Publications
- Authors’ response to “Comment on
adaptive increase in sample size when interim results are promising”.
Statistics in Medicine, 30(28), pp. 3302–3303.
Mehta, C.R. and Pocock, S.J. - Adaptive increase in sample size when interim results are promising: A practical guide with examples.
Statistics in Medicine, 30(28), pp. 3267–3284.
Mehta, C.R. and Pocock, S.J. - Efficient Monte Carlo methods for conditional logistic regression.
Journal of the American Statistical Association, 95(449), p. 99.
Mehta, C.R., Patel, N.R. and Senchaudhuri, P. - Exact logistic regression: Theory and examples.
Statistics in Medicine, 14(19), pp. 2143–2160.
Mehta, C.R. and Patel, N.R. - Exact Permutational tests for group Sequential clinical trials.
Biometrics, 50(4), p. 1042.
Mehta, C.R., Patel, N.R., Senchaudhuri, P. and Tsiatis, A. - A network algorithm for performing Fisher’s exact test in r × c contingency tables.
Journal of the American Statistical Association, 78(382), p. 427.
Mehta, C.R. and Patel, N.R. - Repeated confidence intervals for adaptive group sequential trials.
Statistics in Medicine, 26, 5422-5433.
Mehta, C.R. Bauer, P., Posch, M. and Brannath, W. - Optimizing trial design; sequential, adaptive and enrichment strategies.
Statistical Primer for Cardiovascular Research, 119:597-605.
Mehta, C.R. Gao, P, Bhatt, DL, Harrington, RA, Skerjanec, S and Ware JH. - Exact inference for adaptive group sequential designs.
Statistics in Medicine, volume 32, 3991-4005.
Gao, P., Liu, L., and Mehta, C.R. Biomarker population enrichment for adaptive oncology trials with time to event endpoints .driven
Statistics in Medicine, 33, 4515-4531.
Mehta, C.R., Schäfer, H., Daniel, H. and Irle, S.
Epidemiology and treatment of myasthenia gravis: a retrospective study using a large insurance claims dataset in Germany
Neuromuscular Disorders Journal, Volume 33, Issue 4, p.324-333,
Mevius, A., Jöres, L., Biskup, J., Heidbrede, T., Mahic, M., Wilke, T., Maywald,U., Lehnerer, S., Meisel, A.
- 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:
- 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.
- Mehta CR. Commentary on Friedlin and Korn. Clinical Trials, 2017; vol 14(6), 605-608.
- Marchenko O, Jiang Qi, Chuang-Stein C, Mehta CR, ..., Soomin Park. Statistical Considerations for Cardiovascular Outcome Trials in Patients with Type 2 Diabetes Mellitus. Statistics in Biopharmaceutical Research, 2017; 9(4):347-360.
- Butler J, Hamo CE, ..., Mehta CR,..., Anker DA, on behalf of the EMPEROR Trials Program. The potential role and rationale for treatment of heart failure with sodium-glocose co-transporter 2 inhibitors. European Journal of Heart Failure, 2017; 19(11):1390-1400
- On
Shapes of ADR Report Accumulation Data for Banned Drugs.
Current Science, 113(09), p.1664.
Ghubade, S., Asvalayan, K., Paranjpe, S., Gupta, K. and Gore, A. - Efficacy and Safety of MED2005, a Topical Glyceryl Trinitrate Formulation, in the Treatment of Erectile Dysfunction: A Randomized Crossover Study.
Sex Med
Ralph, DJ., Eardley, I., Taubel, J., Terrill, P. and Holland T. - Serial Measurement of High-Sensitivity Troponin I and Cardiovascular Outcomes in Patients With Type 2 Diabetes Mellitus in the EXAMINE Trial (Examination of Cardiovascular Outcomes With Alogliptin Versus Standard of Care)Clinical Perspective.
Circulation, 135(20), pp.1911-1921.
Cavender, M., White, W., Jarolim, P., Bakris, G., Cushman, W., Kupfer, S., Gao, Q., Mehta, C.R., Zannad, F., Cannon, C. and Morrow, D. - Relationship of glycated
haemoglobin and reportedhypoglycaemia to cardiovascular outcomes in patients with type 2 diabetes and recent acute coronary syndrome events: The EXAMINE trial.
Diabetes, Obesity and Metabolism, 19(5), pp.664-671.
Heller, S., Bergenstal, R., White, W., Kupfer, S., Bakris, G., Cushman, W., Mehta, C.R., Nissen, S., Wilson, C., Zannad, F., Liu, Y., Gourlie, N. and Cannon, C. - Topical safety and vasoconstrictive assay-based bioequivalence of a new reformulated mometasone cream.
Krishna, R., Horowitz, A., Larson, P., Bolognese, J. and Marcantonio, E.Mehta, C.R. - A gatekeeping procedure to test a primary and a secondary endpoint in a group sequential design with multiple interim looks.
Biometrics.
Tamhane, A., Gou, J., Jennison, C., Mehta, C.R. and Curto, T. - Efficiency Considerations for Group Sequential Designs with Adaptive Unblinded Sample Size Re-assessment.
Statistics in Biosciences.
Liu, L., Hsiao, S. and Mehta, C.R. - Optimizing adaptive design for Phase 2 dose-finding trials incorporating long-term success and financial considerations: A case study for neuropathic pain.
Contemporary Clinical Trials, 57, pp.69-86.
Gao, J., Nangia, N., Jia, J., Bolognese, J., Bhattacharyya, J. and Patel, N. - The Case for a Bayesian Approach to Benefit-Risk Assessment.
Therapeutic Innovation & Regulatory Science
Costa, M., He, W., Jemiai, Y., Zhao, Y. and Casoli, C. - Design and monitoring of multi-arm multi-stage clinical trials. Recognized as a top 20 downloaded paper by Wiley
Biometrics.doi :10.1111/biom.12687
Ghosh, P., Liu, L., Senchaudhuri, P., Gao, P. and Mehta, C.R.
- An objective re-evaluation of adaptive sample size re-estimation: Commentary on “Twenty-five years of confirmatory adaptive designs”.
Statistics in Medicine, 35(3), pp. 350–358.
Mehta, C.R. and Liu, L. - A randomized, phase 2 study investigating TRV130, a biased ligand of the μ-opioid receptor, for the intravenous treatment of acute pain.
Pain, 157, no. 1 264-272.
Viscusi, Eugene R., Bolognese, J., et al. - Cardiovascular mortality in patients with type 2 diabetes and recent acute coronary Syndromes from the EXAMINE trial.
Cardiovascular and Metabolic Risk, 39(7), pp. 1267–1273.
White, W.B., Zannad, F., Mehta, C.R., et al. - Angiotensin-converting enzyme inhibitor use and major cardiovascular outcomes in type 2 diabetes Mellitus treated with the Dipeptidyl Peptidase 4 inhibitor Alogliptin Novelty and significance.
Hypertension, 68(3), pp. 606–613.
White, W.B., Wilson, C.A., Mehta, C.R., Nissen, S.E., Zannad, F., et al. - Adaptive designs for clinical trials.
New England Journal of Medicine, 375(1), pp. 65–74.
Bhatt, D.L. and Mehta, C.R. - Addressing challenges and opportunities of “Less Well-Understood” Adaptive Designs.
Therapeutic Innovation & Regulatory Science.
He, W., Gallo, P., Miller, E., Jemiai, Y., et al. - Comments on: “Some Challenges with Statistical Inference in Adaptive Designs”, by Hung, Wang, and Yang.".
Journal of Biopharmaceutical Statistics, 26, no. 2 402-404.
Mehta, C.R. - DIA’s Adaptive Design Scientific Working Group (ADSWG) Best Practices Case Studies for “Less Well-understood” Adaptive Designs.
Therapeutic Innovation & Regulatory Science.
Miller, Eva, Gallo, Paul, Paul, He, Weili, Jemiai, Y., et al. - Methodological extensions of phase 2 trial designs based on program-level considerations: Further development of a case study in Neuropathic pain.
Therapeutic Innovation & Regulatory Science.
