Esmase skisofreenia farmakoteraapia Eestis: võrdlus kohaliku ja rahvusvaheliste ravijuhistega

Psühhooside farmakoteraapia ja ravijuhised on eri riikides erinevad. Uuringu eesmärgiks oli analüüsida ja kirjeldada esmaste skisofreeniaepisoodide farmakoteraapiat Eestis. Töös analüü- siti septembrist 2005 kuni septembrini 2006 Põhja-Eesti Regionaalhaigla ja TÜ Kliinikumi psühhiaatriakliinikutesse hospitaliseeritud skisofreenia ja skisotüüpse häirega patsientide farmakoteraapiat. Vaadeldi manustatud ravimite annuseid, antipsühhootikumide polüteraapia esinemise sagedust ning atüüpiliste ja konventsionaalsete antipsühhootikumide määramise sagedust. Tulemusi võrreldi kohalike ja rahvusvaheliste ravijuhistega. Esmase skisofreenia farmakoteraapia Eestis on suures osas vastavuses Eesti ja rahvusvaheliste ravijuhistega, kuid sageli kasutati konventsionaalseid antipsühhootikume ja levinud oli polüteraapia antipsühhootikumidega. Tõenduspõhise farmakoteraapia soodustamiseks oleks otstarbekas ajakohastada Eesti ravijuhiseid. Eesti Arst 2008; 87(9):601−60

Kolorektaalvähi sõeluuringu kulutõhusus Eestis

Taust. Kolorektaalvähk on seedetrakti kasvaja, millesse haigestub Eestis igal aastal 800–900 inimest. Kolorektaalvähk on enamasti aeglase ja astmelise tekkeprotsessiga; tekkivate adenoomide varane avastamine ja eemaldamine võimaldab ära hoida kolorektaalvähi tekke. Kolorektaalvähist tingitud haigestumuse ja suremuse vähendamiseks on mitmes riigis kasutusel fakultatiivsed ja rahvastikupõhised sõeluuringuprogrammid.Eesmärk. Hinnata teaduskirjanduse ja Eesti andmete alusel tervishoiu rahastaja perspektiivist kolorektaalvähi skriinimise kulutõhusust guajakimeetodil põhineva testi (gFOBT), immunokeemilise testi (FIT) ja ühekordse sigmoidoskoopiaga võrreldes mitteskriinimisega.Metoodika. Koostati Markovi mudel 60aastaste kohordi skriinimiseks 10 aasta perspektiivis, mille alusel võrreldi erinevate skriiningumeetodite tervisetulemeid, kulusid ja kulutõhusust.Tulemused. Võrreldes mitteskriinimisega suudetaks skriinimisel osaleva kohordi kohta Eestis 10 aasta jooksul ära hoida 33–74 kolorektaalvähi surmajuhtu ja võita 71–136 kvaliteetset eluaastat (QALY). Täiendkulu tõhususe määr (ICER) lisanduva QALY kohta on FIT puhul 9919 eurot, gFOBT puhul 13 456 eurot ning sigmoidoskoopia puhul 3759 eurot. Kõige enam mõjutab kulutõhususe hinnangut osalusmäär sõeluuringus, vähi ja vähieelsete seisundite esinemistõenäosused, diskonteerimise mitterakendamine ja hindamise ajaperspektiivi pikendamine.Järeldused. Sõeluuring võib vähendada kolorektaalvähist tingitud suremust ja võita kvaliteetseid eluaastaid. Kolme võrreldud skriinimise testmetoodika täiendkulu tõhususe määrad on samas suurusjärgus.Eesti Arst 2015; 94(11):652–65

Clinical aspects of reimbursement policies for orphan drugs in Central and Eastern European countries

