Budget Impact Analysis of Idecabtagene Vicleucel for the Treatment of Adult Patients with Relapsed or Refractory Multiple Myeloma in the US.

BACKGROUND: Gene therapy is known to be unaffordable to those who need it the most; however, evidence on the financial impact is limited. OBJECTIVE: This study aimed to estimate the budget impact and affordability of the gene therapy idecabtagene vicleucel for the treatment of adult patients with relapsed or refractory multiple myeloma (rrMM) in the US over a 3-year period from the payer healthcare perspective. METHOD: A budget impact model was developed to estimate the budget impact to the US healthcare plan compared with bortezomib-based maintenance therapy. The target population size was based on a hypothetical 1-million-member plan over a 3-year time horizon with and without idecabtagene vicleucel adoption. The cost of drug acquisition, drug administration, and grade 3-4 adverse events (AEs) were calculated for both scenarios. The budget impact of idecabtagene vicleucel was calculated as the difference in costs for these two scenarios. Costs were extracted from IBM-Micromedex Red Book, Centers for Medicare and Medicaid Services, and the literature. A one-way sensitivity analysis was performed to ensure robustness. RESULTS: An estimated 22 patients with rrMM each year would be eligible for idecabtagene vicleucel. The model projected the annual cost per patient in the first year as $19,449 and $517,528.13 for bortezomib and idecabtagene vicleucel, respectively. Introducing idecabtagene vicleucel was predicted to increase the total budget by $13.4, $13.6, and $14 million in the first, second, and third years. There would be an additional $1.1128, $1.1252, and $1.1486 per member per month (PMPM) when using idecabtagene vicleucel over bortezomib. CONCLUSION: This study suggests that the high cost of the gene therapy idecabtagene vicleucel is justifiable due to the low number of rrMM patients.

Cost-effectiveness of adding daratumumab or bortezomib to lenalidomide plus dexamethasone for newly diagnosed multiple myeloma.

BACKGROUND: Multiple myeloma survival rates are steadily increasing due to availability of new drug classes used in combination with corticosteroids and chemotherapy. The latest treatments are daratumumab or bortezomib in combination therapy with lenalidomide and dexamethasone (Rd). Daratumumab, a CD38-targeted, human IgG1k monoclonal antibody, and bortezomib, a proteasome inhibitor, are both approved as regimens for transplant-ineligible relapsed/refractory multiple myeloma (RRMM). There have been cost-effectiveness analyses for daratumumab and bortezomib use in RRMM, but there are limited data regarding cost-effectiveness for daratumumab or bortezomib use in newly diagnosed multiple myeloma patients who are ineligible for stem cell transplantation. OBJECTIVE: To compare the cost-effectiveness of 3 separate regimens-(1) daratumumab, lenalidomide, and dexamethasone triple therapy (DRd); (2) bortezomib and lenalidomide plus dexamethasone triple therapy (VRd); and (3) lenalidomide plus dexamethasone (Rd)-in patients with multiple myeloma ineligible for autologous stem cell transplant. METHODS: A 2-state Markov model was developed using a US health system perspective and lifetime time horizon. Transition probabilities were calculated from the latest progression-free survival data reported in two phase 3 randomized controlled trials-MAIA and SWOG S0777-and extrapolated using a Weibull distribution based on the Hoyle Henley method. National data sources were used to obtain costs in 2019 US dollars, discounted by 3%. Health state utilities from available literature were applied to each health state. Utility decrements for adverse events were individualized in each choice branch with utility decrement weighted by the percentage of patients who experienced the adverse event in the MAIA and SWOG S0777 trials. We assumed a treatment would be cost-effective at a willingness to pay (WTP) of $150,000 per progression-free quality-adjusted life-year ($/PFQALY). One-way and probabilistic sensitivity analyses were conducted. RESULTS: Rd standard therapy had the lowest overall cost at $329,867, followed by VRd at $385,434 and DRd with the highest overall total cost at $626,900. Rd was estimated to result in the least amount (1.24) of PFQALYs, followed by VRd at 1.35 PFQALYs and DRd at 1.52 PFQALYs. With a WTP threshold of $150,000 per PFQALY, VRd was not cost-effective compared with Rd standard therapy, with an incremental cost-effectiveness ratio (ICER) of $530,256 per PFQALY. DRd was not cost-effective compared with VRd (ICER = $1,396,318 per PFQALY), nor as compared with Rd standard therapy (ICER = $1060,832). One-way sensitivity analysis showed that our model was sensitive to cost of DRd, VRd, and Rd drugs. Probabilistic sensitivity analysis showed that only at a WTP threshold of $550,000 was VRd cost-effective for 40% of iterations. There were no reasonable WTP thresholds, up to $800,00, where DRd became more cost-effective than VRd. CONCLUSIONS: This study is the first analysis to directly compare the cost-effectiveness of 3 acceptable chemotherapy treatment regimens for patients with multiple myeloma ineligible for autologous stem cell transplant. Neither DRd nor VRd triple therapy were found to be cost-effective vs Rd. Further cost-effectiveness analyses that include overall survival data for daratumumab and bortezomib triple therapies are needed to demonstrate an ICER in QALYs. DISCLOSURES: No funding was received for this study. At the time of this study, Narsipur was a UCSF-Actelion Clinical Research and Medical Communications Fellow, unrelated to this study. The other authors have nothing to disclose.

