Clinical Trials Succeed When Patients Talk — And The Entire Ecosystem Listens
By Ann Graham, founder and executive director, MIB Agents
When 20-year-old osteosarcoma patient Jacob Knudsen considers joining a clinical trial, the decision is rarely simple. Enrolling in a trial can mean traveling across the country, navigating complex eligibility requirements, and committing to a treatment whose outcomes are still uncertain.
“Clinical trials are good,” he says. “But you really have to do your research. You cannot just jump into one.”
For patients facing rare cancers, these choices are not theoretical. They are deeply personal decisions made while balancing hope, risk, logistics, and quality of life. Jacob has participated in multiple clinical trials during his cancer journey. Finding those opportunities required months of research by his family, reviewing studies online, consulting oncologists across the country, and tracking emerging therapies that might offer the best chance of keeping the cancer at bay.
His experience reflects a broader reality: The success of clinical trials depends on far more than scientific design. It depends on how effectively the entire ecosystem surrounding those trials communicates and collaborates.
I have seen this ecosystem from multiple vantage points, first as a caregiver, then as a patient, and now as the founder of an organization that funds research aimed at improving outcomes for osteosarcoma. Through this work, we partner directly with researchers, clinicians, industry, and regulators to improve outcomes and accelerate progress in osteosarcoma.
Patient Voices Must Be Heard
Most researchers understand the importance of patient-centric trials. The challenge is execution.
Over the past several years, we have worked to bring patient voices directly into the clinical trial process. That has included presenting at the FDA, participating in patient panels alongside young people living with osteosarcoma, and building platforms that connect patients, clinicians, and researchers in real time.
At one FDA meeting, two members of the MIB Agents Junior Advisory Board, both teenagers, shared their experiences navigating treatment and clinical trials. One joined virtually from her living room because she was too ill to travel. She passed away two weeks later. The Mikaela Naylon Give Kids a Chance Act, named in her honor, was signed into law shortly after. Moments like these have demonstrated that when patient voices are not only included but centered, they can influence how decision makers think about urgency, access, and trial design.
We have also focused on creating infrastructure that makes clinical trials more understandable and accessible. Through the MIB Agents OsteoBites webinar series, principal investigators walk through their trials in detail and answer questions from patients, families, and clinicians. This creates a level of transparency that goes far beyond what is available in traditional trial listings.
In parallel, MIB Agents’ Research Bee initiative gathers real-world patient insights through interviews, focus groups, and surveys, allowing researchers to incorporate lived experience directly into trial design. We have seen firsthand how this can change outcomes. In one case, a researcher whose trial failed adjusted the design based on feedback from Research Bee and relaunched the trial with success.
Eugenie Kleinerman, MD, of MD Anderson Cancer Center, shared that this type of engagement can have a direct impact “with facilitating our connection with the patients and their caregivers. Not only was it informative but so motivating to the members of our team. We gained valuable insight which changed the trial design and provided the rationale that we needed.”
These experiences reinforce a consistent lesson: What works is early, direct, and ongoing engagement with the patient community. What does not work is designing trials in isolation and expecting patients to adapt to them.
Patients’ Biggest Pain Points
Some of the most common barriers we see are practical. Trial sites are often limited to a single institution or a small number of locations, requiring patients to travel long distances. For Jacob, participating in trials has meant traveling across the country, with costs that are not covered by insurance.
“Travel is a huge thing,” he says. “A lot of these trials are only in one hospital.”
Eligibility criteria can also unintentionally exclude patients who reflect real-world disease patterns. Osteosarcoma is not uniform, and overly narrow criteria can make it difficult to enroll enough patients to generate meaningful data.
Communication remains another critical gap. Patients often enter trials with limited visibility into how treatments are performing.
“I would like to know more about how it is doing with other patients,” Jacob says. “That is probably the biggest thing.”
Patients understand that clinical research carries uncertainty. They are not asking for guarantees. They are asking for context that helps them make informed decisions about how a trial fits into their lives.
We have also seen the difference strong clinical research coordinators can make. In trials where coordinators are supported and accessible, patients experience clearer communication and greater trust. In trials where these roles are overstretched, confusion and frustration quickly follow.
Speed is another area where the gap between research timelines and patient reality becomes clear. For patients with relapsed disease, waiting years for trial results is not theoretical. It directly impacts treatment decisions in real time.
Participation in clinical trials is often framed as access to treatment. For many patients, it is also about contribution.
“For me, part of doing trials is giving back,” Jacob says. “Maybe by doing them, you are helping the next kid who gets this disease.”
5 Practical Ways To Be Patient-Centric
In rare cancers, every patient who participates contributes to the knowledge needed to move the field forward. That contribution deserves a system designed with equal care. If we want clinical trials to succeed, we need to move beyond broad commitments to patient centricity and focus on practical implementation:
- Engage patient advocates before protocols are finalized, not after.
- Design eligibility criteria that reflect real-world disease.
- Expand access by increasing trial sites and addressing travel barriers.
- Invest in coordinators who can sustain clear communication.
- Create pathways for patients to better understand how trials are progressing.
These are practical approaches we have seen improve trial participation, strengthen collaboration, and, ultimately, move research forward. When researchers, sponsors, clinicians, and patients work in true alignment, clinical trials can do more than generate data; they can create a system that is both scientifically rigorous and genuinely accessible to the people who need it most.
About The Author:
Ann Graham, founder and executive director of MIB Agents, has been working to Make It Better (MIB) for children battling osteosarcoma since she began her own battle with bone cancer in 2010. Although 43 years old at the time, Ann was treated in the pediatric cancer center at Memorial Sloan Kettering in New York City. During treatments, she met Alyssa Divers, a 12-year-old osteosarcoma patient who bravely battled her cancer until her death in 2012. In the years that followed, Ann’s efforts grew from arranging experiences for pediatric cancer patients and their families to actively raising awareness of osteosarcoma in the public, medical, research, and funding communities, and going beyond this to actively working to fund research, provide support programs from diagnosis onward, and create and offer education for doctors, researchers, patients, and families. The collaborations and partnerships that have developed out of MIB Agents’ work are generating hope and creating pathways to progress.