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# Praise, Suggestions for RWE Studies Guidance
- URL: https://www.fdaweb.com/praise-suggestions-for-rwe-studies-guidance/
- Published: 2024-06-20T12:00:00.000Z
- Updated: 2026-09-14T14:33:41.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5157219

Several stakeholders are expressing their appreciation for FDA’s issuance of a draft guidance, *Real-World Evidence: Considerations Regarding Non-Interventional Studies for Drug and Biological Products*. Flatiron Health [says](https://www.regulations.gov/comment/FDA-2023-D-5470-0023?ref=fdaweb.com) the draft will give sponsors and real-world data (RWD) organizations clarity in regulatory expectations in designing a non-interventional study with RWD. “We thank FDA for elaborating on key concepts from earlier Real-World Evidence (RWE) guidance documents, including sponsor engagement with FDA and pre-specification of a study protocol for non-interventional studies,” it says. The letter also submits line-by-line specific comments.

In its [comment](https://www.regulations.gov/comment/FDA-2023-D-5470-0022?ref=fdaweb.com), the Biotechnology Innovation Organization (BIO) says the draft is generally consistent with previous guidances on the appropriate use of RWE and RWD. However, it adds, the draft is notably more high-level and lacking in detail compared with the other recent RWE draft guidances issued by the agency. “The lack of detail limits the utility of the draft guidance for guiding drug sponsors conducting non-interventional clinical trials,” BIO says. “It would be helpful for FDA to provide suggestions regarding recommended best practices for non-interventional studies. We request FDA clarify how to define the population, set the target population for some specific groups, and augment the population based on RWD.” BIO also provides specific technical comments.

EMD Serono [says](https://www.regulations.gov/comment/FDA-2023-D-5470-0020?ref=fdaweb.com) the totality of the now available guidance documents “provides valuable support for the research challenges that currently limit our ability to bring new treatment options to patients, in particular in situations where a randomized controlled clinical trial is not feasible, e.g., when very few patients are available.” The comment letter also has specific comments.

The Real-World Evidence Alliance [commends](https://www.regulations.gov/comment/FDA-2023-D-5470-0026?ref=fdaweb.com) FDA for the approach taken in the draft which, it says, will help RWD and analytics organizations as well as sponsors navigate the opportunities and complexities that come with designing a non-interventional study using RWD. It asks the agency to clarify some of the terms it uses in the draft.

The National Organization for Rare Disorders (NORD) [urges](https://www.regulations.gov/comment/FDA-2023-D-5470-0016?ref=fdaweb.com) FDA to “provide substantially more specific and actionable guidance on how to design the best possible non-interventional and observational studies for rare diseases…. The absence of such specific and targeted information arguably limits the utility and applicability of the draft guidance in its current form to the rare disease field.” NORD makes these specific recommendations:

- tailor the guidance to be more directly responsive to the unique challenges of rare disease drug development;
- provide an appendix or supplementary material with additional details and illustrative examples to further clarify the agency’s thinking and to provide actionable advice for drug sponsors on how to successfully apply the concepts in rare disease drug development;
- meaningfully engage the rare disease patient, provider, and academic community on an ongoing basis to assure awareness and proper use of the guidance and other RWE guidances;
- create a regulatory assistance program for academic investigators and patients; and
- address the use of observational studies or confirmatory evidence in accelerated approvals, including the specific statistical challenges associated with limited populations.

Finally, Regeneron [recommends](https://www.regulations.gov/comment/FDA-2023-D-5470-0024?ref=fdaweb.com) that within the guidance FDA specifically acknowledge the rapidly advancing state of science regarding RWE. It also asks FDA to clarify some guidance provisions that may be outside the control of sponsors. Regeneron includes a table of line-by-line specific comments.