How the FDA Uses Patient-Reported Outcomes (PROs)

How Is the FDA Using PROs?

The FDA has identified PROs¹, also known as patient-generated health data (PGHD), as a focus area for regulatory science, given limitations with data collected in a controlled clinical setting, that patients are the experts in living with their condition, and therefore the FDA increasingly seeks to understand how patients describe their health status.

What Are PROs?

The National Institutes of Health (NIH) defines² PRO as information about a patient’s health that comes directly from a patient.

Examples of PRO information collected include:

• Description of symptoms
• Satisfaction with care
• Impact of a disease or treatment on physical, mental, emotional, spiritual, and social well-being

Use of PROs can guide choice of treatment and improve quality of care.

Regulatory Use of PROs

Regulatory agencies, such as the FDA and the European Medicines Agency (EMA), increasingly value PRO data in supporting labeling claims, demonstrating clinical benefit, and guiding approval decisions. Leveraging robust PRO data can enhance a product’s value proposition by showcasing real-world patient perspectives.

Guidance on Use of PROs

The FDA has issued guidance³ on the use of PROs for regulatory purposes, stressing the importance of early communication since the use and interpretation of PROs require nuance. The guidance outlines standards for developing and validating PRO instruments, focusing on their reliability, validity, and ability to measure treatment impact, ensuring alignment with clinical trial objectives and regulatory requirements.

PROs in Labeling

One review⁴ found that 1 in 5 drugs approved by the FDA from 2006-2015 included PRO-related statements in the drug labeling/package insert. This increased to 1 in 4 over the period 2016 to 2020. Two-thirds of PRO-related labeling was based on primary endpoints and nearly all (98%) was based on disease-related symptoms.

PRO-related statements were most commonly found for drugs approved in diseases of the respiratory system, digestive system, musculoskeletal system, and connective tissue and skin/subcutaneous system. An increase in PRO-related statements was also observed among disease categories that do not traditionally rely on PRO assessment to demonstrate benefit (such as endocrine, nutritional, and metabolic diseases).

Maximize Your Impact With FDA-Recognized PROs

PROs are playing an increasingly vital role in FDA submissions, offering insights into patient experiences that can strengthen your product’s value proposition and support approval. Effectively integrating PROs into your regulatory strategy can enhance patient-centered care and market success.

Contact us today to learn how our team can help you incorporate PROs into your product development, regulatory submissions, and market access strategy to meet FDA standards and drive better patient outcomes.

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References:

1. https://www.fda.gov/science-research/focus-areas-regulatory-science-report/focus-area-patient-reported-outcomes-and-other-clinical-outcome-assessments
2. https://www.cancer.gov/publications/dictionaries/cancer-terms/def/patient-reported-outcome
3. https://www.fda.gov/drugs/development-approval-process-drugs/fda-patient-focused-drug-development-guidance-series-enhancing-incorporation-patients-voice-medical
4. https://www.valueinhealthjournal.com/article/S1098-3015(21)01793-9/fulltext

Magnolia Market Access Authors: Pamela Landsman-Blumberg, Jessica Duchen, Beni Turner