FDA new DRAFT Guidance: Patient-Focused Drug Development: Incorporating Clinical Outcome Assessments Into Endpoints For Regulatory Decision-Making

 The draft guidance is available here.  It is the fourth guidance in a series:

• Methods to collect patient experience data that are accurate and representative of the intended patient population (Guidance 1)

• Approaches to identifying what is most important to patients with respect to their experience as it relates to burden of disease/condition and burden of treatment (Guidance 2)

• Approaches to selecting, modifying, developing, and validating clinical outcome assessments (COAs) to measure outcomes of importance to patients in clinical trials (Guidance 3)

• Methods, standards, and technologies for collecting and analyzing COA data for regulatory decision-making, including selecting the COA-based endpoint and determining clinically meaningful change in that endpoint (Guidance 4)

The scope for this guidance is :

This guidance focuses on COA (clinical outcome assessments) issues associated with clinical trial (study) endpoints, design, conduct, and analysis and will be of most relevance for those designing and conducting trials using COAs as well as analyzing and interpreting the trial data.

 

The draft guidance spends quite a bit of time presenting different types of endpoints, their meaning and planning for assessment by the Agency. Aspects of data collection are reviewed, as well as how to handle 'errors and missing data'.  Fifteen (15) pages are dedicated to "EVALUATING THE MEANINGFULNESS OF TREATMENT BENEFIT" before the last section that discusses a variety of topics related to optimizing the clinical trial design for robust data collection for the safety and efficacy endpoint assessments.