Margaret Sheehan isn’t a scientist, but she is 1 of many patient scientists joining an effort to incorporate Parkinson’s patients’ preference into the design of clinical trials for new treatments.
Sheehan, a Virginia-based lawyer, has had Parkinson’s disease since 2004. She told Drug Delivery Business News that when the folks at the Medical Device Innovation Consortium presented last spring to the Michael J. Fox Foundation’s Patient Council, she was stunned to learn that some clinicians do not involve patients when setting the desired outcomes and risk levels for a clinical trial.
“That was a huge eye-opener for me,” she said.
The MDIC, Massachusetts Institute of Technology, Michael J. Fox Foundation and RTI Health Solutions teamed up last year to launch an effort to re-evaluate the use of statistical measures, such as a p value of 0.05, that don’t take into account patient preference. The statistical figure of 0.05 is widely used in clinical trials to determine if a study’s results are significant.
Stephanie Christopher, program director at MDIC, explained that this “pretty arbitrary” value can leave Parkinson’s patients with few effective treatment options. Christopher’s team hopes to use patient preference to establish statistical levels for Parkinson’s trials other than the conventionally accepted value of 0.05.
The 1st step was to enroll a group of patient scientists, people with Parkinson’s disease, and identify the outcomes that are important to patients. The team narrowed the list to 9 symptoms that patients are most concerned about and what to see addressed with a new product or therapy.
“Parkinson’s is a very complicated disease – there are so many symptoms,” Sheehan said. “A lot of it was prioritization and a lot of it was categorization.”
While most clinical assessments of Parkinson’s patients focus on motor skills, the team and collaborators from the FDA said they were struck by how highly patients prioritized a treatment that could improve cognitive functioning.
“The patients emphasized that while motor skills are important, there are other things that are important to patients that often don’t get captured in those motor skills assessments. Specifically, the cognitive and the psychological symptoms that can come along with Parkinson’s,” Christopher said. “This is exactly the reason why it was so important for us to engage with patients from the earliest stages of this study.”
The MDIC team met again with the Patient Council at their spring meeting last month. Their discussion will inform a patient preference survey which is slated to launch in the summer and stay open until the early fall via Fox Insight – the foundation’s database of patients ready and signed up to participant in surveys relating to patient experience and Parkinson’s disease.
Parkinson’s is a complex disease, marked by “on” and “off” periods of motor and cognitive symptoms. Sheehan pointed out that this makes talking about measuring outcomes difficult.
“It ends up being like 3-dimensional chess,” she said, adding that this is why it’s crucial to acknowledge the preferences of patients that experience the disease every day.
“I’ve come to realize that just the experience of having Parkinson’s makes me an expert in having Parkinson’s. It doesn’t make me an expert in the science of Parkinson’s, but it makes me an expert in being someone with the disease,” Sheehan said. “It’s been incredibly gratifying, personally, to be a part of something that is potentially so profound and groundbreaking.”