In developed nations, public health agencies typically use data from randomized controlled trials to evaluate new drugs. However, these trials routinely exclude populations to which clinicians prescribe approved drugs, meaning some patients are treated with drugs, which were approved on the basis of another group's treatment response. Despite having opportunities to change, some health agencies have not mandated greater inclusion in drug trials and appear to prefer remaining ignorant of some populations' treatment effects when approving a drug. To explore this decision, I introduce a novel mechanism by which a health agency would choose to be intentionally agnostic regarding a population's treatment response. The main contribution of this paper is in showing how ambiguity about on-label and off-label prescription rates could possibly encourage population exclusion in drug approval trials even in the absence of concerns about trial necessity or cost.
Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.1002/hec.3964 | DOI Listing |
Publication Analysis
Top Keywords
Similar Publications
Targeting Protein-Protein Interactions in Hematologic Malignancies.
Annu Rev Pathol
January 2025
Department of Pathology, University of Michigan, Ann Arbor, Michigan, USA; email:
Over the last two decades, there have been extensive efforts to develop small-molecule inhibitors of protein-protein interactions (PPIs) as novel therapeutics for cancer, including hematologic malignancies. Despite the numerous challenges associated with developing PPI inhibitors, a significant number of them have advanced to clinical studies in hematologic patients in recent years. The US Food and Drug Administration approval of the very first PPI inhibitor, venetoclax, demonstrated the real clinical value of blocking protein-protein interfaces.
View Article and Find Full Text PDFDrug-Induced Liver Injury Associated With Emerging Cancer Therapies.
Liver Int
February 2025
Department of Clinical Pharmacology and Toxicology, University Hospital Zürich, University of Zürich, Zürich, Switzerland.
Targeted therapies and immunotherapies have shown great promise as best-in-class treatments for several cancers with respect to efficacy and safety. While liver test abnormalities are rather common in patients treated with kinase inhibitors or immunotherapy, events of severe hepatotoxicity in these patients are rare in comparison with those associated with chemotherapeutics. The underlying mechanisms and risk factors for severe hepatotoxicity with novel oncology therapies are not well understood, complicating the drug-induced liver injury (DILI) risk assessment in the preclinical and clinical phases of drug development.
View Article and Find Full Text PDFMETTL3: a multifunctional regulator in diseases.
Mol Cell Biochem
January 2025
Department of Vascular Surgery, Renmin Hospital of Wuhan University, Wuhan, Hubei, China.
N6-methyladenosine (mA) methylation is the most prevalent and abundant internal modification of mRNAs and is catalyzed by the methyltransferase complex. Methyltransferase-like 3 (METTL3), the best-known mA methyltransferase, has been confirmed to function as a multifunctional regulator in the reversible epitranscriptome modulation of mA modification according to follow-up studies. Accumulating evidence in recent years has shown that METTL3 can regulate a variety of functional genes, that aberrant expression of METTL3 is usually associated with many pathological conditions, and that its expression regulatory mechanism is related mainly to its methyltransferase activity or mRNA posttranslational modification.
View Article and Find Full Text PDFNeuroprotective Effects, Mechanisms of Action and Therapeutic Potential of the Kv7/KCNQ Channel Opener QO-83 in Ischemic Stroke.
Transl Stroke Res
January 2025
Department of Pharmacology, The Key Laboratory of Neural and Vascular Biology, Ministry of Education, The Key Laboratory of New Drug Pharmacology and Toxicology, Hebei Medical University, Shijiazhuang, 050017, Hebei, China.
Ischemic stroke is a worldwide disease with high mortality and morbidity. Kv7/KCNQ channels are key modulators of neuronal excitability and microglia function, and activation of Kv7/KCNQ channels has emerged as a potential therapeutic avenue for ischemic stroke. In the present study, we focused on a new Kv7/KCNQ channel opener QO-83 on the stroke outcomes and its therapeutic potential.
View Article and Find Full Text PDFAging Concerns Related to Sexuality and Gender: HIV Prevention and Healthy Aging.
AIDS Behav
January 2025
Department of Human Development and Family Sciences, University of Connecticut, Storrs, CT, USA.
Healthy aging is an important area of research across many populations, but less work has focused on this area among sexual and gender diverse individuals relative to the general population. On the whole, it is known that as the U.S.
View Article and Find Full Text PDFWant AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!