Rethinking heart failure: patient classification and treatment.

INTRODUCTION: Approaches to treating heart failure (HF), understanding of the most timely and effective interventions, and identification of appropriate patient subpopulations must evolve. HF has emerged as a chronic condition that needs to be managed on multiple fronts. Hospital resources are more limited than ever due to various factors that directly impact staff and hospital space available to manage and treat patients with HF. As a result, there is increasing attention to the current state of this progressive disease and ways to improve patient outcomes. PURPOSE: This paper examines HF and the current and future treatment landscape, the need to reevaluate terms and definitions, and the opportunity to treat HF with the right treatment at the right time. Treatments in development and potential new investigational therapies are also discussed. CONCLUSION: To meet the current challenge, HF treatment must adapt. For other disease states, we have more personalized, nimble, and timely treatment strategies that harness windows of opportunity to help maximize outcomes and reduce overwhelming costs to the health care system. HF treatment is evolving with new guidelines and treatments that hold the promise of greater personalization through additions to existing treatments that are directed by medical guidelines, since each patient is unique and requires more than a one-size-fits-all approach. In addition, advances in remote monitoring, in-home care, and telemedicine are creating a more individualized treatment approach. Therefore, it becomes critical for all health care decision makers to be aware of the tools and resources available in treatment guidelines, individualized treatment options, telemedicine, and other ways of expanding the existing toolbox to enhance patient centricity in HF treatment.

Multiple Sclerosis: Systemic Challenges to Cost-Effective Care.

Background: Multiple sclerosis (MS) is a progressive autoimmune disorder of the central nervous system characterized by symptoms including reduced mobility, pain, fatigue, and spasticity. MS affects nearly 1 million people in the United States, with significant negative impact on a patient's quality of life, and an average lifetime cost of care in excess of $4 million. The cost-effective management of patients with MS faces several challenges. Objective: To review the challenges to the cost-effective management of patients with MS, and to offer healthcare stakeholders a roadmap to address them. Discussion: The cost-effective management of patients with MS, which is driven largely by how quickly a patient receives effective medication therapy, is challenged by a paucity of between-office-visit clinical data, variability of provider expertise with magnetic resonance imaging (MRI), MRI machine quality, lack of standards for MRI machines and reports, misaligned financial incentives, the limited number of available Current Procedural Terminology (CPT) codes for brain MRI, the complexity of disease-modifying therapy (DMT) selection, poor patient adherence to treatment plans, poor communication among providers, and a lack of objective measures of disease progression. Conclusion: Insurers, neurologists, researchers, and patient advocacy groups must address the needs of patients with MS holistically. These efforts should include establishing standards for MRI machines and reports, matching patients with MS specialists, aligning financial incentives, including creating a new CPT code for complex brain MRI, streamlining prior authorization processes of DMTs, using technology to gather patient data and improve coordination of care, and developing better measurement tools of disease activity.

Idiopathic pulmonary fibrosis: exploring the clinical and economic implications of the evolving treatment landscape.

Idiopathic pulmonary fibrosis (IPF) is the most common idiopathic interstitial pneumonia, a form of interstitial lung disease characterized by abnormal wound healing in the lung that leads to progressive scarring and loss of lung function. Comorbidities are highly prevalent in IPF and often lead to further complications and worse outcomes. In fact, undetected and untreated comorbidities are independently associated with poor outcomes. IPF not only affects patient quality of life (QOL) but also requires significant cost for delivering care. Given the potential for rapid progression of IPF and the associated risk for mortality, early diagnosis is critical for retaining the highest lung function and QOL for as long as possible. Delayed diagnosis of IPF is associated with increased costs in terms of investigations performed, and delayed referral can result in lower survival rates independent of disease severity or associated prognostic factors. Significant progress has been made in understanding IPF pathogenesis, which has, in turn, led to the development of novel therapeutic options that improve outcomes, extend life, and minimize disease burden on patients' daily lives. For patients with IPF in the absence of underlying liver disease, pirfenidone and nintedanib are licensed for the treatment of IPF. Additionally, a number of investigational therapeutic options are currently in development. The extent of clinical effectiveness compared with the cost of therapy has led to a lack of consensus on the cost-vs-benefit analyses for the drugs.

