The Center for Cancer Systems Pharmacology

Research

CRS Bibliography

1.
Aboy M, Crespo C, Stern A. Beyond the 510(k): The regulation of novel moderate-risk medical devices, intellectual property considerations, and innovation incentives in the FDA’s De Novo pathway. NPJ Digit Med. 2024 Feb 8;7(1):29. PMCID: PMC10853500
1.
Campbell CM, Webster C, Parisi M, Sherafat-Kazemzadeh R, Braid J, Switzer T, Alves Fávaro M, Sassano C, Coravos A. An aligned framework of actively collected and passively monitored clinical outcome assessments (COAs) for measure selection. NPJ Digit Med. 2024 Mar 16;7(1):71. PMCID: PMC10944461
1.
Choi A, Lee H, Jeong HE, Lee SY, Kwon JS, Han JY, Choe YJ, Shin JY. Association between exposure to antibiotics during pregnancy or early infancy and risk of autism spectrum disorder, intellectual disorder, language disorder, and epilepsy in children: population based cohort study. BMJ. 2024 May 22;385:e076885. PMCID: PMC11109903
1.
Elsallab M, Bourgeois F, Maus MV. National Survey of FACT-Accredited Cell Processing Facilities: Assessing Preparedness for Local Manufacturing of Immune Effector Cells. Transplant Cell Ther. 2024 Mar 16;S2666-6367(24)00289–6. PMID: 38494077
1.
Gardner RA, White C, Elsallab M, Farnia S, Fraint E, Grilley B, Bateman-House A, Grupp SA, Kenderian S, Locke FL, Nikiforow S, Oluwole OO, Rouce RH, Spiegel J, Shah NN, Sharma A, Komanduri K, Gill S. ACT To Sustain: Adoptive Cell Therapy To Sustain Access to Non-Commercialized Genetically Modified Cell Therapies. Transplant Cell Ther. 2024 Aug;30(8):776–787. PMCID: PMC11296902
1.
Guo X, Bourgeois FT, Cai T. Quantifying proportion of treatment effect by surrogate endpoint under heterogeneity. Stat Methods Med Res. 2024 May 8;9622802241247720. PMID: 38717356
1.
Marra C, Stern AD. Tepid Uptake of Digital Health Technologies in Clinical Trials by Pharmaceutical and Medical Device Firms. Clin Pharmacol Ther. 2024 Feb 2; PMID: 38308421
1.
Masanneck L, Stern AD. Tracing Digital Therapeutics Research Across Medical Specialties: Evidence from ClinicalTrails.gov. Clin Pharmacol Ther. 2024 Apr 2; PMID: 38563641
1.
Ryu JH, Choi A, Woo J, Lee H, Kim J, Yoo J, Shin JY. Cardiovascular Safety of COVID-19 Vaccination in Patients With Cancer: A Self-Controlled Case Series Study in Korea. J Korean Med Sci. 2024 Jun 24;39(24):e190. PMCID: PMC11196855
1.
Sinha MS, Stern AD, Rai AK. Four Decades of Orphan Drugs and Priorities for the Future. N Engl J Med. 2024 Jul 11;391(2):100–102. PMID: 38973724
1.
Tang M, Mishuris RG, Payvandi L, Stern AD. Differences in Care Team Response to Patient Portal Messages by Patient Race and Ethnicity. JAMA Netw Open. 2024 Mar 4;7(3):e242618. PMCID: PMC10949096
1.
Tiberi S, Meili J, Cai P, Soneson C, He D, Sarkar H, Avalos-Pacheco A, Patro R, Robinson MD. DifferentialRegulation: a Bayesian hierarchical approach to identify differentially regulated genes. Biostatistics. 2024 Jun 17;kxae017. PMID: 38887902

1.
Arfè A, Narang C, DuBois SG, Reaman G, Bourgeois FT. Clinical development of new drugs for adults and children with cancer, 2010-2020. J Natl Cancer Inst. 2023 May 12;djad082. PMCID: PMC10407707
1.
Avalos-Pacheco A, Ventz S, Arfè A, Alexander BM, Rahman R, Wen PY, Trippa L. Validation of Predictive Analyses for Interim Decisions in Clinical Trials. JCO Precis Oncol. 2023 Feb;7:e2200606. PMCID: PMC10166373
1.
Bourgeois FT, Espinoza JC. Advancing Equity in Medical Device Development for Children. JAMA Pediatr. 2023 Jun 1;177(6):561–562. PMID: 37093608
1.
Brönneke JB, Herr A, Reif S, Stern AD. Dynamic HTA for digital health solutions: opportunities and challenges for patient-centered evaluation. Int J Technol Assess Health Care. 2023 Nov 17;39(1):e72. PMID: 37973549
1.
Elsallab M, Maus MV. Expanding access to CAR T cell therapies through local manufacturing. Nat Biotechnol. 2023 Oct 26; PMID: 37884746
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Elsallab M, Ellithi M, Hempel S, Abdel-Azim H, Abou-El-Enein M. Long-term response to autologous anti-CD19 chimeric antigen receptor T cells in relapsed or refractory B cell acute lymphoblastic leukemia: a systematic review and meta-analysis. Cancer Gene Ther. 2023 Feb 7; PMCID: PMC10281866
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Everhart AO. Time to publication of cost-effectiveness analyses for medical devices. Am J Manag Care. 2023 May;29(5):265–268. PMCID: PMC10214008
1.
Everhart AO, Sen S, Stern AD, Zhu Y, Karaca-Mandic P. Association Between Regulatory Submission Characteristics and Recalls of Medical Devices Receiving 510(k) Clearance. JAMA. 2023 Jan 10;329(2):144–156. PMCID: PMC9857565
1.
Gressler LE, Avila-Tang E, Mao J, Avalos-Pacheco A, Shaya FT, Torosyan Y, Liebeskind A, Kinard M, Mack CD, Normand SL, Ritchey ME, Marinac-Dabic D. Data sources and applied methods for paclitaxel safety signal discernment. Front Cardiovasc Med. 2023;10:1331142. PMCID: PMC10920218
1.
