Net Budget Impact Research Articles

Budget Impact Analysis of the Introduction of Rituximab and Trastuzumab Intravenous Biosimilars to EU-5 Markets.

BackgroundBiologic treatments impose a large financial burden on healthcare payers. Subcutaneous formulations of trastuzumab and rituximab offer administration cost savings relative to the intravenous products through reduced preparation and infusion times. However, intravenous biosimilars have the potential to offset administration costs through lower drug costs.ObjectiveThe objective was to develop a budget impact model (BIM) from a payer’s perspective for the EU-5 countries (UK, France, Germany, Spain, Italy) to demonstrate the economic impact of using intravenous trastuzumab and rituximab biosimilars.MethodsAn incidence-based BIM was developed to estimate the net budget impact utilising epidemiology data from the literature, market research data on the use of relevant treatments in all approved indications, and corresponding costs. The budget impact was estimated for 5 years following introduction of the biosimilars.ResultsAnalysis using the base-case results indicated that adoption of the biosimilars trastuzumab and rituximab would result in net cost savings. At year 5, the net budget saving ranged from €4.05 million to 303.86 million for rituximab and from €19 million to 172 million for trastuzumab. The cost saving could potentially extend treatment to 291–15,671 more patients with rituximab and 622–3688 more patients with trastuzumab.ConclusionThis budget impact analysis emphasised that increased use of intravenous rituximab and trastuzumab biosimilars may result in cost savings from the payer’s perspective across the EU-5 countries.Supplementary InformationThe online version contains supplementary material available at 10.1007/s40259-020-00461-8.

Evolution of the Greater Manchester Nutrition and Hydration Programme.

Older adults in the community are at risk of malnutrition and dehydration. The present article aims to outline an intervention and a population-health approach to raise awareness of the importance of good nutrition and hydration in later life. This was addressed by developing strong partnership working, governance frameworks and local steering committees. Through the Greater Manchester Nutrition and Hydration Programme, 39 500 older people have been asked about appetite and weight loss and/or used the PaperWeight Armband to date. A total of 5586 people from this population were found to be at risk of malnutrition. All were provided resources, advice and signposting to address this issue. The gross fiscal return on investment over a 5-year period was 3.2-fold and the net present budget impact was £800 000. The long-term cashable fiscal return on investment was estimated at 2.69. This very promising approach has potential to enable older adults to extend their healthy life span and deliver cost savings to the health and care system.

Economic evaluation of rotavirus vaccination in children of Bhutan

BackgroundDiarrhoea remains one of the top ten causes of under-five child morbidity in Bhutan, and rotavirus is a significant cause of child diarrhoeal hospitalisations. This study sought to determine the health outcomes, cost-effectiveness, and budget and human resource implications of introducing rotavirus vaccines in the routine immunisation program to inform Bhutan’s decision-making process. MethodsWe used UNIVAC model (version 1.3.41) to evaluate the cost-effectiveness of a rotavirus vaccination programme compared with no vaccination from a government perspective. We also projected the impact of rotavirus vaccination on human resources and budget. Acost-effectiveness threshold was determined to be 0.5 times the gross domestic product (GDP) per capita (equivalent to the United States dollar ($) 1,537) per Disability-Adjusted Life-Year (DALY) averted.One-way deterministic and probabilistic sensitivity analyses, and threshold analyses were performed to capture parameter uncertainties. ResultsIn Bhutan, a rotavirus vaccination programme over 10 years (2020 to 2029) can avert between 104 and 115 DALYs, at an incremental cost ranging from $322,000 to $1,332,000. The incremental cost-effectiveness ratio (ICER) across four vaccination programmes compared to no vaccination scenario were $9,267, $11,606, $3,201, and $2,803 per DALY averted for ROTARIX, RotaTeq, ROTAVAC, and ROTASIIL, respectively. The net five-year budget impact of introducing a rotavirus vaccination programme ranged from $0.20 to $0.81 million. The rotavirus vaccination programme has a potential to reduce the workload of health care workers such as paediatricians, nurses, dieticians, and pharmacists; however, the programme would require an additional 1.93–2.88 full-time equivalent of health assistants. ConclusionAt the current cost-effectiveness threshold, routine rotavirus vaccination in Bhutan is unlikely to be cost-effective with any of the currently available vaccines. However, routine vaccination with ROTASIIL was under the cost-effectiveness threshold of one times the GDP per capita ($3,074). ROTASIIL and ROTAVAC would provide the best value for money in Bhutan.

