NHC Comments on Public Meeting on Reauthorization of the Prescription Drug User Fee Act
August 14, 2025
Dockets Management Staff (HFA-305)
U.S. Food and Drug Administration
Division of Dockets Management
5630 Fishers Lane, Room 1061
Rockville, MD 20852
RE: Reauthorization of the Prescription Drug User Fee Act; Public Meeting; Request for Comments [FDA-2025-N-0816]
To Whom It May Concern:
The National Health Council (NHC) appreciates the opportunity to submit comments to the U.S. Food and Drug Administration (FDA) in response to its July 14, 2025, public meeting on the reauthorization of the Prescription Drug User Fee Act (PDUFA) for fiscal years 2028 through 2032 (PDUFA VIII).
Created by and for patient organizations over 100 years ago, the NHC brings diverse organizations together to forge consensus and drive patient-centered health policy. We promote increased access to affordable, high-value, equitable, and sustainable health care. Made up of more than 180 national health-related organizations and businesses, the NHC’s core membership includes the nation’s leading patient organizations. Other members include health-related associations and nonprofit organizations including the provider, research, and family caregiver communities; and businesses and organizations representing biopharmaceuticals, devices, diagnostics, generics, and payers.
Assessment of PDUFA VII Overall Performance
The NHC recognizes that PDUFA VII has built meaningfully on the commitments made under previous authorizations, particularly in the areas of patient-focused drug development (PFDD), regulatory science, rare disease innovation, and the use of novel evidence tools such as real-world evidence (RWE) and digital health technologies (DHTs). The continued institutionalization of PFDD activities and integration of patient experience data (PED) into regulatory decision-making is a significant achievement. We appreciate the FDA’s efforts to enhance internal reviewer training on PED, facilitate externally led PFDD meetings, and issue guidance supporting the development and submission of clinical outcome assessments (COAs).
PDUFA VII also advanced important pilot programs—such as the Rare Disease Endpoint Advancement (RDEA) initiative, the Chemistry, Manufacturing, and Controls (CMC) Development and Readiness Pilot, and the Split Real-Time Application Review (STAR) pilot—which we view as promising vehicles for generating data on more agile and patient-responsive development pathways. Further, the creation of new meeting types (Type D and INTERACT), the continued implementation of complex innovative trial design (CID) support, and expanded FDA guidance on DHTs have demonstrated responsiveness to the needs of both sponsors and patient communities.
Nonetheless, challenges remain. The incorporation of PED into labeling has been inconsistent, and the impact of PFDD activities on benefit-risk assessments is not always clear to external stakeholders.1 Moreover, resource limitations continue to affect the FDA’s ability to maintain adequate staffing and to fully execute its growing portfolio of patient-facing and science-driven initiatives. As user fee-funded activities now account for more than three-quarters of the agency’s drug review budget, ensuring adequate and predictable resources for PFDD and related programs is critical to fulfilling the public health mission of the FDA.
Features That Should Be Reduced or Discontinued
The NHC does not recommend discontinuation of any major initiatives launched under PDUFA VII. However, we encourage the FDA to review certain processes for potential refinement and improved efficiency.
First, as the landscape of patient engagement becomes more mature, we recommend consolidating duplicative guidance and harmonizing terminology related to PED, patient preference studies, and COA development. Stakeholders continue to report confusion around which types of data are acceptable for regulatory use and under what evidentiary thresholds.2 Streamlining these frameworks could improve clarity and reduce sponsor burden.
Second, as pilot programs launched under PDUFA VII are evaluated, we encourage the FDA to sunset those that do not demonstrate measurable benefit or scalability. The goal should not be proliferation of pilots, but rather transition of successful pilots into formal programs, with clear timelines and performance expectations.
Third, while communication between FDA review staff and sponsors has improved through expanded meeting types, stakeholders have reported inconsistent implementation of meeting formats across review divisions.3 The agency should consider whether certain meeting pathways can be made more predictable and whether internal review timelines can be optimized to reduce scheduling bottlenecks without compromising rigor or transparency.
New Features That Should Be Added in PDUFA VIII
The NHC strongly supports the inclusion of several new features in the PDUFA VIII performance goals to further embed patient-centeredness, strengthen scientific capacity, and enhance regulatory transparency.
We recommend that the FDA support the development of a Patient-Centered Core Outcome Set (PC-COS) program. This initiative should draw upon and be aligned with broader efforts to define patient-centered core impact sets (PC-CIS), such as those developed by the NHC.4 By identifying outcomes that are both measurable and meaningful to patients, this program would enhance consistency in data collection, facilitate meta-analyses, and improve the relevance of study endpoints to patients and caregivers. To maintain flexibility for sponsors, participation in such initiatives should remain entirely voluntary and serve as a resource to complement, rather than constrain, sponsor-led endpoint strategies.
