Research Review: Improving Patient Reported Outcome (PRO) Collection and Transparency in Blood Cancer Research

The Importance of Patient Perspectives in Clinical Trial Research

Research shows that Patient-Reported Outcomes (PROs) are often not well-reported in cancer clinical trials. PROs are data directly from patients about the effects of treatment, including physical symptoms, emotional responses, and overall well-being. A review of studies on solid tumors found that PROs and Health-Related Quality of Life are frequently underreported. Even when reported, these measures often fail to address long-term impacts. Given the serious nature of blood cancer and its potential to affect daily living and quality of life, it is especially important to consider PROs when studying the effectiveness of a treatment

Measuring Data in Clinical Trial Research

In clinical trials, researchers establish endpoints—measurable data that show how effective a treatment is. There are three main types of endpoints: primary, secondary, and exploratory.

Primary endpoints are outcomes that help to determine how safe and effective the treatment is. These endpoints typically address the main research hypothesis. Secondary endpoints provide additional information about a treatment's effects, often evaluating side effects or quality of life. Exploratory endpoints may reveal patterns or trends and help develop new hypotheses for future research.

Endpoints provide crucial information that guides treatment decisions and shapes the future of blood cancer research. A typical endpoint in blood cancer trials is survival, or whether the treatment helps a patient live longer.

Although PROs are Measured in Most Trials, They Are Not Always Reported

A recent study reviewed publications from eight leading oncology journals to examine how often patient-reported treatment impacts are collected during clinical trials and shared in journal articles. The study looked at 90 blood cancer clinical trials to determine whether PROs were used as an endpoint and whether that data was included in primary trial publications.

Out of the 90 clinical trials, 66 included PROs as an endpoint. In these trials, PROs were a primary endpoint in one trial, a secondary endpoint in 50 trials, and an exploratory endpoint in 15 trials. 

 However, although PRO data was collected in the majority of studies, fewer than half of these studies reported the data in the primary publication. Furthermore, the PRO data that was published showed either improvement or no change in symptoms, with no reports of worsening symptoms. This raises the concern that worsening symptoms may be underreported in clinical trial publications.

HealthTree is Dedicated to Ensuring Patient Perspectives Are Shared

PRO data provides important insights for both researchers and patients. While PROs are often included as an endpoint in blood cancer clinical trials, they are frequently not reported in the final publications. Further, when PRO data is reported, it typically shows improvement or no change, indicating bias in the way the data is presented. When PRO data is not shared, essential details about the patient experience are lost.  Including PRO data in studies and publications is vital for helping providers and patients make more informed decisions about care.

HealthTree is committed to elevating patient perspectives. To achieve this, we publish our research at medical conferences and online to ensure that information about patient experiences is widely available to researchers, medical providers, patients, and caregivers.

To participate in research with HealthTree by taking simple surveys to collect patient-reported outcomes like in the above article, follow the link below.

HealthTree Research

Source: 

• Patient-Reported Outcomes in Phase 3 Clinical Trials for Blood Cancers: A Systematic Review