PRO18 BUDGET IMPACT ANALYSIS OF DAMOCTOCOG ALFA PEGOL IN SEVERE HEMOPHILIA A IN GREECE

To estimate the impact on the Greek public payer budget from the use of damoctocog alfa pegol (DaPeg), an extended half-life recombinant factor VIII, for prophylactic and on-demand (OD)-treatment of patients ≥12 years with severe haemophilia A (HemA) having received previous treatment in Greece. A 5-year budget impact model (BIM) was adapted with local data. The BIM compares a market scenario where a proportion of HemA patients receives DaPeg to an alternative market scenario where DaPeg is not available and patients are treated with available comparators, namely efmoroctocog-alfa, turoctocog-alfa, moroctocog-alfa and octocog-alfa (rAHF-PFM, BAY 81-8973). Epidemiologic data were derived by combining data from three Greek hemophilia centers and the World Hemophilia Federation (2017). Assuming equivalent efficacy (bleeding rates) across all comparators, clinical outcomes were not modelled. Current and future market shares for prophylaxis and OD-treatment were provided by the manufacturer. Only treatment costs were considered for prophylaxis and OD-treatment (€, 2019) with the doses obtained from the SPC and clinical trials, respectively. The difference between the total costs in each market scenario was the net budget impact. For a total of 231 patients with severe HemA, the introduction of DaPeg with a market share ranging from 5% to 27% in prophylaxis and 3% to 9% in OD-treatment from year 1 to 5, respectively, results in cost savings compared to the world without DaPeg. The budget saving magnitude increases with each successive year of the model horizon, driven by the lower annual per-patient costs of DaPeg. The cost savings increase from €99,510 in year 1 to €833,567 in year 5. The cumulative cost-savings over the modelled period exceed €2.5million. The introduction of DaPeg into the Greek market of severe HemA results in a negative budget impact, generating cost savings for the public payer in the first 5 years of its use.

PIN80 CEFTAZIDIME-AVIBACTAM FOR THE TREATMENT OF CARBAPENEM-RESISTANT PATHOGENS ASSOCIATED WITH HEALTHCARE-ASSOSIATED INFECTIONS: A BUDGET IMPACT ANALYSIS IN ARGENTINA

Our objective was to estimate the budgetary impact of adding Ceftazidime-Avibactam (CAZ-AVI) as a therapy option for hospitalized patients in Argentina. An excel-based budget impact model was adapted to the Argentinean context to estimate budget impact of adding CAZ-AVI as therapy option in hospitalized patients with cUTI, cIAI, HAP/VAP from the payer perspective in 3-year time horizon. Number of eligible patients was estimated from Argentinian epidemiological data. Cure rates were based on clinical trials, published data and local expert opinion. Hospital length of stay associated with treatment cures/failures was derived from clinical trials of CAZ-AVI. Costs of drugs were obtained from ANMAT's National Drug Vademecum. Costs of hospital stay were based on tariffs from private institutions of Argentina. Costs were expressed in 2018 US-dollars (exchange rate: $1=AR$36.8, November 2018). Current and future market shares were based on market research data and assumptions. Results are reported in terms of annual and per-patient budget impact. In Argentina, the estimated number of patients eligible for CAZ-AVI was 5,995, 6,388 and 6,791, in each year respectively. The estimated net budget impact for the introduction of CAZ-AVI was - $171,513; - $452,487 and -$690,710 for the 1 to 3 years respectively. The cumulative net budget impact was -$1,314,710, representing an impact of -1.07% compared to a scenario without CAZ-AVI, and mainly driven by the reduction in hospital bed days and hospitalization costs. The total cost per treated patient, prior to CAZ-AVI introduction, was estimated to be $6,412 [$7,210 (cIAI), $5,265 (cUTI), $8,072 (HAP/VAP)] and $6,343 [$6,918 (cIAI), $5,199 (cUTI), $8,229 (HAP/VAP)] after the introduction of CAZ-AVI. Our results suggest that the inclusion of CAZ-AVI as a therapeutic option in hospitalized patients with cUTI, cIAI, HAP/VAP could generate budgetary savings compared to current treatment options.

