I recently wrote that the Right-to-Try legislation would inevitably become Right-to-Rob. Although Merck and other pharma incumbents (weakly) opposed the law, it wouldn’t be long until small cash-strapped companies began exploiting it to improve their finances.
That day has arrived. Bloomberg reports that BrainStorm Cell Therapeutics will begin selling its investigational ALS treatment to patients.
Brainstorm has no revenues, net assets of $6M and a burn rate of $5M. Their ALS treatment, NurOwn, is the only product in their pipeline, and they are recruiting for Phase III trials. Think they might be looking for a payday?
According to Bloomberg, the company wants to make a “modest profit” on its RTT patients, at a cost in the neighborhood of $300K per treatment.
In all fairness, NurOwn has made it through Phase II trials, and BrainStorm has reported (but not published) that the treatment is well-tolerated and appears to slow disease progression. $300K is, if anything, on the low side for autologous engineered cell therapy – Kymriah, a CAR-T leukemia therapy, goes for $475K (although it would produce a decent ROI at $160K).
Advocates of RTT claim that providers are allowed to charge for direct costs only. But there is no mention of any cost or profit limitations anywhere in the text of the law. Even if these limitations are incorporated by reference to other laws, there is no mechanism for enforcement. The FDA is specifically forbidden to exercise any oversight. And, of course, BrainStorm’s CEO just came right out and said that they intend to make a profit: “Companies cannot be NGOs. We have to have an incentive.” At least he’s being up front about it.
This case is not egregious. But how about the next one, and the next? RTT extends eligibility to any patient with a life-threatening disease who is under the care of a physician and has exhausted other treatment options. None of theses terms are further defined – and without any enforcement or rule-making mechanisms, patients and doctors will be free to define them as they see fit.
I’m sure advocates of RTT were thinking of diseases like ALS or pancreatic cancer that are uniformly deadly and essentially untreatable. But there are plenty of other diseases that could reasonably meet the broad definitions of the law.
Rheumatoid arthritis, for instance, is well-known to decrease life expectancy and increase the risk of comorbidities like heart disease. RA treatments are not very good and none are curative. What’s to stop a homeopath from running a Phase I trial (a requirement of the law) and then setting up shop? Phase I trials are just about safety and dosing – and since homeopathic remedies contain only magic water, they are undeniably safe. The RTT law does not prohibit advertising treatments or otherwise recruiting patients. Without FDA oversight, quacks are free to make any claims they like, and they can truthfully state that their frauds have been clinically tested.
And treatments that are not outright quackery still pose serious risks. Passing a Phase I trial is no guarantee of safety. About 20% of drugs tested in Phase II and III trials fail due to safety concerns. That’s over and beyond side effects that are considered acceptable – you know, the ones they recite at high speed over visuals of smiling healthy people in drug ads.
RTT fits well with the long-term agenda of the Trumpists – which is to turn our high-trust society into a low-trust one. Power, not norms or decency or common good is all that matters in low-trust societies. Corruption and enfeeblement of institutions is a first step in changing us into a people who trust only those they know – and their Great Leader. That’s the end game here. Enriching quacks and cronies is just a side benefit.