Patient-Reported Outcomes in Pediatric Cancer Registration Trials: A US Food and Drug Administration Perspective

cancer, pediatrics, product labeling, united states food and drug administration, childhood cancer, denosumab, surrogate endpoints, adverse event, patient self-report, pediatric oncology, selumetinib, benefit-risk assessment, tisagenlecleucel, statistica

PROs have been feasible to collect from patients as young as 7 years old and were included in trials from 2013 onward. PROs were used as exploratory endpoints in four product applications but not included in product labeling. To include PRO data in FDA labeling, early interaction with the FDA, c…

Apr 30^th • 12 mins read

Biased by design? Clinical trials and patient benefit in oncology

clinical trial design, drug development, drug registration, patient benefit, risks of bias

The study by Naci et al. raises numerous questions relating to the design characteristics, risks of bias and reporting of pivotal clinical trials in oncology. These findings add to the existing body of evidence that suggests drug development in oncology is not delivering the clinical benef…

Nov 27^th • 3 mins read

Assessment of Food and Drug Administration- and European Medicines Agency-Approved Systemic Oncology Therapies and Clinically Meaningful Improvements in Quality of Life: A Systematic Review

ESMO, MCBS, FDA, ASCO-VF, EMA, QOL

Recent oncology therapies approved by the FDA and EMA often lack evidence of clinically meaningful improvements in quality of life (QOL). Only 40% of FDA-approved and 58% of EMA-approved oncology therapies had published QOL evidence. Clinically meaningful QOL improvements beyond minimal dif…

Feb 11^th • 4 mins read

Real-World Evidence in Support of Oncology Product Registration: A Systematic Review of New Drug Application and Biologics License Application

RWE, Biologics License, FDA, BLA, suplements

The FDA emphasizes early engagement and transparent protocols in Real-World Evidence (RWE) studies. Aligning RWE populations with pivotal trial populations is crucial, often achieved through criteria matching and propensity score methodologies. Major critiques include differences in tumor asse…

Jan 1^st • 12 mins read

How do cancer clinicians perceive real-world data and the evidence derived therefrom? Findings from an international survey of the European Organisation for Research and Treatment of Cancer

real-world evidence, real-world data, oncology, cancer, survey, clinicians, randomized controlled trials, Europe

Real-world evidence (RWE) is increasingly being used in the development and decision-making processes for anticancer therapies, but clinician views on its use are unclear. A survey conducted between May and July 2021 involved 557 clinicians from 30 countries and 13 cancer domains. Most clinician…

Aug 1^st • 45 mins read