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What PFDD guidance can teach us about better real-world research
It sounds obvious. Yet it remains surprisingly rare.
We are in 2026 and development, medical affairs, commercial, and real-world evidence teams still too often work alongside one another rather than together. Patient-centricity, patient-focused medicines development, and patient experience data can fall into the gaps between them, valued by everyone but fully owned by no one.
In my experience this challenge persists despite the multiple reorganizations redrawing the structure seen across the industry in recent years, many of them intended to support a more joined-up, patient-focused and commercially-savvy approach to medicines development.
A false divide between development and the real world
These latter teams recognize the value of patient-centricity, but they are not usually familiar with best scientific practices that have evolved within medicines development.
This is why I refer to the US FDA’s PFDD guidance, the EMA’s PED framework and tools from PFMD into conversations about real-world research with medical affairs and RWE teams. The response is often a reasonable one: why use “medicines development” guidance when alternatives (such as the ISPOR-ISPE Patient-Centered RWE recommendations [3] and the recent ISPOR Real-World PRO Good Practices Report [4]) already exists?
I think there are three good reasons.
1. RWE guidance focuses on PROs, while PFMD guidance embraces the wider PED landscape
2. PFDD guidance represents scientific best practice for generating generalizable, interpretable and un-biased PED which extends beyond clinical trials
However, the ISPOR guidance focuses on what should be considered when collecting PRO data, rather than providing a comprehensive framework for generating high-quality PED. The FDA’s PFDD guidance fills that gap. Most of the principles proposed by FDA holds in an RWE context; it just needs to be considered in a pragmatic way, understanding the differences between the settings.
3. Patient-focused medicines development and RWE/ commercial activities are part of the same evidence journey
High quality, scientifically robust, interpretable research that answers strategic questions across this lifecycle is needed to support a forward-thinking value proposition for a novel intervention.
There are some important differences to consider when conducting patient-focused research in medicines development and the real-world. For example, the rigorous standardization and strict timing of PRO administration is not always possible (or necessary) in RWE contexts, and clinical care infrastructures cannot always cope with the collection of PED [4]. Further, data privacy and security need to be considered in quite a different way [5]. But there are more similarities than differences; patient burden and compliance should be considered across both; patient and clinical team education on the purpose and importance of PRO data collection is vital for high quality data; approaches to missing-data handling must be carefully considered to ensure data interpretability; and robust information technology (IT) infrastructures are a pre-requisite to support data capture [3,4].
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