In addition, we recommend adopting support for regulatory use of externally controlled trials, including through RWE, where appropriate and scientifically justified. This includes issuing clear methodological guidance, expanding internal review capacity, and supporting sponsor engagement earlier in the trial design process. When methodologically rigorous and appropriately validated, these tools can enhance trial feasibility in small populations and rare conditions while maintaining the scientific standards and predictability essential for regulatory decision-making.
To enhance transparency and foster public confidence, we recommend that the FDA explore the feasibility of publishing non-proprietary, aggregate-level metrics on the types and frequency of patient input submitted and how such input is used in regulatory decision-making, while continuing to protect proprietary data. The goal is to provide a high-level, system-wide view of patient engagement without imposing additional administrative burdens on sponsors.
Finally, we support further investment in FDA reviewer training and staffing related to PFDD, DHTs, and RWE, including expanding reviewer expertise in patient-centered methodologies that strengthen the scientific integration of patient-experience data, improve external validity and interpretability, and reinforce evidentiary standards that support timely and efficient review.
Potential Revisions to the Fee Structure
During the July 14 public meeting, FDA leadership raised the possibility of lowering user fees for smaller sponsors to reduce financial barriers to entry. The NHC acknowledges that the cost of drug development presents a substantial challenge, particularly for small and emerging drug sponsors, including academic spinouts and companies developing therapies for rare or ultra-rare conditions.5 While we support policies that promote a diverse and innovative sponsor base, we caution that any changes to the PDUFA fee structure must not undermine the FDA’s core capacity to engage with patients, review complex applications, and execute its performance goals. We appreciate the shared commitment of the FDA and industry to ensure that any structural changes maintain core regulatory functions and do not reduce the FDA’s scientific or patient-engagement capabilities.
If a revised fee structure is pursued, the NHC encourages the FDA and Congress to ensure that appropriated funds are available to maintain staffing levels in priority areas such as PFDD, rare disease review, RWE evaluation, and DHT analysis. The NHC also recommends that any fee exemptions or reductions be carefully structured to ensure a continued focus on meaningful engagement with affected patient communities and compliance with core regulatory expectations.
Conclusion
The PDUFA program has played a transformative role in enhancing the efficiency and effectiveness of human drug review, while also expanding the FDA’s ability to engage patients and promote regulatory science. As FDA prepares for PDUFA VIII negotiations with industry, the NHC urges the agency to prioritize the institutionalization of patient- focused development, the expansion of novel evidence tools, the establishment of a PC-COS program, and the safeguarding of review capacity. These efforts are vital to ensure that future therapies are developed, reviewed, and approved in ways that reflect both high scientific standards and the perspectives and priorities of the patients they are intended to serve. The NHC remains committed to advancing a regulatory system that is transparent, evidence-driven, and accountable to the public it serves.
We thank the FDA for its continued leadership and the opportunity to contribute to this important process. The NHC looks forward to participating in the stakeholder consultation meetings and remains committed to working collaboratively to advance a regulatory system that is scientifically rigorous, transparent, and responsive to patient needs. Please contact Kimberly Beer, Senior Vice President, Policy & External Affairs at kbeer@nhcouncil.org or Shion Chang, Senior Director, Policy & Regulatory Affairs at schang@nhcouncil.org, if you would like to discuss our recommendations in greater detail.
Sincerely,
Randall L. Rutta
Chief Executive Officer
1 Food and Drug Administration, “Methodological Challenges Related to Patient Experience Data; Request for Information and Comments,” Federal Register 88, no. 84 (May 2, 2023): https://www.federalregister.gov/documents/2023/05/02/2023-09265/methodological-challenges-related-to- patient-experience-data-request-for-information-and-comments.
2 Food and Drug Administration, LEADER 3D: Learning and Education to Advance and Empower Rare Disease Drug Developers (March 2024), 12, https://www.fda.gov/media/176557/download.
3 Food and Drug Administration, Independent Evaluation of FDA’s First Cycle Review Performance – Retrospective Analysis Final Report Text (August 17, 2015), https://www.fda.gov/industry/prescription- drug-user-fee-amendments/independent-evaluation-fdas-first-cycle-review-performance-retrospective- analysis-final-report-text.
4 National Health Council, A Blueprint for Developing Patient-Centered Core Impact Sets (PC-CIS) (2022), https://nationalhealthcouncil.org/a-blueprint-for-developing-patient-centered-core-impact-sets-pc- cis/.
5 Pharmaceutical Research and Manufacturers of America (PhRMA), “Research & Development Policy Framework,” January 31, 2025, https://www.phrma.org/policy-issues/research-development.
