How Can Financial Barriers to Patient Participation in Clinical Trials Be Addressed?

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Welcome to this ASCO in Action Podcast. This is ASCO's monthly podcast series where we explore policy and practice issues that impact on oncologists, the entire cancer care delivery team, and the individuals we care for, people with cancer. My name is Clifford Hudis, and I'm the CEO of ASCO, as well as the host of the ASCO in Action Podcast series.

For today's podcast, I'm really delighted to have Dr. Beverly Moy joining us. Dr. Moy is a medical oncologist at the Massachusetts General Hospital who specializes in breast cancer care. She's also an Associate Professor of Medicine at the Harvard Medical School and a longstanding and dedicated ASCO volunteer. She led the ASCO roundtable on addressing financial barriers to clinical trials, and she was one of the authors ASCO's policy statement providing recommendations to address this important issue. So our conversation today will focus on the existing financial barriers to patient participation in clinical trials and how policymakers, trial sponsors, institutional review boards, and other stakeholders can help remove and overcome those barriers Dr. Moy, welcome and thank you for joining me today.

I'm so glad to be here.

By way of background for our audience, only a small percentage of patients with cancer ever participate in clinical research. And yet, we know that certain groups, especially people from low socioeconomic status are underrepresented even in those trials. To give a little bit of shape to that, no more than 3% or 4% of adults with solid tumors participate in research studies. And even then, that number over-represents certain higher socioeconomic groups and underrepresents others. Can you tell us why this issue is important to be addressed, and, furthermore, how we might improve the diversity of clinical trial participation?

So I think it's incredibly important that to improve clinical trial participation among underserved groups. When I think about how we can provide the highest quality cancer care, we really can deliver three basic things-- the best possible standard cancer therapy, the best possible supportive and palliative care, and the best possible novel or experimental therapy. So we know that clinical trials are crucial to the advancement in cancer care. And in the current genomic era, sometimes these trials may even represent the best possible treatment option for some of our patients with cancer. We also know from the medical literature that patients who participate in clinical trials tend to do better and sometimes even live longer.

So when access to the best possible experimental therapy is threatened, optimal cancer care becomes impossible due to financial barriers. And this is an example of social injustice, where poor or underserved patients are being deprived of quality cancer care. So I think that improving the diversity of clinical trial participants is also critically important.

When we determine a new treatment's effectiveness, the cancer clinical trial participants really should reflect the general population of patients with cancer. So as you already said, just a small percentage-- no more than 5%-- of all adult cancer patients in the United States participate in clinical trials, most of whom are white and are from a higher socioeconomic class. That means that the results of our clinical trials are less generalizable, and we need to do better.

So I think that's a longstanding issue, and I know that many listeners are appreciative the efforts to address it. But it raises a question about the underlying reason for this. And one of them, I think, is financial, that is financial barriers to participation, financial barriers to care, and what is now called financial toxicity. As an aside, we recently conducted a national cancer opinion survey-- we do this every year-- and one of the striking observations this year was that 57% of Americans say that if they received a cancer diagnosis, they would be most concerned about financial impact or paying for treatment. And that was compared to 54% who said they'd be most concerned about dying or suffering from the diagnosis. I think it's a remarkable statement that at least a large proportion of people think first and most profoundly about the financial implications of a cancer diagnosis as think about the health consequences.

So if we think about that and then turn back to the clinical trial situation, it is, I think, true that most clinical trial participants will have to face even more additional costs that may prevent them from participating in trials. Can you provide us with an overview of what some of those additional costs are that participants face when they consider a clinical research setting?

So Cliff, I think you're absolutely right and. You know, you're talking about the general financial burden that any cancer patient faces is incredibly high. And I actually would reference listeners to listen to your podcast that you did after the ASCO Quality Symposium, where you actually talked about a few studies looking at really the high rates of financial burden that patients just generally diagnosed with cancer faces.

When you add the complexity of participating in a clinical trial, not only do you have that general financial burden that a cancer patient has, but you add additional potential financial costs that become prohibitive for our poor and underserved patients. So I think of these additional costs related to clinical trials falling into really two basic categories. One is gaps in insurance coverage, and then the second is medical out-of-pocket costs.

So picking the gaps in insurance coverage policies category, we know the Affordable Care Act was passed in 2010, and it does require coverage of routine health care costs for patients participating in clinical trials. But these protections do not extend to patients with Medicare or Medicaid. It's only for patients with private insurers, and there are even restrictions there, which we can go into later. That means our poorer or older patients are more vulnerable to not having these protections.

These potential costs could consist of things like investigational care costs, such as this specific therapeutic drug under investigation, or more likely the cost of additional services that would not have been required if the patient was receiving standard therapy. These services could be things like extra blood draws for safety data or imaging studies that fall outside of the routine staging exams. So these extra services have the danger of either being billed to the patient or forcing the patient to pay more towards their insurance premium due to policies that increase cost sharing to patients in the modern era.

