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

COVID-19 Response

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

Digital Medicine

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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COVID-19 Response

De Novo Powered Air – Purifying Respirator Design and Fabrication for Pandemic Response
Kothakonda, Akshay, Lyla Atta, Deborah Plana, Ferrous Ward, Chris Davis, Avilash Cramer, Robert Moran, et al
Frontiers in Bioengineering and Biotechnology (2021).
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).
3D Printed Frames to Enable Reuse and Improve the Fit of N95 and KN95 Respirator
McAvoy, Malia, Ai - Tram N. Bui, Christopher Hansen, Deborah Plana, Jordan T. Said, Zizi Yu, Helen Yang, et al. “
BMC Biomedical Engineering, 3 (June 7, 2021).
International Analysis of Electronic Health Records of Children and Youth Hospitalized With COVID-19 Infection in 6 Countries
Bourgeois, Florence T., Alba Gutiérrez-Sacristán, Mark S. Keller, Molei Liu , Chuan Hong, Clara - Lea Bonzel, Amelia L. M. Tan, et al.
JAMA Network Open 4, no. 6 (June 1, 2021): e2112596.
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.
A Crisis-Responsive Framework for Medical Device Development Applied to the COVID-19 Pandemic
Antonini, Marc-Joseph, Deborah Plana, Shriya Srinivasan, Lyla Atta, Aditya Achanta, Helen Yang, Avilash Cramer, et al.
Frontiers in Digital Health, 3 (March 29, 2021).
Regulatory Decision-Making on COVID-19 Vaccines During a Public Health Emergency
Avorn, Jerry, and Aaron Kesselheim.
JAMA 324, no. 13 (October 6, 2020): 1284–85.
A Pilot Study of the Impact of Facial Skin Protectants on Qualitative Fit Testing of N95 Masks
Bui, Ai-Tram N., Zizi Yu, Karen Lee, Sara J. Li, William G. Tsiaras, Sherry H. Yu, Nicole R. LeBoeuf, and Arash Mostaghimi.
Journal of the American Academy of Dermatology, June 2020.
Analysis of SteraMist ionized hydrogen peroxide technology in the sterilization of N95 respirators and other PPE
Cramer, Avilash, Deborah Plana, Helen L. Yang, et al.
Sci Rep 11, 2051 (2021).
Assessment of the Qualitative Fit Test and Quantitative Single-Pass Filtration Efficiency of Disposable N95 Masks Following Gamma Irradiation
Cramer, Avilash, Enze Tian, Mitchell Galanek, Edward Lamere, Ju Li, Rajiv Gupta, and Mike Short.
JAMA Network Open 3, no. 5 (May 26, 2020): e209961.
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.
An Overview Of Vaccine Development, Approval, And Regulation, With Implications For COVID-19
Kesselheim, Aaron S., Jonathan J. Darrow, Martin Kulldorff, Beatrice L. Brown, Mayookha Mitra-Majumdar, ChangWon Charlie Lee, Osman Moneer, and Jerry Avorn.
Health Affairs (Project Hope), November 19, 2020, 101377hlthaff202001620.
Origins and Ownership of Remdesivir: Implications for Pricing
Lee, ChangWon C., Jonathan J. Darrow, Jerry Avorn, and Aaron S. Kesselheim
The Journal of Law, Medicine & Ethics: A Journal of the American Society of Law, Medicine & Ethics 48, no. 3 (2020): 613–18.
Internet Searches for Unproven COVID-19 Therapies in the United States
Liu, Michael, Theodore L. Caputi, Mark Dredze, Aaron S. Kesselheim, and John W. Ayers.
JAMA Internal Medicine 180, no. 8 (August 1, 2020): 1116–18.
Regulatory and Safety Considerations in Deploying a Locally Fabricated, Reusable Face Shield in a Hospital Responding to the COVID-19 Pandemic
Mostaghimi, Arash, Marc-Joseph Antonini, Deborah Plana, Philip D. Anderson, Brandon Beller, Edward W. Boyer, Amber Fannin, et al.
Med (New York, N.Y.), June 19, 2020.
Covid-19 – The Law and Limits of Quarantine
16. Parmet, Wendy E., and Michael S. Sinha.
The New England Journal of Medicine 382, no. 15 (April 9, 2020): e28.
Assessing the Quality of Nontraditional N95 Filtering Face-Piece Respirators Available during the COVID-19 Pandemic