Bolognese, J., Bhattacharyya, J., Assaid, C.and Patel, N.R.
- Efficacy and safety of repeated use of ulipristal acetate in uterine fibroids.
Fertil Steril. 2015;103(2):519-527
Donnez, J., Hudecek, R., Donnez, O., Matule, D., Arhendt, HJ., Zatik, J., Kasilovskiene, Z., Dumitrascu, MC., Fernandez, H., Barlow, DH., Bouchard, P., Fauser, BCJM., Bestel, E., Terrill, P., Osterloh, I. and Loumaye, E. - Dynamically optimizing budget allocation for phase 3 drug development portfolios incorporating uncertainty in the pipeline.
Patel, N.R. and Ankolekar, S. - Drug development and the cost of capital.
Chapters from: Optimization of Pharmaceutical R&D Programs and Portfolios pp. 35-47. - Schulz, Kraig F., Sarah T. Bobulsky, Frank S. David, Patel, N.R., and Antonijevic, Z.Bhattacharyya, J.
- Vosaroxin plus cytarabine versus placebo plus cytarabine in patients with first relapsed or refractory acute myeloid
leukaemia (VALOR): Arandomised , controlled, double-blind, multinational, phase 3 study.
The Lancet Oncology, 16, no. 9 1025-1036.
Ravandi, Farhad, Ellen K. Ritchie, Hamid Sayar, Jeffrey E. Lancet, Michael D. Craig, Mehta, C.R., et al. - Heart failure and mortality outcomes in patients with type 2 diabetes taking alogliptin vs. placebo in EXAMINE: a multicentre,
randomised , double-blind trial.
The Lancet, 385, no. 9982 2067-2076.
Zannad, Faiez, Christopher P. Cannon, Mehta, C.R., et al. - A randomized, controlled trial of oral propranolol in infantile hemangioma.
New England Journal of Medicine, 372, no. 8 735-746.
Léauté-Labrèze, Christine, Peter Hoeger, Mehta, C.R., et al. - Impact of phase 2b strategies on optimization of drug development programs.
Optimization of Pharmaceutical R&D Programs and Portfolios, pp. 103–83.
Antonijevic, Z., Bolognese, J., Burman, C., Chuang-Stein, C., Jennison, C., Patel, N.R., et al. - An intra-articular, extended-release formulation of triamcinolone acetonide prolongs and amplifies analgesic effect in patients with osteoarthritis of the knee.
The Journal of Bone & Joint Surgery, 97, no. 11 877-888.
Bodick, Neil, Joelle Lufkin, Bolognese, J., Schoonmaker, C., et al. - Evaluation of an adaptive maximizing design study based on clinical utility versus morphine for TRV130 proof-of-concept and dose-regimen finding in patients with Postoperative pain after Bunionectomy.
Therapeutic Innovation & Regulatory Science, 49(5), pp. 756–766.
Bolognese, J., Subach, R.A., and Skobieranda, F. - Creating value with financially adaptive clinical trials.
Nature Reviews Drug Discovery, 14(8), pp. 523–524.
David, F.S., Bobulsky, S., Schulz, K. and Patel, N.R.
- Individualized vaginal bleeding experience of women with
uterin fibroids in the PEARL I randomized controlled trial comparing the effects of ulipristal acetate or placebo.
Hum Reprod, 2014; 29(3):480-9
Barlow, DH., Lumsden, MA., Fauser, BCJM., Terrill, P. and Bestel, E. - Adaptive Sequential testing for multiple comparisons.
Journal of Biopharmaceutical Statistics, 24(5), pp. 1035–1058.
Gao, P., Liu, L. and Mehta, C.R. - Clinical development approaches and statistical methodologies to prospectively assess the cardiovascular risk of new Antidiabetic therapies for type 2 diabetes.
Therapeutic Innovation & Regulatory Science, 49(1), pp. 50–64.
Geiger, M.J., Mehta, C.R., Gaydos, B, et al. - Research designs for proof-of-concept chronic pain clinical trials: IMMPACT recommendations.
Pain, 155(9), pp. 1683–1695.
Gewandter, J.S., Dworkin, R.H., Mehta, C.R., et al. Biomarker population enrichment for adaptive oncology trials with time to event endpoints.driven
Statistics in Medicine, 33(26), pp. 4515–4531.
Mehta, C.R., Schäfer, H., Daniel, H. and Irle, S.- Early clinical development planning via Biomarkers, clinical endpoints, and simulation: A case study to optimize for phase 3 dose selection.
Therapeutic Innovation & Regulatory Science, 49(3), pp. 405–414.
Musser, B., Bolognese, J., Fayad, G.N., Bhattacharyya, J., Patel, N.R., et al. - Approaches for clinical supply modeling and simulation.
Practical Considerations for Adaptive Trial Design and Implementation, pp. 273–297.
Patel, N.R., Ankolekar, S. and Senchaudhuri, P.
- 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
- Azimpour K, Tremblay G, de Chantal M, Marsolais C. "Cost-Effectiveness Of Tesamorelin In The Treatment Of Lipohypertrophy In The US." Value in Health (2020).
- Tremblay, G., U. Majethia, J. L. Breeze, I. Kontoudis, and J. Park. "Economic Evaluation of Eribulin as Second-Line Treatment for Metastatic Breast Cancer in South Korea." ClinicoEconomics and Outcomes Research : CEOR 8 (September 2016): 485–93. https://doi.org/10.2147/CEOR.S110553.
- Tremblay, G., B. Rousseau, M. Marquis, C. Beaubois, G. Sauvageau, and J. Hebert. "Cost-Effectiveness Analysis of a HMGA2 Prognostic Test for Acute Myeloid Leukemia in a Canadian Setting." Applied Health Economics and Health Policy 17, no. 6 (December 2019): 827–39. https://doi.org/10.1007/s40258-019-00503-5.
- Tremblay, G., M. Dolph, S. Patel, P. Brandt, and A. Forsythe. "Cost-Effectiveness Analysis for Midostaurin versus Standard of Care in Acute Myeloid Leukemia in the United Kingdom." Cost Effectiveness and Resource Allocation: C/E 16 (2018): 33. https://doi.org/10.1186/s12962-018-0153-4.
- Tremblay, G., M. Dolph, S. Patel, P. Brandt, and A. Forsythe. "Cost-Effectiveness Analysis of Midostaurin (MIDO) with Standard Chemotherapy (SOC) for Acute Myeloid Leukemia (AML) in the United Kingdom (UK)." Oral presentation presented at the International Society for Pharmacoeconomics and Outcomes Research (ISPOR), Glasgow, Scotland, November 4, 2017.
- Tremblay, G., C. Cariou, C. Recher, M. Dolph, P. Brandt, A.-S. Blanc, and A. Forsythe. "Cost-Effectiveness of Midostaurin in the Treatment of Newly Diagnosed FLT3-Mutated Acute Myeloid Leukemia in France." The European Journal of Health Economics, January 22, 2020. https://doi.org/10.1007/s10198-019-01149-9.
- Cariou, C., G. Tremblay, M. Dolph, P. S. Brandt, and A. Forsythe. "Cost-Effectiveness Model of Midostaurin (MIDO) Versus Standard of Care (SOC) In Patients With Newly Diagnosed FLT3 Mutation-Positive (FLT3+) Acute Myeloid Leukemia (AML): A French Perspective." Oral presentation presented at the International Society for Pharmacoeconomics and Outcomes Research (ISPOR), Barcelona, Spain, November 10, 2018.
- Tremblay, G., M. Dolph, K. El Ouagari, P. Brandt, and A. Forsythe. "Cost-Effectiveness Analysis of Midostaurin (MIDO) With Standard of Care (SOC) for Acute Myeloid Leukemia (AML) in Canada." Baltimore, MD, USA, 2018. https://doi.org/10.1016/j.jval.2018.04.180.
- Tremblay G, Dolph M, Nidumolu Roy A, Said Q, Forsythe A. "The Cost-Effectiveness of Eltrombopag for the Treatment of Immune Thrombocytopenia in the United States." Clinical Therapeutics, 2020. ISSN 0149-2918. https://doi.org/10.1016/j.clinthera.2020.02.020.
