FDA Reported Use of Patient Experience Data in 2018 Drug Approvals.

Area of Science:

• Drug regulatory science
• Patient-centered research
• Health outcomes research

Background:

• Patient experience data (PED) systematically collects patient perspectives, needs, and priorities.
• The 21st Century Cures Act mandates FDA public statements on PED considered in drug approvals.
• This study assesses the FDA's reported consideration of PED in drug application reviews.

Purpose of the Study:

• To evaluate the extent of patient experience data (PED) use in FDA drug application reviews.
• To establish a baseline understanding of how the FDA incorporates patient perspectives into regulatory decision-making.
• To inform future drug development processes ensuring patient voices are heard.

Main Methods:

• Analysis of FDA documentation for 59 new molecular entities (NMEs) approved in 2018.
• Cross-referencing reported PED with FDA review information and product designations.
• Quantitative description and visualization of PED utilization in drug reviews.

Main Results:

• Patient experience data (PED) was reported in 70.8% of the 48 reviewed NME approvals.
• Patient-reported outcomes (PROs) were the most frequent source of PED, used in 60.4% of approvals.
• Details on PED use varied by FDA review division and regulatory designation.

Conclusions:

• This assessment provides an initial understanding of FDA's integration of patient experience data (PED).
• The findings establish a baseline for FDA's use of PED, aiding future policy development.
• Ensuring adequate consideration of patient experiences is crucial for future drug development and regulatory processes.