This document evaluates the economic impact of home enzyme therapy administration (HETA) compared to in-hospital therapy (iHp) and ambulatory hospitalization (AHp) for patients with Fabry disease and Gaucher disease in Spain. Direct and indirect expenses are estimated for the different treatment strategies. Results show that HETA provides significant cost savings to the health system and avoids lost work productivity costs compared to iHp and AHp. An electronic tool was created to calculate treatment costs for individual patients to help compare treatment strategies and their impact on health systems. The HETA program provides effective treatment with significant economic benefits added to improved patient satisfaction.

1. IntroductionIntroduction
Despite the demonstrated efficacy, security as well as improved quality of life derived form the
administration of specific enzymatic treatment for lysosomal diseases, it is not clear the economical
impact, with scarce and contradictory results probably derived form the different methodology and
highly heterogeneus Health System in each country.
A recent publication in Italy about economical impact of the same national Home Enzymatic
Therapy Administration (HETA) scheme that is being applying now in Spain, has demonstrated that
this starategy is cost-effective.
To estimate the economical impact of home enzymatic therapy (Ht)
administration compared with that in the hospital (Hp) and ambulatory
hospitalization (AHp) for Fabry and Gaucher diseases
In those patients included in the Spanish HEA program
for home infusion of velaglucerase and agalsidase
alpha in the Comunidad Valenciana, a region in the
southeastern coast of Spain.
HETA spanish national program is supported
by Shire Pharmaceuticals Iberica©
ObjectiveObjective
MethodologyMethodology
HETA Protocol
See posters #108 and #109.
Pharmacoeconomical evaluation
Direct and indirect expenses have been estimated
for three possible strategies for enzyme infusion:
In-Hospital (iHp)
Usual Domiciliary-Hospitalitation (DHp)
Home enzymatic Therapy Administration (HETA).
Calculations has been also made taking into account
the disease of each patient (GD vs. FD) and laboral situation.
Expenses were classified as those for the Health System,
the individual and work.
All estimations are calculated in euros for 26 per year infusion.
ResultsResults
Communicated as a poster
Expenditures Expenditure concept Calculation
Direct
For the Health System
Drug preparation, excluding the cost of the
drugs
Ambulance Generalitat Valenciana Official Sanitary Services prices (AM0105 and HS0108)
Work-time of Hospital Pharmacist/s - According to the Generalitat Valenciana Official monthly salaries, the cost
of 1 minute of work was estimated.
- Time for preparation of the drugs was estimated:
For iHp andDHp: 45 and 85 min for Fabry and Gaucher patients respectively.
For HETA: 5 and 25 min for Fabry and Gaucher patients respectively.
Work-time of Hospital Pharmacist/s auxiliar workers
Work-time of Hospital Nursery
Work-time of Hospital Nursery auxiliar workers
Fungible sanitary material (Syringes…) Generalitat Valenciana Official Sanitary Services prices
In-Hospital session Generalitat Valenciana Official Sanitary Services prices
Domiciliary Hospitalization session Generalitat Valenciana Official Sanitary Services prices
Indirect
For the patient
Displacement hospital ↔ home
Displacement (Car taxes, Car wear, Fuel…) Captio Mileage Report 2015
For the society
Loss of working time/productivity
Absence at the work place
Encuesta Trimestral de Coste Laboral (ETCL) del Instituto Nacional de Estadística (INE)
para el 4º trimestre de 2015.
110. Home enzymatic infusion program for Gaucher and
Fabry diseases. Economic impact evaluation of the
experience in the Comunitat Valenciana in Spain.
*Giner-Galvañ V1
, Carrascosa-Piquer O2
, Almela-Tejedo M3
, Perpiñá-Henández J4
, Cornejo-Uixeda S2
, Sarrió-Montes G3
, Sanz-García J1
, Vicente-Navarro D1
.