PCV67 BUDGET IMPACT OF RIVAROXABAN 2.5MG IN COMBINATION WITH ASPIRIN IN TREATMENT OF PATIENTS WITH CORONARY ARTERY DISEASE (CAD) OR PERIPHERAL ARTERY DISEASE (PAD) IN GREECE

To evaluate the budget impact of introducing rivaroxaban 2.5mg (RIV) in combination with aspirin 100 mg (ASA) in patients with CAD or PAD in order to assess the affordability of this therapy for the Greek public healthcare system over a 5-year time horizon. The populations modelled reflect the overall COMPASS (NCT01776424) population (CAD or PAD). The model considers the Greek National Healthcare Payer perspective and as such, only direct costs were included. Model inputs included: risk of main events and associated mortality (based on COMPASS); epidemiology data; current and future market share projections for treatments; and data on the following cost items based on national reimbursed prices and DRGs tariffs (e.g. Official Price Bulletin, Government Gazettes): treatment costs, administration costs, disease-related costs (physician visits and laboratory tests), the management cost of potential associated main events, such as myocardial infarction, ischaemic stroke, intracranial haemorrhage, cardiovascular death, bleeding death and the management of potential health events, such as acute limb ischemia, minor and major amputation, and venous thrombo-embolism. Introducing rivaroxaban for the treatment of patients with coronary artery disease or peripheral artery disease in Greece would result in €639,957 and €13,558,417 budget impact in the 1st and 5th year, respectively. The treatment of CAD or PAD with RIV+ASA would have a total net budget impact of €41.6m in five years. Drug cost is the key driver of the increasing total cost. The introduction of rivaroxaban reduces the cost of acute main events, post main events, health events and mortality by €5,173,990 over five years. The progressive introduction of rivaroxaban would result in a manageable budget impact for the Greek healthcare system and represent elevated savings in the management of acute main events, post main events, health events and mortality due to rivaroxaban’s clinical benefits and associated costs.

PCN240 POTENTIAL BUDGET IMPACT OF ADJUVANT TREATMENTS FOR STAGE III MELANOMA IN IRELAND

To estimate the potential budget impact of adjuvant treatment using checkpoint inhibitors and BRAF targeted treatments, for stage III resected melanoma in Ireland. A budget impact model was developed in line with the Guidelines for Budget Impact Assessment produced by the Health Information and Quality Authority, Ireland. The perspective was that of the drugs budget in the publicly funded health system. Time horizon was 5 years. Included regimens were nivolumab, pembrolizumab, and dabrafenib in combination with trametinib (DAB-TRAM), in line with the dosing schedules in the product licenses. Drug costs were estimated based on median treatment duration in the pivotal trials. Routine care was observation; analysis of national drug reimbursement data showed little use of interferon. It was assumed that 75% patients would receive a checkpoint inhibitor, evenly split between nivolumab and pembrolizumab, and the remaining 25% DAB-TRAM. Eligibility was estimated based on incident melanomas (2015) and a weighted average of the annual percentage change, using National Cancer Registry data. Uncertainty was considered using one-way sensitivity analysis (OWSA) and scenario analyses. Costs are VAT inclusive. The predicted cost per patient treated was €139,032, €99,743 and €138,668 for pembrolizumab, nivolumab and DAB-TRAM respectively. It was estimated that 162 patients would be eligible for treatment in 2019, increasing to 188 by 2023. The estimated 5-year gross budget impact was €108.4 million. Since there were no cost-offsets, this also represents the net budget impact. Cost offsets in the metastatic setting were not considered, given the immaturity and paucity of data on retreatment in the metastatic setting. Eligible patient population and estimated drug costs were drivers of uncertainty in OWSA; estimates ranged from €86.7 million to €138 million. Estimates were robust to scenario analysis. Provision of adjuvant treatments for melanoma in Ireland is expected to be associated with significant drugs budget impact.

Budget Impact Analysis of Rotavirus Vaccine Adoption in the Childhood Immunization Schedule of Iran

ObjectivesTo examine the budget impact of introducing the RotaTeq® vaccine (Merck and Co Inc, West Point, PA) to the national immunization program in Iran. MethodsThe pre- and postvaccine introduction costs were compared. The total annual costs included the vaccination and diarrhea treatment costs. The health outcome was the estimated annual cases of the disease. To evaluate the net budget impact, the annual prevaccine introduction cost was reduced from the postvaccine introduction cost. The sensitivity analysis was done to reduce the uncertainties. ResultsThe total cost of vaccination for 5 years would be more than $184 million. Nevertheless, the financial savings would be about $45 million and $7.5 million because of the reduction in the number of patients after vaccination in inpatient and outpatient sectors, respectively. So the incremental cost would be $131 450 210 during 5 years of immunization. ConclusionsThe results show that the inclusion of rotavirus vaccine in the national vaccination program would have a significant effect on health budgets and would raise government expenditure.