A second important cost category, which is just as important, if not more important, would consist of non-medical out-of-pocket costs resulting from clinical trial participation. So these costs include travel and lodging expenses, or costs due to lost wages because they have clinical trial visits, or the need to pay child care because they have to come into the hospital or the cancer center more often. So patients participating in clinical trials often have far more frequent visits than standard therapy, perhaps at centers that are farther away from their home. If they participate in early phase first in human trials, they could require longer days of things like pharmacokinetic testing that might even require overnight stays in hotels. So these out-of-pocket costs can be considerable, and prohibitive, and make it impossible for poorer patients to enroll into clinical trials.
I know that-- and I'm sure many of our listeners know that-- you're actually a day-to-day clinician. You see breast cancer patients, as well as do research at MGH. And I'm interested in the practical ways that you might have seen these costs directly affect your patients. Do patients ask about trial costs when you're talking to studies with them, talking about studies with them? Have you ever had patients hear about a study and then declined to enroll because they thought it would be too expensive?
So I think that's a really interesting question, Cliff. And I think, in the past, when we've presented clinical trials to patients, the idea of costs never really came up maybe because it was something that patients didn't feel comfortable bringing up to their physicians. But now, either costs are becoming more considerable, or it's being raised into awareness that I think that patients are much more comfortable talking about it, maybe not at first, but maybe towards the end of the process.

So in my own practice, I've seen people bringing it up more, and more, and more, usually even during the consenting procedure when you're talking about all the extra visits and studies that the patient may need to have to undergo the clinical trial. I've also seen patients who've actually enrolled in trials, and they're on study, and they consider even coming off trial because they didn't anticipate some of the costs that might happen.

So, for example, recently, I treated a young woman with high risk breast cancer on an adjuvant CDK4/6 inhibitor trial. And this trial requires more frequent medical visits and blood testing because of potential toxicities. And she found that the extra co-pays and financial costs toward her insurance deductible were completely prohibitive. And despite conversations, she actually prematurely dropped out of the trial after only about nine months of therapy. And this is a single mother of two young kids. That's really not acceptable that a financial reason was the reason why she couldn't continue on the trial.

So I think patients are asking more and more for financial guarantees even before signing consent for clinical trial screening, and I'm afraid we really can't provide financial guarantees because that's really not possible for us to do.

So one of the reasons that we bring together volunteer groups like you've participated in and then publish results at ASCO is not merely to identify, name, and describe the problem, but in fact to offer solutions or at least potential solutions. So I want to turn and talk a little bit about the recommendations that you've made in the policy statement and through your work.

The most recent policy statement on financial barriers to clinical trial participation focused, as I understand, on three key objectives. One was transparency in terms of clinical trial costs and these gaps in coverage that you've been describing. The second is reducing concerns about inducement, that is making sure that whatever we do does itself in an unreasonable way lead to the perception or reality of induction onto the trial. And finally, improving data in the course of participation in trials. That is studying this scientifically like we do other aspects of care.

So let's start with the first objective, transparency. What are some of the ways that lack of transparency has affected participation in trials? And how can clinical trial sponsors, or sites, or investigators help us address the issue of transparency?

So I think that transparency is a very, very critical issue here. And one of the potential problems is that lack of transparency about who is responsible for the specific costs of clinical trial participation can lead to uncertainty from the patient's part about what he or she might be responsible for paying in the end. So often, insurance payers and research sponsors, whether it be industry or other, they might disagree about who is responsible for which costs. They might argue about what really constitute truly safety assessments, what's really routine costs, so generally the research sponsor covers the costs of additional or more frequent services that the insurance payer may disagree about what services are truly additional or more frequent. You know, what is really standard?

So if a health plan denies coverage for the entire trial or individual services within a trial the sponsor considers routine, this could be problematic. So without transparency or protections the patients run the risk of being billed directly for these services. So these costs need to be addressed specifically so that the financial burden doesn't land on the patients in the end.

One of the reasons for the costs being a barrier, of course, is that clinical research is often very expensive, and at least some of the recommendations focused on clinical trial design is a way to reduce costs. What changes do you think sponsors might make to clinical trials that could directly bring down the costs for patients? And I would add, especially with regard to those traditionally facing greater barriers, the populations we're talking about today.

So clinical trial design is really important because we have to be very cognizant that what this means to the patient and how we're burdening them. So clinical trials really need to be more aware of the financial implications of their study design. So, for example, excessive follow up medical visits, or additional laboratory draws, throwing on more imaging studies, or other procedures that are not absolutely critical to the study really ought to be eliminated because these costs add up. And if they're not critical, they really shouldn't be done. There are costs associated with every test or visit performed. So we really need to reduce the excessiveness of what's required of patients on clinical trials.

So in addition to that-- and I mentioned this earlier-- one of the concerns on the other side is about what happens ethically if we pay patients directly to defray these out of pocket costs? This is referred to as an inducement. And around clinical trials, there's really a high degree of, I think, appropriate concern for inducing patients on to studies. What are some of the concerns? Do you think that they're well-founded or overblown? Is there anything that we can do to remove or address those concerns while defraying the course of participation?

