This January, the Food and Drug Administration approved the drug Leqembi to treat Alzheimer’s disease. Leqembi is pathbreaking in two ways. First, while the vast majority of Alzheimer’s drugs can only help slow the progress of the disease, Leqembi can stop and reverse cognitive decline. As an FDA news release states, “This treatment option is the latest therapy to target and affect the underlying disease process of Alzheimer’s, instead of only treating the symptoms of the disease.”
Second, Leqembi is one of only two drugs approved by the FDA to treat Alzheimer’s in the past 17 years. The other one, Aduhelm, reached the market in 2021 after undergoing a long, complicated, and controversial road to approval. Like Legembi, Aduhelm demonstrated the ability to reverse Alzheimer’s symptoms through clinical trial results.
Adding a second treatment method to combat Alzheimer’s disease occurs during a much-needed time. Before Covid-19, medical professionals likened Alzheimer’s disease to a “silent pandemic.” The American Brain Foundation estimates about 5 million Americans currently have Alzheimer’s disease, with another 200,000 living with early-onset Alzheimer’s. Some estimate this figure will more than double by 2050.
Leqembi and Aduhelm provide different ways to combat Alzheimer’s disease, but they have a critical similarity. They both received accelerated approval from the FDA.
Under the agency’s Accelerated Approval Program, The FDA will allow drugs that treat serious conditions where few other treatment options exist to receive special attention from the agency if they have early clinical success. An important part of this program is that drugs selected for this process receive a decision in under six months—allowing treatments to reach patients considerably quicker. Although these drugs still undergo post-market review (which can take a decade), accelerated approval allows potentially life-improving or even life-saving drugs to help patients with little hope of improving their health.
Forgoing the lengthy and expensive FDA drug approval process to allow Alzheimer’s patients access to pathbreaking—but risky—treatment options has garnered criticism. Some consider Leqembi’s ability to treat Alzheimer’s disease to be “modest” and insist “more testing is needed.” Aduhelm’s approval by the FDA was called “highly irregular” and “inappropriate” for how quickly it received approval.
Many of these criticisms fail to recognize that the typical time and resources required to complete the FDA’s approval process are already excessive and provide little return on product safety and effectiveness. A range of studies dating back from 1973 to 2021 finds the FDA’s sluggish and expensive route to new drug approval is more likely to prevent patients from receiving beneficial treatments than prevent them from using ineffective or harmful ones.
Accelerated approvals and other avenues to truncate the FDA’s approval process aren’t trading off safety and effectiveness for urgency. They’re providing all three.
There are numerous examples. As Covid-19 spread across the country in 2020, the FDA issued Expanded Use Authorizations, which strongly weakened the agency’s oversight to regulate Covid-19 tests and treatments. Expanded access to the controversial Covid-19 treatment remdesivir saved countless lives during the first year of the pandemic. In 2016, Ted Harada miraculously improved his ALS symptoms after receiving an experimental medication through Georgia’s right-to-try laws.
Alzheimer’s is a debilitating disease, and developing ways to treat it has been frustratingly slow. Leqembi and Aduhelm give cause for optimism and so does the Accelerated Approval Program’s pathway that prioritizes patients over bureaucratic processes.