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Supplements Cost-Effectiveness of Disease- Modifying Therapies in Multiple Sclerosis: A Managed Care Perspective
Overview and Diagnosis of Multiple Sclerosis
Samuel F. Hunter, MD, PhD
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Economic Burden of Multiple Sclerosis and the Role of Managed Care Organizations in Multiple Sclerosis Management
Gary M. Owens, MD
Cost-Effectiveness of Disease- Modifying Therapies in Multiple Sclerosis: A Managed Care Perspective
Cost-Effectiveness of Disease-Modifying Therapies in Multiple Sclerosis: A Managed Care Perspective

Economic Burden of Multiple Sclerosis and the Role of Managed Care Organizations in Multiple Sclerosis Management

Gary M. Owens, MD
Costs and Cost-Effectiveness of Pharmacotherapy

     Pharmacotherapy contributes substantially to the overall healthcare costs for patients with MS.20 DMTs are agents designed to alter the natural course of disease in MS. They reduce the frequency and severity of attacks and the development of new brain lesions, and slow down the development of disability. Initial biologic agents for this purpose were produced and marketed in
the early 1990s. At that time, prices ranged from approximately $9000 to $12,000 per patient annually. Multiple new and potentially more effective agents have been developed since then; however, these agents come with higher prices, and the pipeline of therapies continues to grow, adding not only to the potential for greater efficacy, but also for significantly higher costs added to the overall
management of MS.24
     The landscape for the management of MS with DMTs has changed dramatically since these drugs were introduced, most notably within the last decade with the approval of several new therapies.25 Currently, there are 12 DMTs approved for use by the FDA in patients with relapsing forms of MS, including secondary-progressive MS for patients who are still experiencing relapses. One is also approved specifically for secondary-progressive MS; however, none of these agents or any others has been approved to treat primary-progressive MS, the type that shows steady progression from disease onset.26

Although DMTs have been proven to be very effective in managing MS per their indications, the costs of these agents remain a subject of much discussion, debate, and even controversy. Despite the development and emergence of newer DMTs for patients with relapsing forms of MS, costs for all DMTs approved by the FDA have increased markedly. The average annual DMT cost per patient with MS in the United States in 2004 was $16,050, comprising approximately half of all direct medical costs for patients with the disease. In comparison, the current average annual cost for the agent interferon (IFN) beta-1b was greater than $60,000, as noted by Hartung et al in 2015. The costs of first-generation DMTs, including IFN beta-1b, IFN beta-1a intramuscular, and glatiramer acetate have risen an average of 21% to 36% annually. Costs of the most recently FDA-approved drugs (eg, fingolimod, teriflunomide, and dimethyl fumarate) have increased 8% to 17% per year since these agents were initially approved for use in patients with MS. Compared with these agents, general and prescription drug inflation increased only 3% to 5% per year during the same time period. Overall, the costs of DMTs in the United States have increased annually at rates 5 to 7 times higher than prescription drug inflation, and substantially above rates for other drugs in similar biologic classes.25

These costs substantially impact patients with MS in several ways. As noted earlier, health insurance carriers have developed tiered formularies. Additionally, another management activity by payers is to require step-therapy trials of DMTs for patients. Because many of the current agents are considered therapeutically equivalent by payer pharmacy and therapeutics committees, the formulary status of an individual agent may be determined by pricing contracts.25-27 Improving clinical outcomes and quality of life for patients with MS may be the overriding priority for health insurance carriers and plans, but decision makers within these plans are now faced with the growing complexity in the MS space with the emergence of new DMTs.

     In addition to tiered formularies and step-therapy management, common current health plan strategies for managing the optimal utilization of agents for MS include: (1) approving initial therapy with 1 or more preferred first-line treatment agents, and (2) prior authorizations that delineate drug appropriateness, the care setting, and the expected duration of therapy. Case-based supervision of each patient is critical, and patient compliance with therapy is a crucial aspect of individualized management.28

The improved efficacy and safety profiles of these newer agents may provide an invaluable benefit to patients with MS; however, their access to these treatments may become limited by costs. On the other hand, ensuring early and appropriate treatment initiation may alleviate some of the costs down the road due to disease exacerbations and progression. Whereas the costs of therapy are high and seem ever-escalating, use of these agents must be assessed in terms of cost-effectiveness. Several analyses have been performed and suggest that the percentage of total (direct and indirect) costs surrounding MS that are attributable to DMTs has risen from 34%, as initially reported by Kobelt et al in 2006, to between 69% and 75% (as noted earlier) of total costs in the current decade.18,19,29,30

Outside of the United States, governmental medical oversight institutions often use quality-adjusted life-years (QALYs) to compare different medications and measure their clinical effectiveness, and determine cost per QALY as a measure of the cost-effectiveness of therapy.18,31,32 In the United States, a cost per QALY value of $50,000 is often put forth as a financial threshold for cost-effectiveness. 18,33,34 In 2011, Noyes et al used data from a longitudinal MS survey to 10-year generate disease-progression paths for a group of patients with MS. Medical costs were estimated using the reimbursement rates from CMS and other sources. Outcomes were measured as gains in QALYs and relapse-free years. Results demonstrated that using DMTs for 10 years provided moderate health gains compared with treatment without a DMT. However, the cost-effectiveness of all DMTs in this analysis was greater than $800,000 per QALY.

