PP14 Budget Impact Of Sapropterin Dihydrochloride For Phenylketonuria

2019 ◽  
Vol 35 (S1) ◽  
pp. 40-40
Author(s):  
Eduardo Mulinari ◽  
Nayara Castelano Brito ◽  
Lays Pires Marra

IntroductionThe National Committee for Health Technology Incorporation (CONITEC) evaluates health technologies to recommend their inclusion or exclusion within the Brazilian Public Health System (SUS), and uses the budget impact assessment to estimate costs to the system. The Ministry of Health (MS) guideline recommends treatment of phenylketonuria (PKU) with restricted phenylalanine diet and phenylalanine-free amino acid formula (PFAAf) supplementation. CONITEC evaluated the inclusion of sapropterin dihydrochloride for PKU in the SUS.MethodsThe population eligible for treatment was evaluated by the number of patients receiving PFAAf between 2014 and 2017 registered in the SUS. Patients were stratified by age/weight and a simple linear regression was performed to estimate the future population. The costs of treatment and testing the responsiveness of sapropterin dihydrochloride were estimated according to the recommended dosage guideline of the MS, leaflet and public purchasing prices. A univariate deterministic sensitivity analysis was performed to evaluate different prices, responsiveness test methods and variations in the reduction of formula use.ResultsThe incorporation of sapropterin dihydrochloride would generate an incremental budget impact in the SUS of around BRL 79 million (USD 21.7 million) in 2019 and BRL 300 million (USD 82.1 million) in five years (2019-2023). The univariate sensitivity analysis estimated that the incremental budget impact could be between BRL 66 and BRL 103 million (USD 18 and USD 28 million) in the first year and between BRL 251 and BRL 388 million (USD 69 and USD 106 million) in five years. Sensitivity analysis showed that the price of sapropterin dihydrochloride was the most sensitive variable in the model.ConclusionsThe incorporation of sapropterin dihydrochloride in the SUS represents a significant budgetary impact and covers a small number of patients. Sapropterin dihydrochloride was recommended by CONITEC for the treatment of women with PKU, with a positive drug responsiveness test, and who are in the preconception period or in the gestational period.

2019 ◽  
Vol 35 (S1) ◽  
pp. 75-75
Author(s):  
Eduardo Mulinari ◽  
Nayara Castelano Brito ◽  
Lays Pires Marra

IntroductionThe National Committee for Health Technology Incorporation (CONITEC) evaluates health technologies to recommend their inclusion or exclusion within the Brazilian Public Health System (SUS), and uses the budget impact assessment to estimate costs to the system. This study estimated the budget impact of the supply of methionine-free amino acid formula (MFAAf) for patients with classical homocystinuria (HCU) in the SUS.MethodsThe incidence of one case per 250,000 live births in Brazil and the registration of a Brazilian association of patients with HCU was assumed to calculate the population. Mortality and responsiveness to pyridoxine rates were applied. The costs of treatment were estimated according to the recommended dosage in literature and public purchasing prices. For calculating the dose of MFAAf patients, a median age of 19 years and weight of 60 kg were assumed, according to Brazilian study data.ResultsThe annual cost of treatment was estimated at BRL 77,000 (USD 21,084) per patient. The incorporation of MFAAf for HCU would generate a budget impact in SUS of around BRL 37 million (USD 10.1 million) in 2019 and BRL 188 million (USD 51.5 million) after five years which considers the epidemiological data, and a budget impact of around BRL 6.4 million (USD 1.75 million) in 2019 and BRL 33 million (USD 9 million) after five years which considers the information of a Brazilian association of patients with HCU. The wide range of values in the incremental budgetary impact is due to the lack of information on the epidemiology of the disease in Brazil.ConclusionsThe incorporation of the MFAAf in the SUS represents an important budgetary impact and covers a small number of patients. CONITEC recommended the incorporation of the MFAAf in the SUS, according to clinical protocol.


