Related Experiment Video
Updated: Nov 19, 2025
Visualizing and Quantifying Pharmaceutical Compounds within Skin using Coherent Raman Scattering Imaging
Published on: November 24, 2021
Demonstrating that Real World Evidence Is Fit-For-Purpose to Support Labeling: Parallels to Patient Reported Outcomes
Cynthia J Girman1, Mary E Ritchey2,3,4, Ann Marie McNeill5
1CERobs Consulting, LLC, Wrightsville Beach, NC, USA. cindy.girman@cerobs.com.
Introduction:
In December 2021, U.S. Food & Drug Administration (FDA) will issue guidance on the use of real-world evidence (RWE) to support new indications or expanded product labeling. While difficult to foresee what FDA will require, learnings can be gleaned from previous paradigm shifts at FDA, such as for patient reported outcomes (PROs) in 2006-2009.
Methods:
We contrast published requirements for justifying PROs as fit-for-purpose for a specific labeling claim with a potential approach to justify RWE as fit-for-purpose to support expanded labeling or a new indication.
Results:
PRO labeling claims require a PRO Evidence Dossier that includes: specific wording of claim, clinical trial hypothesis structure and endpoint model, and justification that the PRO is relevant and meaningful to patients in the target population (content validity) with adequate psychometric properties. FDA's 2018 RWE Framework outlined critical considerations for using RWE to support regulatory decisions, including data quality, relevancy, provenance, and transparency. Strong parallels exist between the evidence required to justify that PROs are fit-for-purpose to support specific labeling claims and evidence to justify RWE as fit-for-purpose for specific research questions and labeling. Early discussion with FDA is encouraged.
Conclusion:
Drawing on parallels with use of PROs in labeling, RWE for regulatory purposes should be evaluated within the context of specific labeling or indication, specific study design and analysis plans, and the data attributes of data source. Sponsors seeking a new indication or labeling expansion based on RWE should justify that a specific data source and specific study design are fit-for-purpose.
Related Concept Videos
01:09Bioequivalence of Drugs: Drugs with Multiple Indications
01:28Drug Products: Biologics, Biosimilars and Interchangeables
01:11Guidelines for Writing Outcome
Patient outcomes reflect the patient's response to the goal rather than what the nurse aims to achieve. Terminology should be observable and measurable to avoid the reader's interpretation. The desired outcome should be realistic and achievable in the designated care timeframe. Expected outcomes should align with adjunctive therapies. The outcome should enhance care...
01:19Pharmacovigilance
This process, termed pharmacovigilance, aims to detect, evaluate, and minimize harmful effects related to medication use. The data collection for pharmacovigilance depends on spontaneous reporting systems, where healthcare professionals or patients voluntarily report suspected ADRs.
In some cases, there...
01:29Clinically Relevant Drug Product Specifications: Methods of Establishment
01:23Types of Biopharmaceutical Studies: Controlled and Non-Controlled Approaches
Non-controlled studies, commonly employed for initial exploration, lack a control group, rendering them susceptible to biases and external influences. In contrast,...