     Reducing the cost of DMTs had the most significant impact on the cost-effectiveness of these drugs. For example, a cost reduction of 67% could improve the probability of 1 agent being cost-effective at $164,000 per QALY to 50%. However, when compared with treating patients at all levels of MS disease severity, the study also found that starting patients on DMT earlier was associated with a lower and more favorable incremental cost-effectiveness ratio. This analysis emphasized the need for early initiation of DMT, preferably when patients with MS are at an EDSS score of 2 or less, as this may be a more cost-effective approach versus starting DMT at later disease stages.31

In comparison, Owens et al performed a retrospective analysis in 2013 of managed care administrative data from a health plan claims database, using claims submitted over a period of 1 year for patients with confirmed MS. This study demonstrated that the mean annual cost of managing MS in the United States in 2009 was $23,434, with some variance depending upon the presence of
comorbidities or complications in the individual patients analyzed. DMTs accounted for 69% of the total costs of disease management and were associated with high incremental cost-effectiveness, ranging widely from $20,000 to more than $1 million per QALY. However, as seen in the Noyes et al analysis, cost-effectiveness improved by initiating treatment during earlier stages of disease.18,31
     The costs of DMTs may be partially offset by their effectiveness in preventing relapses.18,35 In addition, the ultimate goal of therapy for MS is the prevention of disability, which would benefit both the patient and society. The overall costs of MS have been shown to rise significantly with increasing disease progression and severity as measured by EDSS scores. Such cost increases were driven by relapses and productivity costs more than the actual direct costs of DMTs.18,21 With the emergence of new DMTs and therapy strategies, cost-effectiveness studies remain an evolving process. In addition, most health benefit plans are now being designed to require cost sharing by the patient.28

Higher cost sharing on the part of the patient is also a complex issue. Patients are facing ever-increasing financial burdens for treatments because of higher copayments, multitier drug formularies, and the adoption of prescription coinsurance plans. Data suggest that patients may forgo treatment or end treatment prematurely when their cost-sharing burden is higher. In addition, benefit plan designs that negatively impact treatment initiation and adherence may increase health resource use, the risk of relapse, and disease progression and risk of disability. More data are needed to evaluate both the cost-effectiveness of newer and emerging treatments and the impact of cost sharing in the management of MS with DMTs.36

      The current treatment landscape may also be altered in the future with the approval of generic versions of DMTs for MS. In April 2015, the FDA approved the first generic version of glatiramer acetate, an early-generation DMT for MS. This generic became available to the market in June 2015. Although there have been no human studies on this particular version of this DMT, another unapproved generic of this drug was compared with the proprietary version of the agent and demonstrated noninferiority.37


Costs of MS to Employers, Caregivers, and Society

Indirect costs associated with MS can also be substantial, and include lost productivity, health impact on the family, and the economic effects that the disease can place on families and caregivers. Patients with MS frequently have difficulty continuing to work. Data suggest that lost productivity related to loss of employment or the need to retire early may be the largest single factor that contributes to the nonmedical financial cost of MS. The Kobelt et al study from the United States found that among patients with MS taking DMTs, just 41% were working, with 63% of these patients working full time. Approximately one-fifth of these patients changed their work or reduced their working hours, however. In addition, 31.5% took early retirement because of their MS, and an additional 10.9% reported that they ceased working without indicating receipt of any form of early retirement benefits or pension.29,38 Loss of income for patients with MS is also usually linked to a corresponding increase in disability claims to governmental benefit programs and insurance providers.37

Patients with MS also have substantially increased use of long- and short-term sick leave, with this leave often associated with disease relapses.21,38 A US analysis showed that employees with MS had a higher rate of medicallyrelated absenteeism and associated absenteeism days than those without the disease. Employees with MS had more than 6 times the number of sick-leave days compared with employees without MS. Also, the annual costs for disability were 9 times higher for employees with MS, while indirect costs, including the loss of earnings due to the use of unpaid leave, were more than 4 times higher for the MS cohort versus employees without MS.38,39

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