Healthcare resource utilization and cost of care for Gaucher patients in Iran

Abstract

Introduction Gaucher disease (GD) is an inherited recessive enzyme deficiency with a multisystem condition. The Iranian government covers the therapeutic expenditure of GD patients as it is not affordable for the patients. The aim of this study is to identify the main components of the cost of care in Gaucher patients (GPs) in Iran. Methods The Gaucher patients were identified from the Iran Food and Drug Administration (IFDA) national registry database. The direct medical costs, including medication, diagnostic services, and physician visits were considered. The prices of therapeutic and diagnostic services were extracted from Iranian medical tariff book 2014-15. Iran Food and Drug Administration determined the cost of medications. Results 164 Gaucher patients have been registered in Iran. A valid and reliable diagnostic tests are not used to identify the type of GD. The average health care cost per annum was 20,758 USD per patient, which is higher than 4 GDP per capita in Iran. Medication cost constitutes 95.2% of the total cost. The average cost of each GP was $1,473,818 in his/her total life. Conclusion GD is amongst the high-cost diseases and should be managed effectively. The application of oral medication for eligible GPs could improve allocative efficiency in GD management significantly. A sound, valid and reliable national clinical guideline could improve the efficiency of healthcare resources effectively. Selecting appropriate strategies for reducing the birth of a child with Gaucher, could support allocative efficiency of the limited resources effectively.

Economic burden and health related quality of life of ultra-rare Gaucher disease in China

“…Gaucher disease (GD), an ultra-rare multi-organ involvement, inherited recessive enzyme deficiency [ 1 , 2 ], is slowly progressing. Its standardized incidence and prevalence among the general population worldwide vary from 0.30 to 5.80 per 100,000 population, respectively; however, the initial estimate was 0.25 to 1.24 per 100,000 population [ 3 , 4 ] with hundreds of patients diagnosed with GD in China.…”

Analysis of the Situation of Unmet Health Care Need Among Extremely Rare Disease of Gaucher

“…Nine families reported a family history of the same disorder in this study, indicating a need for awareness of genetic disorders to highlight the importance and burden of genetic diseases. Therefore, newborn screening for disorders that meet the criteria for population screening [33], use of reliable diagnostic tools [1,[34][35][36], raising of awareness on the early signs/symptoms [37] or less severe manifestation [21], and initiation of therapy early for rare diseases [30], are essential.…”

“…In particular, long-term treatment with regular monitoring for GD can optimize outcomes [38], alleviate fewer exacerbations [1], and reduce the incidence of complications [39]. [40].…”

“…Gaucher disease (GD), an ultra-rare multi-organ involvement, inherited recessive enzyme de ciency [1,2], is slowly progressing. Its standardized incidence and prevalence among the general population worldwide vary from 0.30 to 5.80 per 100,000 population, respectively; however, the initial estimate was 0.25 to 1.24 per 100,000 population [3,4] with hundreds of patients diagnosed with GD in China.…”

“…Measuring the economic cost of illness can provide better information to policy makers to develop more targeted interventions to GD at various levels of health care system [15]. By measuring the economic and care burdens of diseases to the society will facilitate a prioritized healthcare policies and strategies [1]. However, evidence on the nancial burden (e.g., direct medical costs, direct nonmedical costs, and indirect costs) associated with GD in China is limited [16,17].…”