- Tremblay, G., M. Dolph, A. Roy, J. Neyra, K. El Ouagari, and A. Forsythe. "A Canadian Cost-Effectiveness Analysis for the Treatment of Immune Thrombocytopenia: Assessing the Relative Value of with Eltrombopag Versus Romiplostim." Atlanta, GA, USA, 2017. https://ashpublications.org/blood/article/130/Supplement 1/3410/114517/A-Canadian-Cost-Effectiveness-Analysis-for-the.
- Tremblay, G., A. Forsythe, V. Bal, S. Santra, and A. Briggs. "Cost-Effectiveness Analysis of Ofatumumab As a Treatment for Relapsed Chronic Lymphocytic Leukemia in the United States." San Diego, CA, 2016. https://doi.org/10.1182/blood.V128.22.2366.2366.
- Tremblay, G., M. Dolph, G. Sauvageau, and S. Cohen. "Cost-Effectiveness Model Comparing Expanded Umbilical Cord Blood Transplants With Other HSCT Strategies For Patients With Haematological Malignancies." New Orleans, LA, USA, 2019. https://doi.org/10.1016/j.jval.2019.04.173.
- Tremblay, G., T. Westley, M. Dolph, D. Newman, and G. Lewis. "Cost-Effectiveness Model Comparing Suba-Itraconazole (SUBA-ITRA) Versus Conventional Itraconazole (ITRA) For Treating Systemic Mycoses In The U.S." New Orleans, LA, USA, 2019. https://doi.org/10.1016/j.jval.2019.04.170.
- Tremblay, G., M. Dolph, T. Jones, A. Forsythe, L. Mellor, and M. Sampson. "Cost-Effectiveness Model Comparing Therapeutic Strategies For Treating Iron Deficiency Anaemia (IDA) In Inflammatory Bowel Disease (IBD)." Barcelona, Spain, 2018. https://doi.org/10.1016/j.jval.2018.09.2669.
- Forsythe, A., G. Tremblay, M. Dolph, R. Dhanda, J. Patterson, V. Nadipelli, N. Rashid, L. Citrome, and C. U. Correll. "Cost-Effectiveness Model Comparing the Novel Subcutaneous Antipsychotic RBP-7000 to Intramuscular Paliperidone Palmitate to Treat Patients with Acutely Exacerbated Schizophrenia." Orlando, FL, 2018. https://www.jmcp.org/doi/10.18553/jmcp.2018.24.10-a.s1.
- Tremblay, G., V. Chounta, J. Piercy, T. Holbrook, S. A. Garib, E. K. Bukin, and Y.S. Punekar. "Cost-Effectiveness of Dolutegravir as a First-Line Treatment Option in the HIV-1-Infected Treatment-Naive Patients in Russia." Value in Health Regional Issues 16 (September 2018): 74–80. https://doi.org/10.1016/j.vhri.2018.08.001.
- Punekar, Y.S., N. Guo, G. Tremblay, J. Piercy, T. Holbrook, and B. Young. "Improving Access to Antiretrovirals in China: Economic Analyses of Dolutegravir in HIV-1 Patients." Cost Effectiveness and Resource Allocation : C/E 17 (2019): 26. https://doi.org/10.1186/s12962-019-0195-2.
- Tremblay, G., D. Howard, W. Tsong, V. Patel, and J. De Rosendo. "Cost-Effectiveness of Perampanel for the Treatment of Primary Generalized Tonic-Clonic Seizures (PGTCS) in Epilepsy: A Spanish Perspective." Epilepsy & Behavio : E&B 86 (September 2018): 108–15. https://doi.org/10.1016/j.yebeh.2018.06.002.
COST-CONSEQUENCE MODELS
- Tremblay, G., M. Dolph, M. Bhor, Q. Said, A. Roy, B. Elliott, and A. Briggs. "Cost-Consequence Model Comparing Eltrombopag and Romiplostim in Pediatric Patients with Chronic Immune Thrombocytopenia." ClinicoEconomics and Outcomes Research : CEOR 10 (2018): 715–21. https://doi.org/10.2147/CEOR.S177338.
- Tremblay, G., M. Dolph, M. Bhor, Q. Said, B. Elliott, and A. Briggs. "Cost-Consequence Model Comparing Eltrombopag versus Romiplostim for Adult Patients with Chronic Immune Thrombocytopenia." ClinicoEconomics and Outcomes Research : CEOR 10 (2018): 705–13.
- Tremblay, G., Q. Said, B. Cai, S. A. Garib, D. Tomaras, A. Forsythe, and A. Roy. "Comparing Costs and Consequences of Eltrombopag Plus IST Versus IST Alone for First-Line Treatment in Severe Aplastic Anemia: Results from a Responder Analysis," 132:1041–1041. San Diego, CA, 2018. https://doi.org/10.1182/blood-2018-99-115138.
- Tremblay, G., M. Bhor, A. Roy, B. Elliott, M. Dolph, A. Forsythe, and A. Briggs. "Cost-Consequence Model Comparing Eltrombopag and Romiplostim for Pediatric Patients with Previously-Treated Chronic Immune Thrombocytopenia." Atlanta, GA, USA, 2017. https://ashpublications.org/blood/article/130/Supplement 1/2146/71530
BUDGET IMPACT MODELS
- Tremblay G, Azimpour K. "Clinical Validation Of A Budget Impact Model Using The Established Key Opinion Leader Validation Framework (KOLVF)." Value in Health (2020).
- Tremblay, G., Q. Said, A. N. Roy, B. Cai, S. A Garib, J. Hearnden, and A. Forsythe. "Budget Impact Of Eltrombopag As First-Line Treatment For Severe Aplastic Anemia In The United States." ClinicoEconomics and Outcomes Research: CEOR 11 (2019): 673–81. https://doi.org/10.2147/CEOR.S226323.
- Tremblay, G., T. Westley, M. Dolph, D. Newman, and G. Lewis. "Budget Impact of Suba-Itraconazole Capsules (SUBA-ITRA) For The Treatment Of Systemic Mycoses In The U.S." New Orleans, LA, USA, 2019. https://doi.org/10.1016/j.jval.2019.04.176.
- Forsythe, A., S. A. Garib, G. Tremblay, R. Dhanda, J. Patterson, V. Nadipelli, N. Rashid, L. Citrome, and C. U. Correll. "The Budget Impact of Novel Subcutaneous Once-Monthly Long-Acting Injectable Antipsychotic (LAI) RBP-7000 (Risperidone in the Atrigel® Delivery System) in Patients with Acute Schizophrenia." Orlando, FL, 2018. https://www.jmcp.org/doi/10.18553/jmcp.2018.24.10-a.s1.
- Tremblay, G., V. Barghout, V. Patel, W. Tsong, and Z. Wang. "Budget Impact of Perampanel as Adjunctive Treatment of Uncontrolled Partial-Onset and Primary Generalized Tonic-Clonic Seizures in the United States." Epilepsy & Behavio : E&B 68 (March 2017): 196–202. https://doi.org/10.1016/j.yebeh.2016.12.029.
- Bassali, Jan, Ian Gopal Gould, James A. Kaye, Deirdre Mladsi, and Jyotsna Mehta. "US Budget Impact Model for Selinexor in Relapsed or Refractory Multiple Myeloma." ClinicoEconomics and Outcomes Research. Dove Press, June 19, 2020. https://doi.org/10.2147/CEOR.S251070.
4 ITC/NMA/STC/MAIC
- Tremblay, G., T. Westley, J. C. Cappelleri, B. Arondekar, G. Chan, T. J. Bell, and A. Briggs. "Overall Survival of Glasdegib in Combination with Low-Dose Cytarabine, Azacitidine, and Decitabine among Adult Patients with Previously Untreated AML: Comparative Effectiveness Using Simulated Treatment Comparisons." ClinicoEconomics and Outcomes Research : CEOR 11 (2019): 551–65. https://doi.org/10.2147/CEOR.S203482.
