Prevalence-Based Measurement of the Economic Burden of Rare Diseases: Case Review To Determine the Annual Cost of Acromegaly In France

Abstract

Objectives: To assess pill burden, health care resource utilization (HRU), and costs among patients with long-term immediate release (IR) hydrocodone use. MethOds: We performed a retrospective analysis of health care claims from 2011-2012 Truven MarketScan® Commercial, Medicare supplemental, and Medicaid Multistate databases. Patients with IR hydrocodone prescription for ≥ 90 days during 6 month baseline period (July 2011-December 2011) with continuous enrollment during baseline and 12 month follow-up periods were selected. The final population was sub-categorized by prescribed coverage days (PCD) of IR hydrocodone during baseline into 90-119, 120-179, and ≥ 180 days. Chi-square or ANOVA analyses were used to test pill burden, HRU and costs (standardized to 2013 US dollars) during baseline and follow-up periods across subpopulations. Results: A total of 36,174 commercial, 32,699 Medicaid, and 8,873 Medicare IR hydrocodone users were selected. In the baseline period, subgroups with longer PCD had significantly more average hydrocodone pills per month yet fewer HRU and medical costs (all p< 0.05). However, during the follow-up period, groups with longer PCD had greater increase in number of inpatient hospitalizations and other types of HRU (length of stay, outpatient hospital visits, office visits, and emergency room visits). The subgroup of patients with PCD < 120 days had lower annual all-cause medical costs during follow-up compared with baseline (decreasing $2,624, $2,955, $4,209 per patient per year in Medicaid, Medicare and commercial patients, respectively), while patients with longer PCD during baseline had increased costs (p< 0.05). For example, Medicaid patients with 120-179 PCD had an increase of $1,874 and those with ≥ 180 PCD had an increase of $4,348. These trends were similar for all insurance types. cOnclusiOns: Extended length of PCD, particularly after 120 days, corresponds with higher patient burden including elevated pill burden and rising HRU and costs in both commercial and public insurance patients with long-term IR hydrocodone use.

Economic and pharmaco-economic analysis of acromegaly treatment: a systematic review

“…As it can be seen, direct costs (lab tests, medicines, visits, hospitalizations, diagnostic procedures, etc.) are included in all studies, whereas the indirect costs presented as loss of productivity due to acromegaly, unemployment, assistance to perform household chores and family member loss of income are calculated only in a few studies [26,37,42]. This could be highlighted as a strong limitation especially in cost-of-illness studies where the societal perspective is broader and it is usually taken into account.…”

“…Different costs were also reported in different studies, e.g. only 3 out of 33 presented the indirect costs taking into consideration the societal perspective [26,37,42].…”

“…Therefore, a further step could involve more detailed economic evaluations as well as initiation of common research among the countries from the CEEC region. Economic evaluation studies of acromegaly with high methodological quality have been performed in the United States, the United Kingdom, Italy and other countries from Western Europe, as they precisely inform the society about the significant financial burden of acromegaly and its comorbidities [6,16,18,21,22,25,26,28,29,[32][33][34]42,44]. Sliwczynski et al [35] revealed that comorbidity is associated with approximately 5 times higher costs, which highlights the importance of providing strict control over the main disease and the concomitant diseases.…”

Rare disease curative care expenditure-financing scheme-health provider–beneficiary group analysis: an empirical study in Sichuan Province, China

“…Rare diseases are serious and difficult to diagnose and treat, which can easily lead to damaged tissues and organs, resulting in disability, mental disorders, deteriorated quality of life, and even death. Rare diseases put an extremely heavy burden on patients’ families and society and pose immense challenges in building and improving global public healthcare systems [ 7 – 9 ]. Research has shown that the annual curative expenditure for Hemophilia in the United States is $80,811–$632,088.…”

Rare Disease Curative Care Expenditure- Financing Scheme-Health Provider- Beneficiary Group Analysis — An Empirical Study in Sichuan Province, China

“…However, only 5% of them have treatment methods [6,7]. Since rare diseases are serious and difficult to diagnose and treat, most of them affect all the systems and organs in the human body, which can easily result in disability, mental disorders, deteriorated quality of life, and even death, causing extremely heavy burdens on patient's families and the society while posing immense challenges to the construction and improvement of the global public health service system [8][9][10]. Researches have shown that the annual curative care expenditure(CCE) of Hemophilia in the United States is US$80,811-632,088; European patients with Multiple Sclerosis (MS) spend an average of US$47,888 per year on curative care [11,12]; in Australia, CCE for rare diseases accounts for 4.60%-10.50% of the total inpatients' curative care expenditure, equivalent to that of diabetes or asthma in 2010 [13].…”