Robust Use of Patient Experience Data to Inform Regulatory Decision-Making, Labeling
Patient experience data (PED) has long been recognized as a valuable input in drug development, yet across the medical product lifecycle, it remains inconsistently collected, incompletely analyzed, and underutilized in the decisions it is best positioned to inform. Despite growing regulatory attention and a wealth of available guidance, fundamental questions about evidentiary standards, stakeholder accountability, and downstream use continue to limit PED's impact.
On May 6-7, 2026, the DCRI Think Tanks program brought together regulators, sponsors, methodologists, payers, academics, and patient advocacy representatives to examine how PED can be generated, interpreted, and applied more effectively across the full arc of medical product development.
The Case for Centering the Patient
Discussions opened with a foundational premise: PED captures something that no third-party measure can fully replicate. How patients feel, how they function, and how they weigh the trade-offs of treatment are not reliably surfaced through clinician assessment alone. Patient-reported outcomes (PROs) can reveal symptoms that providers consistently underreport. Patient preference information (PPI) instruments, developed and validated through rigorous processes, can quantify how patients weigh benefit-risk trade-offs in ways that inform regulatory and coverage decisions alike.
Yet despite this value, attendees noted that PED is often treated as supplementary rather than central. It is often added late in development, designed without a clear intended use, and interpreted through frameworks built for different types of evidence. The result is data that is technically collected but rarely decision-ready.
From "Regulatory-Grade" to "Decision-Grade"
A recurring theme during the think tank was the need to reframe how the field considers evidentiary standards. Regulatory approval, attendees emphasized, is only one of several consequential decision points a medical product must navigate. Payer coverage, health technology assessment (HTA) determinations, and real-world adoption each carry their own evidentiary expectations—and those expectations often differ across jurisdictions.
Planning PED around a single regulatory submission, the group argued, is both strategically shortsighted and practically wasteful. A product that clears regulatory review but fails to meet the evidence bar for payer coverage or HTA has not fully succeeded. The group proposed retiring the term "regulatory-grade evidence" in favor of "decision-grade evidence." This framing better reflects the full landscape of decisions PED must support, from first-in-human studies through post-market monitoring.
Greater harmonization across regulatory agencies, HTA bodies, and payers was seen as essential to making this shift viable. Without aligned expectations, sponsors face duplicative and sometimes conflicting evidence requirements that increase cost, slow development, and reduce the overall quality of PED generated.
"We've been using 'regulatory-grade' as shorthand for rigor, but that framing sells short what this data actually needs to do," said Emily O'Brien, PhD, DCRI faculty member and co-director of the Think Tank. "A product that clears FDA review but can't survive a payer coverage decision hasn't fully succeeded, and neither has the evidence behind it."
Planning Early, Planning Intentionally
Much of the discussion centered on a straightforward but frequently violated principle: PED works best when it is planned from the start. Integrating patient experience considerations into the clinical development plan early allows sponsors to align methods to decisions, engage regulators before misalignments become costly, and build a coherent patient narrative that carries through the product lifecycle.
Attendees were clear that no single method is sufficient. Mixed-methods approaches combining qualitative research, PROs, and PPI are often necessary to fully capture the impact of disease and treatment. Qualitative work can surface the concepts that matter most to patients; PROs can track those concepts longitudinally; and PPI can quantify how patients weigh them against each other and against clinical endpoints.
Early and iterative engagement with regulators was identified as particularly important. Multidisciplinary input at the planning stage reduces the risk of misaligned evidence expectations and increases the likelihood that collected data will be usable at the decision points it is meant to inform.
The Burden of Measurement
Careful attention to patient burden emerged as a practical imperative. Trials that stack multiple PRO instruments, administer them at high frequency, or sequence them without regard for respondent fatigue risk degrading the very data they are designed to collect. Disengaged or burdened patients produce lower-quality responses, and low-quality PED, however rigorously analyzed, undermines the evidentiary case it is meant to support.
Attendees also flagged a tension between innovation and familiarity in instrument selection. Legacy endpoints and validated instruments carry the advantage of familiarity and regulatory precedent, but they are not always the most appropriate or sensitive measures for a given therapeutic area or patient population. Where existing instruments lack validity for the intended use, the group agreed they should be adapted or developed de novo—even when that process is more demanding.
Accountability and the Data Sharing Gap
Despite the growing emphasis on PED in regulatory guidance, attendees identified a persistent gap between stated importance and actual practice. Stakeholders across the development ecosystem broadly agree that PED matters, yet accountability for ensuring it is robustly collected and used remains diffuse.
Part of the problem is structural. Consent forms, data ownership provisions, and publication rights frequently constrain the dissemination of PED findings. Negative trial results—which carry substantial scientific value for the field—are particularly unlikely to be published, leaving the broader community without information that could inform future study designs, instrument selection, and evidence expectations.
Attendees called for proactive consent language, data use agreements structured to facilitate rather than restrict sharing, and norms that treat publication of PED findings as a professional and scientific obligation.
Next Steps
The group coalesced around several concrete priorities.
Terminology should shift: adopting "decision-grade evidence" in place of "regulatory-grade" across regulatory communications, guidance documents, and industry practice signals a genuine reorientation toward the full landscape of decisions PED must serve.
Standardization models that work should be translated to other therapeutic areas through practical toolkits with disease-specific guidance. These efforts should be accompanied by broader publication of PRO, PPI, and qualitative findings from both positive and negative trials, supported by data sharing infrastructure such as ClinicalTrials.gov.
Policy and incentive structures need to catch up with stated priorities. Requiring or rewarding robust PED collection during pre- and post-market phases, including after treatment discontinuation, would move accountability from aspiration to expectation. Concrete examples of how PED has shaped regulatory and coverage decisions should be documented and shared to build the case for investment.
Existing guidance should be operationalized into lifecycle planning tools that extend beyond approval. These include practical resources that sponsors can use to align PED strategy with the full arc of evidence needs, from early development through post-market use.
Finally, a working group of international regulators, HTA bodies, and payers should be convened to develop shared evidentiary expectations for PED.
"The goal isn't just better data, it's better decisions for patients," O'Brien said. "That requires the whole ecosystem to treat PED as a shared obligation, not a box to check."