, Yajnik CS.
  • Comparing Effect Estimates in Randomized Trials and Observational Studies From the Same Population: An Application to Percutaneous Coronary Intervention (2021).
    Matthews AA, Szummer K, Dahabreh IJ, Lindahl B, Erlinge D, Feychting M, Jernberg T, Berglund A, Hernán MA.
  • Body weight changes in patients with type 2 diabetes and a recent acute coronary syndrome: an analysis from the EXAMINE trial (2021) pp. 187.
    Ferreira JP, Rossignol P, Bakris G, Mehta C, White WB, Zannad F.
  • Durability of no evidence of disease activity-3 (NEDA-3) in patients receiving cladribine tablets: The CLARITY extension study (2021).
    Giovannoni G, Singer BA, Issard D, Jack D, Vermersch P.
  • Predictive accuracy of prenatal ultrasound findings for lower urinary tract obstruction: A systematic review and Bayesian meta-analysis (2021) pp. 1039-1048.
    Keefe DT, Kim JK, Mackay E, Chua M, Van Mieghem T, Yadav P, Lolas M, Santos JD, Skreta M, Erdman L, Weaver J, Fermin AS, Tasian G, Lorenzo AJ, Rickard M.
  • Phase 1 study in healthy participants of the safety, pharmacokinetics, and pharmacodynamics of enpatoran (M5049), a dual antagonist of toll-like receptors 7 and 8 (2021).
    Port A, Shaw JV, Klopp-Schulze L, Bytyqi A, Vetter C, Hussey E, Mammasse N, Ona V, Bachmann A, Strugala D, Reh C, Goteti K.
  • Maternal vitamin B(12), folate during pregnancy and neurocognitive outcomes in young adults of the Pune Maternal Nutrition Study (PMNS) prospective birth cohort: study protocol (2021).
    Behere RV, Deshpande G, Bandyopadhyay SK, Yajnik C.
  • Multi-proteomic approach to predict specific cardiovascular events in patients with diabetes and myocardial infarction: findings from the EXAMINE trial (2021) pp. 1006-1019.
    Ferreira JP, Sharma A, Mehta C, Bakris G, Rossignol P, White WB, Zannad F.
  • Assessment of Alectinib vs Ceritinib in ALK-Positive Non-Small Cell Lung Cancer in Phase 2 Trials and in Real-world Data (2021).
    Wilkinson S, Gupta A, Scheuer N, Mackay E, Arora P, Thorlund K, Wasiak R, Ray J, Ramagopalan S, Subbiah V.
  • Influence of sex, age and race on coronary and heart failure events in patients with diabetes and post-acute coronary syndrome (2021) pp. 1612-1624.
    Rossello X, Ferreira JP, Caimari F, Lamiral Z, Sharma A, Mehta C, Bakris G, Cannon CP, White WB, Zannad F.
  • A Chatbot to Engage Parents of Preterm and Term Infants on Parental Stress, Parental Sleep, and Infant Feeding: Usability and Feasibility Study (2021).
    Wong J, Foussat AC, Ting S, Acerbi E, van Elburg RM, Mei Chien C.
  • Long-term Safety and Efficacy of the Anti-MAdCAM-1 Monoclonal Antibody Ontamalimab [SHP647] for the Treatment of Ulcerative Colitis: The Open-label Study TURANDOT II (2021) pp. 938-949.
    Reinisch W, Sandborn WJ, Danese S, Hébuterne X, Kłopocka M, Tarabar D, Vaňásek T, Greguš M, Hellstern PA, Kim JS, Sparrow MP, Gorelick KJ, Hoy M, Goetsch M, Bliss C, Gupta C, Cataldi F, Vermeire S.
