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A Budget Impact Analysis of An Increased Uptake of Alemtuzumab From The UK NHS Perspective
A722
VA L U E I N H E A LT H 2 0 ( 2 0 1 7 ) A 3 9 9 – A 8 1 1
carried out. Results: Integrating cfDNA in T21 prenatal screening strategy prior to invasive diagnosis for pregnant women with a risk threshold of at least 1/250, leads to an additional cost of € 440,000, while improving the number of detected prenatal T21s by more than 40, and reducing the number of invasive procedures by 13 300 and the number of related miscarriages by 18. When comparing the modelling strategies integrating cfDNA, the low bounds of 1/250 and 1/1000, and the high bounds of 1 and 1/50, appear to be the most relevant choices based on non-weighted outcomes. Conclusions: Implementation of cfDNA as a contingent test in prenatal screening can improve foetal T21s detection while avoiding procedure related miscarriages. From a health economic point of view, implementation of cfDNA in France seems to be most relevant for threshold risks ranging between [1/250; 1[, [1/1000; 1[, or [1/1000; 1/50[. Final HAS recommendations were based on model results, as well as on ethical, preferential and organisational aspects and expert opinion. PND25 Adequately Reflecting The Clinical Benefits In Rare Disease Economic Modeling Using Sma Type I As A Casestudy Zuluaga S1, Knight C1, Thompson R2, Teynor M3 Health Solutions, Manchester, UK, 2Biogen, Zug, Switzerland, 3Biogen, Cambridge, MA, USA
1RTI
Objectives: Spinal muscular atrophy (SMA) is a rare, hereditary, autosomal recessive neuromuscular disorder caused by deletion of the survival motor neuron 1 gene (SMN1). Type I SMA is one of the most severe forms of SMA that affects infants between 0-6 months of age where they never develop the ability to sit and have a short life expectancy. Nusinersen is the first approved treatment for SMA, and prior management of the disease centred on the symptomatic treatment of respiratory, nutritional, and orthopaedic function decline. The objective of this study was to build a model to adequately reflect the clinical benefits of a novel treatment (nusinersen) in a rare disease which has resulted in patients achieving motor function abilities not previously observed. Methods: A decision analytic model was developed based on the clinical trial outcome measures, registry data and clinical opinion via an advisory board. Health states were based on both motor milestones consistent with Type I SMA and for motor milestones not previously experienced by Type I SMA patients, such as sitting without support and standing with assistance due to the improvement in motor function experienced by patients treated with nusinersen. Due to paucity of data, quality of life utility values were derived from a vignette study. Data for the major clinical events such as scoliosis surgery were based on the literature. Results: The resulting model structure showed that over a 40-year time horizon patients treated with nusinesen gained an average of 6.5 life-years and over 6 quality-adjusted life years (undiscounted). Conclusions: The resulting model structure is a basis on which a full economic model can be developed to support nusinersen in future Health Technology Assessments (HTAs). PND26 A Budget Impact Analysis of An Increased Uptake of Alemtuzumab From The UK NHS Perspective Rog D1, Guo JD2, Nucit A3, Le Bagousse-Bego G2, Chevli M4, Chung L2 1Salford Royal NHS Foundation Trust, Salford, UK, 2Sanofi, Cambrige, MA, USA, 3Ividata, Levallois-Perret, France, 4Sanofi, Guildford, Surrey, UK
Objectives: To estimate the impact of increased use of alemtuzumab 12 mg/day in the treatment of adult RRMS patients (per its marketing authorization) on the UK National Health Service (NHS) budget. Methods: A budget impact analysis was conducted for an average of 121,200 RRMS patients in the UK over a 5-year time horizon (start year: 2017) from the NHS payer perspective. The number of RRMS patients was derived from UK Office of National Statistics (2015) by considering prevalence and incidence of RRMS in the UK. Five direct costs considered were treatment/administration, resource use/monitoring, adverse events, disability progression (assessed by Expanded