Earlier this year, to much fanfare, President Trump signed the “right-to-try” legislation enacted by Congress with the purported goal of helping terminally ill patients who want to try experimental drugs. The issue has captivated the American public, and the right-to-try law has been hailed as a legislative success.
But how does that square with reality?
Certainly, right-to-try has generated a great deal of interest in the news media and among the general public. Unfortunately, along the way a lot of inaccurate information began circulating, confusing many of the key players—patients and drug manufacturers—about what the legislation really does. Instead of faster, easier access to investigational treatments, right-to-try has raised expectations while creating real confusion. Even worse, in the short term, the legislation may actually have harmed access.
Clearing up the misconceptions
The confusion surrounding right-to-try is rooted in misinformation about the Food and Drug Administration’s expanded access program (what I call track 1) and how right-to-try (what I call track 2) could streamline the FDA process. Many believe that right-to-try allows patients to bypass FDA and go directly to drug manufacturers to get instant access to experimental medicines. That’s simply not true.
Unlike track 1, which requires patients and manufacturers to engage FDA early on in the process, track 2 leaves the FDA’s involvement until after the experimental drug is used. The right-to-try legislation requires manufacturers and physicians to file reports about each track 2 use of a drug, including the number of doses, patients treated, and any adverse reactions. The FDA is also required to post an annual summary of this information on a drug-by-drug basis. FDA currently is reviewing the legislation in anticipation of issuing guidance for drug manufacturers.
Proponents of right-to-try argue that involving FDA early in the approval process created a stumbling block. In fact, however, between 2010 and 2017, FDA approved 99 percent of requests for expanded access. Under the new two-track system, since FDA will be included either way, drug manufacturers may prefer to engage FDA early in the process instead of after the fact.
Unfortunately, the right-to-try bill did not address one of the largest challenges to expanded access, which is creating a reimbursement scheme to pay for the experimental medications. Track 2 hasn’t resolved that issue, even though several manufacturers with drugs in clinical trials believed they could start charging for their use before approval.
The bottom line: The FDA expanded access process was working, and that hasn’t changed. It is hard to see how the right-to-try track will improve on that. Moreover, it is unclear who would even use the right-to-try track. Time will tell whether any of this was worth the effort—or the confusion.
A member of the Board of Directors of Clinical Research Pathways, David Farber, J.D., has extensive experience in health policy. He routinely speaks on issues related to expanded access to investigational drugs and devices. The views expressed are his own.