AIM: To evaluate the real-world comparative effectiveness and the cost-effectiveness, from a UK National Health Service perspective, of natalizumab versus fingolimod in patients with rapidly evolving severe relapsing-remitting multiple sclerosis (RES-RRMS). METHODS: Real-world data from the MSBase Registry were obtained for patients with RES-RRMS who were previously either naive to disease-modifying therapies or had been treated with interferon-based therapies, glatiramer acetate, dimethyl fumarate, or teriflunomide (collectively known as BRACETD). Matched cohorts were selected by 3-way multinomial propensity score matching, and the annualized relapse rate (ARR) and 6-month-confirmed disability worsening (CDW6M) and improvement (CDI6M) were compared between treatment groups. Comparative effectiveness results were used in a cost-effectiveness model comparing natalizumab and fingolimod, using an established Markov structure over a lifetime horizon with health states based on the Expanded Disability Status Scale. Additional model data sources included the UK MS Survey 2015, published literature, and publicly available sources. RESULTS: In the comparative effectiveness analysis, we found a significantly lower ARR for patients starting natalizumab compared with fingolimod (rate ratio [RR] = 0.65; 95% confidence interval [CI], 0.57-0.73) or BRACETD (RR = 0.46; 95% CI, 0.42-0.53). Similarly, CDI6M was higher for patients starting natalizumab compared with fingolimod (hazard ratio [HR] = 1.25; 95% CI, 1.01-1.55) and BRACETD (HR = 1.46; 95% CI, 1.16-1.85). In patients starting fingolimod, we found a lower ARR (RR = 0.72; 95% CI, 0.65-0.80) compared with starting BRACETD, but no difference in CDI6M (HR = 1.17; 95% CI, 0.91-1.50). Differences in CDW6M were not found between the treatment groups. In the base-case cost-effectiveness analysis, natalizumab dominated fingolimod (0.302 higher quality-adjusted life-years [QALYs] and £17,141 lower predicted lifetime costs). Similar cost-effectiveness results were observed across sensitivity analyses. CONCLUSIONS: This MSBase Registry analysis suggests that natalizumab improves clinical outcomes when compared with fingolimod, which translates to higher QALYs and lower costs in UK patients with RES-RRMS.

There are several medications used to treat people with relapsing remitting multiple sclerosis, such as interferon-based therapies (Betaferon/Betaseron (US), Rebif, Avonex, Extavia), glatiramer acetate (Copaxone), teriflunomide (Aubagio), and dimethyl fumarate (Tecfidera), collectively named BRACETD. Other treatments for multiple sclerosis (MS) have a narrower use, such as natalizumab (Tysabri) or fingolimod (Gilenya), among others.This study objective was to assess how well natalizumab and fingolimod helped treating MS (clinical effectiveness) and subsequently estimate what the cost of these treatments is in comparison to the benefit they bring to people with rapidly evolving severe MS that use them in the United Kingdom (UK) (cost-effectiveness).We used an international disease registry (MSBase), which collects clinical data from people with MS in various centers around the world to compare the effectiveness of natalizumab, fingolimod and BRACETD treatments. We used a technique called propensity score matching to obtain results from comparable patient groups. People treated with natalizumab had better disease control, namely with fewer relapses and higher improvement on their disability level, than patients on fingolimod or BRACETD. Conversely, there were no differences between each group of people on a measure called disability worsening.Based on these clinical results, we built an economic model that simulates the lifetime costs and consequences of treating people with MS with natalizumab in comparison with fingolimod. We found that using natalizumab was less costly and was more effective compared to using fingolimod in UK patients.

• Klíčová slova
• I, I1, I10, I11, Multiple sclerosis, comparative effectiveness, cost-effectiveness, fingolimod, natalizumab, real-world data,
• MeSH
• analýza nákladové efektivity MeSH
• analýza nákladů a výnosů MeSH
• fingolimod hydrochlorid terapeutické užití   MeSH
• imunosupresiva terapeutické užití   MeSH
• lidé MeSH
• natalizumab terapeutické užití   MeSH
• relabující-remitující roztroušená skleróza * farmakoterapie   MeSH
• roztroušená skleróza * farmakoterapie   MeSH
• státní lékařství MeSH
• Check Tag
• lidé MeSH
• Publikační typ
• časopisecké články MeSH
• Geografické názvy
• Spojené království MeSH
• Názvy látek
• fingolimod hydrochlorid MeSH
• imunosupresiva MeSH
• natalizumab MeSH