- Tremblay, G., T. Westley, S. A. Garib, T. Bell, J. C. Cappelleri, G. Chan, and A. Forsythe. "Indirect Number Needed To Treat: Comparative Effectiveness Of Glasdegib + Low-Dose Cytarabine Vs Azacitidine Or Decitabine In Acute Myeloid Leukemia Patients Ineligible For Intensive Chemotherapy." Amsterdam, UK, 2019. http://journals.lww.com/10.1097/01.HS9.0000562476.96992.f3.
- Tremblay, G., T. Westley, T. J. Bell, J. C. Cappelleri, G. Chan, J. Hearnden, and A. Forsythe. "Indirect Comparative Effectiveness of Treatments for Older Acute Myeloid Leukemia (AML) Patients Ineligible for Intensive Chemotherapy (NIC): Combinations Glasdegib + Low-Dose Cytarabine (GLAS+LDAC) vs Decitabine or Venetoclax + LDAC." Glasgow, Scotland, 2019. https://onlinelibrary.wiley.com/doi/abs/10.1111/bjh.15854.
- Forsythe, A., B. Arondekar, G. Tremblay, G. Chan, and Y. Su. "Systematic Literature Review And Indirect Comparison of Glasdegib Plus Low Dose ARA-C Versus A Hypomethylating Agent For Acute Myeloid Leukemia Patients Ineligible For Intensive Chemotherapy." Madrid, Spain, 2017. https://linkinghub.elsevier.com/retrieve/pii/S1098301517304370.
- Westley, T., A. Forsythe, T. J. Bell, J. C. Cappelleri, G. Chan, and G. Tremblay. "Comparative Effectiveness of Combination Glasdegib+ Low-Dose Cytarabine (GLAS+LDAC) Vs Combination Venetoclax + Low-Dose Cytarabine (VEN+LDAC) Among Older Acute Myeloid Leukemia (AML) Patients Ineligible for Intensive Chemotherapy (NIC): Indirect Treatment Comparison (ITC) Methods." San Diego, CA, 2018. https://ashpublications.org/blood/article/132/Supplement 1/1429/262867/Comparative-Effectiveness-of-Combination-Glasdegib.
- Westley, T., A. Forsythe, T. J. Bell, J. C. Cappelleri, G. Tremblay, and G. Chan. "Clinical Responses of Glasdegibplus Low-Dose Ara-C, Azacitidine, and Decitabine Among Acute Myeloid Leukemia (AML) Patients Ineligible to Receive Intensive Chemotherapy: Comparative Effectiveness Using Indirect Treatment Comparison (ITC) Methods." San Diego, CA, 2018. https://ashpublications.org/blood/article/132/Supplement 1/2709/264110/Clinical-Responses-of-Glasdegibplus-Low-Dose-Ara-C.
- Tremblay, G., B. Arondekar, G. Chan, A. Shor, A. Forsythe, and S. Yun. "Covariate Adjusted Indirect Treatment Comparison (ITC) of Glasdegib Plus Low Dose Ara-C Versus a Hypomethylating Agent for Acute Myeloid Leukemia Patients Ineligible for Intensive Chemotherapy." Blood 130, no. Supplement 1 (December 7, 2017): 5665–5665. https://doi.org/10.1182/blood.V130.Suppl_1.5665.5665.
- Forsythe, A., B. Arondekar, G. Tremblay, G. Chan, and Y. Su. "Systematic Literature Review And Indirect Comparison of Glasdegib Plus Low Dose ARA-C Versus A Hypomethylating Agent For Acute Myeloid Leukemia Patients Ineligible For Intensive Chemotherapy." Glasgow, Scotland, 2017. https://doi.org/10.1016/j.jval.2017.08.103.
- Tremblay, G, T Westley, B Arondekar, J Cappelleri, G Chan, A Forsythe, and A Briggs. "Evaluating Different Indirect Treatment Comparison Approaches: A Case Study in Acute Myeloid Leukemia Patients Ineligible to Receive Intensive Chemotherapy." Baltimore, MD, USA, 2018. https://doi.org/10.1016/j.jval.2018.04.100.
- Forsythe, A., M. Dolph, G. Tremblay, R. Dhanda, J. Patterson, V. Nadipelli, P. Pezalla, L. Citrome, and C. U. Correll. "A Systematic Literature Review and Network Meta-Analysis of Long-Acting Injectable Therapies (LAIs) for Acute Schizophrenia." Orlando, FL, 2018. https://www.jmcp.org/doi/10.18553/jmcp.2018.24.10-a.s1.
- Forsythe, A., T. Westley, G. Tremblay, R. Dhanda, J. Patterson, V. Nadipelli, P. Pezalla, L. Citrome, and C. U. Correll. "Matching-Adjusted Treatment Comparison (MAIC) of RBP-7000 Monthly Subcutaneous Injection and Paliperidone Palmitate Monthly Intramuscular Injection (PP1M) for Acute-Onset Schizophrenia." Orlando, FL, 2018. https://www.jmcp.org/doi/10.18553/jmcp.2018.24.10-a.s1.
- Tremblay, G., D. Chandiwana, M. Dolph, J. Hearnden, A. Forsythe, and M. Monaco. "Matching-Adjusted Indirect Treatment Comparison of Ribociclib and Palbociclib in HR+, HER2- Advanced Breast Cancer." Cancer Management and Research 10 (2018): 1319–27. https://doi.org/10.2147/CMAR.S163478.
- Forsythe, A., D. Chandiwana, M. Dolph, G. Tremblay, and M. Monaco. "Matching-Adjusted Indirect Treatment Comparison of Ribociclib and Palbociclib as First-Line Treatments for HR+, HER2– ABC." Madrid, Spain, 2017.
- Tremblay, G, M Dolph, T Jones, and A Forsythe. "Bayesian Network Meta-Analysis of Treatments in Iron Deficiency Anaemia (IDA)." Baltimore, MD, USA, 2018. https://linkinghub.elsevier.com/retrieve/pii/S1098301518319958.
- Jones, T., A. Forsythe, M. Sampson, G. Tremblay, and M. Dolph. "Using A Bayesian Network Meta-Analysis (NMA) To Compare Ferric Maltol To Treatments For Iron Deficiency And Iron Deficiency Anaemia Excluding CHF And CKD Patients." Barcelona, Spain, 2018. https://doi.org/10.1016/j.jval.2018.09.2596.
- Guyot, P., W. Cheng, G. Tremblay, R. Copher, H. Burnett, X. Li, and C. Makin. "Number Needed to Treat in Indirect Treatment Comparison." Journal of Comparative Effectiveness Research 7, no. 3 (March 2018): 259–69. https://doi.org/10.2217/cer-2017-0023.
- Tremblay, G, G Meier, R Copher, D Misurski, J Pan, M Baig, T Tamai, S Kraljevic, A Shor, and A Forsythe. "Network Meta-Analysis of Treatments For Unresectable Hepatocellular Carcinoma." Glasgow, Scotland, 2017. https://doi.org/10.1016/j.jval.2017.08.096.
- Tremblay, G., G. Meier, D. Misurski, M. Baig, T. Tamai, S. Kraljevic, A. Shor, and A. Forsythe. "Network Meta-Analysis (NMA) of Treatments for Unresectable Hepatocellular Carcinoma (UHCC)." Madrid, Spain, 2017.
- Tremblay, G, SA Garib, G Meir, HJ McElroy, and M Guo. "Comparing ITC Results From Lenvatinib Plus Everolimus For Second-Line Treatment of Advanced/Metastatic Renal Cell Carcinoma: Crossover Versus No Crossover." Glasgow, Scotland, 2017. https://doi.org/10.1016/j.jval.2017.08.106.
- Garib, S., G. Tremblay, G. Meier, H. McElroy, and M. Guo. "Comparing ITC Results from Lenvatinib plus Everolimus for Second-Line Treatment of Advanced/Metastatic Renal Cell Carcinoma: Crossover versus No Crossover." Madrid, Spain, 2017.
