Too Long, you don't have to read . . .

. . . because I'll tell you, as a follow-up to my previous post. David Gorski has good things to say, as he does here, but if brevity is the soul of wit Dr. Gorski is not at all witty. (I think he already knows that but he can't help himself.)

Anyway, so-called "Right to Try" legislation has passed in 33 states, and an effort is underway to pass a federal version. This is one of those policy ideas that looks great on a bumper sticker and is impossible to refute in 30 words or less. Since the voting public gets turned off by wonkery, politicians who know better duck and cover when these proposals come along, so they pass.

The idea is that terminally ill people should have the right to try experimental treatments. Maybe they haven't been proven safe and effective yet, but they got through the first round of trials so it appears they might work, and what have you got to lose? You're dying! Why would the nanny state deprive people of a chance to extend their own lives if that's what they want to do?

Okay. First of all you need to know that the formal process for drug approval consists of three "phases" of trials. Phase one just consists of giving small and gradually escalating doses to a small number of patients, maybe 30, just to establish a level that doesn't produce acute toxicity. Passing this stage is all that is needed for the "right to try" to kick in.

But these drugs may not even have passed phase II trials. These are somewhat larger and last longer. They are underpowered to prove that a drug is safe and effective. They are intended to establish that the trends are in the right direction and that no obvious safety issues emerge in longer term use, so that the much larger investment in a Phase III trial can be justified. Only after successfully completing at least one Phase III trial can a drug be considered for approval.

It turns out that only 30% of drugs that enter Phase II trials even go on to Phase III, let alone win ultimate approval. So the first thing you need to know is that the likelihood that people could benefit from "right to try" is lower than you probably thought. Since the ostensible beneficiaries are already terminally ill, it's actually extremely low. Even safe and effective drugs are almost never miracle cures -- they might extend life for cancer patients if used early enough, but they almost never reverse advanced cancer. And yes, they can indeed harm people.

Terminal illness is actually a pretty loose concept. Doctors are very bad at predicting how long people have to live; it's very common to outlive a prognosis of 6 months to live, by a lot. So it's possible to significantly shorten the life of a person with a supposedly terminal illness. It's also possible to make them sicker. So gambling with an unproven treatment is not a no-cost bet.

And "right-to-try" legislation forbids insurance companies paying for these treatments, at their own insistence. That means the legislation affects only people with the means to pay out of pocket; and the drug companies can charge whatever they want, which is probably a lot. So you're handing over your kids' inheritance for something that is more likely to harm you than to help you.

Finally, the existence of "right-to-try" may deter people from participating in clinical trials in which they might get the placebo. Which means it will be harder for us ever to know for sure whether the stuff works. The proposed federal legislation is even worse because it actively forbids the FDA from taking adverse events in "right-to-try" patients into account in evaluating the treatment. The only point of that is to relieve the drug companies of all risk.

This is nothing but a cynical move to take money from desperate people and give it to drug companies, under cover of fake compassion and the usual nonsensical libertarian arguments. The FDA already has the authority to approve "compassionate use" of experimental medications on a case-by-case basis. That's how it should stay.