Alexandria, VA – July 11, 2023 – The Innovation and Value Initiative (IVI), a non-profit research organization committed to advancing the science, practice, and use of health technology assessment (HTA) in healthcare, submitted comments on the U.S. Food and Drug Administration’s (FDA) Patient-Focused Drug Development: Incorporating Clinical Outcome Assessments into Endpoints for Regulatory Decision-Making Guidance for Industry, Food and Drug Administration and Other Stakeholders.

IVI applauds FDA’s commitment to patient-focused drug development and encourages the FDA to build on this commitment to ensure that patient-centered priorities are a driving consideration in the development of new healthcare technologies. The Patient-Focused Drug Development (PFDD) program is a key step in shifting the overall approach in clinical research.

“Our belief is that value must be seen through the lens of the patient, and it is increasingly clear that estimating patient-centered value and paying for it in the marketplace must prioritize therapies that achieve endpoints important to patients and consequently generates the evidence needed to evaluate them,” states Jason Spangler, MD, MPH, FACPM, Chief Executive Officer at IVI.

IVI submitted the following comments for consideration, among others:

  • PFDD Guidance Should be Based on Principles of Patient-Centricity and Health Equity. Of the principles that guide IVI’s work, two of the most foundational ones are patient-centricity and health equity. We believe that all PFDD guidance and regulatory practice at the FDA should reflect these principles in both theory and practice.
  • PFDD Methods Must Balance Individual Experience Against Generalizability. The current draft Guidance 4, as well as previous PFDD Guidance, reflects the fundamental tension between the individualized experiences of patients—both individually and as groups—and the need for consistent and generalizable measures for use in development and appraisal of therapies.
  • Continue to Advance PFDD. The FDA and policymakers at large should consider establishing a more meaningful role for PFDD guidance in regulatory processes.

Read IVI’s full comments here.


About IVI
The Innovation and Value Initiative (IVI) is a 501(c)(3) tax-exempt, non-profit research organization dedicated to advancing the science and improving the practice of health technology assessment through development of novel methods and the creation and application of enhanced health technology assessment models to support local decision-making needs in healthcare.

Contact: Smita Sanwardeker
Phone: 267.909.7661