A new FDA program is giving patients and providers first-time access to patient-reported outcome data from cancer clinical trials of marketed treatments.
Every day, researchers conduct hundreds of clinical trials in cancer, yielding data about how the latest treatment advances impact the disease. Clinicians look at the results to determine which potential new treatments may or may not be good options for patients. But there are also other assessments that may help determine this: how do patients actually feel and function when they’re taking a medicine? We can get some of these answers from the patient perspective through patient-reported outcomes, or PROs.
While PRO data have historically been analyzed by the US Food and Drug Administration (FDA) during the drug approval process, they have been largely inaccessible to the public. As a result, cancer patients are often left in the dark over how other patients feel and function on treatment – for example, the impact of a therapy on activities of daily functioning, disease symptoms, and symptomatic side effects.
Fortunately, a new FDA program is giving patients and providers first-time access to PRO data from cancer clinical trials of marketed treatments. The FDA’s Oncology Center of Excellence (OCE) launched Project Patient Voice, a pilot website that will evolve to include a list of cancer trials that have available PRO data taken from patient questionnaires. The first cancer trial PRO data being piloted on the site were provided by AstraZeneca.
The pilot website will display a table of patient-reported data collected over time, showing symptoms at baseline and at multiple time points during cancer treatment. The information can give patients and healthcare providers a deeper understanding of the patient experience during cancer trials to help inform treatment decisions.
Project Patient Voice is not meant to replace the clinician-reported safety information that is available as part of a drug's labeling. Data from Project Patient Voice should not substitute for advice from a health care professional. Rather, Project Patient Voice serves as a complement to FDA patient labeling and patient information, not a sole source of information on which to make decisions about medical care. Patients should ask their health care provider if they have any questions about their cancer treatment.
For the first phase of the Project Patient Voice website, AstraZeneca is contributing data from the first clinical trial to report data using the PRO-CTCAE™ patient questionnaire. PRO-CTCAE is a rigorous measurement tool for patients to rate the frequency, severity, interference, or presence/absence of symptomatic adverse events (AEs) that they may experience during a cancer trial. The questionnaire, developed by the National Cancer Institute, asks questions on various AEs, including pain, fatigue, nausea, and other symptoms that can be meaningfully reported from the patient’s perspective during a clinical trial while on an oncology therapy.
As patient-centric healthcare has grown in recent years, more attention is being paid to PRO in cancer trials. With Project Patient Voice, the FDA is aiming to build a publicly accessible repository of PRO data, making it simpler for patients and providers to access and share important information about the patient’s treatment experience.
On July 17th, the FDA will hold a virtual public workshop, co-hosted with the American Society of Clinical Oncology, seeking feedback on the website from stakeholders from across the cancer ecosystem. I invite my fellow healthcare providers to share feedback on the website and data.
The future of PRO in cancer research is exciting. The more it becomes a standard part of cancer clinical trials, the greater the opportunity for it to integrate into clinical practice and help shape cancer care, which may result in better patient outcomes.