Lessons Learned Treating Patients with Multiple Myeloma in Resource-Constrained Settings.

PURPOSE OF REVIEW: Based on personal experiences, recommendations for physicians treating patients with multiple myeloma (MM) in low- and middle-income countries (LMICs) are proposed. RECOMMENDATIONS: (1) Implement strategies to keep the patient in the best possible condition for the longest time, in addition to focusing on ways to avoid financial toxicity; (2) if lenalidomide is unavailable, start treatment with thalidomide and dexamethasone, include, if possible, bortezomib; (3) conduct an outpatient-based autologous stem cell transplantation (ASCT) in all eligible patients; (4) use thalidomide as post-ASCT maintenance treatment if lenalidomide is unavailable for the standard risk patients; (5) monitor monoclonal proteins with serum protein electrophoresis and free light chain measurements; (6) employ novel drugs in cases of relapsed or refractory disease; and (7) do not forget supportive therapy. The therapeutic recommendations to treat patients with MM are somewhat different for physicians working in LMICs, compared with those treating patients in high-income countries. These are relevant since more than 50% of the inhabitants of the world live in LMICs, thus indicating that the vast majority of patients with MM are being treated in resource-constrained settings. As time goes by, physicians may acquire the ability to analyze and express their feelings and experiences about topics in the practice of medicine in which they could have learned lessons (1). Since 1980, we have been treating patients with multiple myeloma (MM); to date, we have been personally involved in the study and treatment of more than 300 patients with this disease (2). Having gained experience dealing with MM patients in underprivileged circumstances, such as those prevailing in our country: México, having explored different ideas, treatments, and methods, and being aware of the financial implications which may impact our selection of therapeutic strategies and recommendations, we felt that it was appropriate to share in this article some of these ideas with practitioners around the world who are involved in the treatment of patients with MM in low- and middle-income countries (LMICs).

Medication use evaluation of the financial and clinical implications of ixazomib compared to bortezomib in the outpatient setting.

In the past decade, several new therapies have been approved for use in multiple myeloma, including the novel oral agent, ixazomib. Ixazomib, like bortezomib and carfilzomib, is a proteasome inhibitor, a class of agents that are a mainstay of treating multiple myeloma in both the frontline and relapsed settings. Ixazomib is administered orally and offers many potential advantages over the subcutaneous or intravenous administration of bortezomib. In this single-center, retrospective medication use evaluation, adult patients with multiple myeloma receiving either ixazomib or bortezomib in the outpatient setting were assessed to evaluate financial implications and tolerability. A total of 28 patients were included. The total wholesale acquisition cost for one cycle of ixazomib was $9942, and $6412 for bortezomib. Average reimbursement per cycle was $9205 for ixazomib and $5664 for bortezomib. Secondarily, the incidence of interruption in therapy was evaluated. Ixazomib was associated with a slightly higher incidence of interruption compared to bortezomib, 42.9% and 35.7%, respectively. It is notable that ixazomib has similar drug reimbursement rates to bortezomib, but slightly higher rates of interruption in therapy. In conclusion, if tolerable for the patient, ixazomib may offer a financially acceptable alternative for the treatment of multiple myeloma.

