Why Families Like Brittany’s Can’t Afford for Rare Trials to Fail

Stories like Brittany’s aren’t rare in rare disease trials. Families absorb the emotional load. They absorb the logistical burden. They absorb the financial cost. And they try to keep showing up. But when clinical trials rely on generic decentralised approaches, those not designed for small populations, fragile journeys, and life-defining decisions, something breaks, and retention and progress suffer. Not because families don’t care. But because the system wasn’t built to support them. We owe them better.

Brittany Ford, Patient Caregiver - Rare Disease Research and Gooseberry Research Testimonial

Although decentralised, home-based strategies should help rare trials, generic DCT approaches tend to struggle to keep these studies on track. 

That’s why FutureMeds and Gooseberry Research formed a partnership to provide a solution that meets the needs of patients, caregivers, sites and Sponsors on both sides of the Atlantic. 

As part of our partnership, we are organising our very first webinar: “Rare Trials Under Pressure: Field-Tested Homecare Strategies to Boost Enrolment & Retention” It brings together a patient caregiver, US & EU Principal Investigators, a patient engagement lead and a site director to share their perspectives to improve study experience and reduce dropouts. 

Brittany Ford's Caregiver Story — Our Journey Into Rare Disease Research

(Full story as provided — unchanged, reflecting the thoughts and opinions of the Caregiver)

Our journey into the world of rare disease research began when my son, Briley, was a spunky, outgoing three-year-old little boy. At first, we simply noticed what seemed like autism-like traits, and I took him to his primary care doctor to be evaluated. Instead of not only did we get the autism diagnosis, we were told his liver enzymes were elevated. He was retested to rule out the basics, and when everything came back clear, we were sent to a GI specialist. After exhaustive testing, the GI doctor shared that the issue appeared to be muscular in nature, and we were referred to a neuromuscular specialist. Two weeks after they ran their tests, I received the phone call that changed our lives forever: Briley tested positive for Duchenne muscular dystrophy. It was the worst phone call of my life—one I will never forget. Even though the moment felt like a blur, I can still hear the doctor’s voice apologizing. I can still feel the scream I let out alone in my car afterward. That same doctor sent us to the MDA Clinic in Cincinnati, Ohio, and that is where our journey truly began. All of this unfolded in the peak of COVID, yet Cincinnati Children’s still pushed forward and referred us for the Mission 51 Rare Disease Research Trial. When we first began exploring the possibility of joining a clinical trial, I was terrified. The phrase “clinical trial” can be frightening for any parent—you immediately imagine your child being used as a test subject or facing unimaginable risks. I didn’t want my son to be experimented on, and the fear of losing him while hoping for a miracle felt overwhelming. But at the same time, we knew that if there was ever going to be a better future for Briley—and for other boys living with DMD—we had to consider the trial. It was a terrifying choice wrapped in the hope that maybe, just maybe, this could make a difference. And it did. One of the most powerful moments of the entire experience came just three months into the trial. We watched Briley go from being unable to stand on both feet and jump, to standing on one foot and jumping again—something he hadn’t been able to do. Seeing progress like that reminded us why these trials matter and why families like ours fight so hard to stay in them. Still, participating in a rare trial is emotionally and financially draining. I wish every sponsor, investigator, and study coordinator deeply understood how frightening this journey already is. We live every day with the fear of the unknown—wondering what tomorrow will bring, when symptoms will worsen, and how much time we truly have. Families need patience, compassion, and understanding. Many of us also face real financial challenges. For us, each visit required traveling nine hours round-trip, not including the hours spent in the clinic. It was stressful, exhausting, and expensive—but still a blessing, because these trials give our children a fighting chance. Through it all, the people behind the research made an enormous difference. When the rare disease research team was using a third-party agency for home health and we faced problems early on, RDR immediately stepped in and worked to fix it. That effort led to Gooseberry—and through that, we were connected with an amazing nurse who has become part of our family. She remains Briley’s home health nurse even now, for a fully FDA-approved treatment. Dr. Phan also made us feel genuinely cared for, often stopping by unannounced during visits just to check on us. The entire RDR care team made every appointment feel welcoming and personal. They even surprised Briley with a birthday celebration and another on his last infusion day. Their kindness will stay with us forever. They didn’t just care for my son—they cared for our entire family. Our journey has been painful, frightening, hopeful, and transformative, but if there is one thing I have learned, it is this: families like mine do not give up. We fight, we advocate, and we keep moving forward—because our children deserve every chance at a brighter future, and we will move mountains to give it to them.

What's Next: A Candid Conversation With All Stakeholders

If you are running or planning to rare disease trials and want to protect the patient and caregiver experience, join us for a candid conversation.

“Hearing from experts working on the ground, whether that's at the coal face of trial recruitment, as a PI responsible for the trial and their patients or a caregiver experienced in trial participation - this discussion is guaranteed to reveal insights into what works and where we can all focus our efforts to recruit and retain patients for rare disease trials.”

- Gemma Whiteley, Managing Director at FutureMeds @home

Reserve your seat here: https://events.teams.microsoft.com/event/6aac4d76-75fd-434a-8844-dc1df68a5ba1@322ea526-1bf2-4a7a-b700-66feb5939200