Objectives: The aim of this study was to characterize the reimbursement policy for orphan drugs (ODs) in Central and Eastern European (CEE) countries in relation to the availability and impact of clinical evidence, health technology assessment (HTA) procedure, selected economic indicators, and the drug type according to indications.Materials and methods: A list of authorized medicines with orphan designation and information about active substance, Anatomical Therapeutic Chemical (ATC) classification, and therapeutic area was extracted from the web-based register of the European Medicines Agency (EMA). A country-based questionnaire survey was performed between September 2021 and January 2022 in a group of selected experts from nine CEE countries (an invitation was sent to 11 countries). A descriptive and statistical analysis was conducted to determine statistical significance, correlations, between the drug or country characteristic and the positive recommendation or reimbursement of ODs.Results: The proportion of reimbursed orphan drugs differed between countries, ranging from 17.7% in Estonia to 49.6% in Hungary (p < 0.001). The odds that ODs were reimbursed were reduced in countries with a “strong” level of impact of drug safety and efficacy on reimbursement decisions (p=0.018), the presence of other additional specific clinical aspects (e.g., genomic data) considered in the reimbursement decision (p < 0.001) and mandatory (without exception) safety assessments (p=0.004). The probability that ODs were reimbursed was increased in countries with a “moderate” level of impact of drug safety and efficacy on reimbursement decisions (p=0.018), when reimbursement decisions are dependent on the EMA registration status and orphan drug designation (p < 0.001), the presence of the “positive HTA recommendation guarantees reimbursement” policy (p < 0.001), higher GDP per inhabitant (p=0.003), and higher healthcare expenditure (p < 0.001).Conclusion: We found that there are differences among CEE countries in the reimbursement of orphan drugs, and we identified aspects that may influence these differences. Safety, efficacy, and specific clinical aspect issues significantly influenced reimbursement decisions. Antineoplastic and immunomodulating agents drugs were the largest group of ODs and increased the chance of getting a positive recommendation. The higher GDP per inhabitant and healthcare expenditures per inhabitant were positively linked to the chance that an OD receives reimbursement

Reimbursement Legislations and Decision Making for Orphan Drugs in Central and Eastern European Countries

BackgroundReimbursement policies influence access of patients to orphan drugs in the European countries.ObjectivesTo provide a comprehensive description of orphan drug reimbursement policies and to assess reimbursement decision-making process in the EU-CEE countries as well as the impact of the type of approval and disease on reimbursement decisions.MethodsFor each drug, the information regarding conditional approval or approval under exceptional circumstances was obtained from the EMA website. The reimbursement status for analyzed drugs was collected in a questionnaire survey performed in a group of experts in reimbursement policy. The agreement between countries was assessed using the κ coefficient, nominal variables tests were compared using the χ2 test or the Fisher exact test. The impact of the EMA’s conditional approval and approval under exceptional circumstances was assessed using logistic regression and presented as an odds ratio (OR).ResultsThe analysis revealed that most orphan drugs were authorized for the treatment of oncological or metabolic diseases [36 drugs (38%) and 22 drugs (23%), respectively]. The shares of reimbursed orphan drugs varied significantly (p = 0.0031) from 6.3% in Latvia to 27.4% in Poland. No correlation (r = 0.02; p = 0.9583) with GDP per capita was observed. The highest agreement in reimbursement decisions was observed between Estonia and Lithuania, and the lowest – between Estonia and Latvia, with kappa of 0.69 and 0.11, respectively. Significant impact of the type of approval and reimbursement status was observed for Czechia, Lithuania and Slovakia where conditional approval and exceptional circumstances negatively influenced reimbursement decision. Type of disease has significant influence on reimbursement decision in 4 out of 10 analyzed countries with significant outweigh of positive decisions for oncological diseases.ConclusionIn considered countries specific regulations on reimbursement of orphan drugs are valid but in Lithuania and Romania no formal HTA process was employed; in case of some countries higher ICER values for orphans are used. The share of reimbursed orphan drugs varied significantly across the countries, but it was not associated with GDP per capita

Comparative Analysis of Legislative Requirements About Patients' Access to Biotechnological Drugs for Rare Diseases in Central and Eastern European Countries