A budget impact analysis of lenalidomide in multiple myeloma Egyptian patients.

INTRODUCTION: The aim of this study was to estimate the budget impact of lenalidomide and dexamethasone (RD) versus bortezomib, cyclophosphamide and dexamethasone (VCD) in newly diagnosed multiple myeloma (NDMM) and relapsed refractory (RR) MM patients, from the perspective of the Egyptian Ministry of health (MoH). METHODS: Two budget impact dynamic models were conducted to assess the budget impact of RD entry over a 3-year period. The clinical data for the modeled cohorts were based on published articles. Total annual medical costs associated with non-progression and progression disease states included the sum of estimated costs for adverse effects management, concomitant treatments, hospitalization and the follow up were measured. Deterministic sensitivity analyses were performed. RESULTS: The target population in a given year was estimated to include 245 patients with RRMM and 291 patients with NDMM receiving RD versus VCD. In RRMM, the annual budget savings of lenalidomide entry were estimated at EGP -1,103,969, -3,362,793 and -5,949,228 at year 1, year 2 and year 3, respectively. In NDMM, the annual budget savings of lenalidomide entry were estimated at EGP869,415, -1,779,776 and -2,139,311 at year 1, year 2 and year 3, respectively, to the payer after lenalidomide entry. The model results in RRMM were most sensitive to variations in patients eligible to transplantation in RRMM. In NDMM, the model results were most sensitive to the market share of VCD in the first year. CONCLUSION: The results of our BI models suggest that not only does RD treatment have an effect on the budget, but also has major cost savings in other areas which are very important while considering the total costs of MM treatment. This study results provided evidence-based information to the MoH that will help in decision making of whether to implement RD as a treatment intervention or not.

Association of adverse events and associated cost with efficacy for approved relapsed and/or refractory multiple myeloma regimens: A Bayesian network meta-analysis of phase 3 randomized controlled trials.

BACKGROUND: Several new treatment options have been approved for relapsed and/or refractory multiple myeloma (RRMM). In this systematic review, associations of the efficacy of each approved regimen with adverse events (AEs) and the total cost per cycle were compared with a Bayesian network meta-analysis (NMA) of phase 3 randomized controlled trials (RCTs). METHODS: Scopus, Cochrane, PubMed Publisher, and Web of Science were searched from January 1999 to July 2018 for phase 3 RCTs of regimens (approved by the US Food and Drug Administration) used in RRMM. The relative ranking of agents was assessed with surface under the cumulative ranking (SUCRA) probabilities. The primary efficacy, safety, and cost outcomes were progression-free survival with the regimen, grade 3 to 4 AEs, and the total cost per cycle (regimen cost plus average cost of managing AEs). RESULTS: Fifteen studies including 7718 patients and evaluating 14 different regimens were identified. Daratumumab, lenalidomide, and dexamethasone were ranked highest for reducing progression (hazard ratio, 0.13; 95% credible interval, 0.09-0.19; SUCRA, 1) but carried the highest probability of total cost per cycle ($41,420; 95% Credible Interval [CrCl], $58,665-$78,041; SUCRA, 0.02). Panobinostat, bortezomib, and dexamethasone were the least effective and least safe (SUCRA, 0.24), whereas bortezomib, thalidomide, and dexamethasone emerged as least effective with the highest total cost per cycle (SUCRA, 0.33). Carfilzomib and dexamethasone emerged as the winner when this regimen was considered in terms of efficacy and safety (SUCRA, 0.61) and efficacy and total cost per cycle (SUCRA, 0.60). CONCLUSIONS: The results of this NMA can provide additional guidance for the decision-making process when one is choosing the most appropriate regimen for RRMM.

