Five states have enacted laws giving terminally ill patients access to experimental drugs, and 25 other states are considering similar legislation. But it’s unclear whether these so-called “right-to-try” laws will help anyone other than state legislators wishing to be seen embracing a politically popular cause.
Before it approves drugs or other treatments for human use, the federal Food and Drug Administration (FDA) requires rigorous clinical trials to ensure the drugs are safe and effective. These clinical trials can take years to complete. People with life-threatening illnesses who might benefit from an experimental treatment that is still a long way from approval have had two options. The first is to enter one of the clinical trials, which are not available to many due to geographic location and stage of illness.
The second way to gain access to an experimental drug is to apply under the FDA’s “expanded access” program. These requests are typically granted; between 2010 and 2014, only 33 of 5,849 individual patients were denied access. But the current application process is viewed by some as overly burdensome and discouraging to applicants (the application alone can require 100 hours to complete).
With the courts so far ruling that terminally ill patients have no constitutional right to experimental therapies and Congress unable to pass legislation on the issue, individual states have stepped in with their own right-to-try measures. The five states that have enacted such laws (as of March 2015) are Arizona, Colorado, Louisiana, Michigan, and Missouri. All five states require that the patient have a terminal illness, have considered all other possible FDA-approved treatments, have received a prescription or recommendation from their doctor for the experimental drug or treatment, and have consented to the risks associated with the experimental treatment. In addition, clinical trials for the drug or treatment must already be underway.
But so far “it is unclear whether any patients have successfully accessed an experimental therapy under the state laws,” according to the journal Health Affairs. One obstacle is that federal law prohibits manufacturers from shipping an experimental drug across state lines without the FDA’s approval. Also, health insurers are not required to pay for the experimental treatments, meaning that often only the well-off or those savvy enough to raise funds through social media will have access under right-to-try laws.
“They’re feel-good laws,” Arthur Caplan, founding director of the Division of Medical Ethics at NYU Langone Medical Center’s Department of Population Health, told USAToday. “They do draw attention to the issue, but they don’t do anything to get you the drugs.”
Such laws are also almost certainly unconstitutional because federal law generally trumps state law whenever the two are in conflict, although the FDA has so far not tested this in court. Critics of right-to-try laws have also raised ethical concerns, warning that the laws could undermine clinical trials and that vulnerable patients have little or no information on a treatment’s effectiveness and they may be exposed to drugs so early in testing that toxicities and side-effects have not yet emerged.
In the meantime, the FDA will soon introduce a new application form for its expanded access program that it estimates can be completed in just 45 minutes. And new federal legislation (HR 5805) is aimed at improving the expanded access program in other ways.
For an in-depth look by Health Affairs at the emergence of right-to-try laws, click here.