The millions of Americans suffering from Alzheimer’s disease face a grim prognosis with disturbingly few options. Roughly 7 million Americans are living with the deadly disease, and that figure is expected to more than double by 2050. Patients received a glimmer of hope on July 2, when the Food and Drug Administration (FDA) approved a new medication called donanemab designed to slow the cognitive decline associated with Alzheimer’s disease. While the FDA deserves applause for approving the drug, the agency’s dismissive attitude toward other medications has cost lives and resulted in unnecessary suffering. The agency must embrace flexibility and give patients suffering from a wide range of diseases more treatment options. After years of dead ends and dashed hopes, donanemab appears to be the real deal. A 2023 studypublished in JAMA and involving more than 1,700 participants found that the medication slowed the progression of Alzheimer's disease by about 35 percent. The finding was the largest statistically significant result from any Alzheimer’s clinical trial ever performed. By comparison, researchers studying another recently approved Alzheimer’s medication (lecanemab) found that the drug reduced progression by 27 percent.
The FDA should have reacted to this incredible result by readying the medication for approval as soon as possible. Instead, it dragged its feet as usual. The agency waited more than 7 months to take any concrete action, breaking its silence in March only to delay its decision on the medication. The agency’s advisory committee finally convened on June 10 and unanimously recommended approval. The agency seemed to share the advisors’ approval and wrote in briefing documents that the drug’s safety profile was acceptable. The FDA then waited three long weeks before finally approving the medication on July 2.
Patients were lucky that the FDA approved donanemab. The agency has recently rejected promising medications for other diseases on flimsy grounds. At the end of June, the FDA denied approval of a gene therapy called Kresladi designed to treat the rare immune disorder leukocyte adhesion deficiency-I. This devastating genetic condition puts patients at risk for recurrent infections, and survival beyond childhood is rare without a bone marrow transplant. The agency seemed convinced by promising clinical trial data, but axed the therapy because of it wanted more information on the company’s manufacturing processes. Surely, the FDA could have granted approval of the therapy and monitored manufacturing to ensure that everything was going smoothly. This is basically how the agency handled COVID-19 vaccines, and any snafus in manufacturing and distribution were closely monitored and corrected as needed. If the FDA had waited until manufacturing plans were “perfect” before giving the green-light, millions of patients would have died while waiting for their shot. Regulators should pursue the “trust but verify” option instead of needless risk aversion.
Another recent rejection targeted a drug called zolbetuximab designed to treat gastric/gastroesophageal cancer. Similar to Kresladi, the issue was neither safety nor efficacy. According to clinical trial results, the medication improved median progression-free and overall survival by 2-3 months compared to just chemotherapy. The drug was held up to “unresolved deficiencies following its pre-license inspection of a third-party manufacturing facility.” These issues apparently weren’t deal-breakers for the FDA’s Japanese counterparts, who green-lit the drug. The FDA likely won’t make a decision until November, leaving cancer patients with little hope and few options.
The FDA should reject risk-aversion and put patients first by embracing choice and flexibility. The agency’s approval of donanemab demonstrates that this is not a pipe dream. Millions of patients cannot afford to wait for endless regulatory delays and blown agency deadlines.
Ross Marchand is a non-resident fellow for the Taxpayers Protection Alliance
