In order to assist clinical research efforts to explore potential treatments for various cancers, the US Food and Drug Administration (FDA) has released a draft guidance document, entitled “Core Patient-Reported Outcomes in Cancer Clinical Trials.” When finalized, the resource is intended to deliver recommendations to sponsors as to which patient-reported outcomes (PROs) concepts should be measured in oncological clinical studies.
Richard Pazdur of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research (CDER), said PROs are important in cancer research but can present problems.
“Patients would like to better understand symptoms they may experience and how a cancer therapy can affect their quality of life; one way to accomplish this is to ask patients in clinical trials about the severity of their symptoms and ability to function using rigorously developed patient-reported outcomes,” Pazdur remarked. “However, achieving sufficient consistency and quality of these data in cancer drug applications submitted to the FDA has been a challenge.”
The federal government, Pazdur added, has responded by looking for solutions that reduce the obstacles to obtaining the consistency and quality of data required to ultimately put treatments within reach of the patients that need it.
"The FDA’s Oncology Center of Excellence has undertaken a sustained effort to identify methods to rigorously collect patient-reported outcomes in cancer clinical trials,” he said. “We’ve been engaging with patients and outcomes research experts through a series of public workshops and publicationsExternal Link Disclaimer on which outcomes to measure, how frequently to assess them, and the tools available to do so.”
The document touches upon ways to incorporate assessment of PRO in clinical trials of drugs and biologics, which are designed to support product labeling describing anti-tumor (preventing or inhibiting the formation or growth of tumors) activity in patients with cancer. The guidance also addresses the frequency of PRO assessments within cancer trials, highlighting that PRO assessments should be of sufficient frequency to take into account the administration schedule of the products being studied; it also discusses labeling considerations for PROs submitted with oncology product applications.
“The draft guidance we’re releasing today is intended to improve the quality and consistency of data in order to inform patients with cancer about the symptoms and impacts they may experience during treatment with a cancer therapy,” Pazdur concluded.