Hwang TJ, Reaman GH, Bourgeois FT. Clinical trials for paediatric cancers under new legislation in the USA. Lancet Child Adolesc Health. 2023 Jul;7(7):e13. PMID: 37349019
1.
Kath J, Du W, Martini S, Elsallab M, Franke C, Hartmann L, Drosdek V, Glaser V, Stein M, Schmueck-Henneresse M, Reinke P, Volk HD, Abou-El-Enein M, Wagner DL. CAR NK-92 cell-mediated depletion of residual TCR+ cells for ultrapure allogeneic TCR-deleted CAR T-cell products. Blood Adv. 2023 Aug 8;7(15):4124–4134. PMCID: PMC10388728
1.
Knox RP, Wang J, Feldman WB, Kesselheim AS, Sarpatwari A. Outcomes of the 340B Drug Pricing Program: A Scoping Review. JAMA Health Forum. 2023 Nov 3;4(11):e233716. PMCID: PMC10665972
1.
Lee LK, Narang C, Rees CA, Thiagarajan RR, Melvin P, Ward V, Bourgeois FT. Reporting and Representation of Participant Race and Ethnicity in National Institutes of Health-Funded Pediatric Clinical Trials. JAMA Netw Open. 2023 Aug 1;6(8):e2331316. PMCID: PMC10469249
1.
Masanneck L, Gieseler P, Gordon WJ, Meuth SG, Stern AD. Evidence from ClinicalTrials.gov on the growth of Digital Health Technologies in neurology trials. NPJ Digit Med. 2023 Feb 10;6(1):23. PMCID: PMC9918454
1.
Olsen A, Beall RF, Knox RP, Tu SS, Kesselheim AS, Feldman WB. Patents and regulatory exclusivities on FDA-approved insulin products: A longitudinal database study, 1986-2019. PLoS Med. 2023 Nov;20(11):e1004309. PMCID: PMC10653475
1.
Ong MS, Wu AC, Bourgeois FT. Enrollment of Pediatric Patients in COVID-19 Interventional Trials. JAMA Health Forum. 2023 Nov 3;4(11):e233939. PMCID: PMC10665967
1.
Quazi S, Narang C, Espinoza JC, Bourgeois FT. Characteristics and Results of Pediatric Medical Device Studies: 2017-2022. Pediatrics. 2023 Aug 11;e2022059842. PMID: 37565273
1.
Raab R, Küderle A, Zakreuskaya A, Stern AD, Klucken J, Kaissis G, Rueckert D, Boll S, Eils R, Wagener H, Eskofier BM. Federated electronic health records for the European Health Data Space. Lancet Digit Health. 2023 Sep 21;S2589-7500(23)00156–5. PMID: 37741765
1.
Tang M, Sharma Y, Goldsack JC, Stern AD. Building the Business Case for an Inclusive Approach to Digital Health Measurement With a Web App (Market Opportunity Calculator): Instrument Development Study. JMIR Form Res. 2023 Jul 26;7:e45713. PMCID: PMC10413230
1.
Tang M, Nakamoto CH, Stern AD, Zubizarreta JR, Marcondes FO, Uscher-Pines L, Schwamm LH, Mehrotra A. Effects of Remote Patient Monitoring Use on Care Outcomes Among Medicare Patients With Hypertension : An Observational Study. Ann Intern Med. 2023 Nov;176(11):1465–1475. PMID: 37931262
1.
Toce MS, Michelson KA, Hudgins JD, Hadland SE, Olson KL, Monuteaux MC, Bourgeois FT. Association of Prescription Drug Monitoring Programs With Opioid Prescribing and Overdose in Adolescents and Young Adults. Ann Emerg Med. 2023 Apr;81(4):429–437. PMCID: PMC10091852
1.
Toce MS, Michelson KA, Hudgins JD, Olson KL, Monuteaux MC, Bourgeois FT. Association of prescription drug monitoring programs with benzodiazepine prescription dispensation and overdose in adolescents and young adults. Clin Toxicol (Phila). 2023 Apr;61(4):234–240. PMID: 36919488
1.
van Kessel R, Srivastava D, Kyriopoulos I, Monti G, Novillo-Ortiz D, Milman R, Zhang-Czabanowski WW, Nasi G, Stern AD, Wharton G, Mossialos E. Digital Health Reimbursement Strategies of 8 European Countries and Israel: Scoping Review and Policy Mapping. JMIR Mhealth Uhealth. 2023 Sep 29;11:e49003. PMCID: PMC10576236
1.
Wang X, Ayakulangara Panickan V, Cai T, Xiong X, Cho K, Cai T, Bourgeois FT. Endovascular Aneurysm Repair Devices as a Use Case for Postmarketing Surveillance of Medical Devices. JAMA Intern Med. 2023 Aug 21;e233562. PMCID: PMC10442779

1.
Khorrami P, Sinha MS, Bhanja A, Allen HL, Kesselheim AS, Sommers BD. Differences in Diabetic Prescription Drug Utilization and Costs Among Patients With Diabetes Enrolled in Colorado Marketplace and Medicaid Plans, 2014-2015. JAMA Netw Open. 2022 Jan 4;5(1):e2140371. PMCID: PMC8760612
1.
Knox R, Curfman G. The Humira patent thicket, the Noerr-Pennington doctrine and antitrust’s patent problem. Nat Biotechnol. 2022 Dec;40(12):1761–1763. PMID: 36477501
1.
Knox RP, Kesselheim AS, Sarpatwari A. Risks to the 340B Drug Pricing Program Related to Manufacturer Restrictions on Drug Availability. JAMA. 2022 May 3;327(17):1647–1648. PMID: 35426909
1.