Systemic sclerosis with associated interstitial lung disease: management considerations and future directions.

Systemic sclerosis (SSc), also referred to as scleroderma, is a rare autoimmune disease associated with vasculopathy, inflammation, and fibrosis of the skin and/or internal organs. Interstitial lung disease (ILD) is a frequent complication and is the leading cause of death in patients with SSc. Although economic data are limited, available data suggest that SSc-ILD is associated with significant cost implications. Treatment of SSc-ILD has historically been with immunosuppressive agents. In 2019, however, the treatment landscape expanded with the FDA approval of the tyrosine kinase inhibitor nintedanib. A lack of codified treatment guidelines for patients with SSc-ILD creates significant challenges in improving outcomes at the patient level and, more generally, in reducing disease burden to the health care system. As the treatment landscape continues to evolve, it is likely that to reduce lung volume loss in patients, a combination of immunosuppressive and antifibrotic approaches will need to be used. Additionally, a greater emphasis on risk-stratification strategies may allow for more efficient follow-up, monitoring, and assessment of treatment response.

The spectrum of progressive fibrosis interstitial lung disease: clinical and managed care considerations.

Progressive fibrosing interstitial lung diseases (ILDs) encompass a wide range of diseases, including hypersensitivity pneumonitis, occupational diseases, granulomatous diseases, drug-induced diseases, and idiopathic pneumonitis. Given the vast number of progressive fibrosing ILDs and the disparities in clinical patterns and disease features, understanding their clinical and economic impact presents significant challenges. Historically, treatment options for progressive fibrosing ILDs include anti-inflammatory drugs and immunosuppressive. The lack of effective options and guideline recommendations, however, has rendered treatment difficult. In March 2020, nintedanib was approved by the FDA for the treatment of patients with chronic fibrosing ILDs with a progressive phenotype, becoming the first therapeutic agent to receive an indication for this set of diseases. The approval was based on data from the phase 3 randomized, double-blind, placebo-controlled, parallel-group INBUILD trial. Questions regarding the cost of medications, their effects on disease and comorbidities, patient selection, and combination strategies remain to be answered.

The economic burden and managed care implications of chronic inflammatory demyelinating polyneuropathy.

Chronic inflammatory demyelinating polyneuropathy (CIDP) is a symmetric demyelinating peripheral neuropathy with either a progressive or relapsing remitting course. CIDP is both debilitating and degenerative, leaving patients with functional impairment due to nerve damage in their extremities. Along with its medical burden, CIDP has a significant economic impact, with disease-related expenses and the often fairly high costs related to the immune therapies used to treat the disease. Costs for patients with this disorder often exceed those of other immune-mediated neuromuscular diseases. For these reasons, it is necessary for clinicians to better determine optimal long-term treatment strategies for patients with CIDP that are designed to address both the clinical and economic burden of its management and improve patient outcomes and quality of life.

Economic Impact of Increased Utilization of Multivariate Assay Testing to Guide the Treatment of Ovarian Cancer: Implications for Payers.