Plana, Deborah, Enze Tian, Avilash K. Cramer, Helen Yang, Mary M. Carmack, Michael S. Sinha, Florence T. Bourgeois, et al.
MedRxiv: The Preprint Server for Health Sciences, July 27, 2020.
Missed Opportunities on Emergency Remdesivir Use
Sarpatwari, Ameet, Anna Kaltenboeck, and Aaron S. Kesselheim.
JAMA 324, no. 4 (July 28, 2020): 331–32.
Personal Protective Equipment for COVID-19: Distributed Fabrication and Additive Manufacturing
Sinha, Michael S., Florence T. Bourgeois, and Peter K. Sorger.
American Journal of Public Health 110, no. 8 (2020): 1162–64.
Return to Work during Coronavirus Disease 2019 (COVID-19): Temperature Screening Is No Panacea
Slade, David H., and Michael S. Sinha
Infection Control and Hospital Epidemiology, September 23, 2020, 1–2.
Considerations for the Selection and Use of Disinfectants Against SARS-CoV-2 in a Health Care Setting
Tyan, Kevin, Adriane Levin, Alejandra Avalos-Pacheco, Deborah Plana, Eleanor A. Rand, Helen Yang, Laura E. Maliszewski, et al.
Open Forum Infectious Diseases 7, no. 9 (September 2020): ofaa396.
False Negative Tests for SARS-CoV-2 Infection – Challenges and Implications
Woloshin, Steven, Neeraj Patel, and Aaron S. Kesselheim.
The New England Journal of Medicine 383, no. 6 (August 6, 2020): e38.
Need for Transparency and Reliable Evidence in Emergency Use Authorizations for Coronavirus Disease 2019 (COVID-19) Therapies
Zhai, Mike Z., Carolyn T. Lye, and Aaron S. Kesselheim.
JAMA Internal Medicine 180, no. 9 (September 1, 2020): 1145–46.
Conversion of Existing UVB Phototherapy Units to UVC Germicidal Chambers for N95 Decontamination: Lessons Learned
Kassamali, Bina, Zizi Yu, Christopher Davis, Mary Carmack, Ai - Tram N. Bui, Jordan T. Said, Deborah Plana, et al.
Photobio modulation, Photomedicine, and Laser Surgery 39, no. 2 (February 2021): 83 – 85.
Analysis of SteraMist Ionized Hydrogen Peroxide Technology in the Sterilization of N95 Respirators and Other PPE
Cramer, Avilash K., Deborah Plana, Helen Yang, Mary M. Carmack, Enze Tian, Michael S. Sinha, David Krikorian, et al
Scientific Reports 11, no. 1 (January 21, 2021): 2051.
Up Is Down – Pharmaceutical Industry Caution vs. Federal Acceleration of Covid-19 Vaccine Approval
Avorn, Jerry, and Aaron S. Kesselheim.
The New England Journal of Medicine 383, no. 18 (October 29, 2020): 1706–8.
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.
Applying the Proportionality Principle to COVID-19 Antibody Testing
Gunnarsdóttir, H. D., M. S. Sinha, S. Gerke, and T. Minssen
Journal of Law and the Biosciences 7, no. 1 (June 2020): lsaa058.
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.
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Pediatric drug and device development

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

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
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

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.
Time to Publication of Cost-Effectiveness Analyses for Medical Devices
Everhart, Alexander O
The American Journal of Managed Care 29, no. 5 (May 2023): 265–68
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).
Dynamic Dossier in the Cloud: A Sociotechnical Architecture for a Real-Time and Metrics-Based Data Tracking System with Gene and Cell Therapies as a Case Study
Nam, Kevin, Kay Larholt, Gigi Hirsch, Paul Beninger, David Fritsche, Diane Shoda, John Ferguson, et al
Therapeutic Innovation & Regulatory Science, October 28, 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.
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Biomarkers and surrogate markers in drug trials

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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