Beyond Numbers: Uses and Value of In-Trial Interview Data for Regulatory and Health Technology Assessment (HTA)...

Area of Science:

• Health Services Research
• Regulatory Science
• Patient-Reported Outcomes

Background:

• The Food and Drug Administration (FDA) and European Medicines Agency (EMA) now emphasize patient experience data in drug approval.
• Qualitative in-trial research (ITR) provides valuable patient insights for decision-making.

Purpose of the Study:

• To evaluate the extent and impact of qualitative in-trial research (ITR) in regulatory submissions and health technology assessment (HTA) reviews.
• To identify challenges and opportunities for integrating patient experience data into drug development.

Main Methods:

• Systematic review of regulatory guidance documents, drug approval records (FDA/EMA), and HTA reports from January 2017 to March 2025.
• Analysis included new drug applications, specific guidance series (e.g., rare diseases, oncology), and HTA methodological guidance from multiple countries.

Main Results:

• Only 10 FDA and 8 EMA approved products incorporated ITR, with 55% for rare diseases.
• ITR data informed insights into symptoms, patient experiences, and meaningful treatment benefits.
• Few HTA bodies have guidance on qualitative research, and its inclusion in reports is minimal.

Conclusions:

• Despite growing recognition, the use of ITR in regulatory and HTA processes is limited.
• Early strategic planning and alignment with regulatory/HTA requirements are crucial for maximizing the value of qualitative evidence.