Review
Medicine, General & Internal
David Zahrieh, Ivana T. Croghan, Jonathan W. Inselman, Sumithra J. Mandrekar
Summary: This article discusses important considerations for data and safety monitoring in pragmatic randomized clinical trials (pRCTs) and provides practical guidelines for generalists conducting pRCTs.
MAYO CLINIC PROCEEDINGS
(2023)
Review
Immunology
Steven Joffe, Abdel Babiker, Susan S. Ellenberg, Alan Fix, Marie R. Griffin, Sally Hunsberger, Jorge Kalil, Myron M. Levine, Malegapuru W. Makgoba, Renee H. Moore, Anastasios A. Tsiatis, Richard Whitley
Summary: The US Federal Government has funded multiple phase 3 trials of COVID-19 vaccines and established a single DSMB to oversee and coordinate these trials, ensuring safety and shared insights. Challenges include the scale and pace of the trials, safety events related to a large number of participants, many of whom have underlying health risks, and the politicized environment in which the trials are conducted.
JOURNAL OF INFECTIOUS DISEASES
(2021)
Review
Gastroenterology & Hepatology
Elliot B. Tapper, Marina Serper, David S. Goldberg
Summary: Patients with chronic liver disease can benefit from pragmatic trial designs, which aim to provide evidence for the adoption of interventions in real-world clinical practice. The efficiency of identifying, recruiting, and following patients, the resemblance of interventions and design to usual clinical care, and the importance of outcomes to patients are key factors in determining the pragmatism of a trial. This review focuses on the promise, trade-offs, and purpose of pragmatic trials in hepatology.
Article
Cardiac & Cardiovascular Systems
Gail A. Van Norman
Summary: Phase I and II clinical trials are integrating therapeutic and toxicity endpoints more, with some therapeutic agents gaining FDA approval based on early phase trials alone. It is suggested that independent monitoring by DSMB is necessary for phase III studies and possibly extended to many phase I and II clinical trials. Minimum qualifications for DSMB service should be established and standardized.
JACC-BASIC TO TRANSLATIONAL SCIENCE
(2021)
Article
Medicine, General & Internal
Jennifer Le-Rademacher, Heather Gunn, Xiaoxi Yao, Daniel J. Schaid
Summary: Clinical trials are important for evaluating the safety and efficacy of new interventions. Traditional explanatory trials focus on exploring the biological causes and effects, while pragmatic trials consider real-world conditions and aim to optimize interventions in diverse clinical practices. It is essential to maintain scientific rigor, increase trial efficiency, ensure generalizability of results, and address health disparities through technology and decentralized approaches.
MAYO CLINIC PROCEEDINGS
(2023)
Editorial Material
Biochemistry & Molecular Biology
Michael J. Grant, Sarah B. Goldberg
Summary: Innovative clinical trial designs with a patient-centered mission are crucial to advancing modern cancer care. The adaptive phase 2 umbrella study CTONG1702 provides one model for this new era of trial design.
Article
Geriatrics & Gerontology
Joan M. Teno, Laura C. Hanson, Julie C. Lima, Debra Saliba
Summary: Pragmatic clinical trials (PCTs) focus on real-world effectiveness methodology to overcome the limitations of explanatory randomized clinical trials (RCTs), but face the challenge of minimizing burdens on patients and healthcare institutions. PCTs prioritize outcome measures from existing data sources, but this may lead to gaps in safety for vulnerable populations. Proposed ranking systems that penalize PCTs with additional data collection burdens often neglect patient experience measures. This article argues for a risk-based approach to collect additional data in PCTs, using real-life examples of end-of-life care interventions to illustrate potential safety and patient experience outcomes captured.
JOURNAL OF THE AMERICAN GERIATRICS SOCIETY
(2023)
Article
Integrative & Complementary Medicine
Jun Liu, Nian Wang, Hai-xia Dang, Bing-wei Chen, Li Zhang, Chong Zou, Cheng-liang Zhong, Ju-kai Huang, Qiong Liu, Ya-nan Yu, Meng Jiang, Wei-xiong Liang, Qi-guang Chen, Yong-yan Wang, Chun-ti Shen, Zhong Wang
Summary: The paper summarizes the key content of the Chinese Medicine Data Monitoring Committee (CMDMC) community standard, issued by the China Association of Chinese Medicine, to help sponsors of clinical studies establish and operate CMDMCs for enhancing the scientific integrity and quality of clinical studies.
