Biosimilar underutilization alone does not foretell a broken biologics market.

Biosimilars offer the potential for cost savings and expanded access to biologic products; however, there are concerns regarding the rate of biosimilar uptake. We assessed the relationship between biosimilar and originator pricing, coverage, and market share by describing four case studies that fall into two categories: (1) sole preferred coverage strategy (ie, aim is to have originator product preferred; biosimilar(s) non-preferred), defined as steep average sales price (ASP) reductions for originator products (decline in net prices by at least 50% following the introduction of biosimilar competition by 2022) and (2) non-sole preferred coverage strategy (ie, aim is to have originator product preferred alongside biosimilar products), defined as moderate ASP reductions for originator products with (net prices did not decline by at least 50% of its pre-biosimilar competition value). We found that originators with sole preferred coverage strategies maintained formulary preference and market share relative to originators with non-sole preferred coverage strategies. Regardless of strategy, the market-weighted ASP for all four product families (originator and biosimilars) declined significantly in the years following the introduction of biosimilars, suggesting that biosimilar uptake alone may not be a complete measure of whether the biosimilar market is facilitating competition and lowering prices.

Balancing Evidence and Need: Variation in US Commercial Payer Coverage of Esketamine.

PURPOSE: Variations in US commercial health plan coverage policies affect how patients access medications. Plans may vary in treatment access criteria, line of therapy, and prescriber requirements. In this study, we examined coverage of esketamine hydrochloride (Spravato) for major depressive disorder (MDD) and treatment-resistant depression (TRD) to answer the following question: how do US commercial health plans cover esketamine, and how do they guide prompt patient access to the drug? METHODS: We used information from the Tufts Medical Center Specialty Drug Evidence and Coverage database, which includes coverage policies issued by 18 large commercial health plans in the United States. Esketamine coverage policies for MDD and TRD active in December 2022 were collated and analyzed. We compared coverage policies according to step therapy protocols, patient subgroup restrictions, and prescriber requirement criteria, evaluating patient access using the number of restrictions and proportion of plans including each criterion. FINDINGS: Plans more often imposed step therapy requirements for access to esketamine for TRD than for MDD, with line of treatment of ≤9 steps for MDD compared with 1 to 5 steps for TRD. Plans also varied with respect to the therapies they required patients to first try and experience treatment failure before granting access to esketamine for both indications. Clinical coverage requirements varied in thresholds and rating scales used to assess severity of depressive symptoms. IMPLICATIONS: Plans vary in terms of line of therapy and clinical coverage requirements for access to esketamine. Variation in health plan coverage policies may result in inequitable access and added complexity for patients and clinicians navigating care, which may delay access to urgent treatment. GOV IDENTIFIERS: Not applicable.

US commercial health plan coverage dynamics in pulmonary arterial hypertension therapies.

BACKGROUND: Health plan coverage is central to patient access to care, especially for rare, chronic diseases. For specialty drugs, coverage varies, resulting in barriers to access. Pulmonary arterial hypertension (PAH) is a rare, progressive, and fatal disease. Guidelines suggest starting or rapidly escalating to combination therapy with drugs of differing classes (phosphodiesterase 5 inhibitors [PDE5is], soluble guanylate cyclase stimulators [sGC stimulators], endothelin receptor antagonists [ERAs], and prostacyclin pathway agents [PPAs]). OBJECTIVE: To assess the variation in commercial health plan coverage for PAH treatments and how coverage has evolved. To examine the frequency of coverage updates and evidence cited in plan policies. METHODS: We used the Tufts Medical Center Specialty Drug Evidence and Coverage database, which includes publicly available specialty drug coverage policies. Overall, and at the drug and treatment class level, we identified plan-imposed coverage restrictions beyond the drug's US Food and Drug Administration label, including step therapy protocols, clinical restrictions (eg, disease severity), and prescriber specialty requirements. We analyzed variation in coverage restrictiveness and how coverage has changed over time. We determined how often plans update their policies. Finally, we categorized the cited evidence into 6 different types. RESULTS: Results reflected plan coverage policies for 13 PAH drugs active between August 2017 and August 2022 and issued by 17 large US commercial health plans, representing 70% of covered lives. Coverage restrictions varied mainly by step therapy protocols and prescriber restrictions. Seven plans had step therapy protocols for most drugs, 9 for at least one drug, and 1 had none. Ten plans required specialist (cardiologist or pulmonologist) prescribing for at least one drug, and 7 did not. Coverage restrictions increased over time: the proportion of policies with at least 1 restriction increased from 38% to 73%, and the proportion with step therapy protocols increased from 29% to 46%, with generics as the most common step. The proportion of policies with step therapy protocols increased for every therapy class with generic availability: 18% to 59% for ERAs, 33% to 77% for PDE5is, and 33% to 43% for PPAs. The proportion of policies with prescriber requirements increased from 24% to 48%. Plans updated their policies 58% of the time annually and most often cited the 2019 CHEST clinical guidelines, followed by randomized controlled trials. CONCLUSIONS: Plan use of coverage restrictions for PAH therapies increased over time and varied across both drugs and plans. Inconsistency among health plans may complicate patient access and reduce the proportion who can persist on PAH treatments.

