The Center for Cancer Systems Pharmacology

Research

Publications by Category

Regulatory Science research conducted at the Center spans a wide range of topics and research disciplines. You can browse some of our work by the focus areas below, a full list of publications can also be seen at https://hmcrs.org/research/all-publications/.

Digital Medicine

Novel clinical trial designs supporting drug and device approval

Emerging considerations for cell and gene therapies

Pediatric drug and device development

Health policies and law in drug regulation

Real world evidence in the lifecycle approach to product evaluation

Biomarkers and surrogate markers in drug trials

Clinical Evidence and Communication

Digital Medicine

BRIDGE Pilot Study: A Bilateral Regulatory Investigation of Data Governance and Exchange
Helen X. Hou, Tom Bisson, Sophia M. Leiss, Julia Thierauf, Ariel D. Stern, Hendrik Strobelt, Felix Nensa, Alena Buyx, Katharina M. Huster, Kira Furlano, Zisis Kozlakidis, Sachin Gupta, Danko Kostadinov, Peter Boor, Anna Slagman, Thorsten Tjardes, Pierre Cholet, Nick K. Schneider, Thorsten Schlomm, Saskia Biskup, Rainer Röhrig, Fruzsina Molnár-Gábor, Uta Schmidt-Straßburger, Katharina Ladewig, Marcel Weigand, Daniel Pinto dos Santos, Jason M. Johnson, Toralf Kirsten, Eric Sutherland, Norman Zerbe, Albert Hofman, Ralf Heyder, Georg Schmidt & Jochen K. Lennerz
NPJ Digital Medicine, ahead of print, February 17, 2026
Data Management and Sharing
Pellen, Claude, Nchangwi Syntia Munung, Anna Catharina Armond, Daniel Kulp, Ulrich Mansmann, Maximilian Siebert, and Florian Naudet
Journal of Clinical Epidemiology 180 (January 20, 2025): 111680.
Mapping the Regulatory Landscape for Artificial Intelligence in Health within the European Union
Schmidt, Jelena, Nienke M. Schutte, Stefan Buttigieg, David Novillo-Ortiz, Eric Sutherland, Michael Anderson, Bart de Witte, et al
NPJ Digital Medicine 7, no. 1 (August 27, 2024): 229
Remote Digital Health Technologies for Improving the Care of People with Respiratory Disorders
Dunn, Jessilyn, Andrea Coravos, Manuel Fanarjian, Geoffrey S. Ginsburg, and Steven R. Steinhubl
The Lancet Digital Health 6, no. 4 (April 1, 2024): e291–98
An Aligned Framework of Actively Collected and Passively Monitored Clinical Outcome Assessments (COAs) for Measure Selection
Campbell, Christine Manta, Courtney Webster, Megan Parisi, Roya Sherafat-Kazemzadeh, Jessica Braid, Thomas Switzer, Marcelo Alves Fávaro, Caprice Sassano, and Andrea Coravos
NPJ Digital Medicine 7, no. 1 (March 16, 2024): 71
Tracing Digital Therapeutics Research Across Medical Specialties: Evidence from ClinicalTrials.Gov
Masanneck, Lars, and Ariel D. Stern
Clinical Pharmacology and Therapeutics, April 2, 2024
Tepid Uptake of Digital Health Technologies in Clinical Trials by Pharmaceutical and Medical Device Firms
Marra, Caroline, and Ariel D. Stern
Clinical Pharmacology and Therapeutics, February 2, 2024
Digital Health Reimbursement Strategies of 8 European Countries and Israel: Scoping Review and Policy Mapping
Kessel, Robin van, Divya Srivastava, Ilias Kyriopoulos, Giovanni Monti, David Novillo-Ortiz, Ran Milman, Wojciech Wilhelm Zhang-Czabanowski, et al
JMIR mHealth and uHealth 11 (September 29, 2023): e49003
Dynamic HTA for Digital Health Solutions: Opportunities and Challenges for Patient-Centered Evaluation
Brönneke, Jan B., Annika Herr, Simon Reif, and Ariel D. Stern
International Journal of Technology Assessment in Health Care 39, no. 1 (November 17, 2023): e72
Effects of Remote Patient Monitoring Use on Care Outcomes Among Medicare Patients With Hypertension : An Observational Study
Tang, Mitchell, Carter H. Nakamoto, Ariel D. Stern, Jose R. Zubizarreta, Felippe O. Marcondes, Lori Uscher-Pines, Lee H. Schwamm, and Ateev Mehrotra
Annals of Internal Medicine 176, no. 11 (November 2023): 1465–75
Building the Business Case for an Inclusive Approach to Digital Health Measurement With a Web App (Market Opportunity Calculator): Instrument Development Study
Tang, Mitchell, Yashoda Sharma, Jennifer C. Goldsack, and Ariel Dora Stern.
JMIR Formative Research 7 (July 26, 2023): e45713.
Evidence from ClinicalTrials.gov on the growth of Digital Health Technologies in neurology trials
Masanneck, Lars, Pauline Gieseler, William J. Gordon, Sven G. Meuth, and Ariel D. Stern
NPJ Digital Medicine 6, no. 1 (February 10, 2023): 23
Trends in Remote Patient Monitoring Use in Traditional Medicare
Tang, Mitchell, Carter H. Nakamoto, Ariel D. Stern, and Ateev Mehrotra
JAMA Internal Medicine 182, no. 9 (September 1, 2022): 1005–6.
Regulatory Considerations to Keep Pace with Innovation in Digital Health Products
Torous, John, Ariel D. Stern, and Florence T. Bourgeois
NPJ Digital Medicine 5, no. 1 (August 19, 2022): 121.
Remote Patient Monitoring – Overdue or Overused?
Mecklai, Keizra, Nicholas Smith, Ariel D. Stern, and Daniel B. Krame
The New England Journal of Medicine 384, no. 15 (April 15, 2021): 1384 – 86.
Ushering in Safe, Effective, Secure, and Ethical Medicine in the Digital Era
Gordon, William J., Andrea R. Coravos, and Ariel D. Stern
NPJ Digital Medicine 4, no. 1 (March 25, 2021): 56.
Building Resilient Medical Technology Supply Chains with a Software Bill of Materials
Carmody, Seth, Andrea Coravos, Ginny Fahs, Audra Hatch, Janine Medina, Beau Woods, and Joshua Corman
NPJ Digital Medicine 4, no. 1 (February 23, 2021): 34.
An Evaluation of Biometric Monitoring Technologies for Vital Signs in the Era of COVID-19
Manta, Christine, Sneha S. Jain, Andrea Coravos, Dena Mendelsohn, and Elena S. Izmailova.
Clinical and Translational Science 13, no. 6 (2020): 1034–44.
Germany’s Digital Health Reforms in the COVID-19 Era: Lessons and Opportunities for Other Countries
Gerke, Sara, Ariel D. Stern, and Timo Minssen.
NPJ Digital Medicine 3 (July 10, 2020): 94.
Modernizing and Designing Evaluation Frameworks for Connected Sensor Technologies in Medicine
Coravos, Andrea, Megan Doerr, Jennifer Goldsack, Christine Manta, Mark Shervey, Beau Woods, and William A. Wood
NPJ Digital Medicine 3 (2020): 37.
Connected Medical Technology and Cybersecurity Informed Consent: A New Paradigm
Tully, Jeffrey, Andrea Coravos, Megan Doerr, and Christian Dameff
Journal of Medical Internet Research 22, no. 3 (30 2020): e17612
Quantifying the Use of Connected Digital Products in Clinical Research
Marra, Caroline, Jacqueline L. Chen, Andrea Coravos, and Ariel D. Stern
NPJ Digital Medicine 3 (2020): 50
Verification, Analytical Validation, and Clinical Validation (V3): The Foundation of Determining Fit-for-Purpose for Biometric Monitoring Technologies (BioMeTs)
Goldsack, Jennifer C., Andrea Coravos, Jessie P. Bakker, Brinnae Bent, Ariel V. Dowling, Cheryl Fitzer-Attas, Alan Godfrey, et al
NPJ Digital Medicine 3 (2020): 55
BioMeT and Algorithm Challenges: A Proposed Digital Standardized Evaluation Framework
Godfrey, Alan, Jennifer C. Goldsack, Pamela Tenaerts, Andrea Coravos, Clara Aranda, Azid Hussain, Marcos E. Barreto, Fraser Young, and Rodrigo Vitorio.
IEEE Journal of Translational Engineering in Health and Medicine 8 (2020): 0700108.
Digital Medicine: A Primer on Measurement
Coravos, Andrea, Jennifer C. Goldsack, Daniel R. Karlin, Camille Nebeker, Eric Perakslis, Noah Zimmerman, and M. Kelley Erb.
Digital Biomarkers 3, no. 2 (May 9, 2019): 31–71.
Developing and Adopting Safe and Effective Digital Biomarkers to Improve Patient Outcomes
Coravos, Andrea, Sean Khozin, and Kenneth D. Mandl.
Npj Digital Medicine 2, no. 1 (March 11, 2019): 14.
Challenges and Opportunities in Software-Driven Medical Devices
Gordon, William J., and Ariel D. Stern.
Nature Biomedical Engineering 3, no. 7 (July 2019): 493–97.
Decentralized Trials in the Age of Real-World Evidence and Inclusivity in Clinical Investigations
Khozin, Sean, and Andrea Coravos.
Clinical Pharmacology and Therapeutics, April 23, 2019.
How Health Care AI Systems Are Changing Care Delivery
Sanders, Samantha F, Terwiesch, Mats, Gordon, William J, and Stern, Ariel S.
NEJM Catalyst, October 17, 2019.
Cybersecurity Features of Digital Medical Devices: An Analysis of FDA Product Summaries
Stern, Ariel Dora, William J Gordon, Adam B Landman, and Daniel B Kramer.
BMJ Open 9, no. 6 (June 2019): e025374.
The Case for a Hippocratic Oath for Connected Medical Devices: Viewpoint
Woods, Beau, Andrea Coravos, and Joshua David Corman.
Journal of Medical Internet Research 21, no. 3 (March 19, 2019): e12568.
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Novel clinical trial designs supporting drug and device approval