Eligibility, Clinical Outcomes, and Budget Impact of PCSK9 Inhibitor Adoption: The CANHEART PCSK9 Study.

BackgroundThe FOURIER (Further Cardiovascular Outcomes Research With PCSK9i [Proprotein Convertase Subtilisin‐Kexin Type 9 Inhibitors] in Subjects With Elevated Risk) trial found a reduction in cardiovascular events in patients with atherosclerotic cardiovascular disease (ASCVD). Our objective was to estimate the eligibility, clinical outcomes, and budget impact of adopting PCSK9i in a large healthcare system.Methods and ResultsOntario, Canada, residents alive in 2011, aged 40 to 85 years, were eligible for inclusion. PCSK9i eligibility was determined on the basis of FOURIER trial definition. Hazard ratios observed in the FOURIER trial were applied to assess the number of events that could be avoided. Budget impact was calculated as the difference between projected costs of treatment adoption and events avoided if PCSK9i were used. Of the 2.4 million included individuals, 5.3% had a history of ASCVD. We estimated that 2.7% of the general population and 51.9% of the patients with ASCVD would be eligible for PCSK9i. Adoption of PCSK9i in all eligible patients with ASCVD was projected to reduce primary events rates by 1.8% after 3 years. Despite cost reduction of $44 million in events, PCSK9i adoption would have a net budget impact of $1.5 billion over 3 years. Potential benefits of PCSK9i varied widely across subgroups, with the largest absolute risk reduction estimated to be 4.3% at 3 years in peripheral artery disease. In this subgroup of 5601 patients, the budget impact of treatment adoption was $116 million.ConclusionsWe estimated that ≈1 in 2 patients with ASCVD would be eligible for PCSK9i. The budget impact of adopting PCSK9i for all patients with ASCVD is substantial. Selective adoption to high‐risk patients will lessen the overall budgetary impact of PCSK9i treatment.

CP3 - PATIENTS-INTERVENTION-COMPARATORS-OUTCOMES-SETTINGS-TIME-EFFECTS-SENSITIVITY (PICOSTEPS): REPRESENTING EVIDENCE-BASED HEALTH ECONOMIC EVALUATIONS (EBHEE) IN THE ORDER OF IMPORTANCE

Evidence-based medicine (EBM) defines clinical questions in terms of the Patients/population-Intervention-Comparators-Outcomes, i.e. PICO. PICO has important role in EBM and is commonly applied in evidence assembly for systematic literature reviews, meta-analyses and clinical care guidelines (e.g. the Finnish Current Care Criteria, CCC, http://www.kaypahoito.fi/web/english/home). However, PICO alone is less suitable for representing the information of evidence-based health economic evaluations (EBHEE). To extend PICO to cover the crucial elements of EBHEEs. A systematic literature review was done to study the applicability of published HEEs for psoriatic arthritis treatments (http://www.kaypahoito.fi/web/kh/suositukset/suositus?id=nix02465&suositusid=hoi50062, http://www.terveysportti.fi/xmedia/nix/nix02465liitteet.pdf) during the work by the Finnish psoriasis CCC working group (http://www.kaypahoito.fi/web/kh/suositukset/suositus?id=hoi50062). During the study, international and Finnish guidelines for HEE were reviewed. Based on the review, Patients, Intervention, Comparators, Outcomes, Settings, Time, Effects and Sensitivity (PICOSTEPS) framework for EBHEEs was established and applied. In comparison to EBM-PICO, the PICOSTEPS Outcomes can include e.g. incremental cost-effectiveness ratios, net benefit, expected value of perfect information, net budget impact or burden avoided depending on the methods and settings applied. The Settings can be incremental, average or summing; depicted e.g. as cost effectiveness/utility, cost benefit, value of information, budget impact or burden of disease/treatment. The Time can vary, from a short within-trial horizon to a lifetime modelling. The Effects are commonly applied based on the trial, meta-analysis or real-world clinical effects, quality of life, resource use and/or productivity loss. The Sensitivity analyses applied for the assessment of uncertainty include e.g. one/multiway deterministic scenarios, probabilistic sensitivity analyses and expected value of partial perfect/sample information. PICOSTEPS has the potential to define HEE in more clinician-familiar way (vs. common HEE guidelines or checklists), and can demonstrate if the minimum information of EBHEE has been reported. PICOSTEPS is being successfully applied to EBHEEs and it represents their crucial information in the potential order of importance.

