Results 41 to 50 of about 757,641 (252)

An empirical analysis of overall survival in drug approvals by the US FDA (2006–2023)

Cancer Medicine
Background The US Food and Drug Administration (FDA) has expanded the use of surrogate markers in drugs approved for oncology/hematology indications. This has likely resulted in a greater number of approvals and possibly drugs coming to market faster ...
Josh Elbaz, Alyson Haslam, Vinay Prasad
doaj   +1 more source

Real‐World Pediatric Blinatumomab Administration: Access to Outpatient Care Delivery and Impact of a Hospital‐Dispensed Model

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Blinatumomab has been shown to be highly effective for patients with pediatric B‐ALL and has recently become standard of care therapy. Due to its past use in the clinical trial setting, there is limited information available about real‐world administration.
Katelyn Oranges, Kelly A. Duffy, Sara McDonough, Evelyn Kurpiewski, R. Jennifer Randall, Allison Barz Leahy, Haley M. Newman, Joyce P. Lee, Stephen P. Hunger, Anne F. Reilly, David T. Teachey, Susan R. Rheingold, Daniel J. Zheng   +12 more
wiley   +1 more source

Ethical Issues in the Drug Approval Process [PDF]

, 2013
The Food and Drug Administration’s (FDA) drug approval process carries great significance for both pharmaceutical companies and for patients. In recent decades, there has been a shift towards expedited approval so that drugs, especially those for ...
McInnis, Meghan
core   +1 more source

An analysis of common ethical justifications for compassionate use programs for experimental drugs [PDF]

, 2016
BACKGROUND: When a new intervention or drug is developed, this has to pass through various phases of clinical testing before it achieves market approval, which can take many years.
Raus, Kasper
core   +1 more source

Survival Outcomes and Complications Among Canadian Children With Retinoblastoma: A Population‐Based Report From CYP‐C

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Purpose Retinoblastoma (RB) is the most common pediatric ocular cancer, yet population‐based data on survival and risk factors remain limited. This study aimed to describe survival in a large national RB cohort and identify predictors of death and complications.
Samuel Sassine, Ofélie Trudeau‐Ferrin, Caroline Laverdière, Patrick Hamel, Rosanne Superstein, Bénédicte Koukoui, Carole Provost, Paul C. Nathan, Vijay Ramaswamy, Furqan Shaikh, Brenda Gallie, Ashwin Mallipatna, Stephanie Kletke, Helen Dimaras, Hallie Coltin   +14 more
wiley   +1 more source

Personalized Selumetinib Dosing in Pediatric Neurofibromatosis Type 1: Insights From a Pilot Therapeutic Drug Monitoring Study

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Objective To evaluate selumetinib exposure using therapeutic drug monitoring (TDM) in pediatric patients with neurofibromatosis type 1 (NF1) and plexiform neurofibromas (PN), assess interpatient pharmacokinetic variability, and explore the relationship between drug exposure, clinical response, and adverse effects.
Janka Kovács, Zoltán Köllő, Balázs Gellért Karvaly, Mahmoud Obeidat, Rita Nagy, Péter Hegyi, Brigitta Teutsch, Eszter Tuboly, Miklós Garami   +8 more
wiley   +1 more source

Sununu\u27s Job Approval Falls From All-Time High; Bipartisan Optimism About NH Overall 4/23/2019 [PDF]

, 2019
Governor Chris Sununu\u27s job approval has declined from an all-time high in February, but his approval mirrors that of his predecessor Maggie Hassan at the same point in her governorship. Only four in ten Democrats approve of Sununu\u27s performance as
UNH Survey Center
core   +1 more source

Adherence to Protocol Recommendations for Children With Wilms Tumour in Two Consecutive Studies in the United Kingdom and Ireland—Does Variation Matter?

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Background and Aims Wilms tumour (WT) has excellent event‐free and overall survival (OS). However, small differences exist between countries participating in the same international study. This led us to examine variation in adherence to protocol recommendations as a potential contributing factor.
Suzanne Tugnait, R. Al‐Saadi, A. Lopez‐Cortes, K. Dzhuma, M. Oostveen, J. Brok, S. Irtan, M. Weeks, J. Bate, S. Baker, R. Williams, M. Powis, D. Saunders, O. Olsen, T. Watson, S. Shelmerdine, S. Vaidya, L. Howell, C. Duncan, G. M. Vujanic, C. Stiller, G. P. Balchin, T. Chowdhury, K. Pritchard‐Jones   +23 more
wiley   +1 more source

Additional safety risk to exceptionally approved drugs in Europe? [PDF]

, 2011
AIMS Regulatory requirements for new drugs have increased. Special approval procedures with priority assessment are possible for drugs with clear 'unmet medical need'.
Arnardottir, Arna H., de Graeff, Pieter A., Eichler, Hans-Georg, Haaijer-Ruskamp, Flora M., Mol, Peter G. M., Straus, Sabine M. J.   +5 more
core   +2 more sources

Sirolimus for Extracranial Arteriovenous Malformations: A Scoping Review of the Evidence in Syndromic and Non‐Syndromic Cases

Pediatric Blood &Cancer, EarlyView.
ABSTRACT Arteriovenous malformations (AVMs) are rare, high‐flow, vascular anomalies that can occur either sporadically or as part of a genetic syndrome. AVMs can progress with serious morbidity and even mortality if left unchecked. Sirolimus is an mTOR inhibitor that is effective in low‐flow vascular malformations; however, its role in AVMs is unclear.
Will Swansson, Ingrid Winship, Gary Hammerschlag, Laura Scardamaglia   +3 more
wiley   +1 more source