Introduction

This overview highlights some of the key themes and discussions from the FDA’s Patient-Focused Drug Development (PFDD) Workshop #2: Methodologic and Other Challenges Related to Patient Experience Data, held on September 18–19, 2025.

This overview highlights aspects that CLINIGMA views would be of interest to stakeholders in Clinical Development, Regulatory Affairs, Health Economics and Outcomes Research (HEOR), and Market Access, rather than offering a comprehensive summary of all discussions from the workshop.

This overview thus represents CLINIGMA’s perspective on the workshop proceedings based on our own notes and observations. It is not an official transcript. For the full context and detailed content, we encourage readers to view the complete two-day workshop recording, which is available on the FDA’s website.

Workshop Objective

The U.S. Food and Drug Administration (FDA) recently hosted a virtual public workshop to discuss methodological and other challenges in collecting and evaluating patient experience data (PED). This event took place on the 18 and 19 of September 2025 and included presentations and panel discussions with a diverse group of industry professionals, alongside FDA officials.

In addition, this workshop presented a draft version of an updated evidence dossier template to facilitate the submission of evidence to FDA to support Clinical Outcome Assessment (COA).

The FDA convened this workshop to advance its commitment to integrating patient perspectives into regulatory decision-making by improving the quality and utility of patient experience data across drug development. As Dr. Theresa Mullin, Associate Director for Strategic Initiatives at FDA’s Center for Drug Evaluation and Research (CDER), explained on Day 1, “[…] our ability to utilize that information in regulatory decision-making is very much tied to the quality of what is collected,” underscoring the need to address specific methodological issues identified by FDA staff and stakeholders. Building on public input received under the Prescription Drug User Fee Act (PDUFA VII) and discussions from prior workshops, this event tackled challenges that can limit PED’s impact on benefit–risk assessments, with a focus on “rigor, collaboration, and transparency” in method development and data interpretation, as noted on Day 1 by Director of Patient-Focused Drug Development at CDER, Robyn Bent. By clarifying methods and introducing tools such as the updated evidence dossier template, FDA aims to maximize the relevance and robustness of PED to better inform regulatory evaluations.

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Essential Takeaways for Stakeholders

This section summarizes four key insights gathered from the  workshop sessions. With contributions from both FDA speakers and industry professionals, the takeaways represent a combination of FDA perspectives and broader viewpoints shared during the discussions. Explicit statements attributed to the FDA reflect official positions, while other key points originate from the combined input of presenters and panelists throughout the workshop.

1.        Focus on early planning to ensure clinical trial endpoints reflect patient experiences  

Stakeholders are encouraged to engage with the FDA and patient communities early in the drug development process to collaboratively identify and prioritize meaningful aspects of health (MAH). Additionally, consideration should be given to patient baseline severity and variability in diverse populations.

This concept was primarily discussed during the first session of the workshop, led by the Division of Clinical Outcome Assessment at the FDA, and was further addressed on Day 2 during the discussion about the COA Evidence Dossier Template. The panel discussion following the Day 1 presentations also emphasized the importance of establishing a solid foundation for meaningful aspects of health. As Michelle Campbell, Associate Director for Stakeholder Engagement and Clinical Outcomes at the FDA, noted, “[…] we have that good foundational meaningful aspects of health work done first to be able to then help inform where we go to minimize any potential issues that we would see in the data when it comes to a severity level.”

Following this approach from an early stage of the trial may help ensure that endpoints and trial designs align with patient-centered outcomes, support regulatory expectations, and incorporate data that reflect patient priorities.

2.        Streamline submissions by using the new COA Evidence Dossier Template

The FDA encourages sponsors to adopt the Draft COA Evidence Dossier Template. As Vishal Bhatnagar, Associate Director for Patient Outcomes at the Oncology Center of Excellence at the FDA, stated, “I think it is important that we reiterate that this is not a requirement. However, the benefits of following this dossier template are that it consolidates and comprehensively organizes information, allowing for efficient review.”

To ensure successful implementation, consider the following points:

• The template consolidates essential knowledge into a structured and transparent format for easier application. The core concepts covered are familiar to industry experts.
•  It is adaptable across various product types, such as pharmaceuticals and medical devices.
• Not every section in the template requires completion; however, sponsors are encouraged to explain the rationale for any omitted sections, as this transparency supports an effective review.
• The dossier preparation process should involve collaboration across multiple departments to ensure comprehensive and coherent documentation.

The FDA emphasized the value of industry professionals and patient advocacy groups providing feedback on the COA Evidence Dossier Template. Feedback will contribute to ensuring that the template effectively addresses challenges in COA evidence submission. Dr. Naomi Knoble, Associate Director, Division of Clinical Outcome Assessment, Center for Drug Evaluation and Research at the FDA, noted:

“The dossier is a chance to show us your work, the very rich and patient-centered work you’ve done to make your thinking transparent and overt to help us follow your logic and ultimately help patients.”