BACKGROUND: Ovarian cancer is the eighth most common cancer among women, but ranks fifth in cancer-related causes of death, the majority of which are detected in late stages, after the cancer has metastasized. The CA125 test is the standard of care for assessing suspicious pelvic masses. However, the primary use of CA125 is to monitor treatment progress rather than to screen for disease, and its sensitivity is exceedingly low, unlike the multivariate assay OVA1. A cost-effective treatment of ovarian cancer requires early and accurate diagnosis of pelvic masses and reduced referrals of patients with benign tumors to a gynecologic oncologist. OBJECTIVE: To analyze the economic impact of increased utilization of a multivariate assay, such as OVA1, to guide the treatment of ovarian cancer. METHODS: The study population was drawn from Medicare and commercial health plan claims data. A budget impact model was constructed to estimate the economic consequences of substituting the multivariate assay OVA1 to replace the single biomarker assay CA125 to assess the likelihood of pelvic mass malignancy in premenopausal and/or postmenopausal women. All patients selected for the analysis had CA125 testing before surgical intervention. RESULTS: A total of 92,843 health plan members were included for analysis, comprising 48,113 commercially insured members and 44,730 Medicare beneficiaries. Estimates of future health plan expenditures, which were calculated from base-case assumptions, projected overall savings of $0.05 per-member per-month (PMPM) for commercially insured members and $0.01 PMPM for Medicare beneficiaries as a result of increased utilization of OVA1. Sensitivity analysis revealed potential savings of up to $0.17 PMPM for commercially insured patients and up to $0.05 for Medicare beneficiaries. CONCLUSION: The results of the budget impact model support the use of OVA1 instead of CA125 by indicating that modest cost-savings can be achieved, while reaping the clinical benefits of improved diagnostic accuracy, early disease detection, and reductions in multiple, and possibly unnecessary, referrals to gynecologic oncologists.

Strategies to manage costs in idiopathic pulmonary fibrosis.

Idiopathic pulmonary fibrosis (IPF) is a diagnostically challenging disease. Clinicians are faced with the need to exclude alternative diagnoses, limited treatment and management guidelines, and few treatment options. Patients with IPF have significantly increased healthcare usage compared with similar patients without the disease. Medicare estimates for this disease are as high as $3 billion, not including cost of treatment. The disease, characterized by worsening dyspnea, declining lung function, nonspecific respiratory symptoms, and a varied clinical course randomly punctuated by episodes of acute exacerbations, is also accompanied by a host of comorbid conditions that contribute significantly to increased healthcare usage and cost. The comorbidities, which increase impairment and disability, and compromise patient quality of life and survival, include pulmonary and cardiac conditions, sleep apnea, gastroesophageal reflux disease, depression and anxiety, and lung cancer. Until recently, palliative care and lung transplant were the only options for management of IPF. Without a lung transplant, the median survival was estimated at 3 to 5 years from the initial diagnosis. Newer treatments, pirfenidone and nintedanib, demonstrate a modest effect on slowing decline in lung function in patients with IPF. Both were approved for the treatment of IPF in 2014. As potentially effective therapies emerge, attention should be given to healthcare resource usage and healthcare processes that ensure patient-centered management with sustainable, cost-effective, and quality care. As such, it is imperative that a structured, comprehensive, multidisciplinary management approach is used in the treatment and management of IPF and its associated comorbidities to limit costs and provide effective and quality healthcare.

Economic burden of multiple sclerosis and the role of managed sare organizations in multiple sclerosis management.

Multiple sclerosis (MS) is disease that has an early age of onset and may intensify and subside with disease relapses or exacerbations interrupted by periods of stability. Because of this, patients, their families and caregivers, employers, and the entire healthcare system carry substantial clinical and economic burdens associated with the disease over of a period of many years. Although most patients with MS are covered by health insurance, the management landscape has become increasingly complex over the past decade with the introduction and approval of several new disease-modifying therapies that, while remarkably effective and well tolerated, usually come with a very high cost. Whereas the main goal of treating patients with MS is to prevent disease progression and disability, healthcare and benefit providers are faced with an ever-tipping balance point between effectively managing the disease and maximizing the value of high-cost disease-modifying therapies in an already overburdened healthcare system. Treatment of MS should be individualized, and shared decision making between patients and healthcare providers must be preserved. Healthcare providers and payers need to collaborate to ensure that resources are used optimally and not wasted, reducing both the clinical and economic burdens related to this complex chronic disorder.

Optimizing Rheumatoid Arthritis Therapy: Using Objective Measures of Disease Activity to Guide Treatment.

