Epidemiology of rare disease mortality in Argentina (1997–2017)

BACKGROUND AND AIMSSaudi Arabia has no precise data on causes of death. We sought to ascertain the commonest causes of death as stated in death certificates of adults and evaluate the completeness of death certificates at a teaching hospital in Riyadh.DESIGN AND SETTINGA cross-sectional study carried out at King Khalid University Hospital in Riyadh, Saudi Arabia, during the year 2008.METHODSAll death certificates that were issued in 2008 were reviewed and data were checked by two reviewers. Causes of death were coded according to specially-designed codes.RESULTSThe mean (SD) age of death was 63.9 (20.7) years. More than 80% arrived alive at the hospital. Among the 410 certificates, 62.2% had the first reported cause of death being classified as “inappropriate” and this tended to be slightly, but significantly more frequent among women. The first most common appropriately reported cause of death was malignancy of any type (7.3%) followed by ischemic heart diseases (4.9%). Accidents and fractures were more common in the younger age groups and among men.CONCLUSIONSThis is the first study that documents the possible gaps among healthcare professionals in Saudi Arabia in their understanding of death and its certification based on the clinical assessment of the deceased. The findings needs to be validated by similar studies from other health care sectors. It is clear, however, that proven educational, system-related and legal interventions to improve the accuracy of death certification are strongly needed if the health care priorities are to be properly identified.

