The FDA’s controversial approval of aducanumab for Alzheimer’s Disease will cost taxpayers dearly, even though there’s no evidence it will benefit patients and caregivers. Stock analysts estimate Biogen’s take from the drug, which it priced at $56,000 a year, could reach $40 billion annually, most of which will automatically get paid by Medicare.
Yet Republicans can’t find it in their hearts to include in the infrastructure bill $400 billion (an average of $40 billion a year for the next decade) to support the people who care for those patients. The Centers for Medicare and Medicaid Services by law must pay for any FDA-approved drug under its “reasonable and necessary” coverage standard. Helping caregivers requires an act of Congress.
Given the two approaches cost about the same, some agency ought to finance a clinical trial comparing these two approaches to helping people with dementia. Fortunately, there is already an agency empowered to conduct such trials.
The 2010 Affordable Care Act created the Patient Centers Outcomes Research Institute specifically to conduct comparative effectiveness research. Unfortunately, due to intense drug industry lobbying, the original legislation prohibited PCORI from funding studies that measure cost-effectiveness or using study results to recommend coverage decisions.
However, in 2019 Congress gave the agency the right to conduct studies that measure the “potential burdens and economic impacts” of health care services in addition to “the full range of clinical and patient-centered outcomes.” What better time to use that power than at the outset of an expensive new drug program whose clinical effects won’t be known for nearly a decade? Biogen told the FDA it will take nearly that long to complete its post-approval trial measuring actual outcomes.
One arm of a PCORI-funded trial would get the drug whose benefits are still unproven (the FDA based its approval on a surrogate marker—clearing plaque from the brain—not a demonstrated clinical benefit). Another arm would receive home care coupled with non-drug interventions like 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 either affects the emotional and financial well-being of the patients’ families. The combined arm would determine if the benefits of the two approaches are additive.
If the outcomes are the same or there are only minimal differences, patients and their families could use the results to make an informed choice about which approach they think appropriate for their situation. Indeed, the government could use the results to turn the $40 billion annual expenditure into a voucher program, a move that should warm the cockles of Republican hearts. Let patients choose. Why should the entitlement only apply to the drug industry’s products?
PCORI already has experience funding comparative trials in Alzheimer’s research. A recently completed study by researchers at the University of Florida compared how patients with mild cognitive impairment responded to five memory-enhancing activities (group classes, a support group, physical exercise, a computer-based brain training app and a paper-based reminder system). Each of the five groups in the trial received four of the five interventions with a different one left out 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 status. There were a few, unsurprising differences. The patients who didn’t take classes reported worse moods. Those who didn’t use the app reported better moods.
Another trial now recruiting patients in California is comparing the best place to deliver supportive Alzheimer’s care aimed at reducing behavior problems. There will be more than 2,000 participants living either at home or in assisted living facilities. They’ll be divided into groups receiving supportive care either in a primary care setting from a nurse practitioner or a physician’s assistant or in a community center from a nurse or a social worker. Results are expected within 18 months.
A few weeks ago, I recommended CMS condition payment for aducanumab on prescribing physicians enrolling their patients in a registry that researchers could later use to judge outcomes well ahead of the company-run post-approval trial. A PCORI-funded comparative effectiveness study testing aducanumab against non-drug interventions would be a welcome addition to physicians’ knowledge base. It would be especially valuable for the families struggling with how best to treat their loved ones suffering from this debilitating disease.
This piece originally appeared in GoozNews, the author’s Substack site.