For a long time, I had one career plan: go to medical school. My mom has myasthenia gravis (MG), a rare disease that causes severe muscle weakness, making it difficult to walk, speak, even swallow food. I wanted to help her and people like her. I thought that meant becoming a doctor.
My mom’s symptoms put limits on her life. She had to leave her job as a teacher. When my siblings and I were young, she wasn’t able to care for us the way she wanted. Some days, she struggles to take full breaths. And my mom’s sub-type of MG, like 95 percent of rare diseases, has no treatment. As a college student, while assisting my mom’s neurologist with clinical research, I realized people with MG didn’t just need another doctor. They needed a drug therapy. That put me on a new career path. Twenty years later, clinical research is still my calling.
A rare disease has reaching effects. I learned this with my mom and then with my sister, whose in-utero sons were diagnosed with rare twin-to-twin transfusion syndrome. In both cases, our whole family — their care circle — felt the impact. So I know when we recruit a patient for a trial, we’re enrolling their care circle, too.
I try to see every protocol through my mom’s eyes. Would this work with her schedule? Have we made compliance as simple as possible? Then I think as a daughter. Would I encourage my mom to join this study? Would I feel good about what she stands to gain? If I answer no to any of those questions, our team gets to work finding better ways to execute.
Though my experiences with rare diseases have been difficult, I’m grateful for the perspective they give me. And I’m grateful that Parexel Biotech teams have the opportunity to build their own rare-disease awareness. Prior to launching a trial, our clinical professionals attend advocacy meetings. They hear from the scientific community, but they also meet patients and families, and care communities. They learn about hurt and hope and they see, like I did, what a therapy would mean to people living with disease. Those moments motivate and elevate our clinical efforts.
When patients participate in a trial, they’re offering us a gift. It might require a caregiver to travel, or get time off from work. That’s a gift. It might take every bit of a patient’s energy that day, every bit of courage. That’s a gift. Each vial of blood, each biopsy, each patient diary: all gifts.
We have the privilege of applying insights to the data patients provide us, and that is no small thing. So I will always advocate fiercely for thoughtful study design and scientific rigor. We can’t let trials become burdensome. Or let a single sample slip through the cracks. Our patients’ data is a gift. I’m proud we treat it that way.