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Can this treatment help me live a longer and better life?

Why was I asked to sit on a panel with two top officials from the U.S. Food and Drug Administration (FDA), two leading neuro-oncologists, an associate professor of neuropsychiatry, and an executive from the biopharmaceutical company, Genentech?

Because, as a five-year brain tumor survivor and volunteer advocate with the National Brain Tumor Society, 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. As one of the FDA officials on my panel so pointedly put it, the “full story” of the drug.

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Allison Bishof

These measures, or goals, for a potential new treatment being tested are known in the field as “endpoints,” and to-date, patient-focused endpoints (known in the field as clinical outcomes assessments) have not been sufficiently included in brain tumor clinical trials. 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 Jumpstarting Brain Tumor Drug Development Coalition is trying to make this happen. Earlier this year, the National Brain Tumor Society started a dialogue with the FDA and asked what the brain tumor field – the researchers and doctors, as well as the drug companies working on brain tumor treatments – could do to design clinical trials that will “jumpstart” brain tumor drug development and approvals. One of the Agency’s answers was to add patient-focused measures into clinical trials for future treatments, as these might provide an additional basis for approval, and are, of course, important to us patients.

Really? the FDA and drug companies want to know what WE think? YES, they do.

So a great, collaborative group of nonprofits and philanthropic organizations that represent the brain tumor community was formed to work together and deliver on this – the already referenced Jumpstarting Brain Tumor Drug Development Coalition, which includes NBTS, Accelerate Brain Cancer Cure (ABC2), the Musella Foundation for Brain Tumor Research and Information, and the Society for Neuro-Oncology. They sent out a survey to patients and caregivers, and, incredibly, more than 1,800 people responded, helping inform the field about what patients and caregivers want!

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Allison (second from left on panel) participates in the Brain Tumor Clinical Trial Endpoints Workshop on Clinical Outcomes Assessments

Fast-forward to mid-October and the recent Brain Tumor Clinical Trial Endpoints Workshop. This workshop, hosted by the Coalition, brought in experts from the FDA; the National Institutes of Health/National Cancer Institute; nonprofit advocacy groups; individual patients and patient advocates; survivors; caregivers; and leaders from the fields of neuro-oncology, neurology, and clinical outcomes assessment.

I happily accepted the invitation from the Coalition to be on one of the panels and represent the patient experience. I was amazed and delighted by the collaborative effort from all the participants (as another panelists said, “One of the more collaborative and fulfilling journeys I’ve been on.”), and the great sense of urgency the community seemed to share about adding clinical outcomes assessments into future brain tumor trials.

These groups used the survey, past research and experiences, and success stories from other disease areas to identify a plan to incorporate clinical outcomes assessments into future clinical trials as acceptable endpoints for approval.

And as testament to how far the field has come in recent years in terms of inclusion, cooperation, and patient-centric focus, I was reminded by an esteemed panelist that, “I don’t think we would have seen Allison here at the table just 10 years ago…I think this is a positive thing.” So do I!

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It’s no simple task to scientifically measure improvement in symptoms, and do it in a standard way across the many medical centers involved in brain tumor clinical trials. But, believe me, these groups are determined to figure it out, and it won’t be long until they do. As one of their pre-Workshop papers said, “Although challenging, the incorporation of [clinical outcomes assessments] into clinical trials is an important goal that will enhance future studies by providing insights not generated by traditional endpoints and efficacy measures and ultimately improve patient care.”

Soon we will have clinical trials designed to find treatments to help us live longer and live better!

Allison Bishof is a volunteer Advocate with the National Brain Tumor Society. She is also active with the Race for Hope – Philadelphia.

Read an official press release of this Workshop here.

  • Andrea Marit Erickson

    This is so absolutely wonderful! I’m a 30 year survivor of brain cancer. I had an Astrocytoma Grade 1 located on the floor of the 4th ventricle near the cerebellum. I can’t begin to summarize all the difficulties that I’ve had since then, but I could use information that could help me live “a better and longer life”. Three cheers for Allison! You go girl!

    • Donna

      My 9yr old son was just diagnosed. I’d love to hear about your journey.

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