The European challenges of funding orphan medicinal products.

BACKGROUND: Funding of orphan medicinal products (OMPs) is an increasing challenge in the European Union (EU). OBJECTIVES: To identify the different methods for public funding of OMPs in order to map the availability for rare disease patients, as well as to compare the public expenditures on OMPs in 8 EU member states. METHODS: Information on the reimbursement status of 83 OMPs was collected in 8 countries by distinguishing standard and special reimbursements. In two consecutive years, the total public expenditures on OMPs were calculated by using annual EUR exchange rates. Annual total public expenditures were calculated per capita, and as a proportion of GDP, total public pharmaceutical and healthcare budgets. Differences between countries were compared by calculating the deviations from the average spending of countries. RESULTS: In 2015 29.4-92.8% of the 83 OMPs were available with any kind of public reimbursement in participant countries including special reimbursement on an individual basis. In Austria, Belgium and France more OMPs were accessible for patients with public reimbursement than in Bulgaria, Czech Republic, Hungary and Poland. Standard reimbursement through retail pharmacies and/or hospitals was applied from 0 to 41% of OMPs. The average annual total public expenditure ranged between 1.4-23.5 €/capita in 2013 and 2014. Higher income countries spent more OMPs in absolute terms. Participant countries spent 0.018-0.066% of their GDPs on funding OMPs. Average expenditures on OMPs were ranged between 2.25-6.51% of the public pharmaceutical budget, and 0.44-0.96% of public healthcare expenditures. CONCLUSIONS: Standard and special reimbursement techniques play different roles in participant countries. The number of accessible OMPs indicated an equity gap between Eastern and Western Europe. The spending on OMPs as a proportion of GDP, public pharmaceutical and healthcare expenditure was not higher in lower income countries, which indicates substantial differences in patient access to OMPs in favour of higher-income countries. Equity in access for patients with rare diseases is an important policy objective in each member state of the EU; however, equity in access should be harmonized at the European level.

HEALTH TECHNOLOGY ASSESSMENT IN EVALUATION OF PHARMACEUTICALS IN THE CZECH REPUBLIC.

OBJECTIVES: In the Czech Republic, the health technology assessment (HTA) approaches have been implemented in evaluation of medicinal products since 2008. The aim of this study was to provide an overview of the implementation of HTA and different levels thereof in the evaluation process conducted by the State Institute for Drug Control (SUKL) and to describe the impact of HTA on the entrance of new medicinal entities into out-patient healthcare system including highly innovative and orphan drugs. METHODS: Materials supporting this overview were collected using the records in the database of administrative proceedings of SUKL, in-house standard operating procedures, and the legislation in force. Based on these sources as well as the hands-on knowledge of the current practice, a brief description of the general rules of administrative proceedings involving HTA of varying complexity was elaborated. Characteristic features of the individual types of proceedings, basic differences in the complexity of HTA employed, and its most important challenges were summarized. RESULTS: In Czech Republic, HTA in the formal administrative proceedings ensures a transparent process of introduction of new medicinal products into clinical practice and leaves space for restriction of reimbursement conditions to minimize budget impact. CONCLUSIONS: As a robust as well as pragmatic HTA methodology has been implemented by SUKL, relevant stakeholders (marketing authorization holders, Health Care Funds, clinical expert groups) are now able to influence reimbursement of new technologies.

The impact of changes in national prescribing conditions for statins on their public expenditure and utilization in the Czech Republic 1997-2013.