Validation of Predictive Analyses for Interim Decisions in Clinical Trials
Avalos-Pacheco, Alejandra, Steffen Ventz, Andrea Arfè, Brian M. Alexander, Rifaquat Rahman, Patrick Y. Wen, and Lorenzo Trippa
JCO Precision Oncology 7 (February 2023): e2200606
Evidence from ClinicalTrials.gov on the growth of Digital Health Technologies in neurology trials
Masanneck, Lars, Pauline Gieseler, William J. Gordon, Sven G. Meuth, and Ariel D. Stern
NPJ Digital Medicine 6, no. 1 (February 10, 2023): 23
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
Elsallab, Magdi, Moataz Ellithi, Susanne Hempel, Hisham Abdel-Azim, and Mohamed Abou-El-Enein
Cancer Gene Therapy, February 7, 2023
Systematic Review of Clinical Drug Development Activities for Neuroblastoma from 2011 to 2020
Nader, Jaclynne H., Florence Bourgeois, Rochelle Bagatell, Lucas Moreno, Andrew D. J. Pearson, and Steven G. DuBois
Pediatric Blood & Cancer, December 2, 2022, e30106
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
Arfè, Andrea, Lewis B. Silverman, and Florence Bourgeois
JAMA Oncology, June 10, 2021
Comparing the Efficacy of Cancer Therapies between Subgroups in Basket Trials
Palmer, Adam C., Deborah Plana, and Peter K. Sorger
Cell Systems 11, no. 5 (November 18, 2020): 449-460.e2.
Optimality of Testing Procedures for Survival Data in the Nonproportional Hazards Setting
Arfè, Andrea, Brian Alexander, and Lorenzo Trippa
Biometrics, June 14, 2020
Pathological Complete Response after Neoadjuvant Chemotherapy and Impact on Breast Cancer Recurrence and Survival: A Comprehensive Meta-Analysis
Spring, Laura M., Geoffrey Fell, Andrea Arfe, Chandni Sharma, Rachel Adams Greenup, Kerry L. Reynolds, Barbara L. Smith, et al
Clinical Cancer Research: An Official Journal of the American Association for Cancer Research, February 11, 2020
Decentralized Trials in the Age of Real-World Evidence and Inclusivity in Clinical Investigations
Khozin, Sean, and Andrea Coravos.
Clinical Pharmacology and Therapeutics, April 23, 2019.
Adaptive Platform Trials: Definition, Design, Conduct and Reporting Considerations
Angus, Derek C., Brian M. Alexander, Scott Berry, Meredith Buxton, Roger Lewis, Melissa Paoloni, Steven A. R. Webb, et al.
Nature Reviews Drug Discovery, August 28, 2019.
A Systematic Review of Feasibility Studies Promoting the Use of Mobile Technologies in Clinical Research
Bakker, Jessie P., Jennifer C. Goldsack, Michael Clarke, Andrea Coravos, Cynthia Geoghegan, Alan Godfrey, Matthew G. Heasley, et al.
Npj Digital Medicine 2, no. 1 (December 2019).
The Central and Unacknowledged Role of the US Food and Drug Administration in the Design and Execution of Medical Device Pivotal Trials
Kaplan, Aaron V., and Ariel D. Stern.
JAMA Cardiology 3, no. 1 (January 1, 2018): 5–6.
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Emerging considerations for cell and gene therapies