Variation in Medicaid and commercial coverage of cell and gene therapies.

Growth in the availability of cell and gene therapies (CGTs) promises significant innovation in the treatment of serious diseases, but the high cost and one-time administration of CGTs has also raised concern about strain on health plan budgets and inequity in access. We used coverage information from the Tufts Medical Center Specialty Drug Evidence and Coverage (SPEC) database for 18 large commercial health plans in the US and information from state Medicaid websites to examine variation in coverage of 11 CGTs in August 2021. We found that US commercial and Medicaid health plans imposed restrictions in 53.5 % and 68.3 % of their coverage policies for the 11 included CGTs, respectively. In addition, we identified significant variation in access to CGTs across commercial plans and across Medicaid plans. Coverage restrictions for certain CGTs were more common than others; clinical requirements were often (but not always) consistent with the inclusion criteria for the clinical trial central to the drug's approval. We conclude that there is variation in access to CGTs, creating differential patient access.

Commercial coverage of specialty drugs, 2017-2021.

Health plans guide their enrollees' access to specialty drugs through coverage policies. We examined a set of health plan policies to determine if they have become more or less stringent over time. We did so by comparing the consistency of policies with Food and Drug Administration (FDA) label indications. We considered coverage policies for the same 187 specialty drugs issued by 17 large US commercial health plans from 2017 through 2021. Overall, the proportion of policies that were consistent with the FDA label declined from 57.1% in 2017 to 45.1% in 2021; the proportion of policies that were more restrictive than the FDA label increased from 39.5% to 51.7%. The proportion of policies excluding drug coverage remained approximately constant (3.4% in 2017; 3.2% in 2021). Trends in coverage restrictiveness varied across plans. For 13 plans, the proportion of policies with restrictions increased over time, while for 4 plans it declined.

Cost-effectiveness of using N-terminal pro-brain natriuretic peptide to guide the diagnostic assessment and management of dyspneic patients in the emergency department.

The cost-effectiveness of N-terminal pro-brain natriuretic peptide (NT-pro-BNP) in dyspneic patients in emergency departments (EDs) is unknown. The objective of this study was to assess the cost-effectiveness of NT-pro-BNP testing for the evaluation and initial management of patients with dyspnea in the ED setting. A decision model was developed to evaluate the cost-effectiveness of diagnostic assessment and patient management guided by NT-pro-BNP, compared with standard clinical assessment. The model includes the diagnostic accuracy of the 2 strategies for congestive heart failure and resulting events at 60-day follow-up. Clinical data were obtained from a prospective blinded study of 599 patients presenting to the ED with dyspnea. Costs were based on the Massachusetts General Hospital cost accounting database. The model predicted serious adverse events during follow-up (i.e., urgent care visits, repeat ED presentations, rehospitalizations) and direct medical costs for echocardiograms and hospitalizations. NT-pro-BNP-guided assessment was associated with a 1.6% relative reduction of serious adverse event risk and a 9.4% reduction in costs, translating into savings of $474 per patient, compared with standard clinical assessment. In a sensitivity analysis considering mortality, NT-pro-BNP testing was associated with a 1.0% relative reduction in post-discharge mortality. The optimal use of NT-pro-BNP guidance could reduce the use of echocardiography by up to 58%, prevent 13% of initial hospitalizations, and reduce hospital days by 12%. In conclusion, on the basis of this model, the use of NT-pro-BNP in the diagnostic assessment and subsequent management of patients with dyspnea in the ED setting could lead to improved patient care while providing substantial cost savings to the health care system.

A cost-effectiveness analysis of a hypothetical catheter-based strategy for the detection and treatment of vulnerable coronary plaques with drug-eluting stents.

