Patients with a rare, chronic disease can find themselves in a conundrum when seeking treatment: Researchers can’t find enough patients to enroll in a statistically rigorous clinical trial and there is uncertainty in the regulatory path. Frustrated by the lack of treatment options on the market, patients may be willing to accept a certain level of risk that device manufacturers normally shy away from.
A collaboration between the Medical Device Innovation Consortium, Mass. Institute of Technology, the Michael J. Fox Foundation and RTI Health Solutions said last month that they launched a study to evaluate the use of patient preference to set a study’s p value – a statistical figure, normally set at 0.05, used to decide if a trial’s results are significant.
But statistical measures such as p value, don’t take into account patient preference and can result in limited access to available treatments for patient populations with complex diseases.
“We recognize that there are situations where that 0.05 value, which is arbitrary to begin with, may not adequately represent the willingness that those patients have to take uncertainty,” MDIC VP of technology innovation Dawn Bardot told MassDevice.com.
In partnership with the FDA, MDIC did a retrospective analysis of patient data from obesity device trials. Andrew Lo, Director of the Laboratory for Financial Engineering at MIT’s Sloan School of Management, demonstrated the utility of patient preference information to explicitly identify the acceptable level of risk that participants were willing to take in a clinical trial.
Using the foundation’s Fox Insight platform to support surveys, the consortium hopes to engage thousands of Parkinson’s patients in a prospective study to evaluate their tool’s effectiveness with a hypothetical device.
“So patient preference informs not the trial design itself per se, but it does inform potentially the end points that could matter, and most importantly in this case, from the model’s perspective, the level of uncertainty, really the p value, what that needs to be in order for a trial to be considered a success from the patient’s perspective,” RTI Health Solutions senior economist & VP of health preference assessment Brett Hauber explained.
While the team is evaluating their tool with Parkinson’s disease, they see it as proof-of-concept work that could eventually be translated into other therapeutic areas. That will mean a serious change in how device companies think about success and risk, MDIC Patient Centered Benefit-Risk Assessment program manager Stephanie Christopher told us.
“At MDIC, we seek to do work that’s transformative, and when you’re doing work that’s transformative, there’s certainly a communication and education component that’s required,” she said.
“We are in an era of innovation, and it’s time that we bring that to how we approach clinical trials as well, and what we are doing with this method is actually adding incredible statistical and methodological rigor around decisions that people make on a daily basis,” Bardot added. “We’re making reproducible the types of questions that panels at the FDA are often asked to consider in an extemporaneous fashion and we’re putting a repeatable and statistical process around it.”
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