I blogged last week about the website Science Based Medicine, where you will find unfailingly interesting articles about the intersection of science and medical practice. I now want to recommend an article by David Gorski about so-called "Right-to-Try" legislation that has been enacted in 33 states and has recently been introduced in Congress. It is a long piece that exposes both the fallacies and dangers inherent in the legislation. In brief, right-to-try laws purport to allow terminally ill patients access to potentially life-saving drugs that have passed Phase I trials. In reality, the state statutes do nothing, because drug approval is an exclusively federal issue under the FDA. Here is how Gorski puts it:
Basically, right-to-try laws are a solution looking for a problem or, as I like to call them, placebo legislation. They make lawmakers feel good, but they do nothing concrete to help actual patients. It’s also important to remember that the real purpose of right-to-try laws is not to help patients, but to neuter the FDA’s ability to regulate certain drugs, consistent with the source of this legislation. Passing state right-to-try laws is part of a strategy to build pressure to pass a federal right-to-try law and thus weaken the FDA.
Proponents of federal right-to-try legislation include President Trump, who said:
“One thing that’s always disturbed me is we come up with a new drug for a patient who is terminal, and the FDA says we can’t have this drug used on the patient. But the patient within four weeks will be dead . . . . They say ‘well we still can’t approve the drug and we don’t know if the drug works or if it doesn’t work but we can’t approve the drug because we don’t want to hurt the patient.’ But the patient is not going to live more than four weeks.”
Gorski, however, explains:
This is a fallacious argument frequently used by right-to-try advocates. Basically patients with only four weeks to live are highly unlikely to be helped by pretty much anything, experimental or otherwise. However, they can be harmed. Right-to-try proponents often ask, “What’s the harm?” or “How can it get worse?” It can. A terminally ill patient could lose those four weeks with his family or could suffer far more than he would have otherwise with palliative care.
Additionally, the nearly all of the right-to-try legislation provide that that insurers do not need to cover the drugs provided under the statutes, or even cover care necessary due to complications. Patients may also lose hospice coverage, which could be devastating to their well-being. Gorski also points out the problems with administering drugs that have only gone through phase 1 trials:
Anyone who knows anything about drug development knows that having completed a phase 1 trial is a dangerously low bar to clear to allow more widespread use of a drug. Basically phase 1 trials are small trials, usually consisting of less than 30 subjects, that look for major toxicities and adverse events. That is not enough to determine safety, nor is it intended to. Phase I trials are designed primarily to identify major side effects and to use a process known as dose escalation to determine what is commonly referred to as the “maximum tolerated dose.” It is utterly impossible for such a small clinical trial to determine the safety of a drug. Phase II and Phase III trials are needed to confirm safety. Think of phase I trials as a screening test looking for the most obvious toxicities, with phase II and III studies confirming them. Indeed, even phase III trials can’t always adequately demonstrate that a drug is safe; it’s not uncommon for less common adverse effects not to show up until post-marketing surveillance, when much larger numbers of patients receive the drug. Moreover, only 5% of all cancer drugs that enter clinical testing are ultimately approved for patient use. Among drugs tested in phase II trials, only 30% go on to phase 3.
Gorski also explains why the FDA's existing "expanded access" program is sufficient to provide drugs to terminal patients for "compassionate use."
There is much more in the article, which is comprehensive, informative, and well-argued. I say this, by the way, as someone who is quite eager to see the FDA approve a potential ME/CFS drug that has thus far not obtained the necessary votes from the relevant committee. But putting my personal interest aside, I think we are all better off when the FDA adheres to rigorous guidelines for drug approval.