Interview: Inclusion of Health-Related Quality of Life (HRQoL) and other Patient-Reported Outcomes (PROs) in the United States Food and Drug Administration (FDA) labels

Sarah Cadarette: The inspiration for this research stemmed from the growing interest in patient-reported outcomes (PROs) over the last decade, particularly within the United States, where historically they have not been a major focus for regulatory decisions. My team frequently encounters questions about PROs and quality of life during literature reviews. This led us to investigate whether these outcomes are being evaluated in registrational trials and if they translate to FDA labels, which are crucial for healthcare providers and patients. The research aimed to understand the role of PROs in various therapeutic areas and how they are perceived in regulatory contexts. 

Sarah Cadarette: While there was no formal hypothesis, we correctly anticipated that there wouldn't be much representation of PROs on FDA labels. This assumption was confirmed through our research, particularly in the therapeutic areas chosen for the study. The team considered a range of conditions, including rheumatoid arthritis, asthma, and of non-small cell lung cancer (NSCLC), to cover the spectrum of where PROs are clinically important. The expectation was that rheumatoid arthritis would show the most PROs, while NSCLC would show the least. This aligned with their findings. 

Sarah Cadarette: One surprising element in the research was the variety of tools used for PROs in NSCLC trials. Although there were not many labels including PROs, the diversity in the tools employed was greater than anticipated. This unexpected finding suggests that while PROs may not be prominently featured on labels, they are being considered in trial designs to a certain extent. This variety indicates a broader interest in exploring different methods for capturing patient experiences, even if they are not yet reflected in labeling practices. 

Sarah Cadarette: This research is a valuable tool for planning clinical trials and developing evidence generation plans. By understanding what PROs have been previously used and their presence on labels, biopharma can better design their trials to include relevant outcomes. This research also serves as a landscape assessment, offering insights into the types of PROs that could be beneficial to include in future trials. The next steps involve using these findings to aid in the strategic planning of trial programs, ensuring that PROs are effectively integrated into drug development processes.