A5074025128- Economic and Financial Impacts of Cancer
- Health Systems, Economic Evaluations, Quality of Life
- Renal cell carcinoma treatment
- Medical Imaging Techniques and Applications
- Cancer survivorship and care
- PI3K/AKT/mTOR signaling in cancer
- Neuroblastoma Research and Treatments
- CAR-T cell therapy research
- AI in cancer detection
- Glioma Diagnosis and Treatment
- Radiation Shielding Materials Analysis
- Statistical Methods and Inference
- Advanced Causal Inference Techniques
- Statistical Methods in Clinical Trials
- Advanced Breast Cancer Therapies
- Lymphoma Diagnosis and Treatment
- Social Media in Health Education
- HER2/EGFR in Cancer Research
- Chronic Lymphocytic Leukemia Research
- Bone health and osteoporosis research
- Neurofibromatosis and Schwannoma Cases
- Statistical Methods and Bayesian Inference
- Infrared Thermography in Medicine
United States Food and Drug Administration
2024
Center for Drug Evaluation and Research
2024
Abstract Introduction As patient-reported outcomes (PROs) are increasingly used in the evaluation of medical treatments, it is important that PROs carefully analyzed and interpreted. This may be challenging due to substantial missing values. The missingness often closely related patients’ disease status. In case, using observed information about intercurrent events (ICEs) such as progression death will improve handling PRO data. Therefore, aim this study was develop imputation models for...
PURPOSE The US Food and Drug Administration (FDA) approved capivasertib in combination with fulvestrant for adult patients hormone receptor–positive, human epidermal growth factor receptor 2 (HER2)–negative, locally advanced, or metastatic breast cancer (MBC) who have received at least one previous endocrine therapy whose tumors harbor more phosphatidylinositol 3-kinase ( PIK3CA )/AKT Serine/Threonine Kinase 1 AKT1 )/phosphatase tensin homolog PTEN ) alterations, as detected by an...
On December 14, 2023, the United States Food and Drug Administration (FDA) approved belzutifan (Welireg, Merck & Co., Inc.) for patients with advanced renal cell carcinoma (RCC) following a programmed death receptor-1 or death-ligand 1 (PD-1/PD-L1) inhibitor vascular endothelial growth factor tyrosine kinase (VEGF-TKI). FDA granted traditional approval based on LITESPARK-005 (NCT04195750), an open-label, randomized, head-to-head trial of 746 RCC that progressed both PD-1/PD-L1 VEGF-TKI....
Abstract In October 2022, the FDA Oncology Center of Excellence hosted an educational symposium entitled, “Considering Functional Outcomes as Efficacy Endpoints in Pediatric Low-Grade Glioma (pLGG) Clinical Trials.” The brought together patient advocates, regulators from and European Medicines Agency (EMA), international group academic thought leaders field pediatric neuro-oncology to discuss potential role functional outcomes, including visual acuity, motor function, neurocognitive...
In April 2023, the U.S. Food and Drug Administration granted regular approval to polatuzumab vedotin-piiq in combination with a rituximab product, cyclophosphamide, doxorubicin, prednisone (pola+R-CHP) for adult patients who have previously untreated diffuse large B-cell lymphoma, not otherwise specified or high-grade lymphoma an International Prognostic Index score of 2 greater. Approval was based on POLARIX, randomized, double-blinded, placebo-controlled trial evaluating superiority...
Abstract Background Patient-reported outcomes (PROs) play an increasing role in the evaluation of oncology treatments. At same time, single-arm trials are commonly included regulatory approval submissions. Because high risk biases, results from require careful interpretation. This benefits a clearly defined estimand , or target estimation. In this case study, we demonstrated how ICH E9 (R1) framework can be implemented SATs with PRO endpoints. Methods For global quality life outcome real...
<title>Abstract</title> <bold>Background</bold> The US Food and Drug Administration (FDA) released a draft guidance document detailing core patient-reported outcomes in cancer clinical trials, including physical function (PF). This study aimed to develop analytic methods visualizations of PF patients with cancer. <bold>Methods</bold> We applied an estimand framework tolerability endpoint data summaries cross-sectionally over time, along visualizations. accomplished this through iterative...
<div>Abstract<p>On December 14, 2023, the U.S. FDA approved belzutifan (Welireg, Merck & Co., Inc.) for patients with advanced renal cell carcinoma (RCC) following treatment a PD-1/PD-L1 inhibitor and VEGF tyrosine kinase inhibitor. The granted traditional approval based on LITESPARK-005 (NCT04195750), an open-label, randomized, head-to-head trial of 746 RCC that progressed both Patients were randomized (1:1) to receive or everolimus. primary endpoints progression-free...