America’s drug approval process is slow, cumbersome, and expensive. The process to bring a new medication to market costs more than $2 billion, and patients have to wait more than ten years to access life-saving drugs. Gene therapies, which modify a person’s genes to treat or cure disease, are too often caught up in this regulatory thicket. That’s why it’s surprising (and nonsensical) that some researchers and regulators argue that the Food and Drug Administration (FDA) is approving therapies too quickly. In a recent op-ed for The Washington Post, scholars affiliated with Harvard Medical School and Brigham and Women’s Hospital claimed the FDA has been pursuing “questionable approvals of drugs targeting devastating neurological conditions.”
This assertion contradicts plenty of evidence to the contrary and detracts from common-sense regulatory reform efforts. The FDA can help patients, but only if it recognizes the real problems plaguing the drug approval process.
In their article, researchers Liam Bendicksen, Edward Cliff, and Aaron S. Kesselheim use the agency’s recent approval of a gene therapy called Elevidys to make the case that the FDA is being too permissive. Elevidys is designed to treat Duchenne muscular dystrophy, a devastating neurodegenerative condition that makes it hard to move and leads to dangerous heart and respiratory complications. Bendicksen, Cliff, and Kesselheim claim that evidence of the drug’s efficacy is murky and lacks statistical significance. This assertion ignores key evidence gleaned from clinical data. In Elevidys’ confirmatory trial of 125 patients, researchers kept track of the medication’s ability to reduce the time it takes for patients to rise from the floor. “Time to rise” was not a primary endpoint for the analysis, but is a key predictor and indicator of the disease’s progression. Taking more than five seconds to rise from the supine position tends to indicate loss in movement from the disease.
Trial results were especially encouraging for this critical measure. Elevidys reduced the chance of patients getting up slowly by more than 90 percent over a 52-week period. These results proved statistically significant across age groups. Bendicksen, Cliff, and Kesselheim minimize these results, describing them as “improvements of less than a second in the time it took patients to rise from the floor or walk 10 meters.” This glib description understates the vast impact that a fraction of a second makes in disease prognosis and progression. The brain signals and muscle contractions involved in standing up take a fraction of a second, and the most minute changes can make all the difference in disease detection and treatment. The op-ed authors downplay a point that researchers cannot afford to ignore.
The FDA deserves applause for green-lighting Elevidys. Unfortunately, other promising medications have been axed by regulators and this faux alarmism may delay more life-saving medications and therapies. 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 can save millions of lives by ditching risk aversion and embracing flexibility. That won’t happen unless everyone is on the same page about the agency’s myriad failures.
Ross Marchand is a non-resident fellow for the Taxpayers Protection Alliance.
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