Value-Based Cancer Care

n engl j med 373;27 december 31, 2015PERSPECTIVE 2593Report from Parisoutside my office, which was set up specially for the families.”Also involved was the Etab-lissement de Préparation et de Réponse aux Urgences Sanitaires (EPRUS), the public agency for preparedness and response to health emergencies. EPRUS sup-plies health care workers to parts of the system that are over-whelmed or experiencing a sud-den surge in need. In this case, it provided medical and psycholog-ical help for the families, as well as help in identifying the bodies.“Identification of everyone involved is no minor task,” Val-let explained. “Sometimes it was very difficult to gather this in-formation in the hospitals be-cause they were overwhelmed.” One reason why early identifica-tion is important is that France offers victims of terrorist at-tacks the same protections as civil casualties of war, automati-cally providing medical, psycho-logical, and social support ser-vices. The health care personnel involved are also taken care of, undergoing first emotional de-briefing and later technical de-briefing about what parts of the process could be improved.“If you want to have a resilient system, you need to have all this in place,” says Vallet. “If you im-provise the medical–psychological care, for example, if you don’t anticipate, then you end up with injured people who don’t function at work or socially, and our soci-ety will be injured for much lon-ger.” It makes a big difference for the victims that needed services are anticipated and provided, and not something they have to fight for later.“This is public health,” Vallet told me as we sat in his office on the sixth floor of the Department of Health, overlooking central Paris. “During that night, I had the feeling I was back in the emergency room or in an inten-sive care facility. But this time, I was curing the body of society.”Disclosure forms provided by the author are available with the full text of this article at .Dr. Haug is an international correspondent for the Journal .This article was published on December 2, 2015, at .DOI: 10.1056/NEJMp1515229Copyright © 2015 Massachusetts Medical Society.Value-Based Cancer CareRobert C. Young, M.D.I n June 2015, the American Soci-ety of Clinical Oncology (ASCO) published a proposed framework for assessing the value of vari-ous cancer treatments.1 The goal was to evaluate selected treatment regimens on the basis of their clinical benefit, toxicity, and cost. ASCO’s ideas about cost had been incubating for a long time: the organization had created a Task Force on the Cost of Cancer Care in 2007, although, like most medical specialty organizations, it had generally remained silent on the issue of cost. But the cost of cancer care has been growing rapidly: though it accounts for a relatively small portion of overall U.S. health care expenditures, it is expected to increase from $125 billion in 2010 to $158 bil-lion in 2020.The costs of cancer drugs amount to only 5 to 20% of the total costs of cancer care, depend-ing on how many of the multiple cost components are included. But the average cost of some newer cancer drugs is now $10,000 to $30,000 per month, and combina-tions of checkpoint inhibitors cost as much as $100,000 per month. Virtually none of these treatments are curative, and some improve only disease-free survival, not overall survival. The costs of co-payments, out-of-pocket expenses, and rising insurance premiums exceed many patients’ capacity to pay. A 20% copayment for check-point inhibitors might cost the patient $60,000 for a full course of treatment. Many patients elect to simply forgo treatment.The momentum behind in-creased attention to costs by phy-sician organizations is attributable in part to the Choosing Wisely campaign, an initiative led by the American Board of Internal Med-icine Foundation that involves identifying each specialty’s “Top Five” opportunities for eliminat-ing overuse and misuse of tests and procedures and highlighting therapies that confer little or no patient benefit. ASCO has pub-lished two such lists, which ad-dress futile therapy, overuse of expensive and unnecessary screen-ing and post-treatment monitor-ing, and misuse of targeted ther-apies, among other sources of waste.2,3 More than 70 subspe-cialty organizations have contrib-uted Top Five lists or included cost or value measures in their publi-cations.The Institute of Medicine has delineated six elements of value in cancer care: safety, effectiveness,PERSPECTIVE n engl j med 373;27 december 31, 20152594patient-centeredness, timeliness, efficiency, and equity.4 ASCO se-lected only three of these for its framework — clinical benefit (effectiveness), toxicity (safety), and cost (efficiency) — believing that the other three, though im-portant, are not easily measured