A requirement of many regulatory bodies around the world, including Canada, is the need to demonstrate the value of new drugs through a pharmaco-economic analysis. In the context of limited resources and an ever-expanding need for health-care services, determining the value for money of new treatments is an important consideration for policy makers, who have to make life and death choices within a constrained budget.
The concept of "opportunity cost" is a fundamental part of economics. In health care, it means that since resources are limited, using any of them for the care of a particular group of patients means someone else, somewhere, loses out. Those losing out on funds could be patients with another disease, young children, or the recipients of a public program for disease prevention that is cut because the province has to find money to cover hospital overruns. Policy makers need to balance, therefore, what is gained against what is lost.
In matters of life-ending ailments, policy makers must balance what is gained from spending more dollars in caring for people with end-stage disease with what we would gain if we spent those same dollars elsewhere.
Until the early part of the 20th century, most people died of infectious diseases, and cancer was a disease without hope. Diagnosis was often at a late stage; no treatments were available that significantly altered the course of disease. Patients with cancer now live much longer, and cancer is considered more of a chronic, debilitating disease, from which most patients eventually die. As the population ages, there will be a disproportionate increase in cancer patients. This poses a daunting resource-allocation challenge to policy makers and the public alike.
Clinical effectiveness, costs, and patient preferences must be weighed when assessing value for money in cancer care. The seriousness of the ailment, the availability of alternative therapies and considerations of equity or fairness may also be factors when deciding whether expensive new drugs are funded.
Recommendations to fund cancer drugs probably often reflect a combination of these factors. But at what point is it no longer "worthwhile" to provide funding for drugs and treatment to prolong life, or alleviate suffering for patients in the end stages of disease? And what is the ethical justification for denying treatment? Isn't life priceless?
End-of-life care for cancer patients is inevitably expensive, largely because people are very sick. Based on Medicare data from the United States, it has been estimated that more than two dollars of every eight spent is spent in the last year of life, and one in eight is spent in the last month. One response to this apparent imbalance would be to try to be more efficient in the provision of end-stage cancer care. For example, home management of terminally ill patients could potentially contribute to decreased costs. A retrospective Canadian study compared the cost of managing cancer patients who required narcotic infusions in hospital and at home. Medical costs in 1991 Canadian dollars averaged $370 a day for inpatient care, and $150 a day for outpatients.
Some would argue that reducing expenditures at the end of life is a justifiable way of cutting wasteful spending and making resources available for other needed health-care services. In the context of these alternatives, it could be argued that interventions for patients whose death is imminent are inherently wasteful, since they neither cure nor stop the progression of disease or disability. For example, one might question the use of an intrathecal pump for administering morphine -- the spinal-access pump has an initial cost of about $20,000 -- to achieve better alertness and pain control, compared to oral or subcutaneous morphine.
What amount of benefit should be considered worthwhile? What is it worth to extend life by a few days? What if this extension in life is accompanied by a decrease in quality of life, because of the toxicity associated with treatment? What is the value of interventions that are not associated with improved survival?
One approach is to set a common threshold of cost effectiveness that should be applied to all health-care interventions. In the United Kingdom, where health-care rationing is practised to a fine art, the National Institute for Health and Clinical Excellence (an agency that provides guidance to the National Health Service) tends not to approve treatments that cost more than £30,000 (about $68,000 Canadian) per year of life gained (adjusted for quality of life).
A similar approach has been proposed in Canada in the past. A blunt instrument, no doubt, but it does force explicit consideration of both benefits and costs.
An important question, of course, is that of how we obtain the data for these assessments. Little attention is given to measuring cost or quality of life in clinical trials. The gold standards for outcomes of cancer treatment remain improved survival, and disease progression.
Decisions about whether to fund expensive therapies in end-stage disease will never be easy and will inevitably raise complex social, ethical and legal issues. It does not make sense, however, to dodge the issue and pretend that resources can be consumed without consequences for other patients, or society at large. We need to generate more data on the cost effectiveness of alternative treatments, in cancer and other diseases, and to have a more open debate about how these difficult choices should be made.
Michael Drummond is professor of health economics at the University of York in the United Kingdom; Deborah Marshall is assistant professor of clinical epidemiology and biostatistics at McMaster University in Hamilton, Ont., and vice-president of Global Health Economics and Outcomes Research.