Online Program

Return to main conference page

All Times EDT

Thursday, September 24
Thu, Sep 24, 12:00 PM - 1:15 PM
Virtual
Roundtables

TL05-Empowerment of Performance Goal (PG) Study in Pivotal Clinical Investigations, Methodology, and Framework (301076)

Chia-Wen Ko, CDRH/FDA 
*Yongping Yan, CDRH/FDA 

Keywords: establishment of performance goal, suitability, key prognostic factors, population comparability

Since FDA issued the Guidance “The Least Burdensome Provisions: Concept and Principles”, many medical device sponsors have proposed to use single-group study with performance goals as the pivotal clinical study supporting a premarketing application. Single-group performance goal studies may be more efficient in relative to traditional randomized controlled studies, but often time it is a challenge to determine whether such studies can provide necessary and adequate (i.e., sufficient and valid) information about a device’s effectiveness and safety. The challenge arose mainly due to lack of consensus on three key elements defining a scientifically appropriate performance goal study: (1) rational justifying the use of a PG; (2) methodology to be used for historical data extraction in deriving a PG; and (3) statistical methods for derivation and validation of PG in presence of influence or bias from prognostic factors or patient heterogeneity.

In this discussion, innovative methods and systematic approach will be explored in suitability, methodology, and bias adjustment for designing a performance goal study. For consideration of suitability, we will discuss a decision-making model on rationale supporting a PG study design. For consideration of methodology, we will explore the application of AI technology in helping with establishment of performance goal. For bias evaluation, we will look at ways to implement sensitivity analysis based upon the nature and the size/extent of study bias.