February 28, 2013 marks the sixth international “Rare Disease Day”. On and around this day, hundreds of patient organizations from more than 60 countries and regions worldwide plan to host awareness campaigns in line with this year's theme, “Rare Disorders without Borders”. Public awareness of intractable and rare diseases has heightened in recent decades. Much progress has also been made worldwide, such as specific legislation to encourage discovery and development of orphan drugs in the United States (US), the European Union (EU), and some parts of Asia. However, there are still many gaps in knowledge with regard to therapeutic tools and strategies. Intractable and rare diseases cause patients substantial physical suffering, psychological despair, and economic hardships due to bleak therapeutic outcomes and the lack of practical support in everyday life. The features of intractable and rare diseases and the increasing number of types of identified diseases make these diseases an important public health issue and a challenge to medical care worldwide. The following are specific aspects of research on intractable and rare diseases that need to be promptly promoted. An International Classification of Diseases (ICD) code to promote the definition and classification of intractable and rare diseases Intractable diseases, or “nanbyo” (literally “hard-to-treat diseases” in Japanese), mainly refer to rare diseases that have resulted mostly from unidentifiable causes and/or a lack of clearly established or curative treatments. According to the World Health Organization (WHO), rare diseases are rare and often debilitating or even life-threatening diseases or conditions with a prevalence of 0.65–1‰. The conventional view is that rare diseases as a whole affect around 10% of individuals worldwide, but the definition and categorization of rare diseases differ slightly by region. In the US, rare diseases are defined as diseases that affect fewer than 200,000 Americans (prevalence of < 0.75‰), while stipulated prevalence rates in other regions are < 0.5‰ in the EU, fewer than 2,000 patients (prevalence of < 0.11‰) in Australia, fewer than 50,000 patients (prevalence of < 0.4‰) in Japan, fewer than 20,000 patients (prevalence of < 0.4‰) in South Korea, or a prevalence of < 0.1‰ in Taiwan. The current outlook for identification of a specific rare disease and estimation of the true burden of rare diseases is bleak given the lack of proper classification and coding of rare diseases. Currently, there is no special coding system for rare diseases. The current ICD code that is used in most countries is not suitable for rare diseases. The absence of a universally recognized coding system is an obstacle for reliable registration of patients in national or international databases, preventing assessment of the economic and social effects of rare diseases. Fortunately, the good news is that the European Rare Disease Task Force of the Health and Consumers Protection Directorate General of the European Commission has set up a working group to collaborate with the WHO on the ICD-10, and the group is considering all other existing classifications to provide the rare disease community with a uniform system. A revised ICD code is urgently needed to both promote the definition and classification of intractable and rare diseases and to obtain accurate epidemiological data on these diseases at the national and international levels. Specific legislation to encourage discovery and development of orphan drugs Currently, orphan drugs — the medicinal products intended for the diagnosis, prevention, or treatment of rare diseases — are a major facet of how rare diseases are dealt with. In the past few decades, many countries have realized that orphan drugs will not lead to substantial sales under normal market conditions because of the high costs and risks of drug development, insufficient knowledge of the pathophysiological mechanisms of rare diseases that the drugs diagnose or treat, and difficulties in conducting clinical trials with small patient populations and a small potential market. Therefore, specific legislation to encourage the discovery and development of orphan drugs was enacted in many countries and regions, including the US in 1983, Japan in 1993, Australia in 1997, the EU in 1999, Taiwan in 2000, and South Korea in 2003. Incentives include financial subsidies, market exclusivity, tax credits, fee waivers, fast track approval, and protocol assistance, resulting in substantial improvements in the treatment of patients with a range of rare diseases. While China is actively preparing to regulate and encourage the development of orphan drugs, it still lags far behind the US, the EU, Japan, and other countries and regions with orphan drug legislation. Evidence has shown that all of the incentives have successfully encouraged the development of new pharmaceutical products to treat rare diseases. Prior to 2010, 352 orphan drugs were approved in the US, helping an estimated 12 million Americans, compared to only 10 such drugs in the decade preceding the Orphan Drug Act (1983). Similarly, 720 drugs had received orphan drug designation from the European Medicines Agency (EMA) and 63 designated orphan medicinal products have been authorized for marketing in the EU. Furthermore, data have shown that an average of 15 new orphan drugs are approved annually in the US and 10-12 new orphan drugs are approved annually in the EU. Thus, China and other countries without orphan drug legislation need to promptly establish domestic legislative regulations and incentives to encourage discovery and development of orphan drugs. Government-funded special biomedical research programs to enhance basic and applied research on intractable and rare diseases Biomedical research on intractable and rare diseases has provided insights into the pathologies of these diseases and revealed their underlying mechanisms. Such work may ultimately reveal possible avenues to therapeutics. Moreover, once biomedical research identifies suitable drug candidates and becomes more translational, it will garner industry attention, potentially leading to safe and effective orphan drugs. In Western countries, many research centers or projects have been established to support special biomedical research programs on rare diseases and development of orphan drugs, such as the Office of Rare Diseases Research (ORDR) established in the US in 1993 within the National Institutes of Health (NIH) and the Rare Disease Task Force (RDTF) established in EU in 2004 within the European Commission Public Health Directorate. In Asian countries, biomedical research on intractable and rare diseases has made great advances in Japan due to the systematic Specified Disease Treatment Research Program established in 1972 with the support of the Ministry of Health, Labor, and Welfare. As a result, special research programs and research grants from government sources to study 130 diseases increased to 10 billion Japanese yen in 2010. Recently, 214 diseases were designated for a second round of special research programs. In China, support for special biomedical programs on intractable and rare disease research comes mainly from the National Natural Science Foundation of China (NSFC). Data showed that 366 projects (involving 32 rare diseases) were funded by the NSFC from 1999 to 2007 with total funding of 89.358 million RMB and annual funding of about 10 million RMB, accounting for just 1/10th of similar funding in the US. Special biomedical research programs that enhance basic and applied research on intractable and rare diseases would benefit patients through better diagnosis and more treatment choices. Government-funded special biomedical research programs need to be promptly implemented in China and other countries to promote research on intractable and rare diseases. Patients' advocacy organizations and disease registry networks to provide vast information on intractable and rare diseases In recent years, progress has been made in the dissemination of knowledge and information by established patients' advocacy organizations, such as the National Organization for Rare Disorders (NORD) in the US and the European Organization for Rare Diseases (EURORDIS) in Europe, but the delay in diagnosis and treatment is still a huge challenge to cope with. A survey of 18,000 individuals found that 25% of patients waited for 5-30 years before being correctly diagnosed and 40% of patients were diagnosed incorrectly before they were correctly diagnosed. Furthermore, clinical studies on orphan drugs also face challenges due to the small size of the trial population and the fact that patients are often geographically dispersed. Disease registry networks need to be established to promote epidemiological and basic research and improve the clinical outcome for patients with intractable and rare diseases. In Western countries, some web-based resources, such as the Rare Diseases Clinical Research Network (RDCRN) in the US and the Orphanet in Europe, have been established in order to facilitate collaboration on clinical outcomes and to share accumulated experience so that patients with intractable and rare diseases are not delayed access to orphan drugs. More patients' advocacy organizations and disease registry networks need to be promptly established to facilitate interaction among patients, clinicians, researchers, the pharmaceutical industry, and governmental bodies with the ultimate goal of promoting intractable and rare disease research worldwide. (February 28, 2013)