- Holbrook, T., G. Milligan, C. Pelletier, P. Rietschel, and G. Tremblay. "Matching-Adjusted Indirect Treatment Comparison in Patients with Radioiodine-Refractory Differentiated Thyroid Cancer." Comparative Effectiveness Research, April 2016, 13. https://doi.org/10.2147/CER.S97885.
- Mehra D et al. "Network Meta-analysis Comparing the Psychiatric Treatment-emergent Adverse Events of Eslicarbazepine Acetate and Brivaracetam." American Epilepsy Society Conference Proceedings (2020) – Accepted for publication at American Epilepsy Society Conference in 2020.
- Westley, T., D. Tomaras, E. Strati, D. Skerrett, A. Forsythe, and G. Tremblay. "Simulated Treatment Comparison (STC) of Human Mesenchymal Stromal Cells (Remestemcel-L, REM) and Ruxolitinib (RUX) for Steroid-Refractory Acute Graft-Versus-Host Disease (SR AGVHD) Pediatric Patients." Orlando, FL, 2019. https://ashpublications.org/blood/article/134/Supplement_1/4545/424492/Simulated-Treatment-Comparison-STC-of-Human.
- Dolph, M., and G. Tremblay. "Covariate Adjustment in Indirect Treatment Comparison (ITC): How To Blend The Useful With The Agreeable." Copenhagen, Denmark, 2019. https://doi.org/10.1016/j.jval.2019.09.1917.
- Tremblay G, "The use of simulated and indirect treatment comparison methods to evaluate therapies' comparative effectiveness: an interview with Gabriel Tremblay."
- Tremblay, G. Daniele, P. Dolph, P. et al.; Comparative Effectiveness of Glasdegib or Venetoclax in Combination with Low-Dose Cytarabine Using Simulated Treatment Comparisons. Blood 2020; 136 (Supplement 1): 17–18. doi: https://doi.org/10.1182/blood-2020-137641
QOL/UTILITY
- Breeze J, Mehta J, Tremblay G. "Minimal Clinically Important Difference (MCID) For The Functional Assessment Of Cancer Therapy - Multiple Myeloma (FACT-MM): Two Approaches." Value in Health (2020).
- Jagannath, S., J. Mehta, J. Breeze, L. Lingling, N. Biran, S. Shacham, M. G. Kauffman, J. J. Shah. "Quality of Life (QOL) Analyses in Patients with Multiple Myeloma: Results from the Selinexor (KPT-330) Treatment of Refractory Myeloma (STORM) Phase 2b Study." ASCO (American Society of Clinical Oncology) Journal Proceedings, (2020).
- Breeze J, Mehta J, Tremblay G, Shacham S, Shah J, Jagannath S. "Quality Of Life Analyses In Patients With Multiple Myeloma: Results From The Selinexor (KPT-330) Treatment Of Refractory Myeloma (STORM) Phase 2B Study." Value in Health (2020).
- Casasnovas RO , Daniele P , Tremblay G et al. "Health Utility in Relapsed/Refractory Dixuse Large B-Cell Lymphoma (RR-DLBCL) Patients - Results of a
Phase II Trial with ORAL Selinexor." Value in Health (2020) – Accepted for poster presentation at ISPOR EU 2020. - Shor, A, A Forsythe, S Li, and A Galaznik. "Systematic Literature Review (SLR) Of Health State Utility Values (HSUV) In Epidermal Growth Factor Receptor Mutant (Egfr-Mutant) Non-Small Cell Lung Cancer (NSCLC) Patients Previously Treated With Targeted Therapies." Baltimore, MD, USA, 2018. https://doi.org/10.1016/j.jval.2018.04.300.
- Forsythe, A., V. BaI, M. Dolph, S. Patel, and G. Tremblay. "Systematic Review Of Health State Utility Values For Economic Evaluation Of Acute Myeloid Leukemia." Madrid, Spain, 2017. https://library.ehaweb.org/eha/2017/22nd/180721/anna.forsythe.systematic.review.of.health.state.utility.values.for.economic.html?f=menu%3D14%2Abrowseby%3D8%2Asortby%3D2%2Amedia%3D3%2Aspeaker%3D619413.
- Forsythe, A., C. S. Kwon, T. Bell, T. A. Smith, and B. Arondekar. "Health-Related Quality of Life in Acute Myeloid Leukemia Patients Not Eligible for Intensive Chemotherapy: Results of a Systematic Literature Review." ClinicoEconomics and Outcomes Researc : CEOR 11 (2019): 87–98. https://doi.org/10.2147/CEOR.S187409.
- Forsythe, A., C. S. Kwon, T. Bell, T. A. Smith, B. Arondekar, and A. P. J. Rabe. "Health Related Quality Of Life (HRQOL) In Acute Myeloid Leukemia (AML) Patients Not Eligible For Intensive Chemotherapy (NIC AML): Results Of A Systematic Literature Review." HemaSphere 2, no. S1 (June 14, 2018): 790–91.
- Forsythe, A., P. S. Brandt, M. Dolph, S. Patel, A. P. J. Rabe, and G. Tremblay. "Systematic Review of Health State Utility Values for Acute Myeloid Leukemia." ClinicoEconomics and Outcomes Researc : CEOR 10 (2018): 83–92. https://doi.org/10.2147/CEOR.S153286.
- Badia, X., P. Trainer, N. R. Biermasz, J. Tiemensma, A. Carreno, M. Roset, A. Forsythe, and S. M. Webb. "Mapping AcroQoL Scores to EQ-5D to Obtain Utility Values for Patients with Acromegaly." Journal of Medical Economics 21, no. 4 (April 2018): 382–89. https://doi.org/10.1080/13696998.2017.1419960.
- Badia, X., N. Biermasz, J. Tiemensma, A. Carreno, M. Roset, and A. Forsythe. "Mapping Acroqol Scores to EQ-5D to Obtain Utility Values for Patients with Acromegaly." Glasgow, Scotland, 2017. https://doi.org/10.1016/j.jval.2017.08.484.
- Tremblay, G, A Forsythe, A Rabe, and T Jones. "Comparing Short Form – 6 Dimension (SF-6D) Derived Utilities and Euroqol Questionnaire – 5 Dimensions (EQ-5D) Mapped Utilities Among Patients with Iron Deficiency in Inflammatory Bowel Disease (ID-IBD) Treated with Ferric Maltol (FM) Vs Placebo." Baltimore, MD, USA, 2018. https://doi.org/10.1016/j.jval.2018.04.585.
- Schmidt, C., D. Baumgart, B. Bokemeyer, C. Büning, S. Howaldt, A. Stallmach, C. Singfield, G. Tremblay, and T. Jones. "Treatment with Ferric Maltol Associated with Improvements in Quality of Life for IBD Patients with Iron Deficiency Anaemia." Vienna, Austria, 2018. https://academic.oup.com/ecco-jcc/article/12/supplement_1/S346/4807586.
- Hudgens, S., A. Forsythe, I. Kontoudis, D. D'Adamo, A. Bird, and H. Gelderblom. "Evaluation of Quality of Life at Progression in Patients with Soft Tissue Sarcoma." Sarcoma 2017 (2017): 2372135. https://doi.org/10.1155/2017/2372135.
- Strati, E., A. Forsythe, and G. Tremblay. "Quality of Life (QoL) in Pediatric Patients Treated with Remestemcel-L for Steroid-Refractory Acute Graft Versus Host Disease (AGVHD) Following Allogeneic Hematopoietic Stem Cell Transplantation (HSCT): Analysis of Msb-GVHD001 Phase 3 Study." Orlando, FL, 2020.
- Tremblay, G., M. de Chantal, A. Forsythe, and C. Marsolais. "The Use Of EQ-5D Visual Analog Scale (EQ-VAS) In Diseases Areas Where EQ-5D Is Insensitive To Changes In The Disease Status: The Case Of Lipodystrophy." Copenhagen, Denmark, 2019. https://doi.org/10.1016/j.jval.2019.09.2632.
RWE
- Ramjee L, Tremblay G. "A Framework For Conducting Probabilistic Sensitivity Analysis (PSA) When Modeling With Real-World Evidence (RWE) - How Good Is The Guidance?" Value in Health (2020).
