Cancer has or will affect nearly every person at some point in their lives, either directly or indirectly. The fight against cancer is central to society; in recent years, treatment advances and prevention strategies have improved outcomes across disease states. New anticancer agents, including immunotherapy and targeted therapies, have fundamentally changed the treatment landscape of certain cancers, though at a price, as pharmaceutical costs now regularly exceed $100,000 per patient. Value measurement strategies and managed care may serve to reconcile the rapid treatment advances occurring in oncology with the attendant high costs of care.
Remember the “Fight for a Cure” campaign? How about the “Great American Smokeout,” which the American Cancer Society started in the 1970s? Whether you recall these programs or not, surely you have spent a Saturday walking for a cure; attended a dinner or an auction to benefit cancer research; or contributed a donation to any number of organizations dedicated to beating cancer. Our society is invested in advancing our ability to beat cancer. We have made significant progress with increasing survival rates in certain disease types, like Hodgkin lymphoma, non-Hodgkin lymphoma, and malignant melanoma, while increasing prevention in others, like cervical cancer.
Innovations in science have led to breakthroughs in treatment. Prior to the development of imatinib (Gleevec, Novartis), fewer than one in three patients with chronic myelogenous leukemia (CML) survived 3 years.1 Now CML patients treated with imatinib have a life expectancy similar to patients without cancer.2 Medical advances that lower treatment-associated toxicities have been just as important. The latest immunotherapies show improvements in survival with fewer toxicities, thus improving quality of life.
Although we have focused primarily on beating cancer, treatment costs remain high, with pharmaceutical costs alone exceeding $100,000 per patient. Even oncologists have taken greater notice of the cost of treatment, and organizations such as the American Society of Clinical Oncology (ASCO) have developed value frameworks to help determine whether the benefits associated with these treatments are worth the cost. This development should not be surprising, as oncologists not only manage patient health, but are increasingly involved in resource allocation. Initiatives such as the Michigan Oncology Quality Consortium and the Centers for Medicare and Medicaid Services’ Oncology Care Model tie provider reimbursement to both quality and cost of care.
Although some argue such programs are hardly voluntary, the fact is that they are clearly growing, much like the pathway programs developed or administered by health plans. With the advent of immunotherapy combinations that cost upwards of $250,000 per patient in the first year of treatment, scrutiny will continue by those paying the bills.3
As these two forces engage in an epic clash, there are fundamental questions to answer that will have a significant role in the outcome—both at an individual and population level.
Will biomarker assessment make treatment more precise, resulting in narrower populations receiving expensive treatment?
Matching treatments to specific effectiveness indicators has played a key role in the advances made in certain disease states, such as trastuzumab (Herceptin, Genentech) for breast cancer. Biomarkers indicating more specific elements of lung cancer, such as the anaplastic lymphoma kinase (ALK+) gene arrangement, can inform which treatments are effective for patients with that particular biomarker. Since ALK+ is believed to be present in approximately 2% to 7% of the non–small cell lung cancer (NSCLC) population,4 the higher treatment cost is for a small segment of the overall NSCLC population. When this treatment cost is spread across the entire NSCLC population and compared to historical nonspecific treatments for those patients, the cost per NSCLC patient is not as drastic. Still, as more targeted therapies come to market, such as those targeting multiple variants of epidermal growth factor receptor, the cost per patient creeps up—especially if the aforementioned immunotherapy combinations further treatment advances.
Will treatment choices be made through patient-physician decision-making or top down value frameworks?
All stakeholders are interested in identifying high-value treatments, but the definition of value varies based on the stakeholder’s perspective.5 Some organizations, such as the Institute for Clinical and Economic Review, measure value from a payer or population-level point of view. Under the payer framework, clinical factors and cost are often the primary factors considered, while treatment benefits and cost are often summarized through an incremental cost-effectiveness ratio. Patients, on the other hand, care little about purported cost and benefits in general, and would rather consider cost and benefits in their own unique scenario. Patients value a treatment based not only on its ability to improve survival for the typical patient, but also on the likelihood that some patients have significantly longer survival durations.6 These durable survival gains allow patients to potentially remain alive until the next advance in therapy.7 Like payers, providers often focus on improvements in survival for the typical patient.8 Value frameworks developed by physician groups such as ASCO can facilitate shared decision-making between the physician and patient based on the latter’s individual preferences and circumstances.