Economic Evaluation of Adding Daratumumab to a Regimen of Bortezomib + Dexamethasone in Relapsed or Refractory Multiple Myeloma: Based on the Latest Updated Analysis of CASTOR.

PURPOSE: Adding daratumumab to a regimen of bortezomib + dexamethasone (Vd) has been reported to provide a benefit of longer progression-free survival in patients with relapsed or refractory multiple myeloma (RRMM). However, it is still unclear whether the addition of daratumumab is cost-effective in RRMM. Based on the latest updated analysis of data from the CASTOR trial, this study performed an economic evaluation of the addition of daratumumab to Vd in patients with RRMM. METHODS: A Markov decision model was used for estimating the long-term costs and efficacy of Vd with or without daratumumab in patients with RRMM. Data on efficacy were taken from the CASTOR trial to compare Vd + daratumumab with Vd. Costs were taken from the US Centers for Medicare & Medicaid Services and from the literature. A series of sensitivity analyses were performed to address the robustness of the model. Variations in the price of daratumumab and subgroup analyses were conducted. FINDINGS: The base-case analysis showed that adding daratumumab to Vd provided an additional 1.256 quality-adjusted life-years (QALYs) or 1.645 life-years (LYs), with incremental 213,164 USD (163,184 USD) per QALY (LY) gained. Univariate sensitivity analyses suggested that the subsequent treatment cost of DVd and the price of daratumumab had the greatest effect on the incremental cost-effectiveness ratio. According to the variations analysis of the price of daratumumab, the addition of daratumumab would be cost-effective when daratumumab was priced at 70% (30%) of the current price at a willingness-to-pay threshold of 200,000 USD/QALY (150,000 USD/QALY). Subgroup analysis indicated that adding daratumumab to Vd was most cost-effective in patients with 1 prior line of therapy. IMPLICATIONS: From a US-payer perspective, daratumumab added to Vd in RRMM is likely to exceed the common accepted values of cost-effectiveness.

Extrapolating Survival Data Using Historical Trial-Based a Priori Distributions.

OBJECTIVES: To show how clinical trial data can be extrapolated using historical trial data-based a priori distributions. METHODS: Extrapolations based on 30-month pivotal multiple myeloma trial data were compared with 75-month data from the same trial. The 30-month data represent a typical decision-making scenario where early results from a clinical trial are extrapolated. Mature historical trial data with the same comparator as in the pivotal trial were incorporated in 2 stages. First, the parametric distribution selection was based on the historical trial data. Second, the shape parameter estimate of the historical trial was used to define an informative a priori distribution for the shape of the 30-month pivotal trial data. The method was compared with standard approaches, fitting parametric distributions to the 30-month data with noninformative prior. The predicted survival of each method was compared with the observed survival (ΔAUC) in the 75-month trial data. RESULTS: The Weibull had the best fit to the historical trial and the log-normal to the 30-month pivotal trial data. The ΔAUC of the Weibull with informative priors was considerably smaller compared with the standard Weibull. Also, the predicted median survival based on the Weibull with informative priors was more accurate (melphalan and prednisone [MP] 40 months, and bortezomib [V] combined with MP [VMP] 62 months) than based on the standard Weibull (MP 45 months and VMP 72 months) when compared with the observed median (MP 41.3 months and VMP 56.4 months). CONCLUSIONS: Extrapolation of clinical trial data is improved by using historical trial data-based informative a priori distributions.

Cost-effectiveness of lenalidomide plus low-dose dexamethasone for newly diagnosed multiple myeloma patients ineligible for stem cell transplantation in China.

Aim: To assess the cost-effectiveness of lenalidomide plus low dose dexamethasone (Rd) relative to bortezomib-contained therapy (BCT) for newly diagnosed multiple myeloma patients ineligible for stem cell transplantation (ndMM) in China. Materials & methods: A literature review was conducted to identify appropriate evidence for developing a cost-effectiveness model comparing Rd with BCT for lifetime health outcomes and direct medical costs in Chinese ndMM patients. Results: The estimated incremental cost-effectiveness ratio per gained quality-adjusted life years for Rd versus BCT was ¥49,793. The chance for Rd to be cost effective, under the cost-effectiveness thresholds of three-times the 2018 Chinese gross domestic goods per capita, was 90.8%. Conclusion: The cost-effectiveness of Rd relative to BCT for ndMM in Chinese patients is highly attractive.

