From Engagement to Evidence: Building a Holistic Measure of Patient-Focused Drug Development

Patient-Focused Drug Development (PFDD) has undeniably advanced how regulators and sponsors listen to patients—from FDA-led PFDD meetings to externally led sessions that surface lived experience and unmet needs. The FDA’s series of PFDD methodological guidance, including those on collecting representative input and developing fit-for-purpose clinical outcome assessments, provide structured frameworks for stakeholders to gather and submit patient experience data across development stages (FDA PFDD Guidance Series).

Advancing the Patient Voice

Patient-Focused Drug Development (PFDD) has made important strides in ensuring patient perspectives shape regulatory science—from FDA-led PFDD meetings to externally led sessions that surface lived experience and unmet needs. The FDA’s guidance series provides structured methods for collecting patient input and developing fit-for-purpose clinical outcome assessments (FDA PFDD Guidance Series).

What We Know From Published Evidence

• Analyses of PFDD data within the esketamine NDA demonstrate that functional and preference data informed benefit-risk assessments (Therapeutic Innovation & Regulatory Science).

• Scholarly reviews highlight opportunities to enhance patient-centric measurements in trial design and regulatory science (PubMed review on PFDD).There is emerging published evidence showing patient experience data can be integrated into regulatory decision-making. For example, analyses of PFDD data within the esketamine NDA illustrate how functional and preference data informed benefit-risk assessments (Esketamine PFDD Analysis, Therapeutic Innovation & Regulatory Science). Broader scholarly reviews highlight opportunities to enhance patient-centric measurements in trial design and regulatory science (PubMed review on PFDD).

Yet despite these advances, the landscape remains piecemeal. The peer-reviewed literature is sparse, most datasets are scattered across FDA Voice of the Patient reports rather than harmonized in research-ready form, and there is little systematic evidence linking PFDD engagement to outcomes like trial advancement, IND submissions, or commercialization success. Patient organizations invest thousands of dollars to convene these meetings, but we lack a holistic picture of impact across disease areas.

The Gaps Remain Significant

Compounding the gap, drug developers rarely publish failures or negative endpoints, leaving critical lessons invisible. We cannot currently verify whether patient-centered outcomes meaningfully influenced trial design, endpoint selection, or benefit-risk decisions. Were outcomes measured only at baseline and study completion, or used actively to guide protocol adjustments? How much weight did patient input carry when endpoints failed? Without transparency, patient-centeredness risks becoming symbolic rather than substantive.

• Peer-reviewed literature is sparse and mostly disease-specific.

• Most datasets are scattered across Voice of the Patient reports rather than harmonized for analysis.

• Drug developers rarely publish negative endpoints or explain why trials fail, leaving critical lessons invisible.

• We cannot currently verify whether patient-centered outcomes influence trial design, endpoint selection, or regulatory decisions.

We do have data sources that can form the foundation for measurement:

• FDA Condition-Specific Meeting Reports – transcripts, Voice of the Patient reports, slides, and webcasts from FDA-led and externally-led meetings (FDA PFDD Meeting Reports)

• Published PFDD case studies – e.g., esketamine analyses in peer-reviewed journals (Therapeutic Innovation & Regulatory Science)

• Systematic academic reviews – e.g., Frontiers in Medicine overview of PFDD methods and outcomes (Frontiers in Medicine)

• ClinicalTrials.gov – public trial registry for comparing development timelines and endpoints across programs (ClinicalTrials.gov)

These sources provide a starting point—but they are not comprehensive. To elevate PFDD from dialogue to measurable influence, we need standardized, interoperable reporting linking PFDD inputs to trial designs, regulatory outcomes, and commercialization milestones. Regulators could require structured post-hoc analyses documenting which patient-centered outcomes were included, how they were measured, their weighting in benefit-risk decisions, and explanations for failed endpoints. Incentives such as priority review credits, extended exclusivity, or public recognition could encourage sponsors to publish negative or null findings in peer-reviewed or FDA-hosted repositories.

A Framework to Measure Impact

We can conceptualize PFDD measurement as a three-tier framework:

PFDD Inputs

• FDA-led and externally led meetings

• Voice of the Patient reports, surveys, COAs, risk-tolerance frameworks

Trial & Regulatory Integration

• Protocol design incorporating patient-prioritized endpoints

• IND/NDA submissions

• Endpoint selection informed by patient input

Measurable Outcomes & Gaps

• Adoption Metrics: % of trials using patient-centered endpoints

• Timeline Metrics: Time to IND, trial modifications, commercialization

• Transparency Metrics: Published failures, endpoint rationale

• Existing resources populate “available data”: FDA reports, published case studies, systematic reviews, ClinicalTrials.gov

• Missing negative/null results and untracked patient input are “critical gaps”

If patient-centered outcomes are to be truly foundational, we must move beyond statements of intent. Measuring when patient input is used, how much it influences decisions, and why it fails is essential. Only with evidence, transparency, and systematic measurement can PFDD fulfill its promise of shaping drug development in ways that truly serve patients.