Right to Try Provides a License to Cash In on Patient Fears
By Peter J. Pitts
More than 1.7 million Americans will be diagnosed with cancer this year. One in three of those patients will eventually succumb to it.
Knowing these odds, patients and their loved ones want quicker access to the latest medicines—even if they are still at an experimental stage.
That's why so many Americans applauded passage of the "Right To Try Act," which was signed by President Trump in May. The measure creates a legal pathway for terminally ill patients to access new medications that have only been through one phase of Food and Drug Administration approval.
While well-intentioned, the law could create opportunities for snake-oil salesmen to prey on those who are vulnerable. As the Food and Drug Administration develops its protocols for right-to-try, the agency must ensure that patients are protected from anyone trying to game the system.
Consider the case of BrainStorm Cell Therapeutics, a drug company with a new experimental stem cell treatment called NurOwn. The treatment is for ALS, the terrifying disease to which baseball superstar Lou Gehrig succumbed. Those suffering from the neurogenerative disorder gradually lose all ability to control muscle movements. Eventually patients are completely isolated in their immobile bodies -- typically until their breathing stops.
NurOwn is harvested from the stem cells of each patient. It's currently under development, and results to date have been inconclusive.
A study involving 48 patients found those given the drug did appear to respond, though the benefit didn't last. It's highly uncertain whether NurOwn will eventually pass the FDA's rigorous safety and efficacy standards.
Nevertheless, NorOwn's developer sought to make it available to patients. But here's the catch: "Right to try" doesn't mean "right to try for free." And that's where BrainStorm Cell Therapeutics had its own brainstorm, proposing to sell its experimental treatment to patients for a cool $300,000.
Insurance doesn't generally cover treatments that haven't been approved by regulators. That means patients would have to pay for the therapies out of pocket.
Right-to-try legislation isn't designed for commercial profit. Yet after Trump signed right-to-try, requests for NurOwn skyrocketed, and BrainStorm's shares closed up 2.8 percent.
No one actually knows if NurOwn works. But desperately ill patients may gamble everything to try it anyway. Such are the unintended consequences of right-to-try.
BrainStorm ultimately bailed out on offering NurOwn under right-to-try, but only after a maelstrom of controversy. Yet the company's initial impulse was likely only the first of many potentially unsafe and exploitative actions, which the FDA must act to prevent.
Let's hope BrainStorm -- and other likeminded companies -- have learned that cashing in on the uncertain hopes of desperate patients is an unacceptable industry practice. And let's make sure the FDA sends that message in no uncertain terms.
Peter J. Pitts, a former FDA associate commissioner, is president of the Center for Medicine in the Public Interest.