Nader JH, Bourgeois F, Bagatell R, Moreno L, Pearson ADJ, DuBois SG. Systematic review of clinical drug development activities for neuroblastoma from 2011 to 2020. Pediatr Blood Cancer. 2022 Dec 2;e30106. PMID: 36458672
1.
Russo M, Scarpa B. Learning in Medicine: The Importance of Statistical Thinking. Methods Mol Biol. 2022;2486:215–232. PMID: 35437725
1.
Tang M, Nakamoto CH, Stern AD, Mehrotra A. Trends in Remote Patient Monitoring Use in Traditional Medicare. JAMA Intern Med. 2022 Sep 1;182(9):1005–1006. PMCID: PMC9344385
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Torous J, Stern AD, Bourgeois FT. Regulatory considerations to keep pace with innovation in digital health products. NPJ Digit Med. 2022 Aug 19;5(1):121. PMCID: PMC9390099
1.
Wunnava S, Miller TA, Bourgeois FT. Improving FDA postmarket adverse event reporting for medical devices. BMJ Evid Based Med. 2022 Feb 17;bmjebm-2021-111870. PMID: 35177481
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Wunnava S, Miller TA, Narang C, Nathan M, Bourgeois FT. US Food and Drug Administration Approval of High-risk Cardiovascular Devices for Use in Children and Adolescents, 1977-2021. JAMA. 2022 Aug 9;328(6):580–582. PMCID: PMC9364120

1.
Antonini MJ, Plana D, Srinivasan S, Atta L, Achanta A, Yang H, Cramer AK, Freake J, Sinha MS, Yu SH, LeBoeuf NR, Linville-Engler B, Sorger PK. A crisis-responsive framework for medical device development applied to the COVID-19 pandemic. Front Digit Health. 2021 Mar;3:617106. PMCID: PMC8064560
1.
Arfè A, Silverman LB, Bourgeois F. Master Protocols and Adaptive Trial Designs to Develop Tumor-Agnostic Drugs for Children: Essential Tools in the Era of the Research to Accelerate Cure and Equity Act. JAMA Oncol. 2021 Jun 10; PMID: 34110376
1.
Barenie RE, Sinha MS, Kesselheim AS. Factors Affecting Buprenorphine Utilization and Spending in Medicaid, 2002-2018. Value Health. 2021 Feb;24(2):182–187. PMID: 33518024
1.
Bourgeois FT, Gutiérrez-Sacristán A, Keller MS, Liu M, Hong C, Bonzel CL, Tan ALM, Aronow BJ, Boeker M, Booth J, Cruz Rojo J, Devkota B, García Barrio N, Gehlenborg N, Geva A, Hanauer DA, Hutch MR, Issitt RW, Klann JG, Luo Y, Mandl KD, Mao C, Moal B, Moshal KL, Murphy SN, Neuraz A, Ngiam KY, Omenn GS, Patel LP, Jiménez MP, Sebire NJ, Balazote PS, Serret-Larmande A, South AM, Spiridou A, Taylor DM, Tippmann P, Visweswaran S, Weber GM, Kohane IS, Cai T, Avillach P, Consortium for Clinical Characterization of COVID-19 by EHR (4CE). International Analysis of Electronic Health Records of Children and Youth Hospitalized With COVID-19 Infection in 6 Countries. JAMA Netw Open. 2021 Jun 1;4(6):e2112596. PMCID: PMC8196345
1.
Bourgeois FT, Avillach P, Turner MA. The urgent need for research coordination to advance knowledge on COVID-19 in children. Pediatr Res. 2021 Aug;90(2):250–252. PMCID: PMC7656217
1.
Bresler J, Sinha MS. The Other Three Waves: Re-assessing the Impact of Industry-Prescriber Relations on the Opioid Crisis. J Leg Med. 2021 Jun;41(1–2):47–81. PMID: 34296970
1.
Bui ATN, Yu Z, Lee K, Li SJ, Tsiaras WG, Yu SH, LeBoeuf NR, Mostaghimi A. A pilot study of the impact of facial skin protectants on qualitative fit testing of N95 masks. J Am Acad Dermatol. 2021 Feb;84(2):554–556. PMCID: PMC7313513
1.
Carmody S, Coravos A, Fahs G, Hatch A, Medina J, Woods B, Corman J. Building resilient medical technology supply chains with a software bill of materials. NPJ Digit Med. 2021 Feb 23;4(1):34. PMCID: PMC7902663
1.
Cherla A, Naci H, Kesselheim AS, Gyawali B, Mossialos E. Assessment of Coverage in England of Cancer Drugs Qualifying for US Food and Drug Administration Accelerated Approval. JAMA Intern Med. 2021 Apr 1;181(4):490–498. PMCID: PMC7900925
1.
