FDA Launches Pilot Website Housing Publicly Available PRO Data From Cancer Trials

Amy Abernethy, MD, PhD

The FDA has launched a new initiative called Project Patient Voice, which is a website that will serve as a source of publicly available information regarding patient-reported symptoms from cancer trials examining marketed treatments, according to a recent announcement made by the regulatory agency.

Although patient-reported outcome (PRO) data has historically been evaluated by the FDA as a part of the drug approval process, this information is rarely released to the public in the form of product labeling.

The new website will house a list of clinical trials conducted in the cancer space in which these data had been collected. Each trial listed on the website will have a table containing patient-reported safety information, and each symptom reported can be selected to display a series of charts reflecting the symptom at baseline as well as over the first 6 months of treatment. Notably, this toxicity information is not currently available in standard FDA safety tables.

“Project Patient Voice has been initiated by the Oncology Center of Excellence to give patients and health care professions unique information on symptomatic side effects to better inform their treatment choices,” Amy Abernethy, MD, PhD, the FDA’s principal deputy commissioner, stated in the press release. “The Project Patient Voice pilot is a significant step in advancing a patient-centered approach to oncology drug development. Where patient-reported symptom information is collected rigorously, this information should be readily available to patients.”

PRO data are gathered through the use of questionnaires that are distributed to patients and completed during clinical trials. The questionnaires are developed to collect significant information pertaining to either disease- or treatment-related events, including the severity of the toxicity and how often it presents, according to the FDA.

These data are also capable of providing health care professionals with additional safety information to discuss with patients when considering the use of particular cancer therapies. Importantly, PRO data offer information that is distinctly different from the clinician-reported safety data that are included in the product labeling, according to the regulatory agency. The data documented in the pilot project are obtained directly from patients and offer insight into events prior to treatment initiation, as well as at different time points throughout the treatment journey.

Notably, this initiative will not replace the clinician-reported safety data that are included on drug labeling; the project is meant to be a complementary source of information. The website should not substitute advice from a health care professional and it should not be used as the sole resource to inform clinical decisions in the cancer community.

“There have long been calls to provide information to patients about how they may feel and function when receiving a cancer treatment. By initiating Project Patient Voice, we are moving toward standardized methods to display these outcomes, starting with patient-reported symptomatic adverse events (AEs),” Paul Kluetz, MD, deputy director of the FDA’s OCE, stated in the press release. “We encourage sponsors to collect this data systematically and look forward to welcoming additional sponsor collaborations as we work to help further serve the patient community.”

The visualizations and data included on the Patient Project Voice website are voluntarily supplied by the pharmaceutical companies that conducted the clinical trials. AstraZeneca was the first company to provide PRO data for one of their FDA-approved cancer agents. The company is working with the FDA to determine approaches for optimally displaying the information for both providers and patients.

For the first phase of this project, the FDA will showcase 1 trial on the Project Patient Voice website to obtain feedback from the public. Using that information, the agency will take steps to further optimize the website for users.

On July 17, 2020, the FDA will seek feedback from the public on the project during a virtual public workshop that is co-sponsored by the American Society of Clinical Oncology. Healthcare professionals, patients, health outcome researchers, industry, advocacy groups, and other stakeholders will be in attendance. Topics of discussion will include trial design considerations to optimize the collection of PRO symptomatic AEs and feedback on the presentation of PRO data on the website.

Reference

1. FDA announces first of its kind pilot program to communicate patient reported outcomes from cancer clinical trials. News release. June 23, 2020. Accessed June 24, 2020. bit.ly/3dBgk4g.