Disability Status Scale scores), and relapse. The model compared the cost difference in each year under two different market scenarios: current market mix, in which the market share of alemtuzumab 12 mg/day was projected to remain consistent with current shares (2017: 1.6%; 2018: 1.8%; 20192021: 1.5%), and an alternative market mix, in which market share of alemtuzumab 12 mg/day was forecasted to increase 0% to 1% per year (2017: 2.6%; 2018: 2.2%; 2019: 2.1%; 2020-2021: 1.5%). Results: Using the alternative market mix in the UK NHS payer perspective, alemtuzumab starts saving in Year 3 (2019) compared with the current market mix, with an average annual total cost saving of £10,976,253 over 5 years for all RRMS population. The annual total costs for the first 2 years increased with the alternative mix, which can be explained by initial high upfront costs of alemtuzumab. However, these increases are offset by the reduction in drug usage in subsequent years, leading to an overall cost saving. Conclusions: From the UK NHS perspective, compared to the current market mix, the alternative market mix with increased uptake of alemtuzumab leads to an average annual saving of £10 million over 5 years, starting from Year 3 (2019). Study Support: Sanofi PND27 Disease-Modifying Therapy Is Associated With Lower Medical Costs In Patients With Multiple Sclerosis With or Without Corticosteroid Treatment: A Us Retrospective Claims-Based Study Sanchirico M1, Caldwell-Tarr A2, Isikwe P1, Hashemi L1, Dufour R2 Health Insights: A Humana Company, Louisville, KY, USA
1Sanofi, Cambridge, MA, USA, 2Comprehensive
Objectives: To compare, using corticosteroid use as a surrogate marker for relapse activity, healthcare utilization and costs among patients with multiple sclerosis (MS) who do/do not receive disease-modifying therapy (DMT). Methods: Healthcare claims analysis included MS patients with or without DMT during the 12 months prior to the most recent claim, stratified by number of corticosteroid treatments (0, 1, or ≥ 2) during this period. Costs were assessed based on claims during the 1-year observation period. Results: Of 7072 patients covered by Medicare, 4689 (66%)
received DMT. Patients with DMT were less likely to have a corticosteroid claim than patients not receiving DMT (39% vs 62%), and less likely to have an ER visit (39% vs 54%) or hospitalization episode (18% vs 29%). Overall medical costs were lower with DMT use. For patients without a corticosteroid claim, total medical costs were $8,037 for DMT vs $13,604 for no DMT; inpatient costs were $15,085 vs $20,802; ER costs were $1,204 vs $1,508. Costs rose with increased corticosteroid treatments. In patients with ≥ 2 corticosteroid treatments, total medical costs (DMT vs no DMT) were $13,688 vs $18,728; inpatient costs were $14,320 vs $17,890; ER costs were $1,674 vs $2,172. Rates of post-steroid DMT switching were low (12%–16%) even in patients with ≥ 2 episodes of corticosteroid use. Conclusions: DMT use was associated with fewer corticosteroid treatments (potentially indicating fewer relapses), fewer hospitalization episodes, fewer ER visits, and lower medical costs, indicative of better health outcomes. DMT use lowered healthcare utilization and costs in patients with and without corticosteroid treatments. Corticosteroid treatments were associated with increasing utilization and costs, but did not prompt increased DMT switching. Study Support: Sanofi PND28 Cost-Effectiveness of Alemtuzumab In The Treatment of Relapsing Forms of Multiple Sclerosis In The United States and Societal Spillover Effects Chirikov V1, Ma I2, Joshi N1, Patel D1, Smith A2, Giambrone C2, Cornelio N1, Hashemi L2 1Pharmerit International, Bethesda, MD, USA, 2Sanofi, Cambridge, MA, USA
