Article
Medicine, General & Internal
Kenji Omae, Akira Onishi, Ethan Sahker, Toshi A. Furukawa
Summary: This study evaluated the accelerated approval program for nononcology drug indications over a 26-year period. The findings showed that the program expedited the approval time for nononcology drug indications but safety-related label modifications were often required after approval, and clinical efficacy was sometimes not confirmed.
Article
Medicine, General & Internal
Jinping Xie, Jinlian Li, Yue Liu, Haoyang Wang, Yifei Wang, Yifan Yang, Yi Chen, Rong Jiang, Rong Shao
Summary: This study analyzed the differences in drug review decisions made by the FDA's accelerated approval (AA) pathway and the EMA's conditional marketing authorization (CMA) pathway for dual-approved oncology drugs. The study found differences in approval decisions, pivotal clinical trials, review speed, and postmarketing obligations. The FDA and EMA have different orientations and considerations for benefit-risk balance in the use of AA or CMA, and the design and implementation of postmarketing studies pose challenges in obtaining the necessary evidence to confirm a drug's benefits.
Letter
Oncology
Ravi B. B. Parikh, Rebecca A. A. Hubbard, Erkuan Wang, Trevor J. J. Royce, Aaron B. B. Cohen, Amy S. S. Clark, Ronac Mamtani
Summary: This study examines patient exposure to oncology drugs that have been withdrawn from the US FDA Accelerated Approval program.
Article
Medicine, General & Internal
Avi Cherla, Huseyin Naci, Aaron S. Kesselheim, Bishal Gyawali, Elias Mossialos
Summary: This study compared FDA accelerated approval decisions for cancer drugs with NICE coverage decisions, revealing that 30 cancer drug indications granted accelerated approval by the FDA were not subsequently reviewed by European regulators or NICE, and 12 drugs were denied authorization or coverage due to insufficient safety, clinical efficacy, or cost-effectiveness. Coverage of cancer drugs by the National Health Service commonly required additional price concessions, restrictions to approved indications, or review of additional data.
JAMA INTERNAL MEDICINE
(2021)
Article
Pharmacology & Pharmacy
Walter Schmitt, Hauke Ruehs, Rolf Burghaus, Christian Diedrich, Sulav Duwal, Thomas Eissing, Dirk Garmann, Michaela Meyer, Bart Ploeger, Joerg Lippert
Summary: The study demonstrates that NT-proBNP can serve as a surrogate for clinical outcomes in HF trials, with a well-described relationship between NT-proBNP concentration and clinical events using an E-max model. Quantitative analysis provided insights into potential risks and risks associated with clinical events. Model-based predictions solely based on NT-proBNP measurements match final trial results, and early NT-proBNP measurements enable shorter yet reliable HF trials.
CLINICAL PHARMACOLOGY & THERAPEUTICS
(2021)
Article
Oncology
Donald C. Moore, Joseph B. Elmes, Scott A. Strassels, Jai N. Patel
Summary: Patient-reported outcomes (PROs) are not consistently utilized and lack harmonization in oncology drug clinical trials leading to initial accelerated approval.
SUPPORTIVE CARE IN CANCER
(2023)
Article
Oncology
Shai Gilboa, Yarden Pras, Aviv Mataraso, David Bomze, Gal Markel, Tomer Meirson
Summary: This study analyzed the patterns of censoring in surrogate endpoints in oncology RCTs between 2010 and 2020. Results showed that censoring imbalance between study arms could potentially affect the validity of the results, especially in trials where new treatments failed to demonstrate survival benefit.
EUROPEAN JOURNAL OF CANCER
(2021)
Article
Oncology
Brooke E. Wilson, Alexandra Desnoyers, Michelle B. Nadler, Eitan Amir, Christopher M. Booth
Summary: This retrospective cohort study found that older adults are under-represented in oncology registration trials, and there were no significant differences in outcomes between age groups in most individual trials. However, the researchers emphasized the need for increased enrollment and further research into differential treatment effects in older patients.
Editorial Material
Multidisciplinary Sciences
Kevin Klein, Gerrit Borchard, Vinod P. Shah, Beat Fluhmann, Scott E. McNeil, Jon S. B. de Vlieger
Summary: The development of regulatory guidelines for complex generic drug products faces challenges, but existing FDA pathways can be used to approve them. Decisions on which application to submit can be based on FDA classification and guidance document. The aim is to increase clarity on regulatory approaches for complex generics and promote sustainability.
