Table 2. Summary of Key Themes and Representative Quotes.
| Theme | Summary | Representative quotea |
|---|---|---|
| EA: a means to an end | Oncologists at academic centers view EA instrumentally, as just another option for obtaining what they view as the best course of treatment for their patients. | “I want the best for my patients, and whatever, in this case, drug, is necessary to do that, I want to get. And as we’ve said, I click through options and [if] this rises to the top, you need to get it before it qualifies, if possible.” (A01) |
| “The way that I think about expanded access, I don’t think about it as research. It’s an access mechanism. Is there a drug you think is going to help somebody? How do you get access to it? And if this is the only access mechanism, then I would consider it.” (A07) | ||
| “It’s simple. If there is an agent that will help your patient, you are obligated to provide a mechanism to get that patient that agent.” (A14) | ||
| “I think our role is to give the patient what we think is going to be the most effective and least toxic therapy and to get that therapy for them from whatever route is necessary. And so if that’s through a product that’s not yet been approved, then that’s still my role.” (P10) | ||
| “And so, for me, expanded access is just another tool to convey to families that we want to do everything we can to care for the patient and to care for them. And it’s just another way to care for them. And sometimes it’s the right way, and sometimes it’s not going to be helpful.” (P07) | ||
| Making treatment decisions: the centrality of data | When considering treatment options, academic oncologists rely heavily on data. In the face of uncertainty, they prefer recommending that patients enroll in available trials. | “Physicians have an obligation to carefully consider whether giving an unapproved drug is the right thing. I know that people get compassionate use for patients where there’s a clinical trial available because the clinical trial randomizes the drug vs placebo. I have never offered a patient compassionate use in that setting because I feel like you have to answer the question. If we knew the right answer, everybody would have access to the drug.” (A02) |
| EA may not be a last resort, if data suggest that an investigational product is the best option for a given patient. | “I’ll say that it’s not necessarily only in people who have exhausted their standard options. If there’s somebody with a target where I think the targeted therapy is better than standard therapy, I would consider a single-patient IND before their standards are exhausted.” (A15) | |
| Academic oncologists express strong confidence in their ability to parse data to decide on the most beneficial therapy, independent of FDA approval status. | “I really am pretty much of a stickler that FDA approval is something that’s given to the drug companies to market a drug. It has nothing to do with whether it’s given to a physician to prescribe the drug, right? So we use a compendium of public literature [and] phase 2 and 3 trials to decide whether to treat somebody with something as a marker of efficacy. But, I always tell my trainees that the FDA approval is the approval to market for an indication, it’s not approval for physician use… I’m not given approval to use something. I get that from my MD.” (A14) | |
| Academic oncologists still value FDA approval, even as they pursue EA. | “The other reasons why we may pursue expanded access is primarily the strength of the emerging data that’s out there, conference abstract presentations, which is really the main source of data for agents that are not approved yet, or if the paper’s been published.” (A08) | |
| “Since at [institution] we’re obviously involved in some of the early trials with the key drugs, we tend to know about these, many patients who we think would benefit from these drugs based on their mutational profile or their disease. If they’re between a phase 1 trial and a phase 3 approval trial, then the only way to get the drug is through compassionate use, and that’s when I’ll bring it up.” (A12) | ||
| “If I think that something sounds like an interesting therapeutic option for a patient and I am aware the data suggests that there’s a reasonable risk-benefit and I can get it, which is the hard part… So how long do they have to wait is driven entirely by data. New mechanism of action. Some evidence in kids that it works. … I think it’s more data-driven than time or where it is in the clinical process.” (P02) | ||
| “I think the approval process is important to define safety, and I think that it’s important to define efficacy, and I think that we should not be using unapproved drugs widely without understanding what the risks and benefits are of those drugs.” (A07) | ||
| “I think one of the more obvious logistical reasons [that FDA approval is needed] is that there are just not enough resources in the world to go through this process for each patient every time that you’re using a drug. And so the way to streamline that is to get a blanket approval for the drug, so that we can use it in all patients.” (P04) | ||