To validate verbal autopsy (VA) procedures for use in sample vital registration. Verbal autopsy is an important method for deriving cause-specific mortality estimates where disease burdens are greatest and routine cause-specific mortality data do not exist. Verbal autopsies and medical records (MR) were collected for 3123 deaths in the perinatal/neonatal period, post-neonatal <5 age group, and for ages of 5 years and over in Tanzania. Causes of death were assigned by physician panels using the International Classification of Disease, revision 10. Validity was measured by: cause-specific mortality fractions (CSMF); sensitivity; specificity and positive predictive value. Medical record diagnoses were scored for degree of uncertainty, and sensitivity and specificity adjusted. Criteria for evaluating VA performance in generating true proportional mortality were applied. Verbal autopsy produced accurate CSMFs for nine causes in different age groups: birth asphyxia; intrauterine complications; pneumonia; HIV/AIDS; malaria (adults); tuberculosis; cerebrovascular diseases; injuries and direct maternal causes. Results for 20 other causes approached the threshold for good performance. Verbal autopsy reliably estimated CSMFs for diseases of public health importance in all age groups. Further validation is needed to assess reasons for lack of positive results for some conditions.

Congenital malformations (CM) represent the second cause of infant death in Argentina. To analyze the secular trend (1980-2018) of infant deaths due to CM at the regional and provincial level. The data come from the DEIS (Ministry of Health) and include the absolute number of deaths and live births and deaths by CM coded according to ICD-10 (Codes Q00-Q99). Infant mortality rate due to CM (IMR-CM) (number of deaths due to congenital malformations / number of newborns) and the proportion of deaths from CM (PD-CM) (percentage of deaths from congenital malformations / deaths from all causes) were calculated by regions (Center, NOA, NEA, Cuyo and Patagonia) and provinces. The period was divided into 7 subgroups of five years and one of 4. Secular trend was analyzed using a Poisson model. A line and bar graph were used to represent graphically the differences in the IRM-CM and PD-CM at the regional level. At the country level, there was a pattern characterized by the significant decrease and increase of the IMR-CM and PD-CM respectively. This pattern is repeated in the Central, Cuyo and Patagonia regions and in the provinces of Buenos Aires, Santa Fé, Entre Ríos, Neuquén, La Pampa, Mendoza and Santa Cruz. In the remaining regions and provinces, the IMR-CM exhibits a heterogeneous behavior. To analyze the secular trend (1980-2018) of infant deaths due to CM at the regional and provincial level.

Rare genetic disease in China: a call to improve clinical services.

European solidarity is changing the face of rare diseases

AimsTo ascertain the number of paediatric deaths (0–14 years) with an underlying rare disease in the Republic of Ireland between the years 2006–2016, and to analyse bed usage by a paediatric cohort of rare disease inpatients prior to in-hospital death.BackgroundRare diseases are often chronically debilitating and sometimes life-threatening diseases, with the majority (69.9%) of rare diseases being of paediatric onset. The Orphanet database contains information on 6172 unique rare diseases. Under-representation of rare diseases in hospital healthcare coding systems leads to a paucity of rare disease epidemiological data required for healthcare planning. Studies have cited variable incidence rates for rare disease, however the burden of rare diseases to healthcare services still remains unclear. This study represents a thorough effort to identify the percentage of child mortality and paediatric bed usage attributable to rare diseases in the Republic of Ireland, thus addressing a major gap in the rare disease field.MethodsRetrospective analysis of paediatric death registration details for the Republic of Ireland in the 11-year period 2006–2016 from the National Paediatric Mortality Register. Data was subcategorised as Neonatal (0–28 days), Post Neonatal (29 days < 1 year) and older (1–14 years). Bed usage data (ICD-10 code, narrative and usage) of paediatric inpatients who died during hospitalisation from January 2015 to December 2016 was extracted from the National Quality Assurance Improvement System of in-patient data. Orphacodes were assigned to rare disease cases from ICD-10 codes or diagnostic narrative of both datasets.ResultsThere were 4044 deaths registered from 2006–2016, aged < 15 years, of these 2368 (58.6%) had an underlying rare disease. Stratifying by age group; 55.6% (1140/2050) of neonatal deaths had a rare disease, 57.8% (450/778) post-neonatal, and 64% (778/1216) of children aged 1–14 years. Mortality coding using ICD-10 codes identified 42% of rare disease cases with the remainder identified using death certificate narrative records. Rare disease patients occupied 87% of bed days used by children < 15 years who died during hospitalisation from January 2015 to December 2016.ConclusionAdditional routine rare disease coding is necessary to identify rare diseases within Irish healthcare systems to enable better healthcare planning. Rare disease patients are overrepresented in paediatric mortality statistics and in-patient length of stay during hospital admission prior to death.