BACKGROUND: Rheumatoid arthritis (RA) affects approximately 1.5 million individuals in the United States, or approximately 1% of the US adult population. In women, RA most often begins between age 30 and 60 years; in men, it often starts later in life. Patients with RA may have rapid declines in physical function that can begin early in the disease course. Disability increases most rapidly during the early years of the disease course, and if patients are not accurately diagnosed and do not receive appropriate care early, substantial functional declines may result. OBJECTIVE: To review strategies and clinical assessment tools that may optimize patient outcomes by using objective measures of disease activity. DISCUSSION: The goal of treatment for patients newly diagnosed with RA should be preventing joint damage from developing by employing early and aggressive approaches to therapy that minimize disease activity. Likewise, for established disease, treatment should be aimed at limiting the progression of existing joint damage. Substantial advances have been made in the treatment of RA over the past 2 decades, in large part as a result of better understanding of the biology of RA and the resultant introduction of biologic therapies. In 2010, an international task force published recommendations for a treat-to-target management approach to RA, much of which was based on the use of biologic drugs. This treatment strategy emphasized that the primary target in the treatment of patients with RA should be clinical remission or low disease activity. The tools necessary to measure RA disease activity are often incomplete, imprecise, or rely on a combination of physician and patient subjective evaluations. There is no one symptom, laboratory measure, or clinical tool that provides a truly accurate assessment of disease activity in patients with RA. CONCLUSION: Thus, there is a large gap between what is recommended in clinical guidelines and the actual practice of rheumatologists. Better methods of assessing RA disease activity are still needed to enable widespread adoption of guidelines in the clinical community.

Revolutionizing treatment outcomes in hepatitis C: managed care implications and considerations--the new and evolving standards of care.

Although the prevalence of hepatitis C virus (HCV) infection is declining, overall costs associated with HCV infection and the burden of advanced liver disease are projected to increase. The recent approval of all-oral, fixed-dose combination treatments for patients with HCV infection has resulted in unprecedented rates of treatment success, and in debate regarding treatment costs and appropriation. With all-oral therapies becoming the standard of care for HCV infection, high drug costs and improved clinical outcomes-now including the eradication of disease-must be weighed when selecting the most appropriate therapy. Patient "warehousing" has reached an all-time high as payers and providers strive to strike the fine balance between clinical efficacy and cost-effectiveness of currently available treatments, and this "wait and see" period may very well continue until an acceptable balance has been achieved. As such, it is imperative that managed care clinicians maintain an informed understanding of the disease burden and current climate of HCV infection in the United States.

Managed care implications in managing rheumatoid arthritis.

Rheumatoid arthritis (RA) is a systemic inflammatory form of arthritis characterized by joint inflammation, pain, swelling, and stiffness. While contemporary treatment strategies based on early diagnosis, aggressive treatment, and regular monitoring have helped a significant number of patients achieve evidence-based treatment goals, RA still presents substantial management challenges to both clinicians and patients, and has the potential to lead to severe disability over time. In addition to its significant clinical consequences, RA has important economic implications. Both direct and indirect medical costs associated with RA are significant, including costs of medications, ambulatory and office-based care, and quality-of-life and productivity costs. In addition, a significant proportion of patients with prevalent RA have associated cardiovascular disease and other comorbidities, further compounding healthcare costs and complicating management of this disorder. Clinically favorable and cost-effective management must focus on prevention of disease progression and the improved patient health status and productivity than can result from optimal disease control. With the myriad of treatment options both available and emerging, managed care organizations are faced with difficult decisions surrounding the most clinically and cost-effective allocation of treatments designed to improve disease outcomes for patients with RA. It is vital that managed care clinicians and providers analyze both the overall burden and the specific costs of RA. This will allow a better understanding of how costs and issues relating to healthcare utilization affect the treatment of patients with RA and impact individualized therapy, care coordination, and outcomes.

Improving transitions of care for patients with thromboembolic disease.

