Next month, Missouri will have a “right to try” law in place that aims to allow terminally ill patients who have exhausted conventional treatments to seek the use of drugs, devices and products that haven’t gone all the way through the federal Food and Drug Administration’s testing process.
John DiPersio, MD, PhD (left) and Show-Me Institute analyst Patrick Ishmael
Governor Jay Nixon (D) signed legislation on Monday to make Missouri the third state to enact such a law, to give patients and their doctors the opportunity to try so-called “investigational” drugs if the makers of those drugs agree.
Proponents of the measure like Show-Me Institute analyst Patrick Ishmael say it will mean a lot to patients.
“I think they’re going to find this legislation as being very helpful to them, being very hopeful for their families,” says Ishmael, “and at the end of the day it provides them an opportunity to pursue treatment and care that before, the state may have been able to step in and try to prevent.”
The bill has critics, however, who say it won’t actually do anything.
“The problem is there’s this thing called the FDA,” says Doctor John DiPersio, Deputy Director of the Siteman Cancer Center in St. Louis and Chief of the Division of Oncology at Washington University in St. Louis.
DiPersio says a mechanism for giving patients access to investigational drugs already exists. He says no matter how the new law reads, the administration to a patient of a drug still under development without FDA approval is illegal.
“If you gave an investigational drug and the FDA found out about it … outside of a clinical trial, outside of their proper mechanism, then it could be potentially catastrophic for the patient, the physician and the institution,” says DiPersio.
DiPersio says it is unlikely pharmaceutical manufacturers would risk development of new drugs by giving them to patients under the terms of the “right to try” law.
“You can’t just give your drug to everybody in the country that wants it and not be in a position to actually, rigorously follow those patients,” says DiPersio, “because if something bad happens then it could undermine the development of the drug, number one. Number two is that there are liability issues.”
HB 1685 was sponsored by Representative Jim Neely (R-Cameron)
Proponents counter that one company, Neuralstem Inc, has expressed interest in making available experimental treatments for Amyotrophic Lateral Sclerosis (ALS, otherwise known as Lou Gehrig’s Disease), under Colorado’s right to try law.
Still, DiPersio says allowing such access to new drugs not only threatens their development, but he criticizes the bill for allowing drug companies to require patients receiving an experimental drug, product or device, to pay for them.
“Under the current practices you’d have to get FDA to approve it, it actually reduces or limits the liability of the company providing it, insurance companies have to pay for the standard of care associated with it and any complications because it’s FDA approved,” says DiPersio, “But now, you’re telling me that the companies can actually ask for a payment for an investigational drug for which there is no known cost associated with it, so they could make up a cost.”
Ishmael says there is no way to be sure what drug manufacturers are going to do.
“Do we know the future about what the FDA will do, about what drug companies will do? This is a new innovation in the law, and really it’s a de-regulation where the state is getting out of the way,” says Ishmael.
Ishmael says he has no doubt the legislation will make a difference.
“There is something to be said for hope. There is something to be said for opportunity,” says Ishmael, “and I think the patients who are in a terminal situation deserve the hope and the opportunity of making sure that the state is not going to stand in their way when they seek this sort of experimental medication.”
The law becomes effective August 28.