To characterise the epidemiological patterns and the spatial-temporal distribution of schistosomiasis-related mortality in Brazil from 2003 to 2018. A national population-based ecological study that used official data from the Mortality Information System. The data included all deaths recorded in Brazil from 2003 to 2018 in which schistosomiasis was mentioned in the death certificate as an underlying or associated cause of death (multiple causes). The municipalities of residence were used as units of geographic analysis, and standardised and smoothed mortality rates (per 100000 inhabitants) were calculated using the local empirical Bayes method. Spatial autocorrelation was evaluated using global and local Moran indexes. To analyse the spatial dependence, the Getis-Ord G and Gi* statistics were used. During the study period, 18421113 deaths were recorded in Brazil. Schistosomiasis was mentioned in 11487 deaths (proportional mortality: 0.06%); for 8141 deaths (70.87%), it was listed as the underlying cause, and for 3346 deaths (29.13%), it was listed as an associated cause. The mean mortality rate was 0.38 deaths/100000 inhabitants. Individuals≥70years of age (RR: 115.34, 95% CI: 68.56-194.03) and residents in the Northeast region (RR: 10.81, 95% CI: 5.95-19.66) presented higher risks related to schistosomiasis. Municipalities with high mortality rates were identified in all regions, and high-risk clusters were found in municipalities located in the Northeast and Southeast regions of the country. Schistosomiasis remains an important cause of death in persistently endemic areas in Brazil, particularly in those with a high prevalence of the disease and a marked parasite load.

Although randomized control trials (RCTs) are the ‘gold standard’ to evaluate treatment effects in health care, they are frequently not practical, ethical or politically acceptable in the evaluation of many health system or public health interventions. A good examplewhere a health system intervention has undergone evaluation using an RCT design is the universal health insurance scheme, Seguro Popular, in Mexico. However, this is a rare exception mainly due to the academic background of the Mexican Health Minister, Julio Frenk, who introduced the scheme. More frequently, randomization is not feasible or practical, particularly when interventions target whole or large subgroups of populations. Because of political considerations, policy makers often want to implement changes quickly and refuse to wait several years to determine a new intervention’s effects. Further, they may be reluctant to be seen to withhold an intervention from a particular community, as was the case with the SureStart programme, which aimed to improve health and educational outcomes in young children in the UK. RCTs may also be unethical where clear evidence of benefit has been demonstrated from observational studies, as was the case with cervical cancer screening. Additionally, lack of funding often poses a hurdle to formal evaluation through an RCT, as RCTs can be very costly to carry out. In the absence of an RCT, evaluations often use quasi-experimental designs such as a pre-post study design with measurements before and after the intervention period. Figure 1, panel A shows an introductory example with an outcome measure subject to a secular time trend and an intervention without any impact. The standard approach to detect a significant impact would apply a t-test to compare the means of the preintervention phase with the post-intervention data. However, a t-test does not consider time but simply separates the data into two groups. In the example in panel A of Figure 1, the t-test would obtain a significant p-value although the difference is not due to the intervention but rather captures the secular time trend. In settings with a secular trend, a t-test (or another statistical test) is prone to false positives, as illustrated in the example, and false negatives in case of a negative secular trend. An interrupted time series (ITS) in contrast, adjusts for secular time trends and should be used instead. Panel B of Figure 1 contains a new data set with an intervention affecting the outcome. An ITS is a segmental linear regression model; preintervention and post-intervention are each modeled as a linear regression. Based on an ITS, a secular trend can therefore be captured in the regression line as shown in panel B of Figure 1. An ITS compares the intercept and slope of the regression line before the intervention with the intercept and slope after intervention. A one-time baseline effect of the intervention without influencing the secular trend can be detected as an intercept change. If the intervention changed the secular trend, there will also be a significant difference in the slope between the two periods. Later, we will see that an ITS also comprises more flexible models. Instead of using an ITS design, many studies use preand post-intervention groups without modeling the secular time trend. Examples include evaluations of major interventions DECLARATIONS

Implementing Real-Time Data Suicide Surveillance Systems.