2010 ◽  
Vol 26 (2) ◽  
pp. 163-169 ◽  
Author(s):  
Timo T. Purmonen ◽  
Päivi K. Auvinen ◽  
Janne A. Martikainen

Objectives:Adjuvant trastuzumab is widely used in HER2-positive (HER2+) early breast cancer, and despite its cost-effectiveness, it causes substantial costs for health care. The purpose of the study was to develop a tool for estimating the budget impact of new cancer treatments. With this tool, we were able to estimate the budget impact of adjuvant trastuzumab, as well as the probability of staying within a given budget constraint.Methods:The created model-based evaluation tool was used to explore the budget impact of trastuzumab in early breast cancer in a single Finnish hospital district with 250,000 inhabitants. The used model took into account the number of patients, HER2+ prevalence, length and cost of treatment, and the effectiveness of the therapy. Probabilistic sensitivity analysis and alternative case scenarios were performed to ensure the robustness of the results.Results:Introduction of adjuvant trastuzumab caused substantial costs for a relatively small hospital district. In base-case analysis the 4-year net budget impact was €1.3 million. The trastuzumab acquisition costs were partially offset by the reduction in costs associated with the treatment of cancer recurrence and metastatic disease.Conclusions:Budget impact analyses provide important information about the overall economic impact of new treatments, and thus offer complementary information to cost-effectiveness analyses. Inclusion of treatment outcomes and probabilistic sensitivity analysis provides more realistic estimates of the net budget impact. The length of trastuzumab treatment has a strong effect on the budget impact.


2020 ◽  
Vol 15 (1) ◽  
pp. 73-82
Author(s):  
A. V. Rudakova ◽  
E. A. Stadnik

Background. Currently, treatment of recurrent / refractory chronic lymphocytic leukemia (CLL) involves the appointment of regimens with innovative drugs, which include ibrutinib and a combination of venetoclax and rituximab. Wherein said combination provides continuing over time high frequency eradication of minimal residual disease. Thereby, this regimen can be canceled if patients do not progress after 2 years from therapy start.The objective of the study was to assess the pharmacoeconomic aspects of therapy with venetoclax and rituximab combination in patients with recurrent / refractory CLL compared with ibrutinib monotherapy.Materials and methods. Analysis was performed by a simulation method from a position of the health care system. In accordance with network meta-analysis results of clinical studies in the recurrent / refractory CLL treatment (MURANO for venetoclax + rituximab combination and RESONATE and HELIOS for ibrutinib), which showed the absence of statistically significant differences in progression-free and overall survival between these treatment options, the cost-minimization method was used in the analysis. In the basic version, the model time horizon is 4 years. The price of venetoclax, rituximab and ibrutinib in the calculation corresponded to that registered (for rituximab – the median of the registered prices) with the value-added tax and the weighted average wholesale allowance taking into account the population in Russia.When analyzing the healthcare system budget impact, the time horizon of the study was 4 years. Therapy with combination of venetoclax + rituximab starting from the first year was suggested in 100 % of newly identified recurrent / refractory CLL patients. In the base case, it was estimated that 100 patients would need therapy every year.In the basic version of analysis, the cost of therapy after progression was not taken into account. In sensitivity analysis, an option taking into account therapy cost after progression, suggesting the appointment of venetoclax in the ibrutinib group and ibrutinib in the venetoclax + rituximab group, was also evaluated. In addition, variants with disease progression were additionally evaluated in 15 % of patients per year in the venetoclax therapy group at the end of the 2-year treatment course, as well as with an increase and decrease in the disease progression rate by 15 % in both comparison groups.As part of the sensitivity analysis, an assessment is made of a 15 % decrease and increase in Venetoclax price, a 30 % decrease in Ibrutinib price compared to registered price and the option with a 3-year study time horizon. When analyzing the budget impact, options with an increase in the number of patients annually identified and requiring treatment by 10, 20, 30 and 50 % were evaluated. Clinical and economic analysis was carried out with a discount rate of 3.5 % per year. A budget impact analysis was performed without discounting.Results. According to the results of cost-effectiveness analysis in the basic version, a regimen including venetoclax can reduce costs by 46.3 % compared with ibrutinib (cost for 4 years per patient is 10.422 and 19.413 million rubles, respectively). Therapy with combination of venetoclax + rituximab in 100 newly identified recurrent / refractory CLL patients annually instead of ibrutinib monotherapy will result in a reduction in therapy costs by 29.0 %, or by 1.579 billion rubles for 4 years per 100 patients starting therapy annually. The sensitivity analysis demonstrated the high reliability of the results. Conclusion. The treatment of recurrent / refractory CLL with a combination of venetoclax and rituximab is comparable in clinical efficacy with ibrutinib monotherapy and can significantly reduce the cost of the healthcare system, and therefore increase the availability of innovative therapy for this group of patients.


2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 1612.2-1613
Author(s):  
J. M. Bello-Gualtero ◽  
O. J. Calixto ◽  
G. Salguedo ◽  
Y. M. Chamorro-Melo ◽  
C. A. Camargo Rodríguez ◽  
...  