Objectives: Caring for patients with relapsing multiple sclerosis (RMS) poses economic and humanistic burdens that are well-documented, but in the United States (US) are seldom incorporated into the cost-effectiveness appraisal of disease modifying treatments (DMTs). Alemtuzumab (ALEM) was compared to ocrelizumab (OCR) and natalizumab (NAT), accounting for societal spillover effects due to RMS treatment. Methods: A Markov model with annual cycles and 20-year time horizon was run separately from US payer and societal perspectives. The societal perspective analysis built upon the payer perspective by including productivity loss costs, informal care costs, and caregiver disutility. For ALEM, the population represented characteristics of pooled treatment-naive and non-naive patients from the CARE MS I and II trials (NCT00530348, NCT00548405). The British Columbia Multiple Sclerosis cohort was used to model the natural history of RMS disease progression. Network meta-analysis provided data on the relative efficacy of DMTs in reducing relapses and slowing disability. Withdrawal rates, treatment waning, resource use, costs and utility inputs were populated using published data and clinical expert opinion. Information on adverse events was derived from package inserts and published sources. Results: From US payer perspective, ALEM dominated comparators by accumulating more quality-adjusted life years (QALYs) and lower costs vs OCR (+0.499; -$486,368) and NAT (+0.516; -$626,578). The benefit of ALEM over comparators further increased when the analysis was conducted from the societal perspective vs OCR (+0.556; -$513,813) and vs NAT (+0.575; -$655,263). Sensitivity analyses identified the DMT withdrawal rate as one of the most influential model parameters. Conclusions: The cost-effectiveness of ALEM was driven by its durable efficacy in the absence of continuous treatment and by savings from treatment acquisition costs over the time horizon. ALEM generated greater positive spillover effects to society by reducing caregiver disutility, informal care, and productivity loss versus comparators. Study Support: Sanofi PND29 Cost-Effectiveness of Brivaracetam As Adjunctive Therapy for Partial-Onset Epilepsy In The Finnish Setting Väätäinen S1, Soini E1, Peltola J2, Charokopou M3, Taiha M4, Kälviäinen R5 Oy, Kuopio, Finland, 2Tampere University Hospital, Tampere, Finland, 3UCB Pharma, Brussels, Belgium, 4UCB Pharma, Espoo, Finland, 5University of Eastern Finland & Kuopio University Hospital, Kuopio, Finland 1ESiOR
Objectives: To evaluate the cost-effectiveness of brivaracetam and perampanel as adjunctive antiepileptic drugs (AED) in the treatment of partial-onset seizures with or without secondary generalization in adult and adolescent patients with epilepsy in Finland. Methods: A discrete event simulation approach was used to model: 1) brivaracetam and perampanel as 3rd concomitant AEDs on top of two AEDs, with perampanel being used after brivaracetam among other adjunctive AEDs, 2) perampanel omitted from brivaracetam arm, 3) brivaracetam and perampanel as adjunctive AEDs on top of only one AED. Comparative treatment efficacy (achieving seizure freedom, ≥ 50% reduction in seizure frequency) and safety (discontinuation due to adverse events) of all AEDs were estimated using a comprehensive network meta-analysis. Perampanel dosing scheme was varied and tested based on a separate meta-analysis of placebo-controlled brivaracetam and perampanel trials. The primary outcomes were direct medical costs (including drug acquisition, monitoring, adverse event management, treatment initiation and switching costs) and quality-adjusted lifeyears (QALYs) gained. Results were discounted at 3% per annum during the 5-year modelling timeframe. Results: Total costs were estimated at € 25,788 and € 25,026 for brivaracetam and perampanel, respectively. Respective QALYs were 3.662 and 3.605, resulting in incremental cost-effectiveness ratio (ICER) of € 13,357 per QALY gained. ICERs ranged between € 7,925 – € 29,170/QALY when varying perampanel dose. When omitting perampanel from the brivaracetam arm, or analyzing brivaracetam and perampanel as adjunctive AEDs on top of only one AED, brivaracetam became dominant (cost saving and more effective than perampanel). Results were robust in both deterministic and probabilistic sensitivity analyses. Brivaracetam had high probability of being cost-effective even at low willingness-to-pay thresholds. Conclusions: BRV not only expands the availability of effective treatment choices, but is also affordable providing value for money in the Finnish settings. PND30 Cost-Effectiveness of Levetiracetam for Patients With Juvenile Myoclonic Epilepsy: A Modeling Approach By The Brazilian Public Health Care System Perspective Kayo M1, Rosim RP2, Duva A2, Ballalai Ferraz AF2, Carmo E1