Objective: To analyze the diagnosis and treatment of children with rare diseases in the pediatric intensive care unit (PICU), the distribution of disease types and populations, clinical characteristics, and the use of orphan drugs. Methods: A retrospective case summary was conducted. Data were collected from 105 children aged 29 days to <18 years with a confirmed diagnosis of rare diseases according to the "First Batch of Rare Disease Catalogue in China" who were admitted to the PICU of Beijing Children's Hospital, Capital Medical University from January 2020 to December 2022. Data including general information, auxiliary examinations, and treatment details for each patient were collected from the hospital's electronic medical record system. Patients were divided into age groups: infancy (29 days to<1 year), early childhood (1 to <3 years), preschool age (3 to<7 years), school age (7 to<13 years), and adolescence (13 to<18 years). The chi-square test was used to compare gender distribution differences among various rare diseases. Results: A total of 105 patients with 130 cases meeting the diagnostic criteria were included, accounting for 4.7% (130/2 754) of the total admissions to the PICU. The age at PICU admission was 5.3 (0.8, 9.5) years and there were 81 cases in male. The 3 most common types of diseases were endocrine, nutritional, and metabolic diseases (37 cases); followed by neurological disorders(32 cases); and congenital malformations, deformities, and chromosomal abnormalities(17 cases). The 5 most common rare diseases were methylmalonic acidemia (14 cases), mitochondrial encephalomyopathy (14 cases), atypical hemolytic uremic syndrome (12 cases), autoimmune encephalitis (12 cases), and idiopathic cardiomyopathy (9 cases). The distributions of common rare diseases varied among different age groups. In infants, atypical hemolytic uremic syndrome was most common (6 children). There was no statistically significant difference regarding gender among children with mitochondrial encephalomyopathy (13.6% (11/81) vs. 6.1% (3/49), χ2=1.77, P=0.184). Respiratory failure (36 cases) was the primary reason for rare diseases children to be admitted to the PICU. A total of 95 cases underwent mechanical ventilation, 39 cases received multidisciplinary collaborative diagnosis and treatment, and only 6 children received orphan drug therapy during their stay in the PICU. Conclusions: Rare diseases are not uncommon in PICU. Endocrine, nutritional and metabolic disorders, neurological disorders, congenital malformations, deformities, and chromosomal abnormalities were common. Methylmalonic acidemia, mitochondrial encephalomyopathy, atypical hemolytic uremic syndrome and autoimmune encephalitis have higher cases. Many children with rare diseases in the PICU have complex conditions those are challenging to treat, requiring multidisciplinary collaboration. The utilization rate of orphan drugs among children with rare diseases in PICU needs to be improved.

BackgroundThere are many public health issues to resolve regarding rare diseases, including a lack of data from large-scale studies. The objective of this study was to explore fundamental data for a list of rare diseases in China, based on a hospitalization summary reports (HSRs) database. The Target Rare Diseases List (TRDL) 2017 was generated using an expert consensus method in which experts listed diseases according to research priorities. Using codes of the 10th revision of the International Statistical Classification of Diseases and Related Health Problems (ICD-10) and key search terms of rare diseases in English and Chinese, data were obtained from HSRs of 96 hospitals, covering a population of over 15 million in China from 2014 to 2015. We extracted and analyzed information on demographics, hospitalizations, and readmissions.ResultsA total 281 rare diseases were included in the TRDL 2017. Altogether, 106,746 hospitalizations for a rare disease were captured from 1 January 2014 to 31 December 2015, accounting for 0.69% of inpatients during the same period. The top 10 rare diseases with most cases on the TRDL 2017 were thalassemia, idiopathic pulmonary arterial hypertension, pulmonary Langerhans cell histiocytosis, moyamoya disease, motor neuron disease, idiopathic pulmonary fibrosis, systemic sclerosis, hepatolenticular degeneration, coarctation of the aorta, and transposition of the great arteries. Among the 24 cities in the database, the five cities with the most types of the rare disease were Beijing, Changsha, Guangzhou, Shanghai, and Chengdu, with 191, 162, 143, 141, and 133 types, respectively. The five cities with most cases of the 281 rare diseases were Beijing, Guangzhou, Shanghai, Nanning, and Chengdu. The age distribution of rare diseases was 52% for the age group 25–64 years, and 27% of cases in the age group of 0–14 years were among children. The 10 highest readmission rates ranged from 35 to 65%.ConclusionsThis study provided the TRDL 2017 and descriptive analysis of 281 rare diseases in a hospitalized population. Our study reveals important fundamental information that will be useful in national policy making and legislation; registry implementation; and diagnosis, treatment, and prevention of rare diseases in China.

