Patient’s Voice Influences Medical Device Labeling
Patient-reported outcomes (PROs) are increasingly used in medical device clinical trials to capture a patient’s perspective on their health, symptoms, and overall well-being beyond what can be observed or measured objectively. The FDA emphasizes the importance of validating PROs to ensure they are "fit-for-purpose" for regulatory submissions, requiring reliability and validity for specific clinical trial uses. This includes demonstrating that the chosen measures are appropriate for the intended use and population, and that the collected data are sufficient to support labeling claims and regulatory decisions.
PROs Capture Patient Experiences:
PROs offer a unique perspective on a device's effectiveness and safety by capturing information directly from patients about their experiences. This information includes aspects like pain levels, symptom severity, functional limitations, and overall quality of life, which may not be fully apparent through traditional clinical assessments.
FDA Guidance on PROs:
The FDA provides guidance on validating PROs to ensure they are reliable, valid, and appropriate for the intended use and population.
“Fit-for-Purpose" and Labeling Claims:
The FDA emphasizes that PROs must be "fit-for-purpose," meaning they are validated for the specific clinical trial setting and intended use. PRO data can be used to support claims about a device's impact on specific symptoms or quality of life, strengthening the evidence for its effectiveness and safety.
Regulatory decisions:
The FDA uses clinical data, including PRO data, to determine if there is a reasonable assurance that a device is safe and effective for its intended use. PROs play a critical role in informing these decisions, particularly regarding the device's impact on patients' daily lives and quality of life.
Collaboration is Key:
The FDA encourages collaboration in developing and validating PROs and other clinical outcome assessments and emphasizes the importance of transparency throughout the regulatory process.
Beyond Objective Measures:
PROs complement traditional clinical measures by capturing subjective experiences, providing a more comprehensive understanding of a device's impact on patients.
Key links to the information referenced:
Inclusion of patient-reported outcome instruments in US FDA medical device marketing authorizations