- Forsythe, A., D. Tomaras, F.J. Badia, O. Pirk, P. Rowe, J. F. Bergman, A. Walker, F. U. Fricke, and G. Tremblay. "Is There A Need For Real World Evidence (RWE) In Health Technology Assessments Of Rare Diseases And Oncology Treatments?" Copenhagen, Denmark, 2019. https://doi.org/10.1016/j.jval.2019.09.2184.
- Ramjee, L., G. Tremblay, and A. Forsythe. "Real World Evidence (RWE) In Partition Survival Modelling: Is It The Real Deal?" Copenhagen, Denmark, 2019. https://doi.org/10.1016/j.jval.2019.09.1923.
- Forsythe, A. "Using Real World Evidence for Decision-Making In Health Technology Assessment: Case Studies." Oral presentation presented at the European Society for Medical Oncology Congress (ESMO), Munich, October 21, 2018.
- Grabner, M., E. Strati, K. Sandman, and A. Forsythe. "Economic Burden of Acute Steroid-Refractory Graft Versus Host Disease in Commercially Insured Pediatric Patients." Journal of Managed Care & Specialty Pharmacy 26, no. 4-a (April 2020). https://doi.org/10.18553/jmcp.2020.26.4-a.s1.
- Grabner, M., E. Strati, K. Sandman, and A. Forsythe. "Economic Burden of Acute Steroid-Refractory Graft Versus Host Disease in Commercially Insured Pediatric Patients." Presented at the AMCP (Virtual), Houston, Texas, April 21, 2020.
- Watson, C., H. Xu, A. Forsythe, S. A. Garib, and A. Barlev. "Younger Patients Are Impacted By Post-Transplant Lymphoproliferative Disorder: Findings from a Systematic Literature Review of Real-World Evidence." Blood 132, no. Supplement 1 (November 29, 2018): 5841–5841. https://doi.org/10.1182/blood-2018-99-113622.
- Forsythe, A., J. Hearnden, G. Lewis, and R. Schreiber. "Real World Evidence on the Burden of Illness Experienced by Patients With Systemic Mycoses," 1, 2018.
- Forsythe, A., J. Hearnden, G. Lewis, and R. Schreiber. "Real World Evidence On The Burden Of Illness Experienced By Patients With Hematological Malignancies And Systemic Mycoses." Stockholm, Sweden, 2018. https://library.ehaweb.org/eha/2018/stockholm/214741/anna.forsythe.real.world.evidence.on.the.burden.of.illness.experienced.by.html?f=menu%3D14%2Abrowseby%3D8%2Asortby%3D2%2Amedia%3D3%2Aspeaker%3D619413.
- Schreiber, R., A. Forsythe, J. Hearnden, and G. Lewis. "Real World Evidence on the Burden of Illness Experienced by HIV Patients with Systemic Mycoses," 2018. https://academic.oup.com/mmy/article/56/suppl_2/S1/5033473.
- Forsythe, Anna, Gareth Lewis, Robyn Jordan, and George R Thompson. "US Database Study: Burden and Healthcare Resource Utilization in Adults with Systemic Endemic Mycoses and Aspergillosis." Journal of Comparative Effectiveness Research, April 22, 2020, cer-2020-0019. https://doi.org/10.2217/cer-2020-0019.
- Forsythe, A., A. Roy, M. Bhor, A. Allepuz, M. D. S. O. Portella, C. Kwon, and G. Tremblay. "Benefit-Risk Comparison in Patients with Immune Thrombocytopenia (ITP) Receiving Eltrombopag or Romiplostim: Real World Evidence (RWE) from 26 Hospital Institutions." Atlanta, GA, USA, 2017. https://ashpublications.org/blood/article/130/Supplement 1/3409/114492/Benefit-Risk-Comparison-in-Patients-with-Immune.
- Forsythe, A, A Roy, M Bhor, and M Socorro O Portella. "Bleeding-Related Episodes (BRE) in Patients With Immune Thrombocytopenia (ITP) Receiving Eltrombopag (EPAG) or Romiplostim (ROMI): Real World Evidence from 26 US Institutions." Glasgow, Scotland, 2017. https://doi.org/10.1016/j.jval.2017.08.834.
- Forsythe, A, M Bhor, A Roy, M Socorro O Portella, C Kwon, and G Tremblay. "Economic Burden of Immune Thrombocytopenia (ITP) In Patients Receiving Eltrombopag (EPAG) And Romiplostim (ROMI): Real World Evidence From 26 US Institutions." Glasgow, Scotland, 2017. https://doi.org/10.1016/j.jval.2017.08.870.
- Kwon, C., A. Forsythe, A. Roy, M. Bhor, M. D. S. O. Portella, and G. Tremblay. "Health Care Resource Use (HCRU) Due To Bleeding Related Episodes (BRE) In Patients With Immune Thrombocytopenic Purpura (ITP) Receiving Eltrombopag (EPAG), Romiplostim (ROMI), Or Rituximab (RITUX): Real World Evidence (RWE) From 27 US Institutions." Glasgow, Scotland, 2017. https://doi.org/10.1016/j.jval.2017.08.903.
- Forsythe, Anna, John Schneider, Timothy Pham, Menaka Bhor, Qayyim Said, Alejandro Allepuz, Maria do Socorro O Portella, Christina S Kwon, and Anuja Nidumolu Roy. "Real-World Evidence on Clinical Outcomes in Immune Thrombocytopenia Treated with Thrombopoietin Receptor Agonists." Journal of Comparative Effectiveness Research, March 16, 2020, cer-2019-0177. https://doi.org/10.2217/cer-2019-0177.
- Gupta, S., P. Kwan, E. Faught, W. Tsong, A. Forsythe, and P. Ryvlin. "Understanding the Burden of Idiopathic Generalized Epilepsy in the United States, Europe, and Brazil: An Analysis from the National Health and Wellness Survey." Epilepsy & Behavio : E&B 55 (February 2016): 146–56. https://doi.org/10.1016/j.yebeh.2015.12.018.
- Kwon, C., P. Brandt, S. Manson, A. Fuentes-Alburo, and A. Forsythe. "Treatment Patterns and Health Care Resources Use (HCRU) in Patients with Acute Myeloid Leukemia (AML): Real World Evidence (RWE) from 30 US Institutions." Blood 130, no. Supplement 1 (December 7, 2017): 5655–5655. https://doi.org/10.1182/blood.V130.Suppl_1.5655.5655.
- Forsythe, A., D. Tomaras, K. Parikh, and R. Copher. "An SLR of Clinical and Real-World Evidence in Relapsed/Refractory Multiple Myeloma (RRMM) Studies to Inform Clinical Decision-Making in the US." Boston, MA, USA, 2019. https://linkinghub.elsevier.com/retrieve/pii/S2152265019318324.
SYSTEMATIC LITERATURE REVIEW AND BURDEN OF ILLNESS
- Shor, A., A. Forsythe, S. Li, and A. Galaznik. "Comparative Effectiveness of Treatments In Epidermal Growth Factor Receptor Mutant (EGFR-MUTANT) Non-Small Cell Lung Cancer (NSCLC) Patients WHO Failed On Prior Targeted Therapies: A Systematic Literature Review (SLR)." Baltimore, MD, USA, 2018. https://doi.org/10.1016/j.jval.2018.04.096.
- Dimitrios Tomaras , Huamao M Lin, Anna Forsythe, Victoria Crossland, Sai-Hong Ou. "Clinical and Real-world Outcomes in Patients With Epidermal Growth Factor Receptor (EGFR) Exon 20 Insertions in Non-small Cell Lung Cancer (NSCLC): a Meta-analysis." Virtual ESMO, 2020.
- Pan, X., C. S. Kwon, S. A. Garib, A. Forsythe, and H. M. Lin. "Epidemiological, Clinical and Humanistic Burden Among ALK+ Non-Small Cell Lung Cancer (ALK+NSCLC) Patients Treated With First-Line ALK Inhibitors: Results Of A Systematic Literature Review (SLR)." New Orleans, LA, USA, 2019. https://doi.org/10.1016/j.jval.2019.04.127.
