What if regulatory decisions reflected not just clinical endpoints, but what patients truly value? What if benefit–risk assessments explicitly accounted for patients’ willingness to accept uncertainty, risk, or trade-offs? The FDA’s adoption of ICH E22 signals its perspective on using patient preferences for regulatory decisions by formalizing how structured patient preference data can be designed, analyzed, and submitted to inform regulatory decision-making across the product lifecycle—from early development through post-marketing evaluation
Patient Preference Studies are designed to systematically assess how patients value different attributes of a treatment, such as efficacy, safety risks, mode of administration, and uncertainty. Importantly, PPS do not replace clinical efficacy or safety data, but rather complement them by providing context for benefit–risk assessment. The guidance emphasizes stated-preference methods, including qualitative, quantitative, and mixed-methods approaches, allowing sponsors to capture not only what matters to patients—but how much it matters.
Several principles stand out:
- Early integration is critical – PPS should be considered as early as possible to inform endpoint selection, protocol design, and recruitment strategies.
- Scientific rigor is non-negotiable – PPS must follow good study design principles, including pre-specified objectives, protocols, analysis plans, and quality checks.
- Patient input shapes the study itself – Patients should be involved in selecting attributes, defining meaningful trade-offs, and contextualizing results.
- Global applicability with justification – PPS conducted in one region may support decisions in another, provided cultural and healthcare differences are addressed.
- Regulatory-ready reporting – PPS belong in CTD Modules 2 and 5, with structured reporting aligned to ICH E3 and M4E expectations.
ICH E22 does not reinvent FDA thinking, it formalizes it. The guidance aligns closely with:
- ICH E8(R1) on quality by design, reinforcing risk-based planning and focus on critical quality factors.
- FDA’s Patient-Focused Drug Development (PFDD) initiatives, which have long encouraged incorporating patient experience data into regulatory submissions.
- ICH M4E(R2) benefit–risk frameworks, where PPS can contextualize clinical outcomes and inform regulatory judgment.
- FDA’s openness to novel evidence, including real-world data and patient-reported insights, when scientifically justified.
In short, E22 operationalizes FDA’s long-standing message: patient perspectives are not anecdotal, they are data.
For drug developers, this guidance raises expectations. PPS must now be methodologically sound, strategically timed, and submission-ready. When executed well, they can de-risk development decisions, strengthen benefit–risk narratives, and support regulatory flexibility—particularly in areas of unmet need or high uncertainty. ICH E22 marks a decisive shift from listening to patients toward systematically measuring their preferences. For sponsors, the message is clear: patient preference data are becoming a credible, reviewable, and influential component of modern regulatory science.