Background:Spondyloarthritis refers to a family of diseases, of which ankylosing spondylitis and non-radiographic axial spondyloarthritis are responsible for axial impairment. Previously, the only treatment available were NSAIDs, which control activity and stop radiological progression, but at the expense of increased adverse effects, such as cardiovascular risk, dyspepsia and chronic renal failure. For the past 2 decades, biological therapy has been available, which means an increase in care costs.Objectives:The objective of this study is to perform a budget impact analysis of biologic therapy.Methods:To do a budget impact analysis from the perspective of the payer, comparing biological therapy with coventional therapy for the treatment of spondyloarthritis. Demographic characterization of the population attended at the Central Military Hospital. Time horizon from 2012 to 2018, taking the activity count according to the hospital’s billing and the prices of the activities of the state body SISMED. Exchange rates at the end of 2018.Results:The patients attended were 117, mostly men (63, 25%), average age 46, 4 years (SD 13), with disease diagnosis time of 9, 8 years (SD 9, 6). In the budget impact analysis, it is observed that 25% of patients were on DMARDs therapy, 22% with NSAIDs and 96% with biologic therapy. The average year/patient cost with NSAIDs alone would be EUR 381, with DMARDs only EUR 9,318 and, if only biological therapy was used, EUR 423. Within the total number of patients, the average annual cost, including the possibility of combining these drugs, amounted to EUR 5,403Conclusion:Including biological therapy in the care of patients with spondyloarthritis can increase up to 24 times the annual cost per patient. This increase is not only due to higher market value, it also relates to the need for more medical procedures and diagnostic follow-up tests.References:[1]Strömbeck, et al. Cost of Illness from the Public Payers’ Perspective in Patients with Ankylosing Spondylitis in Rheumatological Care. J Rheumatol 2010;37;2348-2355.Disclosure of Interests:None declared


2020 ◽  
pp. 147332502097329
Author(s):  
Hamed Mortazavi

As the number of patients infected with the 2019 novel coronavirus disease (nCOVID-19) increases, the number of deaths has also been increasing. According to World Health Organization (WHO), as of 4 October 2020, 34,804,348 cases had tested positive for nCOVID-19 globally, which among them, 1,030,738 confirmed deaths had occurred, equivalent to a case-fatality rate of 2.96%. However, in comparison with global statistics, the incidence and mortality of the nCOVID-19 infection are higher in Iran. As reported by the National Committee on COVID-19 Epidemiology of Ministry of Health of Iran, the total number of patients with confirmed COVID-19 infection has reached 468,119, of which 26,746 have died, equivalent to a case-fatality rate of 5.71%. Currently, there is solid evidence that older adults are at a higher risk of severe disease following infection from COVID-19.


2008 ◽  
Vol 29 (1) ◽  
pp. 16-24 ◽  
Author(s):  
Gary A. Noskin ◽  
Robert J. Rubin ◽  
Jerome J. Schentag ◽  
Jan Kluytmans ◽  
Edwin C. Hedblom ◽  
...  

Objective.To evaluate the economic impact of performing rapid testing for Staphylococcus aureus colonization before admission for all inpatients who are scheduled to undergo elective surgery and providing subsequent decolonization therapy for those patients found to be colonized with S. aureus.Methods.A budget impact model that used probabilistic sensitivity analysis to account for the uncertainties in the input variables was developed. Primary input variables included the marginal effect of S. aureus infection on patient outcomes among patients who underwent elective surgery, patient demographic characteristics, the prevalence of nasal carriage of S. aureus, the sensitivity and specificity of the rapid diagnostic test for S. aureus colonization, the efficacy of decolonization therapy for nasal carriage of S. aureus, and cost data. Data sources for the input variables included the 2003 Nationwide Inpatient Sample data and the published literature.Results.In 2003, there were an estimated 7,181,484 patients admitted to US hospitals for elective surgery. Our analysis indicated preadmission testing and subsequent decolonization therapy for patients colonized with S. aureus would have produced a mean annual cost savings to US hospitals of $231,538,400 (95% confidence interval [CI], -$300 million to $1.3 billion). The mean annual number of hospital-days that could have been eliminated was estimated at 364,919 days (95% CI, 67,893-926,983 days), and a mean of 935 in-hospital deaths (95% CI, 88-3,691) could have been avoided per year. Sensitivity analysis indicated a 64.5% probability that there would be cost savings to US hospitals as a result of preadmission testing and subsequent decolonization therapy.Conclusion.The addition of preadmission testing and decolonization therapy to standard care would result in significant cost savings, even after accounting for variations in the model input values.


