Here are two statistics that may surprise you.
Number one, according to the WIRB-Copernicus Group, the number of oncology trials has skyrocketed over the last decade, almost doubling the number of all other therapeutic areas combined.
Number two, between 2010 and 2020, only 9 out of 108 FDA-approved oncology drugs, a mere 8.3%, included electronic patient-reported outcomes (ePROs) in their labeling.
Clearly, ePRO adoption is lagging in oncology trials. This comes despite recent guidance from the FDA (in 2021 and 2024) explaining how sponsors can use these technologies to ease trials for themselves and their patients.
Regulators expect sponsors to capture the patient’s voice
The FDA’s Patient-Focused Drug Development Guidance Series task force and recent guidance, such as Core Patient-Reported Outcomes in Cancer Clinical Trials, showcase an increasing focus on capturing the patient's voice in drug development. Or, put simply, understanding the impact on the patient’s quality of life as they progress through treatment. To learn more, read on about the symptoms captured during the FDA’s Aura3 project.
The power of ePRO in helping amplify a voice
ePROs give patients a direct voice in reporting their experiences with cancer treatments, providing unfiltered information about symptoms, side effects, and quality of life. Many patients are willing to participate in questionnaires as they understand the importance of documenting treatment toxicity and side effects. However, some may experience survey fatigue, especially when dealing with cancer-related symptoms. ePROs offer a less burdensome way to collect participant data, which enhances participation and helps prevent data loss.
Additionally, when physicians and patients discuss treatment options, having FDA-recognized ePRO data can significantly inform their risk/benefit conversations beyond clinical endpoints like tumor response and survival.
This is because they provide essential insights into:
- How symptoms impact daily life
- Functional status during treatment
- Quality of life considerations
- Treatment side effects from the patient's perspective
For a real-life example, consider the FDA’s “Project Patient Voice,” an initiative that aims to “show patient-reported symptom data consistently from select cancer clinical trials of approved products.”
Their Aura3 data show the changes in quality of life that some participants experienced as they progressed through the trial. According to the FDA, “before starting treatment, 25% of patients who received the trial drug reported having nausea (ranging from rarely to almost constantly).
After beginning treatment, 41% of patients who received the trial drug reported their nausea had worsened (increased by at least 1 point on a 0 – 4 scale) at any time while on treatment. After starting treatment, 6% of patients who received the trial drug reported their nausea had worsened to either occurring frequently or almost constantly at any time while on treatment.”
As the data shows, we can see that participants experience a myriad of effects during a trial that the industry is increasingly expected to capture and report.
Why ePRO and oncology are perfect for capturing the patient’s voice
For several critical reasons, ePRO forms a perfect union when it comes to oncology trials.
First, they provide real-time insights into the patient experience during treatment, capturing nuanced information about symptom burden that might otherwise go unreported. With newer targeted cancer therapies often producing chronic but less severe side effects than traditional chemotherapy, these patient-reported measures become even more valuable.
Second, the digital nature of these tools addresses one of the most significant challenges in cancer trials: patient burden. By allowing patients to report symptoms remotely and at their convenience, ePRO reduces the fatigue associated with travel and participation while also improving data quality and completeness. This becomes particularly important given the lengthy duration of many modern oncology trials.
Third, the standardization offered by systems like the PRO-CTCAE, a type of standardized assessment, provides consistency across studies, enabling meaningful comparisons between different therapeutic approaches. This standardization and electronic data capture create a more complete dataset that can influence regulatory decisions, clinical practice guidelines, and future trial designs.
A step towards better clinical trials and care
As we move forward, integrating ePRO measures into oncology trials isn't just about checking a regulatory box; it's about fundamentally changing how we develop cancer therapies by keeping the patient voice at the center of the process.
With continued refinement and regulatory support, we can expect to see more oncology products with patient-reported outcome-based labeling claims, ultimately leading to treatments that not only extend a patient’s life but also improve its quality.