Real-world treatment patterns, resource use and cost burden of multiple myeloma in Portugal.

OBJECTIVE: We provide a real-world overview of multiple myeloma (MM) treatment patterns, outcomes and healthcare resource use (HRU) in Portugal. METHODS: Data were collected retrospectively from consecutive patients diagnosed/treated at the Portuguese Oncology Institute of Porto (IPO-Porto) between 2012 and 2015. Primary objectives were progression-free survival (PFS) and overall survival (OS), with treatment patterns and HRU secondary. Analysis was by line of therapy (LOT), and post hoc by age (<65/≥65 years). RESULTS: 165, 73 and 32 patients received first, second and third LOTs respectively (N = 187). OS probabilities were 91.5%, 83.2% (<65 years) and 86.6%, 65.3% (≥65 years) at 12, 24 months respectively. PFS decreased from the start of each LOT for both age groups and was less for patients ≥65 years. Younger patients received more combination treatment (immunomodulatory drugs + proteasome inhibitors) and stem cell transplants, and had higher mean costs than older patients (€81,213 vs. €36,864 where three LOTs were received). Cost drivers were medications, transplantations and hospitalisations. CONCLUSION: Our results suggest divergence between younger and older MM patients. Older patients had lower OS and PFS probabilities, HRU costs and fewer stem cell transplantations. The treatment patterns in each LOT may differ from other countries' findings, suggesting treatment heterogeneity.

Cost Offsets in the Treatment Journeys of Patients With Relapsed/Refractory Multiple Myeloma.

PURPOSE: Multiple new regimens are available for the treatment of relapsed/refractory multiple myeloma (RRMM). In this context, it is increasingly important to understand the differential costs of regimens used to treat RRMM. METHODS: A treatment journey for RRMM during a 12-month period of therapy was developed to reflect real-world clinical practice based on current treatment guidelines and input from hematologists/oncologists. The journey incorporated prescreening visits, laboratory tests, regimen-specific premedication, treatment-related costs, medical costs, and indirect costs. A cost model was constructed from the standard RRMM treatment pathway to compare overall, direct, and indirect costs across therapies over a 12-month period from initiation of second-line therapy and to determine cost offsets (incremental costs) associated with use of ixazomib-based therapy versus comparator regimens. According to the clinical input, the standard pathway was modified for patients with high unmet need to determine specific cost offsets in these subgroups. FINDINGS: Total costs ranged from $93,683 for bortezomib-cyclophosphamide-dexamethasone to $315,296 for daratumumab-bortezomib-dexamethasone. Drug cost comprised the highest proportion (83%-98%) of total costs of second-line therapy across regimens, which were generally highest for regimens based on recently approved agents. Indirect costs were higher for regimens that required more frequent or longer durations of drug administration, and lower for all-oral regimens. Costs were reduced among frail patients because of the use of adjusted dosing, whereas indirect costs were increased for regimens that required a greater number of clinic visits among patients with barriers to physician access. IMPLICATIONS: Cost model analyses highlight the differential direct and indirect costs associated with multiple regimens for the treatment of RRMM, including many recent new regimens. The results indicate the lower treatment burden and indirect costs associated with administering all-oral regimens compared with regimens that require frequent and/or lengthy subcutaneous or intravenous infusions. Understanding comparative costs associated with the treatment journeys of different patients with RRMM may help inform payer and patient therapeutic choices.

Costs of administration, travelling, and productivity losses associated with hospital administration of multiple myeloma drugs in Finland.