CHINESE JOURNAL OF INTEGRATIVE MEDICINE
(2021)
Article
Medicine, Research & Experimental
Michael J. Martens, Brent R. Logan
Summary: This article provides a general framework for monitoring safety events in clinical trials, surveys common statistical techniques for creating safety stopping criteria, and offers a software tool for constructing and assessing these rules.
Review
Oncology
Bernard Bright Davies-Teye, Michelle Medeiros, Cynthia Chauhan, Claudia Rose Baquet, C. Daniel Mullins
Summary: Oncology trials are increasingly focusing on pragmatism and meaningful patient engagement. This study offers practical guidance on how to achieve both goals, leading to increased participation, faster recruitment, better retention, and adoption of innovative technologies in community-based care.
Article
Medicine, Research & Experimental
Stephanie R. Morain, Juli Bollinger, Kevin Weinfurt, Jeremy Sugarman
Summary: Sharing data in pragmatic clinical trials presents ethical challenges, such as waivers of informed consent, risks for patient-subjects and health systems, use of electronic health records, and limited control over sensitive data. These challenges raise questions about the suitability of traditional research ethics governance structures.
Article
Multidisciplinary Sciences
Chongliang Luo, Jingcheng Du, Adam Cuker, Ebbing Lautenbach, David A. Asch, Gregory A. Poland, Cui Tao, Yong Chen
Summary: Limited studies have been conducted on severe adverse events (AEs) after COVID-19 vaccination due to their rarity and short follow-up. To monitor the safety of COVID-19 vaccines in the U.S., a study compared the rankings of different vaccines using both randomized controlled trials (RCTs) and the Vaccine Adverse Event Reporting System (VAERS) data, and assessed the risks of local and systemic AEs. Consistent rankings were observed between RCT and VAERS, particularly for systemic AEs.
SCIENTIFIC REPORTS
(2022)
Review
Anesthesiology
David J. Hohenschurz-Schmidt, Dan Cherkin, Andrew S. C. Rice, Robert H. Dworkin, Dennis C. Turk, Michael P. McDermott, Matthew J. Bair, Lynn L. DeBar, Robert R. Edwards, John T. Farrar, Robert D. Kerns, John D. Markman, Michael C. Rowbotham, Karen J. Sherman, Ajay D. Wasan, Penney Cowan, Paul Desjardins, McKenzie Ferguson, Roy Freeman, Jennifer S. Gewandter, Ian Gilron, Hanna Grol-Prokopczyk, Sharon H. Hertz, Smriti Iyengar, Cornelia Kamp, Barbara I. Karp, Bethea A. Kleykamp, John D. Loeser, Sean Mackey, Richard Malamut, Ewan McNicol, Kushang V. Patel, Friedhelm Sandbrink, Kenneth Schmader, Lee Simon, Deborah J. Steiner, Christin Veasley, Jan Vollert
Summary: Many questions regarding pain management and health policy can be answered by pragmatic controlled trials, which aim to generate clinically relevant and widely applicable findings. These trials aim to reproduce elements of routine clinical care and address a broader set of external validity questions critical for stakeholders in considering evidence-based treatments.
Article
Pharmacology & Pharmacy
Jeppe Ragnar Andersen, Christoffer von Sehested, Inger Byrjalsen, Sara Popik, Anne Bo Follin, Asger Reinstrup Bihlet
Summary: This study compared the impact of risk-based monitoring (RBM) and classic monitoring (CM) on data quality in clinical trials (CTs) and analyzed the expected impact on CT costs. The results showed that RBM had lower error rates in major efficacy data and major safety data compared to CM, indicating that RBM improves the accuracy of trial results and subject safety.
BRITISH JOURNAL OF CLINICAL PHARMACOLOGY
(2023)
Review
Medicine, General & Internal
P. Schoffski
Summary: The role and use of independent data monitoring committees (IDMCs) has evolved over the past decades, with guidelines issued by regulatory agencies in place. IDMCs are recommended for large clinical trials involving life-threatening diseases, vulnerable populations, or potential harm to trial participants. Their primary role is to ensure participant safety and maintain trial integrity, particularly important in oncology trials.
