When Lives Hang in the Balance, the FDA Dawdles

Imagine learning that you or a loved one is facing a terminal illness and that the expected lifespan at the time of diagnosis is two years. And imagine that, with every day that passes, you lose physical function and eventually won’t be able to swallow and breathe.

If this scenario isn’t heartbreaking enough, imagine this: a federal agency holds the power to approve a promising treatment that could slow the patient’s decline and extend their life by months—but that same agency is stalling the approval process. Tragically, this is the reality for the 30,000 patients in the U.S. with amyotrophic lateral sclerosis, commonly known as ALS or Lou Gehrig’s disease.

A U.S. Food and Drug Administration (FDA) advisory committee initially considered an ALS drug developed by Amylyx Pharmaceuticals in March. In its small phase 2 study, the drug slowed patients’ functional decline and extended their lives by an average of almost five to 11 months. But the panel wanted more data and voted against approval.

Since then, Amylyx’s ALS treatment received conditional approval in Canada in June. In a strange twist, the same country that notoriously rations care—where patients die while standing by on months-long waiting lists for urgent procedures, and where the affluent and politically connected routinely travel south of their border for care—may now serve as host to those ALS patients who are fortunate enough to still be able to travel and afford the treatment.

It should be noted that the FDA advisory committees aren’t always utilized, such as was the case with the bivalent COVID-19 vaccines for use as booster doses. And their decisions are not binding, allowing the FDA to approve a treatment even when it is in conflict with the committee’s recommendation.

With additional new data, the advisory committee reconvened again this past week to reconsider approval. With an affirmative vote for approval this time around, ALS patients and their loved ones are waiting—and hoping—that the FDA gives the treatment the green light.

While approval would be cause for celebration, the harsh reality is that a government bureaucracy not only wields this much power over Americans’ lives, but that its seemingly arbitrary decisions cause additional suffering and confusion. That is why the Goldwater Institute seeks:

• Right to Try and Right to Try for Individualized Treatments. The federal Right to Try law, signed into federal law in May 2018, is saving lives today. With the rapid medical progress in individualized treatments, we are now proposing to expand the law to include personalized medical care.

• Reciprocity. Some of the world’s leading medical treatments are increasingly found in other countries. The Goldwater Institute is proposing a pathway that would allow the important scientific work that has already been undertaken elsewhere on behalf of patients to be more quickly harnessed. As a result, treatments approved in countries with rigorous clinical evaluation would be available here.

• Transparency. The FDA has a long and well-documented history of obscuring the decision-making process it uses to make life-or-death decisions. We seek to ensure that these crucial decisions are made in a transparent and timely manner, and are not government secrets.

• Medical autonomy. Decisions about one’s medical treatments are among the most personal choices a person can make. When a patient’s life hangs in the balance, the patient under their doctor’s care, not the federal government, should have authority over the practice of medicine.

For these ALS patients, science and technology are not the single obstacle to promising treatments and the gift of more sunsets and time with loved ones; bureaucratic rules, red tape, and dawdling are. Government rules, developed for last century’s treatments, should not stand in the way of getting the right treatment, to the right patient, and at the right time. The FDA has the authority—and responsibility—to make this happen now.

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