| Deciding to pursue EA: weighing risks, benefits, and burdens | When considering EA, oncologists recognize that the benefits of investigational medical products are uncertain, but they will not pursue EA without some reason to expect benefit and will refrain when the risks of harm are substantial. | “We’ve seen again and again drugs that look good in phase 2 trials, [but] in phase 3 trials either the efficacy didn’t look so good or there were some terrible unexpected toxicities… It is the nature of most to overestimate benefits and underestimate harms from investigational compounds.” (A02) |
| Oncologists treating pediatric patients were more likely to consider pursuing EA even with low likelihood of benefit, in part to support parental desire to “leave no stone unturned.” | “I would never go through this process for the sake of just having something to give [the patient]. It really has to be pretty strong evidence that it’s likely to work, or at least has, yeah, or at least has a good chance to work.” (A12) | |
| “And when the risk of doing actual harm is high, it’s not something that I can recommend to an individual patient.” (P08) | ||
| “In pediatrics, I think a lot about parents who are going to survive their children, and they need to live the rest of their lives and be comfortable with the decisions they made around their child’s death. And so I tend to be a little more liberal with what I offer and allow at the end of a child’s life, to make sure that parents don’t have regrets or guilt, or have as little as possible.” (P09) | ||
| EA within the physician-patient relationship | Oncologists report that discussions about EA are typically physician-initiated. | “I’ve had it happen extremely rarely that patients are asking me about drugs if I’m not bringing it up. I’m the initiator here, so I’m not even going to talk about compassionate use unless I think it’s valuable.” (A02) |
| In patient discussions about EA, strong disagreements rarely occur and are usually surmountable with attention to clear explanation and demonstrated support. | “Those discussions are often very hard, but often, with a lot of discussion, families, most families, will understand that causing side effects could end their child’s life sooner than would otherwise have happened.” (P08) | |
| When disagreements persist, patients may seek EA through a different physician. | “I think that patients and their families are afraid of dying, and they’re afraid of being abandoned or left alone in that process. And so, I can’t control whether they live or die, but I have more control over how I position myself and people with whom I work in terms of supporting families so that they are less likely to feel that they’re going to be abandoned or that they’ll be alone in this process.” (P07) | |
| “I do not feel that I ever have any obligation to do compassionate use even if the patient wants it, and they can always find a different physician if I do not feel that it’s in their best interest to do so, that there’s other doctors that might do it for them, but I don’t have to.” (A02) | ||
| “I have had families say that they would like to switch providers to see if they could find a provider who would do something compassionately or on expanded access.” (P08) | ||
| Inequities in access | Oncologists acknowledge structural inequities in accessing the EA pathway. | “There are also equity issues just in terms of where people are seen, because the patients I’ve applied for have been because I have known of some drug or whatever, because I’m an investigator on various trials. So, because they’re seen in an academic center, their doctor has knowledge of some things that other people may not.” (A02) |
| Oncologists generally report the provision of unbiased access to EA for their own patients. | “Because a patient at some level does have to advocate a little bit for him or herself and that is just going to be more possible, greater likelihood of that with higher education, higher socioeconomic status, a better network, a better connection, better connections to everything. I really don’t know what to do about that.” (A03) | |
| “I would hopefully think I would to be as likely to bring it up regardless of the gender, ethnic, racial, socioeconomic issues with the patient. Obviously, I’m a human being, and that’s probably not completely true.” (A12) | ||
| “I don’t think there would be any issue of fairness because we as the provider would be the one who would have to do the process and there’s no financial commitment from the patient whatsoever. There might be a difference in which patients and families are pushing for it based on their education level and their awareness, but ultimately, it’s our responsibility to say yes or no and pursue it.” (A06) | ||
| “If a family comes and asked me about a drug, they have as much chance of getting that drug as anyone else, regardless of the resources on their part.” (P01) | ||
| “I think in a micro-environment, when I think about my own little practice, expanded access... everybody’s treated the same. The protocols you write, they’re just like treating people with anything else.” (A07) |
Abbreviations: A, adult oncologist; EA, expanded access; FDA, US Food and Drug Administration; IND, investigational new drug application; P, pediatric oncologist.
a
For anonymity, respondents are identified by number and whether they were a pediatric or adult oncologist, without reference to their specific institution.