So I could talk about the ethics about this issue all day long, but I'll try not to. But I think the theoretical concern that I've heard often is that financial compensation or reimbursement of clinical trial expenses could represent a form of inducement or coercion to enter into a clinical trial. And this probably stems from FDA and OHRP regulations that clinical trialists should minimize the possibility of coercion or undue influence on patients. There may also be a potential hurdle from CMS that the Social Security Act specifies criminal and civil penalties for offering financial remuneration to a Medicare or Medicaid beneficiary that influences the selection of their medical provider.

So I'm not a lawyer, but I think these concerns are fairly ludicrous because first, coercion, as a principle, is completely irrelevant here. From an ethical perspective, coercion involves a threat that makes a certain choice irresistible, and that is not relevant in the case of cancer clinical trials. Undue inducement is also irrelevant because we have multiple examples in the medical literature that large payments do not disproportionately affect patients' willingness to do medical tasks, for example even donating a kidney.

There was actually even a recent article published in JAMA Oncology, written by some bioethicists from UPenn, that argues that the worry that offering inducements to participate in research is inherently wrong. The authors even go so far as to discuss paying patients for cancer clinical trial participation to make participation more attractive to a wider population of patients. And that's really important for both social justice and trial completion issues.
So the idea of paying patients for trial participation was something that was brought up even by the patient advocates who participated at our ASCO roundtable on this subject. The patients thought, if we're doing this to advance science, shouldn't we be paid for it? So to remove these concerns, ASCO's partner in our roundtable, the Lazarex Cancer Foundation who helps fund our roundtable, they worked with the state of California to sign into law identifying trial-related expenses to be reimbursed and are currently working with several other states to do the same, such as Pennsylvania and even my home state of Massachusetts, and Texas, Florida, Ohio.
But I do believe that we need federal regulations to remove the specter of inducements and coercion out of this field because it simply doesn't belong. So I guess that's my long-winded way of saying that ethical concerns about paying patients for out-of-pocket costs associated with trial participation are completely overblown.
I see.

Sorry if I went on for a while, but I feel pretty strongly about that.
I was going to say, do you have an opinion on the matter? But one of the other areas called out in the paper relates to the economic burden on trial participants versus non-trial recipients and says that this economic burden data is more than 20 years old, that is it's not modern. And so it recommends that organizations should, therefore, pivot and support the building of an evidence base, research, on the true costs of patient participation trials now in the current era. So what exactly is the kind of data that we need and, how would better data, in turn, allow us to reduce barriers to participation?
So I think that, like all research and data issues associated with cancer equity in general, we need more data about effective interventions that reduce the financial and economic burden of clinical trial participation. We also need data in the modern health care era about how burdened our clinical trial participants really are. This data is starting to come in from various single centers across the United States, but we really need a concerted, comprehensive, and collaborative effort to examine this important issue nationwide. But mainly, I do think we need research about interventions that work to help our poor underserved patients enroll into cancer clinical trials.

So with all of this said, what do you think ASCO's next steps on this issue should be? What do you propose? Or what does your group recommend that we do in a concrete way next?

So I think, first, I want to say that historically I've been extremely proud to have worked with ASCO because ASCO's really been a leader in improving access to care for all patients with cancer. And given ASCO's leadership in the oncology community, ASCO's really in the unique position of being able to convene this roundtable that led to this policy statement. And there are multiple stakeholders at this roundtable, including researchers, clinicians, industry, insurance payers, the NCI, FDA, Biden, Moonshot, ethicists, patient advocates, you name it. And ASCO, as a leading clinical cancer society, really can push this issue forward based on its leadership here.

I think ASCO ought to demand change through federal regulatory policy fixes, and disseminate, and possibly even fund relevant research that we just described earlier today. I think what we're doing today with this podcast is that we're increasing awareness of this issue, which is also something that ASCO is doing, and no one can do better. I would want listeners to become really more aware of this issue.

The most underserved patients the United States are being deprived of one of the most important types of cancer treatment options. This is a social injustice that absolutely needs to be corrected, and we need the oncology community to be united in solving this problem.

And that's a really great summary of, I think, the motive and the ethical drive underneath our work in this. Is there anything else you'd like listeners to know about the ASCO recommendations? Are there any parts of this that we've skipped over or failed to mention?
No, I think that the recommendations are kind of a multi-pronged group of recommendations to try to attack this problem, and it's really a first strike in this really important issue. But I think that what our listeners really should understand is that no patients should be denied access to a clinical trial for financial reasons. And no patients should be harmed financially because of their contributions to the advancement of science. So if we're united in this belief, then we really can move forward together.

Well, I love the way you've wrapped that up. And I want to thank Dr. Moy for joining me today for this ASCO in Action Podcast. I want to remind everybody that at ASCO, as you've heard, we are committed to preserving and enhancing access to high-quality cancer care for all individuals with cancer. Our statement on financial barriers to clinical trial participation is just one of many, where ASCO's voice and the collective voice of our members, we hope is helping to share and shape the future of the cancer care delivery system.

I encourage our listeners to read this statement, as well as our other policy and position statements. They're all available on the policy and advocacy pages of our website at asco.org and, in this case, through the JCO. Until next time. I want to thank everyone for listening to this ASCO in Action Podcast and thank Dr. Moy for joining us today.