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What is it about? The study focuses on the use of patient-reported outcome measures and clinician-reported outcome measures in drug labels for orphan drugs approved by the US Food and Drug Administration (FDA) between 2018 and 2024. These measures provide insights into how treatments impact patients' symptoms and quality of life. The research is important because it examines the extent to which these patient-centered tools are used in drug development, especially for rare diseases that affect fewer than 200,000 people in the United States. The problem addressed is the limited use of these outcome measures in orphan drug labels, which hinders the communication of a treatment’s full impact on patients. Previous studies indicated a gap in understanding the trends and implications of these measures' usage, and this article aims to fill that gap by providing updated data and analysis. The paper suggests that increasing the integration of these outcome measures in drug labels can enhance patient care and convey the treatment value more effectively.

How was the research conducted? The research is based on reviewing FDA drug labels to identify trends in the use of patient- and clinician-reported outcome measures. The study examined labels from new drugs and biologic licenses with orphan designation from 2018 to 2024. Researchers looked for mentions of these outcome measures and collected data on various aspects like study design and outcomes measured. They used descriptive and trend analysis methods to compare the findings with data from 2002 to 2017. The study focused on drugs approved for rare diseases, which are often challenging to study due to small patient populations. The researchers chose this method to address the need for patient-centered data in regulatory evaluations.

What were the results? The main finding is that the inclusion of patient-reported outcome measures in orphan drug labels increased from 8.3% in the earlier period to 13.5% in 2018-2024. Clinician-reported outcome measures appeared in 10.1% of the labels during the recent period. Notably, many of these measures were positioned as primary endpoints in clinical trials, indicating their growing importance. An unexpected finding was that although these tools are increasingly used, they are still not specific to rare diseases, which limits their effectiveness.

Why are the results important? These results have significant implications for health technology assessment agencies as they highlight the need for patient-centered tools in evaluating drug efficacy. If these findings lead to more widespread adoption, it could change clinical practice by focusing more on patient experiences. Patients, especially those with rare diseases, would benefit from treatments better tailored to their needs and experiences. Long term, the results could spur further development of specialized outcome measures, improving future drug development processes.

What are the strengths and weaknesses of this study? The study's strength lies in its comprehensive analysis of recent trends in patient- and clinician-reported outcome measures in FDA orphan drug labels, filling a vital knowledge gap. However, a limitation is that it only considers FDA labels, not the broader drug development process where these measures might also be used. Future research could expand by examining these tools' role in global regulatory environments and across different stages of drug development to enhance understanding and application.

Note: This content was created with assistance from artificial intelligence (AI) and has been reviewed and edited by ISPOR staff. For more information or for inquiries on ISPOR’s AI policy, click here or contact us at info@ispor.org.