Why Biogen’s drug, aducanumab, should be studied through an important part of the Affordable Care Act, the Patient Center Outcomes Research Institute.
Controversial FDA approval of aducanumab for Alzheimer’s disease will cost taxpayers expensive, although there is no evidence that it will benefit patients and caregivers. Stock analysts estimate that Biogen’s take of the drug, which was priced at $ 56,000 a year, could reach $ 40 billion annually, most of which will be paid automatically by Medicare.
Yet Republicans don’t find it in their hearts to include $ 400 billion (an average of $ 40 billion a year over the next decade) in the infrastructure bill to support the people who care for those patients. The Centers for Medicare & Medicaid Services are required by law to pay for any FDA-approved drugs based on their “reasonable and necessary” coverage standard. Helping caregivers requires an act of Congress.
Since the two approaches cost roughly the same, some agency should fund a clinical trial comparing these two approaches to help people with dementia. Fortunately, there is already an agency authorized to carry out such judgments.
The Affordable Care Act of 2010 created the Patient Center Outcomes Research Institute specifically to conduct comparative effectiveness research. Unfortunately, due to intense lobbying by the pharmaceutical industry, the original legislation prohibited PCORI from funding studies that measure profitability or using study results to recommend coverage decisions.
However, in 2019, Congress granted the agency the right to conduct studies that measure the “potential burdens and economic impacts” of healthcare services, in addition to “the full range of patient-centered and clinical outcomes.” . What better time to use that power than at the beginning of an expensive new drug program whose clinical effects will not be known for nearly a decade? Biogen told the FDA that it will take almost that long to complete its post-approval trial that measures actual results.
One arm of a PCORI-funded trial would come up with the drug whose benefits have yet to be demonstrated (the FDA based its approval on a surrogate marker, which removes plaque from the brain, not a proven clinical benefit). Another arm would receive home care along with non-pharmacological interventions such as physical and cognitive exercise. And a third arm would get both.
Such a trial could not only measure the impact each approach has on cognitive decline, but also how it affects the emotional and financial well-being of patients’ families. The combined arm would determine whether the benefits of the two approaches are additive.
If the results are the same or there are only minor differences, patients and their families could use the results to make an informed decision about the approach they consider appropriate for their situation. In fact, the administration could use the results to turn the $ 40 billion annual spending into a coupon program, a move that should warm the hearts of Republicans. Let patients choose. Why should the right apply only to products of the pharmaceutical industry?
PCORI already has experience funding comparative trials in Alzheimer’s research. A recent study by researchers at the University of Florida compared how patients with mild cognitive impairment responded to five activities to improve memory (group classes, a support group, physical exercise, a computer brain training application, and a paper reminder system). Each of the five groups in the trial received four of the five interventions and a different one was omitted for each group.
After one year, all patients reported similar improvements in quality of life and ability to perform memory-dependent activities compared to their baseline state. There were some differences, unsurprisingly. Patients who did not take classes reported worse mood. Those who didn’t use the app reported better moods.
Another test now recruitment California Patients is comparing the best place to provide supportive care for Alzheimer’s disease with the goal of reducing behavior problems. There will be more than 2,000 participants living at home or in assisted living facilities. They will be divided into groups that will receive supportive care, either in a primary care setting from a nurse or physician assistant or in a community center from a nurse or social worker. Results are expected within 18 months.
A few weeks ago, i recommended CMS conditions payment for aducanumab to physicians who prescribe and enroll their patients in a registry that researchers could later use to judge results long before the company’s post-approval trial. A PCORI-funded comparative efficacy study testing aducanumab versus non-pharmacological interventions would be a welcome addition to the knowledge base of clinicians. It would be especially valuable to families struggling to find the best way to treat loved ones with this debilitating disease.
This piece originally showed up on GoozNews, the author’s Substack site.