NHC Comments on Public Meeting on Reauthorization of the Prescription Drug User Fee Act
August 14, 2025
Dockets Management Staff (HFA-305)
U.S. Food and Drug Administration
Division of Dockets Management
5630 Fishers Lane, Room 1061
Rockville, MD 20852
RE: Reauthorization of the Prescription Drug User Fee Act; Public Meeting; Request for Comments [FDA-2025-N-0816]
To Whom It May Concern:
The National Health Council (NHC) appreciates the opportunity to submit comments to the U.S. Food and Drug Administration (FDA) in response to its July 14, 2025, public meeting on the reauthorization of the Prescription Drug User Fee Act (PDUFA) for fiscal years 2028 through 2032 (PDUFA VIII).
Created by and for patient organizations over 100 years ago, the NHC brings diverse organizations together to forge consensus and drive patient-centered health policy. We promote increased access to affordable, high-value, equitable, and sustainable health care. Made up of more than 180 national health-related organizations and businesses, the NHC’s core membership includes the nation’s leading patient organizations. Other members include health-related associations and nonprofit organizations including the provider, research, and family caregiver communities; and businesses and organizations representing biopharmaceuticals, devices, diagnostics, generics, and payers.
Assessment of PDUFA VII Overall Performance
The NHC recognizes that PDUFA VII has built meaningfully on the commitments made under previous authorizations, particularly in the areas of patient-focused drug development (PFDD), regulatory science, rare disease innovation, and the use of novel evidence tools such as real-world evidence (RWE) and digital health technologies (DHTs). The continued institutionalization of PFDD activities and integration of patient experience data (PED) into regulatory decision-making is a significant achievement. We appreciate the FDA’s efforts to enhance internal reviewer training on PED, facilitate externally led PFDD meetings, and issue guidance supporting the development and submission of clinical outcome assessments (COAs).
PDUFA VII also advanced important pilot programs—such as the Rare Disease Endpoint Advancement (RDEA) initiative, the Chemistry, Manufacturing, and Controls (CMC) Development and Readiness Pilot, and the Split Real-Time Application Review (STAR) pilot—which we view as promising vehicles for generating data on more agile and patient-responsive development pathways. Further, the creation of new meeting types (Type D and INTERACT), the continued implementation of complex innovative trial design (CID) support, and expanded FDA guidance on DHTs have demonstrated responsiveness to the needs of both sponsors and patient communities.
Nonetheless, challenges remain. The incorporation of PED into labeling has been inconsistent, and the impact of PFDD activities on benefit-risk assessments is not always clear to external stakeholders.1 Moreover, resource limitations continue to affect the FDA’s ability to maintain adequate staffing and to fully execute its growing portfolio of patient-facing and science-driven initiatives. As user fee-funded activities now account for more than three-quarters of the agency’s drug review budget, ensuring adequate and predictable resources for PFDD and related programs is critical to fulfilling the public health mission of the FDA.
Features That Should Be Reduced or Discontinued
The NHC does not recommend discontinuation of any major initiatives launched under PDUFA VII. However, we encourage the FDA to review certain processes for potential refinement and improved efficiency.
First, as the landscape of patient engagement becomes more mature, we recommend consolidating duplicative guidance and harmonizing terminology related to PED, patient preference studies, and COA development. Stakeholders continue to report confusion around which types of data are acceptable for regulatory use and under what evidentiary thresholds.2 Streamlining these frameworks could improve clarity and reduce sponsor burden.
Second, as pilot programs launched under PDUFA VII are evaluated, we encourage the FDA to sunset those that do not demonstrate measurable benefit or scalability. The goal should not be proliferation of pilots, but rather transition of successful pilots into formal programs, with clear timelines and performance expectations.
Third, while communication between FDA review staff and sponsors has improved through expanded meeting types, stakeholders have reported inconsistent implementation of meeting formats across review divisions.3 The agency should consider whether certain meeting pathways can be made more predictable and whether internal review timelines can be optimized to reduce scheduling bottlenecks without compromising rigor or transparency.
New Features That Should Be Added in PDUFA VIII
The NHC strongly supports the inclusion of several new features in the PDUFA VIII performance goals to further embed patient-centeredness, strengthen scientific capacity, and enhance regulatory transparency.
We recommend that the FDA support the development of a Patient-Centered Core Outcome Set (PC-COS) program. This initiative should draw upon and be aligned with broader efforts to define patient-centered core impact sets (PC-CIS), such as those developed by the NHC.4 By identifying outcomes that are both measurable and meaningful to patients, this program would enhance consistency in data collection, facilitate meta-analyses, and improve the relevance of study endpoints to patients and caregivers. To maintain flexibility for sponsors, participation in such initiatives should remain entirely voluntary and serve as a resource to complement, rather than constrain, sponsor-led endpoint strategies.
