Presentation (CTI)

OBJECTIVES: Since the establishment of the Patient-Centered Outcomes Research Institute in 2010, there has been a growing emphasis on integrating outcomes significant to patients, such as HRQoL and other PROs, into regulatory decisions. The study aimed to assess the frequency of PRO assessments in pivotal trials and their representation on FDA labels for drugs approved for rheumatoid arthritis (RA), asthma, and non-small cell lung cancer (NSCLC).
METHODS: Recent FDA labels for drugs approved for RA, asthma, and NSCLC post-2010 were reviewed for any mention of PRO assessments, including multidimensional outcomes combining patient-reported components and clinical assessments. Trials referenced in the labels were cross-checked on clinicaltrials.gov to review the list of assessed outcomes for inclusion of PRO measures.
RESULTS: The review identified 6 approved labels for RA, 19 for asthma, and 34 for NSCLC. As expected, all RA product labels cited PROs, including multidimensional outcomes like the American College of Rheumatology 20/50/70 Response Criteria and Disease Activity Score, sleep and pain assessments, and generic HRQoL measures. Most asthma labels mentioned HRQoL (79%) and/or other PROs (84%). Conversely, NSCLC labels seldom included PRO assessments, with only 1 (3%) mentioning HRQoL and 1 (3%) referencing other PROs. The review of the pivotal trials included in the FDA labels revealed that although PROs were measured in the clinical trials for NSCLC, those were considered secondary/exploratory and therefore, were not included in the FDA labels.
CONCLUSIONS: Among the evaluated therapeutic areas, RA labels most commonly included PROs, aligning with the disease's patient-centered clinical nature. In contrast, NSCLC labels seldom highlighted PROs despite their assessment in pivotal trials. As patient-centered care gains prominence, PROs offer valuable insights into condition impact and treatment effects, warranting consideration in the evaluation of new medicinal products.