Use of suboptimal control arms in randomized clinical trials of investigational cancer drugs in China, 2016-2021: An observational study
CONCLUSIONS: In this study, over one-eighth of randomized trials of cancer drugs registered to apply for regulatory approval in China used a suboptimal comparator. Our results highlight the necessity to refine the design of randomized trials to generate optimal clinical evidence for new cancer therapies.PMID:38085706 | DOI:10.1371/journal.pmed.1004319 (Source: Cancer Control)
Source: Cancer Control - December 12, 2023 Category: Cancer & Oncology Authors: Yichen Zhang Dingyi Chen Siyuan Cheng Zhizhou Liang Lu Yang Qian Li Lin Bai Huangqianyu Li Wei Liu Luwen Shi Xiaodong Guan Source Type: research
Use of suboptimal control arms in randomized clinical trials of investigational cancer drugs in China, 2016 –2021: An observational study
ConclusionsIn this study, over one-eighth of randomized trials of cancer drugs registered to apply for regulatory approval in China used a suboptimal comparator. Our results highlight the necessity to refine the design of randomized trials to generate optimal clinical evidence for new cancer therapies. (Source: PLoS Medicine)
Source: PLoS Medicine - December 12, 2023 Category: Internal Medicine Authors: Yichen Zhang Source Type: research
Kilogram ‐Scale Synthesis of Extremely Small Gadolinium Oxide Nanoparticles as a T1‐Weighted Contrast Agent for Magnetic Resonance Imaging
In this study, polymaleic acid (PMA) is found to be an ideal stabilizer to synthesize ES-GdONs. Compared with ES-GdON-PAA, the PMA-stabilized ES-GdON (ES-GdON-PMA) has a lowerr2/r1 ratio (2.05, 7.0 T) and a lower blood circulation half-life (37.51 min). The optimized ES-GdON-PMA-9 has an exceedingly small particle size (2.1 nm), excellent water dispersibility, and stability. A facile, efficient, and environmental friendly synthetic method is developed for large-scale synthesis of the ES-GdONs-PMA. The weight of the optimized freeze–dried ES-GdON-PMA-26 synthesized in a 20 L of reactor reaches the kilogram level. The ...
Source: Small - November 21, 2023 Category: Nanotechnology Authors: Lihe Wu,
Xuanyi Lu,
Yudie Lu,
Meng Shi,
Shuai Guo,
Jie Feng,
Sugeun Yang,
Wei Xiong,
Yikai Xu,
Chenggong Yan,
Zheyu Shen Tags: Research Article Source Type: research
Retrospective analysis of the biopharmaceutics characteristics of solid oral Modified-Release drug products in approved US FDA NDAs designated as Extended-Release or Delayed-Release formulations
CONCLUSION: The results of this study help the reader understand the design, characteristics, and pharmacological advantages of the ER and DR drug products for patient benefit; as well as the regulations governing the FDA's assessment of ER claims.PMID:37984592 | DOI:10.1016/j.ejpb.2023.11.014 (Source: European Journal of Pharmaceutics and Biopharmaceutics)
Source: European Journal of Pharmaceutics and Biopharmaceutics - November 20, 2023 Category: Drugs & Pharmacology Authors: Ahmed Nadia Ly Holly Pan Amanda Chiang Brian Raines Kimberly Janwatin Timothy Hamed Salaheldin Dave Kaushalkumar Source Type: research
Retrospective analysis of the biopharmaceutics characteristics of solid oral Modified-Release drug products in approved US FDA NDAs designated as Extended-Release or Delayed-Release formulations
CONCLUSION: The results of this study help the reader understand the design, characteristics, and pharmacological advantages of the ER and DR drug products for patient benefit; as well as the regulations governing the FDA's assessment of ER claims.PMID:37984592 | DOI:10.1016/j.ejpb.2023.11.014 (Source: European Journal of Pharmaceutics and Biopharmaceutics)
Source: European Journal of Pharmaceutics and Biopharmaceutics - November 20, 2023 Category: Drugs & Pharmacology Authors: Ahmed Nadia Ly Holly Pan Amanda Chiang Brian Raines Kimberly Janwatin Timothy Hamed Salaheldin Dave Kaushalkumar Source Type: research
Retrospective analysis of the biopharmaceutics characteristics of solid oral Modified-Release drug products in approved US FDA NDAs designated as Extended-Release or Delayed-Release formulations
CONCLUSION: The results of this study help the reader understand the design, characteristics, and pharmacological advantages of the ER and DR drug products for patient benefit; as well as the regulations governing the FDA's assessment of ER claims.PMID:37984592 | DOI:10.1016/j.ejpb.2023.11.014 (Source: European Journal of Pharmaceutics and Biopharmaceutics)
Source: European Journal of Pharmaceutics and Biopharmaceutics - November 20, 2023 Category: Drugs & Pharmacology Authors: Ahmed Nadia Ly Holly Pan Amanda Chiang Brian Raines Kimberly Janwatin Timothy Hamed Salaheldin Dave Kaushalkumar Source Type: research
Patents and regulatory exclusivities on FDA-approved insulin products: A longitudinal database study, 1986 –2019
ConclusionsAmong several strategies that insulin manufacturers have employed to extend periods of market exclusivity on brand-name insulin products are filing patents after FDA approval and obtaining a large number of patents on delivery devices. Policy reforms are needed to promote timely competition in the pharmaceutical market and ensure that patients have access to low-cost drugs. (Source: PLoS Medicine)
Source: PLoS Medicine - November 16, 2023 Category: Internal Medicine Authors: Anders Olsen Source Type: research
Rare oncology therapeutics: review of clinical pharmacology package of drug approvals (2019 –2023) by US FDA, best practices and recommendations
The objective was to understand the expected clinical pharmacology studies and knowledge base in such approvals. Model informed drug development (MIDD) applications were also reviewed, as such approaches are expected to play a critical role in filling clinical pharmacology gaps in rare oncology, where number of clinical trials and size of these trials will perhaps continue to be small. The findings highlighted how clinical pharmacology contributed to the evidence of effectiveness, dose optimization and elucidation of intrinsic and extrinsic factors affecting drug ’s behavior. Clinical pharmacology studies were often inte...
