Value: What Do We Mean, Who Should Decide?

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John F. Smyth, MD

It is unlikely that health care can ever become cheaper, and scientific progress can only add to the burden of the opportunity cost debate—a good problem to have but one of the great contemporary challenges for society as a whole.

—John F. Smyth, MD

Oscar Wilde famously defined a cynic as “a man who knows the price of everything and the value of nothing.” I do not think that oncologists need to be as cynical as this, but it was very appropriate that a major theme of this year’s ASCO Annual Meeting was the concept of “value.” It is clear that there are widely differing attitudes to what we mean by value in oncology, and I would suggest that all members of society need to consider this in a timely fashion—balancing the extraordinary success of medical research vs the pressure of finding the resources to translate this knowledge into practical health care.

Defining Benefit

In the dictionary in my office, value is defined as “the relationship between something that is useful and its exchangeability.” These interesting words are worth pondering. In oncology, we recognize usefulness as benefit, but not everyone agrees as to what benefit really means in any given setting.

In an education session on lung cancer, Rogerio Lilenbaum, MD, from Yale, described the difficulty of agreeing on “clinical benefit” and referred to ASCO’s initiative in encouraging a new focus on “clinically meaningful” goals in the design of clinical trials.1 Many of us have argued that overall survival is not necessarily the best measure of clinical benefit, and certainly in my own experience, progression-free survival is a much more important factor for the majority of patients. Of course, these considerations are disease-specific, but for treatable but incurable cancers—the situation for the majority of solid tumors—relapse represents the lowest point emotionally for patients, even when there are two, three, or more subsequent treatments possible.

Quality of life is not easy to determine meaningfully, and in all the considerations of defining benefit, opinions differ considerably in different parts of the world. Nevertheless, I believe that collectively we are aware of the multitude of factors that contribute to “clinical benefit,” and we have the tools to address and discuss this side of the value argument. I suggest that the other side—cost—is much more problematic.

The expression “exchangeability” in health economic parlance means “opportunity cost”—ie, if you spend on plan A, you cannot spend the same on plan B or C; you have to choose, and this is the hub of the problem. Leaving aside political arguments that health-care budgets should be enhanced at the expense of other areas of government spending, I believe that whatever sum is available for spending on health, the demand is always going to exceed the monies available.

Unstoppable Research, Limited Resources

Scientific research is unstoppable, and as we saw so excitingly illustrated at this year’s ASCO meeting, science is being realistically translated into meaningful progress in programs of prevention, screening, diagnosis, and management. The incidence of cancer is increasing and we are living longer, thereby not only requiring more cancer care, but also stretching demands on other health areas, including cardiology, orthopedics, and geriatrics.

There will never be enough resources, and this problem is especially obvious in countries such as the United Kingdom, where we still try to offer health care free at the point of delivery. In the UK and many European countries, the patient does not have to pay directly, but increasingly, government funds cannot make available all the interventions that we would wish.

All too often, people debate this issue around the cost of drugs, especially the introduction of new drugs, but it is important to remember that opportunity cost refers to all aspects of cancer care—surgery, radiation, etc—and it is not only the cost of goods and services but the availability of staff, time in operating rooms and on machines, and so on.

Who Decides?

The challenge facing us over the next 5 to 10 years is to decide which sectors in society should contribute to the very difficult decisions on what we do or do not fund—these are real choices that have to be faced. As discussed at this year’s ASCO meeting, in the United States, patients and their physicians often have a primary role in these choices, especially, for example, in the use of new, expensive drugs.

Even for an individual family, opportunity costs have to be considered, particularly in situations where life expectancy is short. However, in assessing the overall value of a medical intervention, we can argue that while patients and their advocates are an essential component, they are by definition biased and not well positioned to contribute to the overall opportunity discussion.

In my opinion, politicians are the least suited to make these decisions due to their short termism and the need to appeal to the popular vote. The pharmaceutical industry is pivotal and frequently challenged about the costs of new drugs but, of course, members of this sector argue in defense of their research and development costs and responsibility to shareholders.

Regulators, for example, with the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA), have a role in the value equation. When I served on the EMA’s Scientific Advisory Group for Oncology, I argued (often unsuccessfully) that although we were not involved in pricing issues per say, we nevertheless had a responsibility to avoid granting licenses for drugs with very marginal benefit. If a drug is not licensed, it cannot add to the complexity of the value discussion, but of course there is a constant clamour to make new drugs available as long as they have some proven efficacy.

Drug Licensing and Availability

The hardest problem in opportunity costing lies with health technology assessment programs. I greatly admire the progress made in recent years by both the FDA and EMA to speed up the process of licensing decisions by opening up discussion with drug developers throughout the clinical trial period and adapting their procedures accordingly. In Europe, however, there are also people who decide whether a newly licensed medicine should be made available to the public. Moreover, there are considerable variations in the decisions reached by different health technology assessment programs in different European countries.

In England, the National Institute for Health and Care Excellence (NICE) is responsible for recommendations after a medicine is licensed by the EMA. The National Health Service is under incredible cost pressures, and I do not envy the brave people who sit on NICE committees in having to reach their very difficult decisions. The criteria by which they reach these decisions can be challenged. For example, just recently NICE surprisingly (and controversially) rejected olaparib (Lynparza) for women with ovarian cancer and a BRCA1 or BRCA2 mutation, citing the drug’s high cost and the availability of other treatment options that offer comparable benefits.

Even within the UK, there are different criteria for value decisions, with Scotland having its own counterpart to NICE (ie, the Scottish Medicines Consortium, which also advised against the use of olaparib). In Austria, by contrast, health technology assessment is not such an obstacle, and any newly licensed drug is immediately available to be prescribed.

Finding the Right Mix

So who should decide how we spend limited health budgets—patients, physicians, providers, insurance companies, health technology assessment programs, regulators, industry, or another stakeholder? All have a part to play, but we have yet to get the right mix involved, and we lack criteria for determining an agreed-upon value for any new intervention.

I believe that we need to create new platforms accepting different attitudes and ambitions from the public and physicians in different parts of the world, but these issues need to be debated so that we can better attribute value to new or established interventions and decide on the best opportunity cost. Maybe ASCO, with its global, independent, and comprehensive understanding of cancer, could be the host for discussing the optimum forum where these very pressing issues can be debated.

This problem will not go away. It is unlikely that health care can ever become cheaper, and scientific progress can only add to the burden of the opportunity cost debate—a good problem to have but one of the great contemporary challenges for society as a whole. ■

Disclosure: Dr. Smyth reported no potential conflicts of interest.


1. Lilenbaum R: Statistical and clinical significance in lung cancer trials. 2015 ASCO Annual Meeting. Education Session. Presented May 30, 2015.


Dr. Smyth is Emeritus Professor of Medical Oncology, University of Edinburgh, United Kingdom.

Disclaimer: This commentary represents the views of the author and may not necessarily reflect the views of ASCO.