Patients with or at risk for thromboembolic disease have many transitional interactions within the healthcare system. Transitions of care--when patients move between or within sites of care, or transition from inpatient to outpatient status--create repeated and diverse opportunities for medication errors, rehospitalization, and other adverse events that may increase costs. Although effective antithrombotic therapies are available, these therapies are complex, underprescribed, and frequently suboptimally managed, a situation further exacerbated by poor patient adherence to therapy. Physician and patient education may help address knowledge gaps related to antithrombotic therapy to help ensure that patients receive appropriate therapy and adhere to the therapeutic regimen. Due to the complexities of antithrombotic therapy it is not surprising that when these patients experience transitions of care, the potential for errors and suboptimal outcomes becomes compounded. Efforts are under way to improve the process of transitional care, including the development of protocols for medication reconciliation, improved communication between clinicians at hand-off, the use of electronic medical records, and the introduction of a collaborative approach among different types of healthcare providers, including pharmacists, nurses, and care managers, so that transitional care is provided smoothly and safely.

Cost effectiveness of radiofrequency microremodeling for stress urinary incontinence.

INTRODUCTION AND HYPOTHESIS: Stress urinary incontinence (SUI) is a common and growing problem among adult women and affects individuals and society through decreased quality of life (QoL), decreased work productivity, and increased health care costs. A new, nonsurgical treatment option has become available for women who have failed conservative therapy, but its cost effectiveness has not been evaluated. This study examined the cost effectiveness of transurethral radiofrequency microremodeling of the female bladder neck and proximal urethra compared with synthetic transobturator tape (TOT), retropubic transvaginal tape (TVT) sling, and Burch colposuspension surgeries for treating SUI. METHODS: A Markov model was used to compare the cost effectiveness of five strategies for treating SUI for patients who had previously failed conservative therapy. The strategies were designed to compare the value of starting with a less invasive treatment. The cost-effectiveness analysis was conducted from the health care system perspective. Efficacy and adverse event rates were obtained from the literature; reimbursement costs were based on Medicare fee schedule. The model cycle was 3 months, with a 3-year time horizon. Single-variable sensitivity analyses were conducted to assess stability of base-case results. RESULTS: Two of the five strategies employed the use of transurethral radiofrequency microremodeling and achieved 17-30 % lower mean costs relative to their comparative sling or Burch strategies. CONCLUSIONS: Superior safety and cost effectiveness are recognized when patients are offered a sequential approach to SUI management that employs transurethral radiofrequency microremodeling before invasive surgical procedures. This sequential approach is consistent with treatment strategies for other conditions and offers a solution for women with SUI who want to avoid the inherent risks and costs of invasive continence surgery.

Implications for multiple sclerosis in the era of the Affordable Care Act: the shifting managed care landscape.

Multiple sclerosis (MS) is a disorder of the central nervous system that is associated with disability, reduced quality of life, extensive medical and nonmedical costs, and lost productivity. Specialty medications that are crucial to effective disease management, helping to prevent debilitating episodes of relapse, account for a substantial portion of the medical expenditures associated with MS. Although these therapies are not considered cost-effective by conventional definitions, they are comparable to one another in cost-effectiveness estimates, leaving the complex task of designing cost-efficient formulary management strategies to managed care professionals. Current epidemiologic data suggest that most patients with MS are covered by some form of healthcare insurance, but plan designs and formulary restrictions may still create access barriers for some patients. The Affordable Care Act (ACA) is recent federal legislation that seeks to provide new consumer protections, improve healthcare quality and accessibility while mitigating expenditures, and increase accountability of healthcare insurance companies. The impact of the ACA on specialty pharmaceuticals is unclear at this time, but it does appear to have already begun improving healthcare coverage across the population. Managed care professionals must work within the confines of the ACA to provide better and more affordable care that targets overall cost reductions rather than just pharmacy expenses.

Perspectives for managed care organizations on the burden of multiple sclerosis and the cost-benefits of disease-modifying therapies.