AIM: To estimate the drug administration, travelling, and productivity costs associated with infusion or subcutaneous proteasome inhibitor (PI) treatments (specifically carfilzomib and bortezomib) for multiple myeloma (MM) patients in Finland. MATERIALS AND METHODS: Price tariffs of Finnish hospital districts are used as the basis of invoicing sent to healthcare service payers. A review of these price tariff lists was conducted and obtained data analysed to estimate the mean unit cost of PI administration visit. Travelling costs stratified by areas with different population densities were assessed, based on the national travelling reimbursement register data maintained by the Social Insurance Institution of Finland. Productivity costs due to time spent on administration visits and travelling were estimated based on an expert interview and a spatial healthcare accessibility analysis. RESULTS: Nineteen (95%) of the Finnish hospital districts were included in the review. Relevant unit cost information was found for 15 (75%) of the districts. The mean PI administration cost alone was 270€ (95% CI = 189€-351€) per administration and increased to 371€ when travelling costs were included. Productivity costs added, the mean PI administration costs totalled 405€ for bortezomib and 437€ for carfilzomib. LIMITATIONS: The costing rationale of price tariffs may vary between hospital districts. Productivity costs were estimated conservatively, due to lack of data. CONCLUSIONS: The administration of intravenous or subcutaneous PIs to treat MM in healthcare facilities causes significant and potentially avoidable healthcare, travelling, and indirect costs, and they should be included in all health economic evaluations (HEEs). As the cost estimates utilized in this study represent most of central hospitals in the country, they provide useful information for future HEEs. A broader conclusion is that novel oral medications, such as the first oral PI, have a significant potential for reducing administration-related costs of subcutaneous or intravenous PIs.

Modeling Covariate-Adjusted Survival for Economic Evaluations in Oncology.

BACKGROUND AND OBJECTIVES: In economic evaluations in oncology, adjusted survival should be generated if imbalances in prognostic/predictive factors across treatment arms are present. To date, no formal guidance has been developed regarding how such adjustments should be made. We compared various covariate-adjusted survival modeling approaches, as applied to the ENDEAVOR trial in multiple myeloma that assessed carfilzomib plus dexamethasone (Cd) versus bortezomib plus dexamethasone (Vd). METHODS: Overall survival (OS) data and baseline characteristics were used for a subgroup (bortezomib-naïve/one prior therapy). Four adjusted survival modeling approaches were compared: propensity score weighting followed by fitting a Weibull model to the two arms of the balanced data (weighted data approach); fitting a multiple Weibull regression model including prognostic/predictive covariates to the two arms to predict survival using the mean value of each covariate and using the average of patient-specific survival predictions; and applying an adjusted hazard ratio (HR) derived from a Cox proportional hazard model to the baseline risk estimated for Vd. RESULTS: The mean OS estimated by the weighted data approach was 6.85 years (95% confidence interval [CI] 4.62-10.70) for Cd, 4.68 years (95% CI 3.46-6.74) for Vd, and 2.17 years (95% CI 0.18-5.06) for the difference. Although other approaches estimated similar differences, using the mean value of covariates appeared to yield skewed survival estimates (mean OS was 7.65 years for Cd and 5.40 years for Vd), using the average of individual predictions had limited external validity (implausible long-term OS predictions with > 10% of the Vd population alive after 30 years), and using the adjusted HR approach overestimated uncertainty (difference in mean OS was 2.03, 95% CI - 0.17 to 6.19). CONCLUSIONS: Adjusted survival modeling based on weighted or matched data approaches provides a flexible and robust method to correct for covariate imbalances in economic evaluations. The conclusions of our study may be generalizable to other settings. TRIAL REGISTRATION: ClinicalTrials.gov identifier NCT01568866 (ENDEAVOR trial).

Impact of the affordability of novel agents in patients with multiple myeloma: Real-world data of current clinical practice in Mexico.