PCN70 - ASSESSING THE BUDGET IMPACT OF INCLUDING SUNITINIB FOR THE TREATMENT OF METASTATIC RENAL CELL CARCINOMA IN ARGENTINA

Sunitinib is an oral multi-targeted tyrosine kinase inhibitor approved for the first- and second-line treatment of metastatic renal cell carcinoma (mRCC). The purpose of this study was to assess the budget impact of including sunitinib as first-line therapy (1L) option for mRCC patients, within a 3 year time horizon from the payer perspective in Argentina. An excel-based budget impact model (BIM) was adapted to the context of Argentina to determine the incremental cost of introducing sunitinib in the 1L for patients with mRCC. The analysis was conducted with 2 comparators based on Argentina treatment pattern: pazopanib and sunitinib. Epidemiology data of mRCC was obtained from the literature. The drugs costs were obtained from ANMAT's National Drug Vademecum. The costs of drug administration and patient monitoring, and adverse events costs were estimated through a microcosting approach, based on published data and expert opinion, and were based on tariffs from healthcare institutions (social security and private sector) of Argentina. The market share of the different drugs was based on market studies and assumptions. The budget impact is reported in terms of annual budget impact, per-member per-year (PMPY) and per-patient per-year (PPPY). In Argentina assuming a population of 1 million people, patients eligible for 1L treatment were estimated at 27. The net budget impact estimated for the introduction of sunitinib was -$9,250.08 each year. The cumulative net budget impact was -$27,750.24, representing an impact of -1.94% compared to a scenario without sunitinib. The incremental cost PMPY was -$0.01, and the incremental cost PPPY was -$338.48. In one-way sensitivity analysis the price of sunitinib was the only one that showed a significant influence on the results. Our results suggest that the inclusion of sunitinib as a therapeutic option in patients with mRCC could generate budgetary savings compared to treatment with pazopanib.

Highly Innovative Drug Program in the Czech Republic: Description and Pharmacoeconomic Results—Cost-Effectiveness and Budget Impact Analyses

Highly innovative drugs (HIDs) can be granted 2 to 3 years of temporary reimbursement (TR) to provide timely patient access and to collect real-world evidence through registries in the Czech Republic. A TR applicant does not need to comply with cost-effectiveness (CE) requirements and the willingness-to-pay threshold. It is only when mandatory transition to permanent reimbursement (PR) status occurs does the drug need to comply with CE and willingness-to-pay requirements. To describe and evaluate the HID program in the Czech Republic by analyzing the pharmacoeconomic results when a drug starts with TR status and transitions to PR status. The study was a retrospective analysis of reimbursement decisions of HIDs. All drugs approved for TR (valid from January 2008 to January 2018) were identified. A description of the HIDs and their pharmacoeconomic results were analyzed. Fifty TR drugs were identified. Most (68%) were oncology drugs and 44% were orphan drugs. After the expiration of their TR status, 83% were successfully transitioned to PR status. Cost-utility analysis was used to support CE results in 42% of the TR drugs. The mean incremental cost-effectiveness ratio (cost/quality-adjusted life-year) of drugs that entered TR status was €97,868. When the time came for transition to PR status, the mean incremental cost-effectiveness ratio was €34,086 (lower by 65%). Net budget impact increased by 3% and decreased by 25% in the first and fifth years, respectively, after applying for PR. This analysis provides better insight into the HID program for costly innovative drugs over a 10-year follow-up. A successful transition to PR status was observed for most of the HIDs (83%).

