ALS Drug at Last, but Dying Patients Deserve Better Than FDA’s Stalling

Eight years ago, countless people around the world joined in the Ice Bucket Challenge, dousing themselves with ice-cold water on social media to raise awareness and money to find a cure for amyotrophic lateral sclerosis, commonly known as ALS or Lou Gehrig’s disease. What few probably knew was that their grassroots campaign would hit an iceberg—but not for lack of funds or medical innovation. Instead, it was the slow-moving FDA drug approval process that would keep an innovative, potentially lifesaving drug out of the hands of those who desperately needed it the most.

Thankfully, after significant delay, the U.S. Food and Drug Administration (FDA) finally approved an ALS drug developed by Amylyx Pharmaceuticals—the first treatment funded by the ALS Ice Bucket Challenge to receive the FDA green light. It’s only the third approved ALS treatment overall—and the first that may both slow a patients’ functional decline and extend their lives.

But while this approval is a positive development for the 30,000 Americans with ALS, it highlights a major issue with the FDA’s approval process: namely, that patients facing life-threatening and terminal illnesses often run out of time while bureaucratic rules and red tape delay approval. Not only was this treatment first considered for approval in February of this year only to be stalled because “more data” was needed, but during this delay, the drug was approved in Canada. These bureaucratically driven delays came in addition to an already slow and financially costly drug approval system, one where it takes an average of 14 years and $1.4 billion for a single drug to make its way through the clinical trial process and obtain FDA approval.

Meanwhile, we know that roughly 24,000 people die from ALS every year. Countless more will suffer from the deterioration this cruel illness imposes on their mobility, ability to communicate, and, ultimately, their ability to chew, swallow, and breathe. This heartbreaking disease also upends almost every aspect – emotional, financial, and physical – of the lives of those who love and care for these patients. Dying patients simply do not have time to wait on bureaucratic delays when their lives are hanging in the balance.

The same kind of urgency that we have rightfully seen with the federal government’s willingness and swiftness in responding to COVID-19 and Monkeypox—which is serious but not usually deadly—should be used to address terminal diseases and illnesses for which there is no cure. Removing regulatory obstacles, cutting red tape, and prioritizing the severest of illnesses should be norm, not the exception.

Even though the U.S. leads the globe in drug innovation and development, the risk-averse nature of the FDA—and in this case, the desire for “more data”—meant that a treatment developed in Cambridge, Massachusetts, was first available in Canada.

More importantly, many patients could have benefitted from the treatment in these ensuing months. It didn’t have to be this way.

Naomi Lopez is the Director of Healthcare Policy and a contributor to the Goldwater Institute’s FDA blueprint, Putting Patients First: Unleashing Innovation in American Healthcare.