Cramer AK, Plana D, Yang H, Carmack MM, Tian E, Sinha MS, Krikorian D, Turner D, Mo J, Li J, Gupta R, Manning H, Bourgeois FT, Yu SH, Sorger PK, LeBoeuf NR. Analysis of SteraMist ionized hydrogen peroxide technology in the sterilization of N95 respirators and other PPE. Sci Rep. 2021 Jan 21;11(1):2051. PMCID: PMC7819989
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Gordon WJ, Coravos AR, Stern AD. Ushering in safe, effective, secure, and ethical medicine in the digital era. NPJ Digit Med. 2021 Mar 25;4(1):56. PMCID: PMC7994831
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Gyawali B, Kesselheim AS. FDA approval standards for anticancer agents - lessons from two recent approvals in breast cancer. Nat Rev Clin Oncol. 2021 Jul;18(7):397–398. PMID: 33767454
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Kassamali B, Yu Z, Davis C, Carmack M, Bui ATN, Said JT, Plana D, Yang H, Sorger P, LeBoeuf NR, LaChance AH. Conversion of existing UVB phototherapy units to UVC germicidal chambers for N95 decontamination: lessons learned. Photobiomodul Photomed Laser Surg. 2021 Feb;39(2):83–85. PMCID: PMC8106246
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Kothakonda A, Atta L, Plana D, Ward F, Davis C, Cramer A, Moran R, Freake J, Tian E, Mazor O, Gorelik P, Van C, Hansen C, Yang H, Li Y, Sinha MS, Li J, Yu SH, LeBoeuf NR, Sorger PK. De novo powered air-purifying respirator design and fabrication for pandemic response. Front Bioeng Biotechnol. 2021 Sep 6;9:690905. PMCID: PMC8450396
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McAvoy M, Bui ATN, Hansen C, Plana D, Said JT, Yu Z, Yang H, Freake J, Van C, Krikorian D, Cramer A, Smith L, Jiang L, Lee KJ, Li SJ, Beller B, Huggins K, Short MP, Yu SH, Mostaghimi A, Sorger PK, LeBoeuf NR. 3D Printed frames to enable reuse and improve the fit of N95 and KN95 respirators. BMC Biomed Eng. 2021 Jun 7;3(1):10. PMCID: PMC8182357
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Mecklai K, Smith N, Stern AD, Kramer DB. Remote Patient Monitoring - Overdue or Overused? N Engl J Med. 2021 Apr 15;384(15):1384–1386. PMID: 33853209
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Plana D, Tian E, Cramer AK, Yang H, Carmack MM, Sinha MS, Bourgeois FT, Yu SH, Masse P, Boyer J, Kim M, Mo J, LeBoeuf NR, Li J, Sorger PK. Assessing the filtration efficiency and regulatory status of N95s and nontraditional filtering face-piece respirators available during the COVID-19 pandemic. BMC Infect Dis. 2021 Jul 29;21(1):712. PMCID: PMC8319695
1.
Rome BN, Lee CC, Gagne JJ, Kesselheim AS. Factors Associated With Generic Drug Uptake in the United States, 2012 to 2017. Value Health. 2021 Jun;24(6):804–811. PMID: 34119078
1.
Sahragardjoonegani B, Beall RF, Kesselheim AS, Hollis A. Repurposing existing drugs for new uses: a cohort study of the frequency of FDA-granted new indication exclusivities since 1997. J Pharm Policy Pract. 2021 Jan 4;14(1):3. PMCID: PMC7780607
1.
Srivastava A, Bourgeois FT. Evaluation of Publication of Pediatric Drug Trials. JAMA Netw Open. 2021 Apr 1;4(4):e215829. PMCID: PMC8047779
1.
Stern AD, Chen JL, Ouellet M, Trusheim MR, El-Kilani Z, Jessup A, Berndt ER. Biosimilars And Follow-On Products In The United States: Adoption, Prices, And Users. Health Aff (Millwood). 2021 Jun;40(6):989–999. PMID: 34097520
1.
Wang X, Cai T, Tian L, Bourgeois F, Parast L. Quantifying the feasibility of shortening clinical trial duration using surrogate markers. Stat Med. 2021 Sep 2; PMCID: PMC8595715

1.
Arfè A, Fell G, Alexander B, Awad MM, Rodig SJ, Trippa L, Schoenfeld JD. Meta-Analysis of PD-L1 Expression As a Predictor of Survival After Checkpoint Blockade. JCO Precis Oncol. 2020 Nov;4:1196–1206. PMID: 35050777
1.
Arfè A, Alexander B, Trippa L. Optimality of testing procedures for survival data in the nonproportional hazards setting. Biometrics. 2020 Jun 14; PMID: 32535892
1.
Avorn J, Kesselheim A. Regulatory Decision-making on COVID-19 Vaccines During a Public Health Emergency. JAMA. 2020 Oct 6;324(13):1284–1285. PMID: 32870268
1.
Avorn J, Kesselheim AS. Up Is Down - Pharmaceutical Industry Caution vs. Federal Acceleration of Covid-19 Vaccine Approval. N Engl J Med. 2020 Oct 29;383(18):1706–1708. PMID: 32931162
1.
Barenie RE, Avorn J, Tessema FA, Kesselheim AS. Public funding for transformative drugs: the case of sofosbuvir. Drug Discov Today. 2020 Oct 1; PMCID: PMC7528745
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Barenie RE, Gagne JJ, Kesselheim AS, Pawar A, Tong A, Luo J, Bateman BT. Rates and Costs of Dispensing Naloxone to Patients at High Risk for Opioid Overdose in the United States, 2014-2018. Drug Saf. 2020;43(7):669–675. PMID: 32180134
1.
Beletsky L, Seymour S, Kang S, Siegel Z, Sinha MS, Marino R, Dave A, Freifeld C. Fentanyl panic goes viral: The spread of misinformation about overdose risk from casual contact with fentanyl in mainstream and social media. Int J Drug Policy. 2020 Sep 16;86:102951. PMCID: PMC7492952
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Carmack M, Hwang T, Bourgeois FT. Pediatric Drug Policies Supporting Safe And Effective Use Of Therapeutics In Children: A Systematic Analysis. Health Aff (Millwood). 2020 Oct;39(10):1799–1805. PMID: 33017255
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Conti RM, David FS. Public research funding and pharmaceutical prices: do Americans pay twice for drugs? F1000Res. 2020 Jul 15;9:707. PMCID: PMC7642989
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Coravos A, Doerr M, Goldsack J, Manta C, Shervey M, Woods B, Wood WA. Modernizing and designing evaluation frameworks for connected sensor technologies in medicine. NPJ Digit Med. 2020;3:37. PMCID: PMC7070075
1.
Cramer A, Tian E, Galanek M, Lamere E, Li J, Gupta R, Short M. Assessment of the Qualitative Fit Test and Quantitative Single-Pass Filtration Efficiency of Disposable N95 Masks Following Gamma Irradiation. JAMA Netw Open. 2020 May 1;3(5):e209961. PMCID: PMC7251444
1.