Objectives: The aim of the study was to compare the access of patients with rare diseases (RDs) to biotechnological drugs in several Central and Eastern European countries (CEECs). We focused on the legislative pricing and reimbursement requirements, availability of biotechnological orphan medicinal products (BOMPs) for RDs, and reimbursement expenditures. Methods: A questionnaire-based survey was conducted among experts from 10 CEECs: Bulgaria, Croatia, Estonia, Greece, Hungary, Poland, Romania, Slovakia, Serbia, and Macedonia. The legal requirements for reimbursement and pricing of BOMPs were collected. All BOMPs and medicines without prior orphan designations were extracted from the European list of orphan medicinal products, 2017. The reimbursement status of these medicinal products in 2017 in the public coverage of the included CEECs as well as the share of their costs in relation to the total public pharmaceutical spending for the period from 2014 to 2016 were defined. Results: Our survey revealed that some differences in the legal requirements for pricing and reimbursement of BOMPs amongst the countries included in the study. All European Union countries have developed and implemented pharmacoeconomic guidelines with or without some specific reimbursement requirements for orphan medicinal products. Cost-effectiveness analysis, cost-utility analysis, Markov models, meta-analysis, and discount levels of costs and results were required only in Bulgaria, Poland and Hungary. The number of reimbursed BOMPs and biotechnological medicinal products for RDs without prior orphan designation was the highest in Hungary (17 and 40, respectively). Patient-based reimbursement schemes were available only in Hungary for 11 out of 17 BOMPs. Poland and Greece have the highest pharmaceutical expenditure of reimbursed BOMPs with are similar to 214 million and 180 million EUR, respectively in the observed period from 2014 to 2016. High proportion of the pharmaceutical expenditure on the reimbursed biotechnological medicinal products for RDs for the observed period 2014-2016 is presented in Bulgaria and Slovakia. Conclusions: The non-European Union CEECs face a significant delay in the legal implementation of pharmacoeconomic guideline for assessment of BOMPs. The access to BOMPs is similar among the observed CEECs and the countries with the best access are Hungary and Greece. The influence of BOMP expenditures on the budget in the individual countries is significant

Pricing and Reimbursement of Biosimilars in Central and Eastern European Countries

Objectives: The aim of this study was to review the requirements for the reimbursement of biosimilars and to compare the reimbursement status, market share, and reimbursement costs of biosimilars in selected Central and Eastern European (CEE) countries.Methods: A questionnaire-based survey was conducted between November 2016 and January 2017 among experts from the following CEE countries: Bulgaria, Czech Republic, Croatia, Estonia, Hungary, Latvia, Lithuania, Poland, Slovakia, and Romania. The requirements for the pricing and reimbursement of biosimilars were reviewed for each country. Data on the extent of reimbursement of biologic drugs (separately for original products and biosimilars) in the years 2014 and 2015 were also collected for each country, along with data on the total pharmaceutical and total public health care budgets.Results: Our survey revealed that no specific criteria were applied for the pricing and reimbursement of biosimilars in the selected CEE countries; the price of biosimilars was usually reduced compared with original drugs and specific price discounts were common. Substitution and interchangeability were generally allowed, although in most countries they were at the discretion of the physician after a clinical assessment. Original biologic drugs and the corresponding biosimilars were usually in the same homogeneous group, and internal reference pricing was usually employed. The reimbursement rate of biosimilars in the majority of the countries was the same and amounted to 100%. Generally, the higher shares of expenditures were shown for the reimbursement of original drugs than for biosimilars, except for filgrastim, somatropin, and epoetin (alfa and zeta). The shares of expenditures on the reimbursement of biosimilar products ranged from 8.0% in Estonia in 2014 to 32.4% in Lithuania in 2015, and generally increased in 2015. The share of expenditures on reimbursement of biosimilars in the total pharmaceutical budget differed between the countries, with the highest observed value for Slovakia and Hungary and the lowest—for Croatia.Conclusions: The requirements for the pricing and reimbursement of biosimilar products as well as the access of patients to biologic treatment do not differ significantly between the considered CEE countries. Biosimilar drugs significantly influence the reimbursement systems of these countries, and the expenditure on the reimbursement of biosimilars is increasing as they are becoming more accessible to patients