A Health Economic Model of Breakthrough Pain

Published Online: May 01, 2008
Amy P. Abernethy, MD; Jane L. Wheeler; and Barry V. Fortner, PhD

Although the literature adequately addresses the biologic basis, epidemiology, and management of breakthrough pain (BTP), it does not yet describe the full impact of this troubling, widespread phenomenon. The risks of a scanty understanding of BTP impact are failure to take preventive measures, underdiagnosis, undertreatment, and inappropriate management. Studies to date of the impact of BTP have followed pharmacoeconomic approaches. Building on prior efforts, this paper develops a more comprehensive health economic model that encompasses the full spectrum of costs, outcomes, risks and benefits associated with BTP and its management. The authors provide a rubric within which stakeholders— including providers, institutional leaders, administrators, and policymakers— can systematically balance the myriad potential effects of different treatment scenarios to guide decisionmaking. The paper then extends this model to the population level, providing a template for health economic analysis of alternate strategies for managing BTP, and delineating steps for accomplishing the analysis.

(Am J Manag Care. 2008;14:S129-S140)

Breakthrough pain (BTP) is an abrupt onset, transitory flare of pain occurring in the context of managed, chronic, baseline cancer pain.1 BTP builds to a moderate or severe intensity, usually peaking within 3 to 5 minutes after onset; episodes last approximately 30 minutes. To distinguish it from inadequate background analgesia, BTP is usually defined as 4 or fewer episodes in a 24-hour period.2

BTP prevalence estimates among cancer patients vary from 24% to 95%, depending on the definition of BTP and the setting from which the study sample was drawn. A 63% prevalence of BTP has been observed in patients admitted to hospice with  onmalignant terminal disease.3 This paper focuses on cancer-related BTP—the most frequently studied BTP scenario with the widest portfolio of US Food and Drug Administration–approved or developing pharmacologic interventions.

Background
Pharmacologic and Nonpharmacologic Management of BTP. A solid body of literature establishes the importance of BTP, as well as detailing strategies for its management; however, despite this available information, undertreatment remains a common phenomenon. In a study of guideline-based pain management versus standard care, DuPen et al demonstrated that a substantial reason for undertreatment of cancer pain in general is underdosing of rescue medication for BTP.4 Strategies for managing cancer-related BTP include nonopioid medications (eg, nonsteroidal anti-inflammatory drugs, acetaminophen), short-acting opioids (eg, codeine, hydrocodone, morphine, oxycodone, hydromorphone, fentanyl), and nonpharmacologic strategies (eg, ice, heat, guided imagery). Oral short-acting opioid formulations are most effective for preemptive management of BTP in patients who suffer predictable moderate-to-severe episodes and who do not respond to nonopioid or nonpharmacologic strategies. Rapid-onset transmucosal lipophilic opioids are recommended for patients with unpredictable moderate-to-severe incident or idiopathic BTP.5 Other strategies, such as radiotherapy, neurosurgical procedures, acupuncture,6,7 intrathecal and epidural infusions, neurolytic blocks, and yoga, can factor importantly into the management of cancer pain in general, and improvement in BTP is a corollary outcome in these clinical scenarios.

Impact of BTP
BTP exacts a significant toll on patients, their families, caregivers, and social networks; the healthcare system; and society at large. These consequences are physical, emotional, spiritual, social, and financial in nature. BTP in cancer patients is reported to be associated with decreased functional status, increased levels of anxiety and depression, greater dissatisfaction with opioid treatment, and poorer medical outcomes.8,9 Studies to date, however, have not yet taken a comprehensive approach to delineating the impact of BTP on patients, providers and institutions, and society. Additional work is needed to describe and enumerate the full aspects of suffering caused by BTP; a critical first step will be the development of instruments that are sensitive and specific to quality-of-life (QOL) domains differentially affected by BTP.9

Purpose
The primary purposes of this paper are as follows: (1) to summarize the costs and benefits associated with BTP and its treatment, as described to date in the cancer population; (2) to present a framework that clinicians can use to guide their decision-making for patients with BTP; and (3) to provide a model for health economic analysis, on a population level, of alternate strategies for the management of BTP, and to delineate steps for accomplishing the analysis. We begin by drawing on the pharmacoeconomic approach, which entails systematic quantification of the costs, risks, and benefits of medical interventions.10

