Recently, the Food and Drug Administration (FDA) and American Society of Clinical Oncology (ASCO) held a joint virtual workshop on clinical outcome assessments in clinical trials for cancer treatments. This was a 5th workshop to demonstrate the progress of the FDA’s ongoing initiative to incorporate patient input into drug development.

This workshop provided a forum for productive, collaborative, multidisciplinary discussions to advance the understanding of complex issues surrounding the use of patient reported outcomes (PRO) in clinical trials, bringing together stakeholders from academia, industry, international regulatory, and patient advocacy groups. The specific goal of this meeting was the discussion around regulatory-grade patient-reported data on the tolerability of investigational drugs.

This is an issue we’re passionate about at Koneksa: collecting both objective, as well as patient-reported, measurements in clinical trials is a core part of our business (fully aligned with the FDA’s goals on this topic).

Patient tolerability has become increasingly important to study because, in the emerging plethora of novel anticancer therapies, patients want to discriminate between drugs properly and understand better how they will feel and function taking a certain treatment. Patients want to know the impact that a treatment might have on their cognitive and physical function, their ability to do daily activities, or their ability to maintain their independence.

Moreover, the FDA is looking into ways to make clinical trials more patient-friendly by broadening eligibility criteria, improving trial access and reducing disparities in access. They are learning from healthcare systems and pragmatic clinical trials, decentralized clinical trials, and digital health technology tools for remote data acquisition.

The COVID-19 pandemic has turned out to be a grand experiment for decentralized clinical trials. The recently launched website, “Project Patient Voice,” belongs to the FDA’s Oncology Center of Excellence, and is “an online platform for patients and caregivers along with their healthcare providers to look at patient-reported symptom data collected from cancer clinical trials.” It will provide data visualization tools – tables, graphs, and charts – to help users. The meeting participants discussed how to identify the best methods to assess tolerability and the best frequencies for assessment, and provided some real-life examples (via interactive panel discussions) presenting diverse points of view.

Three important takeaways from this workshop:

1.     The selection of functions to be assessed to determine the tolerability of an investigational drug depends on the specific patient population.

2.     Frequent assessments are needed. Patients are willing to contribute to these assessments, as long as they understand why data collection is important.

3.     Optimization of frequent PRO data collection in clinical trials should be a norm.

The agency has highlighted that technologies are improving researchers’ capabilities to capture ePRO as well as wearable device and sensor data (reference). At Koneksa, we have established academic collaborations (ref, ref) and worked with pharma sponsors  (ref, ref, ref) to collect ePRO and wearable data in cancer patients.

Our experience shows that cancer patients are willing to collect data, and both ePRO and wearable data compliance is very high. These data, unlike conventional clinical-trial endpoints like tumor progression or survival, provide unique insights into a patient’s journey, capture patient input, and objectively measure parameters highlighted by patients, such as physical activity and sleep.

Moreover, these patient-centric measures have a strong potential to differentiate among different therapies and, therefore, guide treatment decisions. We believe, with the FDA, that the extension of life for cancer patients should not be traded for quality of life!