Comparison of Clinical Evidence Submitted to the FDA and EMA for Cell and Gene Therapies
Elsallab, Magdi, Sandra Gillner, and Florence T. Bourgeois
JAMA Internal Medicine, February 3, 2025, e247569
Mapping the Clinical Development Trajectory of Cell and Gene Therapy Products
Elsallab, Magdi, Michelle Ouvina, Andrea Arfe, and Florence T. Bourgeois
Clinical Pharmacology and Therapeutics, December 10, 2024
Neurotoxicity and Rare Adverse Events in BCMA-Directed CAR T Cell Therapy: A Comprehensive Analysis of Real-World FAERS Data
Ellithi, Moataz, Magdi Elsallab, Matthew A. Lunning, Sarah A. Holstein, Smriti Sharma, Jonathan Q. Trinh, Jihyun Ma, Marcela V. Maus, Matthew J. Frigault, and Christopher R. D’Angelo
Transplantation and Cellular Therapy, December 12, 2024, S2666-6367(24)00803-0
ACT To Sustain: Adoptive Cell Therapy To Sustain Access to Non-Commercialized Genetically Modified Cell Therapies
Gardner, Rebecca A., Claire White, Magdi Elsallab, Stephanie Farnia, Ellen Fraint, Bambi Grilley, Alison Bateman-House, et al.
Transplantation and Cellular Therapy 30, no. 8 (August 2024): 776–87
National Survey of FACT-Accredited Cell Processing Facilities: Assessing Preparedness for Local Manufacturing of Immune Effector Cells
Elsallab, Magdi, Florence Bourgeois, and Marcela V. Maus
Transplantation and Cellular Therapy, March 16, 2024, S2666-6367(24)00289-6
Expanding Access to CAR T Cell Therapies through Local Manufacturing
Elsallab, Magdi, and Marcela V. Maus
Nature Biotechnology, October 26, 2023
CAR NK-92 Cell-Mediated Depletion of Residual TCR+ Cells for Ultrapure Allogeneic TCR-Deleted CAR T-Cell Products
Kath, Jonas, Weijie Du, Stefania Martini, Magdi Elsallab, Clemens Franke, Laura Hartmann, Vanessa Drosdek, et al
Blood Advances 7, no. 15 (August 8, 2023): 4124–34
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
Elsallab, Magdi, Moataz Ellithi, Susanne Hempel, Hisham Abdel-Azim, and Mohamed Abou-El-Enein
Cancer Gene Therapy, February 7, 2023
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Pediatric drug and device development

