Centering Patient Experience in Evidence Generation and Trial Design

Advocates emphasized that patient experience is not an add-on to scientific research, rather, a necessary input that shapes whether a clinical trial is feasible and timely, and whether its data can be interpreted in a meaningful way. They described how protocol-defined requirements, such as visit schedules, imaging frequency, expected treatment-related toxicities, and informed consent discussions directly affect a patient’s ability and willingness to participate in clinical research. Kristin noted that treatment decisions are often time-constrained, making transparent and clear communication essential for helping patients and their caregivers make informed decisions. Susan added that trial designs need to consider evolving SOC so that patients assigned to the control arm do not receive an outdated or suboptimal treatment. Their remarks collectively highlighted how communication, timing, and evolving treatment landscapes influence whether patients understand trial options and feel equipped to participate and reinforced that patient experience is a foundational element of trial design.

“We spent a lot of time discussing what happens when the SOC changes during a trial. Adjusting a study mid-trial is difficult, but it matters because patients can end up in arms where the SOC has become obsolete. FDA representatives in our group acknowledged that this is an active area of discussion, and industry partners pointed to real examples where patients were still receiving outdated treatment. Seeing how quickly a trial arm can become misaligned with the current care made the issue very clear.”  – Susan Rouse

What Advocates Brought to the Friends Working Groups

Advocates described the Friends working groups as a space where their lived experiences meaningfully shaped discussions and proposals for innovative, patient-centered trial frameworks and methodologies. Rather than simply reacting to proposals discussed in the Annual Meeting working groups, advocates collaborated with researchers, drug developers, and U.S. Food and Drug Administration (FDA) representatives to develop patient-centered solutions for implementing seamless trials for rare cancers drug development, assessing comparator arm feasibility in global trials, and understanding toxicity in combination therapy studies. Kristin shared how working group discussions considered the scientific, operational, and regulatory factors needed to ensure trials generate high-quality and meaningful data, while also centering practical considerations necessary for high-quality, meaningful data, all while ensuring that trial conduct remains grounded in real . Misha added that across discussions, she noticed a shared sense of urgency among stakeholders to generate reliable evidence without placing unnecessary burden on patients. Through their participation, advocates helped identify where protocol requirements may not be sustainable for patients or their caretakers who are managing ongoing treatment, caregiving responsibilities, financial strain, and the intensive travel and appointment demands often associated with clinical research.

“One thing that really struck me in this working group was the shared sense of urgency. The pharmaceutical teams, FDA representatives, and patient advocates all understood that rare cancer patients do not have time to waste—that urgency is what brought the group together.” – Misha Mehta

Seamless Trial Designs: Improving Timelines and Evidence for Rare Cancers

Misha described seamless trials as an approach that can reduce delays and strengthen the evidence generation in rare cancer settings with small patient populations and limited treatment options. By combining multiple trial phases under a single, continuous protocol, seamless designs allow investigators to refine dosage, expand cohorts, and evaluate early signs of benefit without the pauses required to initiate a new, separate trial, as commonly seen in traditional trial designs. She noted how this structure can help maximize the evidentiary value of each patient and enable timely access to potentially life-saving therapies. Misha also emphasized that patient input is essential when determining the pace of the study and expectations for visits and testing, as these demands need to reflect what participants can realistically manage while living with rare and often aggressive cancers.

“Seamless trials are not a technical improvement; they are a moral one. In rare cancers, especially for pediatric brain tumors, we don’t have the luxury of hundreds of patients or years between each phase. Every patient is precious, and every data point carries a significant amount of weight.” – Misha Mehta

Evaluating Contribution of Effect (COE) for Combination Therapies: Understanding What Each Drug Does

Carol noted that combination therapies have become an important treatment option for patients with cancer due to their ability to target multiple disease pathways simultaneously and improve clinical outcomes. While combination therapies can be effective, each additional drug introduces the potential for added toxicity, making it critical to determine the true contribution of each drug in a combination to the overall therapeutic effect. She emphasized that exposing patients to additional toxicities is acceptable only when each component meaningfully contributes to benefit. As reflected in the , factorial trial designs, in which all possible combinations of the investigational therapy and control are tested, provide the most complete evidence but are often impractical to implement in oncology trials because they are lengthy, resource-intensive, and not feasible for patients with rapidly progressing disease. The working group explored alternative COE strategies, such as leveraging data from other disease settings, evaluating different endpoints, and incorporating patient-reported outcomes into decision making. Carol added that trial timelines directly affect patients, noting that efficient evidence generation is critical because time  in development can translate to lost opportunities for patients.

“Time is money and time is life. I think that’s what’s so important about this (evaluating COE) is the urgency of being able to prove which treatments are contributing to effect, and, how they can benefit patients without any harm.” – Carol Vallett

Comparator Arm Selection in Multi-Regional Clinical Trials (MRCTs): Navigating Global Differences in Care

Panelists discussed how MRCTs introduce additional complexity to trial design and comparator arm selection because of varying SOC, regulatory expectations, and treatment availability across regions. Kristin explained that many patients are unaware of these elements of trial design, yet these differences can shape both the patient experience and the quality of evidence. She stressed that clear and transparent communication about the control arm in the context of evolving SOC is essential when obtaining informed consent, particularly when patients need to make rapid treatment decisions. Susan expanded on this by noting that evolving SOC can intensify these challenges, as MRCTs must be flexible enough to adapt to align with changes in regional best practices, while still preserving scientific rigor. She emphasized that without thoughtful design, patients could inadvertently receive outdated or suboptimal care, further emphasizing the need for MRCT frameworks that can reconcile heterogenous care environments without compromising patient protection, data interpretability, or ethical standards.

“It’s very clear how important the lines of communication are between providers, regulatory bodies, and patients. Decisions are not made in a vacuum, even though from the patient’s side, it can sometimes feel that way if you do not have the full context. Keeping trials patient-centered and safe requires considering many different factors, especially when determining whether a treatment should move to the next phase or toward eventual approval.” – Kristin McJunkins

Key Lessons and Reflections from the Advocacy Perspective

Across discussions, advocates emphasized the ongoing need to balance scientific progress with feasibility, ethical responsibility, and patient safety. Carol reflected on the importance of equipoise, describing the need to ensure that each arm of a trial represents a reasonable, evidence-informed option for participants. Kristin noted that regulatory and clinical decisions occur within a broader ecosystem of providers, regulators, and patients, and must involve continuous dialogue among all stakeholders to ensure early alignment across the board. Misha added that scientific rigor and development speed are not opposing objectives but can be aligned to deliver evidence more efficiently while safeguarding patient welfare. Chris discussed the emotional and human aspects of advocacy, noting that support of cross-functional teams is essential when engaging in work that stems from experiences patients did not choose. Collectively, their reflections reinforce that integrating lived experience into scientific discussions strengthens the relevance, feasibility, speed, and equity of oncology research.

“It all comes back to the patient—the best source comes from those who have lived it, rather than a data point on a poster.” – Chris White