US budget impact of increased payer adoption of the Flexitouch advanced pneumatic compression device in lymphedema patients with advanced chronic venous insufficiency and multiple infections

Aims: To assess the budget impact to a US commercial health plan of providing access to the Flexitouch (FLX) advanced pneumatic compression device (Tactile Medical) to lymphedema (LE) patients with either comorbid chronic venous insufficiency (CVI) or frequent infections.Methods: Budget impact was calculated over 2 years for a hypothetical US payer with 10-million commercial members. Model inputs were derived from published sources and from a case-matched analysis of Blue Health Intelligence (BHI) claims data for the years 2012–2016. To calculate the budget impact, the Status Quo budget (i.e. total cost for LE and sequelae-related medical treatment) was compared to the budget under each of three Alternate Payer Policy scenarios which assumed that a sub-set of patients was redistributed from their initial treatment groups to a group that received FLX. Model outputs included cumulative payer costs, net budget impact, and breakeven point. Sensitivity analyses were performed to assess the impact of model inputs on results.Results: Increasing access to FLX yielded a favorable budget impact in every scenario. For LE patients with comorbid CVI, the three alternate scenarios resulted in cumulative 2-year budget impacts of –$52,841, –$173,317, and –$375,601, respectively. For LE patients with comorbid frequent infections, the three alternate scenarios resulted in cumulative 2-year budget impacts of –$192,729, –$259,339, and –$613,179, respectively.Limitations: Use of claims data assumes accurate coding and does not allow one to control for disease severity or treatment adherence. Also, the distribution of patients between treatment arms was determined using claims data from a specific payer organization, and could differ for health plans with different coverage policies.Conclusions: While previous studies have illustrated cost savings with adoption of FLX, US commercial health plans may also achieve tangible cost savings by expanding access to FLX for LE patients with comorbid CVI and multiple infections.

Implementation of drug-eluting stents for the treatment of femoropopliteal disease provides significant cost-to-system savings in a single-state outpatient simulation

ObjectivesInitial data on drug-eluting stents (DES) shows that they may increase the durability of endovascular treatment of superficial femoral artery disease compared with traditional bare-metal stents (BMS). Observed decreased target lesion revascularization (TLR) rates have potential for cost savings despite an increased initial cost. The purpose of this study was to run a simulation model of progressive transition from BMS to DES over 5 years evaluating the overall cost impact of that transition. MethodsFlorida State Ambulatory Databases were searched for all patients undergoing superficial femoral artery stenting in 2013 using Current Procedural Terminology codes 37226 and 37227. A simulation model was developed to estimate the impact of a progressive transition from BMS to DES over a 5-year horizon in this patient population. Cost estimates were determined from available cost charge ratio data. For the 5-year model, 2013 served as the initial year with each subsequent year based on the expected number of interventions per year. Up to one TLR per patient was assumed for the model. The 5-year TLR rates for DES and other parameter estimates were based on pooled data from the literature. Institutional data were used to estimate that up to 48% of superficial femoral artery lesions would fit the instructions for use for the Zilver PTX (Cook Medical, Bloomington, Ind), which is currently the only DES approved by the U.S. Food and Drug Administration for peripheral interventions. The net budget impact was expressed as the difference in total costs (primary stenting and reinterventions) for a scenario where BMS is progressively replaced by Zilver PTX compared with a scenario of BMS only. Multiple sensitivity analyses were performed on the base scenario. ResultsWe identified 4107 peripheral interventions in the first year that fit our study. The overall cost for these procedures in Florida database was $51,362,142.00. In the base case scenario, DES was introduced slowly into the population at a rate of 8% per year up to 48% at the end of the model. This strategy resulted in an overall cost savings of $1,688,953.72 compared with the model with BMS alone. Sensitivity analyses including slower adoption of DES up to only 24% at 5 years, a 20% increase in TLR rates per year for the DES, and a 10% reduction in TLR rates per year for BMS still resulted in a net savings. As long as the additional cost of a DES compared with BMS is less than $677, the DES model remains less expensive. ConclusionsThe adoption of DES in lieu of traditional BMS can lead to significant cost savings in a single state model over a short time horizon.

The potential price and access implications of the cost-utility and budget impact methodologies applied by NICE in England and ICER in the US for a novel gene therapy in Parkinson’s disease

ABSTRACTBackground: NICE in England, and ICER in the US both use cost-utility analyses (CUA) and budget impact analyses (BIA) to assess value for money and affordability, however the thresholds used differ greatly.Objective: To perform a cross-country comparison of the results of the CUA and BIA and detail the implications for reimbursed price and volumes, for a novel gene therapy for Parkinson’s disease (PD).Methods: A Markov model was built to perform country-specific CUAs and BIAsFindings: The US ceiling price identified through CUA is ~ 1.8 times higher than in England (aligning to our previous US/UK price comparison analysis of high-cost drugs). However, the net budget impact corresponding to these price levels would limit number of patients treated in order not to exceed the BIA threshold. Performance-based annuity payments can increase patient access at launch without exceeding the thresholds while reducing payers’ data uncertainty.Conclusion: Our cost-utility analysis in PD shows a difference in price potential between the US and England that aligns with what is observed in practice for other high-cost drugs. Furthermore, the budget impact threshold operational in England imposes a greater downwards pressure on price and/or volumes than the one applied by ICER in the US.

