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. 2020 Sep 3;113(6):735–741. doi: 10.1093/jnci/djaa137

Table 2.

Areas of uncertainty, misperceptions, and confusion surrounding RTTa

Themes Example quotes
Patients’ rights to investigational drugs Right to Try in my mind says that the patient has the right to try a drug despite it not being maybe rigorously studied…From what it sounds like is that a patient has a right to try. There’s legislation in place. It offers another avenue or some support, advocacy on the patient’s behalf to get a drug that they may not be able to get otherwise. (Participant 1)
The Right to Try is more extensive [than EAP], in my opinion. Basically, a patient wants everything, correct? They can request anything they want. I don’t know if I would support [that] they want any experimental medication. (Participant 3)
Role of drug companies I understand there is some legislation, but I’m not sure whether that legislation trumps—whether or not the drug company’s agreeable to allowing a patient to try it. (Participant 1)
…The name sounds like the company would be forced to share the drug. Right to try sounds like that to me, the patient and the physician agrees that the company has to share the drug, which is not true. (Participant 16)
Differences between EAP and RTT I think there’s one little brain cell that’s saying it’s almost more of a—that there’s some preliminary data or maybe small, low-level study data—those drugs are more fast—I don't know—maybe more fast-tracked through? (Participant 2)
Then right-to-try comes along, and I’m like, ‘Well, that’s compassionate use. We’ve been doing this for a long time.’ Why is it different now other than of course legislation, but now that gets a new name? It’s like, ‘Okay, is that different than what we’ve already been doing?’ (Participant 10)
IRB involvement Sure, what I understood it to be back when I spoke to these folks is that even if a drug or a therapy is not approved by the FDA, a patient is allowed to obtain it if they have been informed and consent to obtaining it if it is I guess something that both the patient and the physician agree is a good idea. I believe the local IRB also has to agree that it’s a good idea. (Participant 13)
I think there’s still a requirement from the IRB protocol. (Participant 17)
Eligibility of drugs Of course Expanded Access would be something where we know there has been record of patient probably benefiting. We know the side effects, and we know what kind of cancers may benefit. With the right-to-try, that becomes a little murky because I wouldn't be able to tell if this patient's population [would be] represented in that particular study of the drug of interest. (Participant 4)
Well, I would think it would just mean if there’s something that’s potentially beneficial that patients can have a right-to-try it, I guess. I guess I don’t know how that would play out in oncology because if something’s truly unproven—I feel like in order to invoke that there would have to be some background data or reason to make you think that it’s going to work…Because if you look at phase I studies which is basically that, the response rate is less than 10 percent, so I feel like, is that appropriate use of resources and cost? (Participant 19)
Unclear rationale for RTT legislation I'm dubious about this new right-to-try law. I think it is showmanship, or I think it's PR, and so…to the extent that I understand it, which is poorly, I'm a little skeptical. (Participant 6)
[In order to share additional thoughts], I’d have to know more about why there’s 2 separate pathways rather than just eating away hurdles. (Participant 17)
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EAP = Expanded Access Program; IRB = institutional review board; RTT = federal Right-to-Try Act.