Do End-of-Life Products Deserve Special Treatment?

July/August 2010, Vol 1, No 3 - ISPOR Annual Meeting

Atlanta, GA—Echoes of the recent contentious American debate over healthcare could be clearly heard in a session at the meeting of the International Society for Pharmacoeconomics and Outcomes Research that addressed issues surrounding the funding of new treatments, technologies, and drugs for patients nearing the end of the life, covering everything from standards for economic evaluations of new drugs to the philosophical questions surrounding treatments near the end of life.

In a wide-ranging discussion, a 3-speaker panel comprised of Nicole Mittmann, PhD, Marc Berger, MD, and Mark Sculpher, PhD, generally agreed that significant tradeoffs occur when healthcare systems fund new treatments, but in a spirited Q&A session that followed, some audience members challenged these basic assumptions.

One audience member took pointed aim at the British healthcare system’s denial of coverage for some new cancer drugs, asking Dr Sculpher, a University of York professor of Health Economics and director of the Program on Economic Evaluation and Health Technology Assessment for the school’s Center for Health Economics, “Would you feel any different about the societal perspective if you yourself were diagnosed with metastatic colon cancer knowing that there are drugs available that could treat you that NICE [National Institute for Health and Clinical Excellence] has denied because of cost?”

“I don’t know what I would think if I were in that position,” Dr Sculpher replied, after dismissing as “unreliable” recent research suggesting that more patients die of cancer in the United Kingdom (UK) because some drugs are not funded, “but ultimately, as a citizen, I believe in a healthcare system which has the purpose of improving health across all patient groups. Every time we say yes to a drug that generates relatively small improvements for high cost, somebody else in that system suffers.”

This exchange highlighted the tension between those who explicitly acknowledge limitations within health care systems and those who do not. Dr Sculpher was eager to illuminate the gulf separating these positions. “Some healthcare systems pretend that there are no opportunity costs, that people don’t suffer when you agree to reimburse new drugs, but in the UK that has become more transparent.”

Frameworks and Tradeoffs

Honestly discussing this tradeoff within healthcare systems and how to manage it equitably was a main theme of the panel discussion. The first panelist, Dr Mittmann, discussed the standards used in Canada to prioritize decisions about funding new cancer treatments. An associate scientist at the Sunnybrook Health Sciences Centre in Toronto, Dr Mittmann walked the audience through the process followed by a 15-member team of investigators as it “cancerized” the Canadian Agency for Drugs and Technologies in Heath’s (CADTH) guidelines for the conduct of economic evaluations. They examined the CADTH guidelines carefully, looking for ways to make the specific cancer guidelines within them more explicit and clear. Working for 3 years and producing 29 different iterations of the document, the team was able to create a sound methodological framework for making funding decisions for new cancer treatments.

The challenges were significant, admits Dr Mittmann. Their initial survey of the problem uncovered a lack of adherence to guidelines used to do economic evaluation, a lack of standardization in economic evaluations, and differential levels of evidence to evaluate cancer products.

“Sometimes you just don’t have a phase 3 trial that is available to you,” observed Dr Mittmann. “So you need to consider phase 2 studies. How do you methodologically deal with this?”

Panelist Marc Berger also grappled with the “framework” for decision-making in a talk that raised important philosophical questions about how healthcare systems evaluate the value of new cancer therapies used for cancer treatment at the end of life. Dr Berger, a vice president of Global Health Outcomes at Eli Lilly, observed that the “number of oncology drugs is about the explode” with a 10-fold increase in number of new cancer products on the horizon, and this plethora of new treatments will force a number of philosophical questions. For example, what is the value of life extension and is it the same for all diseases and medical conditions?

“Human beings are not pure utilitarian creatures,” Dr Berger reminded the audience, “and we do find a lot of people who believe that there is a greater need to intervene there [in the area of life extension].”

Uncomfortable Questions, Expensive Solutions

Berger posed other potentially uncomfortable questions as well: “What value do we assign to a patient’s life beyond direct medical costs and workplace productivity?” Many people getting cancer after they retire will not be adding to the gross domestic product, he observed. Is a purely utilitarian metric appropriate here? What is the value of life extension and is it the same as for other medical conditions? How does a patient benefit from slowing disease progression if he/she still does not live any longer? There is value to having a better quality of life…how do symptoms and disease progression impact a patient’s independence? Dr Berger was realistic about the necessary tradeoffs implied by asking these questions.

“The way that decisions have been made is in the context of a handful of biologics or new cancer therapies,” he observed. “If you imagine a future 10 years from now where you have 50, 75,

a 100 of these, I don’t think we’re going to be paying $20,000 a month or $100,000 a year for a 100 new cancer therapies; that’s just not going to happen. So how that conversation is going to happen is going to be a really interesting evolution in the marketplace.”

In the lively Q&A that followed the session, Dr Berger joined Dr Sculpher in defending NICE as pragmatic and realistic in ways that the American system is not.

“The UK has determined what it’s going to cap its total healthcare expenditures at,” he observed. “Could it be more? Sure. On the other hand, in this country, medical care expenditures are going up and we’re frightened to death by what that means.”

Dr Berger also argued that there is no “Pareto optimal” when making decisions about how to allocate resources in a healthcare system. “We make choices all the time. What we want to have at bottom is a fair and just society in which the process by which those decisions are made, everyone feels like they have a fair shake. I think that NICE has made great strides over its 11 years of existence.”