AIM: Extensive efforts are underway to develop methods for the detection and treatment of vulnerable/high-risk coronary artery plaques. We utilized decision analysis to evaluate the hypothetical clinical benefits and cost-effectiveness of a catheter-based strategy. METHODS AND RESULTS: Currently, stenotic coronary plaques are treated without regard to vulnerability. In a new strategy, vulnerable coronary plaques are detected with a catheter-based test and treated with a drug-eluting stent, regardless of degree of stenosis. A Markov-decision model was developed to compare the new strategy with current practice. Monte Carlo simulations were performed from a societal perspective, costs were converted to year 2003 U.S. dollars, and future costs and outcomes were discounted at 3%. Sensitivity analyses were performed to evaluate the effect of assumptions on variables such as the prevalence of vulnerable plaques and treatment effect. In 60-year-old male patients with coronary stenoses the new strategy would be less expensive and more effective than current practice (37,045 dollars vs 38,257 dollars and 10.23 vs 9.86 quality-adjusted life years (QALYs), respectively). The benefits of the new strategy were robust in sensitivity analyses (e.g., if the prevalence of vulnerable plaques in this patient group was 50% or more and the sensitivity and specificity of the new test were at least 0.80). CONCLUSION: In selected patients with coronary artery stenosis, the detection of vulnerable plaques with a catheter-based test followed by their treatment with a drug-eluting stent could be a less expensive and more effective strategy than current practice. If applied to 1 million such patients in the United States undergoing catheterization, the new strategy would add 370,000 QALYs and save 1.2 billion dollars per year.

Diagnostic imaging costs: are they driving up the costs of hospital care?

PURPOSE: To retrospectively determine how changes in utilization of computed tomography (CT), magnetic resonance (MR) imaging, and other imaging technologies between 1996 and 2002 influenced costs of inpatient hospital care at one large academic medical center. MATERIALS AND METHODS: Institutional review board did not require its approval or patient informed consent for studies with use of billing data. Patient anonymity was protected by removal of potentially identifying information. Data on hospital costs for 17 139 patients admitted to Massachusetts General Hospital, Boston, Mass, between 1996 and 2002 were downloaded from hospital cost-accounting system; sample was restricted to inpatients with diagnoses in diagnosis-related groups 014-015 (Stroke and TIA [transient ischemic attack]), 164-167 (Appendectomy), 082 (Lung Cancer), 182-183 (Upper Gastrointestinal Conditions), 148-149 (Colon Cancer), and 243 (Back Problems). For each patient, data on demographics, all products and services used, and costs associated with each product or service were obtained. By using institutional codes, we calculated costs of CT, MR imaging, and total imaging relative to total hospital costs. Statistical analyses were performed with Student t test and multiple linear regression analysis. RESULTS: Between 1996 and 2002, number of inpatient CT and MR images obtained at the hospital more than doubled. In 2002, hospital costs were 155% those of 1996 levels; inpatient imaging costs were 151% those of 1996 levels. Total costs increased an average of 7.8% per year; imaging costs increased 8.3% per year. Although highly variable over the study period, as a percentage of total imaging costs, CT and MR imaging costs appeared to remain stable relative to costs of other imaging modalities. CONCLUSION: Despite substantial increases in utilization of inpatient CT, MR imaging, and other imaging technologies, diagnostic imaging costs increased at approximately same rate as did total costs for inpatients with several diagnoses. CT and MR imaging do not appear to be driving the cost increases seen between 1996 and 2002.

Cost-effectiveness analysis in the assessment of diagnostic imaging technologies.

In many ways, diagnostic technologies differ from therapeutic medical technologies. Perhaps most important, diagnostic technologies do not generally directly affect long-term patient outcomes. Instead, the results of diagnostic tests can influence the care of patients; in that way, diagnostic tests may affect long-term outcomes. Because of this, the benefits associated with the use of a specific diagnostic technology will depend on the performance characteristics (eg, sensitivity and specificity) of the test, as well as other factors, such as prevalence of disease and effectiveness of available treatments for the disease in question. The fact that diagnostic tests affect short-term, or "surrogate," outcomes, rather than long-term patient outcomes makes evaluation of these tests more complicated than the evaluation of therapeutic technologies. This article will trace the history of technology assessment in medicine, address the role of cost-effectiveness and decision analysis in health technology assessment, and describe unique features and approaches to assessing diagnostic technologies. The article will then conclude with a consideration of the limits of medical technology assessment.

