Clinical Trial Endpoints Workshops

WORKSHOP 1 – IMAGING ENDPOINTS

Objective:
To summarize the discussion sessions from the first Brain Tumor Clinical Trials Endpoints Workshop on imaging-related endpoints held on January 30, 2014 in Bethesda, MD. The workshop was sponsored by the Jumpstarting Brain Tumor Drug Development Coalition with participation by the U.S. Food and Drug Administration (FDA).

Workshop Overview:
The Jumpstarting Brain Tumor Drug Development Coalition (which includes the National Brain Tumor Society, Accelerate Brain Cancer Cure, Musella Foundation for Brain Tumor Research and Information, and the Society for Neuro-Oncology) is sponsoring workshops to evaluate and improve the use of a variety of endpoints in brain tumor clinical trials, with a goal of advancing the development of treatments for glioblastoma multiforme (GBM). These workshops bring together key stakeholders (clinicians, researchers, industry, patient advocates, the National Cancer Institute, and the FDA) to discuss issues related to the development and use of endpoints, and to develop action plans that will ultimately lead to greater clarity and interest in the pursuit of clinical trials that can achieve FDA approval of new brain tumor therapies. The discussions and action items that come of out these workshops are meant to inform and guide the neuro-oncology and clinical trial sponsor community.

The first of these workshops was held in Bethesda, Maryland, on January 30, 2014. This workshop’s discussion was focused on the capability of neuro-imaging to accurately assess GBM response to therapies, and the use of current and emerging imaging-based endpoints in clinical trials. Specifically, stakeholders discussed how to overcome the variables in medical imaging, such as image acquisition parameters, which have hindered the ability to accurately assess brain tumor response to therapies, and how to best incorporate endpoints that rely on imaging into clinical trials.

The workshop began with three formal preliminary presentations to establish context for the day’s discussions: “Efficacy Endpoints in Glioblastoma Multiforme Clinical Trials – A Regulatory Perspective,” “Avastin & the Basis of Approval for GBM in 2009,” and “Current Brain Tumor Imaging Protocols in Multicenter Trials.” After the preliminary presentations, the workshop continued with four panel-led discussions. The panels consisted of experts in neuro-oncology and neuro-radiology including FDA officials, industry representatives, and medical/academic researchers. After a short overview presentation by each panel, a two-part discussion period followed: (1) response by panelists to central questions posed by the moderator, and (2) facilitated audience question and answer.

Click here to read a full PDF summary of the panel discussions and central questions.

Click here for a full list of Workshop participants. Note: the above summary does not necessarily denote the opinions of all individual participants.

Conclusion:
The workshop produced a number of suggested action items intended to represent a starting point for future work in the area of imaging brain tumor response to treatment in clinical trials. The Jumpstarting Brain Tumor Drug Development Coalition will continue to work with our advisors and the FDA to identify outstanding issues needed to move the community closer to more, and better, treatments. There was consensus that the RANO criteria should continue to serve as the standard response criteria in brain tumor clinical trials. However, the imaging elements of RANO would benefit from additional clarification, including a clearer definition of response tailored to the type of therapy under investigation. It was suggested that further research be conducted to understand the RANO criteria's evaluation of imaging based response rate, and the Coalition will aid this effort. Additionally, the Coalition will lead in the formation of a working group that will address and develop guidance materials for the standardization of image acquisition and analysis within brain tumor clinical trials. The goal is for this guidance to be adopted by all sponsors of brain tumor clinical trials, as well as to demonstrate that the brain tumor research and clinical trial environment is innovative, adaptive, and ready for further investment by academia, pharmaceutical companies, and the government.

WORKSHOP 2 – SYMPTOM-RELATED ENDPOINTS

The Second Brain Tumor Clinical Trial Workshop on symptom-related endpoints will be held on October 15, 2014. Updates will follow the Workshop.

NOTE: This workshop was originally scheduled for May 20th, 2014, but re-scheduled to the later date due to scheduling conflicts.