Parkinson’s Patient Benefit-Risk Preference in Trial Design

Last month, a collaboration to determine the benefit and risk tradeoffs Parkinson’s patients are willing to make for a potential new therapy was formed. Led by the Medical Device Innovation Consortium (MDIC), collaborators include RTI Health Solutions, FDA, the Massachusetts Institute of Technology (MIT) and The Michael J. Fox Foundation for Parkinson’s Research (MJFF).

RTI Health Solutions, a business unit of RTI International, will perform the patient-preference elicitation portion of the benefit-risk assessment with the Parkinson’s patients that are participating in the project. This project will represent the first time patient preferences on benefits and risks has been used as an explicit means to set significance levels in a clinical trial design.

Brett Hauber, PhD, senior economist and vice president, Health Preference Assessment at RTI, will lead RTI’s part in the project. Hauber, who has over 20 years experience evaluating patient preferences, spoke to Applied Clinical Trials about the collaborative.

“The MDIC is heading this collaborative as a public/private initiative with CDRH. There are a number of device-aided technologies for Parkinson’s patients, but historically, device trials are smaller. The model the research team will use may likely be applied to smaller populations in clinical trials and optimizing those trials for statistical power, which could be useful for devices,” Hauber explained.

Hauber explained that there are many examples throughout a product lifecycle, where benefit-risk preference information probably makes sense. In this collaborative, the Michael J. Fox Foundation will utilize its patient reach and patient council for both the preference survey and study design. “We will get an understanding from the patient partners at Fox about what matters to them and how much,” Hauber said. “We will understand the endpoints that matter to patients. Then we weight those based on how much they matter relative to the changes in other outcomes.” The collaboration’s methodology is one way of getting to the heart of patient centricity in a scientific way. “We can optimize the trial design in small sample sizes because it’s critical to get to that optimal point.” He added, “Realistically, this is a proof of concept. If we are successful, this approach can be use by industry and FDA to look at trial design.”

Hauber believes that the project will take approximately two years. Hauber added, “In my years of experience, patients are generally very knowledgeable about their disease and they have great insights. I can design what I believe to be a really great preference study, but it really requires patients’ input. I am surprised every time I speak to patients about what they offer and I always learn something from talking with them.”