Patient-Reported Outcomes Labeling for Products Approved by the Office of Hematology and Oncology Products of the US Food and Drug Administration (2010-2014)

Abstract

Although symptoms and functional decrements are common among patients with cancer, PRO labeling is rare in the United States, likely because of logistical hurdles and oncology study design. Recent developments within the FDA OHOP to capture PROs in oncology studies for the purpose of product labeling are encouraging.

Trustworthiness of Patient-Reported Outcomes in Unblinded Cancer Clinical Trials

“…Although there is enthusiasm at the US Food and Drug Administration (FDA) for such a patient-centered approach, evidenced by guidance for industry published on this topic in 2009, 2 most oncology trials and FDA-approved medication labels still do not include information on patient-reported outcomes. 3 …”

“…3 For example, the FDA asserts that patients with pain associated with metastatic prostate cancer might be inclined to report improvements in their pain based simply on the knowledge that they have been assigned to receive a novel therapeutic agent, regardless of the actual properties of that drug or of their actual pain responses. 2,3 The FDA extends this concept to encompass single-arm trials, open-label trials, and blinded trials in which they believe study arm allocation has likely been unmasked owing to imbalances in readily apparent toxic effects between arms. Indeed, in oncology, this scenario now represents most pivotal registration trials.…”

A Review of Patient-Reported Outcome Data for Palliative Cancer Drugs Approved In the European Union

“…Indeed, oncology products may not support PROs likely because of logistical hurdles and/or study design [4] but PRO use is more frequent in clinical trials of products for chronic, disabling conditions where the goal of treatment is not curing but rather an improvement in symptoms, functioning, or quality of life [5]. Low rate of PRO data in the main regulatory document -the SmPC in Europe -may well be one of the reasons why these important measures of patient satisfaction and cost-effectiveness are often under-used in daily practice [6,7].…”

The Spectrum of Functional Rating Scales in Neurology Clinical Trials

“…A recent study found that only 3/40 (7.5 %) of approvals by the Office of Hematology and Oncology Products of the FDA received PRO-related labeling between 2010 and 2014 [81]. Some factors underlying this may include challenges in study design using PROs and the quality of PRO instruments used.…”