Industry Voices—Right to Try law provides a license to cash in on patient fears

Scott Gottlieb FDA
FDA Commissioner Scott Gottlieb has made assurances his agency will protect patients while honoring the rules of the right to try legislation passed earlier this year. However, there are reasons for concern. (FDA)

More than 1.7 million Americans will be diagnosed with cancer this year. Almost one in three of those patients will eventually succumb to it.

Knowing these odds, patients are eager to try anything to improve on them. They and their loved ones want quicker access to the latest medicineseven 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.  

Conference

2019 Drug Pricing and Reimbursement Stakeholder Summit

Given federal and state pricing requirements arising, press releases from industry leading pharma companies, and the new Drug Transparency Act, it is important to stay ahead of news headlines and anticipated requirements in order to hit company profit targets, maintain value to patients and promote strong, multi-beneficial relationships with manufacturers, providers, payers, and all other stakeholders within the pricing landscape. This conference will provide a platform to encourage a dialogue among such stakeholders in the pricing and reimbursement space so that they can receive a current state of the union regarding regulatory changes while providing actionable insights in anticipation of the future.

RELATED: Gottlieb: Agency will adequately protect patients following passage of 'right to try'

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 but inexorably 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.

RELATED: Trump signs Right to Try into law

A midstage study involving 48 patients found those given the drug did appear to respond, though the benefit didn't last for most. It's highly uncertain whether NurOwn will eventually pass the FDA's scientifically rigorous safety and efficacy standards.

Nevertheless, NurOwn'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 hundreds of thousands of dollars

Insurance doesn't generally cover treatments that haven't been approved by regulators or proven to work in clinical trials. That means patients would have to pay for the therapies out of their own pocket. Some could be desperate enough to sell the house to come up with the money.

The 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%. 

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. 

RELATED: BrainStorm mulls making ‘modest profits’ from providing unproven cell therapy on a right-to-try basis

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.

No wonder the American Society of Clinical Oncology opposes right-to-try legislation on both the state and federal levels.

"ASCO supports access to investigational drugs outside of clinical trials, when adequate patient protections are in place," ASCO chief medical officer Richard Schilsky, MD, said. "We don't support right-to-try legislation, however, because these laws ignore key patient protections without actually improving patient access to investigational drugs outside of clinical trials." 

Let's hope BrainStormand other likeminded companieshave 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.

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