BACKGROUND: The treatment of multiple myeloma (MM) has become costly and difficult to access for patients living in low-income to middle-income countries. METHODS: The current retrospective study included 148 patients in Mexico with newly diagnosed MM, and was performed to compare the outcomes of patients with and without access to novel agents. The records of 77 patients admitted to a public hospital (PubC) and 71 patients cared for within private health systems (PrivC) from November 2007 to July 2016 were reviewed. RESULTS: Compared with those treated in PrivC, patients receiving care at PubC were more likely to be diagnosed with advanced disease. A thalidomide-based regimen was the most common induction treatment used at PubC, whereas a bortezomib-based regimen was used most often in PrivC. The median follow-up was 41 months. Patients in PrivC demonstrated better response rates and survival; 65% of patients treated in PrivC versus 41% treated at PubC achieved a very good partial response or better (P = .005). The median progression-free survival and median overall survival were 23 months and 51 months, respectively, for patients treated at PubC and 41 months and 79 months, respectively, for those treated in PrivC (P<.001). More patients underwent autologous stem cell transplantation in PrivC. When adjustments were made for covariates, patients treated at PubC experienced a higher risk of death compared with patients receiving care in PrivC (hazard ratio, 2.0; 95% confidence interval, 1.0-4.3 [P = .04]). CONCLUSIONS: Stage at diagnosis, induction regimen, and autologous stem cell transplantation were found to be contributors to survival disparities between patients with MM treated at PubC compared with PrivC in Mexico. These findings underscore the need to improve access to novel agents and stem cell transplantation in public health systems. Cancer 2018;124:1946-53. © 2018 American Cancer Society.

Healthcare resource utilization and costs in amyloid light-chain amyloidosis: a real-world study using US claims data.

AIM: To estimate healthcare utilization and costs in amyloid light-chain (AL) amyloidosis. PATIENTS & METHODS: AL amyloidosis patients were identified in 2007-2015 claims databases if they had ≥1 inpatient/≥2 outpatient claims consistent with AL amyloidosis and received ≥1 AL-specific treatment. Descriptive statistics were reported. RESULTS: 50.1% (n = 3670) were admitted ≥1 time during the year, 11.3% (n = 827) ≥3 times. From 2007 to 2015, bortezomib use increased from 4.6 to 25.3%; melphalan use decreased from 18.9 to 2.0%; costs increased from 92,866 to $114,030. Among incident patients with at least 2 years of follow-up, healthcare utilization and costs decreased from first to second year post-diagnosis. CONCLUSION: AL chemotherapy-based prescribing practices changed. Total annual healthcare costs increased over time among AL amyloidosis patients.

Potential therapeutic and economic value of risk-stratified treatment as initial treatment of multiple myeloma in Europe.

Biomarkers associated with prognosis in multiple myeloma (MM) can be used to stratify patients into risk categories. An attractive alternative to uniform treatment (UT), risk-stratified treatment (RST) is proposed where high-risk patients receive bortezomib-based regimens while standard-risk patients receive alternative less costly regimens. An early Markov-type decision analytic model evaluated the potential therapeutic and economic value of different RST strategies compared with UT in MM patients in key European countries. Results suggest RST strategies were both cheaper and more effective than UT across all countries, with the molecular marker-only strategy RST-SKY92 producing maximum health gains (0.031-0.039 QALYs). The conclusions remained consistent in the univariate sensitivity analyses. These findings should encourage stakeholders to support the adoption of RST approaches in MM.

Subsidies for Oral Chemotherapy and Use of Immunomodulatory Drugs Among Medicare Beneficiaries With Myeloma.

Purpose The low-income subsidy (LIS) substantially lowers out-of-pocket costs for qualifying Medicare Part D beneficiaries who receive orally administered chemotherapy. We examined the association of LIS with the use of novel oral immunomodulatory drugs (IMiDs; lenalidomide and thalidomide) among beneficiaries with myeloma, who can receive either orally administered or parenteral (bortezomib-based) therapy. Methods Using SEER-Medicare data, we identified Part D beneficiaries diagnosed with myeloma in 2007 to 2011. In multivariable models adjusted for sociodemographic and clinical characteristics, we analyzed associations between the LIS and use of IMiD-based therapy, delays between IMiD refills, and select health outcomes during the first year of therapy. Results Among 3,038 beneficiaries, 41% received first-line IMiDs. Median out-of-pocket cost for the first IMiD prescription was $3,178 for LIS nonrecipients and $3 for LIS recipients, whereas the respective median costs for the first year of therapy were $5,623 and $6, respectively. Receipt of the LIS was associated with a 32% higher (95% CI, 16% to 47%) probability of receiving IMiDs among beneficiaries age 75 to 84 years and a significantly lower risk of delays between refills in all age groups (adjusted relative risk, 0.54; 95% CI, 0.32 to 0.92). Duration of therapy did not significantly differ between LIS recipients and nonrecipients (median, 7.6 months). Patients treated with IMiDs had significantly fewer emergency department visits and hospitalizations compared with patients receiving bortezomib (without IMiDs), but 1-year overall survival and cumulative Medicare costs were similar. Conclusion Medicare beneficiaries with myeloma who do not receive LISs face a substantial financial barrier to accessing orally administered anticancer therapy, warranting urgent attention from policymakers. Limiting out-of-pocket costs for expensive anticancer drugs like the IMiDs may improve access to oral therapy for patients with myeloma.

