Lee N. Newcomer

American Society of Clinical Oncology Statement: A Conceptual Framework to Assess the Value of Cancer Treatment Options

Health care costs in the United States present a major challenge to the national economic well being. The Centers for Medicare and Medicaid Services (CMS) has projected that US health care spending will reach

National Institutes of Health State-of-the-Science Conference: role of active surveillance in the management of men with localized prostate cancer.

4.3 trillion and account for 19.3% of the national gross domestic product by 2019.1 This growth in spending—both in absolute terms and as a proportion of our gross domestic product—has not been accompanied by commensurate improvements in health outcomes, despite expenditures far exceeding those of other countries.2–4 One of the fastest growing components of US health care costs is cancer care, the cost of which is now estimated to increase from

Updating the American Society of Clinical Oncology Value Framework: Revisions and Reflections in Response to Comments Received

125 billion in 2010 to

Health Care Costs for Patients With Cancer at the End of Life

158 billion in 2020.1 Although cancer care represents a small fraction of overall health care costs, its contribution to health care cost escalation is increasing faster than those of most other areas because of several factors: the increasing prevalence of cancer due to the overall aging of the population and better control of some causes of competing mortality; the introduction of costly new drugs and techniques in radiation therapy and surgery; and the adoption of more expensive diagnostic tests. In some cases, the adoption of newer, more expensive diagnostic and therapeutic interventions may not be well supported by medical evidence, thereby raising costs without improving outcomes.5 Coupled with, or even driving, some of these rising costs are sometimes unrealistic patient and family expectations that lead clinicians to offer or recommend some of these services, despite the lack of supporting evidence of utility or benefit.6 Historically, most individuals in the United States were shielded from the acute economic impact of expensive care because they had health insurance. However, current trends suggest that patients will find themselves increasingly responsible for a greater proportion of the cost of their health care. Cost shifting or sharing can occur through the increased use of high-deductible policies and larger copayments. These increased costs are already commonplace and may not be affordable for many families. Indeed, health care expenditures are cited as a major cause of personal bankruptcy,7 and the term financial toxicity has entered the vernacular as a means of describing the financial distress that now often accompanies cancer treatment.8 Like other toxicities of cancer treatment, financial toxicity resulting from out-of-pocket treatment expenses can reduce quality of life and impede delivery of high-quality care.9,10 Patients experiencing high out-of-pocket costs have reported reducing their spending on food and clothing, reducing the frequency with which they take prescribed medications, avoiding recommended procedures, and skipping physician appointments to save money.10,11 These unintended consequences risk an increase in health disparities, which runs counter to some of the key goals of health care reform. In many communities, the high costs associated with cancer care have created a difficult situation for patients and the oncologists who care for them. Addressing this situation will require greater understanding of all the risks and benefits of various treatment options as well as the consequences of specific choices. In this regard, studies have shown that patients specifically want financial information about treatment alternatives along with information about medical effectiveness and treatment toxicity. However, they often do not receive it. Closing this knowledge gap will require educated providers who are able to sensitively initiate a dialogue about the cost of care with their patients when appropriate.12,13 Patients with cancer are often surprised by and unprepared for the high out-of-pocket costs of treatments. They also overestimate the benefits of treatments that sometimes extend life by only weeks or months or not at all. Oncologists are generally aware of this conundrum but uncertain about whether and how the cost of care should affect their recommendations.14 Although raising awareness of costs and providing tools to assess value may help to manage costs while maintaining high-quality care, some oncologists see this as being in conflict with their duty to individual patients.15 Recent American Society of Clinical Oncology Efforts Motivated by our responsibility to help oncologists deliver the highest-quality care to patients everywhere, the American Society of Clinical Oncology (ASCO) formed the Task Force on the Cost of Cancer Care in 2007. Its mission includes educating oncologists about the importance of discussing costs associated with recommended treatments, empowering patients to ask questions pertaining to the anticipated costs of their treatment options, identifying the drivers of the rising costs of cancer care, and ultimately developing policy positions that will help Americans move toward more equal access to the highest-quality care at the lowest cost.16 In 2012, through the work of the Task Force, ASCO responded to the Choosing Wisely Campaign of the American Board of Internal Medicine Foundation and identified specific instances of overuse in the delivery of cancer care. ASCO used a deliberative consensus process to identify five common clinical practices that are not supported by high-level evidence. A second list of five was developed using the same process and submitted to the Choosing Wisely Campaign in 2013. ASCO amplified the evidence basis for both top-five lists in two publications17,18 and is now developing measures to evaluate the use of these practices as part of its Quality Oncology Practice Initiative. These exercises have provided opportunities to develop a rigorous but flexible approach to assessing efficacy across diagnostic and treatment domains.