Drug Development Trajectory of Anticancer Drugs after Initial Pediatric-Eligible Trials
Martin, Samantha D., Danial Pitafi, Florence Bourgeois, and Steven G. DuBois
Cancer 132, no. 9 (2026): e70433
Closing the Evidence Gap for Drugs in Children – Measures to Strengthen the Pediatric Research Equity Act
Liu, Ian T. T., and Florence Bourgeois
The New England Journal of Medicine 394, no. 16 (2026): 1566–68
Characteristics and Safety of Consumer Chatbots for Emergent Adolescent Health Concerns
Brewster, Ryan C. L., Aydin Zahedivash, Gabriel Tse, Florence Bourgeois, and Scott E. Hadland
JAMA Network Open 8, no. 10 (2025): e2539022
Delays in Pediatric Studies Required Under the US Pediatric Research Equity Act
McGonigle, Rylee, and Florence T. Bourgeois
JAMA Network Open 8, no. 10 (2025): e2541176
Describing Inconsistencies in Pediatric Labeling of Medical Devices
Zapotoczny, Grzegorz, Alexis Snitman, Payal Shah, et al
JAMA Network Open 8, no. 12 (2025): e2546517
US Food and Drug Administration Approval of Biologic Drugs for Use in Children and Adolescents
Lyu, Ning, Sebastian Schneeweiss, Florence T. Bourgeois, and Timothy J. Savage
JAMA Pediatrics, December 8, 2025, e255087
Assessment of Oral Anticancer Medicines in Pediatric Patients before the Research to Accelerate Cures and Equity (RACE) Act, 2007-2021
Shahzad, Mahnum, Steven G. DuBois, Rebecca Costa, et al
The Journal of Pediatrics 290 (December 2025): 114949
Pediatric Studies and Labeling Additions Required by the U.S. FDA for Novel Drugs Approved from 2011 to 2023: A Retrospective Cohort Study
McGonigle, Rylee, Huseyin Naci, Maximilian Siebert, et al
https://doi.org/10.1371/journal.pmed.1004651
Impact of COVID-19 Non-Pharmaceutical Interventions on Bacterial Infections in Children: An International Electronic Health Record-Based Study
Zachariasse, Joany M., Alba Gutiérrez-Sacristán, Simran Makwana, et al
BMJ Public Health 3, no. 2 (2025): e002671
FDA Approval of Cardiac Valve Devices Implanted in a National Cohort of Pediatric Patients, 2016-2022
Wunnava, Susmitha, Timothy A. Miller, Meena Nathan, and Florence T. Bourgeois
JAMA Pediatrics, March 24, 2025, e250131
FDA Approval of Orphan Drug Indications for Pediatric Patients, 2011-2023
Kakkilaya, Apoorva, Mahnum Shahzad, and Florence T. Bourgeois
JAMA Pediatrics, December 9, 2024
US FDA Approval of Pediatric Artificial Intelligence and Machine Learning-Enabled Medical Devices
Brewster, Ryan C. L., Matthew Nagy, Susmitha Wunnava, and Florence T. Bourgeois
JAMA Pediatrics, December 16, 2024
Pediatric Exclusivity Revenues for Cancer Drugs
Sarpatwari, Ameet, Liam Bendicksen, Douglas S. Hawkins, Lia Gore, and Florence T. Bourgeois
JAMA Pediatrics, November 11, 2024, e244449
Enrollment of Underserved Racial and Ethnic Populations in Pediatric Asthma Clinical Trials
Geanacopoulos, Alexandra T., Ann Chen Wu, Florence T. Bourgeois, Alon Peltz, Ryan Walsh, Amy Han, and Mei-Sing Ong
The Journal of Allergy and Clinical Immunology. Global 3, no. 4 (November 2024): 100315
Enrollment of Pediatric Patients in COVID-19 Interventional Trials
Ong, Mei-Sing, Ann Chen Wu, and Florence T. Bourgeois
JAMA Health Forum 4, no. 11 (November 3, 2023): e233939
Characteristics and Results of Pediatric Medical Device Studies: 2017-2022
Quazi, Sabrina, Claire Narang, Juan C. Espinoza, and Florence T. Bourgeois
Pediatrics, August 11, 2023, e2022059842
Reporting and Representation of Participant Race and Ethnicity in National Institutes of Health-Funded Pediatric Clinical Trials
Lee, Lois K., Claire Narang, Chris A. Rees, Ravi R. Thiagarajan, Patrice Melvin, Valerie Ward, and Florence T. Bourgeois
JAMA Network Open 6, no. 8 (August 1, 2023): e2331316.
Clinical Trials for Paediatric Cancers under New Legislation in the USA
Hwang, Thomas J., Gregory H. Reaman, and Florence T. Bourgeois
The Lancet. Child & Adolescent Health 7, no. 7 (July 2023): e13
Advancing Equity in Medical Device Development for Children
Bourgeois, Florence T., and Juan C. Espinoza
JAMA Pediatrics 177, no. 6 (June 1, 2023): 561–62
Clinical Development of New Drugs for Adults and Children with Cancer, 2010-2020
Arfè, Andrea, Claire Narang, Steven G. DuBois, Gregory Reaman, and Florence T.
Journal of the National Cancer Institute, May 12, 2023, djad082
US Food and Drug Administration Approval of High-Risk Cardiovascular Devices for Use in Children and Adolescents, 1977-2021
Wunnava, Susmitha, Timothy A. Miller, Claire Narang, Meena Nathan, and Florence T. Bourgeois
JAMA 328, no. 6 (August 9, 2022): 580–82.
The Urgent Need for Research Coordination to Advance Knowledge on COVID – 19 in Children
Bourgeois, Florence T., Paul Avillach, and Mark A. Turner
Pediatric Research, 2 (August 2021).
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
Arfè, Andrea, Lewis B. Silverman, and Florence Bourgeois
JAMA Oncology, June 10, 2021
Inclusion of Children in Clinical Trials of Treatments for Coronavirus Disease 2019 (COVID-19)
Hwang, Thomas J., Adrienne G. Randolph, and Florence T. Bourgeois.
JAMA Pediatrics 174, no. 9 (September 1, 2020): 825–26.
Evaluation of Publication of Pediatric Drug Trials
Srivastava, Ananya, and Florence T. Bourgeois
JAMA Network Open 4, no. 4 (April 1, 2021): e215829.
Pediatric Drug Policies Supporting Safe And Effective Use Of Therapeutics In Children: A Systematic Analysis
Carmack, Mary, Thomas Hwang, and Florence T. Bourgeois
Health Affairs (Project Hope) 39, no. 10 (October 2020): 1799–1805.
Cost Implications of Escalating Intravenous Acetaminophen Use in Children
Bourgeois, Florence T., Dionne A. Graham, Aaron S. Kesselheim, and Adrienne G. Randolph.
JAMA Pediatrics, March 11, 2019.
Promoting Pediatric Drug Research and Labeling – Outcomes of Legislation
Bourgeois, Florence T., and Aaron S. Kesselheim.
The New England Journal of Medicine 381, no. 9 (29 2019): 875–81.
Impact Of The Priority Review Voucher Program On Drug Development For Rare Pediatric Diseases
Hwang, Thomas J., Florence T. Bourgeois, Jessica M. Franklin, and Aaron S. Kesselheim.
Health Affairs (Project Hope) 38, no. 2 (February 2019): 313–19.
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Health policies and law in drug regulation

