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This Year’s FDA-ASCO Workshop Focused on Collection of Patient-Reported Tolerability Data From Clinical Trials


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OCE Insights is an occasional column developed for The ASCO Post by members of the Oncology Center of Excellence (OCE) at the U.S. Food and Drug Administration (FDA). In this installment, Vishal Bhatnagar, MD, Associate Director for Patient Outcomes, Bellinda King-Kallimanis, PhD, Senior Staff Fellow, Paul Kluetz, MD, Deputy Director of the OCE, Heidi Klepin, MD, of Wake Forest School of Medicine, and Kathryn Mileham, MD, of Atrium Health–Levine Cancer Institute (representing the ASCO Cancer Research Committee), provide an overview of the fifth annual FDA-ASCO Clinical Outcomes Assessments in Cancer Clinical Trials (COA-CCT) workshop held on July 17, 2020.

Meeting Goals and Theme

What was the overall theme of this year’s meeting?

Since the inception of the COA-CCT workshop in 2015, the goal has been to provide a forum for productive, topical discussions regarding the use of patient-reported outcome (PRO) measures and, more broadly, clinical outcome assessments (COAs) in cancer clinical trials. This year, we discussed the concept of regulatory-grade patient-reported tolerability data.

Specifically, the morning sessions focused on considerations for the collection of tolerability data (eg, how to select patient-reported symptoms for assessment and timing of assessments) and the importance of assessing overall side-effect bother. The afternoon portion of the workshop was dedicated to introducing and gathering feedback on the new OCE Project Patient Voice pilot website.

Each workshop session was moderated by OCE staff. Panelists included patients, clinicians, industry representatives, academics, and members of global regulatory agencies.

Project Patient Voice

How did the sessions on symptom selection, assessment frequency, and overall side-effect bother relate to Project Patient Voice?

Unbiased selection of which symptomatic adverse events to assess, identifying an optimal assessment frequency, and understanding global side-effect bother in cancer clinical trials allow for high-quality, informative tolerability data, which can then be shared on Project Patient Voice. Project Patient Voice is a Web-based platform for health-care providers to look at patient-reported symptom data collected from cancer clinical trials, in order to discuss them at the point of care with patients and their caregivers. By creating this platform, we hope that sponsors of cancer clinical trials will more rigorously collect patient-reported tolerability data using many of the methods discussed during the workshop.

Improving Data Collection

Based on the workshop discussion, what are some ways to improve collection of patient-reported tolerability data in cancer clinical trials?

In the morning sessions, panelists highlighted the host of potential symptom questions contained in item libraries. Choosing these questions provides the advantage of increased flexibility over existing off-the-shelf static questionnaires. When selecting which patient-reported symptoms to assess from the library, it is best to use a combination of qualitative research, which engages patients, along with mechanistic data and prior clinical data to ensure you are measuring important, high-incidence, expected toxicities. Using this approach will help prevent “cherry picking” symptoms to favor the experimental trial arm and can help establish a succinct set of symptoms.

Another topic discussed was the timing of patient-reported outcome assessments, which is of particular importance when the administration schedule of therapy differs between treatment arms. Using de-identified trial data, workshop presenters showed that the occurrence and severity of patient-reported side effects can differ depending on the timing of assessments, using the example of comparing a daily orally administered drug to an intravenous (IV) infusion given every 3 weeks. Panelists generally concluded that ensuring appropriate timing of patient-reported outcome assessments is critical to making certain we do not miss important toxicities that may occur between assessments when “rest” periods are part of a treatment, as is the case with many infusional treatments.

“Even in its digital format, this workshop generated dialogue from all stakeholders, and the patient voice was included in every session.”
— Kathryn Mileham, MD

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Electronic data capture was raised as a tool to aid in the assessment of patient-reported symptoms. For example, electronic data capture could be used to improve the use of free text boxes to capture additional symptoms (ie, providing drop-down options to minimize variation in reporting of a particular symptom). It can also assist with patient-reported outcome assessments that may be needed between clinic visits to ensure adequate assessment frequency.

Despite the potential benefits, however, some challenges remain in the widespread implementation of electronic data capture. A backup plan where “paper and pencil” can be used is one solution to overcoming the challenge of malfunctioning devices.

New Approaches

Dr. Klepin, as a recurring chairperson for this annual workshop, how was the 2020 workshop different from previous workshops?

