FDA and ERG Release First Public Assessment of New Drug Review Utilizing Patient Experience Data
First Statutory Assessment in 2021
The U.S. Food and Drug Administration (FDA), in accordance with Section 3004 of the 21st Century Cures Act, commissioned the Eastern Research Group, Inc. to conduct an assessment of the utilization of Patient Experience Data (PED). The report was published on June 18, 2021. The analysis covered 1,169 New Drug Applications (NDAs), Biologics License Applications (BLAs), and efficacy addendum applications received from June 12, 2017, to June 12, 2020, and approved by February 5, 2021. This is not the second assessment scheduled for 2026. The FDA is mandated to conduct assessments in 2021, 2026, and 2031; therefore, this document should be considered the first report establishing a baseline for Patient-Focused Drug Development (PFDD).
Key Figures: 68% and 66%
Among the 176 New Molecular Entity (NME) NDAs and BLAs, 68% of FDA review documents mentioned PED. For priority reviews (112 cases), this figure was 70%, and for orphan drug designations (87 cases), it was 66%. The proportion of applications that included PED submitted by the applicant was 66% overall, 66% for priority reviews, and 62% for orphan drugs. These figures differ from the overall rate of 65% and the orphan drug rate of 91.5% reported in the study. While 82% of NME reviews included a table of patient experience data, this does not represent the proportion in which the data influenced the approval decision, but rather a procedural indicator of submission and review.
Changes in Clinical Development Strategy
In FDA reviews, PED is primarily used in the form of Patient-Reported Outcomes (PROs) and Clinical Outcome Assessments (COAs). It contributes most directly to benefit-risk assessments when used as a primary or co-primary endpoint in studies of symptomatic diseases. Conversely, in studies where laboratory values are the primary endpoint, PED is used as supporting evidence to explain tolerability, function, quality of life, and patient priorities. Therefore, companies should proactively design for fit-for-purpose, manage missing data, establish statistical analysis plans, and ensure patient population representativeness across all phases (Phase 1, 2, and 3) of clinical trials and coordinate early with the FDA.
Implications for Regulation and Market Competition
This assessment is a regulatory evaluation of the entire product portfolio and does not address specific drugs, target molecules, indications, or approval dates. Therefore, comparisons of individual brands, generics, or competing therapies, as well as pricing terms, are not applicable. Competition arises from the pharmaceutical companies' ability to develop COAs and the quality of their electronic PRO (ePRO) and electronic Clinical Outcome Assessment (eCOA) data, rather than from comparisons of drug efficacy. The FDA plans to finalize its guidance on the selection and development of fit-for-purpose COAs by October 2025 and its guidance on Patient-Preferred Information (PPI) across the product lifecycle by March 2026.
Given that global new drug development spending exceeds $200 billion annually, PED should be viewed as an underlying infrastructure that influences the risk of clinical failure and the quality of regulatory submissions, rather than as a separate market for therapies.
From an investor's perspective, the key figures are 68% PED mention in 176 NME NDAs/BLAs and 66% in 87 orphan drugs. Using the previously reported figures of 65% and 91.5% in investment assumptions would lead to an overestimation of regulatory utilization. Researchers should establish fit-for-purpose COAs and analysis plans in Phase 1 and 2 trials to ensure that the data can be used in Phase 3 endpoints and for regulatory labeling, as PED has the greatest impact on approval decisions when used as a primary endpoint. The industry will see short-term demand for ePRO/eCOA specialist services and capabilities in validating patient representativeness. In the global new drug development market, which exceeds $200 billion annually, data quality will be a key differentiator. In the medium to long term, the FDA's 2025 COA guidance and 2026 PPI guidance will promote standardization among developers and contract research organizations (CROs), but they will not directly guarantee the approval or sales of specific drugs.
Source: FDA Drug Approvals (rss)