For chronic debilitating and terminal illnesses with no effective treatments, the standard statistical threshold for determining therapeutic effectiveness in clinical trials may be too conservative and may not reflect patients’ perspectives on the potential benefits and risks of a therapy. This webinar will present the results of an MDIC collaborative project to test a novel method for incorporating patient preference information as an explicit means to set significance levels in clinical trial designs, developed as part of a collaboration with MIT, RTI Health Solutions, FDA CDRH and The Michael J. Fox Foundation for Parkinson’s Research. The primary outcome of the project is a method to determine an appropriate p-value threshold and trial size using patient input. This method may help remove barriers to access by giving patients a pathway to breakthrough, lifesaving technologies based on their risk tolerance and the resulting potential for reduced clinical trial size.
In this MDICx, speakers shared the results from the first three aims of the project:
- Aim 1 – Understanding what matters to patients
- Aim 2 – Designing and Conducting the Patient Preference Study
- Aim 3 – Designing methods for clinical trials based explicitly on patient input
For more information on the MDIC project, go to https://live-mdic.pantheonsite.io/spi/pcor
Slides to this webinar are available here.