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Earlier this year, we shared a number of blogs about our Clinical Trial Endpoints Initiative.

National Brain Tumor Society is working with the FDA, the National Cancer Institute, medical academic researchers, clinicians, and the Jumpstarting Brain Tumor Drug Development Coalition (a group of other brain tumor-focused nonprofits and organizations), with the goal of developing action plans to improve the system for measuring and evaluating potential new treatments. We hope this will lead to an accelerated pace and volume of new approvals for brain tumour therapies.

The Coalition is convening the second of its two separate Brain Tumor Clinical Trial Endpoints Workshops to develop the action plans. The first workshop, hosted in January of 2014, focused on developing better standards to improve imaging in brain tumor clinical trails (which in turn improves how biopharmaceutical companies, and subsequently, regulators at the FDA, evaluate a treatment under evaluation). This could potentially lead to the ability of the field to use single-arm trials aimed at achieving accelerated approval for potential new brain tumor therapies.

The second Brain Tumor Clinical Trial Endpoints Workshop, scheduled for October, will focus on how measures related to a patient’s symptoms can be incorporated into double-arm brain tumor clinical trials; as it is well established that, even more so than many other cancers, brain tumors are associated with a significant impact on quality of life.

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Dr. Terri Armstrong (center) with the other leading advisors to the Brain Tumor Clinical Trial Endpoints Workshops, Drs. W.K. Alfred Yung of MD Anderson (left) and Patrick Wen of Dana-Farber Cancer Institute (right)

Helping guide this initiative, as Vice Chair of the National Brain Tumor Society’s Clinical Trial Endpoints Steering Committee is Dr. Terri S. Armstrong, PhD, ANP-BC, FAANP, FAAN, who is a Professor in the Department of Family Health at the UT Health School of Nursing, holder of the Dunn Distinguished Professorship in Oncology Nursing, and an Advanced Practice Nurse in the Department of Neuro-Oncology at MD Anderson Cancer Center.

Dr. Armstrong kindly took the time to explain more about the state of symptom-related endpoints (measures) in brain tumor clinical trials:

Q: From your perspective seeing brain tumor patients at MD Anderson Cancer Center, what frustrations do you see brain tumor patients dealing with most? I.E. what are the common complaints or limitations to patients’ lives that are mentioned?

Dr. Terri Armstrong: It is not uncommon for patients with brain tumors to be healthy, and then have a sudden symptom that leads to their diagnosis over a short period of time. The sudden occurrence and diagnosis often leads to people feeling confused and uncertain. Unfortunately, this uncertainty continues throughout the disease. Common symptoms include difficulty with memory or being able to speak, weakness on one side of the body limiting the ability to walk or care for themselves, and seizures.

Q: As you look at the landscape of brain tumor clinical trials, to what extent are clinical trials adequately including ways to measure if drugs will alleviate symptoms in addition to prolonging life?

TA: It has not been common for clinical trials to include measures of symptom impact in addition to improving survival. However, with the RTOG 0525 study, I was part of a research group that was able to secure a grant to allow us to look at objective neurocognitive functioning, QOL, and symptom reports. Since that time, several other large trials have included these measures as well as other measures (such as steroid reduction and quality of life). These results have shown that it is feasible to do this, that it provides important information on the impact of the tumor and treatment on the person, and may help to fully understand the net impact of the disease and treatment.

Q: What are some of the difficulties faced by drug companies and academic center-based groups in evaluating if treatments are effective at minimizing symptoms? And why, given its importance to patients, do you think the extent of measuring symptom impact of drugs in clinical trials is inadequate?

TA: There are multiple factors. First, it was initially seen as a great burden on the patient. But these recent studies have shown that the time it takes is minimal and that patients are willing to participate. For example, in RTOG 0525 [patient symptom reporting] was not mandatory, but over 90% of patients agreed to do it…Finally, how best to incorporate, and generating acceptance of these outcomes, without making the bar higher for pharmaceutical companies to gain approval, is an ongoing issue (and one we are working on in the upcoming workshop).

Q: You are leading the planning of an upcoming workshop with the FDA that will address the challenges of expanding clinical trials to more robustly measure a potential new drug’s impact on symptoms.  What do you see as the opportunities or potential of this workshop?

TA: This is a fantastic opportunity to bring together clinicians (including those like myself who provide care and are interested in measuring the impact on symptoms), patient advocates, FDA, and the pharmaceutical industry to identify what are the critical symptoms; if tools exist that allow us to measure these symptoms; or begin to discuss development of tools, and develop innovative strategies to incorporate these endpoints into clinical trials. This may provide a standard to include measurement of the symptomatic benefit of therapy for patients, and inclusion of this, in clinical trials going forward. This would be a great benefit to patients.

Q: If we are successful, 3 – 5 years from now, how will brain tumor clinical trials be different with regards to symptom measurement if you had your way?  And how would that vision benefit the brain tumor patient community?

TA: In 3-5 years, a standard set of measures for symptoms would be included in all brain tumor clinical trials as a composite endpoint with tumor response. In addition, further work in not only symptom treatment, but symptom prevention, in the brain tumor population would be common, resulting in improvement in patient outcomes, like quality of life, disease response, and symptomatic improvement.

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While all of the groups who will be in attendance play leading roles in the process of developing and approving new brain tumor treatments, the most important voice in this effort is yours – the voice of patients, caregivers and families. These researchers, companies, and regulators need to know what patients want in terms of treatment options, and what is affecting a patient’s decision to become involved in research of these new treatments. Clearly, living longer and finding a cure is our number one priority. But, there may be treatments that reduce symptoms or improve quality of life, and those treatments need to be evaluated through the clinical trial process. We thank everyone who took our Endpoints Patient & Caregiver Survey to help inform these stakeholders in brain tumor drug development.

 

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