The FDA’s recently released Assessment of the Use of Patient Experience Data in Regulatory Decision-Making offers encouraging evidence that patient experience data (PED) has become an increasingly visible part of drug development and regulatory review. The report found that mentions of PED in reviews of original new molecular entity applications increased over the assessment period of 2021–2025, and approximately 40% of approved product labels associated with these reviews referenced PED. FDA reviewers also described using PED throughout product development, from endpoint selection and trial design through benefit-risk assessment and labeling discussions.

PED is gaining regulatory visibility

These findings reflect the significant progress made since patient-focused drug development (PFDD) became a formal regulatory priority. Sponsors are generating more patient-centered evidence than ever before, and regulators are increasingly incorporating patient perspectives into decision-making.

The next challenge: making PED review-ready

To me, one of the most interesting messages in FDA’s report is not that more PED is being submitted. Rather, it is the suggestion that the next challenge may be ensuring that the right experts can find, evaluate, interpret, and apply that evidence effectively.

In many ways, PED is becoming a victim of its own success.

As the science has evolved, so too has the variety of evidence available to sponsors. Today’s development programs may incorporate qualitative research, natural history studies, patient preference studies, clinical outcome assessments (COAs), psychometric analyses, digital health technologies, and other patient-centered evidence generation activities. FDA reviewers acknowledged that PED now appears across multiple sections of a submission and often serves different purposes throughout development and review.

This growing complexity creates a new challenge. Generating meaningful PED is no longer enough. Sponsors must also ensure that the evidence is organized, contextualized, and positioned in a way that supports efficient regulatory review.

Notably, FDA reviewers reported that applications can contain thousands of pages of documentation and that sponsors do not always clearly identify what patient experience data has been submitted, where it is located, or how it is intended to inform the review. Reviewers noted that relevant information can be inadvertently overlooked when this context is not provided. The FDA has encouraged sponsors to use the Reviewer’s Guide to describe submitted PED and its intended purpose, but reviewers indicated that such information is not consistently included in applications today.

This observation may seem administrative at first glance, but it has important strategic implications.

Consider a development program that includes qualitative research to identify concepts important to patients, a COA selected to measure those concepts in a clinical trial, and additional evidence intended to help contextualize treatment benefit. If these pieces of evidence are submitted without a clear explanation of how they connect to one another and how they are intended to inform regulatory decision-making, reviewers may be left to assemble the story themselves. By contrast, a well-structured Reviewer’s Guide can serve as a roadmap, helping reviewers quickly understand where the evidence resides, why it was generated, and how each component contributes to the overall evidence package. The result is a more reviewable submission.

From evidence generation to evidence integration

This highlights an important evolution in regulatory strategy. Historically, discussions about PFDD have focused primarily on evidence generation. Increasingly, the focus must also include evidence integration. PED should not be viewed as a standalone workstream. It intersects with regulatory affairs, clinical development, biostatistics, psychometrics, qualitative research, patient engagement, and medical writing. Different types of evidence may support different regulatory objectives. A concept elicitation study may inform endpoint development. A patient preference study may help contextualize benefit-risk considerations. A COA may support a key secondary endpoint or labeling objective. Each evidence stream requires specialized expertise and must ultimately contribute to a coherent regulatory narrative.

FDA’s assessment report reinforces this reality. Reviewers described PED review as a multidisciplinary effort involving clinical reviewers, statisticians, and subject matter experts in qualitative and quantitative methods, including psychometricians. The report also notes that, as the range of PED continues to expand, there is not always complete clarity regarding which review disciplines should evaluate specific types of evidence.

What sponsors should do next

For sponsors, this presents an opportunity.

The organizations most likely to realize the regulatory value of PED will not simply be those that collect more of it. They will be the organizations that plan patient-centered evidence strategically, align it with specific regulatory objectives, engage FDA early, and present the evidence in a way that allows reviewers to understand both its purpose and significance.

In other words, the next step in PFDD will be ensuring that patient experience data is review-ready.

As patient-centered evidence continues to mature, sponsors should be asking three fundamental questions:

• Is the evidence scientifically robust?
• Is it fit-for-purpose for the decision it is intended to support?
• Is it organized and communicated in a way that makes its regulatory value immediately clear?

FDA’s 2026 assessment suggests that progress is being made. The next challenge is ensuring that when the patient voice reaches FDA, reviewers can easily locate, interpret, and apply it within the complex multidisciplinary process that ultimately shapes regulatory decision-making.

IQVIA Patient Centered Solutions helps sponsors generate, integrate, and communicate patient-centered evidence in ways that support regulatory decision-making. From COA strategy and qualitative research to psychometrics, patient preference, and regulatory positioning, our experts help make patient experience data more actionable and review-ready. Learn more about our Patient Centered Solutions expertise or contact us to learn how we can support your PED strategy.