Bibliography of Health Informatics and Therapeutic Studies

2025

7. Wunnava S, Miller TA, Nathan M, Bourgeois FT. FDA Approval of Cardiac Valve Devices Implanted in a National Cohort of Pediatric Patients, 2016-2022. JAMA Pediatr. 2025 Mar 24;e250131. PMCID: PMC11933990

6. Elsallab M, Gillner S, Bourgeois FT. Comparison of Clinical Evidence Submitted to the FDA and EMA for Cell and Gene Therapies. JAMA Intern Med. 2025 Feb 3;e247569. PMCID: PMC11791768

5. Kim JH, Choi A, Bae G, Joo EJ, Choi MJ, Huh K, Lee H, Kim J, Kim DH, Yoo MG, Jo IU, Lee PH, Lee GW, Jung HS, Jung J, Shin JY. Selected Acute Safety Events Following the Use of Nirmatrelvir/Ritonavir or Molnupiravir for COVID-19: A Nationwide Cohort Study in South Korea. Clin Pharmacol Ther. 2025 Feb;117(2):445–453. PMID: 39377297

4. Pellen C, Munung NS, Armond AC, Kulp D, Mansmann U, Siebert M, Naudet F. Data management and sharing. J Clin Epidemiol. 2025 Jan 20;180:111680. PMID: 39842522

3. Oh J, Cho Y, Han JY, Choi A, Shin JY. Real-World Utilization Patterns of Oral Corticosteroids During Pregnancy: A Nationwide Population-Based Study in Korea. J Korean Med Sci. 2025 Jan 13;40(2):e80. PMCID: PMC11729232

2. Cho Y, Choi EY, Choi A, Han JY, Ye BD, Kim JH, Shin JY. Anti-Tumor Necrosis Factor Therapy and the Risk of Gestational Diabetes in Pregnant Women With Inflammatory Bowel Disease. Am J Gastroenterol. 2025 Jan 1;120(1):241–250. PMID: 39315687

1. Knox RP, Sarpatwari A. The 340B Drug Pricing Program: Administration, Litigation, and Reform. OKLAHOMA LAW REVIEW. 2025;77.

2024

23. Brewster RCL, Nagy M, Wunnava S, Bourgeois FT. US FDA Approval of Pediatric Artificial Intelligence and Machine Learning-Enabled Medical Devices. JAMA Pediatr. 2024 Dec 16; PMID: 39680415

22. Ellithi M, Elsallab M, Lunning MA, Holstein SA, Sharma S, Trinh JQ, Ma J, Maus MV, Frigault MJ, D’Angelo CR. Neurotoxicity and Rare Adverse Events in BCMA-Directed CAR T Cell Therapy: A Comprehensive Analysis of Real-World FAERS Data. Transplant Cell Ther. 2024 Dec 12;S2666-6367(24)00803–0. PMID: 39672542

21. Elsallab M, Ouvina M, Arfe A, Bourgeois FT. Mapping the Clinical Development Trajectory of Cell and Gene Therapy Products. Clin Pharmacol Ther. 2024 Dec 10; PMID: 39655466

20. Kakkilaya A, Shahzad M, Bourgeois FT. FDA Approval of Orphan Drug Indications for Pediatric Patients, 2011-2023. JAMA Pediatr. 2024 Dec 9; PMID: 39652344

19. 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

18. Sinha MS, Stern AD, Rai AK. Four Decades of Orphan Drugs and Priorities for the Future. N Engl J Med. 2024 July 11;391(2):100–102. PMID: 38973724

17. 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 June 24;39(24):e190. PMCID: PMC11196855

16. 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 June 17;kxae017. PMID: 38887902

15. 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

14. Guo X, Bourgeois FT, Cai T. Quantifying proportion of treatment effect by surrogate endpoint under heterogeneity. Stat Methods Med Res. 2024 May 8;9622802241247719. PMID: 38717356

13. Masanneck L, Stern AD. Tracing Digital Therapeutics Research Across Medical Specialties: Evidence from ClinicalTrails.gov. Clin Pharmacol Ther. 2024 Apr 2; PMID: 38563641

12. 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

11. 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

10. 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

9. 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

8. 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

2023

47. 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

46. 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

45. 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

44. 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

43. 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

42. Elsallab M, Maus MV. Expanding access to CAR T cell therapies through local manufacturing. Nat Biotechnol. 2023 Oct 26; PMID: 37884746

41. 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 Sept 29;11:e49003. PMCID: PMC10576236

40. 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 Sept 21;S2589-7500(23)00156–5. PMID: 37741765

39. 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

38. 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

37. 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

36. 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

35. 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 July 26;7:e45713. PMCID: PMC10413235

34. Hwang TJ, Reaman GH, Bourgeois FT. Clinical trials for paediatric cancers under new legislation in the USA. Lancet Child Adolesc Health. 2023 July;7(7):e13. PMID: 37349019

33. Bourgeois FT, Espinoza JC. Advancing Equity in Medical Device Development for Children. JAMA Pediatr. 2023 June 1;177(6):561–562. PMID: 37093608

32. 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

31. Everhart AO. Time to publication of cost-effectiveness analyses for medical devices. Am J Manag Care. 2023 May;29(5):265–268. PMCID: PMC10214008

30. 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

29. 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

28. 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

27. 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

26. 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

25. 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

24. 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

2022

56. Russo M, Scarpa B. Learning in Medicine: The Importance of Statistical Thinking. Methods Mol Biol. 2022;2486:215–232. PMID: 35437725

55. 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

54. 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

53. 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

52. 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

51. 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

50. 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

49. 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

48. Tang M, Nakamoto CH, Stern AD, Mehrotra A. Trends in Remote Patient Monitoring Use in Traditional Medicare. JAMA Intern Med. 2022 Sept 1;182(9):1005–1006. PMCID: PMC9344385

2021

78. Sahragardjoonegani B,