|
Scientific Rationale and Evidence of Benefit
|
| I have another patient where she was on hospice. There was a drug and I was able to get the drug because it was for a certain targeted mutation. I knew it was a homerun drug. I gave it to the patient. Now we’re a year later and she got out of hospice. She’s alive, doing really well. (Participant 1) |
| Most of the time I’m not looking for something that’s like a nebulous phase-one trial. Usually I’m looking for things that are open in phase two elsewhere that we just don’t have available here, or things that maybe have looked really promising in a phase two or phase three trial but have not been FDA approved, so it’ll be hard for us to push that through insurance. (Participant 11) |
| I do research on [NAMED CLASS OF DRUG], so I knew that this is a very powerful drug and could be life-changing for this patient. It wasn’t a drug—at the time, it wasn’t a drug that might give you maybe one or two months of longer life. This was a major drug that could’ve extended life by a year or more in clinical trials. It was a potentially life-changing drug—no way to get it to patients at that time unless it was on a clinical trial, so that’s the reason why I went through the effort. (Participant 12) |
|
Safety and Functional Status
|
| I thought about it and I really thought about it. I did not think it was ethical. I thought that it was not—the safety wasn’t established. The patient’s clinical status was really poor. I would never have considered giving her therapy if we weren’t already going down this road. Ultimately, I decided to cancel the medication. (Participant 1) |
| I didn’t do it because that patient was advanced, a poor performance status, would not be able to tolerate, his lab studies were abnormal, and so that was never an option for that patient. (Participant 15) |
| The other patient, the drug is part of a class of drugs that’s been under investigation for a couple of years now and have shown a number of results already, and have class of effects that have been mirrored across different agents so that the clinical side effect profile is pretty well known, and it’s pretty well tolerated, so she wasn’t hesitant to go ahead with it. (Participant 8) |
|
Rare Disease and Lack of Options
|
| I think in those situations I’ve told the patient I will reach out to the company and see if I can first and foremost always look for clinical trials first. Th[e] first priority is to gain access to these meds through clinical trials. In the most recent case the patient actually screen failed for a really stupid reason, and so that’s why I reached out to the company to say, “Can we treat her off study?” (Participant 10) |
| These patients that I referred to have extremely rare diseases and extremely poor survival, and the options that we have on the market, none of them work great for them. (Participant 16) |
| Both patients have [name of tumor type], which doesn’t really have a lot of other options from a treatment standpoint, so I think the less options you have from a cancer treatment perspective, the more willing you are to go out on a limb and try other stuff. (Participant 8) |
|
Patient Motivation
|
| It’s not something I typically do unless I really think that there’s a strong reason to do it or a patient’s really pushing for it. (Participant 17) |
| Again, it also depends on what the patient’s experience has been with previous treatments. If they felt miserable and not having quality of life, then they may not wanna do anything more. If they felt good with whatever treatments they had, they wanna do more, so I think it just depends on the patient’s experience. (Participant 4) |
| Well, there’re several calculations that go into this. One is what’s the likely potential for benefit. The second one is what’s the experience with the drug and that is, is it reasonably tolerated. The third is what’s the availability to get the drug or a reasonable surrogate outside of this mechanism. Then what’s the patient think? There are some people that are in go mode and it’s like, ‘Yeah. Even though it’s not on the market yet, yeah, I really want it.’ All those things weigh into the calculation, I think. (Participant 21) |
