Results 31 to 40 of about 159,613 (271)

Amivantamab and Mobocertinib in Exon 20 insertions EGFR Mutant Lung Cancer, Challenge To The Current Guidelines

Translational Oncology, 2022
In 2021, the US Food and Drug Administration (FDA) approved two drugs targeting exon 20 directly: amivantamab and mobocertinib, under the accelerated approval pathway, for adult patients with locally advanced or metastatic non-small cell lung cancer ...
Timothée Olivier, Vinay Prasad
doaj   +1 more source

Accelerated Approval of Highly Expensive Disease-modifying Agents: Lessons Learned From the Aducanumab Approval

Journal of Pharmacoeconomics and Pharmaceutical Management, 2022
Alzheimer’s disease (AD) is a neurodegenerative disease and the most common cause of dementia affecting millions of people yearly. On June 7, 2021, the U.S FDA granted accelerated approval to highly expensive Aducanumab (Aduhelm), the first-ever disease-
Santenna Chenchula, Padmavathi R.
doaj  

Implementation of accelerated research: strategies for implementation as applied in a phase 1 Ad26.ZEBOV, MVA-BN-Filo two-dose Ebola vaccine clinical trial in Uganda

Global Health Action, 2020
Background The 2013–2016 Ebola epidemic in West Africa is the worst ever caused by Ebolaviruses with over 28,000 human cases and 11,325 deaths. The World Health Organisation (WHO) declared the epidemic a public health crisis that required accelerated ...
Jonathan Kitonsa, Abu-Baker Ggayi, Zacchaeus Anywaine, Eva Kisaakye, Laura Nsangi, Vincent Basajja, Mary Nyantaro, Deborah Watson-Jones, Georgi Shukarev, Ine Ilsbroux, Cynthia Robinson, Pontiano Kaleebu   +11 more
doaj   +1 more source

Marketing Authorisation Based on Incomplete Clinical Data: International Experience and Prospects

Регуляторные исследования и экспертиза лекарственных средств, 2020
No new drug can be used in clinical practice without marketing authorisation. Acquisition of the necessary amount of clinical data may take several years, which is especially critical for pernicious diseases for which no alternative therapy is available.
D. V. Goryachev, N. E. Uvarova, G. V. Shukshina   +2 more
doaj   +1 more source

Association of expedited review programmes with postmarketing safety events of new drugs approved by the US food and drug administration between 2007 and 2017

BMJ Open, 2022
Objective To explore the associations between the risks of postmarketing safety events of new drugs and the four expedited programmes of priority review, accelerated approval, fast track and breakthrough therapy established by the US Food and Drug ...
Bao Liu, Xingyue Zhu
doaj   +1 more source

An insider's perspective on FDA approval of aducanumab

Alzheimer’s & Dementia: Translational Research & Clinical Interventions, 2023
Introduction Aducanumab was approved in 2021 by the US Food and Drug Administration (FDA) under the accelerated approval pathway. Since then, there have been many misconceptions about the approval decision despite multiple publications from the FDA to ...
Yaning Wang
doaj   +1 more source

Evaluating the evidence behind the surrogate measures included in the FDA's table of surrogate endpoints as supporting approval of cancer drugs

EClinicalMedicine, 2020
Background: In July 2018, the FDA first published a table listing all surrogate measures that it has used, and may accept for future use, in regulatory approval. However, the strength of surrogacy for those measures was not formally assessed.
Bishal Gyawali, Spencer P. Hey, Aaron S. Kesselheim   +2 more
doaj   +1 more source

A correlation analysis to assess event-free survival as a trial-level surrogate for overall survival in early breast cancer

EClinicalMedicine, 2021
Background: Event-free survival (EFS) has been listed on the FDA Table of Surrogate Endpoints as a surrogate measure that can be considered for accelerated or traditional approval in breast cancer.
Bishal Gyawali, Elvira D'Andrea, Jessica M. Franklin, Aaron S. Kesselheim   +3 more
doaj   +1 more source

Modernising the US FDA's Accelerated Approval pathway

The Lancet Oncology, 2023
On Dec 29, 2022, US President Joe Biden signed The Consolidated Appropriations Act into law. This wide-reaching Act contains notable reforms to the US Food and Drug Administration (FDA) statutory and regulatory frameworks. The legislation amends key regulatory frameworks, including changes to the FDA-expedited approval programme, Accelerated Approval ...
David J, Benjamin, Mark P, Lythgoe
openaire   +4 more sources

Organoids in pediatric cancer research

FEBS Letters, EarlyView.
Organoid technology has revolutionized cancer research, yet its application in pediatric oncology remains limited. Recent advances have enabled the development of pediatric tumor organoids, offering new insights into disease biology, treatment response, and interactions with the tumor microenvironment.
Carla Ríos Arceo, Jarno Drost
wiley   +1 more source