In addition, we recommend adopting support for regulatory use of externally controlled trials, including through RWE, where appropriate and scientifically justified. This includes issuing clear methodological guidance, expanding internal review capacity, and supporting sponsor engagement earlier in the trial design process. When methodologically rigorous and appropriately validated, these tools can enhance trial feasibility in small populations and rare conditions while maintaining the scientific standards and predictability essential for regulatory decision-making.
To enhance transparency and foster public confidence, we recommend that the FDA explore the feasibility of publishing non-proprietary, aggregate-level metrics on the types and frequency of patient input submitted and how such input is used in regulatory decision-making, while continuing to protect proprietary data. The goal is to provide a high-level, system-wide view of patient engagement without imposing additional administrative burdens on sponsors.
Finally, we support further investment in FDA reviewer training and staffing related to PFDD, DHTs, and RWE, including expanding reviewer expertise in patient-centered methodologies that strengthen the scientific integration of patient-experience data, improve external validity and interpretability, and reinforce evidentiary standards that support timely and efficient review.
Potential Revisions to the Fee Structure
During the July 14 public meeting, FDA leadership raised the possibility of lowering user fees for smaller sponsors to reduce financial barriers to entry. The NHC acknowledges that the cost of drug development presents a substantial challenge, particularly for small and emerging drug sponsors, including academic spinouts and companies developing therapies for rare or ultra-rare conditions.5 While we support policies that promote a diverse and innovative sponsor base, we caution that any changes to the PDUFA fee structure must not undermine the FDA’s core capacity to engage with patients, review complex applications, and execute its performance goals. We appreciate the shared commitment of the FDA and industry to ensure that any structural changes maintain core regulatory functions and do not reduce the FDA’s scientific or patient-engagement capabilities.
If a revised fee structure is pursued, the NHC encourages the FDA and Congress to ensure that appropriated funds are available to maintain staffing levels in priority areas such as PFDD, rare disease review, RWE evaluation, and DHT analysis. The NHC also recommends that any fee exemptions or reductions be carefully structured to ensure a continued focus on meaningful engagement with affected patient communities and compliance with core regulatory expectations.
Conclusion
The PDUFA program has played a transformative role in enhancing the efficiency and effectiveness of human drug review, while also expanding the FDA’s ability to engage patients and promote regulatory science. As FDA prepares for PDUFA VIII negotiations with industry, the NHC urges the agency to prioritize the institutionalization of patient- focused development, the expansion of novel evidence tools, the establishment of a PC-COS program, and the safeguarding of review capacity. These efforts are vital to ensure that future therapies are developed, reviewed, and approved in ways that reflect both high scientific standards and the perspectives and priorities of the patients they are intended to serve. The NHC remains committed to advancing a regulatory system that is transparent, evidence-driven, and accountable to the public it serves.
We thank the FDA for its continued leadership and the opportunity to contribute to this important process. The NHC looks forward to participating in the stakeholder consultation meetings and remains committed to working collaboratively to advance a regulatory system that is scientifically rigorous, transparent, and responsive to patient needs. Please contact Kimberly Beer, Senior Vice President, Policy & External Affairs at kbeer@nhcouncil.org or Shion Chang, Senior Director, Policy & Regulatory Affairs at schang@nhcouncil.org, if you would like to discuss our recommendations in greater detail.
Sincerely,
Randall L. Rutta
Chief Executive Officer
1 Food and Drug Administration, “Methodological Challenges Related to Patient Experience Data; Request for Information and Comments,” Federal Register 88, no. 84 (May 2, 2023): https://www.federalregister.gov/documents/2023/05/02/2023-09265/methodological-challenges-related-to- patient-experience-data-request-for-information-and-comments.
2 Food and Drug Administration, LEADER 3D: Learning and Education to Advance and Empower Rare Disease Drug Developers (March 2024), 12, https://www.fda.gov/media/176557/download.
3 Food and Drug Administration, Independent Evaluation of FDA’s First Cycle Review Performance – Retrospective Analysis Final Report Text (August 17, 2015), https://www.fda.gov/industry/prescription- drug-user-fee-amendments/independent-evaluation-fdas-first-cycle-review-performance-retrospective- analysis-final-report-text.
4 National Health Council, A Blueprint for Developing Patient-Centered Core Impact Sets (PC-CIS) (2022), https://nationalhealthcouncil.org/a-blueprint-for-developing-patient-centered-core-impact-sets-pc- cis/.
5 Pharmaceutical Research and Manufacturers of America (PhRMA), “Research & Development Policy Framework,” January 31, 2025, https://www.phrma.org/policy-issues/research-development.