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Objectives: This study aimed to identify different criteria for priority setting of rare diseases to help policy makers in making evidence-informed decisions. Methods: A scoping review was conducted to comprehensively examine the existing various methods and criteria for prioritizing orphan drugs and rare diseases. We performed searching in Scopus, PubMed, Embase, and websites of health technology assessment (HTA) agencies, 2000–21, and data were extracted. Results: From the 1,580 identified publications, eleven articles were included. Multicriteria decision analysis was the most frequent method (seven out of eleven studies) used for priority setting. The extracted criteria for priority setting of orphan products were analyzed based on six main categories as follows: health outcomes and clinical implications (six subsets which showed clinical implications), economic aspects (four subsets that indicated the economic effects of orphan drugs and rare diseases), disease and population characteristics (six subsets that included the characteristics of the rare diseases), therapeutic alternatives and uniqueness of orphan technologies (two subsets which discussed the alternatives and uniqueness of orphan technologies), evidence (three subsets which regarded the quality and availability of evidence), and other criteria (three subsets dealing with social and organizational criteria). Cost-effectiveness, budget impact, and disease severity were the most frequent criteria in the studies. Conclusions: Because of the high price of orphan drugs and limitations of using HTA for reimbursement of them, it is critical to explore them by precise technical methods like multiple criteria decision making in priority setting. [ABSTRACT FROM AUTHOR]

End-stage renal disease (ESRD) is a foremost health issue with major consequences in various parts of the world. In Iran, the prevalence/incidence of ESRD has increased during the past decades. The economic burden of ESRD treatment on patients, their families, and the whole healthcare system is huge as well. Hence ESRD is considered emerging public health problem in developing countries, including Iran, requiring short- and longterm changes in healthcare policies. Developing a national registry system for dialysis patients in Iran now enables us to summarize certain clinical characteristics of these patients and compares the present situation with the late 1990s when dialysis services just began to expand in the country. In this paper, the authors provide information regarding the process of establishing dialysis registry in Iran along with the report of the output of such a registry. Focusing on such an important measure in the whole country of around along with the data that it has produced is a gateway to further progress. [ABSTRACT FROM AUTHOR]

Aim: To bridge the gap in the current knowledge, the present study was conducted to obtain evidences relating to clinical outcomes of the end-stage renal disease (ESRD) population in Iran over the last two decades. Methods: The records of 84 652 incident ESRDpatients from1995 up to and including 2014 in the national registry of ESRDpatientswere analyzed retrospectively. Data were collected from dialysis and transplant centres in Iran from 1995 to 2008 via paper forms and from2009 to 2014 throughweb-based records. Results: Mean age (SD) in incident cases of ESRD (57.7% male) was 52.5 (16.6) years. The prevalence of ESRDpatients grew on average 14.9%and 5.3%annually in the periods of 1995-2004 and 2005-2014, respectively. Adjusted mortality rate among dialysis patients in 1995, 2005, and 2014 was 145, 154, and 177/1000 patient-years, respectively. Median graft half-life (IQR) and median expected remaining life-years (IQR) for dialysis patients in the 2003 incident patient cohort were 4.4 (1.9-8.3) and 4.2 (1.8-8.2) years, respectively. Conclusions: The overall slightly decreasing, and still comparable, survival trend in dialysis patients, despite the accessible and free dialysis treatment, may be the result of the counterbalance of different contributory factors, including increased age and the higher proportion of patients with comorbidities resulting from diabetes and hypertension. The half-lives of renal allografts were generally shorter compared to thus far shared evidence, which may be due to tissue incompatibility. The decreasing trend, in this context, can be attributable to the increased number of transplantation from deceased donors with expanded criteria. [ABSTRACT FROM AUTHOR]

Introduction. Data on risk factors associated with low bone mineral density are limited in patients with end-stage renal disease. This study evaluated the factors deemed associated with lumbar and femoral Z and T scores. Materials and Methods. Clinical and demographic data of 98 patients waiting for kidney transplantation were collected, as well as lumbar and femoral bone densitometries, before transplantation. Osteoporosis and osteopenia and factors associated with bone mineral density were assessed. Results. According to the femoral T score, 38.8% (95% confidence interval [CI], 29.1% to 48.4%), 44.9% (95% CI, 35.1% to 54.7%), and 16.3% (95% CI, 9.0% to 23.6%) of the patients had normal bone density, osteopenia, and osteoporosis, respectively. According to the lumbar T score, 54.1% (95% CI, 44.2% to 63.9%), 33.7% (95% CI, 24.3% to 44.0%), and 12.2% (95% CI, 5.8% to 18.7%) of the patients had normal density, osteopenia, and osteoporosis, respectively. Age, serum levels of creatinine and parathyroid hormone, and use of calcitriol and calcium carbonate were associated with femoral densitometry scores. Serum total protein level, Rh-negative status, and B blood type were associated with the lumbar scores. Conclusions. Parathyroid hormone contributed to bone loss in our kidney transplant candidates, and B and Rh-negative blood types were associated with a higher risk of lumbar osteoporosis while total protein was negatively associated with the risk of bone loss. Calcitriol might improve femoral mineral density, but calcium carbonate was negatively associated with femoral bone density. Age and higher creatinine levels were associated with higher femoral bone densities. [ABSTRACT FROM AUTHOR]

