Plain-language · Issue 3 · 2026-10-28
Stop Writing Guidance About Guidance: A Call to Harmonize and Illustrate Real-World Evidence Standards
Editorially reviewed research summaries, context, and limitations.
Ashley Jaksa
Summary: A published editorial in Value in Health makes a pointed argument: the field of real-world evidence has produced so many overlapping guidance documents — from regulators, health technology assessment bodies, and professional societies — that researchers now spend considerable effort comparing frameworks rather than generating evidence. The author contends that the core principles these documents contain are already broadly consistent across major bodies such as the US Food and Drug Administration and the European Medicines Agency. Instead of issuing more high-level guidance, the author proposes two practical steps: bringing existing standards into better alignment globally, and building a publicly available, annotated collection of real-world evidence case studies that shows how decisions were actually made and why.
Why it matters: For researchers working with patient registries or health records data, navigating competing guidance documents is a real drain on time and resources — without necessarily making the resulting research any better. Regulatory and reimbursement decisions about treatments often depend on reasoning that is never made fully explicit in formal guidance. A collection of clearly explained, real-world examples of how evidence was evaluated would help researchers understand what decision-makers actually expect, reducing uncertainty and inconsistency. For rare diseases, where small datasets and limited studies are the norm, clearer and more consistent evidentiary standards could be especially important.
What to keep in mind: This is an editorial — a professional opinion piece — rather than a research study, so it does not present new data. The author's observation that core principles already converge across major regulators is important if accurate, but it is not independently verified here. The two proposed solutions are reasonable in principle but difficult to deliver in practice. Aligning guidance across agencies in different countries with different legal frameworks requires sustained commitment that the editorial does not map out in detail. The value of an annotated case repository depends entirely on how willing decision-makers are to explain their reasoning openly — something that has historically been limited. Readers should treat this as a well-grounded call for change rather than a proven path forward.
Relevance: Real-World Evidence | Regulatory Science | Data Standards | Observational Research
Source: https://pubmed.ncbi.nlm.nih.gov/42562111/
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