Cost-effectiveness of whole-body CT screening.

PURPOSE: To make preliminary estimates of the effectiveness (in life-years) and cost-effectiveness (in costs per life-year) of whole-body computed tomographic (CT) screening. MATERIALS AND METHODS: Costs and effectiveness (in life-years) of onetime whole-body CT screening relative to those of no screening were calculated by using a decision-analytic model. It was assumed that any benefits from screening were due to earlier detection of disease and improvement in survival relative to survival with routine care. Eight conditions were included in the model: ovarian, pancreatic, lung, liver, kidney, and colon cancer; abdominal aortic aneurysm; and coronary artery disease. Costs of the screening examination, follow-up tests, and patient care were estimated. The base-case analysis was performed for a hypothetical cohort of 500 000 self-referred asymptomatic 50-year-old men. For sensitivity analyses, the age and sex of the cohort were varied. Results were expressed in 2001 U.S. dollars per life-year gained. RESULTS: Compared with routine care, whole-body CT screening provided minimal gains in life expectancy (0.016 6 years or 6 days) at an average additional cost of 2513 dollars per patient, or an incremental cost-effectiveness ratio of 151 000 dollars per life-year gained. Most patients (90.8%) had at least one positive finding, but only 2.0% had disease; work-up in patients with a false-positive result of screening accounted for 32.3% of total costs (1720 dollars of 5332 dollars). Results were sensitive to the prevalence of disease, the effect of screening on stage of disease at diagnosis, the specificity of screening, and the costs of follow-up for false-positive findings. CONCLUSION: Even with assumptions favorable to whole-body CT, implementation of onetime screening would not be cost-effective compared with currently funded medical interventions; follow-up for false-positive findings would add a substantial financial burden to the health care system.

Medicare's national coverage decisions, 1999-2003: quality of evidence and review times.

In 1998 Medicare amended its procedures for making national coverage decisions for new technologies in an attempt to make the process more transparent and evidence based. We examined the quality of evidence for sixty-nine technologies reviewed by Medicare since then. Determinations by the Centers for Medicare and Medicaid Services (CMS) have generally been consistent with the strength of evidence. Good clinical evidence from rigorous studies is usually lacking for the technologies Medicare considers, although in most cases the CMS covers with conditions if there is at least fair evidence that benefits outweigh harms. Decisions referred to the external Medicare Coverage Advisory Committee (MCAC) have averaged eight months longer than non-MCAC decisions.

Metastatic colorectal carcinoma: cost-effectiveness of percutaneous radiofrequency ablation versus that of hepatic resection.

PURPOSE: To evaluate the relative cost-effectiveness of radiofrequency (RF) ablation and hepatic resection in patients with metachronous liver metastases from colorectal carcinoma (CRC) and compare the outcomes, cost, and cost-effectiveness of a variety of treatment and follow-up strategies. MATERIALS AND METHODS: A state-transition decision model for evaluating the (societal) cost-effectiveness of RF ablation and hepatic resection in patients with CRC liver metastases was developed. The model tracks the presence, number, size, location, growth, detection, and removal of up to 15 individual metastases in each patient. Survival, quality of life, and cost are predicted on the basis of disease extent. Imaging, ablation, and resection affect outcomes through detection and elimination of individual metastases. Several patient care strategies were developed and compared on the basis of cost, effectiveness, and incremental cost-effectiveness (expressed as dollars per quality-adjusted life-year [QALY]). Extensive sensitivity analysis was performed to evaluate the impact of alternative scenarios and assumptions on results. RESULTS: A strategy permitting ablation of up to five metastases with computed tomographic (CT) follow-up every 4 months resulted in a gain of 0.65 QALYs relative to a no-treat strategy, at an incremental cost of $2400 per QALY. Compared with this ablation strategy, a strategy permitting resection of up to four metastases, one repeat resection, and CT follow-up every 6 months resulted in an additional gain of 0.76 QALYs at an incremental cost of $24 300 per QALY. Across a range of model assumptions, more aggressive treatment strategies (ie, ablation or resection of more metastases, treatment of recurrent metastases, more frequent follow-up imaging) were superior to less aggressive strategies and had incremental cost-effectiveness ratios of less than $35 000 per QALY. Findings were insensitive to changes in most model parameters; however, results were somewhat sensitive to changes in size thresholds for RF ablation, the number of metastases present, and surgery and treatment costs. CONCLUSION: RF ablation is a cost-effective treatment option for patients with CRC liver metastases. However, in most scenarios, hepatic resection is more effective (in terms of QALYs gained) than RF ablation and has an incremental cost-effectiveness ratio of less than $35 000 per QALY.

