Search Videos by Topic or Participant
Browse by Series:

Surrogate Endpoints for New Drug Discovery in ALL

Panelists: John C. Byrd, MD, Ohio State University; Dan Douer, MD, MSKCC;Stephen P. Hunger, MD, University of Colorado; Brad S. Kahl, MD, University
Published: Tuesday, Feb 04, 2014
For High-Definition, Click
The FDA has been amenable to surrogate endpoints for the approval of novel agents in acute lymphoblastic leukemia (ALL), but hard-set guidance remains vague. In 2012 the FDA granted accelerated approval for vincristine sulfate liposome injection (Marqibo) for the treatment of adult patients with Philadelphia chromosome-negative ALL. This approval was based on the rate of complete remission plus the rate of complete remission with incomplete blood count recovery in a single-arm, single-agent trial of 65 adults in second or greater relapse.

The use of surrogate endpoints, such as getting to transplant in a minimal residual disease setting, remain unclear for accelerated approval of treatments for patients with ALL. At this point, the FDA has not provided clear guidance. As a result, a definitive approval still requires the demonstration of prolonged survival.
Slider Left
Slider Right
For High-Definition, Click
The FDA has been amenable to surrogate endpoints for the approval of novel agents in acute lymphoblastic leukemia (ALL), but hard-set guidance remains vague. In 2012 the FDA granted accelerated approval for vincristine sulfate liposome injection (Marqibo) for the treatment of adult patients with Philadelphia chromosome-negative ALL. This approval was based on the rate of complete remission plus the rate of complete remission with incomplete blood count recovery in a single-arm, single-agent trial of 65 adults in second or greater relapse.

The use of surrogate endpoints, such as getting to transplant in a minimal residual disease setting, remain unclear for accelerated approval of treatments for patients with ALL. At this point, the FDA has not provided clear guidance. As a result, a definitive approval still requires the demonstration of prolonged survival.
View Conference Coverage
Online CME Activities
TitleExpiration DateCME Credits
Clinical Practice Connections™: From Diagnosis to Emerging Immunotherapeutic Options: Understanding the Burden and Risks in Peanut AllergySep 28, 20191.0
Publication Bottom Border
Border Publication
x