HAVE PFDD MEETINGS MEANINGFULLY INFORMED NDA AND BLA REVIEWS BY THE CENTER FOR DRUG EVALUATION AND RESEARCH? FINDINGS FROM ANALYSIS OF PRODUCTS APPROVED FROM JUNE 2017 TO DECEMBER 2025
Author(s)
Ilse Peterson, MPH1, Timothy Franson, MD2, Holly Peay, MS, PhD3, Maya Gerstein, DrPH4, Anne Markus, JD, PhD, MHS5;
1George Washington University Milken Institute School of Public Health, Washington, DC, USA, 2Faegre Drinker Consulting, Indianapolis, IN, USA, 3Faegre Drinker Consulting, Washington, DC, USA, 4Vector Psychometric Group, Chapel Hill, NC, USA, 5The George Washington University Milken Institute School of Public Health, Washington, DC, USA
1George Washington University Milken Institute School of Public Health, Washington, DC, USA, 2Faegre Drinker Consulting, Indianapolis, IN, USA, 3Faegre Drinker Consulting, Washington, DC, USA, 4Vector Psychometric Group, Chapel Hill, NC, USA, 5The George Washington University Milken Institute School of Public Health, Washington, DC, USA
OBJECTIVES: FDA’s Patient-Focused Drug Development (PFDD) program aims to obtain patient perspectives that may inform FDA’s decisions and oversight during drug development and marketing application review. This research examines whether PFDD meeting reports have meaningfully informed review of CDER-approved new molecular entities (NMEs) since reporting on PED use in NDA and BLA reviews began in 2017, including if reports have been considered in benefit-risk assessments and whether meetings have been more frequently cited in reviews of orphan-indicated products than reviews of non-orphan indicated products.
METHODS: A dataset of CDER NME approvals from 6/17/17-12/31/25 with information on approved product uses, designations, PED reporting, PFDD meeting citations in benefit-risk assessments was generated using publicly available datasets and review documentation. Products were matched with PFDD meetings conducted before approval dates, and Stata was used to tabulate approvals matched with a PFDD meeting, containing PED tables, and reflecting meeting reports in benefit-risk assessments. A one-sided Fisher’s exact test was used to test whether meeting reports were more likely to be cited for orphan products, as hypothesized.
RESULTS: 126 NMEs approved by CDER between 6/17/17-12/31/25 were matched with a PFDD meeting held before the approval. Of these, 124 had available review documentation and 118 reviews included PED sections. 35 of the 118 (30%) cited PFDD meeting reports in PED sections and seven indicated in that section that the meeting informed benefit-risk assessment (20% of 35 and 6% of 118). 25 (36%) of 69 approvals for orphan-designated products cited a meeting report, while 10 (20%) of 49 non-orphan designated approvals cited meeting reports (p=0.048).
CONCLUSIONS: These data suggest significant missed opportunities for PFDD meeting reports to inform product review. This raises questions about why more PFDD meetings aren’t cited in reviews and if changes are needed to make PFDD meetings and reports more informative for regulators.
METHODS: A dataset of CDER NME approvals from 6/17/17-12/31/25 with information on approved product uses, designations, PED reporting, PFDD meeting citations in benefit-risk assessments was generated using publicly available datasets and review documentation. Products were matched with PFDD meetings conducted before approval dates, and Stata was used to tabulate approvals matched with a PFDD meeting, containing PED tables, and reflecting meeting reports in benefit-risk assessments. A one-sided Fisher’s exact test was used to test whether meeting reports were more likely to be cited for orphan products, as hypothesized.
RESULTS: 126 NMEs approved by CDER between 6/17/17-12/31/25 were matched with a PFDD meeting held before the approval. Of these, 124 had available review documentation and 118 reviews included PED sections. 35 of the 118 (30%) cited PFDD meeting reports in PED sections and seven indicated in that section that the meeting informed benefit-risk assessment (20% of 35 and 6% of 118). 25 (36%) of 69 approvals for orphan-designated products cited a meeting report, while 10 (20%) of 49 non-orphan designated approvals cited meeting reports (p=0.048).
CONCLUSIONS: These data suggest significant missed opportunities for PFDD meeting reports to inform product review. This raises questions about why more PFDD meetings aren’t cited in reviews and if changes are needed to make PFDD meetings and reports more informative for regulators.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
HPR59
Topic
Health Policy & Regulatory
Topic Subcategory
Approval & Labeling
Disease
No Additional Disease & Conditions/Specialized Treatment Areas