Article
Psychiatry
Ashli Owen-Smith, Christine Stewart, Musu M. Sesay, Sheryl M. Strasser, Bobbi Jo Yarborough, Brian Ahmedani, Lisa R. Miller-Matero, Stephen C. Waring, Irina Haller, Beth E. Waitzfelder, Stacy A. Sterling, Cynthia Campbell, Rulin C. Hechter, John E. Zeber, Laurel A. Copeland, Jeffrey F. Scherrer, Rebecca Rossom, Greg Simon
Article
Medicine, Research & Experimental
Karen L. Margolis, A. Lauren Crain, Anna R. Bergdall, MarySue Beran, Jeffrey P. Anderson, Leif Solberg, Patrick J. O'Connor, Joann M. Sperl-Hillen, Pamala A. Pawloski, Jeanette Y. Ziegenfuss, Dan Rehrauer, Christine Norton, Patricia Haugen, Beverly B. Green, Zeke McKinney, Amy Kodet, Deepika Appana, Rashmi Sharma, Nicole K. Trower, RaeAnn Williams, Benjamin F. Crabtree
CONTEMPORARY CLINICAL TRIALS
(2020)
Article
Substance Abuse
Gwen Lapham, Denise M. Boudreau, Eric A. Johnson, Jennifer F. Bobb, Abigail G. Matthews, Jennifer McCormack, David Liu, Jeffrey H. Samet, Andrew J. Saxon, Cynthia I. Campbell, Joseph E. Glass, Rebecca C. Rossom, Mark T. Murphy, Ingrid A. Binswanger, Bobbi Jo H. Yarborough, Katharine A. Bradley, Brian Ahmedani, Paul J. Amoroso, Julia H. Arnsten, Gavin Bart, Jordan M. Braciszewski, Chinazo O. Cunningham, Rulin C. Hechter, Viviana E. Horigian, Jane M. Liebschutz, Amy M. Loree, Theresa E. Matson, Jennifer McNeely, Joseph O. Merrill, Thomas F. Northrup, Robert P. Schwartz, Angela L. Stotts, Jose Szapocznik, Manu Thakral, Judith I. Tsui, Mohammad Zare
DRUG AND ALCOHOL DEPENDENCE
(2020)
Article
Psychiatry
Jennifer M. Boggs, Richard C. Lindrooth, Catherine Battaglia, Arne Beck, Debra P. Ritzwoller, Brian K. Ahmedani, Rebecca C. Rossom, Frances L. Lynch, Christine Y. Lu, Beth E. Waitzfelder, Ashli A. Owen-Smith, Gregory E. Simon, Heather D. Anderson
GENERAL HOSPITAL PSYCHIATRY
(2020)
Review
Medicine, General & Internal
Carrie D. Patnode, Leslie A. Perdue, Rebecca C. Rossom, Megan C. Rushkin, Nadia Redmond, Rachel G. Thomas, Jennifer S. Lin
JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION
(2020)
Article
Health Policy & Services
Kritzia Merced, Zac E. Imel, Scott A. Baldwin, Heidi Fischer, Tae Yoon, Christine Stewart, Greg Simon, Brian Ahmedani, Arne Beck, Yihe Daida, Sam Hubley, Rebecca Rossom, Beth Waitzfelder, John E. Zeber, Karen J. Coleman
PSYCHIATRIC SERVICES
(2020)
Article
Substance Abuse
Bobbi Jo H. Yarborough, Scott P. Stumbo, Brian Ahmedani, Rebecca Rossom, Karen Coleman, Jennifer M. Boggs, Gregory E. Simon
Summary: In patients with SUD, those who reported "nearly every day" on the Patient Health Questionnaire item 9 were found to have a higher risk of subsequent suicidal behavior. However, clinicians should be aware that individuals reporting "not at all" are not immune to suicide risk.
JOURNAL OF ADDICTION MEDICINE
(2021)
Review
Psychiatry
Olivia E. Bogucki, Mark D. Williams, Leif I. Solberg, Rebecca C. Rossom, Craig N. Sawchuk
CURRENT PSYCHIATRY REPORTS
(2020)
Article
Medicine, Research & Experimental
Thomas E. Elliott, Patrick J. O'Connor, Stephen E. Asche, Daniel M. Saman, Steven P. Dehmer, Heidi L. Ekstrom, Clayton I. Allen, Joseph A. Bianco, Ella A. Chrenka, Laura A. Freitag, Melissa L. Harry, Anjali R. Truitt, JoAnn M. Sperl-Hillen
Summary: This study aims to improve the delivery of primary and secondary cancer prevention services through CPW-CDS and CPW-CDS plus SDMTs, assess the cost, and describe critical facilitators and barriers. The trial and analyses are ongoing with results expected in 2021.
