Medicare’s recent decision to severely restrict access to Aduhelm, a new drug for Alzheimer’s patients who have no therapies available that slow the progression of the disease, sets a dangerous precedent that could delay access to promising treatments for millions of America’s seniors for generations to come.
Biogen’s subsequent decision to effectively shelve the drug provides a cautionary tale for innovators who are working on future groundbreaking treatments.
The Food and Drug Administration last year granted accelerated approval for Aduhelm. When it comes to ensuring the safety and efficacy of commercially available drugs, the FDA is the world’s gold standard. The agency implemented accelerated approval regulations thirty years ago mainly to help quickly bring drugs to market to fill unmet medical needs for the devastating HIV pandemic.
In 2012, Congress passed bipartisan legislation, signed into law by President Obama, affirming its support for accelerated approval within the framework of robust safety and effectiveness standards.
Despite this well-established process, when it came time for the Centers for Medicare and Medicaid Services to decide whether to cover Aduhelm, the agency said it needed more evidence. CMS chose to only cover Aduhelm for Medicare patients who are part of randomized clinical trials, which are typically only held in large academic health centers. Seniors who participate in the trials will still be responsible for their 20% copayment, even if they receive a placebo. This move seriously undermines the agency’s focus on health equity, as clinical trials have historically struggled to enroll minorities and people living in rural areas.
While the Aduhelm decision can be debated, CMS took an unprecedented step and extended its decision to all future Alzheimer’s drugs that target the amyloid protein as their mechanism of action (which all the major Alzheimer’s drugs under development do), and that receive accelerated approval, regardless of the strength of the data.
Effectively, CMS has undermined its own sister agency and upended the entire accelerated approval process for all drugs. The agency is impacting access because it is leaving other payors and states uncertain about whether accelerated approval drugs provide benefits, even though the FDA has deemed them safe and effective. CMS has also made investment and research in innovative treatments a risky venture.
CMS’ coverage with evidence decision for Aduhelm was unusual because the evidence requirement was very steep – a requirement for a randomized clinical trial. Typically when CMS issues such decisions, it allows evidence to be collected by having patients report outcomes to a registry. CMS has never before issued a coverage with evidence decision that required patients to enroll in a randomized trial with a risk of getting a placebo. When CMS requires additional data for a new treatment, the agency typically allows Medicare beneficiaries to access it but requires providers to report outcomes, enabling the agency to collect the data it needs to make a final coverage decision.
During my tenure at CMS, I took a more patient-centered, constructive approach to FDA’s accelerated pathways. My goal was to prioritize patient access to innovative treatments while still requiring additional data. To provide an example, for medical devices that were approved through the breakthrough process, CMS proposed three years of automatic coverage to collect additional evidence. If after that time the data and real-world evidence didn’t show positive patient outcomes, CMS could halt coverage. This same model could be used for drugs that are approved via FDA’s accelerated approval process.
Unfortunately, the Biden administration has chosen a different path for devices and drugs, slamming the door on hope for patients facing incurable diseases. The point of FDA approval is for the agency to convey that the randomized trials have shown that the drug is safe and effective, so patients can access it without having to enroll in a trial where they could get a placebo.
Understandable red flags have been raised about the cost of Aduhelm and these concerns have been used to justify CMS’ action. But there are better ways of handling high-cost drugs that don’t put patients in the middle and preserve patient access. CMS could require manufacturers to guarantee results by requiring value-based pricing, essentially only reimbursing the manufacturer when the drug achieves some clinical endpoint or reducing reimbursement if the drug isn’t effective.
The Aduhelm imbroglio ultimately reveals coordination issues between the FDA and CMS that must be resolved. As it stands now, innovators navigate between the bureaucracy of two different agencies and two teams of scientists – one at FDA for approval and another at CMS for coverage and payment. While CMS and FDA have a process that allows for simultaneous review and approvals by both agencies, it is seldom used. This approach should be the rule not the exception. The existing framework for this coordination should be expanded so that there is a single scientific review of data and evidence by the federal government. Manufacturers of drugs and devices seeking Medicare coverage should be required to provide data for the 65-plus population.
Innovative drugs and devices provide an opportunity to improve Americans’ quality of life and ultimately reduce health care costs. Millions of people living with any type of incurable disease pray for breakthroughs and the federal government should make it easier, not harder, to bring new treatments to patients. Removing bureaucratic obstacles to innovation that discourage investment would be a commonsense move to help patients access medicine that can improve their health and wellness.
Seema Verma served as the Administrator for the Centers for Medicare and Medicaid Services from 2017 – 2021.