Microcephaly in Brazil: confidence builds in Zika connection

to provide the epidemiological framework of those affected by rare diseases resident in the Campania Region (Southern Italy), using the data entered in the Campania Region Rare Disease Registry, acquiring information potentially useful for regional planning. observational retrospective cohort study on patients with rare diseases included in the Regione Campania Rare Disease Registry from 01.01.2022 to 31.12.2022. population included in the Rare Disease Registry and resident in the Campania Region as at 31.12.2022. using the data entered in the Regione Campania Rare Disease Registry, the cumulative incidence (I) of patients with rare diseases resident in Campania was calculated, stratified by age group and rare disease group with the respective 95% confidence intervals (IC95%). Standardised cumulative provincial incidences were also calculated. These are reported using a multiplication factor of 100,000. the incidence of patients with rare diseases in the Campania Region is 50.0 (IC95% 49.4-50.6) per 100,000 inhabitants in the year 2022. Furthermore, the rare disease groups with the highest incidence per 100,000 inhabitants are diseases of the central and peripheral nervous system (I: 8.32 per 100,000 inhabitants) and congenital malformations, chromosomopathies and genetic syndromes (I: 8.52 per 100,000 inhabitants). Moreover, the age groups in which the incidence is highest are in the paediatric age group. an epidemiological framework of the Campania Region on rare diseases such as this one for the year 2022 is fundamental for national and regional planning in order to improve the care and quality of life of people affected by rare diseases, who often feel neglected by society. Sharing this type of information also draws attention to the need for faster diagnosis and the specialisation of new centres.

Медико-соціальне значення ендокринних орфанних захворювань обумовлено труднощами в діагностиці та лікуванні, а також несприятливим прогнозом і високою частотою інвалідизації пацієнтів із цією патологією. Особливо це стосується таких захворювань, як акромегалія та множинна ендокринна неоплазія. В Європі існує спеціальна програма, яка направлена на вирішення різноманітних питань щодо діагностики, лікування та соціального забезпечення пацієнтів з орфанними захворюваннями. Європейський проект «Орфанет» підтримується державними структурами, об’єднує інформацію щодо рідкісних захворювань практично у всіх європейських країнах і включає європейські, інтернаціональні, національні та регіональні реєстри орфанних захворювань. В Україні опубліковано Наказ Міністерства охорони здоров’я (МОЗ) України від 27.10.2014 р. № 778 «Про затвердження переліку рідкісних (орфанних) захворювань», яким офіційно затверджено 302 нозології, що віднесені до рідкісних захворювань. Серед орфанних захворювань вказано 61 рідкісну ендокринну хворобу (зокрема, розлади харчування та порушення обміну речовин), а також природженівади розвитку, хромосомні аномалії та рідкісні новоутворення, у визначенні тактики ведення яких лікар-ендокринолог бере безпосередню участь. Останнім часом зростає частота виявлення орфанних захворювань, зокрема і ендокринних. Причиною цього може бути покращення методів інструментальної та лабораторної діагностики, використання генетичних методів діагностики та більш широка обізнаність лікарів різних спеціальностей щодо ранніх проявів орфанних захворювань. В Україні проблема орфанних захворювань тривалий час перебувала в інертному стані. Ініціація ухвалення Національної стратегії з профілактики, діагностики та лікування рідкісних (орфанних) захворювань в Україні свідчить про визнання проблеми на державному рівні та дає можливість вирішення питань своєчасного виявлення і лікування рідкісних захворювань, а також забезпечення пацієнтів життєво необхідними лікарськими засобами.