VA L U E I N H E A LT H 2 0 ( 2 0 1 7 ) A 3 9 9 – A 8 1 1
carried out. Results: Integrating cfDNA in T21 prenatal screening strategy prior to invasive diagnosis for pregnant women with a risk threshold of at least 1/250, leads to an additional cost of € 440,000, while improving the number of detected prenatal T21s by more than 40, and reducing the number of invasive procedures by 13 300 and the number of related miscarriages by 18. When comparing the modelling strategies integrating cfDNA, the low bounds of 1/250 and 1/1000, and the high bounds of 1 and 1/50, appear to be the most relevant choices based on non-weighted outcomes. Conclusions: Implementation of cfDNA as a contingent test in prenatal screening can improve foetal T21s detection while avoiding procedure related miscarriages. From a health economic point of view, implementation of cfDNA in France seems to be most relevant for threshold risks ranging between [1/250; 1[, [1/1000; 1[, or [1/1000; 1/50[. Final HAS recommendations were based on model results, as well as on ethical, preferential and organisational aspects and expert opinion. PND25 Adequately Reflecting The Clinical Benefits In Rare Disease Economic Modeling Using Sma Type I As A Casestudy Zuluaga S1, Knight C1, Thompson R2, Teynor M3 Health Solutions, Manchester, UK, 2Biogen, Zug, Switzerland, 3Biogen, Cambridge, MA, USA
1RTI
Objectives: Spinal muscular atrophy (SMA) is a rare, hereditary, autosomal recessive neuromuscular disorder caused by deletion of the survival motor neuron 1 gene (SMN1). Type I SMA is one of the most severe forms of SMA that affects infants between 0-6 months of age where they never develop the ability to sit and have a short life expectancy. Nusinersen is the first approved treatment for SMA, and prior management of the disease centred on the symptomatic treatment of respiratory, nutritional, and orthopaedic function decline. The objective of this study was to build a model to adequately reflect the clinical benefits of a novel treatment (nusinersen) in a rare disease which has resulted in patients achieving motor function abilities not previously observed. Methods: A decision analytic model was developed based on the clinical trial outcome measures, registry data and clinical opinion via an advisory board. Health states were based on both motor milestones consistent with Type I SMA and for motor milestones not previously experienced by Type I SMA patients, such as sitting without support and standing with assistance due to the improvement in motor function experienced by patients treated with nusinersen. Due to paucity of data, quality of life utility values were derived from a vignette study. Data for the major clinical events such as scoliosis surgery were based on the literature. Results: The resulting model structure showed that over a 40-year time horizon patients treated with nusinesen gained an average of 6.5 life-years and over 6 quality-adjusted life years (undiscounted). Conclusions: The resulting model structure is a basis on which a full economic model can be developed to support nusinersen in future Health Technology Assessments (HTAs). PND26 A Budget Impact Analysis of An Increased Uptake of Alemtuzumab From The UK NHS Perspective Rog D1, Guo JD2, Nucit A3, Le Bagousse-Bego G2, Chevli M4, Chung L2 1Salford Royal NHS Foundation Trust, Salford, UK, 2Sanofi, Cambrige, MA, USA, 3Ividata, Levallois-Perret, France, 4Sanofi, Guildford, Surrey, UK
Objectives: To estimate the impact of increased use of alemtuzumab 12 mg/day in the treatment of adult RRMS patients (per its marketing authorization) on the UK National Health Service (NHS) budget. Methods: A budget impact analysis was conducted for an average of 121,200 RRMS patients in the UK over a 5-year time horizon (start year: 2017) from the NHS payer perspective. The number of RRMS patients was derived from UK Office of National Statistics (2015) by considering prevalence and incidence of RRMS in the UK. Five direct costs considered were treatment/administration, resource use/monitoring, adverse events, disability progression (assessed by Expanded Disability Status Scale scores), and relapse. The model compared the cost