Introduction. Chronic kidney disease (CKD) is a public health problem that needs an integrated program to be detected, monitored, and controlled. This study reports the results of a CKD program designed and implemented in Shahreza, Iran. Materials and Methods. After initial evaluation of CKD in Shahreza, a CKD management program was developed in the Ministry of Health and the pilot project was started in February 2011 in Shahreza rural areas. The patients at risk, including those with diabetes mellitus and hypertension, were tested with serum creatinine and urine albumin-creatinine ratio. The CKD management program included training, screening, monitoring, and controlling of weight, hypertension, diabetes mellitus, lipids, and vitamin D. Results. This pilot program was organized in the rural population aged over 30 years who were suffering from hypertension, diabetes mellitus, or both, and resulted in the discovery of cases in various stages of CKD. The prevalence of CKD in this high-risk group was 21.5%. Persistent albuminuria and a glomerular filtration rate less than 60 mL/min/1.73 m2 were 13% and 11%, respectively. The rate of CKD stages 1, 2, 3a, 3b, 4, and 5 were 2.75%, 6.82%, 10.08%, 0.92%, 0.31%, and 0.17% respectively. After 1 year of the program implemented, incidence rate of CKD was 24% and improvement rate was 21%. In diabetic patients, the mean of hemoglobin A1c decreased from 8.5 ± 1.9% to 7.5% ± 1.8%. Conclusions. Integration of CKD programs in primary health care is possible and results in improvement in management of CKD patients. [ABSTRACT FROM AUTHOR]

Introduction. Assessment of the hemodialysis adequacy is one of the key factors in evaluating health service system. This would provide a good background for effective future planning by healthcare authorities. In this study, we aimed to evaluate the hemodialysis adequacy in Iran. Materials and Methods. One hundred and twenty-seven hemodialysis centers affiliated to 30 medical universities in Iran participated in this cross-sectional multicenter national study. All demographic data as well as hemodialysis prescription data, including blood flow rate, length of the hemodialysis session, hemodialysis membrane type, and composition of the dialysis solution were recorded for each patient. In addition, urea reduction ratio and Kt/V were calculated to determine the hemodialysis adequacy. Results. A total of 4004 patients were included in this study, 2345 men (58.6%) and 1659 women (41.4%). Bicarbonate-based solutions and low-flux membranes were prescribed for 77.0% and 97.6% of the patients, respectively. The mean blood flow rate was 242.9 ± 39.2 mL/min. The mean length of hemodialysis session was 229.2 ± 22.2 minutes. The mean urea reduction ratio and Kt/V were calculated to be 61.0 ± 11.8% and 1.2 ± 0.4, respectively. A Kt/V less than 1.2 and a urea reduction ratio less than 65% were found in 56.7%, and 65.2% of the hemodialysis patients, respectively. Conclusions. This study showed a substantial inadequate hemodialysis in Iran as compared with the Kidney Disease Outcomes Quality Initiative guidelines. Considering the impact of dialysis adequacy on quality of life and survival rates, as well as healthcare costs, rigorous attempts to achieve the desired goals are necessary. [ABSTRACT FROM AUTHOR]

Introduction. The rapid increase in the prevalence of end-stage renal disease (ESRD) necessitates putting into practice some strategies to prevent its development and progression, especially in the developing world. Detailed chronological changes in the incidence of ESRD may sharpen the focus on its prevention. We, therefore, determined the detailed epidemiological features of ESRD in Iran. Materials and Methods. Data of the national registry of Iran's ESRD provided by the Ministry of Health were used to retrieve the ESRD figures between 1997 and 2006. Results. A total of 35 859 patients who initiated renal replacement therapy (20 633 men and 15 226 women) were registered during the study period from 1997 to 2006. The annual number of patients with ESRD beginning maintenance treatment in Iran increased 130% between 2000 and 2006. During 1997 to 2006, the proportion of new cases of ESRD attributed to diabetes mellitus increased 2-fold from 16% in 1997 to 31% in 2006. The mean age of newly registered men and women increased from 47.0 years and 49.0 years to 52.5 years and 53.0 years, respectively. As for all and major causes of ESRD, age-adjusted incidence rates for men generally were higher than those for women. Male-female ratio was 1.3:1, with no significant changes during this period. Conclusions. We strongly recommend considering chronic kidney disease prevention with initial focusing on strategies and treatment modalities that slow ESRD progression in order to postpone the need for renal replacement therapy. [ABSTRACT FROM AUTHOR]