A Review of Patient-Reported Outcomes Labeling for Oncology Drugs Approved by the FDA and the EMA (2012-2016)

Abstract

To compare US Food and Drug Administration (FDA) and European Medicines Agency (EMA) labeling for evidence based on patient-reported outcomes (PROs) of new oncology treatments approved by both agencies. Methods: Oncology drugs and indications approved between 2012 and 2016 by both the FDA and the EMA were identified. PRO-related language and analysis reported in US product labels and drug approval packages and EMA summaries of product characteristics were compared for each indication. Results: In total, 49 oncology drugs were approved for a total of 64 indications. Of the 64 indications, 45 (70.3%) included PRO data in either regulatory submission. No FDA PRO labeling was identified. PRO language was included in the summary of product characteristics for 21 (46.7%) of 45 indications. European Organisation for Research and Treatment of Cancer and Functional Assessment of Cancer Therapy measures were used frequently in submissions. FDA's comments suggest that aspects of study design (eg, open labels) or the validity of PRO measures was the primary reason for the lack of labeling based on PRO endpoints. Both agencies identified missing PRO data as problematic for interpretation. Conclusions: During this time period, the FDA and the EMA used different evidentiary standards to assess PRO data from oncology studies, with the EMA more likely to accept data from open-label studies and broad concepts such as health-related quality of life. An understanding of the key differences between the agencies may guide sponsor PRO strategy when pursuing labeling. Patient-focused proximal concepts are more likely than distal concepts to receive positive reviews.

Quality of life in patients with relapsed/refractory multiple myeloma during ixazomib-thalidomide-dexamethasone induction and ixazomib maintenance therapy and comparison to the general population

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Prospective development of a patient‐reported outcomes instrument for desmoid tumors or aggressive fibromatosis

“…To date, disease‐specific PROs approved for use in the regulatory setting have been established only for prostate cancer, non–small cell lung cancer, tenosynovial giant cell tumors, and myelodysplastic syndrome . Here, we describe the feasibility and prospective establishment of content validity of the first PRO instrument for DTs.…”

Applying the Behavioural and Social Sciences Research (BSSR) Functional Framework to HIV Cure Research

“…Supportive BSSR may also include patient‐reported outcomes (PROs) and patient‐reported experience measures (PREMs) completed by participants to assess the effects of study participation on various critical domains, such as well‐being, symptoms, functioning, adverse events and experiences with interventions, studies and ATIs . There is a strong precedent for including PROs/PREMs in oncology research testing similar novel innovations, such as immunotherapies, targeted therapies, small molecules and stem cell transplants . Very little data exist about participants’ experiences and quality of life outcomes within HIV cure trials, and their views of these trials after participating .…”