difference in each year under two different market scenarios: current market mix, in which the market share of alemtuzumab 12 mg/day was projected to remain consistent with current shares (2017: 1.6%; 2018: 1.8%; 20192021: 1.5%), and an alternative market mix, in which market share of alemtuzumab 12 mg/day was forecasted to increase 0% to 1% per year (2017: 2.6%; 2018: 2.2%; 2019: 2.1%; 2020-2021: 1.5%). Results: Using the alternative market mix in the UK NHS payer perspective, alemtuzumab starts saving in Year 3 (2019) compared with the current market mix, with an average annual total cost saving of £10,976,253 over 5 years for all RRMS population. The annual total costs for the first 2 years increased with the alternative mix, which can be explained by initial high upfront costs of alemtuzumab. However, these increases are offset by the reduction in drug usage in subsequent years, leading to an overall cost saving. Conclusions: From the UK NHS perspective, compared to the current market mix, the alternative market mix with increased uptake of alemtuzumab leads to an average annual saving of £10 million over 5 years, starting from Year 3 (2019). Study Support: Sanofi PND27 Disease-Modifying Therapy Is Associated With Lower Medical Costs In Patients With Multiple Sclerosis With or Without Corticosteroid Treatment: A Us Retrospective Claims-Based Study Sanchirico M1, Caldwell-Tarr A2, Isikwe P1, Hashemi L1, Dufour R2 Health Insights: A Humana Company, Louisville, KY, USA
1Sanofi, Cambridge, MA, USA, 2Comprehensive
Objectives: To compare, using corticosteroid use as a surrogate marker for relapse activity, healthcare utilization and costs among patients with multiple sclerosis (MS) who do/do not receive disease-modifying therapy (DMT). Methods: Healthcare claims analysis included MS patients with or without DMT during the 12 months prior to the most recent claim, stratified by number of corticosteroid treatments (0, 1, or ≥ 2) during this period. Costs were assessed based on claims during the 1-year observation period. Results: Of 7072 patients covered by Medicare, 4689 (66%)
received DMT. Patients with DMT were less likely to have a corticosteroid claim than patients not receiving DMT (39% vs 62%), and less likely to have an ER visit (39% vs 54%) or hospitalization episode (18% vs 29%). Overall medical costs were lower with DMT use. For patients without a corticosteroid claim, total medical costs were $8,037 for DMT vs $13,604 for no DMT; inpatient costs were $15,085 vs $20,802; ER costs were $1,204 vs $1,508. Costs rose with increased corticosteroid treatments. In patients with ≥ 2 corticosteroid treatments, total medical costs (DMT vs no DMT) were $13,688 vs $18,728; inpatient costs were $14,320 vs $17,890; ER costs were $1,674 vs $2,172. Rates of post-steroid DMT switching were low (12%–16%) even in patients with ≥ 2 episodes of corticosteroid use. Conclusions: DMT use was associated with fewer corticosteroid treatments (potentially indicating fewer relapses), fewer hospitalization episodes, fewer ER visits, and lower medical costs, indicative of better health outcomes. DMT use lowered healthcare utilization and costs in patients with and without corticosteroid treatments. Corticosteroid treatments were associated with increasing utilization and costs, but did not prompt increased DMT switching. Study Support: Sanofi PND28 Cost-Effectiveness of Alemtuzumab In The Treatment of Relapsing Forms of Multiple Sclerosis In The United States and Societal Spillover Effects Chirikov V1, Ma I2, Joshi N1, Patel D1, Smith A2, Giambrone C2, Cornelio N1, Hashemi L2 1Pharmerit International, Bethesda, MD, USA, 2Sanofi, Cambridge, MA, USA