Pharmacoeconomic Analysis: The Foundation for a BTP Health Economic Model. Most pharmacoeconomic analyses employ 1 of 4 methods: costminimization analysis, cost-effectiveness analysis, cost-benefit analysis, and cost-utility analysis. All of these approaches measure costs in monetary units, but they differ in how they value outcomes10 (Table 1). Pharmacoeconomic analyses typically categorize costs into 3 basic types—direct, indirect, and intangible. Direct costs include fixed and variable medical costs (eg, hospital capital expenses, costs of medical treatment) and nonmedical costs (eg, transportation to the clinic). Indirect costs encompass the costs of morbidity and mortality because of the illness or health event, and include lost income and time spent in the waiting room. Intangible costs comprise the toll of psychosocial states resulting from the illness or health event, such as suffering, pain, or depression.11



Once costs have been defined, pharmacoeconomic analyses proceed to assign values to cost items and outcomes, determine outcome probabilities, and compare costs with benefits. One such process, presented by Jolicoeur et al, delineates a method for conducting pharmacoeconomic analysis in 10 steps: (1) defining the problem, (2) determining the study’s perspective, (3) determining the alternatives and outcomes, (4) selecting the appropriate pharmacoeconomic method, (5) placing monetary values on the outcomes, (6) identifying study resources, (7) establishing the probabilities of the outcomes, (8) applying decision analysis, (9) discounting costs or performing a sensitivity or incremental cost analysis, and (10) presenting the results, along with any limitations of the study.10 Other structures may prove equally useful, the critical step in any such analysis being the balancing of costs against benefits. The utility of this step will depend fundamentally on the extent to which both sides of the equation thoroughly capture the impacts of the intervention.

Limitations of Pharmacoeconomic Studies to Date
Most pharmacoeconomic studies in pain have focused on the cost repercussions of chronic pain rather than on BTP. In 1994, the Cancer Pain Panel of the Agency for Healthcare Policy and Research (renamed the Agency for Healthcare Research and Quality; AHRQ) developed a 13-point framework for cost analysis related to cancer pain. This AHRQ framework covers medical costs, cost savings associated with different settings of care, morbidity-related costs, and indirect costs to patients and families and was part of a highly cited monograph on cancer pain management and best practice guidelines (now retired).12 Extending the AHRQ model, Chandler and Payne classified types of cost impact of unrelieved cancer pain into the following categories: direct medical (hospital personnel, overhead, treatment, complications), direct nonmedical (travel, hotels, meals, lost wages), indirect personal (decreased productivity, QOL), and intangible personal (pain, suffering, demoralization).13 In 2003, Fortner et al sought to quantify the costs set out in these 2 models by gathering, directly from patients, data related to the direct and indirect costs of cancer pain.14 In the same year, Abernethy et al published a clinical decision and economic analysis model of cancer pain management that compared the effectiveness and cost of 3 cancer pain management strategies—guideline- based care that incorporated the AHRQ cancer pain management guidelines, oncology-based care, and usual care.6

Overall cancer pain has been the focus of most cancer pain–related pharmacoeconomic studies; the costs of BTP, specifically, remain poorly understood. The lack of a model that depicts the true costs versus benefits of BTP management presents a very real impediment to good clinical practice, hindering consideration of cost-effectiveness and appropriate cost containment. This article builds on prior work in general cancer pain to develop a broader health economic framework that comprehensively captures the costs and other impacts of BTP in 3 key domains. This framework is intended to provide physicians with a guiding tool for clinical decision-making in BTP scenarios, thereby averting the underdiagnosis and undertreatment of BTP that are common today. It may also support institutional and national efforts to maximize cost-effectiveness and to allocate resources in a way that best optimizes patients’ QOL.

A Comprehensive Health Economic Model of BTP
Structure of the Model. Our suggested health economic framework overlays the traditional tripartite cost domains (direct, indirect, intangible) with 3 impact domains (patients, providers, society) and 3 end point domains (costs, outcomes, benefits) (Figure 1). The resulting 3 x 3 x 3 rubric offers healthcare researchers, clinicians, institutions, and consumers a rational schematic depiction of BTP costs, that also integrates the potential outcomes and benefits of BTP management, and itemizes these costs, outcomes, and benefits for the full array of stakeholders affected. It offers a tangible mechanism for organizing evidence in clinical decision-making.



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