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Moving The Field Toward More Patient-Focused Drug Development

Together with our partners in the Jumpstarting Brain Tumor Drug Development Coalition – Accelerate Brain Cancer Cure (ABC2), Musella Foundation for Brain Tumor Research and Education, and the Society for Neuro-Oncology – NBTS has been advocating for the brain tumor research field to take patients’ quality of life into great account during clinical trials.

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Leaders of the Jumpstarting Brain Tumor Drug Development Coalition (L-R): David Arons (NBTS), Al Musella (Musella Foundation), Charles Haynes (Society for Neuro-Oncology), and Max Wallace (ABC2)

As part of that effort, we surveyed patients and caregivers to see what mattered most to them for future treatments. We then took the survey’s findings – the voice of the patients – to a workshop that was planned and carried out with participation from the US Food and Drug Administration (FDA) and included stakeholders from all aspects of the brain tumor community, including clinicians, researchers, biopharmaceutical companies, clinical research organizations, patients, patient advocates, and the National Cancer Institute.

Now, a supplemental issue to the journal Neuro-Oncology – a top scientific and medical journal for brain tumor information – was published to provide details on the outcomes of the workshop so more professionals in the brain tumor field can begin to consider these critical issues. The supplement includes four papers from panels of leaders that took part in the workshop.

The Patient Voice in Drug Development

The process of turning a scientific idea into a new medicine that patients can benefit from is known in biomedical research as “drug discovery and development.” The ‘discovery’ aspect of that term refers to the work done to identify a potential chemical compound or molecular agent that could have a therapeutic effect on targeted mutations in the tumor cells. The ‘development’ aspect refers to taking the crude discovery of chemical compound/molecular agent and developing it into an actual medicine that could be given to patients, travel to its target, and deliver its “payload,” so to speak.

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Allison Bishof (second from left) on a panel at the Brain Tumor Clinical Trial Endpoints Workshop

This development process includes moving the potential new medicine from laboratory into the clinical trial process.

In the traditional clinical trial process, most potential new medicines are evaluated to see if they can improve patient survival and extend life. But, while it will always be the brain tumor field’s main goal to develop life-extending and life-saving treatments, it still is, nonetheless, important to take into account patients’ symptoms, signs and functions during the drug development process.

Measurements of how a brain tumor patient feels and functions – known as “clinical outcome assessments”– have been insufficiently used in brain cancer clinical trials seeking drug approval.

“I want future brain tumor clinical trials to measure both a treatment’s effect on a patients’ tumor, as well as any effects it may have on day-to-day life,” said Allison Bishof, a brain tumor survivor, in a blog post for NBTS. “As one other patient advocate said, this is ‘exactly what has been missing for the last 30 years of work in this field.’ And I agree! I don’t know about you, but I would love a drug that would extend my life AND improve my concentration/fatigue/brain and physical function.”

The publication of the new supplement will further spread the word that it is no longer enough, to measure the length of a patients survival in a clinical trial; all of the clinical outcomes of a potential new treatment need to be taken into account in future drug development. As such, the Jumpstarting Brain Tumor Drug Development Coalition will continue to advocate for the inclusion of COAs into brain tumor clinical trials.

“We know these patients not only want to live longer, but to live comfortably and function as well as they can, for as long as possible,” said Dr. Terri Armstrong of The University of Texas Health Science Center and MD Anderson Cancer Center, and a co-chair of the workshop. “Unfortunately, too often trials do not adequately include rigorous clinical outcome assessments. Yet, COAs are essential to understanding all of the potential patient benefits of a therapy under evaluation, including reducing symptoms, signs, and improving function.”

And an added benefit, in addition to being important to patients, is that during the workshop it was discussed that clinical outcomes assessments actually can help support the primary goal (or “endpoint”) of survival, and thus may actually be a critical rather than optional piece to the process of trial design. Further, these measures alone could also potentially be an additional basis for approval.

As NBTS CEO, David Arons, said at the time of the workshop, “…We need better treatments as soon as possible,” said Arons. “It is our charge to identify ways to improve clinical research and speed the drug development and approval process in a manner that meets patients’ needs and respects what brain tumor patients and their families are going through…”

That sentiment was echoed by Max Wallace, CEO of ABC2, who concluded, “…Although more work needs to be done, we are pleased that this important effort is moving forward and getting the attention not only from the entire field, but also the FDA. This really has the potential to improve patient care going forward.”

Here is a link to the full journal supplement, which includes four papers. Unfortunately, due to copyright laws you’ll need to pay to access the supplement, but a free recap can also be found here, with a summary and more info at the bottom of our Brain Tumor Clinical Trial Endpoints Workshop page. The abstracts are also free to read for each of the four papers.


The Brain Tumor Clinical Trial Endpoints Workshops are part of NBTS’ umbrella initiative, the Clinical Trial Endpoints Initiative.

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