2009 ◽  
Vol 10 (1) ◽  
pp. 19-31 ◽  
Author(s):  
Maurizio Benucci ◽  
Sergio Iannazzo ◽  
Luciano Sabadini

Objective: a Budget Impact analysis was performed to evaluate cost implications for the Italian National Health Service (NHS) of the introduction of rituximab (RTX) in the treatment of rheumatoid arthritis (RA). Methods: RA patients eligible to treatment with a second-line biologic DMARD (Disease Modifying Antirheumatic Drugs) were identified and quantified and available strategies for their management were explored. Costs associated with the different alternatives were estimated, and the impact on the NHS budget was estimated using a cohort simulation based on a Markov chain with a time horizon of 5 years and 1-year cycles. Seven alternative strategies were analyzed, each of them starting after the failure of a first anti-TNFα: 1) the use of a second and a third anti-TNFα; 2) the use of a second anti-TNFα followed by RTX; 3) the use of a second anti-TNFα followed by abatacept (ABAT); 4) the use of RTX as a second biological line, followed by an anti-TNFα; 5) the use of ABAT as a second biological line, followed by an anti-TNFα; 6) the use of RTX as a second biological line, followed by ABAT; 7) the use of ABAT as a second biological line, followed by RTX. Only direct medical costs were considered: drug acquisition, administration, incidental premedication and monitoring exams. Results: Italian patients eligible to second biological line therapies (RA patients refractory or intolerant to at least one anti-TNFα therapy) were estimated in about 650 per year. The adoption of RTX as a second line therapy produced a substantial saving in total costs (-33% at the fifth year) with respect to the strategy with RTX as third line and the one with only anti-TNFα (the last two resulting substantially cost-equivalent). The number of patients in active treatment (biologic DMARD) per unit cost resulted of about 8.1 patient-years/100,000 € with the strategy based only on anti-TNFα and increased of 10% with RTX as a third line. The strategy of RTX as a second line provided a further 41% increase (with respect to RTX as a third line). Conclusions: the introduction of RTX in the treatment of Italian RA patients represents a valuable new therapeutic option for this population especially if anticipated after a first anti-TNFα failure; it can also induce a reduction in health resources consumption for the NHS.


2020 ◽  
Author(s):  
Guido van den berk ◽  
Daoud Ait Moha ◽  
Janneke Stalenhoef ◽  
Marie-Jose Kleene ◽  
Narda van der Meche ◽  
...  

Abstract Background : To support our goal of providing optimal HIV care to our patients, we started applying the value-based health care principle to the HIV care that we offer in our HIV center, measuring relevant health outcomes and costs to allow continuous implementation of improvements (Value-Based HIV Care; VBHiC). Methods : In line with the principles of Michael Porter, our approach consisted of the following steps: 1) Organizing into integrated practice units / describing the HIV care path; 2) Defining an HIV outcome indicator set; 3) Building an enabling information technology platform; 4) Integrating care delivery across separate facilities; 5) Moving to bundled payments for care cycles and 6) Expanding excellent services and interventions for improvement across geographic boundaries. Results : The following set of 9 outcome indicators was developed: undetectable HIV load within the first year of care; quality of life within the first year of care; mortality within the first year of care; retention in care; therapy effectiveness; therapy tolerance; cardiovascular risk; quality of life for every subsequent year and overall annual mortality. These indicators, which were evaluated retrospectively, are shown in figures 1-5. Collection of the underlying data started in January 2016. The HIV care path was also integrated into the electronic file system. Creation of the ability to monitor outcome indicators at patient level, population level and process level allowed us to implement a quality cycle (plan-do-study-act). Conclusion : Our Value-Based HIV Care approach facilitated structured evaluation of parameters that are of value to the patient. It also boosted the quality of the HIV care that we provide and allowed us to increase the number of patients to whom we can offer high quality HIV care.


2020 ◽  
Vol 35 (Supplement_3) ◽  
Author(s):  
Carlo Maria Alfieri ◽  
Paolo Molinari ◽  
Maria Teresa Gandolfo ◽  
Maria Rosaria Campise ◽  
Donata Cresseri ◽  
...  