D’Andrea E, Kesselheim AS, Franklin JM, Jung EH, Hey SP, Patorno E. Heterogeneity of antidiabetic treatment effect on the risk of major adverse cardiovascular events in type 2 diabetes: a systematic review and meta-analysis. Cardiovasc Diabetol. 2020 Sep 29;19(1):154. PMCID: PMC7525990
1.
Darrow JJ, Kesselheim AS. Incentivizing Antibiotic Development: Why Isn’t the Generating Antibiotic Incentives Now (GAIN) Act Working? Open Forum Infect Dis. 2020 Jan;7(1):ofaa001. PMCID: PMC6986775
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Darrow JJ, Najafzadeh M, Stefanini K, Kesselheim AS. Regulatory approval characteristics of antimicrobial versus non-antimicrobial products, 1984-2018: an evaluation of Food and Drug Administration flexibilities. Lancet Infect Dis. 2020;20(7):e159–e164. PMID: 32502431
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Darrow JJ, Chong JE, Kesselheim AS. Reconsidering the scope of US state laws allowing pharmacist substitution of generic drugs. BMJ. 2020 Jun 23;369:m2236. PMID: 32576554
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Darrow JJ, Avorn J, Kesselheim AS. FDA Approval and Regulation of Pharmaceuticals, 1983-2018. JAMA. 2020 Jan 14;323(2):164–176. PMID: 31935033
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Dave CV, Sinha MS, Beall RF, Kesselheim AS. Estimating The Cost Of Delayed Generic Drug Entry To Medicaid. Health Aff (Millwood). 2020 Jun;39(6):1011–1017. PMID: 32479219
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Druedahl LC, Almarsdóttir AB, Kälvemark Sporrong S, De Bruin ML, Hoogland H, Minssen T, van de Weert M, Kesselheim AS, Sarpatwari A. A qualitative study of biosimilar manufacturer and regulator perceptions on intellectual property and abbreviated approval pathways. Nat Biotechnol. 2020;38(11):1253–1256. PMID: 33144720
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Feldman WB, Gagne JJ, Kesselheim AS. Trends in Medicare Part D Inhaler Spending, 2012-2018. Ann Am Thorac Soc. 2020 Nov 18; PMCID: PMC7919157
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Fralick M, Gagne JJ, Patorno E, Levin R, Kesselheim AS. Using Data From Routine Care to Estimate the Effectiveness and Potential Limitations of Outcomes-Based Contracts for Diabetes Medications. Value Health. 2020;23(4):434–440. PMID: 32327160
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Gerke S, Stern AD, Minssen T. Germany’s digital health reforms in the COVID-19 era: lessons and opportunities for other countries. NPJ Digit Med. 2020 Jul 10;3:94. PMCID: PMC7351985
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Godfrey A, Goldsack JC, Tenaerts P, Coravos A, Aranda C, Hussain A, Barreto ME, Young F, Vitorio R. BioMeT and Algorithm Challenges: A Proposed Digital Standardized Evaluation Framework. IEEE J Transl Eng Health Med. 2020;8:0700108. PMCID: PMC7292480
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Goldsack JC, Coravos A, Bakker JP, Bent B, Dowling AV, Fitzer-Attas C, Godfrey A, Godino JG, Gujar N, Izmailova E, Manta C, Peterson B, Vandendriessche B, Wood WA, Wang KW, Dunn J. Verification, analytical validation, and clinical validation (V3): the foundation of determining fit-for-purpose for Biometric Monitoring Technologies (BioMeTs). NPJ Digit Med. 2020;3:55. PMCID: PMC7156507
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Gunnarsdóttir HD, Sinha MS, Gerke S, Minssen T. Applying the proportionality principle to COVID-19 antibody testing. J Law Biosci. 2020 Jun;7(1):lsaa058. PMCID: PMC7454743
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Gyawali B, Hey SP, Kesselheim AS. Evaluating the evidence behind the surrogate measures included in the FDA’s table of surrogate endpoints as supporting approval of cancer drugs. EClinicalMedicine. 2020 Apr;21:100332. PMCID: PMC7201012
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Gyawali B, D’Andrea E, Franklin JM, Kesselheim AS. Response Rates and Durations of Response for Biomarker-Based Cancer Drugs in Nonrandomized Versus Randomized Trials. J Natl Compr Canc Netw. 2020 Jan;18(1):36–43. PMID: 31910385
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Hey SP, Gyawali B, D’Andrea E, Kanagaraj M, Franklin JM, Kesselheim AS. A Systematic Review and Meta-Analysis of Bevacizumab in First-Line Metastatic Breast Cancer: Lessons for Research and Regulatory Enterprises. J Natl Cancer Inst. 2020 Apr 1;112(4):335–342. PMCID: PMC7156929
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Hey SP, Kesselheim AS, Patel P, Mehrotra P, Powers JH. US Food and Drug Administration Recommendations on the Use of Surrogate Measures as End Points in New Anti-infective Drug Approvals. JAMA Intern Med. 2020 Jan 1;180(1):131–138. PMID: 31710344
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Hey SP, Gerlach CV, Dunlap G, Prasad V, Kesselheim AS. The evidence landscape in precision medicine. Sci Transl Med. 2020 Apr 22;12(540). PMID: 32321867
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Hong P, Kesselheim AS, Sarpatwari A. Transformative Models to Promote Prescription Drug Innovation and Access: A Landscape Analysis. Yale journal of health policy, law, and ethics. 2020;19(2):56-.
1.