1
Rare Diseases Unit. General Internal Medicine Department. Hospital Mare de Déu dels Lliris. Alcoy (Alicante). 2
Hospitalary Pharmacy. Hospital La Ribera. Alzira (Valencia). 3
Hospitalary Pharmacy. Hospital Mare de Déu
dels Lliris. Alcoy (Alicante).4
General Internal Medicine Department. Hospital La Ribera. Alzira. Valencia. Spain.
*Presenting and corresponding author: giner_vicgal@gva.es
Internal Medicine
Department
Rare Diseases Unit
Alcoi (Alicante). Spain.
Patient
iHP DHp HETA
Health
system
Displacem
ent
Laboral Total
Health
system
Displacem
ent
Laboral Total
Health
system
Displacem
ent
Laboral Total
Alcoy’s Hospital
F1 3,021.20 84.50 2,148.64 5,254.34 3,422.64 0 2,148.64 5,571.28 0 0 0 0
F2 3,021.20 84.50 2,148.64 5,254.34 3,422.64 0 2,148.64 5,571.28 0 0 0 0
F3 3,021.20 84.50 2,148.64 5,254.34 3,422.64 0 2,148.64 5,571.28 0 0 0 0
F4 3,021.20 84.50 2,148.64 5,254.34 3,422.64 0 2,148.64 5,571.28 0 0 0 0
G1 3,021.20 84.50 0 3,105.7 3,422.64 0 0 3,422.64 0 0 0 0
G2 3,021.20 84.50 0 3,105.7 3,422.64 0 0 3,422.64 0 0 0 0
Total 18,127.2 507 8,594,56 27,228,8 20,535,84 0 8,594.56 29,130.4 0 0 0 0
Alzira’s Hospital
F5 3,021.20 84.50 2,148.64 5,254.34 3,422.64 0 2,148.64 5,571.28 0 0 0 0
F6 3,021.20 84.50 2,148.64 5,254.34 3,422.64 0 2,148.64 5,571.28 0 0 0 0
G3 3,021.20 84.50 2,148.64 5,254.34 3,422.64 0 2,148.64 5,571.28 0 0 0 0
G4 3,021.20 84.50 2,148.64 5,254.34 3,422.64 0 2,148.64 5,571.28 0 0 0 0
Total 12,084.8 338 8,594.56 21,017.2 13.690,6 0 8,594.56 22,285.1 0 0 0 0
Both HospitalsBoth Hospitals
TotalTotal 30,21230,212 845845 17,189.1617,189.16 48,246.048,246.0 34,226.4434,226.44 00 17,189.217,189.2 51,415.551,415.5 00 00 00 00
Table 1. Estimation of expenditures for each enzyme administration strategy (iHP: in-Hospital. DHp: Domicialiry
hospitalization. HETA: Home enzyme Therapy Administration) and concepts for each patient and hospital.
Results are expressed as Euros/year assuming 26 infusion/yr.
Graph 1. Proportional
Comparative potentail savings
for each concept.
A: iHP vs. HETA. B: DHp vs. HETA.
A
B
Laboral
Health System
Laboral
Health System
Displac.
 An electronical Excel©
-based tool has been created to calculate and compare the infusion-related expenditures for each
patient with GD or FD, in each medical center.
 This project is being extended to all patients actually included in the Spanish national HETA program.
ConclusionsConclusions
HETA program t is an effective
strategy for the treatment of
GD and FD with a significant
economical saving to be
added to the very positive
opinion of patients (Poster
#109).
The creation of a very simple
electronical tool to estimate
infusion-related expenditures
with different strategies can be
an interesting way to compare
the convenience of one
infusion strategy respect to the
others, than can be different
dependig on the kind of Health
System organization.
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Guest JF, Concolino D, Di Vito R, Feliciani C, Parini R, Zampetti A.
Modelling the resource implications of managing adults with Fabry
disease in Italy. Eur J Clin Invest. 2011;41(7):710-8.
Guest JF, Jenssen T, Houge G, Aaseboe W, Tøndel C, Svarstad E.
Modelling the resource implications of managing adults with Fabry
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