The New York Times reports on the passage of a “right to try” law, which is a law which permits terminal patients to bypass the FDA mandated testing process and try unapproved drugs for their conditions directly from the suppliers:
A program known as compassionate use, or expanded access, has been in place since the 1970s. It allows patients with a serious disease or condition to obtain experimental medicines; the Food and Drug Administration says it authorizes 99 percent of the requests for expanded access that it receives.
The new national law — like similar laws in more than three dozen states — allows patients and doctors to ask drug companies directly for access to the experimental drugs, rather than wait for approval by the agency.
Yet these laws “do not ensure that manufacturers will provide the drug or that insurance companies will cover the cost,” according to a policy report from Rice University. Obtaining the medicines from manufacturers can be more cumbersome than going through the Food and Drug Administration’s existing program, the report found.
President Trump’s prediction:
“We will be saving — I don’t even want to say thousands, because I think it’s going to be much more, thousands and thousands, hundreds of thousands. We’re going to be saving tremendous numbers of lives.”
The commentary of the blind anti-government ideologue. And the same article supplies a quote from an advocate:
“It’s impossible to tell how many people will be helped by the federal right-to-try law,” said Christina Sandefur, the executive vice president of the Goldwater Institute, the conservative group that spearheaded the effort.
But “if it saves one person, it’s worth it,” she said, adding, “For the patients who are turned away from clinical trials and who are unable to navigate the complex expanded access process, right to try will give them a last chance — and the right to hope.”
Which is naive thinking. Perhaps one life is saved, while another 99 die prematurely, even with their already terminal conditions, and in agony. Would she still stick with her statement? The problem is: she won’t know about the 99.
We’re potentially in a big ol’ social experiment here, and it’s important that we be prepared. Toward this end, we should be collecting data – who and how many people are applying for these unproven drugs? What are the outcomes? How does the fact that this is a transitional process affect the outcome, and how can we compare that to a scenario in which the FDA is little more than a rubber stamp?
In other words, if we’re going to walk down this path, let’s get as much information on how it works out as compared to historical FDA process of having to meet safety and efficacy targets before it can be administered to the general patient population. If I’m a sober citizen of this country, I don’t need to have a few effing anecdotes about how it helped your brother Jose survive his cancer – I don’t even know if Jose exists, I don’t know if had an unpredictable remission, a mis-diagnosis, or if he’s just a figment of someone’s malignant imagination.
No fucking anecdotes. Got it?
Good research studies are the order of the day, no matter how much that might turn the stomachs of the advocates. It’s the only way to find out if the old FDA way of doing things is good, or if letting the desperate plead for drugs of unknown quality is the better way to go.
