Rare Neurological Diseases: an Overreview of Pathophysiology, Epidemiology, Clinical Features and Pharmacoeconomic Considerations in the Treating

Rare diseases (RD) are serious chronic diseases affecting small number of people compared to the general population. There are between 6000 and 8000 RDs, which affect about 400 million people worldwide. Drugs used for causal treatment of RDs are called orphan drugs. RDs bear great clinical and economic burden for patients, their families, healthcare systems and society overall. There are at least two reasons for the high cost of treatment of RDs. First, there is no causal therapy for majority of RDs, so exacerbations, complications, and hospitalizations in those patients are common. The second reason is high price of available orphan drugs, which are not cost-effective when traditional pharmacoeconomic evaluation is employed. The pharmacoeconomic aspect of the treatment of RDs is especially important in the field of neurology, since at least one fifth of all RDs is composed of neurological conditions. The aim of this paper was to provide a concise overview of the pathophysiological, epidemiological and clinical characteristics of some of the most important and common rare neurological diseases, with special reference to their impact on society and economy.

Pharmacoeconomic evaluation of the effectiveness of therapy for metastatic colorectal cancer

Objective: to perform a pharmacoeconomic evaluation of the effectiveness of therapies for metastatic colorectal cancer (mCRC) based on real practical data on medical help for patients with this disease.Material and methods. The authors performed a comparative cost-effectiveness analysis and the calculation of resource consumption in the application of several options of chemotherapy for mCRC: FOLFOX, FOLFIRI, and FOLFOXIRI; targeted therapy: FOLFOX + bevacizumab, FOLFOX + panitumumab, FOLFIRI + cetuximab, FOLFIRI + aflibercept, regorafenib monotherapy.Results. The cost-effectiveness parameter, calculated as a ratio of the cost of therapy to the median survival without progression, for chemotherapeutic schemes varied from 108 to 167 thousand rubles and for the targeted therapy schemes – from 223 to 930 thousand rubles. The calculation of resource consumption showed that in the case of a limited budget, 100% of patients can be treated by FOLFOX scheme, or 26% of patients by FOLFOX + panitumumab, or 47% of patients by FOLFOX + bevacizumab; and 100% of patients by FOLFIRI scheme or 11.5% of patients by FOLFIRI + cetuximab (aflibercept). Besides, it was established that in the case of a similar budget, 100% of patients with mCRC can be treated by chemotherapy schemes or a limited number of patients with regorafenib.Conclusion. The cost of targeted therapy significantly exceeds the cost of chemotherapeutic schemes. Still, considering the gross domestic product per capita in the Russian Federation, they can be an economically feasible investment and the optimum option of therapy

Systematic Review and Quality Evaluation of Pharmacoeconomic Studies on Traditional Chinese Medicines

Objectives: This study was aimed to find and appraise the available published pharmacoeconomic research on Traditional Chinese Medicine (TCM), to identify related issues and make suggestions for improvement in future research.Methods: After developing a search strategy and establishing inclusion and exclusion criteria, pharmacoeconomic studies on TCM were sourced from seven Chinese and English databases from inception to April 2020. Basic information about the studies and key pharmacoeconomic items of each study were extracted. The quality of each study was evaluated by using the British Medical Journal economic submissions checklist for authors and peer reviewers, focusing on factors such as study design, research time horizon, sample size, perspective, and evaluation methods.Results: A total of 431 published pharmacoeconomic articles with 434 studies on topics including cost-effectiveness, cost-benefit, cost-minimization, cost-utility, or combination analyses were identified and included in this review. Of these, 424 were published in Chinese and 7 in English. These studies conducted economic evaluations of 264 Chinese patent medicines and 70 types of TCM prescriptions for 143 diseases, including those of the central nervous, cardiovascular, respiratory, gynecologyical, and other systems. The studied TCMs included blood-activating agents (such as Xuesaitong tablet, Fufant Danshen tablet, and Danhong Injection), blood circulation promoting agents (such as Shuxuetong injection, Rupixiao tablet, and Fufang Danshen injection), and other therapeutic agents. The overall quality score of the studies was 0.62 (range 0.38 to 0.85). The mean quality score of studies in English was 0.72, which was higher than that of studies in Chinese with 0.62.Conclusions: The quality of pharmacoeconomic studies on TCM was relatively, generally low. Major concerns included study design, inappropriate pharmacoeconomic evaluation, insufficient sample size, or non-scientific assessment. Enhanced methodological training and cooperation, the development of a targeted pharmacoeconomic evaluation guideline, and proposal of a reasonable health outcome index are warranted to improve quality of future studies.

Cost minimization analysis of two chemotherapy regimens in the treatment of colorectal cancer in a public reimbursement hospital in Brazil

Objective: To conduct a pharmacoeconomic evaluation between XELOX and mFOLFOX6 in the adjuvant and metastatic treatment of colorectal cancer from the perspective of a public reimbursement hospital. Methods: The cost minimization analysis was conducted for patients who started treatment in 2013 and 2014. The micro-costing technique was used to verify expenditures on drugs, materials, laboratory and imaging tests, ambulatory and daily hospitalization, human and administrative resources and determine the individual cost of each alternative, per patient. To evaluate the robustness of the economic analysis, multivariate sensitivity analysis was performed in six different scenarios. Results: There was an average cost for XELOX of U$ 4,637.14 in adjuvant and U$ 3,831.48 for palliative treatment, and a cost for mFOLFOX6 of U$ 5,474.89 in adjuvant and U$ 4,432.95 in palliative treatment. Sensitivity analysis maintained the dominance of XELOX. Material and drug costs accounted for about 85% of the total cost of XELOX; for mFOLFOX6 this cost was around 36%. On the other hand, the cost of hospitalization and placement of a catheter occured exclusively for mFOLFOX6, which also presented a higher cost with human resources. Conclusion: From the perspective of the hospital, XELOX proved to be the least costly alternative on the treatment of colorectal cancer.

Pricing and Reimbursement Pathways of New Orphan Drugs in South Korea: A Longitudinal Comparison

This study aimed to analyze four current pathways affecting the listing and post-listing prices of new orphan drugs (ODs) in South Korea. These mechanisms were: (1) essential OD, (2) pharmacoeconomic evaluation (PE) waiver OD, (3) weighted average price OD, and (4) PE OD. We analyzed the ratio of the listing price of 48 new ODs to the average adjusted price (AAP) of seven advanced countries and examined the change in the post-listing price. Descriptive statistics were used to analyze the listing and post-listing price changes. The mean and median ratios of the listing price of total new OD to AAP were calculated to be 69.4% and 65.4%, respectively. Essential OD showed the highest mean (93.8%) and median (80.8%) ratios. The mean cumulative price discount rate of the new OD was 7.2% in the third year and 5.7% in the fifth year. The rarity of diseases impacts the listing price of OD, but the political effects of the benefits of OD on the post-listing price of these drugs could not be verified. Further research should be conducted to develop measures that facilitate the practical sharing of budget risks and increase patient access to new ODs.