When I wrote a week ago about the sham that is “right-to-try”, one criticism (among many) that I made of these misguided, profoundly patient-unfriendly laws was that I have as yet been unable to find a single example of a patient who has managed to obtain access to an experimental therapeutic through such a law, much less been helped by it. So-called “right-to-try” laws, of course, claim to provide a mechanism by which patients with terminal illnesses can obtain access to experimental therapeutics not yet approved by the FDA but still in clinical trials. They are, as I’ve pointed out, a cruel sham, placebo legislation that makes lawmakers feel as though they’ve done something good but do nothing of substance for patients while providing them with false hope. The federal government through the FDA controls drug approval, which means that states can’t compel a drug company to provide a drug to a patient, and most drug companies would not want to risk jeopardizing approval of their drug, which is what could happen if they grant access to an investigational drug under right-to-try and the patient suffers an adverse event. After all, the success rate for drugs that have passed phase 1 (which is all that right-to-try requires) in phase 3 trials is only on the order of 9-12%, meaning that that’s the most optimistic probability that such drugs would benefit a patient. In reality, it’s almost certainly much, much lower.
In a recent “Perspective” article in The New England Journal of Medicine, three physicians (Drs. Cox, Borio, and Temple) make a strong case for not letting the rush to save Ebola patients tempt us to deviate from good science and skip the randomized controlled trial (RCT). Their arguments cut to the essence of the scientific approach to medicine, and they deserve careful consideration.
Ebola is uniquely scary
Ebola is the kind of threat that really gets our attention. The virus was first identified in 1976, and prior to 2013 there were several small outbreaks in Africa with death rates as high as 90%. This time the death rates are lower, but the numbers are much greater. It has spread to several African countries, and a few cases have even reached the US and Europe due to infected travelers and health care workers. We face a risk that Ebola may become endemic, smoldering along as a constant presence in Africa.
There is no known effective treatment. Fear of Ebola has sparked bizarre conspiracy theories and claims of “natural” cures and prevention kits from homeopaths, alternative medicine advocates like Mercola, and purveyors of remedies like colloidal silver and essential oils. These have been covered on SBM here, here, and here. (more…)
Ed. note: Please read disclaimer in Dr. Gorski’s profile!
There are times when supporting science-based health policy and opposing health policies that sound compassionate but are not are easily portrayed as though I’m opposing mom, apple pie, and the American flag. One such type of misguided policy that I’ve opposed is a category of bills that have been finding their way into state legislatures lately known as “right to try” bills. Jann Bellamy and I have both written about them before, and with the passage of the first such bill into law in Colorado in May, I had been meaning to revisit the topic. Although “right-to-try” laws are a bad policy idea that’s not new, versions of such bills having been championed by, for example, the Abigail Alliance for at least a decade, the recent popularity of the movie Dallas Buyers Club appears to have given them a new boost, such that Colorado state Senator Irene Aguilar even frequently referred to her state’s right-to-try bill as the “Dallas Buyers Club” bill. It’s a topic I’ve been meaning to revisit since the news out of Colorado, but apparently I needed a nudge, given that it’s two months later now.
Unfortunately, that nudge came in the form of a right-to-try bill (Senate Bill 991) being introduced into the legislature in Michigan by Senator John Pappageorge and unanimously passing, almost without comment by the committee and certainly with minimal news coverage, through the first hurdle, the Michigan Senate Health Policy Committee. In parallel, the same legislation (House Bill 5651) has been introduced into the Michigan House of Representatives.