Navigating Drug Costs: Tools to Aid in Determining Comprehensive Drug Value
Jennifer MacDonald, PharmD
PGY-2 Oncology Pharmacy Resident
University of Colorado
Aurora, CO
In an era of both expanding oncologic therapeutic options for patients and escalating drug prices, it is important to consider not only efficacy and safety but also the drug costs for the patient. Although a newer therapy may be indicated, if it comes at extreme cost to the patient, a comprehensive review should be undertaken before the therapy is initiated. In 2016, 21 oncologic agents were approved for the treatment of various malignancies. These approvals consisted mostly of rebranding medications like daratumumab, pembrolizumab, or nivolumab to encompass a broader array of indications.1 What do these approvals and expanded indications have in common? High cost.
In recent years, the American public has grown increasingly concerned about escalating drug costs and has urged the government to institute price controls.2 These growing concerns led a number of groups—including the American Society of Clinical Oncology (ASCO), the National Comprehensive Cancer Network (NCCN), and Memorial Sloan Kettering Cancer Center (MSKCC)—to develop tools evaluating the utility of various therapies for a patient.2 These tools take into account several aspects that are important when one is considering a treatment regimen and attempting to determine a drug’s overall value; some factors considered are quality of clinical data, likelihood of serious adverse events, magnitude of treatment effects, cost-effectiveness, product costs, treatment benefits, and effects on the healthcare system.2 These tools mark an important transition in health care toward a value-based framework. But how do we incorporate these tools to guide clinicians and patients in making value-based treatment decisions?
Before any tool is used, its associated strengths and limitations should be evaluated. Of equal importance is the definition of the value used to develop the tool. In its simplest form, value equals outcomes divided by cost.3 This vague definition leaves much to interpretation. Outcomes differ for each treatment regimen but also vary significantly from patient to patient. Take, for example, the use of high-dose interleukin-2 (IL-2) for a patient who has metastatic melanoma. The monthly cost is roughly $22,000, based on 2014 drug prices. Only 10% of patients derive a durable response to treatment, and almost 50% of patients experience grade 3–4 adverse events.4,5 Though outcomes may be acceptable to the 10% of patients who achieve a durable response and the benefits of the regimen may greatly outweigh the associated costs and toxicity experienced, the treatment would likely not have value for a patient who dies during treatment or has no response. In this case, a majority of patients are likely not to associate high-dose IL-2 with an outcome that outweighs the costs.
Faced with the need for a value-based framework tool caused by this drug-cost crisis, a number of organizations have developed scoring tools for providers and patients. The tools developed by NCCN, ASCO, and MSKCC are described in (Table 1-see PDF). All these tools offer a means to facilitate dialogue between providers and patients regarding a given regimen’s place in therapy that is customized to meet individual patients’ goals in accordance with their personal definition of value. Although components of the scoring systems vary, it is clear that cost entails more than just the exchange of money. Cost includes toxicity and loss of quality of life, among other measures. The NCCN Evidence Blocks tool has been incorporated into all previously existing guidelines and is perhaps the simplest scoring tool available but is much less specific to a given patient.6 ASCO recently adapted its net health benefit (NHB) tool in response to feedback about limitations and suggestions for improvement. The revised tool now emphasizes evidence that includes overall survival benefit and gives more weight to this benefit than, for example, to progression-free survival.7 The ASCO scoring system is complex, but comparisons of NHB score and drug acquisition cost are made graphically, allowing an easier grasp of the differences. The MSKCC DrugAbacus is probably the most complicated tool and is less personalized.3
Each tool for measuring drug value is unique, and providers may find one tool more appealing for their patients in general or for a specific patient. No matter which tool is selected, their incorporation into treatment discussions should become commonplace. In an era of expanding targeted agents and personalized oncologic medicine, and as drug toxicities are minimized and drug costs escalate, value-based discussions will become increasingly important. The place of these agents in practice (i.e., as first- or second-line treatments) and the therapeutic intent behind their use (i.e., for curative vs. palliative care) greatly affect the perceived value of each agent.
The incorporation of these tools into guidelines and clinical practice is growing. Understanding the tools’ limitations and applications is fundamental to using them in discussions with patients about treatment options. These tools may allow pharmacists and providers to make a comprehensive comparison of second- or third-line treatment options. In addition, they fulfill a crucial function in facilitating discussions of value-based care that encompass all aspects of treatment, particularly in the palliative setting. As the healthcare system continues to shift toward a value-based framework, adaptation of these tools will continue. More studies are needed regarding the use of these tools in clinical practice and multidisciplinary team recommendations. Perhaps some combination of all the available tools will prove to be the most appropriate course.
With the continuing development of oncologic therapies and understanding of indications, these tools can facilitate shared decision making about treatment that is personalized to each patient. Value-based decision tools allow therapy choices to be tailored to individual patients’ financial circumstances, goals, and preferences.
References
1. US Food & Drug Administration. Hematology/oncology (cancer) approvals & safety notifications. US Food & Drug Administration website. www.fda.gov/drugs/informationondrugs/approveddrugs/ucm279174.htm. Updated May 1, 2017. Accessed April 16, 2017.
2. Neumann PJ, Cohen JT. Measuring the value of prescription drugs. N Engl J Med. 2015;373(27):2595-97.
3. Dalzell M. Considerations for designing “value calculators” for oncology therapies. Am J Manage Care. 2016;22-29. www.ajmc.com/journals/evidence-based-oncology/2016/peer-exchange-oncology-stakeholders-summit/considerations-for-designing-value-calculators-for-oncology-therapies/P-1. Accessed April 16, 2017.
4. Amaria RN, Reuben A, Cooper ZA, Warga JA. Update on the use of aldesleukin for treatment of high-risk metastatic melanoma. Immunotargets Ther. 2015;4:79-89.
5. Akins MB, Lotze MT, Dutcher JP, et al. High-dose recombinant interleukin 2 therapy for patients with metastatic melanoma: analysis of 270 patients treated between 1985 and 1993. J Clin Oncol. 1999;17(7):2105-16.
6. National Comprehensive Cancer Network. NCCN unveils evidence blocks for CML and multiple myeloma. National Comprehensive Care Network website. https://www.nccn.org/about/news/newsinfo.aspx?NewsID=546. Updated October 16, 2015. Accessed April 16, 2017.
7. Schnipper LE, Davidson NE, Wollins DS, et al. Updating the American Society of Clinical Oncology value framework: revisions and reflections in response to comments received. J Clin Oncol. 2016;34(24):2925-34.