Objectives: Caring for patients with relapsing multiple sclerosis (RMS) poses economic and humanistic burdens that are well-documented, but in the United States (US) are seldom incorporated into the cost-effectiveness appraisal of disease modifying treatments (DMTs). Alemtuzumab (ALEM) was compared to ocrelizumab (OCR) and natalizumab (NAT), accounting for societal spillover effects due to RMS treatment. Methods: A Markov model with annual cycles and 20-year time horizon was run separately from US payer and societal perspectives. The societal perspective analysis built upon the payer perspective by including productivity loss costs, informal care costs, and caregiver disutility. For ALEM, the population represented characteristics of pooled treatment-naive and non-naive patients from the CARE MS I and II trials (NCT00530348, NCT00548405). The British Columbia Multiple Sclerosis cohort was used to model the natural history of RMS disease progression. Network meta-analysis provided data on the relative efficacy of DMTs in reducing relapses and slowing disability. Withdrawal rates, treatment waning, resource use, costs and utility inputs were populated using published data and clinical expert opinion. Information on adverse events was derived from package inserts and published sources. Results: From US payer perspective, ALEM dominated comparators by accumulating more quality-adjusted life years (QALYs) and lower costs vs OCR (+0.499; -$486,368) and NAT (+0.516; -$626,578). The benefit of ALEM over comparators further increased when the analysis was conducted from the societal perspective vs OCR (+0.556; -$513,813) and vs NAT (+0.575; -$655,263). Sensitivity analyses identified the DMT withdrawal rate as one of the most influential model parameters. Conclusions: The cost-effectiveness of ALEM was driven by its durable efficacy in the absence of continuous treatment and by savings from treatment acquisition costs over the time horizon. ALEM generated greater positive spillover effects to society by reducing caregiver disutility, informal care, and productivity loss versus comparators. Study Support: Sanofi PND29 Cost-Effectiveness of Brivaracetam As Adjunctive Therapy for Partial-Onset Epilepsy In The Finnish Setting Väätäinen S1, Soini E1, Peltola J2, Charokopou M3, Taiha M4, Kälviäinen R5 Oy, Kuopio, Finland, 2Tampere University Hospital, Tampere, Finland, 3UCB Pharma, Brussels, Belgium, 4UCB Pharma, Espoo, Finland, 5University of Eastern Finland & Kuopio University Hospital, Kuopio, Finland 1ESiOR
Objectives: To evaluate the cost-effectiveness of brivaracetam and perampanel as adjunctive antiepileptic drugs (AED) in the treatment of partial-onset seizures with or without secondary generalization in adult and adolescent patients with epilepsy in Finland. Methods: A discrete event simulation approach was used to model: 1) brivaracetam and perampanel as 3rd concomitant AEDs on top of two AEDs, with perampanel being used after brivaracetam among other adjunctive AEDs, 2) perampanel omitted from brivaracetam arm, 3) brivaracetam and perampanel as adjunctive AEDs on top of only one AED. Comparative treatment efficacy (achieving seizure freedom, ≥ 50% reduction in seizure frequency) and safety (discontinuation due to adverse events) of all AEDs were estimated using a comprehensive network meta-analysis. Perampanel dosing scheme was varied and tested based on a separate meta-analysis of placebo-controlled brivaracetam and perampanel trials. The primary outcomes were direct medical costs (including drug acquisition, monitoring, adverse event management, treatment initiation and switching costs) and quality-adjusted lifeyears (QALYs) gained. Results were discounted at 3% per annum during the 5-year modelling timeframe. Results: Total costs were estimated at € 25,788 and € 25,026 for brivaracetam and perampanel, respectively. Respective QALYs were 3.662 and 3.605, resulting in incremental cost-effectiveness ratio (ICER) of € 13,357 per QALY gained. ICERs ranged between € 7,925 – € 29,170/QALY when varying perampanel dose. When omitting perampanel from the brivaracetam arm, or analyzing brivaracetam and perampanel as adjunctive AEDs on top of only one AED, brivaracetam became dominant (cost saving and more effective than perampanel). Results were robust in both deterministic and probabilistic sensitivity analyses. Brivaracetam had high probability of being cost-effective even at low willingness-to-pay thresholds. Conclusions: BRV not only expands the availability of effective treatment choices, but is also affordable providing value for money in the Finnish settings. PND30 Cost-Effectiveness of Levetiracetam for Patients With Juvenile Myoclonic Epilepsy: A Modeling Approach By The Brazilian Public Health Care System Perspective Kayo M1, Rosim RP2, Duva A2, Ballalai Ferraz AF2, Carmo E1