  • Safety and Efficacy of GFB-887, a TRPC5 Channel Inhibitor, in Patients With Focal Segmental Glomerulosclerosis, Treatment-Resistant Minimal Change Disease, or Diabetic Nephropathy: TRACTION-2 Trial Design (2021) pp. 2575-2584.
    Walsh L, Reilly JF, Cornwall C, Gaich GA, Gipson DS, Heerspink HJL, Johnson L, Trachtman H, Tuttle KR, Farag YMK, Padmanabhan K, Pan-Zhou XR, Woodworth JR, Czerwiec FS.
  • The Critical Need for Pooled Data on Coronavirus Disease 2019 in African Children: An AFREhealth Call for Action Through Multicountry Research Collaboration (2021) pp. 1913-1919.
    Sam-Agudu NA, Rabie H, Pipo MT, Byamungu LN, Masekela R, van der Zalm MM, Redfern A, Dramowski A, Mukalay A, Gachuno OW, Mongweli N, Kinuthia J, Ishoso DK, Amoako E, Agyare E, Agbeno EK, Mohammed Jibril A, Abdullahi AM, Amadi O, Mohammed Umar U, Ayele BT, Machekano RN, Nyasulu PS, Hermans MP, Otshudiema JO, Bongo-Pasi Nswe C, Kayembe JN, Mbala-Kingebeni P, Muyembe-Tamfum JJ, Aanyu HT, Musoke P, Fowler MG, Sewankambo N, Suleman F, Adejumo P, Tsegaye A, Mteta A, Noormahomed EV, Deckelbaum RJ, Zumla A, Mavungu Landu DJ, Tshilolo L, Zigabe S, Goga A, Mills EJ, Umar LW, Kruger M, Mofenson LM, Nachega JB; Investigators in the AFREhealth COVID-19 Research Collaboration on Children and Adolescents.
  • Zariffa, N., Haggstrom, J. & Rockhold, F. Open Science to Address COVID-19: Sharing Data to Make Our Research Investment Go Further. Ther Innov Regul Sci (2020). https://doi.org/10.1007/s43441-020-00250-z
  • Harari O, Hsu G, Dron L, Park JJH, Mills EJ, Thorlund K. Utilizing Bayesian Predictive Power in Clinical Trial Design. Pharmaceutical Statistics. 2020; 1– 16. https://doi.org/10.1002/pst.2073
  • Forrest JI, Rayner CR, Park JJH, Mills EJ. Early Treatment of COVID-19 Disease: A Missed Opportunity. Infectious Diseases and Therapy. 2020; DOI: 10.1007/s40121-020-00349-8
  • Dillman A, Park JJH, Zannat NE, Lee Z, Dron L, Hsu G, Smith G, Khakabimamaghani S, Harari O, Thorlund K, Mills EJ. Reporting and Design of Randomized Clinical Trials for COVID-19: A Systematic Review. Contemporary Clinical Trials. 2021;101:106239.
  • Dillman A, Zorratti M, Park JJH, Hsu G, Dron L, Smith G, Harari O, Rayner CR, Zannat NE, Gupta A, Mackay E, Arora P, Lee Z, Mills EJ. The landscape of emerging randomized clinical trial evidence for COVID-19 disease stages: A systematic review of global trial registries. Infection and Drug Resistance. 2020. Accepted
  • Accelerating Clinical Evaluation of Repurposed Combination Therapies for COVID-19 Craig R. Rayner, Louis Dron, Jay J. H. Park, Eric H. Decloedt, Mark F. Cotton, Vis Niranjan, Patrick F. Smith, Michael G. Dodds, Fran Brown, Gilmar Reis, David Wesche, Edward J. Mills
  • Lancet Global Health 4 series on “Series in Clinical Trials in Global Health” – Forthcoming: 
Park JJH, Grais R, Taljard M, Nakimuli-Mpungu E, Jehan F, Nachega J, Ford N, Xavier D, Kengne A, Ashorn P, Socias ME, Bhutta ZA, Mills EJ. Global health clinical trials: Urgently seeking efficiency and sustainability. Lancet Global Health, 2020. Accepted
 
