Table 1.
Five Case Examples of Using PPI in Clinical Trial Design (compiled and assessed by MDIC [12])
| Title | Researcher | Focus |
|---|---|---|
| Patient-centered outcomes research project for parkinson’s disease | MDIC in collaboration with FDA-CDRH, the Michael J. fox foundation for Parkinson’s research (MJFF), Massachusetts institute of technology (MIT), and RTI health solutions | A patient preference study was conducted in PD and the PPI was incorporated in a BDA tool as an explicit means to set significance levels during clinical trial design [13, 14] |
| Quantifying benefit-risk preferences for heart failure devices: a stated-preference study | MDIC & six device sponsor companies in collaboration with FDA-CDRHa, Duke clinical research institute, and QLS advisors consulting group |
A patient preference study examined the acceptability of risks and benefits associated with devices used for HF, rather than for a specific device development program [15] A follow-on project computed the BDA-optimal statistical significance threshold that maximizes the expected value to patients with HF [16] |
| Selecting endpoints for clinical trials (heart failure) | Abbott Vascular | This project provided quantitative PPI on benefit-risk tradeoffs relevant to transcatheter mitral valve repair versus medical therapy for patients with HF and symptomatic secondary mitral regurgitation [17] |
| Establishing clinically relevant trial/study thresholds for success using PPI data | Edwards Lifesciences | PPI and a BDA model were planned to identify the optimal non-inferiority margin and statistical significance threshold from a patient perspective for an ongoing clinical trial [18, 19] |
| A PPI-based model for determining endpoint thresholds | FDA-CDRH | A proof-of-principle study was conducted on the preferences of obese participants regarding the use of medical devices to achieve weight loss and the results were retrospectively analyzed using a BDA model [20, 21] |
BDA Bayesian design analysis, FDA-CDRH food and drug administration-center for devices and radiological health, HF heart failure, MDIC medical device innovation consortium, PD parkinson’s disease, PPI patient preference information
aFunded partially through a broad agency announcement grant from FDA-CDRH