RESEARCH TRIANGLE PARK, NC– RTI Health Solutions, a business unit of RTI International, will join a collaborative led by the Medical Device Innovation Consortium (MDIC) that will work with Parkinson’s patients to determine the benefit and risk tradeoffs they are willing to make for a potential new therapy, and use that information in designing a clinical trial.
This project will represent the first time patient preference on benefits and risks has been used as an explicit means to set significance levels in a clinical trial design.
The project is a collaboration of MDIC, RTI Health Solutions, FDA, the Massachusetts Institute of Technology (MIT) and The Michael J. Fox Foundation for Parkinson’s Research (MJFF). RTI will perform the benefit-risk assessment with the Parkinson’s patients that are participating in the project.
“This work represents a unique opportunity to make significant advances in three key areas – giving patients a meaningful voice in how medical technologies are evaluated, incorporating preferences into key product-development decisions, and demonstrating the value of patient input into clinical study design,” said Brett Hauber, Ph.D., senior economist and vice president, Health Preference Assessment at RTI, who will lead RTI’s part in the project.
For chronic debilitating and terminal illnesses with no effective therapies, the standard statistical threshold for determining therapeutic effectiveness in clinical trials may be too conservative and may not reflect patients’ perspectives on the trade-off between the risk of endorsing an ineffective therapy (Type I statistical errors) and the risk of rejecting an effective therapy (Type II statistical errors).
Clinical trial designs based on patient preferences may expedite trials, allow smaller trials, and enable groups representing rare disease states or smaller patient populations to participate in trials they can actually complete.
The direct incorporation of patient preferences into clinical trial design will address the longstanding conundrum that, in an enterprise devoted to easing the burden of disease, those affected with the disease often have no input into the process. By designing clinical trials that reflect patients’ urgency and risk tolerance, scarce resources can be allocated more efficiently, bringing more therapies to more patients faster and more cost-effectively.
This project marks the first time MDIC has partnered directly with a patient group to engage patients as research partners. Representatives from The Michael J Fox Foundation Patient Council are serving on this project as patient scientists and will give direct input on the design and execution of the project.
“We view this project as potentially transformative in medical device clinical trials and we are thrilled to work with The Michael J Fox Foundation, who are the recognized leaders in mobilizing Parkinson’s patients to participate in clinical trials,” MDIC President and CEO Bill Murray said. “We couldn’t ask for a better patient partner.”
This method incorporates patient preference information as an explicit means to set significance levels in clinical trial design. While this project focuses on a specific disease state, the method is generalizable to other diseases and has the potential to 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.