Breaking a Vicious Cycle

National Institutes of Health (NIH) Consensus and State-of-the-Science Statements are prepared by independent panels of health professionals and public representatives on the basis of 1) the results of a systematic literature review prepared under contract with the Agency for Healthcare Research and Quality, 2) presentations by investigators working in areas relevant to the conference questions during a 2-day public session, 3) questions and statements from conference attendees during open discussion periods that are part of the public session, and 4) closed deliberations by the panel during the remainder of the second day and morning of the third. This statement is an independent report of the panel and is not a policy statement of NIH or the U.S. government. The statement reflects the panel’s assessment of medical knowledge available at the time the statement was written. Thus, it provides a “snapshot in time” of the state of knowledge on the conference topic. When reading the statement, keep in mind that new knowledge is inevitably accumulating through medical research. The following statement is an abridged version of the panel’s report, which is available in full at http://consensus.nih.gov/2011/prostatefinalstatement.htmNational Institutes of Health (NIH) Consensus and Stateof-the-Science Statements are prepared by independent panels of health professionals and public representatives on the basis of 1) the results of a systematic literature review prepared under contract with the Agency for Healthcare Research and Quality, 2) presentations by investigators working in areas relevant to the conference questions during a 2-day public session, 3) questions and statements from conference attendees during open discussion periods that are part of the public session, and 4) closed deliberations by the panel during the remainder of the second day and morning of the third. This statement is an independent report of the panel and is not a policy statement of NIH or the U.S. government. The statement reflects the panel’s assessment of medical knowledge available at the time the statement was written. Thus, it provides a “snapshot in time” of the state of knowledge on the conference topic. When reading the statement, keep in mind that new knowledge is inevitably accumulating through medical research. The following statement is an abridged version of the panel’s report, which is available in full at http: //consensus.nih.gov/2011/prostatefinalstatement.htm In 2011, more than 240 000 men are projected to receive a diagnosis of prostate cancer and 33 000 are projected to die of this condition. More than 2.5 million men in the United States are long-term survivors of prostate cancer. Men with a strong family history of prostate cancer and African American men are at increased risk for prostate cancer. Most cases of prostate cancer are localized at diagnosis and detected as a result of screening with prostatespecific antigen (PSA) testing. Most of these screendetected cases of cancer are low risk and are unlikely to cause death. The natural history of prostate cancer has changed dramatically in the past 3 decades because of PSA screening. Although most cases of prostate cancer are slowgrowing and unlikely to spread, most men receive immediate treatment with surgery or radiation. These therapeutic strategies are associated with shortand long-term complications, including impotence and urinary incontinence. Only a few men choose observational strategies, thereby delaying the initiation of curative therapy or avoiding it completely. Given the high prevalence of low-risk prostate cancer, the roles of active surveillance and other observational strategies as alternatives to immediate treatment need to be clarified. The National Cancer Institute, the Centers for Disease Control and Prevention, and the NIH Office of Medical Applications of Research convened a State-of-the-Science Conference on 5 to 7 December 2011 to assess the available scientific evidence about active surveillance for men with localized prostate cancer. The conference, which addressed 5 key questions, was informed by a formal evidence report commissioned through the Agency for Healthcare Research and Quality, data presented by speakers, and input from attendees.