BACKGROUND: Breast cancer (BC) poses a public health challenge as the most commonly diagnosed cancer among women globally. While BC mortality has declined across Europe in the past three decades, an opposite trend has been reported in some transitional European countries. This analysis estimates the mortality burden and the cost of lost productivity due to BC deaths in nine Central and Eastern Europe (CEE) countries: Bulgaria, Croatia, Czech Republic, Hungary, Poland, Romania, Serbia, Slovakia, and Slovenia, that have defied the favorable cancer mortality trends. These estimates may provide relevant evidence to aid decision-makers in the prioritization of BC-targeted policies. METHODS: The human capital approach (HCA) was used to estimate years of life lost (YLL) and productivity losses due to premature death from BC (ICD-10 code: C50 Malignant neoplasm of breast). YLL and present value of future lost productivity (PVFLP) were calculated using age and gender-specific mortality, wages, and employment rates. Data were sourced from the World Health Organization (WHO), Eurostat, and the World Bank. RESULTS: In 2019, there were 19,726 BC deaths in the nine CEE countries. This study estimated BC deaths resulted in 267,184 YLL. Annual PVFLP was estimated to be €85 M in Poland, €46 M in Romania, €39 M in Hungary, €21 M in Slovakia, €18 M in Serbia, €16 M in Czech Republic, €15 M in Bulgaria, €13 M in Croatia, and €7 M in Slovenia. CONCLUSION: Premature death from BC leads to substantial YLL and productivity losses. Lost productivity costs due to premature BC-related mortality exceeded €259 million in 2019 alone. The data modeled provide important evidence toward resource allocation priorities for BC prevention, screening, and treatment that could potentially decrease productivity losses. Careful consideration should be given to BC-specific policies, such as surveillance programs and the availability of new treatments in CEE countries to decrease the medical and financial burden of the disease.

• Klíčová slova
• Health economics, I, I1, I10, I18, TNBC, breast cancer, health policy, indirect costs,
• MeSH
• lidé MeSH
• nádory prsu * MeSH
• Check Tag
• lidé MeSH
• ženské pohlaví MeSH
• Publikační typ
• časopisecké články MeSH
• Geografické názvy
• Česká republika MeSH
• Evropa epidemiologie   MeSH
• Polsko MeSH
• východní Evropa epidemiologie   MeSH

AIMS: This study assessed the cost-effectiveness of the subcutaneous RANKL inhibitor, denosumab, vs the intravenous bisphosphonate, zoledronic acid, for the prevention of skeletal-related events (SREs) in patients with prostate cancer, breast cancer, and other solid tumors (OST) in the Czech Republic. MATERIALS AND METHODS: A lifetime Markov model was developed to compare the effects of denosumab and zoledronic acid on costs (including drug costs and administration, patient management, SREs, and adverse events), quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios from a national payer perspective. Different discount rates, time horizons, SRE rates, distributions, and nature (asymptomatic vs all SREs), and the inclusion of treatment discontinuation were considered in scenario analyses. The robustness of the model was tested using deterministic and probabilistic sensitivity analyses. RESULTS: Across tumor types, denosumab was associated with fewer SREs, improved QALYs, and higher total costs over a lifetime. The incremental cost per QALY gained for denosumab vs zoledronic acid was 382,673 CZK for prostate cancer, 408,450 CZK for breast cancer, and 608,133 CZK for OST. Incremental costs per SRE avoided for the same tumor type were 54,007 CZK, 51,765 CZK, and 94,426 CZK, respectively. In scenario analyses, the results remained similar to baseline, when different discount rates and time horizons were considered. At a non-official willingness-to-pay threshold of 1.2 million CZK, the probabilities of denosumab being cost-effective vs zoledronic acid were 0.64, 0.67, and 0.49 for prostate cancer, breast cancer, and OST, respectively. LIMITATIONS: The SRE rates used were obtained from clinical trials; studies suggest rates may be higher in clinical practice. Additional evidence on real-world SRE rates could further improve the accuracy of the modeling. CONCLUSIONS: Compared with zoledronic acid, denosumab provides a cost-effective treatment option for the prevention of SREs in patients with prostate cancer, breast cancer, and OST in the Czech Republic.