Hwang TJ, Ross JS, Vokinger KN, Kesselheim AS. Association between FDA and EMA expedited approval programs and therapeutic value of new medicines: retrospective cohort study. BMJ. 2020 Oct 7;371:m3434. PMCID: PMC7537471
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Hwang TJ, Randolph AG, Bourgeois FT. Inclusion of Children in Clinical Trials of Treatments for Coronavirus Disease 2019 (COVID-19). JAMA Pediatr. 2020 Sep 1;174(9):825–826. PMID: 32379296
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Jung EH, Sarpatwari A, Kesselheim AS. Novelty of Active Ingredients in High-Cost Brand-Name Drugs. J Gen Intern Med. 2020 Jul;35(7):2219–2221. PMCID: PMC7352024
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Kesselheim AS, Darrow JJ, Kulldorff M, Brown BL, Mitra-Majumdar M, Lee CC, Moneer O, Avorn J. An Overview Of Vaccine Development, Approval, And Regulation, With Implications For COVID-19. Health Aff (Millwood). 2020 Nov 19;101377hlthaff202001620. PMID: 33211535
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Kurzrock R, Kantarjian HM, Kesselheim AS, Sigal EV. New drug approvals in oncology. Nat Rev Clin Oncol. 2020;17(3):140–146. PMID: 32020042
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Lauenroth VD, Kesselheim AS, Sarpatwari A, Stern AD. Lessons From The Impact Of Price Regulation On The Pricing Of Anticancer Drugs In Germany. Health Aff (Millwood). 2020 Jul;39(7):1185–1193. PMID: 32634355
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Lee CC, Darrow JJ, Avorn J, Kesselheim AS. Origins and Ownership of Remdesivir: Implications for Pricing. J Law Med Ethics. 2020;48(3):613–618. PMID: 33021169
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Lee CC, Kesselheim AS, Sarpatwari A. Clinical Development Times for Biosimilars in the United States. Mayo Clin Proc. 2020;95(10):2152–2154. PMID: 33012346
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Lee CC, Kesselheim AS, Avorn J. Variations in Generic Combination Opioid Use Across State Medicaid Programs. J Gen Intern Med. 2020 Nov 2; PMCID: PMC8481421
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Lin KJ, Dvorin E, Kesselheim AS. Prescribing systemic steroids for acute respiratory tract infections in United States outpatient settings: A nationwide population-based cohort study. PLoS Med. 2020;17(3):e1003058. PMCID: PMC7108689
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Lite S, Gordon WJ, Stern AD. Association of the Meaningful Use Electronic Health Record Incentive Program With Health Information Technology Venture Capital Funding. JAMA Netw Open. 2020 02;3(3):e201402. PMCID: PMC7093764
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Liu M, Caputi TL, Dredze M, Kesselheim AS, Ayers JW. Internet Searches for Unproven COVID-19 Therapies in the United States. JAMA Intern Med. 2020 Aug 1;180(8):1116–1118. PMCID: PMC7191468
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Liu M, MacKenna B, Feldman WB, Walker AJ, Avorn J, Kesselheim AS, Goldacre B. Projected spending for brand-name drugs in English primary care given US prices: a cross-sectional study. J R Soc Med. 2020;113(9):350–359. PMCID: PMC7488930
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Luo J, Kesselheim AS, Sarpatwari A. Insulin access and affordability in the USA: anticipating the first interchangeable insulin product. Lancet Diabetes Endocrinol. 2020;8(5):360–362. PMCID: PMC9036474
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Manta C, Jain SS, Coravos A, Mendelsohn D, Izmailova ES. An Evaluation of Biometric Monitoring Technologies for Vital Signs in the Era of COVID-19. Clin Transl Sci. 2020;13(6):1034–1044. PMCID: PMC7719373
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Marra C, Chen JL, Coravos A, Stern AD. Quantifying the use of connected digital products in clinical research. NPJ Digit Med. 2020;3:50. PMCID: PMC7125096
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Molto C, Hwang TJ, Borrell M, Andres M, Gich I, Barnadas A, Amir E, Kesselheim AS, Tibau A. Clinical benefit and cost of breakthrough cancer drugs approved by the US Food and Drug Administration. Cancer. 2020 Oct 1;126(19):4390–4399. PMID: 32697362
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Mostaghimi A, Antonini MJ, Plana D, Anderson PD, Beller B, Boyer EW, Fannin A, Freake J, Oakley R, Sinha MS, Smith L, Van C, Yang H, Sorger PK, LeBoeuf NR, Yu SH. Regulatory and safety considerations in deploying a locally fabricated, reusable face shield in a hospital responding to the COVID-19 pandemic. Med (N Y). 2020 Dec 18;1(1):139-151.e4. PMCID: PMC7304404
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Naci H, Kesselheim AS. Specialty Drugs - A Distinctly American Phenomenon. N Engl J Med. 2020 Jun 4;382(23):2179–2181. PMID: 32492301

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Adaptive Platform Trials Coalition. Adaptive platform trials: definition, design, conduct and reporting considerations. Nat Rev Drug Discov. 2019 Oct;18(10):797–807. PMID: 31462747
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Bakker JP, Goldsack JC, Clarke M, Coravos A, Geoghegan C, Godfrey A, Heasley MG, Karlin DR, Manta C, Peterson B, Ramirez E, Sheth N, Bruno A, Bullis E, Wareham K, Zimmerman N, Forrest A, Wood WA. A systematic review of feasibility studies promoting the use of mobile technologies in clinical research. NPJ Digit Med. 2019;2:47. PMCID: PMC6554345
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Beall RF, Darrow JJ, Kesselheim AS. Approximating Future Generic Entry for New Drugs. J Law Med Ethics. 2019 Mar;47(1):177–182. PMID: 30994060
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Beall RF, Hwang TJ, Kesselheim AS. Pre-market development times for biologic versus small-molecule drugs. Nat Biotechnol. 2019 Jul;37(7):708–711. PMID: 31213674