Changing Physician Incentives for Affordable, Quality Cancer Care: Results of an Episode Payment Model

The mission of American Society of Clinical Oncology (ASCO) is to conquer cancer through research, education, and promotion of the highest quality patient care. Toward fulfillment of this goal and at the direction of its board of directors, the ASCOValue in Cancer Care Task Force set out to develop a framework that would enable a physician and patient to assess the value of a particular cancer treatment regimen given the patient’s individual preferences and circumstances. The rationale that served as the impetus for this initiative is many faceted. Substantial progress has been made in translating our knowledge of the biologic characteristics of cancer into novel therapies. Some of these therapies have led to major improvements in outcomes for specific diseases, and others have produced only modest advances. There is now a wide array of choices for treating many cancer types, and these treatment choices often differ by only small degrees in clinical effectiveness and toxicity. Yet, there is often a wide disparity in cost to patients and payers. Because patients are often confronted with enormous expenses when receiving cancer care, the goal of describing a relationship between the cost of an agent or regimen and the clinical benefits it delivers takes on great importance. As the primary advisor to the patient, the oncologist has an important role in providing a comparative assessment of the various treatment options available; in the spirit of shared decision making, the patient should have transparent information about the clinical impact that can be expected from the different options presented and their relative financial implications. The value framework has been constructed as a conceptual model that incorporates the elements of clinical benefit, toxicity, and symptom palliation as derived from a comparative clinical trial and combines these elements into a score termed the net health benefit (NHB). Ultimately, deployment of the framework as a software application is planned, enabling a patient to modify the weight attributed to any of the elements included in the NHB depending on his or her personal preferences and circumstances. The final NHB will therefore reflect the priorities that are most important to the patient and will be arrived at through guidance from the physician. Information on the cost of the regimens will also be presented so the patient can consider the relative financial impact of his or her treatment options. Two versions of the framework have been created: one for advanced disease and the other for potentially curable (adjuvant therapy) clinical presentations. The original framework versions are shown in Appendix Tables A1 and A2 (online only). The key elements included in the framework— namely, clinical benefit and toxicity—are also those that are regularly reported in the scientific literature when discussing the outcome of a clinical trial that compares two or more therapies. The importance of relying on high-quality, quantifiable evidence cannot be overstated, and this is most often provided by a well-designed, well-conducted prospective randomized trial. The task force recognizes that a limitation of this approach is that it does not readily permit cross-trial comparisons. Such analyses are important to patients and remain a goal for future versions of the value framework. The task force is well aware that there are many elements that might be important to individual patients in assessing the relative value of their treatment options that are not taken into account in our model. These include the convenience of receiving therapy, the avoidance of interrupting the flow of activities of daily living, and the impact of a treatment on quality of life

The Patient-Centered Medical Home in Oncology: From Concept to Reality

PURPOSE With rising health care costs in the United States, clearly defined end-of-life (EOL) cancer costs are needed to help health administrators proactively manage this important care. Our objective was to examine EOL health care resource costs among oncology patients in a US commercial insurance population. METHODS A retrospective claims database affiliated with OptumInsight was analyzed. Included patients had: a medical claim with cancer diagnosis between July 1, 2002, and December 31, 2009; death on or before December 31, 2009; continuous enrollment with medical/pharmacy benefits from diagnosis until death; ≥ 180 follow-up days; and active cancer in the last 6 months before death (MBD). Death was captured from facility discharge codes or Social Security Administration death files. Costs were determined by summing paid amounts on all services utilized within the last 6 MBD: cancer-related inpatient (IP) stays, cancer- related hospice care, and cancer-related outpatient (OP) services (ie, chemotherapy, erythropoiesis-stimulating agents, granulocyte colony-stimulating factors, radiation, cancer-related office or emergency room visits, cancer-related hospital OP procedures, and other services with cancer diagnosis). RESULTS A total of 28,530 patients met inclusion criteria. Mean total cancer-related costs in the last 6 MBD were

Innovative payment models and measurement for cancer therapy.

74,212 (standard deviation,

Unsupported off-label chemotherapy in metastatic colon cancer

112,740), comprising IP costs of

The Responsibility to Pay for Cancer Treatments: A Health Insurer's View of Value

40,702 (55%), OP costs of