Closing the Evidence Gap for Drugs in Children – Measures to Strengthen the Pediatric Research Equity Act
Liu, Ian T. T., and Florence Bourgeois
The New England Journal of Medicine 394, no. 16 (2026): 1566–68
Assessment of Oral Anticancer Medicines in Pediatric Patients before the Research to Accelerate Cures and Equity (RACE) Act, 2007-2021
Shahzad, Mahnum, Steven G. DuBois, Rebecca Costa, et al
The Journal of Pediatrics 290 (December 2025): 114949
Pediatric Studies and Labeling Additions Required by the U.S. FDA for Novel Drugs Approved from 2011 to 2023: A Retrospective Cohort Study
McGonigle, Rylee, Huseyin Naci, Maximilian Siebert, et al
https://doi.org/10.1371/journal.pmed.1004651
The 340B Drug Pricing Program: Administration, Litigation, and Reform
Knox, Ryan P, and Ameet Sarpatwari
OKLAHOMA LAW REVIEW 77 (2025).
FDA Approval of Orphan Drug Indications for Pediatric Patients, 2011-2023
Kakkilaya, Apoorva, Mahnum Shahzad, and Florence T. Bourgeois
JAMA Pediatrics, December 9, 2024
US FDA Approval of Pediatric Artificial Intelligence and Machine Learning-Enabled Medical Devices
Brewster, Ryan C. L., Matthew Nagy, Susmitha Wunnava, and Florence T. Bourgeois
JAMA Pediatrics, December 16, 2024
Pediatric Exclusivity Revenues for Cancer Drugs
Sarpatwari, Ameet, Liam Bendicksen, Douglas S. Hawkins, Lia Gore, and Florence T. Bourgeois
JAMA Pediatrics, November 11, 2024, e244449
Naloxone Access Laws and Opioid-Related Overdose Deaths in Youths
Toce, Michael S., Kenneth A. Michelson, Scott E. Hadland, Michael C. Monuteaux, and Florence T. Bourgeois
JAMA Pediatrics, October 14, 2024
Mapping the Regulatory Landscape for Artificial Intelligence in Health within the European Union
Schmidt, Jelena, Nienke M. Schutte, Stefan Buttigieg, David Novillo-Ortiz, Eric Sutherland, Michael Anderson, Bart de Witte, et al
NPJ Digital Medicine 7, no. 1 (August 27, 2024): 229
The Challenges of Regulatory Pluralism
Gillner, Sandra, Katharina Elisabeth Blankart, Florence Tanya Bourgeois, Ariel Dora Stern, and Carl Rudolf Blankart
Health Policy (Amsterdam, Netherlands) 149 (November 2024): 105164
Biosimilar Approval Pathways: Comparing the Roles of Five Medicines Regulators
Knox, Ryan P., Vineet Desai, and Ameet Sarpatwari
Journal of Law and the Biosciences 11, no. 2 (2024): lsae020
Four Decades of Orphan Drugs and Priorities for the Future
Sinha, Michael S., Ariel D. Stern, and Arti K. Rai
The New England Journal of Medicine 391, no. 2 (July 11, 2024): 100–102
Patents and Regulatory Exclusivities on FDA-Approved Insulin Products: A Longitudinal Database Study, 1986-2019
Olsen, Anders, Reed F. Beall, Ryan P. Knox, Sean S. Tu, Aaron S. Kesselheim, and William B. Feldman
PLoS Medicine 20, no. 11 (November 2023): e1004309
Outcomes of the 340B Drug Pricing Program: A Scoping Review
Knox, Ryan P., Junyi Wang, William B. Feldman, Aaron S. Kesselheim, and Ameet Sarpatwari
JAMA Health Forum 4, no. 11 (November 3, 2023): e233716
Federated Electronic Health Records for the European Health Data Space
Raab, René, Arne Küderle, Anastasiya Zakreuskaya, Ariel D. Stern, Jochen Klucken, Georgios Kaissis, Daniel Rueckert, et al
The Lancet. Digital Health, September 21, 2023, S2589-7500(23)00156-5
Clinical Trials for Paediatric Cancers under New Legislation in the USA
Hwang, Thomas J., Gregory H. Reaman, and Florence T. Bourgeois
The Lancet. Child & Adolescent Health 7, no. 7 (July 2023): e13
Association of Prescription Drug Monitoring Programs with Benzodiazepine Prescription Dispensation and Overdose in Adolescents and Young Adults
Toce, Michael S., Kenneth A. Michelson, Joel D. Hudgins, Karen L. Olson, Michael C. Monuteaux, and Florence T. Bourgeois
Clinical Toxicology (Philadelphia, Pa.) 61, no. 4 (April 2023): 234–40
Association of Prescription Drug Monitoring Programs With Opioid Prescribing and Overdose in Adolescents and Young Adults
Toce, Michael S., Kenneth A. Michelson, Joel D. Hudgins, Scott E. Hadland, Karen L. Olson, Michael C. Monuteaux, and Florence T. Bourgeois
Annals of Emergency Medicine 81, no. 4 (April 2023): 429–37
The Humira Patent Thicket, the Noerr-Pennington Doctrine and Antitrust’s Patent Problem
Knox, Ryan, and Gregory Curfman
Nature Biotechnology 40, no. 12 (December 2022): 1761–63
US Food and Drug Administration Approval of High-Risk Cardiovascular Devices for Use in Children and Adolescents, 1977-2021
Wunnava, Susmitha, Timothy A. Miller, Claire Narang, Meena Nathan, and Florence T. Bourgeois
JAMA 328, no. 6 (August 9, 2022): 580–82.
Risks to the 340B Drug Pricing Program Related to Manufacturer Restrictions on Drug Availability
Knox, Ryan P., Aaron S. Kesselheim, and Ameet Sarpatwari
JAMA 327, no. 17 (April 15, 2022): 1647 - 1648.
FDA Approval Standards for Anticancer Agents – Lessons from Two Recent Approvals in Breast Cancer
Gyawali, Bishal, and Aaron S. Kesselheim
Nature Review s. Clinical Oncology 18, no. 7 (July 2021): 397 – 98.
Biosimilars And Follow – On Products In The United States: Adoption, Prices, And Users
Stern, Ariel Dora, Jacqueline L. Chen, Melissa Ouellet, Mark R. Trusheim, Zeid El - Kilani, Amber Jessup, and Ernst R. Berndt
Health Affairs (Project Hope) 40, no. 6 (June 2021): 989 – 99.
Factors Associated With Generic Drug Uptake in the United States, 2012 to 2017
Rome, Benjamin N., ChangWon C. Lee, Joshua J. Gagne, and Aaron S. Kesselheim
Value in Health: The Journal of the International Society for Pharmacoeconomics and Outcomes Research, 6 (June 2021)
Assessment of Coverage in England of Cancer Drugs Qualifying for US Food and Drug Administration Accelerated Approval
Cherla, Avi, Huseyin Naci, Aaron S. Kesselheim, Bishal Gyawali, and Elias Mossialos
JAMA Internal Medicine 181, no. 4 (April 1, 2021): 490 – 98.
Repurposing Existing Drugs for New Uses: A Cohort Study of the Frequency of FDA – Granted New Indication Exclusivities since 1997
Sahragardjoonegani , Babak, Reed F. Beall, Aaron S. Kesselheim, and Aidan Hollis
Journal of Pharmaceutical Policy and Practice 14, no. 1 (January 4, 2021): 3.
FDA Approval and Regulation of Pharmaceuticals, 1983-2018
Darrow, Jonathan J., Jerry Avorn, and Aaron S. Kesselheim
JAMA 323, no. 2 (January 14, 2020): 164–76
Reconsidering the Scope of US State Laws Allowing Pharmacist Substitution of Generic Drugs
Darrow, Jonathan J., Jessica E. Chong, and Aaron S. Kesselheim
BMJ (Clinical Research Ed.) 369 (June 23, 2020): m2236
Incentivizing Antibiotic Development: Why Isn’t the Generating Antibiotic Incentives Now (GAIN) Act Working?
Darrow, Jonathan J., and Aaron S. Kesselheim
Open Forum Infectious Diseases 7, no. 1 (January 2020): ofaa001.
Regulatory Approval Characteristics of Antimicrobial versus Non-Antimicrobial Products, 1984-2018: An Evaluation of Food and Drug Administration Flexibilities
Darrow, Jonathan J., Mehdi Najafzadeh, Kristina Stefanini, and Aaron S. Kesselheim
The Lancet. Infectious Diseases 20, no. 7 (2020): e159–64
A Qualitative Study of Biosimilar Manufacturer and Regulator Perceptions on Intellectual Property and Abbreviated Approval Pathways
Druedahl, Louise C., Anna Birna Almarsdóttir, Sofia Kälvemark Sporrong, Marie Louise De Bruin, Hans Hoogland, Timo Minssen, Marco van de Weert, Aaron S. Kesselheim, and Ameet Sarpatwari
Nature Biotechnology 38, no. 11 (2020): 1253–56
Association between FDA and EMA Expedited Approval Programs and Therapeutic Value of New Medicines: Retrospective Cohort Study
Hwang, Thomas J., Joseph S. Ross, Kerstin N. Vokinger, and Aaron S. Kesselheim
BMJ (Clinical Research Ed.) 371 (October 7, 2020): m3434
Association of the Meaningful Use Electronic Health Record Incentive Program With Health Information Technology Venture Capital Funding
Lite, Samuel, William Joseph Gordon, and Ariel Dora Stern
JAMA Network Open 3, no. 3 (02 2020): e201402
Insulin Access and Affordability in the USA: Anticipating the First Interchangeable Insulin Product
Luo, Jing, Aaron S. Kesselheim, and Ameet Sarpatwari
The Lancet. Diabetes & Endocrinology 8, no. 5 (2020): 360–62
Clinical Benefit and Cost of Breakthrough Cancer Drugs Approved by the US Food and Drug Administration
Molto, Consolación, Thomas J. Hwang, Maria Borrell, Marta Andres, Ignasi Gich, Agustí Barnadas, Eitan Amir, Aaron S. Kesselheim, and Ariadna Tibau
Cancer 126, no. 19 (October 1, 2020): 4390–99
Market Exclusivity Length for Drugs with New Generic or Biosimilar Competition, 2012-2018
Rome, Benjamin N., ChangWon C. Lee, and Aaron S. Kesselheim
Clinical Pharmacology and Therapeutics, July 12, 2020
Assessment of Variation in State Regulation of Generic Drug and Interchangeable Biologic Substitutions
Sacks, Chana A., Victor L. Van de Wiele, Lisa A. Fulchino, Lajja Patel, Aaron S. Kesselheim, and Ameet Sarpatwari
JAMA Internal Medicine, August 31, 2020
The Wrong Cure: Financial Incentives for Unimpressive New Antibiotics
Sinha, Michael S., John H. Powers Iii, and Aaron S. Kesselheim
The Journal of Infectious Diseases, August 22, 2020
Reputation and Authority: The FDA and the Fight over U.S. Prescription Drug Importation
Thomas J Bollyky, and Aaron S Kesselheim
Vanderbilt Law Review 73, no. 5 (2020): 1331–1400
Association of State-Level Opioid-Reduction Policies With Pediatric Opioid Poisoning
Toce, Michael S., Kenneth Michelson, Joel Hudgins, Michele M. Burns, Michael C. Monuteaux, and Florence T. Bourgeois
JAMA Pediatrics 174, no. 10 (October 1, 2020): 961–68
Decision Making Under Uncertainty: Comparing Regulatory and Health Technology Assessment Reviews of Medicines in the United States and Europe
Vreman, Rick A., Huseyin Naci, Wim G. Goettsch, Aukje K. Mantel-Teeuwisse, Sebastian G. Schneeweiss, Hubert G. M. Leufkens, and Aaron S. Kesselheim
Clinical Pharmacology and Therapeutics 108, no. 2 (August 2020): 350–57
FDA and EMA Biosimilar Approvals
Jung, Sarpatwari, Kesselheim, and Sinha. 2019.
Journal of General Internal Medicine, October 21, 2019.
Experiences With and Challenges Afforded by Expedited Regulatory Pathways
Liu, Sheng, and Aaron S. Kesselheim.
Clinical Pharmacology & Therapeutics 105, no. 4 (2019): 795–97.
Is Health-Care Data the New Blood?
Perakslis, Eric, and Andrea Coravos.
The Lancet Digital Health 1, no. 1 (May 1, 2019): PE8-E9.
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Real world evidence in the lifecycle approach to product evaluation

