New trial design guidelines address ‘inappropriate’ patient exclusion

By Melissa Fassbender contact

- Last updated on GMT

(Image: Getty/Zinkevych)
(Image: Getty/Zinkevych)

Related tags: Cancer

Exclusion of brain metastasis patients from clinical trials of anti-cancer drugs could remove up to two-thirds of the stage IV population, say researchers who have published new guidelines aimed at bringing attention to opportunities for improvement.

The new guidelines​ were developed by the Response Assessment in Neuro-Oncology – Brain Metastases (RANO-BM) group and describe how to address cancer patients with CNS involvement in clinical trials of anti-cancer drugs.

Spread of solid tumors to the brain is becoming an increasing problem, particularly in more common cancers, such as lung cancers, melanoma, and breast cancer, explained Ross Camidge, MD, PhD, Joyce Zeff Chair in Lung Cancer Research at the University of Colorado Cancer Center, director of Thoracic Oncology at the CU School of Medicine, and lead author of the trial design guidelines.

As we have got better at controlling advanced cancers, the need to address the central nervous system directly in clinical trials lead to these guidelines​,” Camidge told

Partly this is about who to include and exclude and when that’s appropriate – but it’s also about drawing attention to how we have done things in the past and how we need to do better – shifting the field through a shared desire to improve,"​ he explained.

Camidge said the guidelines were developed in response to the “inappropriate exclusion​” of patients whose cancer has spread to their brain, but which wouldn’t affect the study’s outcome.

According to the guidelines: "Exclusion of [brain metastasis] patients could remove half to two-thirds of the stage IV population."

The goal is to provide explicit wording to suggest who to exclude, so as to not narrow the trial eligible population, said explained Camidge.

The guidelines also provide direction on how specific dosing schedules could help optimize efficacy in the CNS – “so we know what to do if a standard dose or schedule works in the body, but then the brain becomes a problem on treatment​,” Camidge added.

As drugs start to show activity in the brain, we have to design trials to capture activity in the brain robustly and separately from activity in the rest of the body​,” he explained.

The US Food and Drug Administration was involved in the development of the guidelines, which were recently published the guidelines in the journal Lancet Oncology.

Camidge said the guidelines were written to create pragmatic, “how-to-do-it” suggestions, as doctors, scientists, and consumers of trial data increasingly demand data to make informed decisions in the future.

We hope the adoption of the suggestions in this paper will happen organically – driven by that shared desire to improve​,” he said.


Title​: Clinical trial design for systemic agents in patients with brain metastases from solid tumours: a guideline by the Response Assessment in Neuro-Oncology Brain Metastases working group
Author​: Camidge, D Ross et al.
Publication​: The Lancet Oncology, Volume 19, Issue 1, e20 - e32
Date​: January 2018

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