• Klíčová slova
• Bone, Breast cancer, Cost-effectiveness, Czech Republic, Oncology, Prostate cancer,
• MeSH
• analýza nákladů a výnosů MeSH
• bisfosfonáty aplikace a dávkování   ekonomika   MeSH
• denosumab aplikace a dávkování   ekonomika   MeSH
• dvojitá slepá metoda MeSH
• ekonometrické modely MeSH
• imidazoly aplikace a dávkování   ekonomika   MeSH
• inhibitory kostní resorpce aplikace a dávkování   ekonomika   MeSH
• kvalita života MeSH
• kvalitativně upravené roky života MeSH
• kyselina zoledronová MeSH
• lidé MeSH
• Markovovy řetězce MeSH
• nádory prostaty komplikace   MeSH
• nádory prsu komplikace   MeSH
• nádory komplikace   MeSH
• nemoci kostí etiologie   prevence a kontrola   MeSH
• Check Tag
• lidé MeSH
• mužské pohlaví MeSH
• ženské pohlaví MeSH
• Publikační typ
• časopisecké články MeSH
• klinické zkoušky, fáze III MeSH
• randomizované kontrolované studie MeSH
• Geografické názvy
• Česká republika MeSH
• Názvy látek
• bisfosfonáty MeSH
• denosumab MeSH
• imidazoly MeSH
• inhibitory kostní resorpce MeSH
• kyselina zoledronová MeSH

OBJECTIVE: Complications contribute largely to the economic gravity of diabetes mellitus (DM). How they arise and are treated differs substantially between countries. This paper assesses the total annual, direct, and indirect cost of severe hypoglycemia events (SHEs) in nine European countries: Bulgaria, Croatia, the Czech Republic, Greece, Hungary, Macedonia/the former Yugoslav Republic of Macedonia (MK), Poland, Slovenia, and Spain. METHODS: Data was collected on epidemiology, treatment structure, SHE-driven resource consumption, and unit costs. Two systematic reviews-on the SHE rates and the resources used for treatment-and data on the days-of-work lost due to SHE along with salaries and employment rates were used. The total SHE cost in each country was calculated and how the differences are driven by individual parameters was analysed. RESULTS: The annual costs of SHEs varied in absolute terms from €379,951.25 in MK up to €58,429,684.40 in Spain, or-when expressed per one drug-treated DM patient-from €5.47 in Bulgaria up to €17.74 in Spain. Indirect cost constituted between 6.01% (MK) and 26.49% (Hungary) of the total cost. The differences between countries are driven mostly by the cost of treating a single event, and this is related to general differences in prices. LIMITATIONS: The main limitation is the lack of good quality data in some parts, and the necessity to use mean-value imputations, experts' opinions, etc. Additionally, we only considered DM treatment as the SHE driver, while other elements, e.g. style of living, may contribute substantially. CONCLUSIONS: A common framework can be applied to estimate the economic burden of SHE in various countries, allowing one to identify the drivers of differences in cost. Treating DM is complex, and so no resolute conclusions ought to be drawn as to whether SHE management is better in one country than another.

• Klíčová slova
• Diabetes mellitus, cost study, direct cost, indirect cost, severe hypoglycemia,
• MeSH
• hypoglykemie ekonomika   patologie   MeSH
• lidé MeSH
• náklady a analýza nákladů * MeSH
• náklady na zdravotní péči trendy   MeSH
• osobní újma zaviněná nemocí * MeSH
• stupeň závažnosti nemoci MeSH
• Check Tag
• lidé MeSH
• Publikační typ
• časopisecké články MeSH
• Geografické názvy
• Evropa MeSH

Objective Patients with bone metastases or lesions secondary to solid tumors or multiple myeloma often experience bone complications (skeletal-related events [SREs]-radiation to bone, pathologic fracture, surgery to bone, and spinal cord compression); however, recent data that can be used to assess the value of treatments to prevent SREs across European countries are limited. This study aimed to provide estimates of health resource utilization (HRU) and cost associated with all SRE types in Europe. HRU data were reported previously; cost data are reported herein. Methods Eligible patients from 49 centers across Austria (n = 57), the Czech Republic (n = 59), Finland (n = 60), Greece (n = 59), Portugal (n = 59), and Sweden (n = 62) had bone metastases or lesions secondary to breast, lung, or prostate cancer, or multiple myeloma, and ≥1 index SRE (a SRE preceded by a SRE-free period of ≥ 6.5 months). SRE-related costs were estimated from a payer perspective using health resource utilization data from patient charts (before and after the index SRE diagnosis). Country-specific unit costs were from 2010 and local currencies were converted to 2010 euros. Results The mean costs across countries were €7043, €5242, €11,101, and €11,509 per radiation to bone, pathologic fracture, surgery to bone, and spinal cord compression event, respectively. Purchasing power parity (PPP)-adjusted mean cost ratios were similar in most countries, with the exception of radiation to bone. Limitations The overall burden of SREs may have been under-estimated owing to home visits and evaluations outside the hospital setting not being reported here. Conclusions All SREs were associated with substantial costs. Variation in SRE-associated costs between countries was most likely driven by differences in treatment practices and unit costs.