- Pan, X., C. S. Kwon, S. A. Garib, A. Forsythe, and H. M. Lin. "Burden Of Brain Metastases (BM) In ALK+ Non-Small Cell Lung Cancer (ALK+ NSCLC) Treated With First-Line ALK Inhibitors: Results Of A Systematic Literature Review (SLR)." Copenhagen, Denmark, 2019. https://doi.org/10.1016/j.jval.2019.09.219.
- Forsythe, A., E. Kim, K. Parikh, and R. Copher. "Comparative Effectiveness of Induction Treatment Regimens in NDMM: Results of an SLR to Inform Clinical Decision-Making in the US." Boston, MA, USA, 2019. https://linkinghub.elsevier.com/retrieve/pii/S2152265019317276.
- Forsythe, A., E. Kim, K. Parikh, and R. Copher. "Effectiveness of Maintenance Treatment in Newly Diagnosed Multiple Myeloma (NDMM): Results of an SLR to Inform Clinical Decision-Making in the US." Boston, MA, USA, 2019. https://linkinghub.elsevier.com/retrieve/pii/S2152265019318749.
- Forsythe A, Kwon CS, Kim ME et al. "Systematic Literature Review (SLR) of Evidence on the Humanistic Burden of Relapsed/Refractory Multiple
Myeloma (RRMM)." Value in Health (2020) – Accepted for poster presentation at ISPOR EU 2020. - Forsythe A, Kim ME, Kwon CS et al. "A Systematic Literature Review (SLR) of Health State Utility Values (HSUVS) in Relapsed/Refractory Multiple Myeloma (RRMM)." Value in Health (2020) – Accepted for poster presentation at ISPOR EU 2020.
- Tomaras, D., E. Strati, and A. Forsythe. "The Economic And Humanistic Burden Of Graft-Versus-Host Disease (GVHD) In Pediatric Patients: A Systematic Literature Review (SLR)." Amsterdam, UK, 2019. http://journals.lww.com/10.1097/01.HS9.0000561156.61407.1f.
- Tomaras, D., M. Dolph, P. Caudrelier, M. Dumont-Lagacé, and A. Forsythe. "Allogeneic Hematopoietic Stem Cell Transplantations (ALLO-HSCT) Are Associated With Poor Quality Of Life (QOL) And Economic Burden Due To Graft-Versus-Host Disease (GVHD) Complications: Results Of A Systematic Literature Review (SLR)." In Value in Health. New Orleans, LA, USA, 2019. https://doi.org/10.1016/j.jval.2019.04.409.
- Tomaras, D., M. Dolph, P. Caudrelier, M. Dumont-Lagacé, and A. Forsythe. "Acute Graft Versus Host Disease (GVHD), Chronic GVHD And Non-Relapse Mortality (NRM) Associated With Mismatched Allogeneic Hematopoietic Stem Cell Transplantation (ALLO-HSCT): A Systematic Review." New Orleans, LA, USA, 2019. https://doi.org/10.1016/j.jval.2019.04.410.
- Tomaras, D., M. Dolph, P. Caudrelier, M. Dumont-Lagacé, and A. Forsythe. "Comparative Effectiveness Of Allogeneic Hematopoietic Stem Cell Transplantations (ALLO-HSCT) From Different Graft Sources In Acute Myeloid Leukemia (AML): Results Of A Systematic Literature Review." New Orleans, LA, USA, 2019. https://doi.org/10.1016/j.jval.2019.04.150.
- Xu, H., C. Watson, S. A. Garib, A. Forsythe, and A. Barlev. "Treatment Patterns for Patients with Post-Transplant Lymphoproliferative Disorder Who Fail Rituximab after Allogeneic Hematopoietic Stem Cell Transplantation: Findings from a Systematic Literature Review." San Diego, CA, 2018. https://ashpublications.org/blood/article/132/Supplement 1/4777/262154/Treatment-Patterns-for-Patients-with-Post.
- Xu, H., A. Forsythe, A. Barlev, N. Rashid, and C. Watson. "A Systematic Literature Review of Real-World Evidence in Post-Transplant Lymphoproliferative Disorder." Blood 132, no. Supplement 1 (November 29, 2018): 5839–5839. https://doi.org/10.1182/blood-2018-99-113583.
- Sandman K, Bell T. "Patient-Focused Benefits Of At-Home Vs In-Clinic Administration Of Cancer Therapy." Value in Health (2020).
- Tomaras D, Marshall S, Forsythe A. "The Humanistic And Economic Burden Of Myasthenia Gravis (MG) - A Debilitating & Costly Disease." Value in Health (2020).
- Forsythe A, Vieweg DC, Marshall S, Tomaras D, Kim E. "Symptoms, Delayed Diagnosis And Quality Of Life (QOL) Deterioration - Exploring The Burden Of Amyotrophic Lateral Sclerosis (ALS)." Value in Health (2020).
- Forsythe A, Kim ME, Tomaras D, Kielhorn A. "Amyotrophic Lateral Sclerosis (ALS) Results In High Direct And Indirect Costs To Patients, Caregivers, And Healthcare Systems" Value in Health (2020).
- Hearnden, J., K. Dudoit, E. Kim, G. Tremblay, and A. Forsythe. "Use Of Computer-Assisted Methods To Realize The Concept Of A Living Systematic Review Via An Online Platform." Copenhagen, Denmark, 2019. https://doi.org/10.1016/j.jval.2019.09.1736.
- Kwon, C. S., L. J. Lee, A. Forsythe, and C. Mamolo. "Clinical And Humanistic Burden Among Non-Muscle Invasive Bladder Cancer Patients: Results Of A Systematic Literature Review." Copenhagen, Denmark, 2019. https://doi.org/10.1016/j.jval.2019.09.231.
- Kaufman, P. A., C. S. Kwon, J. Feliciano, T. Westley, A. Forsythe, P. Garfin, and L. Brown. "Systemic Therapy in Second-Line Metastatic Triple Negative Breast Cancer (MTNBC): A Systematic Literature Review (SLR) and Meta-Analysis (MA) of Efficacy." Barcelona, Spain, 2019.
- Hitron, M., Smith, G., Forsythe, A., Harricharan, S., Shah, MA. "An evaluation of the efficacy and safety of second line (2L) treatments in metastatic colorectal cancer (mCRC)" Journal of Managed Care & Specialty Pharmacy (2020) – Accepted for presentation at AMCP Nexus in October 2020.
- Forsythe A, Zhang WW, Strauss UP, Fellous M, Korei M, Keating KN. "Challenges of Descriptive Epidemiology in Rare Diseases: A Systematic Literature Review (SLR) and Meta-Analysis (MA) of Solid Tumors with Neurotrophic Tyrosine Receptor Kinase (NTRK) Gene Fusions." Value in Health (2020) – Accepted for poster presentation at ISPOR EU 2020.
- Crotty, C., A. Forsythe, and M. Magestro. "Unmet Needs & Burden of Recurrent Pericarditis (RP): Results of A Systematic Literature Review (SLR)." New Orleans, LA, USA, 2019. https://doi.org/10.1016/j.jval.2019.04.445.
- Forsythe, A., J. Hearnden, K. Dudoit, C. Crotty, and G. Tremblay. "Real-Time Systematic Literature Reviews (SLR) - Dream or Reality With Computer-Assisted Methods: A Case Study In Acute Myeloid Leukemia (AML)." New Orleans, LA, USA, 2019. https://doi.org/10.1016/j.jval.2019.04.364.
- Tomaras D , Harricharan S , Tremblay G , Forsythe A. "Understanding the Clinical Burden of Acute Respiratory Distress Syndrome (ARDS) in a Covid-19 Era: a Targeted Search and Epidemiological Model." Value in Health (2020) – Accepted for poster presentation at ISPOR EU 2020.
