Sacco P, Raspa M, Leahy L, Cabo R. A literature review of the economic burden of Fragile X syndrome. Poster presented at the 2018 AMCP Managed Care & Specialty Pharmacy Annual Meeting; April 23, 2018. Boston, MA. [abstract] J Manag Care Pharm. 2018 Apr; 24(4-a):S7. Previously presented at the 70th AAN Annual Meeting.

OBJECTIVE: Published data were evaluated to understand the economic burden of Fragile X syndrome (FXS).

BACKGROUND: Individuals with FXS experience intellectual disability and reduced functional abilities, including problems with attention and anxiety. Evidence is beginning to converge on the use of medical specialists, allied health professionals, and prescription medication among individuals with FXS. Resource utilization and costs are critical to populate economic models to demonstrate the value of new health technologies for the treatment of FXS.

A targeted review searched PubMed (2006–2016) using search terms on the following topics: resource utilization (e.g., medical services, medical tests, procedures, therapy, hospitalization) direct and indirect costs (e.g., economic burden, productivity, absenteeism, long term care).

RESULTS: The original search yielded 730 unique abstracts. Sixteen articles were retained; full text evaluation identified 13 studies for this review. Study geographies included: 10 United States (US), 2 European Union (EU), 1 Canada. US administrative claims data found FXS patients had median all-cause direct costs ranging $2222 to $2955 (commercial/Medicare), $4548 to $9702 (Medicaid) and annualized total all-cause mean direct costs of $14,677 (privately insured). Healthcare cost drivers in the US were outpatient visits, home-care and long-term care services, medical procedures, hospitalizations, medications, other out of pocket expenses. In a survey in 5 EU countries, mean total annual direct cost per patient varied widely (€4951 to €58,862); mean annual direct healthcare costs per patient ranged €110 to €2,675. Healthcare cost drivers in the EU were medical visits, hospitalizations, medications. We will report on studies which investigated the association of functional limitations on economic burden.

CONCLUSIONS: This review suggests FXS poses substantial economic burden. The cost of care varies across payer types, geographies, symptom severity. Further study is warranted to better characterize symptom-related cost drivers and impact of economic burden for all stakeholders.

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