A Caregiver's Perspective on Rare Disease Clinical Trials

A Caregiver's Perspective on Rare Disease Clinical Trials

Posted by Juli Greenwood

3003_zaggocare_logo.jpgSupporting rare disease research is a big part of our work here at BBK and rare disease recruitment is an expertise that our sponsors and CRO customers have come to expect from us.

While progress has been made to improve the lives of patients with rare disorders, there is much that still needs to be done to reach patients, further research, find new treatments and improve outcomes. Our most recent eBook, “Rare Disease Research. What’s Next?” is a collection of insights from a variety of rare disease stakeholders, including Roberta Carson, Founder and President of Zaggo, Inc. – a national nonprofit devoted to helping patients and families be more engaged members of their medical teams.

She founded Zaggo after losing her teenage son to a terminal brain cancer, hoping that by sharing her personal experiences and lessons learned she might help others. Managing an illness or injury is overwhelming, and thrusts patients and their caregivers into emotional, unchartered territory. But we know that improved engagement is linked to better outcomes – so isn’t it worth helping patients and caregivers be more engaged?

In this excerpt, Roberta describes her study experience:

At the age of 17 my son Zach was diagnosed with DIPG, a rare and aggressive brain cancer. His diagnosis was so grim we had to choose his treatment at our first oncologist appointment. Since there are no known treatments, a clinical trial was our only option. Our doctor gave us three study options; none of which we could fully understand. Coping with Zach’s diagnosis was impossible enough, now we would have to make an important decision that would impact Zach tremendously – how long he might live and what side effects he might face. We did what many others in our shoes do. We asked our doctor what he would do and we followed his suggestion.

We were lucky to have a very supportive doctor and care team. They always made time for us, never rushed us through an appointment and answered every question thoughtfully and completely.

The first study held the tumor at bay for 15 months, but the treatment stopped working and he was taken off the trial. A month later he was placed in a new trial. This was a devastating time for us.

Soon thereafter, Zach’s high-dosage steroid to combat brain swelling caused tissue to build in his spinal column, leaning on his spinal cord. As Zach became immobile, we were desperate for alternatives and felt lucky to be enrolled in a separate trial for a medication to reduce brain swelling without steroids. We knew Zach had a 50:50 chance of getting a placebo, but we felt it was worth the risk. Unfortunately, Zach’s paralysis worsened due to the extra steroid he was given along with his placebo. The paralysis led to a pulmonary embolism, in the end causing his death from respiratory failure. As his parents and his caregivers we had to make the best decisions under the worst of circumstances, knowing that in the end we might only extend his life by a little or improve what time he had left. Devastated to lose him of course, we did take some comfort knowing that we were contributing to research that might someday lead to a treatment or a cure.

This is just one portion of Roberta’s contribution. To read more about Roberta’s experiences and to learn from our other contributors including experts from the National Organization for Disorders (NORD), Landon Pediatric Foundation and Atlantic Research Group, you can download the full eBook here.


Topics: Patient Engagement, Patient Advocacy, Patient Experience