Park JJH, Ford N, Xavier D, Ashorn P, Grais R, Bhutta ZA, Goossens H, Thorlund T, Socias ME, Mills EJ. Global health clinical trials: Randomised trials at the level of individual. Lancet Global Health, 2020. Accepted

 

Dron L, Taljard M, Cheung YB, Grais R, Ford N, Thorlund K, Jahan F, Nakimuli-Mpungu E, Xavier D, Bhutta ZA, Park JJH, Mills EJ. Global health clinical trials: Role and challenges of cluster randomized trials for global health. Lancet Global Health, 2020. Accepted
 
Park JJH, Mogg R, Smith GE, Nakimuli-Mpungu E, Jehan F, Rayner CR, Condo J, Decloedt EH, Nachega J, Reis G, Mills EJ. How COVID-19 fundamentally changed clinical research in global health. Lancet Global Health, 2020. Accepted

  • Chen, Xianwen MD; Ren, Cuiping MD; Li, Juan MD; Wang, Shangpei MD; Dron, Louis MSc; Harari, Ofir PhD; Whittington, Craig PhD The Efficacy and Safety of Piribedil Relative to Pramipexole for the Treatment of Early Parkinson Disease: A Systematic Literature Review and Network Meta-Analysis, Clinical Neuropharmacology: 7/8 2020 - Volume 43 - Issue 4 - p 100-106 doi: 10.1097/WNF.0000000000000400
  • Ghosh P, Liu L, Mehta CR. Adaptive Multiarm Multistage Clinical Trials. Statistics in Medicine, 2020; 39:1084-1102.
  • Ferreira JP, Mehta CR, Sharma A, Nissen SE, Rossignol P, Zannad F. Alogliptin after acute coronary syndrome in patients with type 2 diabetes: a renal function stratified analysis of the EXAMINE trial. BMC Medicine, 2020; 18:165.
  • Elharram M, Sharma A, White W, Bakris G, Rossignol P, Mehta CR, Ferreira JP, Zannad, F. Timing of Randomization after an Acute Coronary Syndrome in Patients with Type-2 Diabetes Mellitus. American Heart Journal, 2020 (in Press).
  • Ferreira JP, Rossignol P, Sharma, A, White WB, Mehta CR, Bakris G, Zannad, F. Multi-proteomic approach to predict specific cardiovascular events in patients with diabetes and myocardial infarction: findings from the EXAMINE trial. Clinical Research in Cardiology, 2020. Published on-line, DOI 10.1007/s00392-020-01729-3.
  • Ankolekar S., Mehta C., Mukherjee R., Hsiao S., Smith J., Haddad T. (2020) Monte Carlo Simulation for Trial Design Tool. In: Piantadosi S., Meinert C. (eds) Principles and Practice of Clinical Trials. Springer, Cham. https://doi.org/10.1007/978-3-319-52677-5_251-1

 