This workshop was different from previous workshops in several ways. First and most obvious was the transition to a digital format. To adapt to the digital platform, each segment was designed to enhance an exchange of ideas between panelists through moderated discussion. This format allowed for a rich discussion of important considerations, practical challenges, and strategies to overcome those challenges on topics such as item selection and timing of assessments. The opportunity for dynamic dialogue and problem-solving from varied stakeholder perspectives was an important feature of this meeting.

The content of this workshop differed from prior workshops but was built on the foundation laid previously, to advance the discussion of how to approach the integration of patient-reported outcomes into clinical trials. This workshop, in particular, was infused with the patient perspective and highlighted very clearly the importance of “starting with the end in mind.” Each consideration in study design—from exactly what to measure, when and how to measure it, how to analyze it, and ultimately, how to interpret the information—should be informed by the perspective of patients.

The final sessions of the workshop brought the previous methodologic discussions full circle by demonstrating how visualization of patient--reported outcome data collected in clinical trials can inform discussions about treatment tolerability among patients, caregivers, and their -providers.

Strong Patient Participation

Dr. Mileham, what was your perspective on this workshop?

Having not previously participated in this workshop, I was most impressed with the progress made in identifying patient-reported outcomes as a priority and determining mechanisms for integrating clinical outcome assessments into clinical trials. In its fifth year, the COA-CCT workshop continues to drive interest, with 60% of this year’s record attendance being first-time attendees, many of whom were patients.

Even in its digital format, this workshop generated dialogue from all stakeholders, and the patient voice was included in every session. Having such strong patient participation was unique and yet imperative to enhance the discussion. I appreciated that the workshop addressed practical challenges in the rigorous collection of patient-reported symptoms. Despite these challenges, the FDA-ASCO COA-CCT workshop participants recognized the ongoing need for patient-reported outcome implementation, data management, and result interpretation as a part of its evolution to improve the patient experience.

In my practice, Project Patient Voice has already provided adjunct guidance in assessing tolerability of therapy. The terms are often more relatable to patients, and the longitudinal reports assist in point-of-care decisions. As a thoracic medical oncologist, I have referred some of my patients being treated with osimertinib to the Project Patient Voice website. This workshop successfully unites all stakeholders in addressing the complexities in collecting and reporting patient tolerability assessments, while also creating an action plan for next steps in enhancing those assessment reports through Project Patient Voice.

Additional Feedback

Did patients and health-care providers provide feedback regarding Project Patient Voice?

The value of Project Patient Voice as an important resource was highlighted throughout the entire workshop, but discussion of the details and direct feedback from panelists were elicited in the afternoon sessions. As more anticancer therapeutics are added to the website, there will be greater potential for Project Patient Voice to be a useful tool for shared decision-making between patients and providers. Panelists also emphasized that the data presented on Project Patient Voice are not intended to replace the FDA product label, but rather, to serve as a complement to labeled safety information. Of note, Project Patient Voice provides a publicly facing resource for accessing data provided by patients enrolled in clinical trials, which typically are not freely accessible to all.

Three different types of data presentations (tables, stacked bar charts, and pie charts) are included on the Project Patient Voice website. Both clinician and patient panelists commented that although there is a substantial amount of information presented, the various data visualizations could help in facilitating patient-provider conversations.

Panelists also acknowledged that using consistent visualization formats across studies will increase website users’ familiarity with the visual displays, which will lead to easier interpretation over time. Given the fast pace of a typical clinic workflow, clinician panelists commented that they would likely focus on the specific side effects of concern to the individual patient being seen. Finally, although there were comments about the complexity of the data presented, when asked to complete a survey with questions assessing the interpretability of Project Patient Voice data, the majority of the workshop audience was able to answer the interpretation questions correctly.

Website Optimization

What is the current status of Project Patient Voice?

Over the coming months, the OCE will review feedback received during the workshop and directly via the Project Patient Voice website to consider improvements. Forthcoming manuscripts will provide additional details regarding feedback on Project Patient Voice and the process for sponsors to volunteer for the next version of the pilot.

Although there will be continued improvements and enhancements with the website, the OCE envisions Project Patient Voice to remain in a pilot phase as the website is optimized and scalability issues are assessed. During the workshop, stakeholders and workshop panelists expressed a desire to add additional treatments to populate the Project Patient Voice website.

For More Information

Is there a recording of the workshop?

Yes, all of the workshop materials can be found here: https://www.fda.gov/drugs/news-events-human-drugs/fda-asco-public-workshop-2020-clinical-outcome-assessments--cancer-clinical-trials-fifth-annual. The available content includes slides and recordings from all of the sessions. 

DISCLOSURE: All authors reported no conflicts of interest.


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