The U.S. Food and Drug Administration recently launched an important patient engagement-based initiative called Project Patient Voice, an initiative of the FDA’s Oncology Center of Excellence (OCE). Project Patient Voice develops a consistent source of publicly available information describing patient-reported symptoms from cancer trials for marketed treatments through a new website. While this patient-reported data has historically been analyzed by the FDA during the drug approval process, it is rarely included in product labeling and, therefore, is largely inaccessible to the public. AstraZeneca stepped up to be the first major pharma to participate in this important step toward incorporating a patient voice into drug development.
TrialSite News offers a brief breakdown of this FDA initiative. The implications of this pilot initiative are significant.
What is the point of Project Patient Voice?
According to FDA Principal Deputy Commissioner Amy Abernethy, MD, PhD, “Project Patient Voice has been initiated by the Oncology Center of Excellence (OCE) to give patients and health care professionals unique information on symptomatic side effects to better inform their treatment choices.” Dr. Abernethy commented, “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.”
Paul Kluetz, MD, deputy director of the FDA’s OCE, declared, “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 towards standardizing methods to display these outcomes, starting with patient-reported symptomatic adverse events.”
How is this Patient-reported outcome (PRO) data usually collected?
PPO data is collected using questionnaires that patients complete during clinical trials. These questionnaires are designed to capture important information about disease-or-treatment-related symptoms. This includes how severe or how often a symptom or side effect occurs. These questionnaires can be part of a digital application on a smartphone app.
Why is Patient-reported data usually collected?
Historically, this data was collected to offer additional and complementary information for health care professionals to discuss with patients, especially when talking about the potential side effects of a particular cancer treatment.
How is this different than what goes on the FDA Label?
The data that becomes part of the set of information associated with the product label involves clinician-reported safety data.
Is this new pilot meant to change the clinician-reported safety information available as part of a drug’s labeling?
No. It will serve as a compliment.
How is the FDA pilot visionary?
With Project Patient Voice, the data that goes on the product label is obtained directly from the patient. This more real-time, dynamic, and patient-centered pilot starts collecting data directly from patients and can reveal symptoms before treatment starts and at multiple time points while receiving cancer treatment.
Are there examples of clinical trials with this information?
Yes, on the Project Patient Voice website, the FDA plans on including a list of cancer clinical trials that have available patient-reported symptom data.
How will these clinical trials work?
Each clinical trial will include a table of the patient-reported symptoms collected. Each patient-reported symptom can be selected to display a series of bar and pie charts describing the patient-reported symptom at baseline (before the treatment commences) and over the first six months of treatment.
Of course, moving forward for these pilot studies, the information now offers insight into side effects not currently available in standard FDA safety labels, including existing symptoms before the start of treatment, symptoms over time, and the subset of patients who did not have a particular symptom prior to starting treatment.
What does the First Phase of the pilot look like?
In the first phase of the pilot website, only one trial will be included while the FDA seeks public feedback on how the information is presented. The FDA will use this feedback to consider improvements to the website in order to make the information as user-friendly as possible.
What is the drug company’s role?
The drug companies will voluntarily provide data used for visualizations on the website.
Who is the first drug company to step up and participate?
AstraZeneca. They have stepped up and are providing patient-reported outcome data for one of their FDA-approved drugs. They have collaborated with the FDA to identify methods to display the information in a way that is informative to health care professionals and patients.
Is this required or just a pilot?
This effort is a pilot for now, although the FDA is encouraging participation. TrialSite News suggests the FDA become even more aggressive in “urging” participation.
Call to Action: The FDA will seek public feedback regarding the Project Patient Voice pilot effort at a virtual public workshop co-sponsored by the American Society of Clinical Oncology on July 17. The “Clinical Outcome Assessments in Cancer Clinical Trials” workshop will include health care providers, patients, health outcome researchers, industry, advocacy groups, and other stakeholders interested in the rigorous measurement of symptom and functional outcomes. The FDA also seeks to obtain feedback on how the patient-reported outcomes symptomatic side effect data is presented on the website. Visit the workshop link.