  • Park, Jay JH, et al. "Association of early interventions with birth outcomes and child linear growth in low-income and middle-Income countries: Bayesian network meta-analyses of randomized clinical trials." JAMA network open 2.7 (2019): e197871-e197871.
  • Mehta, C. R., L. Liu, and C. Theuer. "An adaptive population enrichment phase III trial of TRC105 and pazopanib versus pazopanib alone in patients with advanced angiosarcoma (TAPPAS trial)." Annals of Oncology 30.1 (2019): 103-108.
  • Hsiao, Samuel T., Lingyun Liu, and Cyrus R. Mehta. "Optimal promising zone designs." Biometrical Journal 61.5 (2019): 1175-1186.
  • Mehta, C. R., et al. “Exact Inference for Adaptive Group Sequential design,” in Liu, Ray, and Yi Tsong, eds. Pharmaceutical Statistics: MBSW 39, Muncie, Indiana, USA, May 16-18, 2016. Vol. 218. Springer, 2019.
  • Park JJH, Siden E, Zoratti MJ, et al. Systematic review of basket trials, umbrella trials, and platform trials: a landscape analysis of master protocols. BMC Trials 2019; 20(1): 572.
  • Siden EG, Park JJ, Zoratti MJ, et al. Reporting of master protocols towards a standardized approach: A systematic review. Contemporary Clinical Trials Communications 2019; 15: 100406.
  • Park JJ, Harari O, Dron L, Mills EJ, Thorlund K. Effects of biomarker diagnostic accuracy on biomarker-guided phase 2 trials. Contemporary Clinical Trials Communications 2019; 15: 100396.
  • Mills EJ, Nsanzimana S. Have clinical trials in HIV finally matured? Lancet HIV 2019; 6 (9): 561-563.
  • Zhang K, Arora P, Sati N, Béliveau A, Troke N, Veroniki AA, Rodrigues M, Rios P, Zarin W, Tricco AC. Characteristics and methods of incorporating randomized and nonrandomized evidence in network meta-analyses: a scoping review. Journal of Clinical Epidemiology 2019; 113:1-10.
  • Welch VA, Hossain A, Ghogomu E, Riddle A, Cousens S, Gaffey M, Arora P, Black R, Bundy D, Castro MC, Chen L. Deworming children for soil-transmitted helminths in low and middle-income countries: systematic review and individual participant data network meta-analysis. Journal of Development Effectiveness. 2019 Dec 7:1-9.
  • Dron L, Golchi S, Hsu G, Thorlund K. Minimizing control group allocation in randomized trials using dynamic borrowing of external control data - An application to second line therapy for non-small cell lung cancer. Contemporary Clinical Trials Communications 2019; 16: 100446.
  • Gupta, A., Slater, J. J., Boyne, D., Mitsakakis, N., Béliveau, A., Druzdzel, M. J., … Arora, P. (2019). Probabilistic Graphical Modeling for Estimating Risk of Coronary Artery Disease: Applications of a Flexible Machine-Learning Method. Medical Decision Making 2019; 39(8):1032-1044. 
  • Arora P, Boyne D, Slater JJ, Gupta A, Brenner DR, Druzdzel MJ. Bayesian Networks for Risk Prediction Using Real-World Data: A Tool for Precision Medicine. Value Health 2019; 22(4):439-445.
  • Jafar R, Labrosse M, Weaver JD, Retta SM, Wu C, Duraiswamy N. A Computational Study on Deformed Bioprosthetic Valve Geometries: Clinically Relevant Valve Performance Metrics. J Biomech Eng 2019.
  • Khakabimamaghani S, Kelkar YD, Grande BM, Morin RD, Ester M, Ziemek D. SUBSTRA: Supervised Bayesian Patient Stratification. Bioinformatics 2019; 35(18):3263-3272.
  • Gupta A, Arora P, Brenner D, Edmonson-Jones M, Parkhomenko E, Stevens W, Wagner S, Borrill J, Wu E. Application of a Machine Learning Model to Predict Survival Outcomes for Patients with Advanced Renal Cell Carcinoma (aRCC) Treated with Nivolumab. In41st Annual Meeting of the Society for Medical Decision Making 2019 Oct 23. 
  • Zhang K, Arora P, Sati N, Béliveau A, Troke N, Veroniki AA, Rodrigues M, Rios P, Zarin W, Tricco AC. Characteristics and methods of incorporating randomized and nonrandomized evidence in network meta-analyses: a scoping review. Journal of Clinical Epidemiology 2019; 113:1-10.
  • Arora P, Thorlund K, Brenner DR, Andrews JR. Comparative accuracy of typhoid diagnostic tools: A Bayesian latent-class network analysis. PLoS Neglected Tropical Diseases 2019; 8;13(5):e0007303.
  • Park JJH, Fang ML, Harari O, et al. Association of Early Interventions With Birth Outcomes and Child Linear Growth in Low-Income and Middle-Income Countries: Bayesian Network Meta-analyses of Randomized Clinical Trials. JAMA Netw Open 2019; 2(7): e197871.
  • Gupta A, Brenner DR, Arora P. PMU12 Scalable Decision-Analytic Modelling with Influence Diagrams. Value in Health. 2019 May 1;22:S251.
 
