A 15 Year Review (2006-2020) of Patient-Reported Outcome (PRO) in United States Oncology Product Labeling and Trends in Sponsor Size and Oncology Experience.

Objectives: Despite wide use of PRO tools in clinical development, resulting data is rarely incorporated into the US label. This study reviewed oncology product labels approved by the Food and Drug Administration (FDA) between 2006 and 2020 to determine if the number of PRO included in labeling has meaningfully changed. Sponsors were assessed to identify demographic trends in achieving PRO label success.

Methods: FDA-approved drugs were searched utilizing the Drugs@FDA database by month from January 2015 to December 2020 for novel drug and biologic approvals. Products approved between 2006-2014 were identified utilizing the Gnansakthy et al., 2012 and 2016 publications. Labels were reviewed for inclusion of PRO data in the label and product summary basis of approval (SBA). Sponsor size and experience were measured for each year of product approval.

Results: 155 oncology products received initial approval between 2006-2020, of which only 7 contained PRO data in the label. More than half (53.5%) of products had PRO data described in the SBA. Over time, PRO information has increasingly been included in the product marketing application. Sponsors utilizing PRO data tend to be experienced in oncology development and larger in size.

Conclusions: There has been a small increase in inclusion of PRO data in oncology product labeling over the past 15 years. Utilization and analysis of appropriate PRO tools and data remains a challenge to sponsors. Further collaboration with FDA is needed for the development of disease specific PRO tools that provide meaningful data to the targeted patient population.