ANNALS OF THE NEW YORK ACADEMY OF SCIENCES
(2021)
Article
Medicine, General & Internal
Mark P. Lythgoe, Aakash Desai, Bishal Gyawali, Philip Savage, Jonathan Krell, Jeremy L. Warner, Ali Raza Khaki
Summary: This study compared the market authorization dates for new oncology therapies approved in the US and Europe over the past decade, and examined and contrasted the regulatory activities of the FDA and EMA in the approval of new cancer medicines. The results showed that the US approved oncology therapies earlier than Europe, with the FDA receiving licensing applications sooner and having shorter review times.
Article
Immunology
Amanda Minter, Lorenzo Pellis, Graham F. Medley, T. Deirdre Hollingsworth
Summary: As countries strive to meet WHO goals for reducing morbidity and eliminating disease transmission, they will need to plan for the next stages of surveillance and control in low prevalence settings. Going beyond simple predictions of treatment program effectiveness, more nuanced intervention choices informed by quantitative analyses will be necessary for sustainable gains in the long term. Identifying policy goals and finding the right balance between intervention and surveillance constraints will be key challenges in achieving multiple aims such as minimizing morbidity and costs simultaneously.
CLINICAL INFECTIOUS DISEASES
(2021)
Article
Oncology
Timothee Olivier, Alyson Haslam, Vinay Prasad
Summary: This study aimed to estimate the prevalence of unequal rules for dose modification or the use of myeloid growth factors in head-to-head registration trials. The results showed that about 55% of the trials favored the experimental arm, raising questions about the superiority of new molecules and the impact of dosing or growth factor support.
EUROPEAN JOURNAL OF CANCER
(2022)
Article
Psychology, Multidisciplinary
Pia Berlin, Nico Leppin, Katharina Nagelschmidt, Carola Seifart, Winfried Rief, Pia von Blanckenburg
Summary: The study aimed to provide a valid instrument to assess people's readiness for end-of-life conversations. Exploratory factor analysis in a community sample identified three subscales: readiness, communication, and values. Structural equation modeling in a population affected by cancer led to adjustments in the questionnaire structure with a focus on readiness.
FRONTIERS IN PSYCHOLOGY
(2021)
Article
Medicine, General & Internal
Joel Lexchin
Summary: This study examined multiple aspects of new drug approval, including drug characteristics, patient demographics in clinical trials, trial characteristics, and review types. The results showed that there have been changes in regulatory standards and some improvement in data transparency.
FRONTIERS IN MEDICINE
(2023)
Article
Economics
Mackenzie Mills
Summary: Conditionally approved drugs are more likely to face rejection or delays in HTA approval compared to drugs with standard marketing authorisation. These drugs have lower probabilities of receiving HTA approval and face moderate delays in the approval process. Uncertainties in clinical and economic evidence are raised more frequently for conditionally approved drugs.
Article
Hematology
Aaron M. Goodman, Myung S. Kim, Vinay Prasad
Summary: Over the past decade, there have been advancements in the treatment of multiple myeloma and its precursor diseases, including expanding the definition to include patients without end organ damage and treating high-risk, smoldering cases. Despite the potential benefits, such as improved survival and quality of life, the implications of early treatment in this context remain uncertain.
Editorial Material
Medicine, General & Internal
Kerrington Powell, Vinay Prasad
EUROPEAN JOURNAL OF CLINICAL INVESTIGATION
(2022)
Review
Medicine, General & Internal
Audra N. Iness, Jefferson O. Abaricia, Wendemi Sawadogo, Caleb M. Iness, Max Duesberg, John Cyrus, Vinay Prasad
Summary: This review examined the impact of hospital visitor restrictions during the COVID-19 pandemic and found that blanket policies failed to address the unique needs of patients, visitors, and healthcare providers. A patient-centered and thoughtful approach to visitor policies is recommended for maximum benefit to all stakeholders.
