Sunday, January 7, 2024

Is the FDA protecting us adequately?

The high point of the FDA (Food and Drug Administration) showing its worth came in 1960, when Dr. Frances Kelsey refused to let thalidomide be marketed in the U.S. She prevented the U.S. from experiencing the epidemic of babies born with deformed limbs to mothers who were prescribed the drug for morning sickness in Europe.

Do we still have similar guardians? Recent years have seen a raft of drugs and devices of dubious value approved by the FDA.

Example: Zurzuvae was approved in August 2023 to treat postpartum depression. This is a major depressive disorder that begins during pregnancy or within 4 weeks after delivery. Standard recommended care is psychotherapy or the well-studied group of antidepressants called SSRIs (selective serotonin reuptake inhibitors). Zurzuvae works on the same brain receptors as benzodiazepines (think Valium, Xanax) and barbiturates, and so not surprisingly causes sedation, sleepiness and dizziness, and most people who take it for over a week suffer withdrawal symptoms when they stop.

Two studies of Z were done in women with severe postpartum depression, and in both it was compared to placebo, not SSRIs. It was superior, but the placebo group saw about 75% of the improvement seen in active drug users. The real test would have been comparison with a safer SSRI, not a placebo.

Moreover, even though only patients with severe depression were studied, the drug was approved for all patients with the disorder. I’d avoid it.

Example: In Dec 2019, the FDA approved the oral drug Olaparib for treatment of pancreatic cancer. This drug is priced at $12,000 PER MONTH. The study leading to approval showed a very modest improvement in survival: 18.9 months vs. 18.4, with no quality-of-life benefit. Statistically significant perhaps, but clinically? I’d also question using placebo rather than existing approved drugs as the comparison.

In addition to drugs, I could describe many devices of dubious value, but will only mention one in the interest of brevity.

In 2023 the FDA approved two devices that destroy nerves to the kidney to treat high blood pressure without medication. Two studies have been done. One showed no benefit and one showed statistical lowering, but by an average of 3 mm – less than the usual variation I would see between two measurements in the same visit. Is that worth the estimated $6000 that using the device will cost? Do we have data showing long-term benefit? (No!)

We should require that the pharmaceutical and medical device industries fund studies that clearly define which patients will benefit from new drugs and devices. We should then also require our regulators to protect the public by forcing new innovations to pass a proper bar before being allowed on the market. “Might help some people,” at high cost and with potential side effects is not a high-enough bar, not when lives are at stake.

Let the FDA know they need more Dr. Kelseys and fewer rubber stamps.

Prescription for Bankruptcy. Buy the book on Amazon

1 comment:

  1. As a kidney cancer patient advocate after my own Stage IV diagnosis, now 19 years later, I am acutely aware of the value of new medications,
    However, I am shocked to hear of the approval of Olaparib with that it was approved with those pitiful trial results, and even more so to think of using a placebo as a comparator.

    In my kidney cancer world where there were few drugs, we patients and advocates fought against the use of placebo arms, as the natural history of the disease was well known! Also hard to believe that the "results" were statistically significant.