- Hubscher, E. Sikirica, S. Bell, TJ. et al.; Patterns of Undertreatment Among Patients with Acute Myeloid Leukemia (AML) Not Receiving Standard Intensive Induction Chemotherapy. Blood 2020; 136 (Supplement 1): 18–19. doi: https://doi.org/10.1182/blood-2020-137425
EARLY ECONOMIC MODELS AND VALUE FRAMEWORKS
- Azimpour K, Tremblay G, Hagemeister N, Cagnin A, Fuentes A. "Conceptual Framework For Economic Evaluation Of Knee Kinesiography For The Management Of Knee Osteoarthritis." Value in Health (2020).
- Tremblay G, Lemay W. "Economic Value Drivers In Severe Red Blood Cell Disorders: A Review And Development Of An Economic Conceptual Framework." Value in Health (2020).
- Tomaras, D., W. Lemay, and G. Tremblay. "Economic Evaluation of Severe Anaemia: Review-Based Recommendations and a Conceptual Framework." European Medical Journal 5, no. 3 (September 17, 2020): 45–54. https://doi.org/10.33590/emj/20-00102.
- Tremblay G, Tomaras D, Mearns ES, Copher R. "A Conceptual Framework For Economic Evaluation Of Beta-Thalassemia." Value in Health (2020).
- Tomaras, D., L. Ramjee, A. Forsythe, and G. Tremblay. "The Role Of Early Value Framework In A Successful Reimbursement Journey." Copenhagen, Denmark, 2019. https://doi.org/10.1016/j.jval.2019.09.2177.
- Dolph, M., A. Roy, M. Bhor, J. Hearnden, C. S. Kwon, A. Forsythe, G. Tremblay, and A. Briggs. "A Decision Framework for Treating Chronic Immune Thrombocytopenia with Thrombopoietin Receptor Agonists." Journal of Comparative Effectiveness Research 7, no. 8 (August 2018): 775–84. https://doi.org/10.2217/cer-2018-0034.
HEALTH ECONOMICS METHODS
- Dolph M, Tremblay G. "Using Cure Models In Economic Analyses: A Review And Conceptual Guideline." Value in Health (2020).
- Azimpour, K., G. Tremblay, and A. Forsythe. "Clinical and Key Opinion Leaders Validation In Health Economics Model And Comparative Effectiveness: A Guide For Non-Expert Audience." Copenhagen, Denmark, 2019. https://doi.org/10.1016/j.jval.2019.09.2197.
- Azimpour, K., G. Tremblay, and A. Forsythe. "What Is A Good or Bad Number Need to Treat (NNT) Value? A Literature Review." Copenhagen, Denmark, 2019. https://doi.org/10.1016/j.jval.2019.09.1623.
- Tremblay, G., K. Azimpour, and A. Forsythe. "Per-Member Per-Month (PMPM) Expenditure Value in Oncology: A Quick Literature Review And Opinion." Copenhagen, Denmark, 2019. https://doi.org/10.1016/j.jval.2019.09.2198.
- Cariou, C., G. Tremblay, M. Dolph, P. S. Brandt, and A. Forsythe. "Incremental Quality-Adjusted Survival Analysis When No Head To Head Data Are Available: A Case Study Of Midostaurin (MIDO) Versus Standard Of Care (SOC) In Patients With Advanced Systemic Mastocytosis (ASM)." Barcelona, Spain, 2018. https://doi.org/10.1016/j.jval.2018.09.2791.
- Tremblay, G., T. Westley, A. Forsythe, C. Pelletier, and A. Briggs. "A Criterion-Based Approach to Systematic and Transparent Comparative Effectiveness: A Case Study in Psoriatic Arthritis." Journal of Comparative Effectiveness Research 8, no. 15 (November 2019): 1265–98. https://doi.org/10.2217/cer-2019-0064.
- Tremblay, G., A. Forsythe, C. Pelletier, and A. Briggs. "A Criterion-Based Approach to Systematic and Transparent Comparative Effectiveness Research: A Case Study in Psoriatic Arthritis." Dallas, TX, 2017. https://www.jmcp.org/doi/10.18553/jmcp.2017.23.10-a.s1.
- Tremblay, G., C. Livings, L. Crowe, V. Kapetanakis, and A. Briggs. "Determination of the Most Appropriate Method for Extrapolating Overall Survival Data from a Placebo-Controlled Clinical Trial of Lenvatinib for Progressive, Radioiodine-Refractory Differentiated Thyroid Cancer." ClinicoEconomics and Outcomes Research Volume 8 (June 2016): 323–33. https://doi.org/10.2147/CEOR.S107498.
- Briggs, Andrew, Bruno Daniele, Katherine Dick, Thomas R. Jeffry Evans, Peter R. Galle, Richard A. Hubner, Carlos Lopez, Uwe Siebert, and Gabriel Tremblay. "Covariate-Adjusted Analysis of the Phase 3 REFLECT Study of Lenvatinib versus Sorafenib in the Treatment of Unresectable Hepatocellular Carcinoma." British Journal of Cancer, April 8, 2020, 1–6. https://doi.org/10.1038/s41416-020-0817-7.
ENDPOINT VALIDATION
- Forsythe, A., D. Chandiwana, J. Barth, M. Thabane, J. Baeck, A. Shor, and G. Tremblay. "Is Progression-Free Survival a More Relevant Endpoint than Overall Survival in First-Line HR+/HER2- Metastatic Breast Cancer?" Cancer Management and Research 10 (2018): 1015–25. https://doi.org/10.2147/CMAR.S162714.
- Forsythe, A., D. Chandiwana, J. Barth, M. Thabane, J. Baeck, and G. Tremblay. "Progression-Free Survival/Time to Progression as a Potential Surrogate for Overall Survival in HR+, HER2- Metastatic Breast Cancer." Breast Cancer (Dove Medical Press) 10 (2018): 69–78. https://doi.org/10.2147/BCTT.S162841.
- Forsythe, A., D. Chandiwana, J. Barth, M. Thabane, J. Baeck, A. Shor, and G. Tremblay. "PFS/TTP as a Potential Surrogate for OS in HR+, HER2– MBC." Madrid, Spain, 2017.
- Forsythe, A., D. Chandiwana, J. Barth, M. Thabane, J. Baeck, A. Shor, and G. Tremblay. "Is PFS a More Relevant Endpoint than OS in 1L HR+, HER2– MBC? A Systematic Literature Review." Madrid, Spain, 2017.
HEALTH TECHNOLOGY ASSESSMENTS
- Forsythe, A., J. Schneider, A. Shor, P. Patel, and A. Briggs. "Application of Oncology Value Frameworks to the 2nd Line Treatments in Renal Cell Carcinoma (2L-RCC)." Boston, MA, USA, 2017. https://doi.org/10.1016/j.jval.2017.05.005.
- Forsythe, A., and E. Pezalla. "Defining the Value: Do the New Oncology Frameworks in the Us Aim at Objective Valuation? – A Case Study in Multiple Myeloma." Oral Presentation presented at the 2nd Annual Pharma Pricing, Reimbursement and Marketing Access 2016, London, England, June 8, 2016.
- Vu BK, Forsythe A, Nguyen K, Tomaras D. "Health Technology Assessment (HTA) Review Of Cell & Gene Therapies: The Road To Reimbursement." Value in Health (2020).
- Nguyen K, Forsythe A, Azimpour K, Vu BK, Tomaras D. "Global Oncology Access - The Relative Difficulty Of Reimbursement Across Four Health technology Assessment (HTA) Agencies." Value in Health (2020).
- Forsythe, A. "From Regulatory Approval to Reimbursement of Car-Ts: Can Real World Evidence Help Close The Data Gap?" Oral Presentation presented at the 1st European CAR T Cell Meeting of the European Heamatology Association, Paris, France, February 15, 2019.
- Richard ME , Harricharan S , Forsythe A. "Always up-to-Date Systematic Literature Reviews (SLRS Now a Reality - an Example in NEWLY Diagnosed Acute Myeloid Leukemia (ND AML) and Relapsed/Refractory Acute Lymphoblastic Leukemia (RR ALL)." Value in Health (2020) – Accepted for poster presentation at ISPOR EU 2020.