Benzodiazepine or Z-Hypnotic Use during Pregnancy and Risk of Psychiatric Disorders in Children: Population Based Cohort Study
Cho, Yongtai, Ahhyung Choi, Jieun Woo, Jung Yeol Han, Jae-Won Kim, and Ju-Young Shin
BMJ (Clinical research ed.) 393 (April 2026): e088671
Anti-Tumor Necrosis Factor Therapy and the Risk of Gestational Diabetes in Pregnant Women With Inflammatory Bowel Disease
Cho, Yongtai, Eun-Young Choi, Ahhyung Choi, et al
The American Journal of Gastroenterology 120, no. 1 (2025): 241–50
Impact of COVID-19 Non-Pharmaceutical Interventions on Bacterial Infections in Children: An International Electronic Health Record-Based Study
Zachariasse, Joany M., Alba Gutiérrez-Sacristán, Simran Makwana, et al
BMJ Public Health 3, no. 2 (2025): e002671
Ocrelizumab versus Natalizumab in Relapsing-Remitting Multiple Sclerosis: A Registry-Linked Electronic Health Records Study
Huang, Feiqing, Wen Zhu, Jue Hou, et al
medRxiv: The Preprint Server for Health Sciences, December 30, 2025, 2025.12.01.25341331
Advancing the Use of Longitudinal Electronic Health Records: Tutorial for Uncovering Real-World Evidence in Chronic Disease Outcomes
Huang, Feiqing, Jue Hou, Ningxuan Zhou, Kimberly Greco, Chenyu Lin, Sara Morini Sweet, Jun Wen, et al
Journal of Medical Internet Research 27 (May 12, 2025): e71873
Real-World Utilization Patterns of Oral Corticosteroids During Pregnancy: A Nationwide Population-Based Study in Korea
Oh, Jeongin, Yongtai Cho, Jung Yeol Han, Ahhyung Choi, and Ju-Young Shi
Journal of Korean Medical Science 40, no. 2 (January 13, 2025): e80
Neurotoxicity and Rare Adverse Events in BCMA-Directed CAR T Cell Therapy: A Comprehensive Analysis of Real-World FAERS Data
Ellithi, Moataz, Magdi Elsallab, Matthew A. Lunning, Sarah A. Holstein, Smriti Sharma, Jonathan Q. Trinh, Jihyun Ma, Marcela V. Maus, Matthew J. Frigault, and Christopher R. D’Angelo
Transplantation and Cellular Therapy, December 12, 2024, S2666-6367(24)00803-0
COVID-19 Infection and Severe Clinical Outcomes in Patients with Kidney Disease by Vaccination Status: A Nationwide Cohort Study in Korea
Woo, Jieun, Ahhyung Choi, Jaehun Jung, and Ju-Young Shin
Epidemiology and Health 46 (2024): e2024065
Anti-Tumor Necrosis Factor Therapy and the Risk of Gestational Diabetes in Pregnant Women With Inflammatory Bowel Disease
Cho, Yongtai, Eun-Young Choi, Ahhyung Choi, Jung Yeol Han, Byong Duk Ye, Ju Hwan Kim, and Ju-Young Shin
The American Journal of Gastroenterology, September 24, 2024
Selected Acute Safety Events Following the Use of Nirmatrelvir/Ritonavir or Molnupiravir for COVID-19: A Nationwide Cohort Study in South Korea
Kim, Ju Hwan, Ahhyung Choi, Gihwan Bae, Eun-Jeong Joo, Min Joo Choi, Kyungmin Huh, Hyungmin Lee, et al
Clinical Pharmacology and Therapeutics, October 8, 2024
Cardiovascular Safety of COVID-19 Vaccination in Patients With Cancer: A Self-Controlled Case Series Study in Korea
Ryu, Ji Hwa, Ahhyung Choi, Jieun Woo, Hyesung Lee, Jinkwon Kim, Joonsang Yoo, and Ju-Young Shin
Journal of Korean Medical Science 39, no. 24 (June 24, 2024): e190
Data Sources and Applied Methods for Paclitaxel Safety Signal Discernment
Gressler, Laura Elisabeth, Erika Avila-Tang, Jialin Mao, Alejandra Avalos-Pacheco, Fadia T. Shaya, Yelizaveta Torosyan, Alexander Liebeskind, et al.
Frontiers in Cardiovascular Medicine 10 (2024)
Endovascular Aneurysm Repair Devices as a Use Case for Postmarketing Surveillance of Medical Devices
Wang, Xuan, Vidul Ayakulangara Panickan, Tianrun Cai, Xin Xiong, Kelly Cho, Tianxi Cai, and Florence T. Bourgeois
JAMA Internal Medicine, August 21, 2023, e233562.
Association Between Regulatory Submission Characteristics and Recalls of Medical Devices Receiving 510(k) Clearance
Everhart, Alexander O., Soumya Sen, Ariel D. Stern, Yi Zhu, and Pinar Karaca-Mandic
JAMA 329, no. 2 (January 10, 2023): 144–56
Improving FDA Postmarket Adverse Event Reporting for Medical Devices
Wunnava, Susmitha, Timothy A. Miller, and Florence T. Bourgeois
BMJ Evidence-Based Medicine (February 17, 2022).
Decentralized Trials in the Age of Real-World Evidence and Inclusivity in Clinical Investigations
Khozin, Sean, and Andrea Coravos.
Clinical Pharmacology and Therapeutics, April 23, 2019.
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Biomarkers and surrogate markers in drug trials

