Photo: Dr. Ziya Gokaslan and chordoma survivor, Susie Rinehart
Clinical trials are essential to finding better treatments for chordoma. They help the medical community learn whether a promising treatment is safe and effective, and they can give patients access to new options that could potentially prolong their lives.
For a trial to answer the questions it was designed to address, enough patients need to be able to participate and stay involved. For a rare cancer like chordoma, that can be especially challenging: the number of patients eligible for any given trial is small, and they’re spread across the world. Many are also managing the effects of chordoma or prior treatment, which can make frequent trips to a trial site difficult. These and other barriers can make it harder for trials to enroll enough patients and reach completion.
To help more trials succeed, we wanted to hear directly from chordoma patients and caregivers: What matters most when considering clinical trials? And what would make it easier to join a trial and stay enrolled?
We partnered with Edge Research, an independent research firm, to explore these questions through small-group discussions, surveys, and interviews with chordoma patients and caregivers. More than 350 people from 39 countries shared their experiences, priorities, questions, and concerns.
The findings, now available in a new white paper, can help researchers design trials around the realities of patients’ lives. They also point to ways that care teams can better support patients and caregivers as they explore their options.
What we heard from patients and caregivers
- They see hope in clinical trials, but also have understandable concerns. Many are motivated by the possibility of controlling their tumor, improving their quality of life, and helping advance treatments for others with chordoma. At the same time, side effects, costs, travel, and the time required can make the decision to participate complicated.
- Clear information makes a difference. Patients and caregivers want straightforward explanations of how a trial works, its potential risks and benefits, what participation involves, and where to turn with questions. Among patients who were familiar with or had heard about chordoma trials, only 15 percent said they understood the information they received “very well.”
- Doctors play an important role in conversations about trials. Patients value their doctors’ guidance and want to hear about trials even early in their disease course, before a trial may be a care option. These conversations give patients and caregivers time to learn and ask questions before facing a decision.
- Practical details matter. Travel, expenses, time away from home, and coordination with a patient’s existing care team all shape whether a trial feels possible. When appropriate, offering local lab work or scans, combining appointments, and helping with logistics could make participation more manageable.
- Patients want to feel supported throughout the experience. Participants described the importance of responsive communication, careful management of side effects, and being treated as individuals.
- The results that matter to patients go beyond tumor shrinkage. Many also value treatments that keep their tumor from growing, relieve symptoms, or improve their quality of life. Measuring these outcomes can help researchers understand whether a treatment makes a meaningful difference in patients’ lives.
This work supports aims in our 2030 Strategic Plan, including enabling more patients to participate in clinical trials in order to advance better treatments. It will also help inform our near-term efforts to develop a chordoma platform trial, an approach designed to make it possible to study promising treatments more efficiently.
Resources and next steps
Our Patient Navigators can answer questions, help patients and caregivers explore relevant trials, and support them through the process of joining a study. Our Clinical Trial Assistance Program can also help patients with travel-related costs for eligible trials.
We’re sharing what we learned with researchers, clinicians, trial sponsors, and study teams to help guide the design of future trials and strengthen the information and support available to patients.
We’re deeply grateful to the patients and caregivers who shared their perspectives, and to the trial teams working to advance better treatments. Together, we’re making trials more responsive to patients and generating the evidence needed to move promising therapies forward.