NHC Comments on PDUFA VII Public Stakeholder Meeting


By Maddie Mason, Senior Associate, Policy 

The National Health Council (NHC) submitted comments last week to the Food and Drug Administration (FDA) on their July 23 Public Stakeholder Meeting on the Prescription Drug User Fee Act (PDUFA) VII where NHC Chief Executive Officer Marc Boutin presented. In the past, the NHC has supported a multitude of provisions to bring the patient perspective into drug development and modernize the development and regulation of treatments for people with chronic diseases and disabilities, and we look forward to continue our work with the FDA on the development of PDUFA VII.  

The NHC recommended the FDA address the following in PDUFA VII: 

Expansion and enhancement of core outcome set program to increase patient-centricity and impact 

The FDA announced a small pilot program to fund external organizations to develop publicly available core outcome assessments (COAs), which are agreed upon, standardized sets of outcomes that should be gathered and reported at a minimum in research of specific areas of health or health care. This work should be expanded through greater funding with a goal of creating a systemic approach beyond pilot phase, with a focus on a greater level of patient involvement and an expansion beyond COAs only to include all concepts important to patients.  

The NHC proposes the concept of Patient-Centered Core Outcome Sets to ensure greater patient centricity and expanded utility to go beyond COA measures. This process would encourage patient and stakeholder engagement to identify a broad set of concepts that matter the most to patients in specific disease states.  

Continued examination of the potential regulatory uses for real-world evidence  

PDUFA VI implemented the use of realworld evidence (RWE), and the FDA has since funded pilot programs on the usage of RWE and realworld data (RWD) to support clinical development. With science continually evolving, the learnings from these pilots can inform additional work and potentially more formalized pilots under PDUFA VII. We believe that the expansion of the pilot program should require or strongly encourage patient engagement and include more transparent reporting requirements to ensure that the lessons learned can be utilized by all stakeholders.   

Decreasing burden through digital clinical trials 

It has been a long and shared goal of the NHC, FDA, and industry to implement ways to reduce the burden of clinical trials and promote clinical trial diversity. The NHC urges the FDA to include funding in PDUFA VII for pilot programs designed to help better understand how patient engagement can improve the use of mobile technology to create clinical-trial efficiency, reduce participant burden, and improve patient centricity.  

Embedding patient-experience data throughout the knowledge management system  

Currently, the FDA is developing a knowledge management system and portal to provide FDA staff access to and analysis of prior regulatory decisions. The FDA also developed a template that reviewers can use to denote what patient experience data was submitted by sponsors. We would like to see these two efforts incorporated together by implementing the knowledge management system in a way that ensures patient-experience data and other emerging data is systemically captured for use in future decisions.  

Evolving the science of patient engagement through increased FDA staff capacity  

The provisions in that were included in PDUFA VI to provide additional funding to hire new FDA staff should be continued and improved. We would like to see a greater investment in staff in both numbers and skills to evaluate sponsors’ patient-engagement plans, hold earlier and more frequent meetings to discuss the plans with industry, and ensure sponsors collect the right patient-experience data to meet the FDA’s expectations.