Tennis legend Arthur Ashe once said, "Start where you are, use what you have, do what you can."
And that may be a good way to begin addressing cancer-drug costs and their benefits, according to an essay by Robert C. Young, MD, of RCY Medicine, in Philadelphia, Pennsylvania, that was published online November 19 in the New England Journal of Medicine.
Prescription drug prices in the United States have been among the highest in the world and have evoked much discussion among physicians, the general public, professional organizations, and advocacy groups. The escalating costs have spurred a number of initiatives designed to help physicians, payers, and patients better understand the value of new therapies and thus make better choices about their use.
In his new essay, Dr Young, who is a former president of the American Society of Clinical Oncology (ASCO) and former chairman of the Board of the National Comprehensive Cancer Network, reviews some of the major initiatives.
ASCO, for example, has introduced its Value Framework, which defines value as a measure of clinical benefit, toxicity, and cost. Peter Bach, MD, director of the Center for Health Policy and Outcomes at Memorial Sloan Kettering Cancer Center, in New York City, designed the DrugAbacus, a tool that may help determine a more appropriate price for a specific agent, based on what experts feel are possible components of a drug's value. And the National Comprehensive Cancer Network (NCCN) has released its new value initiative — the NCCN Evidence Blocks, which uses "blocks" to visually represent five key value measures — efficacy, safety, quality of evidence, consistency of evidence, and affordability — to provide important information about specific recommendations found in the NCCN guidelines.
"The goal of all these initiatives is to address the challenge of mushrooming cancer-drug costs by providing cost–benefit analyses in a format that enables a conversation between the patient and physician that incorporates consideration of costs," writes Dr Young. "Each group envisions its tool being used in discussions driven by individual patient preferences."
Dr Young points out that none of the organizations have suggested that these tools be used as a part of a broadly applied administrative mechanism to control cancer-drug costs or use, and he suspects that they would not support that type of application. But nothing prevents others from trying to use them for that purpose, he said.
"For people in our society who believe that cost should never be a consideration in decisions about the treatment of serious illnesses, these initiatives will be unsettling," he says. "Although these remain works in progress, it is clear that the cost-benefit dialogue has come of age."
Positive Step, but Refinement Needed
In a related essay in the same issue of the NEJM, Peter J. Neumann, ScD, and Joshua T. Cohen, PhD, both of Tufts Medical Center, Boston, Massachusetts, applaud the efforts of these initiatives.
They emphasize that the move to value-based frameworks for assessing drugs and other interventions is a positive step. Although anger over escalating drug costs may be understandable, it has led to calls for price setting that will reflect research, development, and production costs for manufacturers. This is "a strategy we believe is misguided," write Dr Neumann and Dr Cohen.
"By instead focusing on a drug's benefits, value-based approaches can encourage firms to produce more of what people want — products that improve health — and thereby further stimulate innovation," they writeHowever, the initiatives all need refinement before they can be broadly implemented.
"It's a big challenge but one that is welcome and evolving," Dr Neumann told Medscape Medical News. "They are works in progress, and we will have to see if the payers, physicians, and patients — the people who are supposed to be the targets — are going to find them useful."
The value-based frameworks that highlight overall cost-effectiveness are most attractive because cost-effectiveness provides a common scale for comparing products. But cost-effectiveness analysis is an incomplete tool, because it does not "convey all of a drug's salient characteristics," they point out. Therefore, more work is needed to determine how to best take into consideration factors such as side effects and ancillary benefits that are important to patients.
Also, cost-effectiveness analysis does not always adequately address the full cost impact, because it focuses on individual therapies and not the potential system-wide effect. As an example, the authors point to the hepatitis drug sofosbuvir (Sovaldi, Gilead Sciences, Inc), which studies have found to be "acceptably" cost-effective (ie, $50,000 to $100,000 per quality-adjusted life-year), but the high price tag makes it too expensive for several million Americans.
"All of these initiatives will be subject to ongoing discussion," said Dr Neumann. "But most important is if a payer finds this useful, and that will be telling. Private groups are offering up these frameworks, but they are not the ones footing the bill."
The ideal would be for policy makers to reduce spending on lower-value services so that higher-value ones could be better accommodated. Efforts are under way to identify low-value interventions, such as the Choosing Wisely program, but more initiatives are needed, write the authors.
This in turn could produce its own set of challenges. Even when lower-value services are identified, reducing spending on them is exceedingly difficult because potential income for physicians, hospitals, and product manufacturers would be threatened, Dr Neumann explained.
"These frameworks look at one intervention at a time, and they come up with a score of what is good value and bad value," he said. "But while this is useful, it's not a complete picture."
Despite similarities, the initiatives and frameworks differ from one another as well. Despite the challenges, Dr Neumann pointed out that they are helping to "change the conversation and move the focus onto value."
The authors have various ties to industry, foundations, and organizations, all of which are described in the original published articles.
N Engl J Med. Published online November 18, 2015. Full text