Results 11 to 20 of about 49,931 (273)

Progression-free survival as a surrogate endpoint in myeloma clinical trials: an evolving paradigm [PDF]

Blood Cancer Journal
Measurement of overall survival (OS) remains the gold standard for interpreting the impact of new therapies for multiple myeloma in phase 3 trials. However, as outcomes have improved, it is increasingly challenging to use OS as the primary endpoint if ...
Charlotte Pawlyn, Fredrik H. Schjesvold, David A. Cairns, L. J. Wei, Faith Davies, Omar Nadeem, Haifaa Abdulhaq, Maria-Victoria Mateos, Jacob Laubach, Katja Weisel, Heinz Ludwig, S. Vincent Rajkumar, Pieter Sonneveld, Graham Jackson, Gareth Morgan, Paul G. Richardson   +15 more
doaj   +2 more sources

Imputation of a true endpoint from a surrogate: application to a cluster randomized controlled trial with partial information on the true endpoint

BMC Medical Research Methodology, 2003
Background The Anglia Menorrhagia Education Study (AMES) is a randomized controlled trial testing the effectiveness of an education package applied to general practices.
Duffy Stephen W, Nixon Richard M, Fender Guy RK   +2 more
doaj   +3 more sources

A surrogate endpoint-based provisional approval causal roadmap, illustrated by vaccine development. [PDF]

Biostatistics
For many rare diseases with no approved preventive interventions, promising interventions exist. However, it has proven difficult to conduct a pivotal phase 3 trial that could provide direct evidence demonstrating a beneficial effect of the intervention ...
Gilbert PB, Peng J, Han L, Lange T, Lu Y, Nie L, Shih MC, Waddy SP, Wiley K, Yann M, Zafari Z, Ghosh D, Follmann D, Juraska M, Díaz I.   +14 more
europepmc   +2 more sources

The perils of surrogate endpoints [PDF]

European Heart Journal, 2015
Our goals in medicine are (i) to improve the quality of patients' lives, (ii) help them to live longer, and (iii) to do so at a reasonable cost. These are our true endpoints: health status, survival, and cost. It is thus entirely consistent with this point of view that these are the fundamental concepts that can be united in a formal cost–utility ...
Weintraub, William S, Lüscher, Thomas F, Pocock, Stuart   +2 more
openaire   +5 more sources

Establishing a Public Resource for Acceptable Surrogate Endpoints to Support FDA Marketing Applications

Frontiers in Medicine, 2022
Following a comprehensive and coordinated effort between CBER and CDER, FDA established a table of acceptable surrogate endpoints (SEs) to support drug marketing applications.
Abena S. Agyeman, Jeffrey N. Siegel, Christopher Leptak   +2 more
doaj   +1 more source

A meta-analysis of GFR slope as a surrogate endpoint for kidney failure. [PDF]

Nat Med, 2023
Glomerular filtration rate (GFR) decline is causally associated with kidney failure and is a candidate surrogate endpoint for clinical trials of chronic kidney disease (CKD) progression. Analyses across a diverse spectrum of interventions and populations
Inker LA, Collier W, Greene T, Miao S, Chaudhari J, Appel GB, Badve SV, Caravaca-Fontán F, Del Vecchio L, Floege J, Goicoechea M, Haaland B, Herrington WG, Imai E, Jafar TH, Lewis JB, Li PKT, Maes BD, Neuen BL, Perrone RD, Remuzzi G, Schena FP, Wanner C, Wetzels JFM, Woodward M, Heerspink HJL, CKD-EPI Clinical Trials Consortium.   +26 more
europepmc   +2 more sources

Food and Drug Administration approvals in phase 3 Cancer clinical trials

BMC Cancer, 2021
Background Phase 3 oncologic randomized clinical trials (RCTs) can lead to Food and Drug Administration (FDA) approvals. In this study, we aim to identify trial-related factors associated with trials leading to subsequent FDA drug approvals.
Joseph Abi Jaoude, Ramez Kouzy, Marc Ghabach, Roshal Patel, Dario Pasalic, Elie Ghossain, Austin B. Miller, Timothy A. Lin, Vivek Verma, C. David Fuller, Vivek Subbiah, Bruce D. Minsky, Ethan B. Ludmir, Cullen M. Taniguchi   +13 more
doaj   +1 more source

The Relationship Between Short-Term Surrogate Endpoint Indicators and mPFS and mOS in Clinical Trials of Malignant Tumors: A Case Study of Approved Molecular Targeted Drugs for Non-Small-Cell Lung Cancer in China

Frontiers in Pharmacology, 2022
Objective: Due to the initiation of the priority review program in China, many antitumor drugs have been approved for marketing based on phase II clinical trials and short-term surrogate endpoint indicators.
Mingjun Rui, Mingjun Rui, Zijing Wang, Zijing Wang, Zhengyang Fei, Zhengyang Fei, Yao Wu, Yao Wu, Yingcheng Wang, Yingcheng Wang, Lei Sun, Ye Shang, Ye Shang, Hongchao Li, Hongchao Li   +14 more
doaj   +1 more source

Surrogate endpoint evaluation using data from one large global randomized controlled trial

BMC Medical Informatics and Decision Making, 2021
Background Robust identification of surrogate endpoints can help accelerate the development of pharmacotherapies for diseases traditionally evaluated using true endpoints associated with prolonged follow-up.
Milan Geybels, Benjamin Ole Wolthers, Frederik Flindt Kreiner, Søren Rasmussen, Robert Bauer   +4 more
doaj   +1 more source

Intermediate endpoints as surrogates for outcomes in cancer immunotherapy: a systematic review and meta-analysis of phase 3 trialsResearch in context

EClinicalMedicine, 2023
Summary: Background: Cancer immunotherapy shows unique efficacy kinetics that differs from conventional treatment. These characteristics may lead to the prolongation of trial duration, hence reliable surrogate endpoints are urgently needed.
Zhishan Zhang, Qunxiong Pan, Mingdong Lu, Bin Zhao   +3 more
doaj   +1 more source