and are infrequently reported in clinical trials. ASCO has used its framework to analyze drug costs in a few cancers in which random-ized trials have compared new therapies with standard regimens. It has presented two versions of the framework, one for advanced disease and the other for adjuvant therapy. Analysts used a clinical-benefit score derived from com-parisons of overall survival, pro-gression-free survival, or response rates, as well as comparative tox-icities of the two regimens to de-fine a “net health benefit” (NHB). In some cases, such as that of the newer three-drug regimen for multiple myeloma, the NHB justi-fied substantially greater monthly cost than that of the previous stan-dard regimen ($7,042 vs. $279). In other cases, such as that of one of the regimens for non–small-cell lung cancer, there was no NHB difference despite huge cost dif-ferentials ($9,193 vs. $811).As the drafters of the frame-work emphasize, the model is pre-liminary, and ASCO has requested feedback from stakeholders. One drawback is that the model uses similar clinical-benefit measure-ments for outcomes that have vast-ly different value to the patient. For example, improved survival rates greatly eclipse increased re-sponse rates in terms of patient benefit. Many of the weights placed on the components of risk–benefit analysis are arbitrary, and bonus points were included for palliation and time off therapy. Of the many components of drug costs, only acquisition costs andpatients’ out-of-pocket expenses were included. The designers pro-pose in subsequent iterations to use alternative databases, nonran-domized trials, and more robust cost analyses. Their task will not be easy. For such a model to be useful in a busy office practice, comparisons of a broad spectrum of cancer treatments and prior calculations of the NHB of com-mon regimens will be needed. Many commonly used regimens have never been compared in randomized trials with all other seemingly equivalent regimens.Other oncology groups have presented alternative approaches. Memorial Sloan Kettering Cancer Center has developed a tool () that ana-lyzes therapeutic impact, toxici-ties, and cost and has used it to produce a cost–benefit analysis for 54 cancer drugs approved by the Food and Drug Administra-tion since 2001. According to the Sloan Kettering analysis, some of the drugs appear to be appropri-ately priced, others grossly over-priced, and others underpriced for the benefit achieved. This analysis, while interesting and useful, could have unintended consequences: drug manufacturers might con-sider increasing the prices of the drugs deemed underpriced and leave the others unchanged.The National Comprehensive Cancer Network (NCCN) has de-veloped a patient-oriented value formula that adds evidence on af-fordability to information on ef-ficacy, safety, quality of evidence, and consistency of evidence for the many alternative regimens included in its 66 comprehensive cancer treatment guidelines (www ). The information is pre-sented in a column format, which compares these five aspects of each regimen using stacked shad-ed blocks representing values ranging from 1 (palliative only) to 5 (highly effective), with scores based on analyses by panels of physician experts. The shaded blocks provide a visual summary of the overall benefit of the regi-men, according to Robert Carl-son, NCCN chief executive officer.The goal of all these initia-tives is to address the challenge of mushrooming cancer-drug costs by providing cost–benefit analy-ses in a format that enables a con-versation between the patient and physician that incorporates consid-eration of costs. Each group en-visions its tool being used in discussions driven by individual patient preferences. None of them has suggested that these tools be used as a part of a broadly applied administrative mechanism to con-trol cancer-drug costs or use, nor, I suspect, would they support such an application. Unfortu-nately, nothing prevents others from trying to use the informa-tion for such a purpose. For people in our society who believe that cost should never be a consider-ation in decisions about the treat-ment of serious illnesses, these initiatives will be unsettling. But I believe all these groups should be commended for pioneering efforts to address unsustainable drug costs with credible data-driven analyses. Although these remain works in progress, it is clear that the cost–benefit dia-logue has come of age.If a rational consideration of cancer-drug costs in the selection of effective cancer treatment is achieved, physicians and their pa-tients will need to play a central role. As Michael Porter and Thomas Lee, early proponents of value-based health care, have written, “The challenge of be-coming a value-based organiza-tion should not be underestimat-ed, given