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# FDA Officials Leery About ‘Real World Evidence’ Potential
- URL: https://www.fdaweb.com/fda-officials-leery-about-real-world-evidence-potential/
- Published: 2016-12-09T12:00:00.000Z
- Updated: 2026-09-14T21:53:35.000Z
- Author: David McFarland
- Tags: Drugs, Devices, #legacy-id-D5137529

FDA officials writing in the 12/8 *New England Journal of Medicine* express caution about “real world evidence” and its potential to contribute efficiently to current knowledge gained from traditional clinical trials, “whose well-known limitations make it difficult to generalize findings to larger, more inclusive populations of patients, providers, and health care delivery systems or settings that reflect actual use in practice.” Written by a who’s who at FDA, including FDA commissioner **Robert Califf**, CDER director **Janet Woodcock**, CDRH director **Jeff Shuren** and CDER medical policy director **Robert Temple**, the authors caution against expecting any “quick wins” from using such evidence. Apparently writing to head off a negative impact on FDA review standards from the [fast-moving](https://www.fdaweb.com/senate-passes-cures-act-and-sends-to-obamas-desk/) 21st Century Cures Act, they say “other analytic methodologies with varying levels of evidentiary requirements, such as historical controls or study designs with an open-label phase in which all patients receive the investigational product, fall within the spectrum of potentially useful approaches that will require careful consideration before they can be appropriately applied to answer important questions about the effects of treatment with medical products in real-world settings, including issues involving latent or rare outcomes and treatments for rare diseases.”

The authors look forward to advancing “robust policy development under the proposed reauthorization of the Prescription Drug User Fee Act VI (user-fee program) for drugs and biologic products. This commitment includes convening public workshops involving participants on all bands of the research spectrum — from patients to providers to sponsors — to gather input on the use of real-world evidence in regulatory decision making. With this information, the agency will initiate activities to address key concerns and publish draft guidance on how such evidence can be used to assess safety and effectiveness in both premarketing and postmarketing regulatory requirements. Complementary efforts are included in Medical Device User Fee Amendments IV for devices.”

The article is [available here](http://www.nejm.org/doi/full/10.1056/NEJMsb1609216?query=TOC&ref=fdaweb.com).