Patient-reported outcomes in the post-approval environment: Opportunities and challenges

This paper explores the US context of the opportunities and challenges of collecting and using patient-reported outcomes (PROs) in the post-approval environment. Firstly, an overarching goal of real-world comparisons of therapies based on their benefits and harms is borne in mind whilst placing PROs in the landscape of comparative effectiveness research. Secondly, a review of opportunities and challenges relating to the collection and use of real-world patient-reported outcome (PRO) data in clinical and community settings is presented, highlighting patient monitoring such as in post-marketing/post-approval (phase IV) studies, through formulary management and reimbursement systems, and the utilization of available technologies. The paper ends with a summary of policy considerations in optimizing these opportunities and addressing the related challenges. Throughout this paper, the use of the term ‘pre-approval environment’ is intended to refer to the time and efforts preceding the regulatory approval of a pharmaceutical product, and ‘post-approval environment’ on the other hand refers to the time and efforts proceeding the attainment of product approval and the respective labeling claims. ‘Real-world’ is intended to refer to settings other than clinical trials (i.e., clinical practice and community settings, and similar patient monitoring programs).