Biphasic insulin aspart 30 treatment for people with type 2 diabetes: a budget impact analysis based in Thailand

Objective: To assess the financial consequences of different adoption rate of Biphasic Insulin Aspart (BIAsp) 30 instead of Biphasic Human Insulin (BHI) 30 for people with type 2 diabetes (T2DM) in Thailand from the payer’s perspective.Methods: The Excel-based International T2DM Budget Impact Model over a 3-year period was used. The cohort was the T2DM patients who received treatment from government hospitals under the Universal Health Coverage Scheme. Demographic, the adverse events, and the costs were derived from published studies in Thailand. Efficacy was based on meta-analysis. Adoption rates were assumed to increase each year. Net budget impact (NBI) and one-way sensitivity were analyzed.Results: Hypoglycemia costs were lower in BIAsp 30 compared with BHI 30. The NBI per year was 26,511,269 THB (771,349 USD) for year 1, 52,181,133 THB (1,518,218 USD) for year 2, and 76,189,608 THB (2,216,747 USD) for year 3. The NBI per insulin user per year was 33.45 THB (0.97 USD), 67.27 THB (1.96 USD), 101.49 THB (2.95 USD) from year 1 to year 3, respectivelyConclusions: Lower rate of hypoglycemia with BIAsp 30 than those treated with BHI 30 generates cost savings resulting in significant deduction in the additional acquisition cost of BIAsp 30. Therefore, the NBI per insulin user per year has become small.

Pharmacoeconomic Results Of Highly Innovative Drugs Approved For Temporary Reimbursement In The Czech Republic

Highly innovative drugs can be granted two plus one year of temporary reimbursement (TR) to provide timely access and to collect additional real-world evidence through registry. TR applicant does not need to comply with strict cost-effectiveness (CE) requirements and willingness-to-pay threshold (WTP) when there is no sufficient data about use in clinical practice or about CE at a time. After two/three years when switching to permanent reimbursement (PR), a drug must comply with strict CE and WTP requirements. The main objective was to analyse pharmacoeconomic results at the entry of drug into TR and compare them with results presented in consecutive PR procedure. All drugs approved for TR until 5/2017 were identified. Pharmacoeconomic results (i.e. type of analysis, incremental cost-effectiveness ratios (ICERs), net budget impact (netBI)) at the time of TR and consecutive PR application were analysed. We also examined therapeutic class, and compared the length of approval of TR versus PR. 40 TR decisions were identified. Majority (70%) belonged to oncology drugs, 33% were orphans. After the expiration of TR, 92% had positive subsequent decision on PR. Price negotiation with payers was present in 50% of cases. 8% did not manage to receive PR. Cost-utility analysis was used to present results of cost-effectiveness in 44% of TR procedures. Mean ICER (cost/QALY) of TR products was €93,087 (SD €64,550). In the subsequent PR procedure, the mean ICER was €40,937 (lower by 56%). NetBI lowered by 32% and 50% in 1st and 5th year respectively when applying for PR. Mean decision time about TR was longer (404 days; SD 194) than consecutive decision about PR (259 days; SD 251). Costly innovative drugs are accepted for a TR with higher ICERs and netBI than usually accepted in PR procedure. 92% of these drugs succeed in consecutive PR procedure.

Impact of Comprehensive Genomic Profiling of Patients With First Line Non-Small Cell Lung Cancer In The UK