the entrenched interests Value-Based Cancer Caren engl j med 373;27 december 31, 2015PERSPECTIVE 2595Value-Based Cancer Careand practices of many decades. The transformation must come from within. Only physicians and provider organizations can put in place the set of interdepen-dent steps needed to improve value because ultimately value is determined by how medicine is practiced.”5The collective efforts now un-der way to address cancer-drug costs and benefits are preliminary but should not be undervalued. Perhaps a lesson from tennis leg-end Arthur Ashe is apt: “Start where you are, use what you have, do what you can.”Disclosure forms provided by the author are available with the full text of this article at .From RCY Medicine, Philadelphia.This article was published on November 18, 2015, at .1. Schnipper LE, Davidson NE, Wollins DS, et al. American Society of Clinical Oncology statement: a conceptual framework to as-sess the value of cancer treatment options. J Clin Oncol 2015;33:2563-77.2. Schnipper LE, Smith TJ, Raghavan D, et al.American Society of Clinical Oncology iden-tifies five key opportunities to improve careand reduce costs: the Top Five list for oncol-ogy. J Clin Oncol 2012;30:1715-24.3. Schnipper LE, Lyman GH, Blayney DW, etal. American Society of Clinical Oncology2013 Top Five list in oncology. J Clin Oncol2013;31:4362-70.4. Levit L, Balogh E, Nass S, Ganz PA. Deliv-ering high-quality cancer care: charting anew course for a system in crisis. Washing-ton, DC: National Academies Press, 2013.5. Porter ME, Lee TH. The strategy that willfix health care. Harvard B usiness Review.October 2013 (https:///2013/10/the-strategy-that-will-fix-health-care ).DOI: 10.1056/NEJMp1508387Copyright © 2015 Massachusetts Medical Society.Measuring the Value of Prescription DrugsPeter J. Neumann, Sc.D., and Joshua T. Cohen, Ph.D.E scalating drug prices have alarmed physicians and the American public 1,2 and led to calls for government price con-trols. Less visibly, they have also spawned a flurry of private-sector initiatives designed to help physi-cians, payers, and patients un-derstand the value of new thera-pies and thus make better choices about their use. Pro-grams recently introduced or ad-vanced by nonprofit organiza-tions, including leading medical professional societies, represent an important innovation in the United States, but they have also revealed numerous analytic and implementation challenges.The most prominent players include the American College of Cardiology and the American Heart Association (ACC–AHA), the American Society of Clinical Oncology (ASCO), the Institute for Clinical and Economic Re-view (ICER), Memorial Sloan Kettering Cancer Center (MSKCC), and the National Comprehensive Cancer Network (NCCN). Their initiatives have different missions — for example, ASCO, MSKCC, and NCCN focus on cancer drugs, and ICER’s purview is broader and not specific to pharmaceuti-cals. B ut each organization’s framework accounts for factors underlying value, such as the quality of clinical data support-ing the therapy’s use, the magni-tude of its treatment effects, the likelihood of severe adverse events, and the product’s costs, ancillary benefits, cost-effectiveness, and effects on the health system bud-get (see table).Several lessons are emerging. First, the move to value-based frameworks for assessing drugs and other interventions is a posi-tive step. Anger over rising drug prices may be understandable, but it has led some observers to call for setting prices to reflect research, development, and pro-duction costs for drug firms, a strategy we believe is misguided. B y 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 in-novation. Consider the purchase of an automobile. Consumers don’t ask dealers about a car’s manufacturing costs. Instead, they decide whether to buy a par-ticular car by comparing its price and features to those of other ve-hicles, in the process spurring companies to develop ever better alternatives.Second, whereas the govern-ments of many countries use their regulatory and buying pow-er to control drug prices, these U.S.-based initiatives represent private-sector solutions. They are a response to three realities in the United States: increasing pre-scription-drug costs, political op-position to giving Medicare au-thority to negotiate drug prices, and the fact that most Americans have private health insurance. The initiatives reveal potential for pri-vate organizations to challenge the patent-protected monopoly pow-er enjoyed by drug manufacturers, though the groups are advisory in nature and simply provide infor-mation for the marketplace.Third, the frameworks reveal numerous analytic challenges. Value is an elusive target, and there’s no consensus about what dimensions should be taken into account. For example, only the。