Leber hereditary optic neuropathy in Slovenia: quality of life and costs from patient perspective.

INTRODUCTION: Leber hereditary optic neuropathy (LHON) is the most commonly diagnosed mitochondrial disorder, resulting in colour vision abnormalities and rapid but painless deterioration of central vision. While numerous studies have assessed the impact of LHON on the quality of life (QoL) of LHON patients, the financial impact of the disease remains unexplored. This study attempts to calculate both the direct non-medical costs and the indirect costs associated with productivity losses experienced by people with LHON and their unpaid caregivers in Slovenia, in addition to assessing their QoL. Due to the rarity of the disease, the study involved a small sample size, which is important to note for interpreting the results. METHODS: The analysis was conducted on nine adult participants diagnosed with LHON, representing one-third of the total number of known Slovenian patients with this condition. To thoroughly assess the economic and social impact of LHON, tailored questionnaires were designed to collect information on demographics, socioeconomic status, LHON severity, and associated non-medical and indirect costs. RESULTS: The mean age of the study participants was 48.8 years (SD 13.3; n = 9). The annual productivity loss attributable to LHON, taking both absenteeism and relative presenteeism into account, was calculated to be EUR 11,608 per person affected. The mean VFQ-25 score, a measure of vision-related quality of life, for adult LHON patients was 30.4 (SD 12.9). CONCLUSION: The findings highlight the significant economic and social burden of LHON on patients and their families. Ensuring prompt, accurate diagnosis, access to treatment, financial support, and psychological counselling and services are critical to helping individuals cope with and mitigate the profound challenges of vision loss and living with LHON.

Leber hereditary optic neuropathy in Czechia and Slovakia: Quality of life and costs from patient perspective.

Introduction: Leber hereditary optic neuropathy (LHON) is the most frequent mitochondrial disease causing dyschromatopsia and progressive central visual loss that is subacute in progression and painless. Several studies have been published assessing QoL in patients with LHON, but no estimate of the economic burden has been reported to date. This study aims to quantify direct non-medical and indirect costs (productivity loss) incurred by LHON patients and their informal caregivers in Czechia and Slovakia, as well as to assess their quality of life. Methods: The study was performed in 27 adults and children with LHON. To determine the socioeconomic burden of LHON, separate questionnaires for adults, children, and their parents were developed, including demographic and socioeconomic data. The following data were collected: age, education, family size, severity of LHON, non-medical direct and indirect costs of LHON. Results: The mean age of adult respondents was 36.1 years (SD 13.1; n = 21). The total cost of absenteeism was EUR 1003 per person/year in adult employees, and EUR 2711 per person/year in children's parents. The productivity loss as a consequence of LHON due to combined relative absenteeism and relative presenteeism was estimated at EUR 9840 per an adult patient/year, and EUR 6298 per a parent/year, respectively. The mean cost of informal care was estimated at EUR 4502 (SD 4772; n = 6) per person/year. The mean VFQ-25 score for adult patients with LHON was 43.47 (SD 15.86). Conclusion: The results of this study clearly show that patients with LHON and their families face an extensive socioeconomic burden related to this rare disease. Early, timely and appropriate access to diagnosis, treatment, and reimbursement decisions, but also to psychological counselling and services may help the patients and their relatives adapt and cope with the challenging aspects of vision loss and life with the disease.

The Checklist for Standard Methodological Requirements and Reporting of Economic Evaluation of Medicines in Slovakia.

OBJECTIVES: We have developed a scientifically well-grounded, methodological, and reporting checklist for economic evaluation (EE) of medicines in the Slovak health technology assessment process, which serves as a supplement to the Slovak pharmacoeconomic guidelines. METHODS: The checklist was developed using an iterative process in which items were generated and gradually added to the baseline checklist based on shortcomings identified in an analysis of Slovak EEs, using relevant published checklists, and Slovak, as well as international, methodological guidance that was identified in the systematic literature review. The selection of checklist recommendations, their clarity, and relevance to the Slovak setting were validated in the online survey. RESULTS: From the sample of 151 price and reimbursement submissions published between January 2018 and July 2021, almost half of them (n = 73) received at least 1 request from the Ministry of Healthcare to justify or modify the methodology used in the EE; and in 18 proceedings, a negative opinion was issued because of shortcomings identified in the EE. The 25-items preliminary checklist, resulting from an iterative working process, has been validated in an online survey conducted among members of ISPOR Chapter Slovakia. After incorporating relevant comments, the final proposal for the Slovak checklist consists of 55 recommendations. CONCLUSIONS: The research represented the first attempt to create a Slovak EE checklist, which serves as a part of ISPOR Slovakia pharmacoeconomic guidelines. Implementation of the checklist allows checking whether EE meets legislative and methodological requirements and thus helps in improving the appropriateness and standardization of EEs in Slovakia.

Drug Policy in Slovakia.

Slovak law sets clear rules and timelines in the process of approving the price and reimbursement of drugs. During the last decade, the Ministry of Health adopted several cost-containment measures in the price and reimbursement policy. The most effective measures were the implementation of the external referencing of drug prices in 2008 and the reimbursement law in 2011. The new act introduced several regulations such as making stricter rules for the referencing of prices, setting cost per quality-adjusted life-year threshold, and defining new rules for the setting of reimbursements. On one side, implementation of these measures helped to achieve visible cost savings, but, on the other side, cost-containment policies have had some unintended consequences. In recent years, Slovakia has been facing a decreased availability of drugs because of parallel exports. As a result of the government's effort, Slovakia is the only country in the European Union that implemented a legal ban on the re-export of medicines. During the decade before 2011, many innovative drugs were included in the reimbursement system. Because of stricter legal conditions introduced in 2011, there has been a gradual shift in reimbursing innovative drugs from the standard reimbursement system to reimbursement by way of exceptions of health insurance companies. Recently, there has been an ongoing discussion on possible changes to the reimbursement law.