To estimate the impact of increased use of Comprehensive Genomic Profiling (CGP) together with targeted and immuno-therapies consistent with NCCN Guidelines®, versus conventional molecular diagnostic testing, on cost and overall survival (OS) in the UK. A 5-year budget impact forecasting model was developed to determine the financial implications of incorporating CGP to guide first-line treatment options in an incident cohort of patients with advanced lung carcinoma. Costs including molecular diagnostics, associated medical and treatment expenses were calculated for the duration of first-line treatment. Future treatment regimens were forecasted based on published biomarker status. Costs were identified from literature and technology assessment reports. Extensive sensitivity analyses were conducted to investigate the impact of key model parameters on results. The incremental budget impact with 10% uptake of CGP was estimated to be £0.02 per member per month. This increased usage of CGP equates to a net total budget impact of £17.5M for eligible patients in the UK (15% related to cost of CGP;62% due to treatment costs;23% costs due to improved survival). Number-Needed-to-Treat for 1 additional life year is approximately 3 patients. Patients receiving CGP had longer survival by 2.62 months. Cost offsets are achieved in repetitive biopsy procedures and non-matched drug treatment costs. That is in contrast to increases in costs of molecular testing and matched drug treatment costs. CGP may be an affordable option to identify the most appropriate treatment regimen for an individual patient. Further research is warranted better understand how current therapies can be best utilized to improve overall survival. Furthermore, health systems may need to move from a tumour type based treatment approach to a mutation specific based indication as mounting evidence suggest cancer is driven by genomically matched indications rather than anatomical location.

A Budget Impact Model of The Addition of Telotristat Ethyl Treatment In Patients With Uncontrolled Carcinoid Syndrome

Carcinoid syndrome (CS) is a rare condition in patients with neuroendocrine tumors (NETs), characterized by flushing and diarrhea that severely impact patients’ quality of life. Though typically treated by somatostatin analogs (SSAs) as standard of care, many patients nevertheless experience uncontrolled CS symptoms despite therapy. Telotristat ethyl (TE) is a novel tryptophan hydroxylase inhibitor granted EMA orphan designation that has been shown in phase 3 studies to effectively reduce bowel movement frequency in CS. We developed a model to evaluate the 5-year budget impact of introducing TE in combination with SSAs (TE+SSAs) in patients with uncontrolled CS in Sweden. Results from the phase 3 TELESTAR trial (NCT01677910) informed TE efficacy in the model, based on the 12-week treatment response, after which health states were captured by a Markov model using 4-week cycles. The model also allowed for TE discontinuation based upon treatment response data from the TELESTAR open-label extension period. Systematic and targeted literature reviews informed CS and carcinoid heart disease (CHD) prevalence, incidence and mortality. These data were used to estimate the number of patients in Sweden eligible for treatment. Real-world CS and CHD-related costs (healthcare resource use, drug acquisition, SSA dosage) were obtained from a Swedish database study. Market share of TE was assumed to increase annually, from 2% to 10% uptake by Year 5. The net 5-year budget impact of TE+SSAs was 11,898€, which translated to a cost of less than 4.88€ per patient per month in each year of the analysis. As a much-needed treatment for a rare and potentially debilitating condition, the 5-year budget impact of the addition of TE to existing standard of care was projected to be 11,898€ according to our model, suggesting that TE may be an affordable treatment option for patients with this rare disease.

Pembrolizumab as second-line therapy in non-small cell lung cancer in northern Norway: budget impact and expected gain—a model-based analysis

BackgroundPembrolizumab is a new drug approved in several countries for second-line therapy in non-small cell lung cancer (NSCLC) being programmed cell death ligand (PD-L1) positive. This drug has a high cost, and the cost-effectiveness ratio has been debated.Patients and methodsThe budget impact to the Northern Norwegian Regional Health Authority trust of implementing pembrolizumab in second-line therapy in patients with PD-L1-positive NSCLC was calculated. A model was developed employing data from the Cancer Registry of Norway, the KEYNOTE-010 study, the price list from The Hospital Pharmacy of North Norway, the cost of analysing PD-L1 expression and the cost of travelling. Today's cost of second-line therapy was compared with the new standard employing pembrolizumab. The sale price of pembrolizumab in Norway was not published due to price confidentiality. Norwegian krone (NKr) was converted into Euros (€) at a rate of 1€=Nkr 8.8138. (Bank of Norway, 21 February 2017).Results105 new patients were identified available for pembrolizumab per year. The annual cost of pembrolizumab was €5.2 million, hospital pharmacy administration costs €0.1 million, PD-L1 testing €0.3 million, oncologist/pulmonologist/nurses €0.2 million, radiology €0.06 million and transportation €0.4 million. Savings due to avoided present second-line therapy was calculated €0.4 million. Consequently, the cost of implementing pembrolizumab was €5.5 million and the annual budget impact was €5.0 million. A mean gain of at least 9 months per patient treated was necessary to make pembrolizumab cost-effective.ConclusionsThe net budget impact of pembrolizumab was €5.0 million. The expenditure could not be indicated cost-effective. Price confidentiality is a growing problem in health economics and it has become a ‘menu without prices’ setting.