Would you share your thoughts on the New Bill signed the other day by POTUS called RIGHT TO TRY?I wonder how the BTs will answer all the new requests which are End of Life requests so they will NOT fit into current studies which have strict entrance criteria..they will need to come in under some sort of Compassionate use study and who pays for this?Is this a net positive or net negative for BTs and Pharmas?
Right to Try laws have been enacted by most states in the country, in response to what the libertarian Goldwater Institute perceives as a bureaucratic nightmare for patients with terminal illnesses to get access to medication that might save their lives. Many proponents of these laws echo that sentiment. One person I discussed this issue with put it in this colorful way: “If I’m going to die, I don’t care. Strap me up to a car battery and let’s go.”
A national Right to Try Law has generally been turned into yet another political football, one that happens to be carried by the conservative crowd in this case. From the perspective of investing in cancer research, we need to pay attention to these kinds of laws, though. Will they have an impact on your company of choice? This is particularly important now that a national law has been enacted.
What Is Right to Try?
At the national level, this legislation does a few key things. First, it defines who can receive drugs. Second, it defines which drugs qualify. Third, it codifies the protection of companies who provide their medications from liability. This last point is perhaps the most important for wider uptake. Why would a company participate in giving their experimental medications to patients outside of the context of a clinical trial if they could be sued, or if they could be set back with the FDA?
In essence, there are two protections laid out in the bill: nobody along the production chain of the experimental medicine is to be held liable. Furthermore, the FDA cannot use the outcomes from Right to Try usage to delay the review or approval of a drug. This removes at least one crucial impediment to the longstanding practice of Compassionate Use. The issue of liability at this time is not pressing, since nobody has yet sued a drug developer who provided experimental medication.
So who will benefit? The bill prohibits the Federal Government from stopping a patient with a terminal illness from receiving an experimental drug, biological product, or device. Patients are allowed to possess said drug or device as long as a physician in good standing has certified that the patient has exhausted all other options.
Who will pay?
In spirit, I don’t have much trouble with Right to Try laws as they stand. Adults can make their own decisions about care. Furthermore, this legislation appears to respect the doctor/patient relationship. And this communication and respect are the patient’s best chance at achieving better outcomes. I like this.
However, the bill doesn’t really do much to improve access to patients. In the past, companies that wanted to provide their drugs had the opportunity to use expanded access programs or compassionate use. The latter just required approval from the FDA. And you might say that avoiding the bureaucracy is kind of the point here. But let’s not forget that the FDA serves a pivotal role in keeping people safe. When CAR-T cell therapy led to the death of 4 patients in 2016, the FDA acted as gatekeeper to prevent other people from being at risk. This helped prioritize the development of safer CAR-T cell therapies, which have gone on to be approved. The hyped nature of CAR-T cell therapy means that more patients will be aware of them, and they may demand their right to try.
But who will pay for these medications? The bill does not lay that out. Clearly it does not compel insurers, who would not normally reimburse for experimental treatments. It does not compel the company to pay. It won’t be paid for by the government.
Patients are going to be left to foot the bill for these experimental treatments. And I’m not trying to pull your heart strings; I’m just stating that it’s going to severely impair the implementation of Right to Try. In 2017, a pair of scientists from NYU published a paper analyzing the impact of the Right to Try laws that had been passed in 32 states at the time. They critiqued these laws because manufacturers don’t have to participate. And they cited that there was no documented evidence of any patients having received a single medication through Right to Try laws that they could not have gotten through traditional compassionate use overseen by the FDA.
Long story short: this is not going to dramatically expand the use of experimental medications outside of the context of a clinical trial. So our cancer biotechs are not going to see any significant revenue from this. They’re not going to be able to speed up clinical development. Ultimately, this law is not going to have much of an impact on business, and I’m not considering it seriously when evaluating clinical-stage biotech companies.
What is the bill really about?
The author of the national Right to Try law, Ron Johnson, prepared a letter to FDA commissioner Scott Gottlieb criticizing the FDA’s position on the law. This was in response to recent comments Gottlieb had made about the challenges of implementing the bill from the FDA’s perspective.
For me, this is the key quote from Senator Johnson’s letter (emphasis mine):
“This law intends to diminish the FDA’s power over people’s lives, not increase it. It is designed to work within existing FDA regulations, definitions, and approval processes. It is not meant to grant the FDA more power or enable the FDA to write new guidance, rules, or regulations that would limit the ability of an individual facing a life-threatening disease from accessing treatments. Under this law, the FDA’s oversight with respect to patient safety within a Phase I trial remains unchanged; the current thresholds for successful completion of such a trial phase remain in place.”
While there is a thread of altruism written into the law, the senator makes few bones about the law’s true purpose. That is to undermine the FDA’s power.
Frankly, I have a problem with that notion in this political climate. We’re coming out of a tumultuous decade filled with misinformation about “death panels” and debates on the role of government in healthcare. This, I’m afraid, is not the sanest environment to start taking away from one of the more competent agencies in the government. This particular law does not do much against the FDA, but we need to be vigilant. The FDA serves a very important function for the country, particularly with respect to patients with terminal or life-threatening diseases. If we undermine that mandate in the chase for libertarian utopia, we will come to regret it. Such was the case in the 1980s and 1990s when the FDA caved to public pressure to allow wider access to stem cell transplantation before the data were conclusive. A lot of breast cancer patients were unnecessarily harmed, and for no benefit.
I’m all for having a nice debate on the role of the government in healthcare. And I am absolutely for allowing adults to make their own decisions about their bodies. But we should remain cautious about the ideological chase for personal freedom. The FDA is just about the only protection we have in cancer medicine from charlatans and snake oil salesmen. And we should protect this status.
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