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Beall RF, Hwang TJ, Kesselheim AS. Major Events in the Life Course of New Drugs, 2000-2016. N Engl J Med. 2019 Mar 14;380(11):e12. PMID: 30865793
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Beall RF, Kesselheim AS, Sarpatwari A. New Drug Formulations and Their Respective Generic Entry Dates. J Manag Care Spec Pharm. 2019 Feb;25(2):218–224. PMCID: PMC10398232
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Bourgeois FT, Kesselheim AS. Promoting Pediatric Drug Research and Labeling - Outcomes of Legislation. N Engl J Med. 2019 29;381(9):875–881. PMID: 31461599
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Bourgeois FT, Graham DA, Kesselheim AS, Randolph AG. Cost Implications of Escalating Intravenous Acetaminophen Use in Children. JAMA Pediatr. 2019 Mar 11; PMCID: PMC6503505
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Coravos A, Khozin S, Mandl KD. Developing and adopting safe and effective digital biomarkers to improve patient outcomes. NPJ Digit Med. 2019;2(1):14. PMCID: PMC6411051
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Coravos A, Goldsack JC, Karlin DR, Nebeker C, Perakslis E, Zimmerman N, Erb MK. Digital Medicine: A Primer on Measurement. Digit Biomark. 2019;3(2):31–71. PMCID: PMC7015383
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D’Andrea E, Hey SP, Ramirez CL, Kesselheim AS. Assessment of the Role of Niacin in Managing Cardiovascular Disease Outcomes: A Systematic Review and Meta-analysis. JAMA Netw Open. 2019 Apr 5;2(4):e192224. PMCID: PMC6481429
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Darrow JJ, Beall RF, Kesselheim AS. The Generic Drug Industry Embraces a Faster, Cheaper Pathway for Challenging Patents. Appl Health Econ Health Policy. 2019 Feb;17(1):47–54. PMID: 30141133
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Dave CV, Brill G, Kesselheim AS. Changes in Price for Generic Drugs in the USA, 2008-2016. J Gen Intern Med. 2019 Sep;34(9):1677–1679. PMCID: PMC6712128
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Desai RJ, Sarpatwari A, Dejene S, Khan NF, Lii J, Rogers JR, Dutcher SK, Raofi S, Bohn J, Connolly JG, Fischer MA, Kesselheim AS, Gagne JJ. Comparative effectiveness of generic and brand-name medication use: A database study of US health insurance claims. PLoS Med. 2019 Mar;16(3):e1002763. PMCID: PMC6415809
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Feldman WB, Avorn J, Kesselheim AS. Potential Medicare Savings on Inhaler Prescriptions Through the Use of Negotiated Prices and a Defined Formulary. JAMA Intern Med. 2019 Dec 2; PMCID: PMC6902106
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Feldman WB, Hey SP, Franklin JM, Kesselheim AS. Public Approval of Exception From Informed Consent in Emergency Clinical Trials: A Systematic Review of Community Consultation Surveys. JAMA Netw Open. 2019 Jul 3;2(7):e197591. PMCID: PMC6659147
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Fischer MA, Kesselheim AS, Lu Z, Ross KM, Tessema FA, Avorn J. Physician Perceptions of Step Therapy Prescribing Requirements. J Manag Care Spec Pharm. 2019 Nov;25(11):1210–1224. PMCID: PMC10398190
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Fralick M, Kesselheim AS. The U.S. Insulin Crisis - Rationing a Lifesaving Medication Discovered in the 1920s. N Engl J Med. 2019 Nov 7;381(19):1793–1795. PMID: 31693804
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Fralick M, Schneeweiss S, Wallis CJD, Jung EH, Kesselheim AS. Desmopressin and the risk of hyponatremia: A population-based cohort study. PLoS Med. 2019 Oct;16(10):e1002930. PMCID: PMC6802819
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Gordon WJ, Stern AD. Challenges and opportunities in software-driven medical devices. Nat Biomed Eng. 2019 Jul;3(7):493–497. PMID: 31278389
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Growdon ME, Sacks CA, Kesselheim AS, Avorn J. Potential Medicare Savings From Generic Substitution and Therapeutic Interchange of ACE Inhibitors and Angiotensin-II-Receptor Blockers. JAMA Intern Med. 2019 Aug 5; PMCID: PMC6686978
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Gyawali B, Kesselheim AS. The promise of ESCAT: a new system for evaluating cancer drug-target pairs. Nat Rev Clin Oncol. 2019 Mar;16(3):147–148. PMID: 30317250
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Gyawali B, Kesselheim AS. US Food and Drug Administration Approval of New Drugs Based on Noninferiority Trials in Oncology: A Dangerous Precedent? JAMA Oncol. 2019 Mar 28; PMID: 30920591
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Gyawali B, Hey SP, Kesselheim AS. Assessment of the Clinical Benefit of Cancer Drugs Receiving Accelerated Approval. JAMA Intern Med. 2019 Jul 1;179(7):906–913. PMCID: PMC6547118
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Gyawali B, Tessema FA, Jung EH, Kesselheim AS. Assessing the Justification, Funding, Success, and Survival Outcomes of Randomized Noninferiority Trials of Cancer Drugs: A Systematic Review and Pooled Analysis. JAMA Netw Open. 2019 Aug 2;2(8):e199570. PMCID: PMC6724156
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Gyawali B, Bouche G, Pantziarka P, Kesselheim AS, Sarpatwari A. Lung Cancer Survival Gains: Contributions of Academia and Industry. J Law Med Ethics. 2019 Sep;47(3):465–467. PMID: 31560624
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Hey SP, Feldman WB, Jung EH, D’Andrea E, Kesselheim AS. Surrogate Endpoints and Drug Regulation: What Is Needed to Clarify the Evidence. J Law Med Ethics. 2019 Sep;47(3):381–387. PMID: 31560631
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Hey SP, D’Andrea E, Jung EH, Tessema F, Luo J, Gyawali B, Kesselheim AS. Challenges and Opportunities for Biomarker Validation. J Law Med Ethics. 2019 Sep;47(3):357–361. PMID: 31560620