Disease-modifying therapies (DMTs) are a core component of multiple sclerosis (MS) management. Given current constraints on health care expenditures, the relative cost-effectiveness of these therapies needs to be considered when making treatment decisions. The objective of this article is to review the burden of illness of MS, discuss the cost-effectiveness data for DMTs, and summarize the implications for payers. For the burden of illness in MS, a retrospective analysis of managed care administrative data from the IMS LifeLink Health Plan Claims Database was performed. Data from claims submitted for patients with confirmed MS (ICD-9-CM code 340) over a period of 1 year (2009) were analyzed. A literature review was conducted to put these data into perspective. The retrospective analysis determined that the mean annual cost of treating MS in the United States in 2009 was $23,434, which varied according to the presence of comorbidities/complications. Overall, DMTs accounted for 69% of the total costs of managing the disease. According to the literature review, the typical first-line DMTs (interferon beta [IFNß] formulations and glatiramer acetate [GA]) are generally associated with incremental cost-utility or cost-effectiveness ratios in excess of $100,000 per quality of life year gained. Natalizumab may have cost benefits over other agents in patients with more aggressive disease. According to the available data, studies indicate that DMT cost-effectiveness (specifically cost per quality-adjusted life years) appears to improve with treatment initiation during the early stages of the disease. In relapsing-remitting MS, there is currently little evidence to differentiate between the DMTs that are typically used first-line (IFNs and GA) based on cost-effectiveness or cost-utility studies. Presently, optimal therapy decisions for DMT-naïve patients are likely to be made individually based on patient and provider preference, adherence, and medication risk-benefit profiles. For patients with more advanced disease, natalizumab appears to have greater efficacy and to be more cost-effective than other agents.

Translating clinical guidelines into practice: the effective and appropriate use of human growth hormone.

There are 9 recombinant human growth hormone (rhGH) products currently available for 10 US Food and Drug Administration-approved indications; each rhGH product is approved for 1 or more indications. Adult and pediatric patients with the various conditions for which rhGH is indicated, from idiopathic short stature (ISS) and growth hormone (GH) deficiency to short bowel syndrome and HIV/AIDS wasting, may benefit from rhGH treatment. In clinical practice, pediatric patients with GH deficiency or ISS make up the majority of the population receiving treatment with rhGH. Most rhGH products are provided through specialty pharmacies that often have to balance the needs of the patient, their own utilization objectives, and the availability of the rhGH on formulary from a particular payer. Often, a payer will prefer only 2 or 3 rhGH products to cover all 10 indications. As such, managed care professionals need to be more informed about the options available and should be familiar with the different indications to help educate patients about treatment. Additionally, healthcare providers should endeavor to identify and manage the care of appropriate patients who would potentially benefit from rhGH therapy, and should be aware of formulary options. Because many of the patients are children and young adults, adherence to treatment is a concern; patient education on the importance of treatment adherence should be ongoing. Various mechanisms are in place (eg, prior authorization requirements and case manager follow-up) to help ensure that rhGH products are used, and used appropriately. This publication includes highlights from a roundtable discussion by key opinion leaders (clinicians and managed care professionals) on how managed care policies and clinical guidelines on appropriate use of rhGH translate into real-world practice. Also discussed are the efficacy and safety of rhGH therapy for its pediatric indications, and the role of specialty pharmacies in managing patient access to therapy.

Managed care aspects of managing multiple sclerosis.

Multiple sclerosis (MS) is a chronic disease of the central nervous system usually diagnosed in the second or third decade of life; MS is more common among women than men by a ratio of 3 to 1. With its relatively early age of onset and symptoms that impair patients' quality of life, MS requires lifelong, dynamic treatment, and places a substantial economic burden on individuals, healthcare systems, and society. The costs associated with providing benefits for MS therapy are growing rapidly and the increasing complexity of the MS market is impacting disease management for payers. Employers are also increasingly aware of the costs associated with MS and are asking health plans to advise on the most appropriate and cost-effective ways to manage both pharmacologic and non-pharmacologic therapies for MS. Health plans, by necessity, must therefore balance appropriate access to treatments for MS with the need to manage rising treatment costs. To meet this goal, payers require population-based solutions, guidelines, and treatment algorithms for the management of MS that can be used in clinical and formulary management decision making in the context of an evolving therapeutic landscape. Further, comparative studies are necessary for payers to determine which agents may work best on a population basis. Due to the current lack of appropriate clinical guidance and insufficient head-to-head data on disease-modifying drugs, strategies for health plans and clinical management have been designed using the best available evidence. Undoubtedly, management of this class will continue to evolve with the launch of newer agents.