3.        Integrate patients’ voices in MSRs and MSDs development

It is important to establish and apply Meaningful Score Regions (MSRs) to interpret clinical trial data effectively. As highlighted in the third session of Day 1 of the workshop, MSRs provide clarity on the clinical significance of COA scores, potentially aiding in regulatory decision-making. Monica Morell, Senior Statistician, Division of Biometrics III, Office of Biostatistics, Center for Drug Evaluation and Research, FDA, underscored that MSRs are “approximate points of reference that reflect regions of meaningfully distinct scores that are likely to vary from patient to patient.” This variability demonstrates the necessity of employing multiple methods to estimate a range of MSRs, rather than relying on a single metric.

To enhance the interpretative accuracy of MSRs and Meaningful Score Differences (MSDs), engaging patients during the development process may help gather additional contextual insights about their experiences. According to Morell, “the methods used should directly consider patient voice”, incorporating both quantitative methods like anchor-based approaches and qualitative methods such as cognitive interviews.

Using a multifaceted strategy can enhance the interpretation of observed treatment effects and help align outcomes with patients' experiences.

4.        Standardize data practices for improved reliability

Effective implementation of COAs and Patient-Reported Outcomes (PROs) in clinical trials relies on standardized data practices and collaborative efforts among stakeholders. This standardization can improve workflows, support regulatory submissions, and enhance the use of patient-centered data for decision-making.

As Laura Lee Johnson, Division Director, Division of Biometrics III, Office of Biostatistics, Center for Drug Evaluation and Research at the FDA, highlighted, the Technical Conformance Guide (TCG) and the accompanying FDA data standards resource page provide essential technical specifications for consistent submissions. These resources are intended to improve communication between sponsors and FDA reviewers, streamline the transfer of clinical and nonclinical research data, and address challenges like converting COA evidence into statistical code.

As Flora Mulkey, Lead Mathematical Statistician, Division of Biometrics V, Office of Biostatistics, Center for Drug Evaluation and Research at the FDA, notes, the variability in how PRO data is collected and submitted has created challenges for consistent analysis. To address these longstanding issues, Mulkey referred to the FDA's guidance issued in November 2023, which aims to promote standardization in PRO submission data for cancer clinical trials. Mulkey further notes, “As standards are adopted and data is consistently collected and collated to address these standards, creating and disseminating code to analyze this data becomes increasingly attractive, enabling more adaptable use across diverse.”

Effective statistical programming and planning are essential for regulatory submissions, impacting review timelines and decision-making. A consistent framework for planning and execution can ensure reproducibility and scientific reliability. Standardized data structures and clear source codes help FDA reviewers efficiently replicate analyses and resolve discrepancies that may arise during the review process. Ki Chung, Statistician, Division of Analytics and Informatics, Office of Biostatistics, Center for Drug Evaluation and Research at the FDA, stated, “Standardized data structures and clear source codes […] will significantly accelerate the review timelines, helping reviewers address potential discrepancies between submitted analyses and their replications.”

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Next steps

Although the workshop concluded on September 19, Robyn Bent (Director of PFDD at CDER) emphasized that “the work does not” end there, as the agency continues to advance efforts to strengthen the collection and use of patient experience data. The federal docket for public comment, which closed on November 18, provided an opportunity for stakeholders to submit input that will “help sharpen the tools and approaches we discuss, and inform FDA’s ongoing work”, as Bent noted.

A primary focus of this next phase is refining the draft Clinical Outcome Assessment (COA) evidence dossier template. While the template is not a requirement, it is intended to be useful to both sponsors and FDA reviewers to make submissions more efficient and to reduce unnecessary information requests. Feedback from the docket will guide decisions on the next steps for finalizing the template.

Alongside this effort, FDA is continuing to move the PFDD guidance series forward, with two guidances now finalized and the remaining drafts expected to be issued in final form in the coming months.

These actions reflect the workshop’s core themes: “rigor in our methods, collaboration across disciplines and sectors, [and] transparency in how evidence is developed and applied”, with the shared goal of ensuring patient experience data are “robust, meaningful, and capable of shaping both regulatory and clinical decision-making”, as articulated by Bent in her closing remarks.

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Concluding Remarks  

The FDA’s Patient-Focused Drug Development Workshop #2 provided a platform to discuss challenges and potential solutions for the collection, analysis, and application of patient experience data in clinical trials. Key discussions focused on incorporating patient voices, standardizing data practices, and leveraging tools like the COA Evidence Dossier Template to support clinical outcome assessments and regulatory submissions.

The workshop reinforced the value of collaboration among stakeholders—including regulators, industry professionals, and patient advocacy groups—in addressing these challenges. Continued efforts to explore and refine these strategies, along with stakeholder feedback, could further support the integration of patient experience data in drug development and regulatory decision-making processes.