Earlier today (o5 April 2023) USFDA's Center for Drug Evaluation and Research, Center for Biologics Evaluation and Research, and Center for Devices and Radiological Health released a draft guidance on"Patient-Focused Drug Development: Incorporating Clinical Outcome Assessments Into Endpoints for Regulatory Decision-Making".
There are four methodological patient-focused drug development (PFDD) guidance documents in the series.
The objective of this guidance is to describe how stakeholders (patients, caregivers, researchers, medical product developers, and others) can collect and submit data from patients and caregivers for regulatory decisions.
This guidance focuses on Clinical Outcomes Assessment (COA) issues related to clinical trial (study) endpoints, design, conduct, and analysis and is of most relevance to those who design and conduct clinical trials using COAs, analyze trial data, and interpret the results.
In addition, this guide discusses how COA-based endpoints can be aligned with clinical study objectives to improve study and analysis planning, as well as improve study interpretation.
The guidance describes how to interpret treatment effects on COA-based endpoints in light of patient's perceptions of the product's effect. It is important to note that statistical significance alone does not indicate if the detected effect corresponds to a clinically meaningful one.