Plain Language Summary

This study investigates the growing use of clinical outcome assessments in drug labeling by the US Food and Drug Administration (FDA) and in health technology assessments (conducted by key European agencies in France, Germany, and England. Clinical outcome assessments are tools used in clinical trials to measure patients' health outcomes, such as symptoms, quality of life, and daily functioning. They provide insights into how patients experience diseases and treatments, which is vital for developing patient-centered healthcare interventions.

From 2012 to 2022, the inclusion of nonmandatory clinical outcome assessments—those not required for primary endpoint assessment or cost-effectiveness analyses—in FDA labels increased from 18.6% to 28.4%. Similarly, their inclusion in health technology assessments rose from 45.2% to 64.9%. This upward trend reflects a growing recognition of the importance of capturing patient experiences beyond the mandatory requirements, indicating a shift towards more patient-centric approaches in drug development and market access decisions.

The study highlights differences between FDA labels and health technology assessments in terms of the types of clinical outcome assessments used. In FDA labels, most clinical outcome assessments focused on signs and symptoms, aligning with the FDA's emphasis on measuring direct disease manifestations and treatment effects. Conversely, health technology assessments predominantly used clinical outcome assessments that measured health status and quality of life, reflecting their broader evaluative goals.

The trend varied across therapeutic areas. For FDA labels, respiratory diseases showed the most significant increase in clinical outcome assessment inclusion, while oncology and blood and immune system diseases saw decreases. In health technology assessments, cardiovascular diseases experienced the largest increase, with substantial growth also noted in areas like endocrine and metabolic, skin, respiratory, and digestive diseases.

For patients, these developments mean that their experiences are increasingly considered in regulatory and market access processes, potentially leading to more effective and tailored healthcare solutions. Healthcare decision makers can leverage these insights to prioritize patient-centered data in evaluations and recommendations, ensuring that new treatments align with patient needs and preferences. Researchers are encouraged to continue integrating diverse clinical outcome assessment measures in clinical trials to enhance the understanding of patient experiences and improve treatment outcomes. Overall, the study underscores the evolving landscape where clinical outcome assessments are increasingly recognized for their value in capturing patient experiences, shaping both regulatory approval and market access strategies.

^{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.}