  • An adaptive population enrichment phase III trial of TRC105 and pazopanib versus pazopanib alone in patients with advanced angiosarcoma (TAPPAS trial)
    Annals of Oncology
    C R Mehta, L Liu, C Theuer
  • ADaM Intermediate Dataset: how to improve your analysis traceability
    PhUSE US Connect 2018, Paper SD16
    Tinazzi, A. Curto, T. and Aggarwal, A.
  • Confidence, credibility and prediction.
    METRON, 76(2), pp.251-268
    Aitkin, M. and Liu, C.
  • Liu L, Hsiao S, Mehta CR. Efficiency Considerations for Group Sequential Designs with Adaptive Unblinded Sample Size Re-assessment. Statistics in Biosciences, 2018; 10(2):405-419.
  • Hwang Y-C, Morrow DA, ..., Mehta CR, ..., White WB. High-sensitivity C-reactive protein, low-density lipoprotein cholesterol and cardiovascular outcomes in patients with type 2 diabetes in the EXAMNE trial. Diabetes, Obesity and Metabolism, 2018; 20(3):654-659.
  • Cavender M, White WB, ..., Cannon CP. Total Cardiovascular Events Analysis of the EXAMINE Trial in Patients with Type 2 Diabetes and Recent Acute Coronary Syndrome. Clinical Cardiology, 2018; vol 41, pages 1022-1027.
  • Green SJ, Mentz RJ, ..., Mehta CR, ...O'Connor CM. Reassessing the Role of Surrogate Endpoints in Drug Development for Heart Failure. Circulation, 2018; 138(10):1039-1053.
  • White WB, Jalil F, ..., Mehta CR, Zannad F. Average Clinician-Measured Blood Pressures and Cardiovascular Outcomes in Patients with Type 2 Diabetes and Ischemic Heart Disease in the EXAMINE Trial. Journal of the American Heart Association, 2018; 7(20), e009114.
  • Menon V, Nichols SJ, ..., Mehta CR, ... Nissen SE. Fasiglifam-induced liver injury in patients with type-2 diabetes: results of a randomized controlled cardiovascular oucomes safety trial. Diabetes Care, 2018; 41:2608-2609.
  • Multiplicity: discussion points from the Statisticians in the Pharmaceutical Industry multiplicity expert group.
    Pharmaceut. Statist., 2013, 12 255–259
    Phillips, A., Fletcher, C., Atkinson, G., Channon, E., Douiri, A., Jaki, T., Maca, J., Morgan, D., Roger, JH. and Terrill, P.
  • PEARL I Study Group. Ulipristal acetate versus placebo for fibroid treatment before surgery.
    N Engl J Med., 2012; 366(5):409-20.
    Donnez J, Tatarchuk TF, Bouchard P, Puscasiu L, Zakharenko NF, Ivanova T, Ugocsai G, Mara M, Jilla MP, Bestel E, Terrill, P., Osterloh I and Loumaye E.
  • PEARL II Study Group. Ulipristal acetate versus leuprolide acetate for uterine fibroids.
    N Engl J Med., 2012; 366(5):421-32.
    Donnez J, Tomaszewski J, Vázquez F, Bouchard P, Lemieszczuk B, Baró F, Nouri K, Selvaggi L, Sodowski K, Bestel E, Terrill, P., Osterloh I and Loumaye E.

COST-EFFECTIVENESS ANALYSES

COST-CONSEQUENCE MODELS

BUDGET IMPACT MODELS

4 ITC/NMA/STC/MAIC

QOL/UTILITY

RWE

SYSTEMATIC LITERATURE REVIEW AND BURDEN OF ILLNESS

EARLY ECONOMIC MODELS AND VALUE FRAMEWORKS

HEALTH ECONOMICS METHODS

ENDPOINT VALIDATION

HEALTH TECHNOLOGY ASSESSMENTS