Evidence Supporting European Medicines Agency Drug Approvals (2020-2023): A Cross-Sectional Study of Trial Design and Outcomes
Siebert, Maximilian, Laura Caquelin, Florian Naudet, Joseph S. Ross, and Reshma Ramachandran
Clinical Trials (London, England), April 7, 2026, 17407745261437363
Participants in the FDA’s Biomarker Qualification Program
Shahzad, Mahnum, and Ariel D. Stern
Clinical Pharmacology and Therapeutics, April 9, 2025
Quantifying Proportion of Treatment Effect by Surrogate Endpoint under Heterogeneity
Guo, Xinzhou, Florence T. Bourgeois, and Tianxi Cai
Statistical Methods in Medical Research, May 8, 2024, 9622802241247719
Quantifying the Feasibility of Shortening Clinical Trial Duration Using Surrogate Markers
Wang, Xuan, Tianxi Cai, Lu Tian, Florence Bourgeois, and Layla Parast
Statistics in Medicine (September 2, 2021).
Meta-Analysis of PD-L1 Expression As a Predictor of Survival After Checkpoint Blockade
Arfè, Andrea, Geoffrey Fell, Brian Alexander, Mark M. Awad, Scott J. Rodig, Lorenzo Trippa, and Jonathan D. Schoenfeld
JCO Precision Oncology, 4 (November 2020): 1196–1206
Response Rates and Durations of Response for Biomarker-Based Cancer Drugs in Nonrandomized Versus Randomized Trials
Gyawali, Bishal, Elvira D’Andrea, Jessica M. Franklin, and Aaron S. Kesselheim.
Journal of the National Comprehensive Cancer Network 18, no. 1 (January 1, 2020): 36–43.
Evaluating the Evidence behind the Surrogate Measures Included in the FDA’s Table of Surrogate Endpoints as Supporting Approval of Cancer Drugs
Gyawali, Bishal, Spencer P. Hey, and Aaron S. Kesselheim.
EClinicalMedicine 21 (April 2020): 100332.
US Food and Drug Administration Recommendations on the Use of Surrogate Measures as End Points in New Anti-Infective Drug Approvals
Hey, Spencer Phillips, Aaron S. Kesselheim, Pranav Patel, Preeti Mehrotra, and John H. Powers.
JAMA Internal Medicine 180, no. 1 (January 1, 2020): 131–38.
Digital Medicine: A Primer on Measurement
Coravos, Andrea, Jennifer C. Goldsack, Daniel R. Karlin, Camille Nebeker, Eric Perakslis, Noah Zimmerman, and M. Kelley Erb.
Digital Biomarkers 3, no. 2 (May 9, 2019): 31–71.
Innovation Incentives and Biomarkers
Stern, Ariel D., Brian M. Alexander, and Amitabh Chandra.
Clinical Pharmacology and Therapeutics 103, no. 1 (January 2018): 34–36.
Developing and Adopting Safe and Effective Digital Biomarkers to Improve Patient Outcomes
Coravos, Andrea, Sean Khozin, and Kenneth D. Mandl.
Npj Digital Medicine 2, no. 1 (March 11, 2019): 14.
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Clinical Evidence and Communication

