Top considerations when determining whether to approve expensive drugs

Weighing the ethics of approving expensive cancer drugs for patients facing terminal disease is a multifaceted and inherently multidisciplinary endeavor.

Managed Healthcare Executive (MHE) recently spoke with ethicist Jacqueline J. Glover, PhD, a professor in the University of Colorado Department of Pediatrics and Center for Bioethics and Humanities, about this issue.

Glover spoke at the AMCP Managed Care & Specialty Pharmacy Annual Meeting, in Denver, during the March 29 session, “Ethical Dimensions of Approving Expensive Drugs for Patients with Terminal Cancer.”

MHE: What are some of the ethical considerations pharmacies must weigh when it comes to approving expensive drugs (adding them to institutional formularies) for patients diagnosed with terminal cancers? 

GloverGlover: Firstly, good ethics starts with good facts. When you are weighing the benefit and burdens to see whether it is worth the increased burden of increased cost, it totally depends on the quality of the data. There can be problems with the data. Overall survival-the time from drug assignment to death from any cause-is meaningful to patients. But people who fund trials are interested in other things too; they want a shorter trial duration and to minimize the number of participants, enabling rapid access to the market. So many trials use surrogate endpoints: overall response rate of the tumor, early tumor shrinkage, progression-free survival (the time from drug assignment to progressive disease or death from any cause). These things don’t necessarily correlate with overall survival. 

Also the impact on quality of life is very unclear and when there are studies about increased survival, the range is often two to three months, not years. It is a very good question, how these clinical trials really compare to the real world, the general population in the “real world.”

A second example is how to weigh how much benefit is worth the burdens. In our individualist society where respect for individual autonomy is very important, some would want to say that that determination is always left to the patient to decide for himself or herself. But our clinicians do not usually talk about costs as a matter of informed consent-here are the potential benefits, here are the risks-and left off is how much it is going to cost you and your family-or mostly probably, your estate, after you die. They don’t normally talk about cost and they don’t really know how to do that.

A third consideration is the role of the clinician as a professional. They shouldn’t consider cost, right? Clinicians should be the kind of gatekeepers that only keep the gate open. Only consider the needs of one patient at a time, the patient before them.

But I don’t think that’s true. Clinicians have obligations of justice. All professional codes of ethics include something about obligations of justice.

And uncontrolled spending by some puts at risk availability and access for others. Clinicians have obligations of justice because we all do as members of families, communities and nations.

I realize that human needs are limitless but our resources to address them are limited. So that makes cost an ethical issue: how best to use our limited resources for both the individual and what we call the common good.

A fourth ethical consideration is the value that we place on innovation and pushing limits. Our cancer centers are excellent and they participate in cutting edge research. We want our cancer centers to have access to new drugs for our patients. We use war metaphors-we are battling cancer and we want to win-and we don’t want to limit the weapons available to our warriors.

So ethical considerations include good data, how to weigh it, the role of clinicians in talking about costs, and intentions to benefit others.

Next: What about drug-cost transparency?

MHE: What about drug-cost transparency?

Glover: The authors of a recent Nature Reviews paper talk about transparency and what goes into drug pricing. They would dispute the R&D costs explanation for drug prices. They go into why on earth do we forbid Medicare from negotiating on drug prices.

In balancing costs, people think the benefits are all on the side of receiving the drug. But that is not true. There are burdens associated in terms of side-effects and cost. Cost is certainly a burden. Increased costs are harmful to patients. You will not likely be as compliant-you won’t start taking a drug right away. So high cost is related to patient harm in not taking the drug as prescribed. They’re unlikely to start when they should and to be compliant. They drop off.

There are substantial individual harms from high cost; not least is bankruptcy. Soon, the rises in cost will be unsustainable.

MHE: What are the biggest challenges for ethical drug approval decision-making? Is cost transparency and informed consent about costs, a major one?

Glover: Yes, in addition to the challenge of good data, to be able to weigh benefits and burdens, cost transparency and informed consent are major challenges.

It can be hard to do, to bring that to scale, in larger practices. A smaller hospital in our system has a designated pharmacist for our oncology practice who looks up the out-of-pocket expenses for our patients. That’s absolutely best practice. Some in their electronic health records have embedded alerts about cheaper alternative drugs that can help clinicians and patients make better choices together.

The data is clear: people want to know the out-of-pocket expenses for themselves. They don’t particularly care about costs in general to the insurance companies or Medicare or Medicaid. But they do want to know about costs to themselves.

I think doctors are not necessarily the appropriate people to talk about costs because they often don’t know. But they should set the stage; they should be aware of the options and who to talk to and to make it OK for patients and their families to bring up cost issues, and know where to refer them. They have to be prepared to help the patient find information, even to the point of asking, “What other concerns do you have? Do you have concerns about costs?”

Next: What does an ethics review for drug pricing, the process of doing that, look like?

MHE: What does an ethics review for drug pricing, the process of doing that, look like? How does it work?

Glover: If there is a formal consult, one [example] is like some “rationing” decisions about certain very expensive drugs or drugs that are simply not available.

The ethics team, we gather information, we meet with team-clinicians, administrators, and, if possible, somebody representing the patient and family perspective-to develop consensus about an approach that is ethically justified. We write it up for ethics records and send to key stakeholders.

The ethics team brings knowledge of an ethical framework-respect for autonomy, beneficence, non-maleficence, justice, fidelity, truth-telling, transparency-to a discussion with clinicians and administrators who are already committed to behaving ethically.

People have the wrong idea about ethics teams. We don’t come in and run an ethics “X-ray”; we are not ethics police. We assume that people want to do the right thing and we help them sort out what that means: what options are better and which are less good, and why. The ethics team also brings a connection to a literature about these difficult topics-and where possible, guidelines.

I am a representative from the ethics committee that sits on the P&T (Pharmacy and Therapeutics) committee. I speak to individual choices, ask questions, compare those to other decisions and the literature. I usually expand it to improved processes like informed consent.

MHE: Should every hospital or facility involved in prescribing decisions establish an ethics committee?

Glover:  Every hospital is required by the Joint Commission to have a “mechanism” in place for patients, families and staff to have assistance when faced with ethical issues. And I think ethics committees can be a very effective mechanism, to not only react to the need to individual consults, but also to be involved in policy development and review and also education.

I think ethics committees can be an important part of establishing an ethical climate or culture, where at all levels discussion routinely involves values and what to do when values are in tension-from the bedside to the boardroom, if you will.

I think that ethics committees can be part of that change.

If you are a company involved in healthcare, you should be concerned about an ethical climate or culture, where the language of ethical values is included. And an ethics committee can help. But perhaps the biggest challenge of all is how to have more public dialogue about healthcare and healthcare costs.

You have to have a moral compass. But healthcare professionals also need ethics. Sometimes your personal morality can be in tension with your professional responsibilities.

Drug cost is a “wicked problem”-one that does not have a technological fix, but one that involves a tension among deeply-held value commitments. And we have to talk about it, among families, communities, and as a nation.

We are prone to using “rights” language-is there a right to healthcare? I don’t think rights language is useful, and it can be very polarizing.

We use it as though it answers our ethical questions. But it really just starts them. A right is a justified claim: something so important that others should do it or refrain from doing it. A right involves a correlative obligation on the part of someone. It is a very important obligation. So let’s begin talking about our obligations: Who is my neighbor and what do I owe him or her in terms of public health measures and healthcare. And the answer cannot ethically be nothing or everything.