Cost-effectiveness of uterine artery embolization and hysterectomy for uterine fibroids.

PURPOSE: To compare the cost-effectiveness of uterine artery embolization (UAE) with that of hysterectomy for women with symptomatic uterine fibroids. MATERIALS AND METHODS: The authors developed a decision model to compare the costs and effectiveness of UAE and hysterectomy. In the model, a cohort of women aged 40 years with a diagnosis of uterine fibroids and no desire for future pregnancy was followed up until menopause. The analysis was performed from a societal perspective, including all costs and effects, regardless of who incurs them. Transition probability and quality-of-life estimates were obtained from the literature and a gynecologist, whereas costs (in 1999 U.S. dollars) were estimated by using rates of Medicare reimbursement for hospital costs and physician fees. Sensitivity analyses of key estimates were performed. Results were expressed in costs per quality-adjusted life-year (QALY). RESULTS: UAE was more effective (8.29 vs 8.18 QALYs) and less expensive (US dollars 6916 vs US dollars 7847) than hysterectomy. Cost-effectiveness results, with the exception of quality-of-life data, were robust to changes in most model assumptions. When the quality-of-life adjustment was eliminated, the two procedures were equally effective. CONCLUSION: UAE is a cost-effective alternative to hysterectomy across a wide range of assumptions about the costs and effectiveness of the two procedures. However, the study results were sensitive to changes in quality-of-life values.

Abdominal aortic aneurysms: cost-effectiveness of elective endovascular and open surgical repair.

PURPOSE: To evaluate the cost-effectiveness of elective endovascular and open surgical repair of infrarenal abdominal aortic aneurysms (AAAs) by taking into account short- and long-term outcomes. MATERIALS AND METHODS: A Markov decision model was developed to evaluate quality-adjusted life-years (QALYs) and lifetime costs of endovascular and open surgical repair. The incremental cost-effectiveness ratio (CER) was calculated for endovascular repair relative to open surgery in a cohort of 70-year-old men with an AAA between 5 and 6 cm in diameter. Clinically effectiveness data were derived from the literature. Cost data were derived from Medicare reimbursement rates, the hospital database, and the literature. One- and multiple-way sensitivity analyses were performed on uncertain model parameters. Costs were converted to year 2000 U.S. dollars; future costs and outcomes were discounted at 3%. RESULTS: The incremental CER of endovascular repair was 9,905 dollars per QALY. QALYs and lifetime costs were higher for endovascular repair than for open surgery (6.74 vs 6.52 and 39,785 dollars vs 37,606 dollars, respectively). In sensitivity analyses, the incremental CER was insensitive to immediate conversion rate and procedure mortality rate. The incremental CER was sensitive (ie, more than 75,000 dollars per QALY or endovascular repair was ruled out by dominance) to systemic-remote complications, long-term failures, and ruptures. CONCLUSION: The results suggest that endovascular repair is a cost-effective alternative compared with open surgery for the elective repair of AAA. The benefits and cost-effectiveness are highly dependent on uncertain outcomes, however, particularly long-term failure and rupture rates.

Hospital costs of uterine artery embolization and hysterectomy for uterine fibroid tumors.

RATIONALE AND OBJECTIVES: The purpose of this study was to compare the total actual hospital costs of uterine artery embolization (UAE) and hysterectomy for treatment of uterine fibroid tumors and to evaluate factors that might influence cost. MATERIALS AND METHODS: Total actual hospital costs were collected from the institution's cost accounting system on patients who underwent UAE (n = 57) or hysterectomy (n = 300) for uterine fibroids between 1998 and 2001. Electronic medical records were reviewed to collect clinical information. Standard statistical techniques were used to determine which factors influenced hospital costs. RESULTS: The mean total actual hospital costs of UAE were significantly higher than hysterectomy ($8,223 vs $6,046, P < .0001), but the mean length of stay was shorter (0.95 vs 2.6 days, P < .0001). In linear regression analyses, complications were predictive of increased costs of UAE; length of stay, complications, and laparoscopic hysterectomy were predictive of increased costs of hysterectomy. CONCLUSION: Hospital costs of UAE were higher than hysterectomy for the treatment of uterine fibroids, but the hospital stays were shorter.