While all eyes are on Biogen’s FDA-required Aduhelm trial that will determine whether the treatment actually aids Alzheimer’s disease patients, the company is involved in another real-world study in the works assessing its worth in an array of people coping with the memory-robbing disease.
That phase 4 observational study was first organized by Biogen ahead of Aduhelm’s nod in June, James Galvin, M.D., a professor of neurology at the University of Miami and the study’s presenting author, told Fierce Pharma. The study is intended to discover the drug's safety and impact on the disease in 6,000 patients treated with the antibody, according to details released during the Alzheimer’s Association International Conference on Thursday.
To be sure, it’s not the highly anticipated confirmatory trial the FDA required as part of Aduhelm’s accelerated approval. Rather, the observation trial outlined on Thursday—dubbed ICARE AD-US—will be seen as “complementary” to Biogen’s mandatory evaluations, Galvin noted.
The FDA gave Aduhelm the go-ahead based on its ability to clear amyloid plaque in a patient’s brain—a hallmark of Alzheimer’s disease. As part of the agency's approval conditions, Biogen will have to show that the amyloid clearance also leads to a slowing down of cognitive decline.
In comparison, ICARE’s goal will be to determine how Aduhelm, also known as aducanumab, performs in the real world, rather than in controlled clinical trials.
“I think something like this is incredibly important to do,” Galvin, who also served as an investigator for Biogen’s late-stage Aduhelm trials, told Fierce Pharma.
All the sites are ready to begin dosing patients, but enrollment isn’t expected to pick up until Medicare and other third-party payers decide which patients will be reimbursed for the $56,000-per-year treatment.
“I don’t think this study will get up and rolling until we understand that,” Galvin said.
The primary objective of the study will be to evaluate how well Aduhelm slows the progress of Alzheimer's in dosed patients, measured by a patient’s cognition, function and behavior as well as their performance through various assessment tools.
Researchers will also look at how Aduhelm impacts healthcare resources, quality of life and overall disease burden. The study will enroll patients in line with the FDA’s narrowed label, aiming to do so over four years at roughly 200 clinical sites across the U.S. Researchers will collect data over five years, although the timeline could be extended, Galvin said.
One gray area researchers intend to investigate is Aduhelm’s impact on diverse patients, including at least 500 African American and 500 Hispanic people. Biogen has faced pushback over the lack of patient diversity in its controversial phase 3 studies, known as EMERGE and ENGAGE.
ICARE will also include people who may not typically qualify for clinical trials, like those with diabetes, high blood pressure and cancer.
As part of the observational study, researchers will also collect blood plasma and serum from patients so Biogen can measure changes and, potentially, improvement via blood samples, Galvin said. Currently, the only way of doing that is through PET scans or lumbar punctures that could cost patients thousands.
“I think there’s a lot of amazing information that could be gleaned from this, but you have to know what it can’t do,” Galvin said. “And what it can’t do is be used as a confirmation study for the FDA because there’s no control group. There’s no placebo.”