Presentation (CTI)

OBJECTIVES: Clinical outcome assessments (COAs) are essential for evaluating treatment benefits from a patient-centred perspective. Between 2010 and 2019, only 2% (n=10) of asthma products in Japan incorporated patient-reported outcomes. This study aims to follow up on that analysis by examining the progress of COA inclusion in asthma drug development between 2020 and 2024.
METHODS: Asthma treatments approved or with label changes between 2020 and 2024 were identified from the Pharmaceutical and Medical Devices Agency website. Corresponding labels, interview forms (IFs), and patient guidance documents were reviewed for COA-related content.
RESULTS: A total of 175 newly approved or relabelled asthma products (excluding generics) were reviewed, including both adult and paediatric formulations. COAs were identified in one product label and thirteen IFs, but no relevant content was found in patient guidance documents. COAs were often included as secondary endpoints, with the Asthma Control Questionnaire (ACQ) being the most frequently used instrument across both adult and paediatric studies. Compared to products from 2010-2019, more trials from 2020-2024 included Japanese participants. While many trials incorporated COAs, such as the ACQ, to assess treatment effectiveness from the patient’s perspective, the use of COAs in effectiveness evaluation varied across studies. One product used patient asthma diaries for physician-based assessment, though how patient-reported information was considered was unclear. Another paediatric study reported using patient or caregiver impressions to evaluate treatment benefit, but did not distinguish between sources.
CONCLUSIONS: Compared to the 2010-2019 period, the inclusion of COAs in asthma product development has increased, and their use in paediatric trials reflects growing recognition of patient-centred evaluation within Japan’s regulatory and development landscape. However, the overall adoption remains limited, highlighting the need for further integration and transparency of COAs in asthma drug development.