Ending the Requirement to Show Drug Efficacy
By David Henderson
In my January 14 post, “Why Are Drug Prices So High?“, I referenced Charley Hooper’s and my proposal that Congress go back to pre-1962 law and have the FDA be a certifier of only safety, not efficacy. In the longer piece that I linked to, we also suggested having drugs automatically legal in the United States if they are approved by a similar regulatory agency in any of a few developed countries.
That post led to much discussion–58 comments so far, which is close to a record for me. Fortunately most of it was civil and much of it was thoughtful. I particularly appreciated the comments of Jim Glass, who backed much of what I wrote. I also appreciated the comments of AlanG, who was critical of parts of my post and parts of Charley’s and my short study.
Rather than reply in comments, it makes sense to reply in a new post.
We had written:
Who would want to take a drug that has not been shown, to the FDA’s satisfaction, to be effective? Almost everyone.
Saying that almost everyone would not [DRH note: Alan agreed in a comment that he didn’t mean to say write “not”] want to take a drug that has not been proven effective is an astounding statement. If this only refers to drugs where there is a documented history of use maybe I can buy it. But what evidence is there? I well remember the case of laetrile and its use as an oncology agents. It never worked. Just because there is a journal article or blogs doesn’t mean a drug works.
His response makes sense only if we had written that everyone would want to take any drug that has not been shown, to the FDA’s satisfaction, to be effective. Of course, we didn’t write that. We wrote “a,” not “any.” And we went on to establish that many people in fact do take such drugs. Alan writes that “Just because there is a journal article or blogs doesn’t mean a drug works.” That’s true and it certainly doesn’t contradict anything we wrote. Moreover, Alan himself admitted that he has taken drugs that the FDA has not deemed effective. In a later comment, he wrote: “Yes, I’ve taken one or two drugs off label but I consider myself an [sic] super-educated consumer and I always read the full prescribing information of any new drug that I am considering.”
We also wrote:
Market success would establish efficacy.
That was an overstatement and, quite rightly, commenter Stuart Buck jumped on it. The longer a drug is on the market, the more likely it is to be efficacious, but the term “establish” is too strong.
Stuart Buck reminded us that no drug is ever shown to be completely safe but then he went too far. He wrote:
This is a common suggestion, but it fails for the basic reason that no drug is “safe.” There is simply no such thing. All drugs have side effects, and some are particularly dangerous. Cancer drugs are the best example. Chemotherapy was originally developed from mustard gas used in World War I, and some of the “best” chemo drugs we have today are incredibly toxic (google “adriamycin” and “heart failure”). The ONLY reason we would ever give a chemo drug to someone is NOT that it’s safe, but that there is rigorous evidence that there might be a benefit large enough to outweigh the toxic and sometimes fatal side effects. Saying that we should approve chemo drugs for safety without efficacy is impossible nonsense.
Clearly, safety is contextual. But his last sentence, even though correct, can’t be used to argue reasonably for an FDA requirement that efficacy be established. If it were so used, many anti-cancer drugs would not be used in their current uses: they have been shown to be efficacious for a particular cancer, but not for others. I wasn’t clear whether Buck was claiming that doctors should not be allowed to prescribe and/or administer those drugs for uses where they have not been shown, to the FDA’s satisfaction, to be efficacious. If he doesn’t disagree with us, then our differences are smaller than he appears to think.
Late in the comment thread, the issue of reciprocity came up. Charley and I had discussed that in our piece, although it was not an idea I highlighted in the post. I think AlanG misunderstood our proposal. He wrote:
the idea of reciprocity of approvals has been around for over 20 years. My former employer was advocating for this back in 1992. While the idea was interesting back then, it would not have much of an impact as regulatory filings are usually done simultaneously and companies want a US approval as quickly as possible because of the lack of price controls here relative to other countries (though this may change because of Trump’s announcement that he wants to negotiate pricing for Medicare and Medicaid).
This does not establish at all that the proposal would not have much impact. Even if drug companies file simultaneously, as he says, if one agency approves it well before another, then, under our proposal, it’s legal here.