Source: Journal of Pharmacokinetics and Pharmacodynamics - November 4, 2023 Category: Drugs & Pharmacology Source Type: research
FDA Accepts NDA for Investigational RA Agent
In August, the U.S. Food & Drug Administration (FDA) accepted an investigational new drug application for MYMD-1, an oral tumor necrosis factor (TNF) α inhibitor that crosses the blood-brain barrier. Currently in pre-clinical studies, MYMD-1 is designed to regulate the immune system by selectively blocking TNF-α when it becomes overactivated during a cytokine storm and... (Source: The Rheumatologist)
Source: The Rheumatologist - November 1, 2023 Category: Rheumatology Authors: Natasha Yetman Tags: Biologics/DMARDs Drug Updates tumor necrosis factor (TNF) inhibitors U.S. Food and Drug Administration (FDA) Source Type: research
Preclinical studies supporting a first-in-human trial of pulmonary macrophage transplantation therapy of CSF2RA-associaited PAP
CSF2RA mutations cause hereditary pulmonary alveolar proteinosis (hPAP), a disorder of alveolar macrophage dysfunction, surfactant accumulation, and hypoxemic respiratory failure, and, in some patients, serious infections or pulmonary fibrosis - without an approved disease-specific therapy. After reporting hPAP in 2008, we developed diagnostics, identified a patient cohort, and defined its pathogenesis, presentation, and natural history. We discovered that lentiviral-mediated CSF2RA gene transfer restored GM-CSF signaling in hPAP patient macrophages. We developed and validated Csf2raKO mice as a model o...
Source: European Respiratory Journal - October 27, 2023 Category: Respiratory Medicine Authors: Arumugam, P., Carey, B., Trump-Durbin, L., Wessendarp, M., Suzuki, T., Spoorthi, F., Ma, Y., Shima, K., Stock, J., Chalk, C., Wikenheiser-Brokamp, K., Lutzko, C., Trapnell, B. Tags: Rare ILD/DPLD Source Type: research
Feasibility of Emulating Trials Supporting Approvals of Drugs and Biologics
This cross-sectional study evaluates the supporting clinical trials for supplemental new drug applications and supplemental biologics license applications from 2017 to 2019. (Source: JAMA Internal Medicine)
Source: JAMA Internal Medicine - October 2, 2023 Category: Internal Medicine Source Type: research
Use of patient-reported outcome measures for oncology drugs receiving accelerated approval
The objective was to assess whether the trials leading to such an approval included PROs. Between 2013 and 2022, the FDA approved 59 unique drugs for an oncology indication via the accelerated app roval pathway, and 35 (59%) included a PRO assessment in the clinical trial. A median of 1 PRO measurement was used in each trial, with 23 different types of PRO assessment tools were used across the 59 new drug applications. In summary, we found that PRO measurements are inconsistently utilized in trials leading to initial accelerated approval of oncology drugs, and there seems to be a lack of harmonization of different PRO meas...
Source: Supportive Care in Cancer - September 29, 2023 Category: Cancer & Oncology Source Type: research
Characterization of the US Food and Drug Administration Post-Marketing Commitments and Requirements for Pregnancy and Lactation
Clin Pharmacol Ther. 2023 Sep 26. doi: 10.1002/cpt.3059. Online ahead of print.ABSTRACTEnactment of the Food and Drug Administration Amendments Act (FDAAA) in 2007 and the Pregnancy and Lactation Labeling Rule (PLLR) in 2015 are important milestones giving the FDA the authority to request studies in pregnant and lactating women. Our objective was to characterize trends in pregnancy and lactation related post-marketing commitments (PMCs) and post-marketing requirements (PMRs) for new molecular entities approved by the FDA between 2000-2022. Approval letters of original New Drug Applications (NDAs) for new molecular entities...
Source: Clinical Pharmacology and Therapeutics - September 26, 2023 Category: Drugs & Pharmacology Authors: Charul Avachat Islam R Younis Angela K Birnbaum Source Type: research
Characterization of the US Food and Drug Administration Post-Marketing Commitments and Requirements for Pregnancy and Lactation
Clin Pharmacol Ther. 2023 Sep 26. doi: 10.1002/cpt.3059. Online ahead of print.ABSTRACTEnactment of the Food and Drug Administration Amendments Act (FDAAA) in 2007 and the Pregnancy and Lactation Labeling Rule (PLLR) in 2015 are important milestones giving the FDA the authority to request studies in pregnant and lactating women. Our objective was to characterize trends in pregnancy and lactation related post-marketing commitments (PMCs) and post-marketing requirements (PMRs) for new molecular entities approved by the FDA between 2000-2022. Approval letters of original New Drug Applications (NDAs) for new molecular entities...
Source: Clinical Pharmacology and Therapeutics - September 26, 2023 Category: Drugs & Pharmacology Authors: Charul Avachat Islam R Younis Angela K Birnbaum Source Type: research