• Klíčová slova
• Bone metastases, Cost, Europe, Skeletal-related events, Supportive care,
• MeSH
• ekonometrické modely MeSH
• fraktury spontánní ekonomika   etiologie   MeSH
• komprese míchy ekonomika   etiologie   MeSH
• lidé středního věku MeSH
• lidé MeSH
• nádory kostí komplikace   ekonomika   sekundární   MeSH
• senioři MeSH
• výdaje na zdravotnictví statistika a číselné údaje   MeSH
• zdravotnické služby ekonomika   statistika a číselné údaje   MeSH
• Check Tag
• lidé středního věku MeSH
• lidé MeSH
• mužské pohlaví MeSH
• senioři MeSH
• ženské pohlaví MeSH
• Publikační typ
• časopisecké články MeSH
• Geografické názvy
• Evropa MeSH

PURPOSE: The Czech Republic is faced with making choices between pharmaceutical products, including depot injectable antipsychotics. A pharmacoeconomic analysis was conducted to determine the cost-effectiveness of atypical depots. METHODS: An existing 1-year decision-analytic framework was adapted to model drug use in this healthcare system. The average direct costs to the General Insurance Company of the Czech Republic of using paliperidone palmitate (Xeplion®), risperidone (Risperdal Consta®), and olanzapine pamoate (Zypadhera®) were determined. Literature-derived clinical rates populated the model, with costs adjusted to 2012 Euros using the consumer price index. Outcomes included quality-adjusted life-years (QALYs), days in remission, and proportions hospitalized or visiting emergency rooms. One-way sensitivity analyses were calculated for all important inputs. A multivariate probability analysis was used to examine the stability of results using 10,000 iterations of simulated input over reasonable ranges of all included variables. RESULTS: Expected average costs/per patient treated were €5377 for PP-LAI, €6118 for RIS-LAI, and €6537 for OLZ-LAI. Respective QALYs were 0.817, 0.809, and 0.811; ER visits were 0.127, 0.134, and 0.141; hospitalizations were 0.252, 0.298, and 0.289. Results were generally robust in sensitivity analyses. PP-LAI dominated RIS-LAI and OLZ-LAI in 90.2% and 92.1% of simulations, respectively. Results were insensitive to drug prices but sensitive to adherence and hospitalization rates. CONCLUSIONS: PP-LAI dominated the other two drugs, as it had a lower overall cost and superior clinical outcomes, making it the preferred choice. Using PP-LAI in place of RIS-LAI for chronic relapsing schizophrenia would reduce the overall costs of care for the healthcare system.

• MeSH
• analýza nákladů a výnosů MeSH
• analýza rozptylu MeSH
• antipsychotika ekonomika   terapeutické užití   MeSH
• benzodiazepiny ekonomika   terapeutické užití   MeSH
• chronická nemoc MeSH
• dospělí MeSH
• ekonomika farmaceutická MeSH
• isoxazoly ekonomika   terapeutické užití   MeSH
• kvalitativně upravené roky života MeSH
• léky s prodlouženým účinkem ekonomika   terapeutické užití   MeSH
• lidé MeSH
• metody pro podporu rozhodování MeSH
• mladý dospělý MeSH
• multivariační analýza MeSH
• náklady na léky * MeSH
• olanzapin MeSH
• paliperidon palmitát MeSH
• palmitany ekonomika   terapeutické užití   MeSH
• risperidon ekonomika   terapeutické užití   MeSH
• schizofrenie diagnóza   farmakoterapie   ekonomika   MeSH
• Check Tag
• dospělí MeSH
• lidé MeSH
• mladý dospělý MeSH
• mužské pohlaví MeSH
• ženské pohlaví MeSH
• Publikační typ
• časopisecké články MeSH
• práce podpořená grantem MeSH
• srovnávací studie MeSH
• Geografické názvy
• Česká republika MeSH
• Názvy látek
• antipsychotika MeSH
• benzodiazepiny MeSH
• isoxazoly MeSH
• léky s prodlouženým účinkem MeSH
• olanzapin MeSH
• paliperidon palmitát MeSH
• palmitany MeSH
• risperidon MeSH