he newly enacted right-to-try law allows drug makers to earn a profit by selling unproven therapies to desperate and dying patients. The FDA is powerless to stop it.
You’d think no drug maker would be dumb enough to actually try to make money this way, given prevailing public opinion that already views the drug industry as greedy, price-gouging profiteers.
But you’d be wrong. Enter BrainStorm Cell Therapeutics, which intends to start a “semi-commercial enterprise with modest profits” that would sell its experimental stem cell therapy to patients with amyotrophic lateral sclerosis, or ALS, according to a story from Bloomberg.
BrainStorm’s treatment, called NurOwn, is a bespoke therapy made from stem cells harvested from each patient. BrainStorm CEO Chaim Lebovits told Bloomberg he’s considering charging $300,000 for each NurOwn treatment offered through a new pathway set up as a result of the right-to-try law.
“We have to have an incentive,” he said.
To be fair, BrainStorm is trying to develop NurOwn in the traditional way. The therapy is currently being vetted in a large Phase 3 clinical trial. Lebovits said the company only considered offering NurOwn through right-to-try because of intense demand from ALS patients who have no other treatments available.
But the precedent that BrainStorm will set by turning what should be an altruistic act into a money-making venture is extremely dangerous and potentially exploitive. Unscrupulous drug companies will be incentivized to skip the traditional clinical trial and FDA review process altogether. Drugs no better than snake oil — but deemed safe — could be sold to desperate patients at a profit. The FDA would have no power to stop it from happening.
Under the agency’s preexisting compassionate use program, the FDA had oversight of eligible treatments and companies could only recoup their costs, not make money.
There’s little evidence that ALS patients, especially those with advanced disease, will benefit from NurOwn. In BrainStorm’s Phase 2 study, NurOwn was unable to slow the progression of ALS compared with a placebo. The company turned to a responder analysis to eke out a signal of efficacy, which it’s now trying to confirm in the Phase 3 study.
The idea that BrainStorm could make a profit from NurOwn before the treatment is proven effective and approved by the FDA is a bad look for the company and the entire pharmaceutical industry. It reeks of opportunism, even when couched in compassionate rhetoric.
Right-to-try should not be right-to-die-poorer, but that’s what the law will end up being for patients if profit motive takes hold.