Reporting and Publication of Postmarket Clinical Trials for Novel Drugs Approved by the FDA
Maximilian Siebert, Joshua D. Wallach, Florence T. Bourgeois
JAMA Netw Open. 2026;9(7):e2624832.
Patient Perspectives on Approval Speed vs Evidentiary Certainty in US Cancer Drug Approvals
Robin Forrest, Ajay Aggarwal, Michelle Tregear, Melanie Wyne, Holly Fernandez Lynch, Anita K. Wagner, Emily Jackson, Huseyin Naci
JAMA Netw Open. 2026;9(6):e2617450.
New Cancer Drug Approvals: Less Than Half Of Important Clinical Trial Uncertainties Reported By The FDA To Clinicians, 2019–22
Avi Cherla, Steven Woloshin, Anita K. Wagner, Olivier J. Wouters, Courtney Davis, Elias Mossialos, and Huseyin Naci
Health Aff (Millwood). 2025 Jul;44(7):830-838.
Reporting of Clinical Trial Uncertainties With New Cancer Drugs in Journal Publications and Clinical Guidelines
Avi Cherla, Anita K. Wagner, Olivier J. Wouters, Steven Woloshin, Courtney Davis, Elias Mossialos, Huseyin Naci
JAMA. 2025;334(16):1480–1482. doi:10.1001/jama.2025.13917
Completion and outcomes of postmarketing requirements and commitments issued by the US Food and Drug Administration for new drugs, 2016-24: cross sectional evaluation of postmarketing studies
Siebert M, McGonigle R, Gutiérrez-Sacristán A, Naci H, Wagner AK, Bourgeois FT.
BMJ Medicine. 2026
Closing the Evidence Gap for Drugs in Children – Measures to Strengthen the Pediatric Research Equity Act
Liu, Ian T. T., and Florence Bourgeois
The New England Journal of Medicine 394, no. 16 (2026): 1566–68
Delays in Pediatric Studies Required Under the US Pediatric Research Equity Act
McGonigle, Rylee, and Florence T. Bourgeois
JAMA Network Open 8, no. 10 (2025): e2541176
Assessment of Oral Anticancer Medicines in Pediatric Patients before the Research to Accelerate Cures and Equity (RACE) Act, 2007-2021
Shahzad, Mahnum, Steven G. DuBois, Rebecca Costa, et al
The Journal of Pediatrics 290 (December 2025): 114949
Pediatric Studies and Labeling Additions Required by the U.S. FDA for Novel Drugs Approved from 2011 to 2023: A Retrospective Cohort Study
McGonigle, Rylee, Huseyin Naci, Maximilian Siebert, et al
https://doi.org/10.1371/journal.pmed.1004651
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