OBJECTIVES: In the Czech Republic (CZ) extensive price regulation and prescribing conditions are common instruments often employed with new drugs. Since the introduction of statins onto the market in 1990s the originally strict conditions gradually relaxed while the prescription rates and public costs were rising. The aim was to analyze long-term utilization trends of statins, changes in their reimbursement prices and prescribing conditions, and the evolution of the market. METHODS: From January 1997 to December 2013 statin use was measured in terms of defined daily doses per 1000 insured per day (DDD/TID). The prescription-based database of the General Health Insurance Company of the Czech Republic in 1997 covering 7825,216 inhabitants, i.e. 76% of CZ population, was used as the administrative data source. Also the overall expenditure, unit prices, and reimbursement criteria were analyzed. RESULTS: Between 1997 and 2013 the utilization of statins rose from 2 to 96 DDD/TID while the expenditure rose 5.5-fold. The rise of prescription for each molecule was always observed after the liberation of the prescribing criteria. In the study period reimbursement prices of simvastatin and atorvastatin gradually decreased to just 5% of their initial values. CONCLUSIONS: The rising consumption of statins in CZ clearly corresponds in time with the liberation of prescribing conditions allowing for prescription by general practitioners and with the introduction of generics accompanied by a swift and repeated reimbursement price cuts.

Medical and Productivity Costs of Rheumatoid Arthritis in The Czech Republic: Cost-of-Illness Study Based on Disease Severity.

OBJECTIVE: International pharmacoeconomic studies suggest that functional impairment can be a significant predictor for the evaluation of direct and productivity costs for rheumatoid arthritis (RA). We calculated the direct and productivity costs for five Health Assessment Questionnaire (HAQ) groups of patients (HAQ scores <0.6, 0.6 ≥ 1.1, 1.1 ≥ 1.6, 1.6 ≥ 2.1, and ≥2.1) in the Czech Republic. METHODS: This was a retrospective cross-sectional study. We included 261 patients with RA, aged 18 to 84 years. We applied a bottom-up method by retrospectively reviewing individual patient medical records. Patients' demographic characteristics, patient-reported outcome, and clinical parameters were gathered at the time of data collection. For the calculation of productivity costs, we used the friction cost approach, based on patient absenteeism with a friction period of 130 workdays, with average monthly income used as the denominator. Costs were expressed as a mean value per patient with RA in each HAQ group. RESULTS: Mean patient age was 56.4 years. average time from diagnosis was 14.5 years, the mean HAQ score was 1.15, and the Disease Activity Score in 28 joints was 3.45. A total of 47.5% patients were treated with biologics. Mean annual direct medical costs for each HAQ group were €5315, €7357, €7697, €7716, and €8968, respectively. The mean annual indirect costs associated with productivity loss were €1414, €1459, €1610, €1876, and €2307, respectively. CONCLUSIONS: Direct costs and productivity costs for patients with RA are closely related to the value of the HAQ score. The annual mean total (direct plus productivity) costs per patient 1) treated with biologics, 2) without biologic treatment, and 3) from the overall cohort were €14,763, €3,559, and €8,882, respectively.

The economic burden of the ankylosing spondylitis in the Czech Republic: comparison between 2005 and 2008.

To investigate the burden of ankylosing spondylitis in the Czech Republic as a baseline for future health economic evaluations. Data were obtained from two cross-sectional studies Beda I (2005) and Beda II (2008), performed in 1,008 and 509 patients, respectively. Methodology used was Cost-of-Illness prevalence-based analysis bottom-up approach. Analysis was performed from payer (health insurance companies) and societal perspective (including productivity costs using friction cost approach). Mean age of sample in Beda I and Beda II was 50.2 and 52.5 years, male were present by 61.0 and 62.7 %; average disease duration was 23.0 and 26.4 years, respectively. Mean total annual costs per patient in the sample were €4,782 in Beda I and €5806 in Beda II. Average direct costs per patient in the sample per year are estimated at €1,812 (Beda I) and €2,588 (Beda II) with the average productivity costs €2,970 (Beda I) and €3,218 (Beda II). We observed a small decrement in percentage (6.7 %) of productivity costs for Beda II as an influence of higher consumption of biologic drugs, hence higher direct costs and possible productivity preservation. The largest direct cost burdens were spa procedures (45.3 %, Beda I) and biological drugs (52.8 %, Beda II). Unique analysis of the burden of the AS in the Central-Eastern Europe presents health care resource and cost consumption by comparing two cross-sectional prevalence-based studies. Further analysis should be carried to obtain data connecting health status with costs consumption in order to analyse the AS from this perspective.