AMERICAN JOURNAL OF MEDICINE
(2022)
Editorial Material
Medicine, General & Internal
Kerrington Powell, Vinay Prasad
EUROPEAN JOURNAL OF CLINICAL INVESTIGATION
(2022)
Letter
Oncology
Timothee Olivier, Vinay Prasad
JOURNAL OF CLINICAL ONCOLOGY
(2022)
Editorial Material
Medicine, General & Internal
Kerrington Powell, Vinay Prasad
Summary: Randomized controlled trials are highly valued in biomedicine for their ability to minimize confounding factors. However, there are misconceptions in oncology regarding the difficulty, time, cost, industry influence, and ethical standards of conducting these trials. Potential solutions include improving trial design, establishing regulatory and strategic frameworks, and addressing ethical concerns.
EUROPEAN JOURNAL OF CLINICAL INVESTIGATION
(2022)
Article
Oncology
Kristin Wright, Daniel E. E. Meyers, Timothy M. M. Chisamore, Matthew D. F. McInnes, Sergio Sismondo, Bishal Gyawali, Vinay Prasad, Christopher M. M. Booth
Summary: A small number of medical oncologists receive very high payments from the pharmaceutical industry, and they hold major leadership roles within oncology. Further research is needed to explore the potential impact of these conflicts of interest on clinical practice and policy.
JCO ONCOLOGY PRACTICE
(2022)
Review
Medicine, General & Internal
Benjamin Knudsen, Vinay Prasad
Summary: Myocarditis is a significant adverse event associated with COVID-19 vaccination, particularly for men under 40. The risk of myocarditis may be underestimated for high-risk groups and overestimated for low-risk groups if not stratified by relevant risk factors. Our study assessed how the risk of myocarditis is reported in the literature.
EUROPEAN JOURNAL OF CLINICAL INVESTIGATION
(2023)
Editorial Material
Oncology
Kerrington Powell, John Marquart, Timothee Olivier, Vinay Prasad
Summary: In this paper, a framework is proposed for conducting randomized controlled trials (RCTs) to assess the effectiveness of metastasectomy, regardless of histology or anatomy. The focus is on factors such as growth rate kinetics to determine the therapeutic efficacy of surgical intervention. This strategy may help identify target patients who will benefit from surgery, regardless of their specific tumor type.
Article
Medicine, General & Internal
Alyson Haslam, Timothee Olivier, Vinay Prasad
Summary: There is currently little consensus on the specific definition of long COVID, but various organizations have proposed different definitions. This study aimed to examine the definition of long COVID used in current clinical trials. The findings showed that 54.3% of the studies only confirmed COVID-19 diagnosis through laboratory testing. There were eight different time durations specified for symptom persistence, and a total of 57 symptoms were identified across the studies. Only 8.7% of the trials adhered to NICE or WHO definitions.
EUROPEAN JOURNAL OF CLINICAL INVESTIGATION
(2023)
Editorial Material
Oncology
Anushka Walia, Vinay Prasad
BLOOD CANCER JOURNAL
(2023)
Article
Health Care Sciences & Services
Audrey A. Tran, Vinay Prasad
Summary: Total knee arthroplasty is a common and costly elective operation, with more than 600,000 surgeries performed annually in the USA alone. Despite the high patient satisfaction rate, there is a lack of solid evidence supporting the effectiveness of this procedure. Sham-controlled surgical trials are needed to provide a more accurate assessment of its necessity.
JOURNAL OF COMPARATIVE EFFECTIVENESS RESEARCH
(2023)
Article
Oncology
Jordan Tuia, Alyson Haslam, Vinay Prasad
Summary: The purpose of this study was to determine the prevalence of attrition and the frequency of transition from clinical to industry-related roles among oncology physicians. The results showed that 21% of the oncologists had stopped billing by 2022, and 35% of the sampled physicians were working in the industry.
JCO ONCOLOGY PRACTICE
(2023)
Article
Oncology
Anushka Walia, Jordan Tuia, Vinay Prasad
Summary: Composite outcome measures are commonly used in oncology research, but they have limitations such as vague definitions and complicated result comparisons. Knowing the breakdown of component events is essential for accurate interpretation of trial results and assessing the true benefit of interventions.
NATURE REVIEWS CLINICAL ONCOLOGY
(2023)
Article
Medicine, General & Internal
Tracy Beth Hoeg, Shamez Ladhani, Vinay Prasad
BMJ EVIDENCE-BASED MEDICINE
(2023)