Estimated generic prices of cancer medicines deemed cost-ineffective in England: a cost estimation analysis.

OBJECTIVES: The aim of this study was to estimate lowest possible treatment costs for four novel cancer drugs, hypothesising that generic manufacturing could significantly reduce treatment costs. SETTING: This research was carried out in a non-clinical research setting using secondary data. PARTICIPANTS: There were no human participants in the study. Four drugs were selected for the study: bortezomib, dasatinib, everolimus and gefitinib. These medications were selected according to their clinical importance, novel pharmaceutical actions and the availability of generic price data. PRIMARY AND SECONDARY OUTCOME MEASURES: Target costs for treatment were to be generated for each indication for each treatment. The primary outcome measure was the target cost according to a production cost calculation algorithm. The secondary outcome measure was the target cost as the lowest available generic price; this was necessary where export data were not available to generate an estimate from our cost calculation algorithm. Other outcomes included patent expiry dates and total eligible treatment populations. RESULTS: Target prices were £411 per cycle for bortezomib, £9 per month for dasatinib, £852 per month for everolimus and £10 per month for gefitinib. Compared with current list prices in England, these target prices would represent reductions of 74-99.6%. Patent expiry dates were bortezomib 2014-22, dasatinib 2020-26, everolimus 2019-25 and gefitinib 2017. The total global eligible treatment population in 1 year is 769 736. CONCLUSIONS: Our findings demonstrate that affordable drug treatment costs are possible for novel cancer drugs, suggesting that new therapeutic options can be made available to patients and doctors worldwide. Assessing treatment cost estimations alongside cost-effectiveness evaluations is an important area of future research.

Guideline Adherence Regarding the Use of Expensive Drugs in Daily Practice: The Examples of Trastuzumab in Breast Cancer and Bortezomib in Multiple Myeloma.

BACKGROUND: The present study was designed to obtain insights into guideline adherence regarding the use of expensive drugs in The Netherlands in daily practice and into the patients' perspective on the decision-making process. MATERIAL AND METHODS: A retrospective review of medical charts regarding the use of trastuzumab in early and metastatic breast cancer (EBC/MBC) and bortezomib in multiple myeloma (MM) was conducted. Prescription according to clinical practice guidelines was assessed. The review was supplemented with patient interviews. RESULTS: Of 702 adjuvant-treated EBC patients, 97% had a documented human epidermal growth factor receptor 2 (HER2) testing (23% HER2 positive). 92% (147/160) of the HER2-positive EBC patients were treated with trastuzumab. Of 594 MBC patients, 81% had a documented HER2 testing (19% HER2 positive). 82% (75/91) of the HER2-positive MBC patients were treated with trastuzumab. Of 68 MM patients, 50% were treated with bortezomib. Reasons not to treat were consistent with the guidelines. Patients were generally satisfied with the decision-making process; improvements in patient education were suggested (e.g., repeating the information given, adding information on side effects). CONCLUSIONS: Guidelines were generally well followed with respect to trastuzumab and bortezomib, indicating that funding did not influence the treatment decisions of physicians. In view of the growing numbers of both cancer patients and expensive new anticancer drugs, and increasing budget constraints, it is unclear whether the present-day policies will guarantee a similar level of guideline adherence. Patient involvement in decision-making could be increased by improving the patient education on treatment.