How do we account for the positive impacts on society that cannot be easily quantified?
Recommendations from the Second Panel on Cost Effectiveness provide some guidance by enumerating a broader set of societal components of value that should be included in any value assessment.9 For instance, societal value measures should incorporate a treatment’s benefits to caregivers. Typically, value from treatments that improve a patient’s functional status and allow caregivers to return to work are not incorporated in baseline value assessments. Treatments that can decrease a patient’s use of social services also result in significant cost savings to the government. For children, the treatment benefits that allow them to return to school should also be incorporated into any value assessment.
How do we deal with uncertainty?
Although statisticians can measure uncertainty in any given statistical model, a treatment’s true real-world benefit is uncertain. Treatment cost is well known at drug launch, but studies to measure additional treatment benefits (eg, caregiver burden) do not typically occur until after coverage decisions are made. Further, differences in the characteristics of real-world patients using a given treatment and those enrolled in a clinical trial may result in differences between a treatment’s efficacy and effectiveness estimate.10 One option for payers to address this uncertainty is to implement creative pricing mechanisms. Indication-specific pricing, licensing unlimited use of a treatment or treatments for a fixed time period, and outcomes-based pricing should all be considered.
Value measurements and the use of pricing mechanisms may serve as partial solutions to get high-value treatments to the individual patients who need them. Creative solutions will be required from each stakeholder to prevent the “clash” between realizing advances in oncology outcomes and breaking budgets.
1. National Cancer Institute. A story of discovery: Gleevec transforms cancer treatment for chronic myelogenous leukemia. https://www.cancer.gov/research/progress/discovery/gleevec. Accessed March 9, 2017.
2. Gambacorti-Passerini C, Antolini L, Mahon FX, et al. Multicenter independent assessment of outcomes in chronic myeloid leukemia patients treated with imatinib. J Natl Cancer Inst. 2011;103(7):553-561.
3. Loftus P, Winslow R. FDA approves Bristol-Myers’s Yervoy, Opdivo for treatment of melanoma. Wall Street Journal. https://www.wsj.com/articles/fda-approves-bristol-myers-yervoy-opdivo-for-treatment-of-melanoma-1443711746. Updated October 1, 2015. Accessed March 9, 2017.
4. National Comprehensive Cancer Network (NCCN). NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines): Non-Small Cell Lung Cancer. NCCN. 2014;3:1-148.
5. Chandra A, Shafrin J, Dhawan R. Utility of cancer value frameworks for patients, payers, and physicians. JAMA. 2016;315(19):2068-2070.
6. Lakdawalla DN, Romley JA, Sanchez Y, Maclean JR, Penrod JR, Philipson T. How cancer patients value hope and the implications for cost-effectiveness assessments of high-cost cancer therapies. Health Aff (Millwood). 2012;31(4):676-682.
7. Sanchez Y, Penrod JR, Qiu XL, Romley J, Thornton Snider J, Philipson T. The option value of innovative treatments in the context of chronic myeloid leukemia. Am J Manag Care. 2012;18(11 suppl):S265-S271.
8. Shafrin J, Schwartz TT, Okoro T, Romley JA. Patient versus physician valuation of durable survival gains: implications for value framework assessments. Value Health. 2017;20(2):217-223.
9. Sanders GD, Neumann PJ, Basu A, et al. Recommendations for conduct, methodological practices, and reporting of cost-effectiveness analyses: second panel on cost-effectiveness in health and medicine. JAMA. 2016;316(10):1093-1103.
10. Shafrin J, Brookmeyer R, Peneva D, et al. The value of surrogate endpoints for predicting real-world survival across five cancer types. Curr Med Res Opin. 2016;32(4):731-739.