Abstract Background CMV disease (CMVd) is a frequent complication in renal transplanted patients (RTxp), especially during the first year of transplantation (RTx). CMVd impacts on long term graft and patients outcomes is still debated. Our observational retrospective study aims to evaluate: 1) the prevalence of CMVd during the first year of RTx; 2) the factors related to CMVd; 3) the relationship between CMVd and early and long term graft and patients survival. Method In 505 RTxp (age: 50[41;58]yrs – 292 males), up to the 616 transplanted in our unit between 2004 and 2016, clinical, blood and urinary parameters were recorded after 1 (T1) and 12 (T12) months of RTx. eGFR was estimated by MDRD formula. Donor (D) and Recipient (R) CMV serology was tested at the moment of RTx. CMV IgG-D+/IgG-R- RTxp (12%) and high risk patients (second or more RTx, ATG induction therapy), received prophylaxis therapy until the 3rd month of RTx. CMVd, was defined by the presence either of CMVr (CMV replication without clinical signs of CMV disease) or CMVs (CMV replication with signs of disease and/or need of antiviral therapy/reduction of immunosuppressive therapy). Median follow up (FU) was 8[5-11]yrs. The following outcomes were investigated: 1) graft: reduction of eGFR >20% between T1 and T12; reduction of eGFR >50% between T1 and end of FU (eGFRr>50%); graft loss (GL); eGFRr>50% + GL.; 2) RTxp survival at the end of FU. Results Ninty percent of RTxp had a kidney from a deceased D; 73% and 21% received haemodialysis (HD+) and peritoneal dialysis before RTx. Dialysis vintage was 50[33-75] months. In 12% of RTxp, induction therapy included ATG. Cumulative steroids dose was 880[840-105]mg and 2272[2598-3223]mg at T1 and T12 respectively. During the first year of RTx, 45% of patients had CMVd (CMVd+). CMVd+ were older than CMV free RTxp (CMVd-). Female gender, HD+ and CMV IgG-D+/IgG-R- were more prevalent in CMVd+. In addition, at T1, CMVd+ had lower albumin, haemoglobin and higher PTH, uric acid and reactive C protein levels than CMVd- and, both at T1 and T12, received higher steroid dose. In multivariate analysis, albumin-T1 was the most significant modifiable factor in determining CMVd+ (p=0.009 OR 0.50 – IC 0.29-0.84). Albumin-T1 maintained its significance also after the addiction of CMV serology IgG-D+/IgG-R- to the model (albumin T1: p=0.008 OR 0.48 – IC 0.29-0.84; CMV serology IgG-D+/IgG-R-: p=0.01 OR 2.16 – IC 1,18 -3,95). Among CMVd, 19% and 25% had respectively CMVr and CMVs. CMVs were characterized by lower dialysis vintage and higher prevalence of CMV IgG-D+/IgG-R- than CMVr. GL and death were observed in 11% and 8% of RTxp respectively. In univariate and survival analyses, CMVd, CMVr and CMVs didn’t show any impact on the graft and patients outcomes examined. Conclusion Our retrospective study confirms the high prevalence of CMVd during the first year of RTx, and identifies albumin at T1 as the most impacting parameter in influence CMVd insurgence. This might reflect the importance of the pre-RTx status in CMVd development after RTx. Nevertheless, CMVd seems not impact significantly on early and long term outcomes, experienced however in a small part of the cohort studied. Future studies, possibly prospective and including higher number of patients might better elucidate this issue.


2013 ◽  
Vol 3 (3) ◽  
pp. 17 ◽  
Author(s):  
Dan Brun Petersen ◽  
Thomas Andersen Schmidt

Background: Hospitals in countries with public health systems have recently adopted organizational changes to improve efficiency and resource allocation, and reducing inappropriate hospitalizations has been established as an important goal, as well as avoiding or buffering overcrowding in Emergency Departments (EDs). Aims: Our goal was to describe the impact of a Quick Diagnostic Unit established on January 1, 2012, integrated in an ED setting in a Danish public university hospital following its function for the first year. Design: Observational, descriptive and comparative study. Methods: Our sample comprised the total number of patients being admitted and discharged from the Department of Internal Medicine in 2011 and 2012, with special focus on the General Medicine Ward. Results: Compared with 2011 the establishment of the Quick Diagnostic Unit integrated in the Emergency Department resulted in the admittance and discharge of fewer patients (40%; p < .0001) to the hospital’s General Medicine Ward and 11.6% (p < .0001) fewer patients in the whole Department of Internal Medicine. Conclusions: A Quick Diagnostic Unit integrated in an ED setting represents a useful and fast track model for the diagnostic study and treatment of patients with simple internal medicine ailments, and also serves as a buffer for overcrowding of the ED.


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