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Hong P, Sarpatwari A, Kesselheim AS. Orphan Drug Designation and Exclusivity for “Same Drugs.” J Law Med Ethics. 2019 Jun;47(2):347–349. PMID: 31298092
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Hudgins JD, Porter JJ, Monuteaux MC, Bourgeois FT. Trends in Opioid Prescribing for Adolescents and Young Adults in Ambulatory Care Settings. Pediatrics. 2019;143(6). PMID: 31138669
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Hwang TJ, Jain N, Lauffenburger JC, Vokinger KN, Kesselheim AS. Analysis of Proposed Medicare Part B to Part D Shift With Associated Changes in Total Spending and Patient Cost-Sharing for Prescription Drugs. JAMA Intern Med. 2019 Jan 14; PMCID: PMC6439690
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Hwang TJ, Kesselheim AS, Vokinger KN. Lifecycle Regulation of Artificial Intelligence- and Machine Learning-Based Software Devices in Medicine. JAMA. 2019 Nov 22; PMID: 31755907
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Hwang TJ, Bourgeois FT, Franklin JM, Kesselheim AS. Impact Of The Priority Review Voucher Program On Drug Development For Rare Pediatric Diseases. Health Aff (Millwood). 2019 Feb;38(2):313–319. PMID: 30715972
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Hwang TJ, Sinha MS, Dave CV, Kesselheim AS. Prescription Opioid Epidemic and Trends in the Clinical Development of New Pain Medications. Mayo Clin Proc. 2019;94(12):2437–2443. PMID: 31685265
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Jiao K, Gupta R, Fox E, Kesselheim A, Ross JS. Characteristics of Recent Generic Drug Approvals by the US Food and Drug Administration. JAMA Netw Open. 2019 Oct 2;2(10):e1913029. PMCID: PMC6804024
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Jung EH, Sarpatwari A, Kesselheim AS, Sinha MS. FDA and EMA Biosimilar Approvals. J Gen Intern Med. 2019 Oct 21; PMCID: PMC7280431
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Kesselheim AS, Sinha MS, Rausch P, Lu Z, Tessema FA, Lappin BM, Zhou EH, Dal Pan GJ, Zwanziger L, Ramanadham A, Loughlin A, Enger C, Avorn J, Campbell EG. Patients’ Knowledge of Key Messaging in Drug Safety Communications for Zolpidem and Eszopiclone: A National Survey. J Law Med Ethics. 2019 Sep;47(3):430–441. PMID: 31560634
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Kesselheim AS, Sinha MS, Campbell EG, Schneeweiss S, Rausch P, Lappin BM, Zhou EH, Avorn J, Dal Pan GJ. Multimodal Analysis of FDA Drug Safety Communications: Lessons from Zolpidem. Drug Saf. 2019 Jul 13; PMID: 31302895
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Kesselheim AS, Woloshin S, Lu Z, Tessema FA, Ross KM, Schwartz LM. Physicians’ Perspectives on FDA Approval Standards and Off-Label Drug Marketing. JAMA Intern Med. 2019 Jan 22; PMCID: PMC6503563
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Khozin S, Coravos A. Decentralized Trials in the Age of Real-World Evidence and Inclusivity in Clinical Investigations. Clin Pharmacol Ther. 2019 Apr 23; PMID: 31013350
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Lee TT, Solomon DH, Kesselheim AS. Fool Me Twice? The Reemergence of Rofecoxib and the Orphan Drug Act. Ann Intern Med. 2019 Oct 1; PMID: 31569216
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Li DG, Najafzadeh M, Kesselheim AS, Mostaghimi A. Spending on World Health Organization essential medicines in Medicare Part D, 2011-15: retrospective cost analysis. BMJ. 2019 17;366:l4257. PMCID: PMC6635813
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Liu S, Kesselheim AS. Experiences With and Challenges Afforded by Expedited Regulatory Pathways. Clin Pharmacol Ther. 2019 Apr;105(4):795–797. PMID: 30883712
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Luo J, Khan NF, Manetti T, Rose J, Kaloghlian A, Gadhe B, Jain SH, Gagne JJ, Kesselheim AS. Implementation of a Health Plan Program for Switching From Analogue to Human Insulin and Glycemic Control Among Medicare Beneficiaries With Type 2 Diabetes. JAMA. 2019 29;321(4):374–384. PMCID: PMC6439763
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Luo J, Kulldorff M, Sarpatwari A, Pawar A, Kesselheim AS. Variation in Prescription Drug Prices by Retail Pharmacy Type: A National Cross-sectional Study. Ann Intern Med. 2019 Oct 1; PMID: 31569218
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Monteleone PP, Banerjee S, Kothapalli P, Stern AD, Fehder D, Ginor R, Vollmar D, Fry ETA, Pirwitz MJ. The Market Reacts Quickly: Changes in Paclitaxel Vascular Device Purchasing Within the Ascension Healthcare System. J Invasive Cardiol. 2019 Oct 15; PMID: 31611426
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Morten CJ, Kesselheim AS, Ross JS. The Supreme Court’s Latest Ruling on Drug Liability and its Implications for Future Failure-to-Warn Litigation. J Law Med Ethics. 2019 Dec;47(4):783–787. PMID: 31957583
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Nayak RK, Avorn J, Kesselheim AS. Public sector financial support for late stage discovery of new drugs in the United States: cohort study. BMJ. 2019 Oct 23;367:l5766. PMCID: PMC6812612

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Kaplan AV, Stern AD. The Central and Unacknowledged Role of the US Food and Drug Administration in the Design and Execution of Medical Device Pivotal Trials. JAMA Cardiol. 2018 Jan 1;3(1):5–6. PMID: 29094158
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Stern AD, Alexander BM, Chandra A. Innovation Incentives and Biomarkers. Clin Pharmacol Ther. 2018 Jan;103(1):34–36. PMID: 29034452

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Palmer AC, Sorger PK. Combination cancer therapy can confer benefit via patient-to-patient variability without drug additivity or synergy. Cell. 2017 Dec 14;171(7):1678-1691.e13. PMCID: PMC5741091