CONTEMPORARY CLINICAL TRIALS
(2021)
Article
Health Care Sciences & Services
Judith Tsui, Mary A. Akosile, Gwen T. Lapham, Denise M. Boudreau, Eric A. Johnson, Jennifer F. Bobb, Ingrid A. Binswanger, Bobbi Jo H. Yarborough, Joseph E. Glass, Rebecca C. Rossom, Mark T. Murphy, Chinazo O. Cunningham, Julia H. Arnsten, Manu Thakral, Andrew J. Saxon, Joseph O. Merrill, Jeffrey H. Samet, Gavin B. Bart, Cynthia Campbell, Amy M. Loree, Angela Silva, Angela L. Stotts, Brian Ahmedani, Jordan M. Braciszewski, Rulin C. Hechter, Thomas F. Northrup, Viviana E. Horigian, Katharine A. Bradley
Summary: This study investigated the prevalence of diagnosed OUD and treatment among primary care patients with HCV or HIV, finding that patients with HCV and HIV were more likely to have OUD compared to those without, and HIV patients were less likely to receive medication treatment for OUD.
JOURNAL OF GENERAL INTERNAL MEDICINE
(2021)
Article
Psychiatry
Deepak Prabhakar, Edward L. Peterson, Yong Hu, Simran Chawa, Rebecca C. Rossom, Frances L. Lynch, Christine Y. Lu, Beth E. Waitzfelder, Ashli A. Owen-Smith, L. Keoki Williams, Arne Beck, Gregory E. Simon, Brian K. Ahmedani
Summary: This study examines suicide attempts requiring medical attention as a risk factor for suicide death in the US general population. The results show that prior suicide attempts associated with medical visits significantly increase the risk of suicide death, especially for women and children aged 10-14 years.
CRISIS-THE JOURNAL OF CRISIS INTERVENTION AND SUICIDE PREVENTION
(2021)
Article
Psychology, Clinical
Joan M. Cook, John E. Zeber, Vanessa Simiola, Rebecca Rossom, Jeffrey F. Scherrer, Ashli A. Owen-Smith, Brian K. Ahmedani, Kiumars Zolfaghari, Laurel A. Copeland
Summary: The study compared the clinical characteristics and health care utilization of PTSD patients diagnosed in primary care versus specialty mental health care. Patients diagnosed by specialty mental health care providers had higher rates and longer duration of psychotherapy, while prescription fill levels for psychotropics were generally higher for patients in specialty mental health care. Strategies are needed to better align patient needs with access and treatment modality in primary care settings.
JOURNAL OF CLINICAL PSYCHOLOGY IN MEDICAL SETTINGS
(2021)
Article
Health Care Sciences & Services
Rachel Gold, Mary Middendorf, John Heintzman, Joan Nelson, Patrick O'Connor, Joann Sperl-Hillen, Deepika Appana, Erik Geissal, Vijay Thirumalai, Christina Sheppler, Maryjane Dunne
HEALTHCARE-THE JOURNAL OF DELIVERY SCIENCE AND INNOVATION
(2020)
Article
Psychology, Clinical
Denise M. Boudreau, Gwen Lapham, Eric A. Johnson, Jennifer F. Bobb, Abigail G. Matthews, Jennifer McCormack, David Liu, Cynthia Campbell, Rebecca C. Rossom, Ingrid A. Binswanger, Bobbi Jo Yarborough, Julia H. Arnsten, Chinazo O. Cunningham, Joseph E. Glass, Mark T. Murphy, Mohammad Zare, Rulin C. Hechter, Brian Ahmedani, Jordan M. Braciszewski, Viviana E. Horigian, Jose Szapocznik, Jeffrey H. Samet, Andrew J. Saxon, Robert P. Schwartz, Katharine A. Bradley
JOURNAL OF SUBSTANCE ABUSE TREATMENT
(2020)
Article
Substance Abuse
Frances L. Lynch, Edward L. Peterson, Christine Y. Lu, Yong Hu, Rebecca C. Rossom, Beth E. Waitzfelder, Ashli A. Owen-Smith, Samuel Hubley, Deepak Prabhakar, L. Keoki Williams, Arne Beck, Gregory E. Simon, Brian K. Ahmedani
ADDICTION SCIENCE & CLINICAL PRACTICE
(2020)